Mora Barbara, Maffioli Margherita, Rumi Elisa, Guglielmelli Paola, Caramella Marianna, Kuykendall Andrew, Palandri Francesca, Iurlo Alessandra, De Stefano Valerio, Kiladjian Jean-Jacques, Elli Elena M, Polverelli Nicola, Gotlib Jason, Albano Francesco, Silver Richard T, Benevolo Giulia, Ross David M, Devos Timothy, Borsani Oscar, Barbui Tiziano, Porta Matteo G Della, Bertù Lorenza, Komrokji Rami, Vannucchi Alessandro M, Passamonti Francesco
Department of Oncology, ASST Sette Laghi Ospedale di Circolo Varese Italy.
Department of Molecular Medicine University of Pavia Pavia Italy.
EJHaem. 2023 Jul 17;4(3):679-689. doi: 10.1002/jha2.745. eCollection 2023 Aug.
Myelofibrosis (MF) is a clonal malignancy frequently characterized by anemia and in 10%-20% of cases it can evolve into blast phase (BP). Anemia in MF is associated with reduced survival and -in primary MF- also with an increased probability of BP. Conventional treatments for anemia have limited effectiveness in MF. Within a dataset of 1752 MF subjects largely unexposed to ruxolitinib (RUX), BP incidence was 2.5% patients per year (p-y). This rate reached respectively 4.3% and 4.5% p-y in case of patients with common terminology criteria for adverse events (CTCAE) grade 3/4 and grade 2 anemia, respectively, that represented together 32% of the cohort. Among 273 MF cases treated with RUX, BP incidence was 2.89% p-y and it reached 4.86% p-y in subjects who started RUX with CTCAE grade 2 anemia (one third of total). Within patients with red blood cell transfusion-dependency at 6 months of RUX (21% of the exposed), BP rate was 4.2% p-y. Our study highlights a relevant incidence of BP in anemic MF patients, with a similar rate whether treated with or without RUX. These findings will help treating physicians to make decisions on the safety profile of innovative anemia treatments.
骨髓纤维化（MF）是一种克隆性恶性肿瘤，常表现为贫血，在10%-20%的病例中可进展为急变期（BP）。MF患者的贫血与生存率降低相关，在原发性MF中还与BP发生概率增加有关。贫血的传统治疗方法在MF中的疗效有限。在一个包含1752例基本未接受芦可替尼（RUX）治疗的MF患者的数据集中，BP发生率为每年2.5%患者（每患者年）。在不良事件通用术语标准（CTCAE）为3/4级和2级贫血的患者中，该发生率分别达到每年4.3%和4.5%患者，这两类患者共占队列的32%。在273例接受RUX治疗的MF病例中，BP发生率为每年2.89%患者，在开始使用RUX时CTCAE为2级贫血的患者（占总数的三分之一）中达到每年4.86%患者。在接受RUX治疗6个月时依赖红细胞输血的患者（占暴露患者的21%）中，BP发生率为每年4.2%患者。我们的研究强调了贫血MF患者中BP的相关发生率，无论是否接受RUX治疗，发生率相似。这些发现将有助于治疗医生就创新贫血治疗的安全性做出决策。
