Clinical end points for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms

• Verfasst von: Barosi, Giovanni [VerfasserIn]
• Verfasst von: Hehlmann, Rüdiger [VerfasserIn]
• Verfasst von: Reiter, Andreas [VerfasserIn]
• Titel: Clinical end points for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms
• Titelzusatz: consensus statements from European LeukemiaNET (ELN) and Internation Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)
• Verf.angabe: 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. Reiter, R. T. Silver, S. Verstovsek, G. Tognoni and T. Barbui
• Jahr: 2015
• Jahr des Originals: 2014
• Umfang: 7 S.
• Fussnoten: Published online: 19 September 2014 ; Gesehen am 27.06.2017
• Titel Quelle: Enthalten in: Leukemia
• Ort Quelle: London : Springer Nature, 1997
• Jahr Quelle: 2015
• Band/Heft Quelle: 29(2015), 1, Seite 20-26
• ISSN Quelle: 1476-5551
• DOI: doi:10.1038/leu.2014.250
• Datenträger: Online-Ressource
• Sprache: eng
• K10plus-PPN: 1560238275

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.