Cassano Cassano Raffaella, Bonadio Angelo Giovanni, Del Giudice Maria Livia, Giannese Domenico, Galimberti Sara, Buda Gabriele
Department of Clinical and Experimental Medicine, Hematology, University of Pisa, 56126, Pisa, Italy.
Department of Surgical, Medical, Molecular Pathology and Critical Area, Division of Surgical Pathology II, University of Pisa, Via Paradisa 2, 56124, Pisa, Italy.
Ann Hematol. 2025 Apr;104(4):2083-2093. doi: 10.1007/s00277-024-05911-9. Epub 2024 Aug 28.
Light chain deposition disease (LCDD) is a rare hematologic disorder characterized by the deposition of non-amyloid monoclonal light chains in several organs. Together with renal impairment is being the primary morbidity associated with this disease. Due to its rarity, randomized clinical trials lack to explore treatment strategies and there are no approved or universally accepted standard of care treatment options. We aimed to provide a systematic summary of histological and clinical aspects of LCDD and treatment options of available literature therapies strategies. Currently, drugs used to treat multiple myeloma are recommended when LCDD patients also presented multiple myeloma. Anyway, in patients with LCDD that is not associated to multiple myeloma, haematopoietic stem cell transplantation (ASCT) and chemotherapy with thalidomide, dexamethasone, bortezomib are also recommended. In eligible patients, bortezomib-based chemotherapy followed by ASCT appears to be an effective treatment option with durable hematologic remission and organ responses. Although it appears that the patients undergoing ASCT seem to achieve deeper and durable hematologic remissions and organ responses, no statistically significant superiority can be demonstrated over non-transplant or standard chemotherapy-based approaches. As retrieved by our review, bortezomib-based therapy appears to be favorable strategy as long as no dose modification is required for renal impairment, and early hematologic responses as a recovery of renal function. Encouraging data were also demonstrated by treatment lenalidomide or melpalan based. Moreover, new myeloma treatment strategies, as monoclonal antibody Daratumumab, seem to be effective in LCDD. Instead, renal allograft is not recommended, due to high incidence of relapse.
轻链沉积病(LCDD)是一种罕见的血液系统疾病,其特征是在多个器官中沉积非淀粉样单克隆轻链。肾功能损害是与该疾病相关的主要发病情况。由于其罕见性,缺乏随机临床试验来探索治疗策略,也没有批准的或普遍接受的标准治疗方案。我们旨在对LCDD的组织学和临床方面以及现有文献治疗策略的治疗选择进行系统总结。目前,当LCDD患者同时患有多发性骨髓瘤时,推荐使用治疗多发性骨髓瘤的药物。无论如何,对于与多发性骨髓瘤无关的LCDD患者,也推荐进行造血干细胞移植(ASCT)以及使用沙利度胺、地塞米松、硼替佐米进行化疗。在符合条件的患者中,基于硼替佐米的化疗后进行ASCT似乎是一种有效的治疗选择,可实现持久的血液学缓解和器官反应。尽管接受ASCT的患者似乎能实现更深且持久的血液学缓解和器官反应,但与非移植或基于标准化疗的方法相比,尚无统计学上的显著优势。根据我们的综述,只要肾功能损害不需要调整剂量,基于硼替佐米的治疗似乎是一种有利的策略,并且早期血液学反应可恢复肾功能。基于来那度胺或美法仑的治疗也显示出令人鼓舞的数据。此外,新的骨髓瘤治疗策略,如单克隆抗体达雷妥尤单抗,似乎对LCDD有效。相反,由于复发率高,不建议进行肾移植。