Costs of MPN Clinical Trials May Hinder Enrollment

While clinical trials are paving the way for new treatments to treat hematologic malignancies, but many patients are apprehensive to participate in them after considering the costs.
Clinical trials are leading the way in developing new treatments for hematologic malignancies, but the cost associated with them may be holding many patients back from participating, according to a recent international study that was published in the European Journal of Hematology.

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FDA grants marketing authorization to genomic sequencing kit for suspected PV

The FDA granted marketing authorization to ipsogen JAK2 RGQ PCR Kit for the detection of mutations affecting the JAK2 gene, according to the device’s manufacturer.

The ipsogen JAK2 RGQ PCR Kit (Qiagen) is a qualitative in vitro diagnostic test for real-time polymerase chain reaction on the Rotor-Gene Q MDx instrument (Qiagen) to detect the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood.

The kit is the first FDA–authorized test intended to help physicians evaluate patients for suspected polycythemia vera. Harboring JAK2 mutations is one of the major criteria for a clinical confirmation of polycythemia vera, with the V617F/G1849T allele present in more than 94% of patients.

Marketing authorization was based on a clinical study of the kit used in 216 individuals with suspected polycythemia vera compared with results obtained with Sanger sequencing.

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Geron survives but Jury at J&J is still on the future of imetelstat

Geron has survived another scrape with disaster, but it’s still operating under a dark cloud.

This morning the biotech announced that J&J’s review of the data from two studies of its drug imetelstat warranted continued work in myelodysplastic syndromes and myelofibrosis. But the pharma giant $JNJ is still reserving the right to quit if the data don’t hold up later in the year.

That’s not a big vote of confidence.

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A Patient’s Story: My Clinical Trial Experiences

 At what point in your MPN diagnosis did you join a clinical trial?  What trial (drug) were you in?

I was diagnosed with PV in 2001.  Only on phlebotomy and 81mg aspirin until 2007 when I began hydroxyurea because I turned 60 in 2006 and was now “high risk.”  In 2009 my spleen was enlarging. I had been hearing that Interferon was best taken in the early stage of MF and might reduce myelofibrosis.  My hematologist advised me he would be having a trial with Pegasys (Interferon) and I should wait for that. I actually waited until 2012 when the trial became available.  I was now showing signs through bone marrow biopsies of secondary MF.
Did you feel as though you received accurate information getting into the trial?

Yes, I had been reading about Interferon treatment and knew I wanted to try it.  Perhaps I didn’t know how quickly I could be removed from the trial because of my worsening symptoms.
What didn’t you know going into the trial that would be useful for others to know or questions they might ask?

It would have helped to know at what point you may be pulled from the trial and what happens after you are removed.  Also, I did not know that the FDA can shut a trial down at any point due to adverse reactions to few patients.

How many clinical trials have you participated in?

I am currently on my 3rd clinical trial.  (First trial wasn’t improving my symptoms, second trial was shut down by the FDA and the pharma company decided not to proceed , and the third trial I have begun my 4th year.)

Were you able to get the drugs after the trial(s) if you were doing well?

I can continue with the drug I am currently on as long as I continue to have benefit and as long as there is funding for this drug by the pharma company, and that it doesn’t get shut down prior to FDA approval

What advice would you impart to those who are skeptical about clinical trials who are not doing well on available treatments?

What do you have to lose?  If you are currently not well, you certainly may have good results and feel better.  If you don’t show benefit you will be removed to try something else.  We do not know the future after taking these drugs.  That is a gamble we must take.

Would you participate in other trials knowing what you know now?  

If I was not feeling well and I met the qualifications, I would try again.

Marylin C. has been living with her diagnosis for many years.  She has graciously volunteered for trials not only for her own reasons, but for the sake of MPN patients and their futures.

 

Thrombopoietin Measurement as a Key Component in the Evaluation of Pediatric Thrombocytosis

Abstract

JAK2, MPL, and CALR mutations, which underlie essential thrombocythemia (ET) in most adults, are infrequent in children. Consequently, additional tests are needed to confirm pediatric ET diagnoses. We report a child with suspected ET and normal JAK2, MPL, and CALR analyses. Serum thrombopoietin (TPO) was markedly elevated, leading to analysis of the TPO gene, TPHO, which contains an upstream open reading frame (uORF) known to repress THPO translation. Sequencing revealed a de novo, germline stopgain mutation in the uORF, explaining the elevated TPO and thrombocytosis. This finding suggests that screening TPO levels and, if elevated, THPO 5′ UTR sequencing could be diagnostic.

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NCCN May Already Be Updating MPN Guidelines

After publishing them in October 2016,the National Comprehensive Cancer Network (NCCN) is already seeking to update and expand their guidelines for diagnosing and treating patients with myeloproliferative neoplasms (MPNs).
The guidelines focused initially on diagnosing each of the MPNs and treatment options for patients with myelofibrosis, as that was the disease type with the greatest unmet need, Ruben A. Mesa, M.D., explained in an interview with CURE following his presentation at the 2017 NCCN Annual Conference.

BLOG-Springtime: Season for Renewal and Growth

flowersSigns of springtime are visible everywhere as daylight lengthens, plants bud and bloom, and more people are out and about.

Living with a chronic illness, it is easy to forget that some experiences are not symptoms of the serious; rather they are part of life for most people. I recall one visit to my hematologist because I was convinced my PV triggeblog springred a serious, unusual inflammation response. My eyes, nose, and skin were so itchy my husband teased that I had fleas. The normal beige bags under my eyes looked more like pink suitcases because I rubbed them so much.

I explained to my doctor the itchiness seemed to build up slowly over a week and then just exploded over the weekend. (This was well documented in my symptom journal). I took Benadryl so I could sleep without scratching myself raw. When she asked about my activities, I recalled planting flowers in my front yard on Saturday.

BINGO!! The pollen count in our area was over 1,500 that entire time (extraordinarily high) and my symptoms were consistent with seasonal allergies. No fever and my CBC (complete blood count) results were stable. She referred me to an allergist.

Well didn’t I feel silly. I’ve had seasonal allergies for years, but never this extreme. The allergist said that it is possible to gain new allergies and there were a couple of trees and grasses that were particularly strong that season. He changed up my antihistamines (H1 and H2 blockers), instructed me to wear a mask when outdoors to reduce inhaling the pollens, and to change out of my “street clothes,” shower, and wear clean indoor-only clothing when inside.

Morals of the story:

  1. Sometimes an allergy is just an allergy.
  2. We are not immune from everyday health issues.
  3. Every health concern does not automatically blossom into an MPN-related condition.
  4. Pollen, like those awkward middle school years, are part of the circle of life. The discomfort is temporary and the growth is worth it.
  5. Take time to smell the roses anyway.