骨髓纖維化為一種預後不佳的骨髓增生性腫瘤，常見症狀為疲勞、發燒、夜間盜汗及體重減輕，嚴重的骨髓纖維化病人可能需進行異體造血幹細胞移植，藥物治療方面，過去的藥物選擇多為hydroxyurea、danazol、類固醇等，近年來，詹納斯激酶 (Janus kinase, JAK) 抑制劑成為新的治療選擇，包含ruxolitinib、fedratinib、pacritinib 及momelotinib，這些藥物已被2024 年美國國家癌症資訊網指引納入第一線治療選擇。臺灣衛生福利部食品藥物管理署已核准ruxolitinib 作為第一個用於骨髓纖維化的JAK 抑制劑，而fedratinib 經由臨床試驗證明可能作為ruxolitinib 治療失敗後的藥物選擇，pacritinib 和momelotinib 則分別適用於嚴重血小板減少症及貧血的病人。雖然JAK 抑制劑可有效控制骨髓纖維化，但可能伴隨血液學不良反應，例如貧血、血小板減少症及嗜中性白血球減少症，其他常見的不良反應包含腹瀉、噁心嘔吐等。本文蒐集彙整骨髓纖維化現今的藥物資訊，希望藉此提供醫療人員加深對骨髓纖維化的認識，並對往後的臨床實務能有所幫助。

 

 

Myelofibrosis is a poor-prognosis myeloproliferative neoplasm characterized by symptoms such as fatigue, fever, night sweats, and weight loss. Patients suffering from severe progressive myelofibrosis may require allogeneic hematopoietic cell transplantation. As for pharmacologic management, choices in the past were mostly hydroxyurea, danazol, steroids, etc. In recent years, Janus kinase (JAK) inhibitors, including ruxolitinib, fedratinib, pacritinib and momelotinib, have become new therapeutic options, all of which have been included as first-line treatment options in the 2024 National Comprehensive Cancer Network guidelines. Taiwan Food and Drug Administration has approved ruxolitinib as the first JAK inhibitor for myelofibrosis. Fedratinib has been proven through clinical trials to be a possible option after failure of ruxolitinib treatment. Additionally, pacritinib and momelotinib are suitable for patients with severe thrombocytopenia and anemia respectively. Although JAK inhibitors can effectively control myelofibrosis, they may be accompanied by hematological adverse reactions, such as anemia, thrombocytopenia, and neutropenia. Other common adverse reactions include diarrhea, nausea, and vomiting. The authors collected recent pharmaceutical information and conducted a literature review aimed at providing clinicians with a better understanding of myelofibrosis and its pharmacologic management.

 

 

Submitted for publication: 2023.11.18; Accepted for publication: 2024.6.25