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)
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 m...
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| Main Authors: | , , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
2015
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| In: |
Leukemia
Year: 2014, Volume: 29, Issue: 1, Pages: 20-26 |
| ISSN: | 1476-5551 |
| DOI: | 10.1038/leu.2014.250 |
| Online Access: | Verlag, Volltext: http://dx.doi.org/10.1038/leu.2014.250 Verlag, Volltext: http://www.nature.com.ezproxy.medma.uni-heidelberg.de/leu/journal/v29/n1/full/leu2014250a.html 
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| Author Notes: | 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 |
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| 520 | |a 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. | ||
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