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Which patients with myelofibrosis should receive ruxolitinib therapy? ELN-SIE evidence-based recommendations
M Marchetti1, G Barosi2, F Cervantes3
1Ospedale Cardinal Massaia, Asti, 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.
Insights
Ruxolitinib is strongly recommended for treating myelofibrosis symptoms and splenomegaly in specific patients. However, evidence for improving survival with ruxolitinib is weak, and it may not apply to transplant candidates.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Ruxolitinib, a JAK1/JAK2 inhibitor, is approved for myelofibrosis treatment.
- Regulatory agencies and scientific societies have provided discordant indications for its use.
- There is a need for evidence-based recommendations for selecting appropriate candidates for ruxolitinib therapy.
Purpose of the Study:
- To develop evidence-based recommendations for ruxolitinib use in myelofibrosis.
- To clarify optimal patient selection for ruxolitinib therapy based on clinical outcomes.
- To utilize the GRADE methodology for formulating recommendations.
Main Methods:
- Conducted a systematic review and evidence synthesis using the GRADE methodology.
- Defined 18 patient-intervention-comparator-outcome profiles.
- Focused on clinical outcomes including splenomegaly, disease-related symptoms, and survival.
Main Results:
- Strongly recommended ruxolitinib for symptomatic or severe splenomegaly in intermediate-2 or high-risk myelofibrosis (IPSS/d-IPSS).
- Strongly recommended ruxolitinib for systemic symptoms (MPN10 score >44, severe itching, weight loss, fever).
- Did not recommend ruxolitinib for improving survival due to weak evidence.
Conclusions:
- Ruxolitinib is recommended for symptom and splenomegaly management in specific myelofibrosis patients.
- Ruxolitinib is not recommended for survival improvement in myelofibrosis based on current evidence.
- Recommendations may not apply to patients eligible for allogeneic stem cell transplant.
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