The fixer of everything.
Her name was Voncille but we knew her as Peppe, to the extent we knew her at all.
For years Peppe would pop up on Facebook pages or MPN email lists.
She would smile, volunteer help, offer advice, complain, dig in and fight for something she believed in, open her heart and always burst on the scene with a big smile and high energy.
She was diagnosed with myelofibrosis in 2005. Two years later she was referred to MD Anderson for a stem cell transplant. She was approved by the transplant specialist but denied coverage by Medicare.
That was the start. She and her hematologist would struggle for nine years to find alternative therapies when few options existed. In October, 2016 Medicare finally approved a clinical trial that incorporated stem cell transplant.
In the summer of 2014, in her writing about the PRM 151 clinical trial, we all got to know Peppe a lot better. She was on the PRM-151 trial and had been on three others before that. She believed in the drug. It was helping her and she wrote a bit about it in the Forum.
Her narrative is a good place to start telling her story in her words.
He sent me to do all the testing and I got a diagnoses of MF. He watched my counts for 6 months and finally decided to send me to MDACC for a transplant.
I was seen by Dr. V for a diagnosis. After we sat down and spoke about options and what I was there for, Dr. V sent me to the Transplant Dept. to see Dr. Popat. At that time he was not able to answer my questions about transplants in MF patients because there was no data. This was breaking ground. So I wasn’t sure where I stood with my decision.
Here’s the wonderful part of this trip to Houston. I passed all the mandatory testing for a transplant, and was approved by Dr. Popat. Then when it came to insurance, I have Medicare and they will not pay for a transplant for MF. and still won’t till this day. I was devastated. I couldn’t talk . I had to walk away. It was out of my hands. I was always a fixer of everything in our family, but Mama was broken and there was nothing I could do about it. I got that part right away. So next day is another appointment with Dr. V and he gave me options and hope.
(In her article, she talks about her Revlimid trial with prednisone, 6 months, pomalidomide in 2009, BMS 911543…all terminated for adverse effects or lack of efficacy. She has become transfusion dependent. In October 2013 she and her doctor discuss the PRM-151 trial.)
I could see the excitement in his eyes, face and his actions. I said to myself, I’d hate myself if I don’t try this and it’s a success. So after our conversation I told him I was with him, lets do it.
PRM-151 is different than all the other drugs, in the fact that it’s not pills, but given through an IV or port, so you have to be at the clinic each month to receive the drug. You wait about 2 hours for a bed and it wears on you. The days are long, the time on the road driving is long, it’s all a huge pain in the butt, but knowing that I am finally having positive results after all these years is so exciting for me. I’m going to hang in there as long as I can.
I was on Jakafi with the PRM and asked to stop the Jakafi. I was getting blood transfusions every month. I didn’t feel well and I felt it was the Jakafi. So he said ok. That was 4/15/2014. While on Jakafi and PRM-151 I was receiving blood transfusions about every month. Once Jakafi was removed my blood counts slowly began coming up with no transfusions and I felt better.
About 6 weeks with no Jakafi hgb is now 10.5, usually it stayed in the 8’s and dropping. I think this is what helped my breathing to be honest.
It’s so easy to assume that the study drug is causing this or that. But with a great doctor in your corner to help you understand the ins and outs and one that genuinely cares about you makes you feel a little more in control and not falling apart because you don’t know what’s going on.
My thanks and gratitude to Dr. V, his team and to Promedior Company. I finally have hope!
(In the Summer of 2015, she is optimistic about PRM-151. Here she responds to a Comment on her article:)
I know exactly what you’re saying and feel. This is the best I’ve felt in a long time. When I was dx, I had been <10 many many times. I was given a prognosis of 2 – 5 yrs. Here I am at 9 yrs. Not every day is great, but I’m thankful for what I have.
(On Mon, Dec 14, 2015 at 4:03 PM, Voncille Fryou responded to an MPNforum question.)
So good to hear from you. Well this is my think after listening to Dr V at the end of the ASH Conference with Andrew. I think our doctors keep us pumped up about all these drugs that [are] all in clinic[al trial]. But you and I both know it takes years and years to get these drugs to the FDA for approval. I think they’re trying to keep our hopes alive. Prm-151 does work Zhen, I’m living proof and so are my BMBS I’ve done every 3 months for over 2 yrs. Is it still working now? When I seen Dr V in Oct two years ago, he was more excited about PRM-151 than any other drug I ever seen him speak of….
The first order of business is trying to keep us alive and a better quality of life. I had that for a while. Counts were mostly in the normal, except platelets. They’re presently at 29,000, after Houston last month I didn’t feel well so I went to the doctor and they ordered a CBC. Everything was still stable, but the platelets dropped in a matter of days, from 34,000 to 27,000 and pretty much held there all month. This is the scariest thing I deal with…
There’s no drug here that works forever, and I have been through them. I’m afraid the drug is starting to fail me. I’m tired so much, lots of pain, muscle spasms that are torture, I can’t sleep by a few hours at a time. Perhaps the stress in my life is overwhelming me and bringing about all these symptoms. I just don’t know Zhen. The drug does work Zhen, but perhaps two years maybe all it will be doable for me. As always, wait and see….
(By April, 2016 Peppe posts about complications to an MF Facebook page.)
My visit with Dr V was very informative. It seems my huge spleen has caused me to have cirrhosis of the liver. The spleen size has become very serious. We have to reduce the size quickly. If we don’t I’m at high risk that the pressure in my liver will cause the blood in the liver to back flow into my esophagus. I have veins that are forming to help release the pressure but it’s not enough. I have portal hypertension. Dr V and I agree not to remove my spleen, unless last resort.
(April 17 2016. In an email Voncille talks about her need to reach out.)
From the very moment I began to learn about my dx I always had the interest of others in mind as well as my own. What if my son’s, my future grandchildren and beyond would be dx with this? There was no cure, no drugs, barely any studies. Local doctors barely new of the cancer, what about all those after me? Someone had to help the doctors. I felt after meeting Dr V, it was me. We needed something besides transplants, when transplant doctors could not give you data, because there was none past 5 yrs.
My current situation has caught me uninformed. I had no idea about this as a possible end to my life. I should have! Zhen, besides looking a little tired I’m the picture of health.
(April 18, 2016, from a letter Voncille wrote to a patient who had undergone SCT and raised funds on-line)
I’m not ready to leave my family, I feel even in my limited body life has just begun. I have a granddaughter named Grace.
She was born right about the time I was Dx in 2006. She has been my life line to fight and not give up. Now I have another grandchild, an adorable blue eyed boy named Gabe. He has lite that fire in my heart and desire to be here to watch him grow, see him walk and talk, and watch my youngest son being a father. Life is good, I need a transplant to live longer. I’ve lived 13 yrs with PMF that we know of through labs. But I have been sick about 20 yrs. I have done 4 studies and Jakafi. Dr V said, if we don’t reduce my spleen size which is putting extreme pressure in my liver, the blood flow will go up and into my esophagus, or the huge veins will rupture and I’ll bleed and possibly die. I’m running out of time. Medicare is going to pay for a study they want SCT vs drugs. Dr Popat said in his email it will start in the next 4 to 6 months. In the mean time we will start a new study to try to shrink the spleen. It’s possible the drug might work, but for how long?
After a while they all stop working. So I need to take this small window of time and do this Medicare SCT study. I am on disability and receive about $750.00 a month. My rent is $400.00 and with other personal expenses I can not save for living in Houston. I have asked people here that have the ability to raise money, but there have been no answers. If you can help me in anyway, I would be so grateful. My life has been a difficult one and finally my heart has found happiness and love in my grandchildren and two sons, what a shame to loose it now. Gezzzz while I was typing to you a snake came sliding by me on the patio. A Louisiana Water Moccasin. About 5′ long. Thank god my Yorkie wasn’t out…
(That Fall she wrote she wanted to help patients avoid what she had endured. She was concerned not to mention her doctor, for whom she had great affection and respect.)
October 15, 2016
I know you’re not a fan of clinical studies. I have always felt none of the studies have hurt me in any way. But after having an ultra sound done of my spleen and liver, I now know otherwise. I would like to discuss this with you over the phone in maybe the next few days. You know I have great respect for Dr.____ and would never do anything to hurt him in any way. So I have to have your word we will not use his name, perhaps mine either.
I feel like I have been sacrificed for research Zhen. When I tell you my story you will understand. Perhaps, he was trying to give me as many days, weeks, month or years he could. But, test don’t lie. My reason for coming out of the shadows is to educate others before what happens to me happens to them.
The Stem Cell Transplant, too late.
Voncille’s stem cell transplant took place October 26. She showed remarkable early improvement as donor cells engrafted. She continued to improve through November. Despite the improvement in blood counts, she could not survive the toll of her myelofibrosis, co-morbidities, the impact of many clinical trials, and the rigors of stem cell transplant. Voncille died a week before Christmas, 2016.
Her son Garrett’s posting is an eloquent tribute to our Peppe.
Endnote: Honoring Voncille’s request to protect the doctor she loved and respected, I sent her last emails to him at MD Anderson for permission to reveal his identity. Dr. Srdan Verstovsek responded immediately:
“Thank you Zhen, Peppe’s story needs to be told as it happened, using her words without a change. no worries from me about any negative ways or anything similar. she fought this war with myelofibrosis head on, for long time, with amazing family support, and I was just trying to help to the best of my abilities. No shame in that…. Peppe passing away was tragic loss, indeed. We are all very saddened with what had happened, since she was improving until two days before her death, so this was very unexpected to me.
“I agree with you. (Editor’s note: I had concluded my email to Dr. Verstovsek with a strong caveat against entering early stage clinical trial if other options are possible.)
“This is why she was first on established meds, revlimid, prednisone, pomalidomide, and only then on investigational meds (and did well for long time on PRM).”
Family comment… None of us can go through a stem cell transplant on our own. Beyond the transplant team we need personal support to help us navigate the many obstacles along the way. When we sent the draft if this tribute to the Peppe’s family for fact check and review we got back a note and a photo of Gary Comeaux with Peppe, a photo taken at the 2015 PatientPower meeting: “If it weren’t for him, she would have never made it this far. He was the one by her side through all these trips to Houston every month and her caretaker for the transplant. He stepped in where none of us were able to because of work and our families, and they were divorced all this time. Only last July right before the transplant did they remarry. “ — Brittani Comeaux, Peppe’s daughter-in-law
Standing in Voncille’s shoes.
We share with our doctors the predictable inability to stop the ravages of myelofibrosis with existing drugs. Our meds are designed to relieve symptoms, ease pain and in time the rampaging blood lines damage tissue and organs, deposit collagen fibers, swell our organs, stress our blood-bone niche and circulatory system beyond bearing. The greater danger is progression to end stage, blast phase, acute myeloid leukemia. And at that point a stem cell transplant is almost hopeless.
The absence of drugs to treat MPNs can only be filled by research and development, translation of science into therapy. And for that we require clinical trials. And there’s the rub. Trials are always long and frequently exhausting and occasionally toxic or deadly.
So long as drug makers are simply looking for a piece of Incyte’s JAK2 inhibitor action, the risk of clinical trial far outweighs any possible gain. And for those Phase I/II dosage and toxicity trials of highly experimental compounds the risk of weakening our already compromised immune system is far greater than any possible gain, Miracles are few and far between.
So what do we do when we stand in Voncille’s shoes? We depend on our doctor to help us sort through our best options. Sometimes there are simply no good options. When Medicare shut the door on Peppe’s transplant in 2007 it took courageous efforts by her, her family and her medical team to sustain her life.That Medicare denial was a severe blow but out of it emerged a sustaining relationship with a dedicated, skilled hematologist. Together they pushed the boundaries of available medical technology to greatly extend her life.
If we were in Voncille’s shoes would we not rather take a pill or needle in a promising clinical trial and postpone the radiation, diarrhea, nausea, chemo, isolation and recovery associated with SCT? Of course, but As Voncille has tried so hard to tell us, there is a price to pay in lost time and stress on body and soul.
Our participation in a Phase I/II dosage and toxicity trial must be our very last option, the Hail Mary pass as time has almost run out. When existing meds fail, our choices usually boil down to clinical trial of a promising drug or stem cell transplant. Helping us selecting one of those trials needs all the skill and experience of an MPN specialist.
But if the stars are aligned, if we are young and strong enough and a qualified stem cell donor is available. If we have an experienced transplant team in place and available insurance, social and economic resources, our chances to emerge at the other end fully cured, free of MF and suffering only minimal GVHD are excellent.
For a review of our Clinical Trial versus Stem Cell Transplant options, here’s The Choice: Clinical trial vs. Stem Cell Transplant that concludes with a comment by Voncille.
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Comments on: "Peppe – The fixer of everything" (14)
WOW, I am just seeing this. I knew her way back in the day when her and Carl where married. They lived next door to my 2nd mom on Emma street. That was in the late 70’s, early 80’s. I had no idea ………………:(
Zhen and Lindy Sutton, Thanks for your replies regarding Medicare coverage of SCT for myelofibrosis. My husband was NOT in a clinical trial for the stem cell transplant. (By the way, I mistyped his name which is Irv not Irw). So I looked up Medicare information on SCT coverage for myelofibrosis. It turns out it is not a clinical trial that is required but the facility must be willing to report outcome statistics to CMS.
Here is the relevant quote from the article found on Hematology.org dates November 12, 2015: (http://www.hematology.org/Advocacy/Policy-News/2015/4662.aspx
This proposed change would add those three conditions to covered status under the process known as coverage with evidence development. Coverage with evidence development is used by Medicare when the agency finds that the scientific evidence of a procedure’s efficacy is lacking in a particular population. Medicare agrees to pay for the service in exchange for those providing it gathering data, often using a registry. Coverage with evidence development is intended to be a temporary evidence-gathering step but some coverage with evidence development policies have been in place for many years.
I think this is an important distinction.
Thanks, Robin, an important clarification, The Coverage with Evidence Development paradigm is quoted in the final decision in 2016, linked below, but seemed to require a clinical trial setting for the CED. In any case, Irv’s direct experience should be able to guide our MF patients seeking Medicare coverage of a SCT. Much appreciated.
What a beautiful story for a beautiful lady rip peppe
My Heart is aching, My dear friend Peppe, What a wonderful tribute to you and gift you gave to all of us to share your story. I open FB every day and I miss your smiling face. We had many discussions about our journeys, we were scared together, gave each other faith to keep on trying and not to give up. You were a Champion to me. Loved all your stories about Gracie and then Gabe and they will have all your wonderful comments to know how much you loved them and the rest of your family. Thank you for always being there for us and I know you are looking down from heaven and telling us do not give up. Find a Cure. I too, am on a new trail. Momelotinib. I am happy, Oh yes I have good and bad days. Extreme tired, but no Transfusions for 1 year. Yeah. I am blessed. Yes I am sure it will not last forever but I have been given this time to enjoy my family. In beginning I thought 3-5 Years and now it has been 7. Very Blessed I Believe we will find a cure. I believe I walk with God, Thank You. Love you Peppe, we will keep trying.
I don’t know if Peppi’s story would have any impact on the Medicare claims department, but I feel her story should be sent to them. Why are some Bone Marrow Transplants covered by Medicare (Irw Dubinsky) and others are not. They should be aware of Peppi’s long wait for transplant resulted in her untimely demise. So sad….
You can certainly forward the story to CMS, Lindy. In October, 2015 the Center for Medicare and Medicaid services issued its final rule on HSCTs under which Peppe qualified. It’s a highly restricted approval requiring participation in an approved clinical trial.
CMS will cover items and services necessary for research under §1862(a)(1)(E) for allogeneic HSCT for certain Medicare beneficiaries with myelofibrosis (MF) using the Coverage with Evidence Development (CED) paradigm. We are finalizing the following decision:
Allogeneic HSCT for myelofibrosis will be covered by Medicare only for beneficiaries with Dynamic International Prognostic Scoring System (DIPSSplus) intermediate-2 or High primary or secondary MF and participating in an approved prospective clinical study. There must be appropriate statistical techniques in the analysis to control for selection bias and potential confounding by age, duration of diagnosis, disease classification, DIPSSplus score, comorbid conditions, type of preparative/conditioning regimen, graft vs. host disease (GVHD) prophylaxis, donor type and cell source.
A prospective clinical study seeking Medicare coverage of allogeneic HSCT for myelofibrosis pursuant to Coverage with Evidence Development (CED) must address the following question:
Prospectively, compared to patients who do not receive allogeneic HSCT, do Medicare beneficiaries with MF who receive allogeneic HSCT have improved outcomes as indicated by:
• Graft vs. host disease (acute and chronic);
• Other transplant-related adverse events;
• Overall survival; and
• Quality of life (optional).
Thanks Zhen I will see what I can do. It sure is complicated. Thanks again, Lindy
My husband, Irw Dubinsky was diagnosed with MF in 2010. Last fall 2016, at age 76, he had a SCT a few weeks before Peppe. He and Peppe were in very close contact throughout both of their procedures. We were so saddened by her loss.
The SCT was suggested by Dr. V when we were out of options. Dr. V said that functional status is a better parameter than age so they accepted him even though he was technically “too old”. So it appears age is no longer a barrier. We decided on Sloan Kettering in NYC for the SCT because it was closer to home and he could spend his 100 days in Hope Lodge, a residence for cancer patients getting treatment away from home. This made it affordable. One thing that confuses me is that Medicare paid for it. Does medicare coverage vary by state?
My husband had been very despondent that he was not eligible for any clinical trials. It turns out the lack of prior treatments allowed him to go into the SCT with no pre-existing organ damage. He basically sail ed through his recovery. We have been very lucky. We wish this could have been the case for Peppe as well.
Robin your story precisely sums up our dilemma as MPN patients. I am so happy your husband didn’t qualify for a clinical trial and was able to transcend barriers to a stem cell transplant thanks to the good offices of Dr. Verstovsek. I hope his health continues to improve and you keep in touch.
Yes, we need to grieve over losing Peppe. But we also need to get angry and resist the forces that would make victims and grieving survivors of us all; greed and government indifference.
WE are victimized by our own desire to survive and the predatory practices of the pharmaceutical industry. Encouraged by our instincts, drug company propaganda and often our own doctors, we are convinced that our salvation lies in a pill or needle when, in fact, no clinical trial of any drug has been fielded as curative.
MPN clinical trial drugs are at best palliative, designed to reduce symptoms (temporarily) and earn extraordinary profits for drug companies. With the single exception of one JAK inhibitor, virtually all have so far failed in even this limited objective… and, along the way these human experiments have injured, killed, or weakened MPN patients who have aged in the process.
Dr. Verstovsek who has as much clinical trial experience as any of our hematologists, heads a large MPN section at MD Anderson that is also dependent on drug dollars invested in clinical trial at his institution. He emerges as a hero in this tragic story because he has consistently demonstrated his solidarity with patient needs. He endorsed Peppe’s original bid for a stem cell transplant…and when that was denied he worked with every available tool to prolong her life.
In that dark era before gene therapy – an era that only now is beginning to come to an end – the only options were our limited meds and experimental drugs. Peppe didn’t die of her stem cell transplant. She died of organ failure having absorbed a decade of biochemical l stress in clinical trial.
We need a patient advocate at every stage of clinical trial.
I didn’t know Pepe or her story but her words give me hope that I might be around longer than I had anticipated. I would say she faced her fate with bravery and dignity and it’s unfortunate that the SCT did not come soon enough to let her spend more time with her grandchildren.
I was diagnosed with primary myelofibrosis in August of 2015 and had my spleen removed in June of 2016 (7lbs, 7oz) and was beginning to rupture. I know Pepe’s Dr avoided spleen removal but, I had no other option. I just hope I can hang on as long as she did and with the dignity that she exhibited.
Peppe was such a gallant soul. I followed her journey with MF over the same years that Joe had MF. Peppe was much younger than joe. I also believe that if in 2007 Peppe had been able to have a SCT that she would be here today. There is no way of knowing, Peppe did what most patients would do…..follow HOPE where there is none. Jakafi was a blessing to Joe in that it gave him a quality of life he desired. YEs, it took a toll on his kidneys along with the chemo for MCL. His immune system was shot. He had no regrets taking Jakafi and I know if he had to do it all over again, he would do it again. He was on Jakafi from 2008 to 2015 with many of the years he enjoyed life and seeing the world. I miss Peppe. I miss Joe. I miss way too many people that we lost. My heart has taken a toll each time we have to say goodbye to another fighter. Praying for a CURE.
Peppe was one of my courage-giving mentors at at time when we MPNs in Norway did not have anywhere else to turn, but to the Internet. I was deeply saddened to read her story, but also impressed by her courage and fighting spirit. Her story gives my own story some perspective, and I feel a very strong connection across the world between us MPN sufferers, we are good at finding pleasure in the really important things in life; such as family and friends that lift us up. Luckily Norway has a very good health care service, so I am blessed that way, but the MPN “curse” is not always fixed anyway. Thank you for this story, it helps me to hear about people like Peppe who are true fighters; her family shall know that she has been there for me too.
Peppe was such a brave soul. Of course I cried as I read her story. I feel fortunate to have reached some of the milestones Peppe missed. Since I was diagnosed at an older age, I have been fortunate to have enjoyed my children and grandchildren. I too considered trial drugs but was unable to try the first one, because my platelets went too high for the trial. So I live, and I wait, and I hope, as Peppe did. Thank you Peppe! I know you are at peace now, and are looking down on your grandchildren with love.