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Leukemia. 2017 Apr;31(4):882-888. doi: 10.1038/leu.2016.283. Epub 2016 Oct 14.

Which patients with myelofibrosis should receive ruxolitinib therapy? ELN-SIE evidence-based recommendations.

Author information

1
Ospedale Cardinal Massaia, Asti, Italy.
2
IRCCS Policlinico San Matteo Foundation, Pavia, Italy.
3
Hospital Clinic, IDIBAPS, Barcelona, Spain.
4
Uppsala University Hospital, Uppsala, Sweden.
5
Johannes Wesling Klinikum Minden, Minden, Germany.
6
Guy's and St Thomas' NHS Foundation, London, UK.
7
Medizinische Fakultät Mannheim, Universität Heidelberg, Mannheim, Germany.
8
Centre d'Investigations Cliniques - CIC 1427, Paris, France.
9
University Hospital Hamburg-Eppendorf, Hamburg, Germany.
10
Queen's University Belfast, Belfast, UK.
11
Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
12
Ospedale di Circolo e Fondazione Macchi, Varese, Italy.
13
Research Foundation, Ospedale Papa Giovanni XXIII, Bergamo, Italy.

Abstract

Ruxolitinib is an oral Janus-activated kinase 1 (JAK1)/JAK2 inhibitor approved for the treatment of patients with myelofibrosis based on the results of two randomized clinical trials. However, discordant indications were provided by regulatory agencies and scientific societies for selecting the most appropriate candidates to this drug. The European LeukemiaNet and the Italian Society of Hematology shared the aim of building evidence-based recommendations for the use of ruxolitinib according to the GRADE methodology. Eighteen patient-intervention-comparator-outcome profiles were listed, each of them comparing ruxolitinib to other therapies with the aim of improving one of the three clinical outcomes: (a) splenomegaly, (b) disease-related symptoms, and (c) survival. Ruxolitinib was strongly recommended for improving symptomatic or severe (>15 cm below the costal margin) splenomegaly in patients with an International Prognostic Scoring System (IPSS)/dynamic IPSS risk intermediate 2 or high. Ruxolitinib was also strongly recommended for improving systemic symptoms in patients with an MPN10 score >44, refractory severe itching, unintended weight loss not attributable to other causes or unexplained fever. Because of weak evidence, the panel does not recommend ruxolitinib therapy for improving survival. Also, the recommendations given above do not necessarily apply to patients who are candidates for allogeneic stem cell transplant.

PMID:
27740634
DOI:
10.1038/leu.2016.283
[Indexed for MEDLINE]

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