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Clinical end points...
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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Barosi, G (author)
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Tefferi, A (author)
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Besses, C (author)
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- Birgegård, Gunnar (author)
- Uppsala universitet,Hematologi
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Cervantes, F (author)
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Finazzi, G (author)
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Gisslinger, H (author)
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Griesshammer, M (author)
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Harrison, C (author)
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Hehlmann, R (author)
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Hermouet, S (author)
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Kiladjian, J-J (author)
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Kröger, N (author)
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Mesa, R (author)
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Mc Mullin, M F (author)
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Pardanani, A (author)
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Passamonti, F (author)
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- Samuelsson, J (author)
- Karolinska Institutet
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Vannucchi, A M (author)
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Reiter, A (author)
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Silver, R T (author)
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Verstovsek, S (author)
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Tognoni, G (author)
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Barbui, T (author)
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(creator_code:org_t)
- 2014-08-25
- 2015
- English.
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In: Leukemia. - : Springer Science and Business Media LLC. - 0887-6924 .- 1476-5551. ; 29:1, s. 20-26
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https://www.ncbi.nlm...
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Publication and Content Type
- ref (subject category)
- art (subject category)
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Leukemia
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To the university's database
- By the author/editor
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Barosi, G
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Tefferi, A
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Besses, C
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Birgegård, Gunna ...
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Cervantes, F
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Finazzi, G
-
show more...
-
Gisslinger, H
-
Griesshammer, M
-
Harrison, C
-
Hehlmann, R
-
Hermouet, S
-
Kiladjian, J-J
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Kröger, N
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Mesa, R
-
Mc Mullin, M F
-
Pardanani, A
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Passamonti, F
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Samuelsson, J
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Vannucchi, A M
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Reiter, A
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Silver, R T
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Verstovsek, S
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Tognoni, G
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Barbui, T
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Hematology
- Articles in the publication
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Leukemia
- By the university
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Uppsala University
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Karolinska Institutet