骨髓纤维化（MF）是一种预后不良的慢性骨髓增生性肿瘤，不仅因脾肿大和衰弱的全身症状而损害生活质量，而且具有很高的急性髓系白血病进展率。 Ruxolitinib 是一种 JAK 抑制剂，可增强 MF 的全身症状并延长生存期；然而，它不能有效抑制骨髓纤维化和白血病进展，仍需要异基因造血干细胞移植来治疗。因此，目前正在开发许多治疗 MF 的新药物。许多类似药物已被证明与鲁索替尼联合使用可增强治疗效果，特别是 BCL-2/BCL-XL 抑制剂、溴结构域和末端外结构域抑制剂以及人双分钟同源物 2 抑制剂，以改善骨髓纤维化。这项研究概述了目前在日本进行的临床试验中使用的药物。

Myelofibrosis (MF) is a chronic myeloproliferative tumor with a poor prognosis that not only impairs the quality of life because of splenomegaly and debilitating systemic symptoms but also has a high acute myeloid leukemia progression rate. Ruxolitinib, a JAK inhibitor, enhances systemic symptoms in MF and prolongs survival; however, it is not effective in suppressing bone marrow fibrosis and leukemia progression, and allogeneic hematopoietic stem cell transplantation remains needed for treatment. As a result, many new drugs for MF are presently being developed. Many similar drugs have been demonstrated to enhance therapeutic effectiveness if combined with ruxolitinib, particularly BCL-2/BCL-XL inhibitors, bromodomain and extra-terminal domain inhibitors, and human double-minute homolog 2 inhibitors, to improve bone marrow fibrosis. This study provides an overview of drugs currently employed in clinical trials being performed in Japan.