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<p>Calreticulin (<i>CALR</i>) 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 <i>JAK2</i>-inhibitors, most commonly ruxolitinib. Recently, new therapies specifically targeting the <i>CALR</i> mutant clone have entered clinical investigation. To collect information on efficacy and safety of ruxolitinib in <i>CALR</i>-mutated patients, we report a sub-analysis of the “RUX-MF” (NCT06516406) study, comprising 135 <i>CALR</i>-mutated and 786 <i>JAK2</i>-mutated ruxolitinib-treated patients. Compared to <i>JAK2</i>-mutated patients, <i>CALR</i>-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, <i>p</i> &lt; 0.001). At 6 months, spleen responses were numerically inferior in <i>CALR</i>-mutated patients, who also had significantly lower rates of symptom responses (56.1% versus 66.7%, <i>p</i> = 0.04).</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年1月20日

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  • Francesca Palandri,
  • Filippo Branzanti,
  • Erika Morsia,
  • Alessandra Dedola,
  • Giulia Benevolo,
  • Mario Tiribelli,
  • Eloise Beggiato,
  • Mirko Farina,
  • Bruno Martino,
  • Giovanni Caocci,
  • Novella Pugliese,
  • Alessia Tieghi,
  • Monica Crugnola,
  • Gianni Binotto,
  • Francesco Cavazzini,
  • Elisabetta Abruzzese,
  • Alessandro Isidori,
  • Emilia Scalzulli,
  • Domenico D’Agostino,
  • Santino Caserta,
  • Antonella Nardo,
  • Roberto Massimo Lemoli,
  • Daniela Cilloni,
  • Monica Bocchia,
  • Fabrizio Pane,
  • Florian H. Heidel,
  • Giuseppe A. Palumbo,
  • Massimo Breccia,
  • Elena M. Elli,
  • Massimiliano Bonifacio

摘要

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 < 0.001). 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).