Study Demonstrates Survival Benefit In Patients With MDS

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Myelodysplastic Syndromes

Patients with myelodysplastic syndromes should find great hope in the results of a recently completed, large, randomized Phase 3 study of azacitidine (Vidaza - a demethylating drug) versus conventional care.

This study demonstrated a two-year survival of 50.8% vs. 26.2% for Vidaza and conventional care, respectively. Patients receiving Vidaza lived 9.4 months longer (24.4 months vs. 15 months for Vidaza and conventional care, respectively) than patients who received conventional care. This is the first time that any drug has been shown to lengthen the lives of MDS patients.

The patients who were enrolled in this study were predominantly 'higher- risk' MDS patients, i.e., those more likely to develop acute myeloid leukemia and whose survival is normally estimated in weeks to months rather than years. These patients either received Vidaza plus best supportive care or low-dose cytarabine plus best supportive care or standard chemotherapy plus best supportive care.

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Dr. Alan List, Chief, Malignant Hematology Division and Deputy Physician in Chief at the H. Lee Moffitt Cancer Center and Research Institute in Tampa, Florida said that, "With these very exciting results for Vidaza, survival should now be the standard by which we evaluate treatment options for higher- risk MDS and importantly, this is the first and only epigenetic therapy to demonstrate a survival benefit in any cancer. These findings should accelerate exploration of Vidaza in other malignancies where hypermethylation is believed to play a key role in tumor development and progression."

This study is a multi-center (US and ex-US), randomized, open-label, parallel-group, Phase 3 trial of subcutaneous Vidaza given at a dose of 75mg/m2 daily for seven consecutive days every 28 days plus best supportive care versus conventional care plus best supportive care. The treating physician was asked to select one of three 'conventional care' regimens as patient need dictated: best supportive care alone, low-dose cytarabine plus best supportive care or standard chemotherapy plus best supportive care. Erythropoietin stimulating agents (ESAs or EPO) and G-CSF use was not allowed under this protocol. 358 higher-risk MDS patients were enrolled at sites within the United States, Australia, and Europe. Patients were stratified by French-American-British (FAB) Classification subtypes (either RAEB or RAEB-T) and by International Prognostic Scoring System subgroups of Intermediate-2 or High risk. The conventional care regimen was selected for the individual patient prior to randomization on a 1:1 basis. The primary end point of the trial was to demonstrate a survival benefit with Vidaza plus best supportive care. Secondary assessments included transfusion independence, hematologic status, hematologic response and improvement, improvement of number of infections requiring IV antibiotics, time to relapse after a complete response (CR) or a partial response (PR), time to disease progression, time to transformation to AML, time to transformation or death from any cause, safety, toxicity, and pharmacoeconomics.

Until May of 2004 when Vidaza was approved for the treatment of MDS, followed by Revlimid (lenalidomide, an immunomodulatory drug) in December 2005 and Dacogen (decitabine, another demethylating agent) in May 2006, MDS patients had little hope of effective treatment. More progress has been made in the past 5 years than in the 30 years since MDS was differentiated from the acute leukemias.

Kathy Heptinstall, Operating Director, the MDS Foundation, Inc., said, "This study provides great hope to MDS patients and their families. The progress in treatment of MDS is phenomenal. Fifteen years ago, when the Foundation began to work with MDS patients, we had little to tell them regarding effective treatment. Now we can offer them hope to not only improve their quality of life but the possibility to extend their lives with effective treatment. It is a giant step on the "Journey to Hope" that is shared by patients, their families and the healthcare providers who treat them."

The MDS Foundation is a multi-disciplinary organization devoted to assisting patients with MDS, providing education for physicians, and support for research activities worldwide that will provide impetus for the development of new treatments for MDS.

The MDS Foundation is a publicly supported organization, exempt from Federal Income Tax under Section 501 (C)(3) of the I.R.S. code. Our US office is located at 36 Front Street, P.O. Box 353, Crosswicks, NJ 08515 and our EU office is located at King's College in London, England (UK). Please do not hesitate to contact the Foundation at 800-MDS-0839 (within the US) or outside the US at 609-298-1035 if you would like additional information or if you have any questions regarding the Foundation and our programs.

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