A drug previously approved for adults with certain types of lymphoma or leukemia has become the first FDA approved medication for chronic graft-vs-host disease (GVHD). Ibrutinib, marketed as Imbruvica, inhibits Bruton tyrosine kinase, a protein that plays a role in the survival and spread of malignant B cells. The medication is indicated for adults whodevelopchronicGVHDafterahematopoietic stem cell transplant but don’t respond to systemic therapy. An estimated 30% to 70% of patients who undergo these transplants subsequently develop chronic GVHD. The drug’s approval is based on an ongoing, single-arm phase 1b and phase 2 trial that evaluated ibrutinib’s safety and efficacy in 42 patients with chronic GVHD who didn’t respond to first-line corticosteroid therapy. The most common malignancies that led to the study participants’ transplants were acute lymphocytic leukemia, acute myeloid leukemia, and chronic lymphocytic leukemia. Nearly 90% of patients had at least 2 organs—most often the mouth or skin—affected by their disease. According to a statement from AbbVie Inc, which developed the drug with Pharmacyclics LLC and Janssen Biotech Inc, 67% of the patients responded to the drug. Among them, 21% had complete responses and 45% had partial responses. Some 48% of patients had sustained responses lasting for at least 20 weeks. Lead investigator David Miklos, MD, PhD, a specialist in blood and marrow transplantation at Stanford University Medical Center, said in a statement that ibrutinib’s approval “represents a major advance and provides physicians with a new option for adults with steroidrefractory [chronic] GVHD.” The most common adverse events reported include fatigue, bruising, diarrhea, and thrombocytopenia. More serious adverse effects include hemorrhage, infections, cytopenias, and secondary primary malignancies.

中文翻译：

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胃内气球手术后报告的死亡人数

一种先前批准用于患有某些类型的淋巴瘤或白血病的成年人的药物已成为 FDA 批准的第一种治疗慢性移植物抗宿主病 (GVHD) 的药物。依鲁替尼，以 Imbruvica 销售，抑制布鲁顿酪氨酸激酶，一种在恶性 B 细胞的存活和扩散中起作用的蛋白质。该药物适用于造血干细胞移植后出现慢性 GVHD 但对全身治疗无反应的成人。估计有 30% 至 70% 接受这些移植的患者随后会发展为慢性 GVHD。该药物的批准基于一项正在进行的单臂 1b 和 2 期试验，该试验评估了依鲁替尼在 42 名对一线皮质类固醇治疗无反应的慢性 GVHD 患者中的安全性和有效性。导致研究参与者移植的最常见恶性肿瘤是急性淋巴细胞白血病、急性髓细胞白血病和慢性淋巴细胞白血病。近 90% 的患者至少有 2 个器官（最常见的是口腔或皮肤）受到疾病的影响。根据与 Pharmacyclics LLC 和 Janssen Biotech Inc 共同开发该药物的 AbbVie Inc 的一份声明，67% 的患者对该药物有反应。其中，21%有完全反应，45%有部分反应。大约 48% 的患者有持续至少 20 周的反应。首席研究员 David Miklos，医学博士，斯坦福大学医学中心血液和骨髓移植专家，在一份声明中表示，依鲁替尼的批准“代表了一个重大进步，并为医生提供了一种治疗类固醇难治性 [慢性] GVHD 成人的新选择。” 报告的最常见的不良事件包括疲劳、瘀伤、腹泻和血小板减少症。更严重的副作用包括出血、感染、血细胞减少和继发性原发性恶性肿瘤。