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- Gugliotta, L., et al.
(författare)
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Treatment Of Essential Thrombocythaemia In Europe : An Observational Study Of 3649 High-Risk Patients In Exels
- 2015
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Ingår i: Haematologica. - St Orsola Malpighi Hosp, Dept Hematol, L&A Seragnoli, Bologna, Italy. Univ Bari, Hematol Transplantat, Bari, Italy. Osped Maggiore della Carita, Novara, Italy. IRCCS Casa Sollievo Sofferenza, Div Ematol, San Giovanni Rotondo, FG, Italy. Arcispedale S Maria Nuova, Reggio Emilia, Italy. Hop St Louis, APHP, Ctr Invest Clin, Paris, France. Hosp del Mar IMIM, Dept Hematol, Barcelona, Spain. Johannes Wesling Med Ctr, Hematol & Oncol, Minden, Germany. Guys & St Thomas NHS Fdn Trust, Dept Haematol, London, England. Shire Pharmaceut, Global Biometr, Wayne, NJ USA. Shire Int GmbH, Res & Dev, Zug, Switzerland. Uppsala Univ, Dept Haematol, Uppsala, Sweden.. - 0390-6078 .- 1592-8721. ; 38, s. 216-216
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)
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4. |
- Barosi, G, et al.
(författare)
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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)
- 2015
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Ingår i: Leukemia. - : Springer Science and Business Media LLC. - 0887-6924 .- 1476-5551. ; 29:1, s. 20-26
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Tidskriftsartikel (refereegranskat)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.
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