An early Christmas
A little more than three years ago,
Howard Lewis – retired IBM executive and active North Carolina business consultant – backed into the murky MPN world. He felt an abdominal swelling, feared a tumor and went in for a physical exam. He ended the day with a diagnosis of chronic Myeloid leukemia…later corrected to polycythemia vera/
After suffering fatigue, splenomegaly, disturbed sleep, weight loss and all the rest, he was able to move over to Jakafi after last year’s FDA approval of that JAK inhibitor. It worked for a time but drove his platelets so low he was soon back at his doctor’s office to explore alternatives. A momelotimib trial was one possibility except by then his platelets were below protocol levels. That’s when his wife Sharon following up discussions on the Myelofibrosis Support Facebook group happened on pacritinib. With a little digging into www.clinicaltrials.gov she came up with the trial description and trial sites. They contacted the investigator at the D.C. center and were enrolled in the study
Problem was Howard pulled the short straw and was consigned to the Best Available Therapy (BAT) arm of the trial. If his spleen increased by 25% during the trial he could cross over to the pacritinib side …otherwise he would have to wait 24 weeks. Meantime his BAT drug, low dose Jakafi, helped maintain his counts while his spleen swelled.
December 21, an early Christmas. That was the day he crossed over to pacritinib and almost instantly improved. By week four his spleen had measurably reduced, platelets increased, he had gained weight andwas feeling much better.
”It was unbelievable,” he recalled. I felt better every day. “In terms of the electronic diary patients on trial keep, on a scale of 10 I went from a miserable 9 down to a reasonable 4 when the trial suddenly halted.” 
The date was February 4. After waiting six month to get on pacritinib Howard got to enjoy the benefits of the drug for only a few weeks before the FDA yanked it to review data.
Looking at his options now that his spleen is once again swelling and his symptoms have returned, he and Sharon are looking at a possible stem cell transplant or a new trial. “Because we were doing so well, our doctor said he would apply for an exception,” he said.
“Howard wasn’t “officially” informed that he needed to stop pacritinib,” said Sharon,” until February 10… in plenty of time to ruin Valentines Day.”
“I would go back on the drug if it becomes available,” said Howard.
Coming off has been rough
Jack Miller started the pacritinib trial about 10 months ago at the Cleveland Clinic. A father of two, “married to his childhood sweetheart,” Jack is in his early forties and, beyond his myelofibrosis, in comparatively good health.
He had been working at the city’s Convention and Visitor’s Bureau and studying to become a radiation therapist. His advancing myelofibrosis led him to revise his plans. He had to wait six months before he could cross over from the comparator Best Available Therapy (BAT) arm of the trial and start taking pacritinib.
He stopped taking Jakafi two week before entering the trial as required by the protocol. Randomly selected for the BAT arm he began taking Jakafi again after about a month and continued until he crossed over.
He was on pacritinib for three months and had good response. His only side effects, beyond some gastro-intestinal response in the beginning was petechiae which resolved as soon as he went off pacritinib.
The impact of being suddenly taken pacritinib off due to the FDA clinical hold “has been rough, fatigue, spleen building up. Once I stopped taking the drug it was readily apparent how well it had been working because my symptoms rebounded so strongly.”
He and his regular hematologist are now assessing treatment options.
On the trial for 26 weeks
Michael Spiegel, a 70 year old electrical engineer, married, with two grand-daughters, was doing terribly. His spleen would balloon up and he felt intense fatigue. 
He tried for a stem cell transplant but couldn’t find a perfect match and was then, in any case, turned down for the procedure by Medicare. He was on Danazol when his physician Dr. Moshe Talpaz at the University of Michigan, found the pacritinib trial.
“I was on the trial from July 2015. My doctor had to adjust the dosage (from 4 pills down to 3) to achieve the best result but my spleen rapidly shrunk from 21cm to 15, I felt much more energetic My counts were low but steady and I was very thankful that I could stay on Pacritinib for two or three more years till something new and better will be available.
“So the cancellation of the trial was a shock. Tomorrow I will see my doctor and I hope he will share my concern
“I feel so bad since getting off pacritinib, These pills are like poison. My body got used to taking poison every day and did well on it. Now I feel so tired, no energy at all. I lost 12 pounds in the last eight days.”
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Pain levels through the roof
Diagnosed with MF last year after suddenly collapsing, Bob Wanamaker’s Cleveland Clinic doctor recommended he start the pacritinib trial.
“I’ve been on pacritinib since September 9, ” said Bob Wanamaker, a married, 54 year old chief technology officer. “At first I thought it wasn’t working at all. My spleen didn’t shrink, I still had night sweats, still transfusion dependent. I did notice the time between transfusions got longer. I think I’m just a slow responder…
“Once I stopped pacritinib though my pain levels went through the roof. Fatigue has been intense. My spleen has gotten harder.”
While trying to sort out his next therapeutic options, limited because of his low platelet levels, Bob is using opioids and Tylenol to help control the pain.
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