Clinical end points 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)

G. Barosi, A. Tefferi, C. Besses, G. Birgegard, F. Cervantes, G. Finazzi, H. Gisslinger, M. Griesshammer, C. Harrison, R. Hehlmann, S. Hermouet, J. J. Kiladjian, N. Kröger, R. Mesa, M. F. Mc Mullin, A. Pardanani, F. Passamonti, J. Samuelsson, A. M. Vannucchi, A. ReiterR. T. Silver, S. Verstovsek, G. Tognoni, T. Barbui

Research output: Contribution to journalReview article

27 Scopus citations

Abstract

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 end points. Accordingly, a response able to capture the long-term effect of the drug should be selected as the end point 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 end points, should measure efficacy in phase III studies. New drugs should be tested for preventing disease progression in myelofibrosis patients with early disease in randomized studies, and a time to event, such as progression-free or event-free survival should be the primary end point. Phase III trials aimed at preventing vascular events in polycythemia vera and essential thrombocythemia 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 that facilitates communication between academic investigators, regulatory agencies and drug companies.

Original languageEnglish (US)
Pages (from-to)20-26
Number of pages7
JournalLeukemia
Volume29
Issue number1
DOIs
StatePublished - Jan 10 2015

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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    Barosi, G., Tefferi, A., Besses, C., Birgegard, G., Cervantes, F., Finazzi, G., Gisslinger, H., Griesshammer, M., Harrison, C., Hehlmann, R., Hermouet, S., Kiladjian, J. J., Kröger, N., Mesa, R., Mc Mullin, M. F., Pardanani, A., Passamonti, F., Samuelsson, J., Vannucchi, A. M., ... Barbui, T. (2015). Clinical end points 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). Leukemia, 29(1), 20-26. https://doi.org/10.1038/leu.2014.250