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)
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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). / 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; Reiter, A; Silver, R T; Verstovsek, S; Tognoni, G; Barbui, T.
In: LEUKEMIA, 25.08.2014.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - 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)
AU - Barosi, G
AU - Tefferi, A
AU - Besses, C
AU - Birgegard, G
AU - Cervantes, F
AU - Finazzi, G
AU - Gisslinger, H
AU - Griesshammer, M
AU - Harrison, C
AU - Hehlmann, R
AU - Hermouet, S
AU - Kiladjian, J-J
AU - Kröger, N
AU - Mesa, R
AU - Mc Mullin, M F
AU - Pardanani, A
AU - Passamonti, F
AU - Samuelsson, J
AU - Vannucchi, A M
AU - Reiter, A
AU - Silver, R T
AU - Verstovsek, S
AU - Tognoni, G
AU - Barbui, T
PY - 2014/8/25
Y1 - 2014/8/25
N2 - 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.Leukemia advance online publication, 19 September 2014; doi:10.1038/leu.2014.250.
AB - 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.Leukemia advance online publication, 19 September 2014; doi:10.1038/leu.2014.250.
U2 - 10.1038/leu.2014.250
DO - 10.1038/leu.2014.250
M3 - SCORING: Journal article
C2 - 25151955
JO - LEUKEMIA
JF - LEUKEMIA
SN - 0887-6924
ER -