Real-world effectiveness and safety of ruxolitinib in myelofibrosis: A multicenter cohort study (CHART-MF)


Tarkun P., Sönmez M., Yokuş O., Tuğlular A. T., Özkurt Z. N., Saydam G., ...Daha Fazla

Blood Global Hematology, ss.1-21, 2026 (Hakemli Dergi)

  • Yayın Türü: Makale / Tam Makale
  • Basım Tarihi: 2026
  • Doi Numarası: 10.1016/j.bglo.2026.100162
  • Dergi Adı: Blood Global Hematology
  • Derginin Tarandığı İndeksler: Directory of Open Access Journals
  • Sayfa Sayıları: ss.1-21
  • Eskişehir Osmangazi Üniversitesi Adresli: Evet

Özet

Ruxolitinib is a Janus kinase inhibitor for treatment of intermediate or high-risk primary and secondary myelofibrosis (MF). The CHART-MF multicenter, retrospective, non-interventional study aimed to describe the characteristics of MF patients (≥18 years) treated with ruxolitinib and to assess its 12-month effectiveness and safety in 355 patients (median age: 63 years) classified into Group 1 (intermediate-1 risk) and Group 2 (intermediate-2 or high-risk) based on the Dynamic International Prognostic Scoring System plus scores. Patients in Group I were younger (61 vs 65 years; p=0.047), had longer time since diagnosis to onset of ruxolitinib (6.70 vs 5.47 years; p=0.045) and more favorable karyotypes (6.3% vs 22.7%; p=0.003). The proportion of patients with secondary MF was higher in Group I than in Group 2 (64.7% vs 45.3% p<0.001). Among patients with spleen size data available at baseline and month 12, splenomegaly resolved in 66.3% (58/91) by month 12. Serial hemograms revealed a transient decline in hemoglobin and thrombocyte levels, which did not require dose adjustment in most cases. While anemia caused treatment discontinuation in 7.4% of subjects, any patient ceased ruxolitinib because of thrombocytopenia. Overall, 253 adverse events (AEs) occurred in 135 patients (38%), 82 (60.7%) of whom experienced hematological AEs including anemia (30.3%; 41/135) and thrombocytopenia (21.5%; 29/135). Most reported non-hematological AEs were pneumonia and urinary tract infections (6.7%; 9/135 each). The CHART-MF showed that, with close monitoring, ruxolitinib was safely and effectively used as a second-line treatment for MF in clinical practice in Türkiye, regardless of patients’ risk categories.