骨髓纤维化 (MF) 是一种罕见的 BCR-ABL 阴性骨髓增生性肿瘤，其特征是干细胞克隆性增殖，并伴有 JAK2、CALR 或 MPL 基因突变。 MF 有原发性和继发性两种形式，常见症状包括脾肿大、贫血和血小板减少。诊断标准包括骨髓检查和突变研究。目前的治疗方法有限，同种异体干细胞移植是唯一的治疗选择。 FDA 最近批准的 Momelotinib (MMB) 为患有贫血的 MF 患者带来了新的希望。 MMB 是一种 JAK1/2 和 ACVR1 抑制剂，可有效缩小脾脏大小，提高血红蛋白水平，并减少输血依赖性。 MOMENTUM 试验在 JAK 抑制剂治疗的贫血 MF 患者中比较了 MMB 与达那唑，显示 MMB 具有优异的症状缓解和输血独立率。此外，SIMPLIFY-1 和 SIMPLIFY-2 试验分别在未使用过 JAK 抑制剂的患者和有经验的患者中评估了 MMB，证实了 MMB 在减少脾脏体积方面不劣于鲁索替尼，并强调了其在输血需求方面的益处。 MMB 独特的双重抑制机制通过抑制铁调素的产生来解决贫血问题，从而增强红细胞生成。这些试验共同表明 MMB 是治疗 MF 的有效方法，可以改善生活质量并为贫血患者提供生存优势。尽管面临试验设计限制和不良事件等挑战，MMB 代表了 MF 管理的重大进步，为以前服务不足的患者群体提供了新的治疗选择。版权所有 © 2024 Furqan 和 Oduoye。

Myelofibrosis (MF) is a rare BCR-ABL negative myeloproliferative neoplasm characterized by clonal proliferation of stem cells, with mutations in JAK2, CALR, or MPL genes. MF presents in primary and secondary forms, with common symptoms including splenomegaly, anemia, and thrombocytopenia. Diagnostic criteria involve bone marrow examination and mutation studies. Current treatments are limited, with allogeneic stem cell transplant as the only curative option. Recent FDA approval of Momelotinib (MMB) offers new promise for MF patients with anemia. MMB, a JAK1/2 and ACVR1 inhibitor, effectively reduces spleen size, improves hemoglobin levels, and decreases transfusion dependency. The MOMENTUM trial compared MMB to danazol in JAK inhibitor-treated MF patients with anemia, showing MMB's superior symptom relief and transfusion independence rates. Additionally, the SIMPLIFY-1 and SIMPLIFY-2 trials evaluated MMB in JAK inhibitor-naïve and experienced patients, respectively, confirming MMB's non-inferiority to ruxolitinib in spleen volume reduction and highlighting its benefits in transfusion requirements. MMB's unique dual inhibition mechanism addresses anemia by suppressing hepcidin production, thus enhancing erythropoiesis. These trials collectively suggest MMB as an effective treatment for MF, improving quality of life and offering a survival advantage for patients with anemia. Despite challenges, such as trial design limitations and adverse events, MMB represents a significant advancement in MF management, providing a new therapeutic option for a previously underserved patient population.Copyright © 2024 Furqan and Oduoye.