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Clinical endpoints for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms: consensus statements from European LeukemiaNET (ELN) and Internation Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).

Barosi G, Tefferi A, Besses C, Birgegard G, Cervantes F, Finazzi G, Gisslinger H, Griesshammer M, Harrison C, Hehlmann R, Hermouet S, Kiladjian JJ, Kröger N, Mesa R, Mc Mullin MF, Pardanani A, Passamonti F, Samuelsson J, Vannucchi AM, Reiter A, et. al.

Leukemia. 2014 Aug 25


The discovery of somatic mutations, primarily JAK2V617F and CALR, in classic BCR-ABL1-negative myeloproliferative neoplasms (MPNs), has generated interest in the development of molecularly targeted therapies, whose accurate assessment requires a standardized framework. A working group, comprised of members from European LeukemiaNet (ELN) and International Working Group for MPN Research and Treatment (IWG-MRT), prepared consensus-based recommendations regarding trial design, patient selection, and definition of relevant endpoints. Accordingly, a response able to capture the long-term effect of the drug should be selected as the endpoint of phase II trials aimed at developing new drugs for MPNs. A time-to-event, such as overall survival, or progression-free survival or both, as co-primary endpoints, should measure efficacy in phase III studies. New drugs should be tested for preventing disease progression in MF patients with early disease in randomized studies, and a time to event, like progression-free or event-free survival should be the primary endpoint. Phase III trials aimed at preventing vascular events in PV and ET should be based on a selection of the target population based on new prognostic factors, including JAK2 mutation. In conclusion, we recommended a format for clinical trials in MPNs which facilitates communication between academic investigators, regulatory agencies and drug companies.Leukemia accepted article preview online, 25 August 2014 doi:10.1038/leu.2014.250.


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