Impact of calreticulin mutations on treatment and survival outcomes in myelofibrosis during ruxolitinib therapy.

• Tác giả: Elisabetta Abruzzese, Eloise Beggiato, Giulia Benevolo, Gianni Binotto, Monica Bocchia, Massimiliano Bonifacio, Filippo Branzanti, Massimo Breccia, Giovanni Caocci, Santino Caserta, Francesco Cavazzini, Daniela Cilloni, Monica Crugnola, Domenico D'Agostino, Alessandra Dedola, Elena M Elli, Mirko Farina, Florian H Heidel, Alessandro Isidori, Roberto Massimo Lemoli, Bruno Martino, Erika Morsia, Antonella Nardo, Francesca Palandri, Giuseppe A Palumbo, Fabrizio Pane, Novella Pugliese, Emilia Scalzulli, Alessia Tieghi, Mario Tiribelli
• Ngôn ngữ: eng
• Thông tin xuất bản: Germany : Annals of hematology , 2025
• Bộ sưu tập: NCBI
• ID: 730559

Tóm tắt

 Calreticulin (CALR) mutations are detected in around 20% of patients with primary and post-essential thrombocythemia myelofibrosis (MF). Regardless of driver mutations, patients with splenomegaly and symptoms are generally treated with JAK2-inhibitors, most commonly ruxolitinib. Recently, new therapies specifically targeting the CALR mutant clone have entered clinical investigation. To collect information on efficacy and safety of ruxolitinib in CALR-mutated patients, we report a sub-analysis of the "RUX-MF" (NCT06516406) study, comprising 135 CALR-mutated and 786 JAK2-mutated ruxolitinib-treated patients. Compared to JAK2-mutated patients, CALR-mutated patients started ruxolitinib with a more severe disease (higher peripheral blast counts, lower hemoglobin levels and worse marrow fibrosis) and after a longer median time from diagnosis (2.6 versus 0.7 years, p <
  0.002). At 6 months, spleen responses were numerically inferior in CALR-mutated patients, who also had significantly lower rates of symptom responses (56.1% versus 66.7%, p = 0.04).