Methodological challenges in the development of endpoints for myelofibrosis clinical trials
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Methodological challenges in the development of endpoints for myelofibrosis clinical trials. / Barosi, Giovanni; Tefferi, Ayalew; Gangat, Naseema; Szuber, Natasha; Rambaldi, Alessandro; Odenike, Olatoyosi; Kröger, Nicolaus; Gagelmann, Nico; Talpaz, Moshe; Kantarjian, Hagop; Gale, Robert Peter.
in: LANCET HAEMATOL, Jahrgang 11, Nr. 5, 05.2024, S. e383-e389.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Review › Forschung
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TY - JOUR
T1 - Methodological challenges in the development of endpoints for myelofibrosis clinical trials
AU - Barosi, Giovanni
AU - Tefferi, Ayalew
AU - Gangat, Naseema
AU - Szuber, Natasha
AU - Rambaldi, Alessandro
AU - Odenike, Olatoyosi
AU - Kröger, Nicolaus
AU - Gagelmann, Nico
AU - Talpaz, Moshe
AU - Kantarjian, Hagop
AU - Gale, Robert Peter
N1 - Copyright © 2024 Elsevier Ltd. All rights reserved.
PY - 2024/5
Y1 - 2024/5
N2 - Myelofibrosis is a myeloid neoplasm characterised by the presence of JAK2, CALR, or MPL mutations (with a 90% mutation frequency) and trilineage myeloid proliferation with prominent megakaryocyte atypia. People with myelofibrosis have a lower survival rate and poorer quality of life than healthy individuals. Therapy for myelofibrosis uses Janus kinase inhibitors, which reduce splenomegaly and alleviate symptoms. Regulatory approvals for Janus kinase inhibitors have focused on this dual endpoint. In this Viewpoint, we discuss the validity of using spleen reduction as a surrogate endpoint for the disease-modifying activity of candidate drugs for myelofibrosis. We suggest alternative endpoints addressing unmet patient needs, including progression-free survival and overall survival. Moreover, we highlight the importance of selecting a core set of crucial outcomes with which we can individualise clinical decision making and standardise reporting of clinical trials results. We propose selecting patient-reported outcomes and anaemia response. We also suggest integrating economic considerations in the process of evaluating new drugs for myelofibrosis.
AB - Myelofibrosis is a myeloid neoplasm characterised by the presence of JAK2, CALR, or MPL mutations (with a 90% mutation frequency) and trilineage myeloid proliferation with prominent megakaryocyte atypia. People with myelofibrosis have a lower survival rate and poorer quality of life than healthy individuals. Therapy for myelofibrosis uses Janus kinase inhibitors, which reduce splenomegaly and alleviate symptoms. Regulatory approvals for Janus kinase inhibitors have focused on this dual endpoint. In this Viewpoint, we discuss the validity of using spleen reduction as a surrogate endpoint for the disease-modifying activity of candidate drugs for myelofibrosis. We suggest alternative endpoints addressing unmet patient needs, including progression-free survival and overall survival. Moreover, we highlight the importance of selecting a core set of crucial outcomes with which we can individualise clinical decision making and standardise reporting of clinical trials results. We propose selecting patient-reported outcomes and anaemia response. We also suggest integrating economic considerations in the process of evaluating new drugs for myelofibrosis.
U2 - 10.1016/S2352-3026(24)00067-X
DO - 10.1016/S2352-3026(24)00067-X
M3 - SCORING: Review article
C2 - 38604205
VL - 11
SP - e383-e389
JO - LANCET HAEMATOL
JF - LANCET HAEMATOL
SN - 2352-3026
IS - 5
ER -
