2017年8月4日,美国食品和药物管理局今天扩大了Imbruvica（依鲁替尼ibrutinib）应用范围，批准用于治疗一项或多项治疗失败后的慢性移植物抗宿主病（cGVHD）的成年患者。 这是FDA批准用于治疗慢性移植物抗宿主病cGVHD的治疗方法。
慢性移植物抗宿主病cGVHD是一种危及生命的病症。患者为治疗血液或骨髓疾病，接受造血干细胞移植，cGVHD 发生在患者接受干细胞移植后，cGVHD发生在干细胞移植细胞侵袭患者组织中的健康细胞时。
cGVHD的症状可能发生在皮肤，眼睛，口腔，肠道，肝脏和肺部。大约有30-70％接受HSCT的患者发生cGVHD。
FDA的血液和肿瘤学产品办公室主任Richard Pazdur博士说：“cGVHD患者对其他形式的治疗（通常是皮质类固醇抑制其免疫系统）目前没有有效的治疗方案。“这项批准强调了已知的癌症治疗如何在接受干细胞移植的血液癌患者中的严重和危及生命的病症中找到新的用途。
FDA批准Imbruvica（依鲁替尼ibrutinib）治疗cGVHD的疗效和安全性是根据一项cGVHD患者的42例的单臂试验结果。这42例cGVHD患者尽管用皮质类固醇进行了标准治疗，但仍有症状持续存在。大多数患者的症状包括口腔溃疡和皮疹，超过50％的患者有两个或更多器官受cGVHD影响。加入Imbruvica治疗后，67％的患者经历了cGVHD症状的改善。在48％的试验患者中，症状改善持续5个月或更久。
Imbruvica依鲁替尼对cGVHD患者的常见副作用包括疲劳，瘀伤，腹泻，血小板（血小板减少），肌肉痉挛，口腔肿胀和溃疡（口腔炎），恶心，严重出血（出血），低血红蛋白血细胞（贫血）和肺部感染（肺炎）。
Imbruvica依鲁替尼的严重副作用包括严重出血（出血），感染，血细胞减少（血细胞减少），心律不齐（心房颤动），高血压（高血压），新癌症（第二原发性恶性肿瘤）和代谢异常（肿瘤裂解）综合征）。怀孕或母乳喂养的妇女不应服用维生素，因为它可能会伤害发育中的胎儿或新生婴儿。
Imbruvica（ibrutinib依鲁替尼），是Pharmacyclics/Johnson & Johnson共同开发的BTK抑制剂，是一种激酶抑制剂，以前被批准用于治疗慢性淋巴细胞白血病以及在套细胞淋巴瘤，Waldenström的巨球蛋白血症和边缘区淋巴瘤的某些适应症。
FDA批准了这一应用优先审查和孤儿药物资格。FDA批准Imbruvica授予Pharmacyclics LLC。
IMBRUVICA Rx
Generic Name and Formulations:
Ibrutinib 70mg, 140mg; caps.
Company:
Pharmacyclics and Janssen Biotech
Indications for IMBRUVICA:
Mantle cell lymphoma (MCL) in patients who have received at least one prior therapy. Chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL). CLL/SLL in patients with 17p deletion. Waldenstrom's macroglobulinemia (WM). Marginal zone lymphoma (MZL) in patients who require systemic treatment and have received at least one prior anti-CD20-based therapy.
Adult:
Swallow whole with water. MCL and MZL: 560mg once daily. CLL/SLL (with or without bendamustine/rituximab) and WM: 420mg once daily. All: treat until disease progression or unacceptable toxicity. Hepatic impairment (mild): 140mg once daily; (moderate): 70mg once daily; (severe): avoid. Dose modifications for toxicities or concomitant CYP3A inhibitors (eg, voriconazole, posaconazole): see full labeling.
Children:
Not established.
Warnings/Precautions:
Risk of hemorrhage; consider the benefit/risk of withholding treatment for 3–7 days pre-and post-surgery. Monitor for fever and infections; eva luate promptly if occurs. Monitor for myelosuppression; obtain CBCs monthly. Periodically monitor for cardiac arrhythmias (esp. in those with cardiac risk factors, hypertension, acute infections, history of cardiac arrhythmias); do ECG if arrhythmic symptoms or new onset dyspnea develop. Monitor for new onset or uncontrolled hypertension; adjust and/or initiate anti-hypertensives as appropriate. Risk of second primary malignancies (eg, non-melanoma skin cancer or others). Monitor for tumor lysis syndrome in patients at risk (eg, high tumor burden). Hepatic impairment. Maintain adequate hydration. Embryo-fetal toxicity. Pregnancy; avoid during and for 1 month after treatment cessation. Nursing mothers.
Interactions:
Avoid concomitant strong CYP3A inhibitors (eg, boceprevir, clarithromycin, cobicistat, conivaptan, danoprevir/ritonavir, diltiazem, elvitegravir/ritonavir, idelalisib, indinavir/ritonavir, itraconazole, ketoconazole, lopinavir/ritonavir, nefazodone, nelfinavi |
