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Details

Autor(en) / Beteiligte
Titel
A Double-Blind, Placebo-Controlled Trial of Ruxolitinib for Myelofibrosis
Ist Teil von
  • The New England journal of medicine, 2012-03, Vol.366 (9), p.799-807
Ort / Verlag
Waltham, MA: Massachusetts Medical Society
Erscheinungsjahr
2012
Link zum Volltext
Quelle
Free E-Journal (出版社公開部分のみ)
Beschreibungen/Notizen
  • This trial showed clinically significant responses in spleen size and quality of life among patients with myelofibrosis receiving ruxolitinib, a JAK1 and JAK2 inhibitor. The agent has some myelotoxicity, but this study showed a survival advantage with ruxolitinib. Myelofibrosis, a myeloproliferative neoplasm, is manifested by abnormal blood counts (anemia, thrombocytosis or thrombocytopenia, and leukocytosis or leukopenia), splenomegaly, and debilitating symptoms (e.g., fatigue, weakness, abdominal pain, cachexia, weight loss, pruritus, night sweats, and bone pain), which are thought to be caused by the combined effects of massive splenomegaly and elevated levels of proinflammatory cytokines. 1 Survival ranges from approximately 2 to 11 years, depending on defined prognostic factors. 2 Traditional therapeutic options, including splenectomy, have limited benefit. 3 Although allogeneic stem-cell transplantation may cure myelofibrosis, few patients are eligible for this treatment. Although the gain-of-function mutation in the gene encoding Janus kinase . . .

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