An Opportunity to improve measurement of health related quality of life symptoms using MPN Patient reported outcomes

A Patient’s Perspective – Methods to Improve MPN Research by Collecting data and Blood test results from Patients

by David Wallace

As a PV patient, my treatment has run a wide spectrum during the last 6 years.  Starting with phlebotomy only, Hydroxyurea (short time frame), Pegasys (nearing 2 years) and now the “cutting edge combo” of Pegasys and Jakafi for the last 5 months.  I personally believe this combo may be the Best Available Therapy (BAT) for highly symptomatic PV patients who do not respond well to Pegasys alone, but that is not directly the point of this article….as always, be sure to consult an MPN Specialist and keep in mind these are the views of a patient.

23andme Research Study

23andMeWhen 23andme (self described as a saliva-based DNA service) asked for MPN patients to “volunteer their saliva” in hopes of helping research, I readily accepted, as did many of my friends online.  It would be a safe assumption that hundreds of MPN Facebook group members did as well.  In return, we received a detail genetic report showing us what diseases we had health risks for, drug response, inherited conditions, traits and a detailed ancestry overview.  Their goal was 1000 patients (over 1500 participated), seemingly a lofty goal given the presumed” rarity of our clonal abnormalities (MPNs).  Through active collaboration and fellow patients spreading the word on social media, that goal was achieved.  This is reflective of our tight knit community and speaks volumes regarding our eagerness to help out for the common good.

Would you be willing to share your Blood Tests and complete surveys to help MPN Research?

I know I would and my guess is many of you would be willing to help out as well, assuming the necessary systems were in place.

In my opinion, there is presently an “uncollected data gap” (opportunity) in the study of MPN patients undergoing various treatment regimes.  We need to know what treatments are working today (untapped information is out there – in our own lab results), how well, how long until we should expect results, what are the side effects, the pitfalls, dangers?….in addition to the continuing study of new treatments.

First of all, I should note that I am very fortunate to finally bond with a first class hematologist who knows his stuff, respects my opinion and works with me in harmony. If you follow my newsletters over the last 2 years, you may have read about some of my struggles trying to find a hematologist locally who really understood PV; the available treatment options….and me.  Now steering back to the point.

The reason I mentioned my treatment with Pegasys and now the combo, was the realization “where is my data going?”  No doubt, my diligent doctor tracks progress, perhaps shares results with colleagues and maybe one of the drug companies involved.  That is the way the system is setup today.  We don’t have the opportunity to volunteer and no system is in place to share our data for wider analysis and better evaluation.

No “formal clinical trial” is required to collect data from patients who are willing and able to share valuable information TODAY!  There are literally 100’s (maybe more) of patients taking Pegasys in the U.S. alone, yet there is little consensus among MPN experts as to the value (efficacy) of this treatment.  Outside of the pioneering efforts of Dr Richard Silver and a few renowned hematologists (in the U.S.), many MPN specialists are hesitant to recommend Pegasys, as clinical trials in the U.S. lag on at a snails pace.

The clinical trial at MD Anderson Cancer Center, NCT00452023, has dragged on for 10 years and the completion date is December 2017 (click on the image below or this link for details).  Do we really need 12 years to complete this study?  Dr Heinz Gisslinger in Vienna (along with European colleagues) have been successfully treating MPN patients with Interferon for 30 years!pegasys clinical trial mpn

Combination Therapy

As far as the combo (Pegasys and Jakafi), only a handful of patients in the U.S. are on this treatment, yet it may be the best available treatment we have today…..who knows?  There are patients begging for this treatment (I have been contacted by a few personally) as Pegasys alone is not quite delivering the desired results for some.

Many patients are waiting 2-3 years for Pegasys to become effective in reducing the need for phlebotomy (a highly desirable outcome in PV), reduce spleen size and normalize counts, while the addition of Jakafi may produce the same outcome in months.  So you have the potential for halting/slowing the progression of MPN’s with Pegasys, while Jakafi has proven effective in reducing symptoms in Polycythemia Vera and Myelofibrosis.

The combo therapy allowed me to significantly reduce Pegasys dosage and frequency….thereby reducing some of the nagging (putting it kindly) side effects.

Very few doctors would even consider prescribing this “cutting edge therapy” because of the lack of formal clinical data.  The truth is….it makes sense, appears to help, at least in the short term, but does require close monitoring (regular CBC’s) while tweaking doses of each drug.  The synergistic effect of the two medications pack a potent punch against PV/MF.  Shouldn’t data be gathered from patients taking these drugs if they are willing to participate?

A Potential Solution

blood test results mpnWhat if a leading University or Research Institution, with financial backing by an independent party were to band together and develop a similar internet based solution to gather patient data?  If patients readily climbed on board and shared their blood tests and symptom assessments, we would know just how well MPN treatments are working today….not 3-5 years from now when the clinical trials are complete.  The following report presents a novel method of gathering patient data using a web based tool.

Thanks to Nana Brochmann for contributing the Following Research Study.  Participants were recruited from the outpatient clinic at the Department of Haematology, Roskilde University Hospital, Denmark.  Click the following link to go to the PDF study or the Yumpu Reader below and allow full screen mode.

Please be sure to read the article below:

A new internet-based tool for reporting and analysing patient-reported outcomes and the feasibility of repeated data collection from patients with myeloproliferative neoplasms


About David Wallace

Founder of PV Reporter, a resource for Myeloproliferative Neoplasm (MPN) patients and caregivers. After being diagnosed with Polycythemia Vera (PV) in 2009, I utilized social media to connect with "informed patients" and develop a better understanding of emerging treatment options. My philosophy on patient care is straight forward - "educating the patient is essential, so the patient can guide their physician to meet his or her needs." PV Reporter is a comprehensive resource hub giving visitors vital tools to become "empowered patients."

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