The Critical Importance of Clinical Trials

June 4, 2014

Haematologica recently published a thought-provoking article by Drs. John Mascarenhas and Ronald Hoffman of Mt. Sinai, Dr. Ruben Mesa of the Mayo Clinic, Scottsdale, and Dr. Josef Prchal of the University of Utah, (Optimal therapy for polycythemia vera and essential thrombocythemia can only be determined by the completion of randomized clinical trials) which gives a history of treatments for essential thrombocythemia and polycythemia vera, and also makes the case that there is insufficient scientifically valid evidence on the treatment options currently available. The article provides an overview of the data from studies on Pegasys, Hydroxyurea, phlebotomy and other treatments dating back 40 years.

Dr. Mascerenhas points to the availability of Pegasys to patients - which the Foundation has helped facilitate - as a reason some of the existing randomized trials have not hit their recruitment goals. We agree with Dr. Mascerenhas' point that the only way to truly know how Pegasys measures up against Hydroxuyrea and other therapies is to complete a randomized clinical trial. At the same time, we will continue to assist patients any way we can while we fund research that could deliver more and better treatment options.

To find more about the ongoing randomized trial for Pegasys in ET and PV click here, or visit our complete list of current MPN clinical trials.

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