Critical Reviews in Oncology/Hematology ( IF 6.2 ) Pub Date : 2021-07-31 , DOI: 10.1016/j.critrevonc.2021.103435 Hamza Hassan 1 , Faiz Anwer 2 , Anum Javaid 3 , Hamza Hashmi 4
Outcomes for patients with systemic light-chain (AL) amyloidosis have improved over the last two decades with timely diagnosis, use of novel chemotherapeutic agents, risk stratification and better patient selection criteria before hematopoietic autologous stem cell transplant (ASCT). However, majority of patients have advanced stage disease at initial presentation and at relapse rendering them ineligible for intensive cytotoxic chemotherapy or ASCT. Daratumumab (Dara) with or without standard chemotherapy appears to be an excellent treatment option for newly diagnosed and relapsed refractory AL amyloidosis. This is largely due to its tolerable safety and remarkable efficacy as seen in multiple retrospective, small phase II studies as well as a phase III randomized controlled trial. Here we review published clinical trials and retrospective data of Dara in AL amyloidosis that explore its role as a valuable addition to the treatment armamentarium for this challenging disease.
中文翻译:
研究进展:Daratumumab 改善 AL 淀粉样变性患者的治疗结果
在过去的 20 年中,系统性轻链 (AL) 淀粉样变性患者的预后随着及时诊断、新型化疗药物的使用、风险分层和更好的患者选择标准而在造血自体干细胞移植 (ASCT) 之前有所改善。然而,大多数患者在初次就诊和复发时已处于晚期疾病,使他们不适合进行强化细胞毒性化疗或 ASCT。Daratumumab (Dara) 联合或不联合标准化疗似乎是新诊断和复发难治性 AL 淀粉样变性的绝佳治疗选择。这主要是由于其在多项回顾性、小型 II 期研究以及 III 期随机对照试验中所见的可耐受的安全性和显着的疗效。