Vaxman Iuliana, Dispenzieri Angela
Division of Hematology, Mayo Clinic, Rochester, MN.
Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Petah-Tikvah, Israel.
Oncology (Williston Park). 2021 Aug 12;35(8):471-478. doi: 10.46883/ONC.2021.3508.0471.
Autologous stem cell transplantation (ASCT) has been an essential part of the treatment armamentarium in light chain (AL) amyloidosis for several decades. Patients who achieve a complete hematologic response following ASCT have a long overall survival. However, only 1 randomized controlled trial compared ASCT with the standard of care used at the time, which was melphalan and dexamethasone, and the results did not support the use of ASCT in AL amyloidosis. These results are of limited significance due to the unexpected high transplant-related mortality (TRM) (24%). TRM is a major concern in AL amyloidosis, but its incidence can be lessened by better patient selection and by patients receiving ASCT in specialized centers. ASCT in AL amyloidosis is performed only in selected patients; approximately 20% of patients with AL amyloidosis are transplant eligible up front or after bortezomib (Velcade) based conditioning. The introduction of newer agents such as bortezomib and daratumumab (Darzalex), which lead to deep responses and have good safety profiles, encourage revisiting the benefit and timing of ASCT in the modern era. This review provides a comprehensive assessment of eligibility criteria for ASCT in AL amyloidosis, conditioning dosing, efficacy in terms of hematologic and organ response, and future areas of research.
几十年来，自体干细胞移植（ASCT）一直是轻链（AL）淀粉样变性治疗手段的重要组成部分。接受ASCT后实现完全血液学缓解的患者总体生存期较长。然而，仅有1项随机对照试验将ASCT与当时使用的标准治疗方案（美法仑和地塞米松）进行了比较，结果并不支持在AL淀粉样变性中使用ASCT。由于意外的高移植相关死亡率（TRM）（24%），这些结果的意义有限。TRM是AL淀粉样变性中的一个主要问题，但通过更好的患者选择以及患者在专业中心接受ASCT，其发生率可以降低。AL淀粉样变性的ASCT仅在选定的患者中进行；大约20%的AL淀粉样变性患者在初始时或基于硼替佐米（万珂）的预处理后符合移植条件。新型药物如硼替佐米和达雷妥尤单抗（兆珂）的引入，可带来深度缓解且安全性良好，这促使人们重新审视现代ASCT的获益及时机。本综述全面评估了AL淀粉样变性中ASCT的入选标准、预处理剂量、血液学和器官反应方面的疗效以及未来的研究领域。
