Science & Medicine

On trial: Hydroxyurea versus Interferon

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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.

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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.

Dr. Ronald Hoffman, Mount Sinai, MPD-RC

Dr. Ronald Hoffman, Mount Sinai, MPD-RC

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.

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Why we need this trial.

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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.)

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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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Comments on: "On trial: Hydroxyurea versus Interferon" (6)

  1. Linda frank said:

    My Doctor has recommended pegasys, i would much prefer pill hydroxyurea. High price in pegasys verses affordable hydroxyurea. Can they both equally achieve the same results?

    • The evidence is both incomplete and compelling, Linda. This really is an issue to discuss at length with your doc after reading the many arguments raised in these pages.,;…. plus do look at the Graphic Archives (link on the MPNforum Home Page, bottom).
      What we think we know: Yes HU is much less expensive than INF; it generally works faster to calm blood counts, improve symptoms; Over the course of a year they are about equal in efficacy. Beyond that, in selective instances, interferon is superior in addressing the underlying disease and relieving its symptoms.
      Toxicity varies by individual — some can’t tolerate INT, some develop adverse reactions to HU — but short term both are considered safe. Claims for long term disease modification and durable responses for Interferon are many but not yet clinically accepted. Longer term toxicity of HU, cytoreductive, possibly leukemogenic, has not been established in the absence of triggering meds. (Many MPN patients have been on HU for decades without progression to more acute conditions.
      Again, Linda, an open, frank and informed discussion with your physician will incorporate your own, individual clinical status in the discussion… and may help in your decision. At an early enough stage, with your doc’s assistance, you can always try one, monitor the results, and decide on your options at that time. Good luck! Keep in touch.

      • Linda A frank said:

        Thank you for your response, i am getting ready to make a decision that i feel will suit my highly active lifesyle. I am a symptomatic at this time, other than periodic fatigue i feel perfectly healthy and do not want to become the sick girl because of long term drugs.

  2. I have had PV for 6 1/2 years. At diagnosis hematocrits were 77. Now platelets over 1.17 million and WBC is 20.2. My hematologist never told me about Pegasys interferon as an option.

  3. My husband is on this trial doing very well on peg interferon. Very lucky to have Dr. Hoffman as his doctor he is extremely knowledgable and caring.

  4. Medhat Aziz said:

    I took the both pegasys interferon 180 one shot every 3 weeks plus 1 hydroxyurea 500 day after day , so far the disease under control thanks God :)

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