Palladini Giovanni, Perfetti Vittorio, Obici Laura, Caccialanza Riccardo, Semino Alessandra, Adami Fausto, Cavallero Giobatta, Rustichelli Roberto, Virga Giovambattista, Merlini Giampaolo
Amyloid Center Biotechnology Research Laboratories, Department of Biochemistry, University Hospital, Istituto di Ricovero e Cura a Carattere Scientifico-Policlinico San Matteo, Pavia, Italy.
Blood. 2004 Apr 15;103(8):2936-8. doi: 10.1182/blood-2003-08-2788. Epub 2003 Dec 18.
The most efficient therapeutic approach for immunoglobulin light chain amyloidosis (AL) is autologous stem cell transplantation (ASCT); however, the toxicity of ASCT limits its feasibility to a minority of patients. Patients ineligible for ASCT are usually treated with standard oral melphalan and prednisone, but the response rate to this regimen is unsatisfactory, and time to response is long. High-dose dexamethasone provides a rapid response time in patients with AL. We evaluated the combination of oral melphalan and high-dose dexamethasone (M-Dex) in 46 patients with AL ineligible for ASCT. Thirty-one (67%) achieved a hematologic response and 15 (33%) a complete remission. In 22 (48%) of the responsive patients functional improvement of the organs involved was observed. Five patients (11%) experienced severe adverse events, 3 required hospitalization, and no treatment-related deaths were observed. M-Dex represents a feasible and effective therapeutic option for patients with advanced AL who are ineligible for ASCT.
免疫球蛋白轻链淀粉样变性（AL）最有效的治疗方法是自体干细胞移植（ASCT）；然而，ASCT的毒性限制了其仅适用于少数患者。不符合ASCT条件的患者通常采用标准口服美法仑和泼尼松治疗，但该方案的缓解率不尽人意，且起效时间长。大剂量地塞米松可使AL患者起效迅速。我们评估了口服美法仑与大剂量地塞米松（M-Dex）联合方案对46例不符合ASCT条件的AL患者的疗效。31例（67%）患者获得血液学缓解，15例（33%）完全缓解。在22例（48%）缓解患者中观察到受累器官功能改善。5例（11%）患者发生严重不良事件，3例需要住院治疗，未观察到与治疗相关的死亡。M-Dex是不适用于ASCT的晚期AL患者一种可行且有效的治疗选择。
