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I know my Pegasys experience was very unusual but I can’t be the only one and I believe if I had started at 22.5 instead of the usual 45, I might have tolerated it better.  Just before I stopped taking it, I read an article in one of the journals that said they were afraid that some interferon effects could be permanent and that frightened me.  So, I believe more research should be done and am happy to help

The winged horse – An ET patient’s Pegasys journey.

by Elizabeth Goldstein

According to Greek mythology, Pegasus was a white winged horse. An ancient hero, Bellerophon, attempted to ride Pegasus to Olympus, home of the gods, but Zeus saw them and sent a horse fly to bite Pegasus.  Pegasus bucked and Bellerophon fell back to earth.  Zeus then put Pegasus in the night sky where he transports thunderbolts for Zeus.  If this myth was a metaphor for my experience with the drug, Pegasys, I would be Bellerophon because it surely threw me to the ground.

After reading several articles by Dr. Richard Silver, Dr. Hans Hasselbalch and other experts in MPNs, I was convinced that Pegasys (peginterferon alpha-2a) would be a far better drug for me than the hydroxyurea (HU) that I had been taking for four years to reduce my platelet count. I have ET, JAK2+ and because I was 64 years old when diagnosis was confirmed by a bone marrow biopsy, my hematologist started me on HU immediately as my platelets had risen rapidly from 450 to over 700 and it appeared that they would continue to rise without suppressive therapy.

HU is an oral chemo drug and I took 500mg. daily, a low dose, to bring my platelets down to 475-525 fairly quickly.  The experts said that Pegasys would not only do a better job of lowering platelets, in most cases, but it could put my disease in remission and possibly heal my damaged bone marrow.  My hematologist agreed that it would be good for me but since I was tolerating HU, she said my insurance company would never approve it.  I gave up the idea, resigned to taking HU forever.

A few months later, I began having diarrhea often and then often became every day and trips to the bathroom were becoming more and more frequent. At my next appointment, I told my hematologist about this problem and her face lit up and she said, “You can no longer tolerate HU, now we can ask for Pegasys!” I got excited about the drug again but didn’t anticipate my insurance company’s resistance to paying for a much, much higher priced drug, about $3,700 per month without insurance.  After several denials from the company, one after a letter from my hematologist, I started doing research.  I found as many pro-Pegasys articles as I could, copied them and took them to my hematologist.  I wrote on the top of one a quote from Dr. Hasselbalch, “It is immoral not to offer a patient the best treatment available.”  Now, armed with an inch thick packet of articles, my hematologist applied once more.  Both she and I received a very confusing letter from the insurance company’s oncology consultant stating that since I had failed Peg Intron (another form of interferon), I was not eligible for Pegasys.  I had never taken Peg Intron but we reasoned that if that was supposedly approved, there was no reason not to approve Pegasys.  We had them on a technicality, and the first week of July, 2014, I received my first shipment of the drug and began injecting 45mg, the starting dose, weekly.

Although injecting myself was surprisingly easy, my fatigue immediately worsened and I felt slightly nauseated much of the time but I was willing to take that. Then, I began actually vomiting, with no prior nausea or warning.  Several times I was in our family room and knowing I couldn’t possibly make it to the bathroom, yelled “trash can” and my husband got the trash can under my face just in time.  That went away after a month or so and then I began having severe, nightly diarrhea, getting up 4-5 times nightly.  I began losing weight and early in the morning one day, my dog licked my nose to wake me up.  I was in a pool of excrement.

Still, I soldiered on. This drug was supposed to be healing my bone marrow and if I was lucky, could put my disease in a complete molecular remission. I must have inherited my father’s iron will.  He had been a fighter pilot in WWII, was shot down and spent 13 months in a German prison camp.  All of our lives he told my siblings and me that we could do anything, bear any physical inconvenience, if we just put our mind to it.  For instance, he refused pain medications or Novocain, no matter how bad the procedure was, at the dentist’s office.  He said that if he told himself it wouldn’t hurt, it didn’t (don’t try this at home – it didn’t work for me).  So, because I didn’t want to “wash out”, I stayed with Pegasys.

Toward the end of 14 months, I had a series of strange side effects. First, I had sudden, total muscle weakness.  One night, I couldn’t get in the bed that I had gotten in for 18 years.  I had one leg up but was incapable of pulling the other up and fell to the hardwood floor. I devised a way to get in the bed by using a stool and holding on to the headboard.  But one evening, returning from the bathroom, I turned right instead of left, the way to my bed.  I ran into the wall, very hard and fell on the floor again but could not get up, even holding on the leg of the bed.  My husband tried to get me up but I was dead weight so I told him to just give me a pillow and a blanket so we both could get some sleep.  That lasted a few weeks, then went away and was replaced by loss of coordination.  I didn’t get dizzy but would just fall, usually onto a chair or sofa.  I was okay if I could hold on to something, like a grocery cart and okay if I was seated so I could drive but getting in and out of the store was treacherous.  This, too, disappeared in a few weeks to be replaced by the strangest effect of all, a change in my gait.  Instead of my normal stride, I began taking small, rapid steps like a Geisha girl in a big hurry.  My husband noticed it and was astounded but it only lasted a week and it, too, was gone.  My hematologist worried about a TIA but I told her that I never lost consciousness, never had any trouble speaking and never felt confused or acted confused and I didn’t think I’d had anything but side effects of Pegasys.  However, I told her I had reduced my dose to 22.5mg and felt better, which she approved.

Then, the final blow was the worst depression I could ever have imagined. I had been taking Wellbutrin since starting Pegasys to try to avoid this side effect but Wellbutrin was no match for this depression.  By January, 2016, I knew I was depressed and gave myself my last injection on January 6. About ten days later, I was so depressed that I couldn’t, literally, get out of bed.  I stayed there for two weeks in absolute agony, then began getting up a few times a day but was still terribly depressed and cried much of the time.  I didn’t leave my house until March 1, when I had my appointment with my hematologist and I was worried about where my platelets were and if she would be angry that I hadn’t called her.  My platelets were 425 after two months – they had been about 340 in January – and my doctor understood why I didn’t call.  She said to come back in 4 weeks and we’d see where we were.  In April and May, platelets were 416 and 429 in June.  At that time my hematologist said she believed I was in a remission but neither of us had any idea of how long it would last so we continue to monitor it.

My depression finally lifted completely in April but on May 9, my father died at age 99 from a stroke which left him completely unresponsive and then he passed ten days later. My amazing father who stayed in the Air Force after the war and joyously flew every jet made until he retired.  Then he flew a corporate jet until he was 71 and raced stock cars as a hobby until he was 77 and my brother took the reins.  He was known as the Silver Fox to local pilots and race drivers and often referred to as “the original Joe Cool”.  He stood on his head and then walked on his hands across the floor, to entertain us, until he was 80 years old.  I suppose my siblings and I thought he would live forever.  His death made me slightly depressed but it was the normal depression caused by grief.  I miss him terribly but know how fortunate we were to have had him as our father, for so long.

I am still monitoring platelets and still in a remission as of July. If, and when, my platelets get over 525, I will be on the lowest dose of HU once again.  Since so many people do so well on this drug, I wonder if there’s something in my particular genetic profile that would offer a clue as to why I had such a hard time on it.  I will never know, I don’t think Medicare pays for extensive genetic testing and it really doesn’t matter now.  Looking back, I stayed on it far too long, 18 months, but I’m my father’s daughter and I don’t give up or give in easily but that beautiful white winged horse dropped me to the ground and I finally called “uncle.”

Response from Dr. Hans Carl Hasselbalch…

 Certainly an unpleasant journey with Pegasys. 

I would never permit a patient to continue treatment with Pegasys or PegIntron with such a severe burden of side effects . As we underscore in all of our papers some patients do not tolerate IFN and have to discontinue the drug – even on low-dose Pegasys ( 45 ug x 1 sc /week ) – actually about 20-30 % of the patients .Accordingly, 70- 80 % fortunately tolerate IFN very well .  About 25 % of patients on HU discontinue HU due to side effects or they are refractory to the drug .

One of the many challenges in the future is to unravel the reasons for IFN intolerance in a subset of patients. In the near future we hope to be able to contribute with novel information in regard to  the mechanisms behind IFN intolerance in some MPN patients – by current gene expression profiling and molecular studies in our Danish Low-Dose IFN HU – DALIAH –  cohort of 200 newly diagnosed ET, PV and hyperproliferative MF patients who have been randomized to Pegasys 45 ug x 1 sc/ week or PegIntron 35 ugx1 sc/ week ( below 60 years ) and in those above 60 years a third HU arm is added. All 200 patients have been enrolled and data analysis is ongoing . Our second cohort is our COMBI Trial in which we combine IFN and Ruxo in patients intolerant of or refractory to IFN . Hopefully, this study will also yield novel information   On ” The Mystery behind IFN- Intolerance .”

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