International MPN News, Science & Opinion

The Jakafi Report

Report from Jakafi patients

Jakafi gets mixed grades
Results mostly fast and dramatic, nasty side effects cited by some.

 In an anecdotal survey of  21 MPN patients taking Jakafi for relief of MF symptoms, first results vary from highly positive to unacceptable.

This is by no means a scientifically controlled study. The results were derived from an MPNforum Magazine on-line request for first hand responses from the early adopters of Jakafi.  This qualitative study supplements scientific reports with fresh testimony from the patient pioneers in this revolutionary MF treatment.  (Jakafi is the first drug  approved by the FDA and EHA to treat myelofibrosis therapeutically and the drug that clinically demonstrated the efficacy of kinase inhibition.)

The survey sample, too small to yield any statistically relevant data, is made up of self-selected respondents.   Every entry submitted by September 15, 2012 —  for which we had permission to publish —  appears below.  The accounts  are essentially unedited.

The stories that follow extend from a paragraph or two to several pages.  In practical terms, while we can’t as patients draw conclusions about our own likely response to  Jakafi, these accounts present a valuable range of first hand experience.      Our thanks to participants for their courage and willingness to step forward.

 Swelling causes discontinuation

I have PV and the worst itching and stabbing pains. Had to really drug up in order to take a shower. Now I can take a shower itch free. I was hoping to have relief from fatigue but after 2 weeks on the drug, still no relief there After the 3rd week of being on Jakafi, my face, neck and hands swelled up.
I had a bit of difficulty breathing last night. So even though it helped
tremendously with the itching, I think I will have to get off. It also is
not controlling my counts at all….

I ended up in the ER last night bc. the swelling of the neck started causing breathing constrictions, and my dr. wanted to know if I am not throwing a super vena cava blood clot. I am not. I did however for the 1st time ever get hives from the contrast. Jakafi did help with the itching, that is why I am upset that I got such a side effect to force me off the drug.

*  Update: My nurse wants me to stay on 10 mg. of  Jakafi since it does help the itch. And to add back 25 mcg of Pegasys to help the platelet and wbc counts. My bp dropped to 97/57 and we don’t know what caused that, but I felt like passing out.

– Monique

Combination of Pegasys and Jakafi Seems to Work

I am 52 and was diagnosed with early PMF in 1/11.  I was a bit anemic and had a large spleen of 9cm.  I began Pegasys a few months later at the suggestion of RS, and ramped up my dose to 90mcg before beginning a trial for SR Jakafi.  My spleen was measured at 15cm at this time and my decision to try Jakafi was based on my enlarging spleen.  I am now currently on 20mg Jakafi and 90mcg Pegasys.  I think I’m doing well, but other than feeling good on this combination, is there any way to tell if I am benefiting from it?

I have tracked my counts back a year before diagnosis, and they have been pretty much stable up until starting Jakafi which lowered my platelets and hgb.  My platelets are now stable at around 113k and hgb is back up to pre jakafi days at 12.2.  Most people I speak with see a lowering of counts while on Pegasys, and I can’t tell it’s made any difference.  No change the 4 months on Pegasys alone before starting Jakafi.

I know a bmb may provide some answers, but I want to wait a bit longer.  The benefits I see from Jakafi are reduction in spleen (now at 7cm) and reduction in night sweats.  What I see from Pegasys is reduction of bone and joint pain and an increase in energy.

So has this combination helped keep me stable, or would I have been stable without it?  I think my spleen would be a whole lot bigger now.  Latest CBC wbc 5.9 rbc 4.06 hgb 12.2 hct 35.5 rdw 17.6  plt 113.

I am not sure I have much to offer anyone yet on my combination,  and I am concerned about giving others with MF a false hope.  I may be a very unique case.  I only know that I feel better taking Pegasys.  From what I’ve read, most patients see a reduction in counts from it, but I never have.  Jakafi reduces your platelets and Hgb, and a combination therapy may cause further drops.

I have seen my platelets drop from 198k to 100k while on Jakafi.  My Hgb initially dropped a point, but has now rebounded back.  There was no real change when I added Pegasys. My disease seems to be stable, but unless I see a further reduction in my spleen, or have another bmb that shows a reduction in fibrosis, the jury is still out on this combination.

…  I started[Jakafi] last September as a member of a drug trial for a sustained release version.  I was converted to the standard version when the trial ended in January.  I started the combination therapy in February.
The combination therapy I’m on was my idea.  My doctors of course have gone along with it, but I would be reluctant to recommend this to anyone without close supervision.  I seem be be doing well with this, but others have seen some counts lowered by both drugs and this combination is not been tried before.

Dave (pseud)

At brink of splenectomy, fresh start with Jakafi

I was diagnosed in March of 2006, age 59. I also was diagnosed with prostate cancer in the same month along with a DVT in my right leg.

After much soul searching and consultation at the University of North Carolina,  I was scheduled for a spleenectomy and Bone Marrow Transplant in the spring of 2011.  I had a heart attack that April and a stent placed in my heart. I postponed my surgery and BMT as I felt better and did not want to risk those procedures.

I have been on Thalimid, Revlimid and Prednisone.  I stopped the Revlimid and am coming off the Prednisone.  I started Jakafi, 5mg twice a day, on September 26, 2012.

I am hopeful the Jakafi will reduced my enormous spleen and enable me to eat better.   As of October 2, it appears my spleen is smaller and I feel much better.

– David G. Dickover

 

I felt renewed energy to enjoy life

On day twelve after entering the Jakafi clinical trial experience I changed the home smoke alarm in my apartment. The automatic beep that lets you know that the alarm is getting to the end of its life had been beeping at me off and on for weeks and I had ignored it, then detached it so that it hung awkwardly from the ceiling but in silence. After that I just pretended it was not there and all because I had become so fatigued over the previous eighteen months to do much of anything beside the essentials. I tell you this to illustrate what can happen to you when you suffer from extreme fatigue as many MF patients do. You just don’t care any more. You do what you have to do through acts of tremendous will, but mostly you live life though a kind of mental and physical fog. But on the 12th day of jakafi I went out and bought a new smoke alarm and installed it myself!

I felt the renewed energy to enjoy life within days of  beginning Jakafi, mentally first as I suddenly had the energy to answer e mails, return phone calls, read books, and then physically as I again took up my favourite exercise, swimming. I even did some creative writing, which I had pretty well given up. My desire to meet my social obligations and my ability to carry out household tasks and a modest exercise program with renewed energy had returned. Yes, my spleen also eventually shrunk and my night sweats and bone discomfort moderated to the point that I did not need nightly ibuprofen to go to sleep, but for me it was all about having my brain back.

There have been some ups and downs in the adjustment period. At first I ate too much as my returned appetite made me enjoy lots of everything. Indigestion taught me to moderate. I quickly returned to my ambitious hiker’s stride only to find in the third month that my haemoglobin had dropped and I got breathless when walking quickly. By the sixth month my hemoglobin came back to just within the normal range and my other blood numbers came into line and I am learning not to walk so fast and to exercise some good sense and not overdo things just because I have enthusiastic energy again. I am grateful for the clarity of mind that has been mine since Jakafi has calmed the cytokine storm that was my immune system’s reaction to increasing fibrosis. I am grateful for the much decreased level of blasts in my peripheral blood. Long may it last.

The science says that Jakafi may not give me a longer life prognosis, only relief of symptoms, but since most MF patients succumb not to the fibrosis itself but to opportunistic infections and increasing stress to all body systems caused by being in poor health I am hoping that good health will keep these secondary morbidities at bay for a long time. We live in hope, for this drug and the ones that will come from what science has learned from the Jakafi experience of MF patients.

* (I was part of the Canadian third phase trials to have the drug approved by Health Canada. I was a patient in the Vancouver cohort. The trial ended when Health Canada approved the drug for MF and now I receive my prescription free of charge from Novartis, a promise they made to all those participating in the Canadian trials. ).

— Helen M. Buss

 Long journey to Jakafi…feel great

The short version: With the Jakafi I feel the best I have felt in years! I have more energy then ever – though I still take naps. I am not what I used to be before all this but I feel amazing!

The Details:
I was a person who never got sick -not even a cold. then in 2001 I felt exhausted all the time. I was diagnosed with PV/ET. I survived fine with phlebotomies for two years, then in 2003 I had a Budd Chiari. I made it through this shocking surgery but was given 3 days to live because unfortunately I am HIT +. (allergic to Heprin)  Once I got past 3 days I decided in that moment there was no way I was dying in winter(!) in a hospital, at 42!
 A long month later I went home, it was tough but after a years recovery I made many changes to my life. I eat healthier (I tried being a vegan but that was a disaster – now I reluctantly eat meat.) , I sleep more, don’t let the little stuff get to me (and most of the stuff in life is the little stuff), get alternative healing, and generally live my life more in the moment. I do not dwell on being a “sick” person. and most people have no idea I am “sick.” The one thing I have left to do is add more physical activity to my life.
 I am very, very, lucky and have an amazing hematologist Dr. Jeannie  Callum (SunnyBrook Hospital in Toronto) who supports me with unbelievable care, and I have many other amazing women healers on my side. (I.E. Acupuncture, Chiropractor, Physio for my circulation in my legs, and Shamanic crystal healing)

 The short medical history is this: dates are approximate. I don’t remember all the doses.
2001 – 2003 Phlebotomies only
2003  HU
2009 Interferon injections for about 8 months. this was awful. I was sick two days a week and I ended up on anti-depressants. (I went off them as soon as I switched back to HU.)\2010 5 or 6 Blood transfusions. (needed them about every 7 weeks)
2011 Back to HU as the interferon was not helping. I was not able to be considered for the trial because my blood levels for my liver were not good enough. This was because the Budd Chiari had compromised my liver.
2012  HOWEVER! Surprisingly this changed and I was in a place that my doctor applied for me to be on Jakafi. I have been on since about April – I take 10mg in the morning and 5mg at night.
 Note that in Canada Jakafi was not approved so under a compassionate release, I was getting it for free from Novartis for the first 5 months. However Jakafi has just been approved here. Also to note: Novartis would have kept giving it to me  for Free or covered any co payments, if my insurance didn’t cover. Lo and behold I am so very lucky because our extra insurance (not the provincial) through my partner covers it 100%!
 In some funny way I feel lucky to have gotten this wake up call. I have changed so much from before I was “sick.” I was too serious and worried about every thing, I was consumed with stress, and had no real goals. Now I feel like I am way more focused and live my life with so much gratitude. So that is my story. If you want more details about something let me know. I am happy to share my story. But please no complaining. that just doesn’t work for me any more.
– Melissa Levin

 

My quality of life greatly improved… I can tell when the meds quit working

My Jakafi experience…I am a 62 year old woman. I was diagnosed in October 2003 with polycythemia. Treatment monitored my CBC and phlebotomies were ordered as needed. Eventually it was necessary to be prescribed hydrea urea. I did not respond well. I was on it for less than 6 months & it was a constant roller coaster of too much or too little. I dropped 15 lbs, my hair thinned, my skin was hanging off my bones, lights on..no one home.

I was screened for the trial and accepted with early stages of mylelofibrosis in March 2010. The trial was not unblinded until December 2010 but I had immediate results. Within 24 hours my foggy brain receded leaving me much more alert. I began sleeping comfortably again without being drugged on atarax to keep the itching & twitching at bay. My CBC #’s dropped within 2 weeks and were in the normal range in a month. My spleen size reduced the 25% the trial was looking for and has been normal for over a year. My hair and skin condition also improved. I experience no discernible side effects.

I can tell when the meds quit working. I take 20mg 2x a day 12 hours apart. Approximately 2 hours before I’m due to take my next dose I can feel the heat kick in. The medicine seems to act like an on/off switch. My quality of life is greatly improved. My bone marrow tests show no advance in tissue scarring.

I’m still in the trial. Week 132 is coming up.  I know my insurance will not cover the cost of the drug. So far Incyte has been an excellent partner and my trial nurse & doctor superb. Of the four patients in my trial location, I have responded the best… possibly because my disease was less advanced. It is my opinion that early intervention with Jakafi slows the progression of the disease …normalizes CBC… alleviating symptoms.
— AJ,  Palm Springs

Jakafi really helps but blood counts not stable, transfusions required.

I started Jakafi 20mg twice daily April 9th of this year after a Bone Marrow Biopsy in February revealed that I had converted to Post Polycythemia vera Myelofibrosis. I had had Polycythemia vera for over 22 years which was controlled very well by Hydroxyurea and phlebotomies.

One of the first things I noticed with my monthly blood counts was change in the differentials. I watched those and. then within a few months, the other counts began to fall. I had not noticed any particular change in spleen size. I was surprised when my Hem/Onc told me how much it had enlarged. I had been doing so well all those years, that I never paid much attention to postings on Myelofibrosis.

I took the 40mg of Jakafi daily for about a month.

Marilyn and Ed at Old Faithful Lodge

My spleen shrank almost immediately,my Hem/Onc was amazed. Then my blood counts started on a downward spiral, which required a transfusion of two units of blood. I was put on 20mg of Jakafi daily May 14.,which has helped and my blood counts have been much better, but still not steady.

I fell and have a broken back, “in a turtle shell “ now for the past two months…one more month to go if I behave myself. While in the rehab hospital, I had daily blood draws and it is amazing how the blood counts vacillate daily. I received one unit of blood while in the hospital. My hemoglobin has continued to drop., and I am sure that I will need another to boost it higher. It has been in the 8-9 range lately and I can have one any time of my choosing.

The Jakafi really helps me: My head is so much clearer, no more “fuzzy” brain. Also, my energy level has definitely improved and the itching, which started again after I converted, has gone away. I do feel good and feel that my quality of life has improved since starting on Jakaf. I understand that Jakafi will not prolong my life, but it has certainly improved my quality of life, thus far.

I think it will be interesting to see how I am doing after my back is healed. I had given up a number of things, such as line dancing, that required energy and stability in the few months prior to starting Jakafi and then falling.. I am definitely feeling stronger now with physical therapy and “cabin fever” that has set in.

— Marilyn Morgan

No side effects…my life is truly back.

I am now on my 28th day of Jakavi and can truly say that my life is back.  At a recent hockey game a couple of friends commented to my wife on how much I have changed in the past few weeks.  They thought I was more outgoing and participated much more in a meeting they attended with me.  These comments were pleasing to hear as I have only known these friends during my illness.  They haven’t known me in my previous life when I was a “borderline extrovert”.

I was diagnosed, in late 2007 at the age of 58, with primary myelofibrosis even though I suspect the disorder probably started in 2005.  In mid-2008 I started Hydrea (500 mg.) to try and get my energy back and control the white blood cell counts.  Itching, which was the first symptom, was also becoming a prevalent part of my life.  Morning showers, an important part of my ritual, became a big “no-no.  I had an enlarged gut and shortness of breath but my friends convinced me that I was just out of shape and should cut back on the beer.  Cutting back on showers was quite enough.

The Hydrea helped a lot but I still needed to nap every day and then couldn’t sleep at night.  After a year or so my hematologist recommended that I investigate the possibility of getting a bone marrow transplant while I was still relatively healthy.  I had several visits at the transplant unit of the Cancer Agency in Vancouver, 4 hours away from my home.  Apparently all of my other functions checked out fine but I didn’t get a suitable donor from my immediate family.  I was told that a wider search could be conducted if my condition worsened.  I was given the literature on bone marrow transplants.  Probably out of fear, having never spent any time in a hospital, I decided that a transplant wasn’t for me.  In particular I didn’t think removing my spleen was a good idea.

As an alternative, the BMT hematologist told me about a clinical trial that was supposed to start later that year (2009) and I should consider volunteering.  I agreed, reasoning that if I could delay the progress of the disorder long enough, researchers would find something less intrusive than a major operation.

The year passed and the clinical trial wasn’t approved.  I was feeling pretty good so I didn’t think much about it anyway.  We had just finalized plans to go to the first week of the 2010 Olympics when I got word that the clinical trial was approved.  I had to go through a rigorous qualification process, which the research hematologist conveniently arranged while I was at the Olympics in Vancouver.  I received word shortly thereafter that I was accepted in the clinical trial.  The clinical trial was a phase III trial and was to be conducted as a double blind test.  Results from previous trials were promising and side effects didn’t seem onerous.

Considering that it was probably my duty as a reasonably healthy person to help with the research on this disorder, I signed up.  Over the next few months my condition deteriorated and I became anemic, my lethargy increased and my attitude became rather negative. I was becoming quite a grump.  I shared my suspicion that I was on the placebo with the research hematologist and eventually she agreed.  After five months I discontinued the trial and re-started on Hydrea.

The summer of 2010 wasn’t great, either from a weather perspective or a health perspective.  The Hydrea didn’t seem to have its old punch.  My white blood cell counts continued to rise after a short period of being “normal” and my hemoglobin fell gradually to under 100.  In March 2011 my hematologist increased the Hydrea dosage to 1000 mg.  The WBC counts declined slowly and my hemoglobin began to rise.  I still didn’t feel good and I’m sure I was quite negative.  My business was deteriorating maybe because of the economy but likely because of my negative attitude and lack of drive.

In November I hit the bottom with the added bonus of a back problem that wouldn’t go away.  My usual remedy for back pain is Ibuprofen so I started that regime along with the other cocktail of drugs.  At this point in time the cocktail of drugs had gotten extensive for a guy who didn’t even take aspirin before the diagnosis.  I was on Hydrea, Allopurinol, Nexium, Aspirin, Vitamin D and Synthroid (albeit briefly).  In addition I chew Nicorette gum.  This time the Ibuprofen didn’t seem to work so I increased the dosage and went to a chiropractor, to no avail.  I reasoned that my spleen had gotten so big, 30 cm. below my rib cage, that it was throwing my back out.

Just about this time I received a phone call from the research Hematologist telling me that Novartis was conducting another clinical trial for Jakavi and this one would not have a control (placebo) group.  If I was finding that Hydrea was losing its effect I was welcome to join the trial.  This call couldn’t have come at a better time.  I made an appointment to get checked out in mid-April of this year.

The blood tests showed of all things that my platelets had fallen to 85.  Platelets!  Are you kidding, that’s where it all started with platelets in the 600 to 900 range?  How is that possible?  The doctors suggested I cut back on the meds and get re-tested at the end of May.  I suspected that Ibuprofen was part if not all of the problem.  Nobody agreed with me but one month later, after quitting Ibuprofen and Nexium and cutting back on the Hydrea, the platelet count was back in the 277 range.  Success!

No, we aren’t out of the woods just yet!  Jakafi was approved by the FDA in the US in November 2011 but not in Canada.  Thinking that I was now going to get on the trial I was told that Canadian approval would likely be received in June 2012 and it would be impractical for me to start the trial as it would be stopped once the approval was received.  My heart stopped!  The hematologist must have noticed because she then said that she could apply to put me on the compassionate program.  I didn’t hesitate, “let’s do it!”

The paper work took some time and I don’t really know what was going on but I suspect summer holidays played a big part in the delay.  In early August the pharmacy for Novartis delivered my prescription.  I started taking 10 mg. twice a day on August 11, 2012.

The first day I noticed that the itching stopped but I thought it was just in my head.  I slept better than usual that night and had an “itch free” shower the next afternoon.  Jakavi must be working.

In the month I have been on Jakavi many of my symptoms have disappeared.  No more night sweats, very few itch episodes (and then only minor) and even my acid reflux has disappeared.  I’ve also noticed that the joint pain that I had attributed to old age has all but ceased to be.  Particularly satisfying is my “yes” attitude.  I’m sure it has a lot to do with getting a sound sleep but I find myself saying yes to most everything these days.  In the first week on the medication I painted the inside of the garage, a job I have been planning for several years.  The next week I cut down a filbert tree in an inaccessible part of our yard that was interfering with the view.  Golf has become fun again and I’ve managed to play six rounds in the last two weeks.  That’s twice as many rounds as the whole season prior.

In the old days, prior to MF, I fell asleep as my head touched the pillow and reportedly snored with some passion.  Early mornings before everyone else got up were my favourite time of the day.  Post MF, my sleep patterns were erratic as I was awake most of the night and I didn’t snore at all. My best sleep was after 5:00 AM so my mornings didn’t start until 9:00 or 10:00 AM.  My wife on the other hand has always slept poorly and one time I said “maybe that’s all the sleep you needed”.  Boy have I heard that comment a lot over the last several years.

What a relief; I sleep soundly for 8 hours a night and put in a good days work.  I still have the odd nap every few days but nothing like the 1 or 2 naps every day before Jakavi.  Other benefits include getting my taste and smell back, no more night (sometimes day) sweats and a better memory.  At my last check-up my spleen size has shrunk from 30 cm. to 20 cm. 

My weight hasn’t changed much and I am still short of breath at the beginning of a walk.  My latest blood test shows my WBC falling back into normal and fewer H & L’s noted in the column.  I am toying with the idea of playing hockey again and fleetingly thought I could do the bike portion of an Ironman race.

I haven’t felt any of the side-effects listed in the literature but did have a “bout of gout” after the first week.  I also had some extended bleeding when I cut my shin.  I found it interesting that when my platelets were in the stratosphere I could watch while a cut healed, it only took a minute or so.  This time I bled for a few hours and the scab is still noticeable after two weeks.

Today was very interesting and serves as an example of the improvement in my quality of life.  At breakfast this morning I was mentally planning my day.  I had two papers to write and a golf time at 10:00 AM.  My horoscope said it was a good day for participating in a sport and writing reports.  I was thinking I should skip the golf because I would run out of energy.  Naw, I’ll do it all.  The golf was fun and I’m just finishing the second report (this one).

My life is truly back and I am enjoying every minute of it.

— Ben Minuk

 

“A weight has been lifted from me… “

I was diagnosed with PV in 1994.  For some years I had venesections every month or so, in the later years I was on and off ifn and hydroxyurea.

Then in 2000 I was diagnosed with MF and I was then on ifn 6mu every 2nd day and my blood counts were normal and they stayed that way until my spleen started growing in December 2010.

In January 2011 I took part in a clinical trial called CYT387.  I persevered with the trial for 6  months but not only did my spleen enlarge even more (19 cms) but my platelets rose to 950 and it was then I decided that drug was not for me.  I then saw my regular hem/onc and he was amazed at how my counts had changed.  He then put me on hydroxy urea which over time reduced my spleen but I had side effects from the hydroxy i.e. hair falling out, skin changes and loss of appetite .

Then my spleen started enlarging again and that was when I started the Jakafi and my spleen reduced to such an extent that you can’t feel it.  My appetite has increased and I’ve put on the 14 lbs that I had lost.  Back to 112 lbs.  But unfortunately my hb has been slowly decreasing, last week it was 10.5, down from 10.9 and previously 11.2.  So my doctor is now thinking about reducing my dose of Jakafi.

I am currently on Jakafi and have been on it since July 2012 I have had very good results with it; my main problem was a huge spleen, measuring 19cms in length and causing me a lot of discomfort.  Since taking the drug (20mg twice a day – 40mg) I can no longer feel my spleen, it has completely disappeared.

Before going onto the Jakafi I had a minor infarct of the spleen which caused me great pain and hospitalisation until the doctors discovered the problem.  As the drug is not approved in Australia at this stage, my hem/onc achieved getting it for me on compassionate grounds.  I think it is wonderful and I feel like a weight has been lifted from me.  I can recommend this drug very highly.

.I’m having another blood test today and will ring him tomorrow to find out the results and he’ll then decide what to do.  At present I am on 20 gm twice a day.,

– Joy Swedosh

 Increased energy…but problems adjusting dosage

I was diagnosed with PMF in mid-February of this year.  I did not notice any significant symptoms prior to February.    At the time of diagnosis, my spleen was enlarged and I had anemia but no other symptoms (except night sweats which could also be attributable to menopause).

It was decided at the time of diagnosis to do regular cbcs every two weeks and “watch and wait” and I was also referred to NorthwesternMedicalCenter to discuss an eventual bmt.

By late March, my spleen slze had increased considerably and was causing a good deal of discomfort.  I was aware that Jakafi had been approved to treat symptoms of MF (I work for a very large drug retailer). My doctor had never prescribed it for another patient so he had no experience with it ,and we discussed the possible risks and side effects prior to deciding to try it.   It took about a week for the prescription to be approved, filled and shipped and I started at a dose of 20mg twice per day (based on the dosage recommendations from Incyte) on April 18.  Within two days, my spleen size started reducing and I called Incyte to see if it could indeed be working that fast (and I was told that it could).  Within about a week it had shrunk considerably and I felt more energetic than I had in months.

My hgb and platelets started to drop about two weeks later, but stayed within the range where I did not have to stop taking Jakafi.

On May 8, my platelets dropped to 45 and my doctor instructed me to stop taking the Jakafi.   My hgb on 5/8 was 9.5.  Three days later (5/11) my platelets dropped to 24 and hgb was 6.2 and I had my first transfusion totaling four units of rbc and two units of platelets all during an overnight hospital stay.    During the late night on 5/12, I started experiencing fever , chills, headache and body aches which lasted for about three days….I was later told that they were likely a result of my stopping the Jakafi.    My spleen also started enlarging again almost immediately after the transfusions.

My platelet level increased over the next few weeks and on 5/21 I re-started Jakafi at 5mg twice per day.  That was increased to 10 mg twice per day a few weeks later and then the platelets and hgb dropped again and we reduced the Jakafi back to 5 mg twice per day.  Three days later the hgb dropped to 7.6 and platelets dropped to 64 and we stopped Jakafi again and I was transfused with two units of rbc.  The counts continued to drop and I had another rbc transfusion a week later and remained off Jakafi.

On June 20 I had a follow up appointment with my doctor of Northwestern. By then my spleen had again increased considerably and I was quite uncomfortable.  He suggested re-starting the Jakafi at 5mg one time a day to allow my system to get used to the drug and then increase it as needed.   I’ve been on that dosage since June 22.

Since this latest dose adjustment (5mg 1X per day), I’ve had two additional rbc transfusions, but the periods between transfusions is increasing.  My hgb level is staying higher, longer (last week it was 11!!!) and my platelet count has fluctuated but not to the extent where I have to stop Jakafi or have another platelet transfusion.

I think the Jakafi is working well for me.  My spleen is not as small as it was at the higher levels of Jakafi, and I frequently have discomfort in my spleen area but nothing severe.    I have increased energy.  I’ve been able to continue to work full time and, while I take frequent rest/nap periods, I am able to enjoy activities in my leisure time.  I’ve gained some weight back and don’t look so gaunt, all things that make my quality of life better on a regular basis.

— Barb Flohr

Rapid results on Jakafi…

 I am 54 years old and began with PV in 1999. I had much itching and my HCT was 59!!!

The treatment was through phlebotomies, in a first moment, and HU especially.

But in 2009 my marrow became fibrotic and some blood’s values got down, without becoming anaemic.

About a month ago I began to use Ruxolitinib (HCB 14,1 and platelets 106 thousand) with a spleen 25 cm. long. Now my hemoglobin is 12,4 and platelets are 142  thousand, my spleen has reduced and I feel better.

—  TG (Italy)

Jakafi worked at first…and then didn’t…

My name is Pat Hensley and I was diagnosed with primary myelofibrosis in 2007. I was asymptomatic for the first two years and then as my blood started to deteriorated,   my disease advanced.  I started to get  an enlarged spleen as well as reduced platelets, hemoglobin counts and white blood cell counts.

I had been following the Jak-2 inhibitors that were coming on the market for the preceding couple of years and wanted to get into the Incyte 18424 trial which later became Jakafi. I did so in November 2011  in a trial in Florida.

I began with a dose of 25 mg per day  and got immediate results within the first few weeks.

Pat with wife, Patty

My spleen started to shrink, my itching got better and my night sweats improved.  The first two months was a  pretty amazing experience as everything improved  and I thought that was I was doing great.

After the first two months,  my blood levels started to deteriorate and my spleen started to enlarge again.   Over the next two months, they reduced the dosage down to 15 mg a day and  I experienced a return  of my most of my symptoms. At 16 weeks I had a follow-up MRI on my spleen and it was essentially the same size as when I started the trial , 21 cm .

My hemoglobin went down to 7.7 and I had to have a transfusion for the first time. It was decided at that time that I should go ahead and drop out of the trial as it was no longer effective for me and in fact was now making me worse off. I decided to continue with my ultimate plan of curing my disease by getting a stem cell transplant.   I was successfully transplanted at  Seattle Cancer Care Alliance   on May 24, 2012.

— Pat Hensley

From clinical trial to “the birthday I never expected to have.”

On Monday my oldest granddaughter started kindergarten.  In most families that is a routine event, albeit a special step into the school-age years.  But for me and for my family, this was an exceptional and special event, one that I feared I would never experience.

In 2008 when my Myelofibrosis suddenly became much more intense than it had been in the previous seven years since my diagnosis at age 60, I began to deal with the reality that my 2 year old granddaughter might grow up without ever remembering me.  Her younger sister, then barely 1 year old, wouldn’t have known me at all.

I was losing weight, dealing with an ever-enlarging spleen and lack of appetite as well as intense night sweats and ever-present fatigue.  I realized that, as they say in old movies, “my days might be numbered.”  I remembered my hematologist telling me in 2001 that Myelofibrosis was a “Ten Year Disease,” and I was now fast approaching that “deadline.”  There was no more denial of my desperate situation.

In the next year my symptoms worsened, I became weaker and weaker, and was unable to do even daily tasks in my home.  At the grocery store, I had to drive a “shopping basket car” because I could not  walk  through the store.

In  the summer of 2009, my wonderful oncologist/hematologist, Dr. Thomas Beck, at Mountain States Tumor Institute in Boise, Idaho, took on a new role as my “guardian angel.”  He told me about a clinical trial that would begin in “a few months,” and one he thought might be of great benefit to me.   He then became my medical advocate and continued to gather and share information on the Comfort 1 Study.  After a myriad of tests, physical exams, forms to complete, interviews, etc., I finally qualified for the study in the spring of 2010.

At that point my spleen had reached a horrendous size of 25 cm below the point on my ribs that the doctors use as a starting point.  I could eat very little, weighed only 105 pounds, had minimal energy, could barely get out of bed in the morning and took several naps throughout the day.  I was seriously ill and dealing with my own mortality.  I could not believe that I would not live to see my granddaughters at least start school, let alone become young women.

My family was more realistic than I was about my situation and they helped me cope, but they were suffering as well.  Our daughter hosted a big party for our 45th wedding anniversary because it was feared that I would not be here for our fiftieth.  Jack and I took a celebratory cruise to Alaska.

Then the first dose of Ruxolitinib was given to me on April 19, 2010.  While I understood that Comfort 1 was a double blind study and thus my chances of getting the drug rather than a placebo were 50-50, I had faith that I would know if I had been given the “real” drug.  Within a few days I experienced some symptoms that seemed different and unusual to me.  Hoping these weren’t just “halo effect” symptoms as the result of my wishing to feel something to tell me I was getting the drug, I became more and more certain that my body was reacting to a new substance.

Four weeks after I began the drug, and numerous (almost daily) lab tests, I had my first scheduled appointment with Dr. Beck to check my spleen.  He happily reported that my spleen had now reduced in size by a whopping 25%.  There were a lot of happy tears at that moment by all of us and I called my husband to report the astonishing news even before I got off the examining table.  We were all astounded, thrilled, and too happy for words.

That wonderful progress continued steadily.  In six more months, my spleen size had reached the 6 cm size it remains today, more than two years after the first dose.  While my spleen was shrinking rapidly, my appetite was returning (with a vengeance) and I started gaining weight.

Another highlight of the past few years was the day in November, 2011, when Jakafi received its FDA approval and the drug could be now available to other MF patients who could now also live past the “Ten Years.”  Two weeks after the FDA approval, my family and friends helped me celebrate my seventieth birthday—-the birthday I never expected to have.

Needless to say, Jakafi has saved my life!  I am now approaching Day 900 of the clinical trial and am grateful for every day Jakafi has brought me.  When the trial ends in January, 2013, I will continue on the drug with my medical insurance paying the bills.

Today I have gained back all of my lost weight and “a few more pounds.”  Now I have energy to do all the things I love.  I volunteer at the NICU at our local St. Luke’s Hospital where my second granddaughter was a preemie who spent her first seven weeks there.

My husband Jack and I also volunteer at the Warhawk Air Museum in nearby Nampa, Idaho.  We enjoy helping with their educational programs for students as well as assisting with services for veterans of all ages.  In addition, I play Mah Jongg every Thursday with girlfriends, do a lot of sewing of baby blankets for the NICU, and am learning to do quilting.  I am also working with MPN to develop a patient support group here in Idaho.  Best of all, Jack and I spend a lot of time with our granddaughters who are now 4 and 5.  They are the joy of our lives.  We look forward to their helping us celebrate our fiftieth anniversary in 2014.

Thanks to Jakafi, I had the opportunity to see my first granddaughter start kindergarten!

Next long-term milestone—her high school graduation!

— Susan Hill

On earliest Ruxo trial…now back on hydroxyurea

I have written about the Incyte trial before, so I will just add some of the later experience.

I was doing well on Jakafi for about three plus years.  I went to my regular six month appointment in the last part of December 2011.  I was feeling a lot more tired than usual and I wondered if I should fly the long distance alone, and be able to manage my suitcase by myself.  I told myself that I was okay.  I looked forward to discussing a few things with my doctor, but at the appointment time I only saw the doctor briefly and he didn’t examine me at all, but left that up to the nurses.  The regular BMB went fine and I was able to finish the trip without incident.

Shortly after I came home I had a severe pain under my left rib, which traveled to my shoulder.  I relieved the pain with heat, but I was sick.  It lasted about three days.  I called the Houston nurse for their opinion but of course, I wasn’t there for them to examine.

Later in the month, I was eating lunch with my husband and son, when all of a sudden what I was saying was garbled.  I knew I was speaking gibberish, but didn’t know what was happening.  My husband saw that my soup was running down my chin (charming, huh?).   Immediately they called 911.  The medics took me to the hospital and I found that I had had a TIA.  By the time I got to the hospital, it seemed to be over.

I stayed in the hospital for a couple of days.  After I came home from the hospital I started having severe night sweats, itching, I hurt all over–all the things that happened before I started the Incyte trial.  I called the nurse again.  She talked to the doctor, who just said that he didn’t think it was caused by my earlier pain.  I became sicker and sicker.  They wanted me to come to MDACC but I was too sick.

I had been taking Jakafi all the while, but I decided that if it wasn’t working I might as well stop taking it.  Gradually, I eased off Jakafi.  However, within a week, my spleen had grown enormously.  It looked like a big round ball was in my stomach, and I could only eat about a half a cup of food at a time.  Also, aside from a couple of things, everything tasted weird. I hurt all over, and I looked awful. My primary doctor thought I was in the end stage, and I did, too..

She prescribed Tramadol for my pain. It helped a lot.  I tried to call for an appointment with a local hem-onc, but I was sick and the receptionist asked silly questions (in my irritable state of mind} and I gave up.  I stayed in bed, and only got up when necessary.

Finally I decided I needed to try again, and I got an appointment to see a hem-onc.  He started me on hydroxyurea, which had given me leg ulcers years ago.  But this time it seems to be working, and my spleen is down about half.  I am still weak, but stronger. I am very thin and need to buy new clothes.   I haven’t tried to drive again, because all of a sudden I will feel completely exhausted and have to lie down.  I am able to go out some, and I drive the grocery store carts, to the endangerment of all.

I hear that Jakafi will start working again after not taking it for a while, but I don’t have the courage to try and then have to go off of it again.

— Barbara Beckman

Glad to discontinue…

 I was dx Angiogenic Myeloid Metaplasia.   My spleen has been growing a lot, up to 26 cm. My hem had discussed Jakafi with me for nearly 2 years. My blood counts have been pretty stable since I was dx in ’97.

I was fully informed as to possible side effects before starting it. However, about 2 weeks in, my fatigue became serious, I couldn’t get out of bed and walk to the bathroom without becoming short of breath. I had headaches. My blood counts showed my platelets had dropped from 177 to 38, and RBC dropped a point, HGB 3 points, and HCT 10 points. I developed an infection in the armpit where I’d had a mastectomy 2 years ago.

I took it a month and was glad to discontinue. I am glad it helps some patients, but it was not for me    I have started Revlimid, 2 weeks ago. I hope it does better for me.

– Crystie L. Riley

Jakafi saved my life…

I am 84 with MF for the past t5 years after 10 years of ET. When diagnosed with MF I went to MD Anderson and entered their experimental Jakafi program. Spleen at that time was about 20 cms. and Platelets were over 1 million. Within two weeks all counts except Hgb were normal and have stayed so. I am on 20 mg daily with absolutely no side effects. i have become transfusion dependent every 3 weeks – 2 units.

Feel good and have a great quality of life – Jakafi saved my life.

–  Harry Newman

Jakafi delayed – Hydrea works…

Mine is no great story. A yr ago when my PV turned into MF, I was ill. Enlarged spleen got up 25.4 cm’s and I had the miserable bone pain. I was scheduled to start on Jak Ruxo, but it took almost 2 mos for me to get the med. So, in the interim, my team of HEMAC’s kept me on HYDREA, upping the dose to 1500 mg’s daily from 500 mg’s daily. One month later, all my symptoms of MF were gone and I was asymptomatic of MF. My team chose to KEEP me OFF RUXO due to the toxicity of the drug and the very debilitating and harsh side effects. ……A yr later, my counts are stable and maintaining within norm range on 1500 Hydrea daily. Doc said that I respond so well to Hydrea that they will keep me on it.

— Mimi McGuire

“Jakafi gave Joe his life back…”

Joe’s transformation of PV into MF in 2008 produced adverse effects that affected his way of life. He had a consistent low grade fever, severe exhaustion, soaking body sweats when horizontal and a huge spleen.

A BMB in early summer 2008 confirmed that he had MF-3. Dr. Alan Freedman, Joe’s local hematologist, recommended the INCYTE trial under Dr. Verstovsek at MD Anderson Cancer Center in Houston, Texas.

One week later we flew to Houston, Texas for a week of tests and consultations. Most of the MF patients were approved to enter the trial. Joe was patient number 81.

Joe started the INCYTE on 9/12/2008. He noticed within less than one week that he had more energy. Within two weeks his sweats and fever subsided. Joe was thrilled that he had his life back. At the end of October Joe’s CBC results showed the he was anemic. Dr. V told him to cease the INCTYE. Joe and I were both apprehensive about that since we were given no indication he would go back on it.

Two weeks later Joe’s blood counts improved. He was put on a lesser dose and gradually went back up to 15 mg twice a day. Joe’s quality of life continued on the INCYTE. All the adverse effects of MF were gone and he started to regain weight since his spleen had decreased in size.

Springtime in 2009 Joe went anemic again and the INCYTE was stopped again for a few weeks till his counts rebounded.

Joe continued on the INCYTE trial until September 2011 when he had to stop taking it so he could receive intense chemotherapy for Mantle Cell Lymphoma. The MPD lists emphasized the danger of Joe going off the INCYTE and that some people had died when they went off of it too abruptly. I researched as much as I could in a short period of time and went with my instinct to taper Joe off INCYTE. He had no adverse effects coming off it.  We were told that Joe would likely not be allowed back on the INCYTE trial once he had completed chemo since now he had another cancer.

I personally appealed to the INCYTE research physician and the PR department. They told me that they would see what they could do when the time came.

With Joe in remission in June 2010, we had a new consultation with Dr. Verstovsek. We held our breaths for a few days when we joyfully received the news that Joe was being allowed back on the trial. It was decided that he was considered on the trial but had a lapse of treatment like when he was anemic. Joe restarted the trial at 15 mg twice a day and remains on it.

Joe’s Quality of Life has been excellent. He is the one who cheers what INCYTE and Jakafi have done for him.

 — Bonnie Evans

 My Jakafi trials…the jury is still out.

To give you a little background, I have been dealing with MPNs for at least 35 years, probably longer, and am now 57.

When I had my diseased gallbladder removed in 1977, my platelets were over 900,000.  They never even mentioned this to me, so I wasn’t actually diagnosed with ET until I was admitted to the hospital in June 1982 with pre-eclampsia.   My blood pressure was so high, that they did an emergency C-Section at 28 weeks gestation.  I had an enormous hematoma from that surgery which took over 6 months to dissipate.

It was then that they did my first bone marrow biopsy, and with a platelet count of over two million, diagnosed me with ET.  I was put on Hydroxyurea for about three weeks and my platelets dropped to 750,000.  Since I was only 27, they took me off the Hydroxyurea, my platelets went up to 1.3-1.4 million, and I went on “Watch & Wait”, for the next 23 years.

When I turned 50, my platelets dropped to 750,000 and then to 450,000.  I was excited, thinking my body was finally normalizing, only to find out they wanted to do another bone marrow biopsy which confirmed I had progressed to MF.

They put me back on the Hydroxyurea to bring down my high white count, hopefully shrink my spleen and slow MF progression.  My bone pain worsened over the next three years so I had a consult with Dr Mesa, who told me about the Ruxolitnib clinical trial.  I went off the Hydroxyurea in August 2009 in preparation for starting the clinical trial.  At this point my spleen took up most of the left side of my body, crossing mid-line in width and going clear down to my groin and starting back up again in length.

My Jakafi journey began with me not qualifying for the trial because no blasts showed up in my first blood draw, which was one of the requirements.  My Doctor had seen a couple on my bone marrow biopsy report so she told me to come back in a week for another blood draw.  It was weird that I went home and prayed for one blast to show in my next blood draw.  Well, prayers were answered and the next draw showed one blast.  I was accepted into the trial in October 2009!

Keep in mind that at this time, I had a huge spleen causing constant discomfort and intense pain when I would bend or twist at the waist, fatigue, mild anemia, bruising and bone pain.  I didn’t have the intense itching or wasting that many others with MF experience.  I was overweight when I started the trial.

Unfortunately, I was one of the unlucky ones who received the placebo for the next 15 months.  With no treatment, my fibrosis worsened from a 2 to a 3 on the WHO Prognostic Scoring System.  I was now taking Oxycotin and Oxycodone daily to lessen the bone pain.

The week before Christmas 2010, I received the call that they were finally unblinding the clinical trial and they scheduled to see me December 23, 2010.  What an awesome Christmas present!  I was finally going to receive the actual drug and hopefully experience a better quality of life.

I was started on a dose of 20 mg twice per day since my platelets were over 400,000 and my hemoglobin was over 12.  During the next three months my spleen shrunk dramatically.  It was no longer in my pelvic area and was about ½ the original width.  I was feeling even more fatigued so my Doctor suggested that I cut back on the pain medication.  To my surprise, I was able to stop the Oxycotin and Oxycodone completely an only needed occasional Tylenol.  This did help with the fatigue.  I also was rarely having spleen pain now.

The big negatives for me were the excessive weight gain…45 pounds…so I am now obese, and severe swelling of my hands and feet.  My platelets dropped to around 126,000 and my hemoglobin to around 10.  We decided to lower my dose to 15 mg twice per day for about a month, then 10 mg twice a day and finally 5 mg twice per day in June 2011 to see if it would help with the swelling, but it didn’t.  Instead my spleen rebounded my counts dropped and I ended up in the emergency room June 12, 2011 with intense abdominal pain and a hemoglobin of 7.

We immediately increased my dose to 15 mg twice per day and I had my first and only transfusion of 2 bags of packed red blood cells on June 14th, 2011.  Upon closer review of my blood counts, we realized my fasting glucose was 199 and my Triglycerides were over 400.  I also had sinus tachycardia and an occasional erratic heart rate.  This prompted my wearing a Holter Monitor and having an Echo, which revealed several minor heart issues.  I went on several new meds and Presto, the swelling was gone!  When the next lab, in September 2011, still showed a high fasting glucose, I went to my GP and am now on meds to lower my Glucose.

Things were going well until mid November 2011, when I was rushed to the hospital with breathing problems.  It was my 1st and hopefully last ambulance ride.  They kept me for 4 days on steroids and breathing treatments.  We don’t know what caused this episode, but I now add COPD to my growing list of medical conditions and carry a rescue inhaler everywhere I go.

During the next few months, I worked hard at trying to eat healthier and lost 20 of the 45 pounds I’d gained.  I started having some sporadic spleen pain again and the MRI in February 2012 revealed that my spleen had increased in size so we upped my Jakafi back to 20 mg, twice per day.  The good news is that I rarely have spleen pain now so I think we can safely assume my spleen shrunk again.  The bad news is that I gained back 12 of the original 20 pounds I worked so hard to lose even though I didn’t change my eating habits.  My platelets also dropped to 109,000.

In April 2011, I came down with Shingles.  I wouldn’t wish that on my worst enemy!  I am happy to report the pain is finally gone.

As I write this I am waiting on results of last weeks’ blood draw.  Things have been pretty stable, “knock on wood,” with only a slight increase in bruising.  I am currently still in the Jakafi clinical trial.  They expect it to close later this year.  I am currently checking into new clinical trials as I assume the recent need to increase my dose may mean it will stop working for me in the near future.

Am I still glad to be in the Jakafi clinical trial?…Yes, definitely.  Do I think my heart issues, Diabetes and COPD were caused by Jakafi?…Probably, indirectly.  I believe the weight gain was the reason for the added health problems and that Jakafi caused the weight gain.  Would I recommend it to others?  Definitely!  My Doctor said that it seems to help the elderly and those dealing with wasting the best.  I think anyone who is transfusion dependent may want to look into other clinical trials and those who have lower counts, should probably start on a low dose of 5-10 mg twice per day and gradually increase as their body tolerates.

I will end this with I am very glad to be alive after dealing with MPNs for over 35 years and hopeful that better meds are on the horizon and maybe even a cure in our lifetime.

— Diane Blackstock


One year later, doing well…

I was diagnosed with myelofibrosis 29-Dec-2010 by my hematologist.  He  informed my wife, Nathalie,  and I that a bone marrow transplant was the only potential cure and referred us to a transplant doctor for evaluation.  He also informed us that drugs were available to help alleviate symptoms; but they did not cure the disease.

We had no idea about bone marrow transplants nor about myelofibrosis – except for the little we knew about a colleague who had recently returned to work after a one year absence.  Coincidentally, he underwent a stem cell transplant to treat myelofibrosis.  Needless to say, we were very anxious for him to return from his overseas Christmas vacation, so we could learn from his experience.  But, that would be a couple of weeks away.

In the meantime, we scoured the internet for information and came across a very helpful group MPD-NET, as well as numerous articles on the disease, clinical trials and treatment options. We read the articles with great interest and difficulty.  Many required knowledge of the medical and scientific terminology; it was like reading science fiction in some foreign language.

Through MPD-NET we obtained the name of a specialist in our area and also learned of an upcoming patient-doctor conference in Scottsdale.  We immediately made arrangements to see Dr. Verstovsek  on 20-Jan and to attend the Scottsdale conference 12-Feb.

My colleague never did return from Christmas vacation.  We had news that he had passed away due complications from his stem cell transplant. That sad news also delivered a heavy dose of reality around the risks involved with stem cell transplants.  The statistics on first year mortality, suddenly had a face and a name. I was not anxious to add mine to those numbers.  It was a heavy blow.

20-January-2011, we went to see Dr. V at the MDACC.  The hospital’s sheer size is impressive and it was somewhat comforting to see many other patients with MPNs in the large waiting room. I was not the only one with this rare disease.
The first visit was an all-day affair, with admission formalities, blood tests, MRI and a bone marrow biopsy.

Dr. V lived up to his reputation as a kind caring, considerate and professional person. I realized my good fortune to have landed here, with this facility and this doctor and his team.  Couldn’t ask for better.  Many patients travel great distances to come here; with good reason.

We agreed on an initial treatment with Danazol, to help improve anemia and fatigue, then consider participation in one of the  clinical trials for JAK2 inhibitors.  A transplant was considered too risky.

After leaving Dr. V’s office I met with my hematologist to review the plan.  He agreed that I continue under the care of Dr V.  I cancelled the appointment with the transplant doctor.

Three weeks after our initial visit with Dr. V, we attended the Scottsdale Conference.  The timing was perfect and the conference, was of tremendous help in our decision making.  We met other patients with various MPNs, on various treatment plans,including stem cell transplants, and learned much from their personal experiences. We also met some of the worlds leading experts, including our own Dr. V, in MPN research and treatment. Their presentations and patient/doctor discussions were extremely enlightening.

At the same time, we began to realize that there is still much to learn about this disease and not even the experts agree on the best treatment options. But, we left the conference with a strengthened sense of optimism thanks to those kind and caring patients and doctors.

After returning home, the next 6 months were fairly uneventful, apart from continuing weight loss, which I found worrisome.  I was unable to maintain my skinny weight in spite of a high-calorie-everything diet. I was starting to look like a starving person and had very little energy. Little by little we found some good MPN Support Networks: MPNResearchFoundation,Leukemia and Lymphoma Society, MPNForum, MPN Private Support Group.  There are others, but these work well for me.

September 2011, I started in a clinical trial for a sustained release version of Ruxolitinib.   Magically, the night sweats disappeared within the first few days.  My spleen size shrank, after slight dosage adjustment, over the next few weeks and fatigue improved significantly along with the motivation to do things.

Blood counts dropped slightly initially but did improve with time.  Other than that I suffered absolutely no side effects.  Oops – there is one – weight gain!  I began to put on some of the pounds that I had lost.

Things were progressing nicely, when out of the blue, a scathing attack on the use of Ruxo appeared in one of the prominent medical journals by one of the prominent specialists in MPN.  It disturbed us greatly, because it came from one of the leading specialists.  But on reflection we had seen signs of passionate disagreement, at the Scottsdale conference.

We did more Internet research, more reading, had more questions for my doctor and after carefully evaluating the facts available to us – We decided that this was still the best path forward – for us. Then some Great News! November 2011 – the FDA approves Ruxolitnib for the treatment of myelofibrosis.

In January 2012, I transitioned from the clinical trial to the commercially available Jakafi.

September 20 was the one year mark since starting Jakafi.  I’m doing very well and am grateful to many, for that.

– Charles Nielsen

Dosage issues, side effects….

Here is my story; My name is Michael,I am 50yrs old father of 4 children ages 24-21-17-7yrs old.I have 20 yrs in the transportation industry. I was born in Boston Ma. and grew up right next door to Logan International Airport.

There is a reason why I need to let You know where I grew up .I lost my dad (also 30yrs in transportation industry) to acute leukemia when he was 58yrs. old. He also grew up in the same area as did his parents, both died from bowel cancer, both where late 70’s.

I was diagnosed with Myelofibrosis and the JAK2 mutation through a bone marrow biopsy in 2005. Symptoms that had me go to the local clinic were a loss of 30lbs, no energy and a painful blue baby toe of all things.Thats when I was sent to an oncologist

I was put on hydroxyurea…I remained on this until March 2012.My  oncologist had me see Dr. David Stone@ Dana Farber In Boston,and confirmation of the Jak2 mutation and Primary Myelofibrosis were determined through an updated bone marrow biopsy. I was looking to participate in a clinical study, my counts were unstable, had bone pain,fatigue, spleen was 17 cm.

Dr. Stone informed me of the new med JAKAFI and I have been on 20mgs 2x a day since March 27 2012. Since my last visit approx 4wks ago, my blood counts were good , spleen reduced 20%,  still have bone pain, some fatigue, night sweats but not as frequent.  I have been going through EMG testing( having pain,cramps,weakness in legs & back pain) Had EKG testing and recently,3 days ago,  had a Dibutamine (stress test) waiting on results.

I started feeling extreme tightness in right arm and chest roughly 8-10 times in the past 45 days and I’m getting frequent severe headaches very strange feelings.

I have also have had lately high blood pressure and now take 75mg Attenenol and 5mg Lisiniprol. I havent been able to work and Im still waiting for SSDI approval. I have a couple of questions: 1-Do MPNs cause heart disease? 2-What does JAKAFI cause? 3-Living near jet fuel,diesel fuel and other carcinogens have a major role in the breeding of MPNs and gene mutations? 4- and what can be done to stop it? …

…Just updating info in regards to my last appointment on 7/17. My doctor @danafaber has reduced my Jakafi to once a day(20mg) due to extreme dizziness and headaches. Blood counts were a bit higher also and he is having me see a neurologist,,,to see if they can figure out why

— Mike Masello

Burning, swelling. Allergic to Jakafi

My Jakafi experience was shortlived but none the less eventful.

Getting there started Feb. 29, 2012 when I finally started on the clinical trial for Pegasys.  I  had PV for 11 years and had been on HU for 5 years and waited for 2 years for this trial to open up as I had read so many good things about Pegasys being able to reverse early stages of MF if started early enough.

For four months I thought things were going relatively well as my WBC finally started to come down (still not in the normal range).  My platelets had come down nicely also and my RBCs were being kept down.  I hadn’t needed a phleb the whole time.  I was experiencing an enlarged spleen since 2009, but after 4 months on Pegasys, I started having considerable pain on the left side under my ribs and my back.  It was extremely uncomfortable to lay on my back or side. I was also having pain when I was eating.

An ultrasound showed that my 20 cm spleen had now grown to 30 cm in these four months and a BMB showed that I had converted to sMF, so I was pulled from the trial.  Now wondering what my options were, I had a consult appointment at the Mayo Clinic and it was there that my options were narrowed down to trying Ruxolitinib since it had good results shrinking spleens.

Even though Ruxolitinib has been FDA approved for MF, it is such a new expensive drug that it needed approval from my drug insurance company.  Now about 5 weeks after stopping the Pegasys, I had 20mg of Ruxolitinib in hand.  Since my platelets were well above the low limit I was started on 20mg twice a day.

I took one dose on the evening of Aug. 7.  In the morning I noticed that my face was slightly flushed but didn’t think much about it.  Took the second dose and throughout the day noticed that my face became increasingly red till it looked like I had a severe sunburn and it felt like it too.  It was burning.  Before taking the third dose, I called my hematologist to ask his opinion.  I was told to discontinue the drug and come in the next morning.

Throughout the evening my face continued to burn.  At 9:30 pm called the pharmaceutical company.  They called this redness a “rash.”  I was advised to go to the ER if I began to run a fever.  I began running a fever and went to ER.  Since it is such a new drug, no one knows anything about its side effects.  Was breathing, walking, and talking so was sent home.  Went to bed wearing a cool wash cloth on my face all night.

When I awoke the next morning and looked in the mirror, I looked like a freak!  Every part of my face was swollen except right around my mouth.

After my immediate frightened response I received a call from the pharmaceutical nurse to ask how I was.  I now told her about the swelling.  Then I discovered that the rash now covered my arms and upper legs.  She told me to immediately see my hematologist.  She even asked for the name and phone number and she called them and called me back immediately with an appointment within 40 minutes.  Was put on Benadryl. If it didn’t look better or became worse in the next 24 hours to go back and would put me on steroids.  Swelling and redness started to subside.  Within about 6 days it was gone.

It was then decided I should try the drug again but at the lowest dose of 5mg.  Two weeks after my first dose, I now took a 5mg dose but premedicated with benedryl.  I awoke at 5am and discovered that my face was once again bright red.  I took more benedryl.  Two hours later my face started to swell slightly.  Didn’t take a 2nd dose!  Continued with the benedry; and by the next day I was back to normal.  It was decided that I did have an allergic reaction to the drug.  This was nothing they had experienced before.  I guess there really is a first for everything.  Can’t wait for the next chapter in my sMF saga!!!

Note:  Currently trying to get on the Sanofi trial for MF people intolerant to Jakafi.

– M.C.

Take me back to the Contents

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Comments on: "The Jakafi Report" (10)

  1. Lucille Whalen said:

    I have been on Jakafi for a year 10 mg twice a day. My spleen was quite swollen and uncomfortable but after starting the Jakafi my spleen shrunk and is now at a reasonable size. I see my Oncologist every two months and my life has returned to normal. While I am not as full of energy as I was before being diagnosed with Myelophybrosis disorder five years ago, I am feeling well, drive, go to my exercise class three times a week, join friends for lunch three times a month. In general my life has returned to normal for someone my age of 87.

  2. Can someone tell me how to get this medication willing to do anything. Have no insurance. Please I would pay whatever the cost. Please email me at andreaveerasammy2@gmail.com

  3. Joy Swedosh said:

    I found these entries very interesting and informative; particularly as I was one of the participants. I was very interested in finding out how others dealt with their MF and how Jakafi helped them. Thank you Zhen for a job well done.

  4. Hi, Melissa here – (my story is above.) I am having great results with Jakavi. It was so interesting to see all the different reactions to the drug. Thanks everyone. I too believe that soon there will be something even better! I wanted to add that I also do a visualization when ever I remember. ( I used to do it every night) I imagine a giant computer screen like in a sci-fi movie or Disk Doctor on my MAC. Here is what I visualize: I see my DNA running through a check up on a GIANT computer screen. one DNA at a time – but going very quickly doing the check up. suddenly a light flashes and the computer image stops at DNA 617F and flashes “Alert, Alert”. Then a scientist in a white lab coat checks the computer and presses a giant button that says “REPAIR”! She pushes the button and the screen flashes “617F REPAIRED”. then the computer continues to scan my DNA strands.

    they say what you think has an effect on your well being…..

    My best to all. I hope those who are not helped by Jakavi find something else.
    Melissa

  5. This collection of authentic “Jakafi Stories” is an up to date compendium of the effects of the drug on real people and friends whom we know. I second my friend Barbara Kurtz’s comments and deplore with much love and concern the plight of my other ‘Barbara friend’, Barbara Beckman. A.

  6. mimi mcguire said:

    Hello to ALL: Thanks for sharing all your stories with your experience with JAK RUXO. They were very enlightening and all so varied and interesting. I find all the MPN conditions to be so puzzling–they react so differently with everyone, and some patients still struggle with all different drugs to find relief to no avail?? (I am MIMI who was not put on JAK RUXO and am still doing well with 1500 mg’s daily HU) I respond so well to HU, doc decided it best to keep me on Hu.)
    Also, I thank Zhen for his great job on the MPNForum magazine–great job and I know how hard you worked on it. KUDOS, ZHEN!….You should be quite proud–the magazine is great and refreshing.
    Happy Trails,
    mimi

  7. Barbara Kurtz said:

    Thank you all for sharing your stories and as always I like the pictures. I recognize some of names and seeing a face just adds something. Also thank you all for being on the front lines of a new drug. I am happy for those it helped and sorry for those it didn’t work out for. With all the research going on I’m sure something will come along.

  8. BonniecEvans said:

    Great to see that many have had good results form Jakafi. There is no doubt in my kind that in a few years something better will come along. just wait and see!

  9. Thank you all for sharing your stories. I wish I had known about Jakafi before I had my spleen removed, still, it has given me my life back.

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