Yun Nicole K, Alrifai Taha, Miller Ira J, Shammo Jamile M
Department of Internal Medicine, Rush University Medical Center, Chicago, Illinois, USA.
Division of Hematology, Oncology, and Cell Therapy, Rush University Medical Center, Chicago, Illinois, USA.
Case Rep Oncol. 2022 Feb 14;15(1):126-132. doi: 10.1159/000521889. eCollection 2022 Jan-Apr.
Myelofibrosis (MF)-associated anemia and transfusion dependency are associated with inferior quality of life and poor prognosis. JAK2 inhibitors and TGF-β superfamily ligand traps are being explored as treatment options for MF-associated anemia. Here, we present the case of a 66-year-old man with heavily pretreated intermediate-2 (INT-2) risk primary MF who had an exceptional response to combination fedratinib and luspatercept therapy. He achieved transfusion independence and experienced a reduction in spleen size from 20 cm to 12 cm, with remarkable improvement in performance status. Compared with other JAK inhibitors, the mechanism of action of fedratinib may explain its milder effect on anemia. It is possible that the addition of luspatercept may result in an additive or synergistic effect of one or both medications. Although the exact biological pathways have not yet been elucidated, combination fedratinib and luspatercept nevertheless is a promising therapy for anemia in patients with transfusion-dependent INT-2 risk MF.
骨髓纤维化（MF）相关贫血和输血依赖与生活质量低下及预后不良相关。JAK2抑制剂和转化生长因子-β超家族配体陷阱正被探索作为MF相关贫血的治疗选择。在此，我们报告一例66岁男性患者，其患有经过大量前期治疗的中危-2（INT-2）原发性MF，对fedratinib和罗特西普联合治疗有显著反应。他实现了输血独立，脾脏大小从20厘米缩小至12厘米，体能状态有显著改善。与其他JAK抑制剂相比，fedratinib的作用机制可能解释了其对贫血的影响较轻。添加罗特西普可能会使一种或两种药物产生相加或协同效应。尽管确切的生物学途径尚未阐明，但fedratinib和罗特西普联合用药仍是输血依赖型INT-2风险MF患者贫血的一种有前景的治疗方法。
