Blood Res.  2021 Dec;56(4):322-331. 10.5045/br.2021.2021101.

Real-life ruxolitinib experience in intermediate-risk myelofibrosis

• Arikan F
• Toptas T
• Atagunduz IK
• Ercan T
• Oruc O
• Yilmaz F
• Tuglular T

• KMID: 2524092
• DOI: http://doi.org/10.5045/br.2021.2021101

Abstract

Background
In this retrospective cohort of patients with primary, post-polycythemia vera, or post-essential thrombocythemia myelofibrosis, 57 patients with MF who received ruxolitinib for MF-related symptoms or symptomatic splenomegaly were evaluated.
Methods
The median age of the patients in this cohort was approximately 58 years. Of these, there were 33 patients (57.9%) in INT-1, 23 patients (40.4%) in INT-2, and 1 patient (1.8%) at high risk. Overall, spleen size reduction of at least 35% (spleen response) was achieved in 56.6% and 63.3% of all cohort and INT-1 risk at any time, respectively.
Results
Symptom response and clinical improvement were observed in 21.7% and 60.7% of patients, respectively. Anemia and thrombocytopenia were prevalent, but manageable. About 73.7% of patients continued treatment during a median follow-up of 22 months. Two-year OS probability was approximately 84.5% (95% CI, 63.1‒94.0%) and 62.3% (95% CI, 37.5‒79.6%) for the intermediate-1 and -2 risk groups, respectively.
Conclusion
Real-life experience in a community-based hospital confirms the efficacy and safety profile of ruxolitinib in intermediate-risk myelofibrosis. Treatment discontinuation rates were lower than those in clinical trials.

Keyword

Primary myelofibrosis; Ruxolitinib; Spleen response

Figure

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  Fig. 1 Spleen response at 6 months of treatment in the cohort (A). Spleen response at any time during the follow up in the cohort (B).

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  Fig. 2 Overall survival in the cohort (A). Overall survival in intermediate-1 and -2 risk groups (B). Impact of baseline anemia on survival (C).

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