Is this a clinical trial for you?
Interferon and Hydroxyurea go head to head.
By far the most commonly prescribed workhorse MPN drugs for ET and PV are interferon and hydroxyurea (HU). Neither is approved for MPN use, setting the stage for contentious debate over relative effectiveness, presumed toxicities, and all the rest.
MPNforum has provided ample coverage of interferon (INT) in 2014, featuring the work of eminent MPN specialists. We provided little coverage of HU, mostly because it is such an old and heavily used medicine in MPN treatment that there seemed to be little to report beyond the occasional refutation of its alleged leukemogenicity.
We have also staked out an extreme position on clinical trial.
Following death and severe injuries during recent Phase III and Phase I/II trials of experimental new MPN drugs, we stepped up our resistance. Briefly, MPNforum’s position has been that clinical trials are by their nature experimental and MPN patients, whose health is already compromised, should generally avoid all Phase I/II dosage and toxicity trials and clinical trials of any kind unless clear, measurable benefit is offered.
To provide a more balanced view of our Interferon vs. Hydroxyurea options and the important role clinical trials play in MPN therapy, we turned to two world-renowned hematologists and investigators we know and respect. Both are scientists and clinicians heavily published in scientific journals, both have deep clinical MPN experience.
Here they present their views on why a head to head clinical trial of these two drugs is necessary… and why patients, with the support of their hematologists, should consider participating in clinical trials.
In their presentations, both Dr. Claire Harrison and Dr. Ronald Hoffman advance different arguments to reach the same conclusion: There are personal and altruistic reasons to consider participating in the Interferon vs. Hydroxyurea clinical trials, now open across 28 study centers.
A link to the clinical trial protocols that include contact information is provided at the conclusion. If you’re planning to participate it might be a good idea to download that document and bring it along as you discuss your treatment options with your hematologist.
MPN treatment at a crossroads….
– Dr. Ronald Hoffman
I believe that this field is currently at a crossroads as it relates to the therapy of individuals with PV and ET.
There are two drugs, hydroxyurea and interferon, that are widely used for treating these patients but at present we cannot distinguish which drug is superior.
We will only obtain this information by completing phase 3 randomized trials comparing interferon to hydroxyurea. The completion of such trials is entirely dependent upon the willingness of the patients themselves to participate.
Frequently patients refuse to participate because they have been told that one of these drugs is more effective than the other.The truth is that we have little information which indicates with an acceptable degree of certainty that one of these drugs is better than the other. Other experts in this field strongly disagree with such statements but their passionate and equally well-meaning pleas are based upon impressions not science.
As to the patient making the difficult decision as to whether to participate or not, it is important to emphasize that both drugs are effective treatment and if you participate and receive either of the drugs you will not be receiving inferior treatment. Most importantly your participation is voluntary and you can always withdraw from the study if you and your physician feels this is justified. Since each of these drugs have been used for several decades and few if any of the patients treated with either of these drugs have been cured, the randomization to either of the arms should not be considered “bad.”
The opinions of seasoned clinicians are of great value and should not be ignored but they do not replace the power of science. In medicine, science always wins and ultimately leads to better patient care. The sole reliance on opinions to make difficult clinical decisions will surely lead to confusion and anxiety on the part of the patients and scientific stagnation rather than progress.
These trials when completed will answer a very important question which will serve as a foundation to accelerate further scientific research in this field which will ultimately help all patients with PV and ET.
Why we need this trial.
Dr. Claire Harrison
(This short video has been extracted from Dr. Claire Harrison’s longer discussion of MPN drugs at ASH-San Francisco last month. That full video is available here.)
Clinical Trial NCT01259856: Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET)
Recruiting participants is another Phase III head-to-head comparison of the two drugs. Clinical trial NCT01949805, is the Dr. Heinz Gisslinger study of AOP2014 (Pegylated interferon) vs hydroxyurea in polycythemia vera, (PROUD-PV). Details of this European-based trial, open in 58 study locations, can be found here.
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