Zhong Qing-Xiu, Meng Fan-Yi, Chen Hong-Yu, Li Xin, Wang Xiao-Guo, Huang Shun-Hua, Wu Ming-Yan, Yu Jian-Hua, Xue Ying, Wu Yi-Hong, Yao Da-Na, Long Jia-Xin, He Xiao-Hong
Department of Hematology, Kanghua Hospital, Dongguan, Guangdong, P.R. China.
Department of Oncology, Kanghua Hospital, Dongguan, Guangdong, P.R. China.
Ann Hematol. 2025 Jan;104(1):793-800. doi: 10.1007/s00277-025-06219-y. Epub 2025 Jan 24.
The efficacy and safety of total marrow irradiation (TMI) plus a reduced dose of melphalan as autologous stem cell transplantation (ASCT) preconditioning for multiple myeloma (MM) patients were evaluated. The 11 patients with MM had a median age of 57 (range: 46-75) years; six of them were at standard risk and five of them were at high risk based on the Mayo Stratification of Myeloma and Risk-adapted Therapy (mSMART) standard risk factors. Before ASCT, three patients achieved stringent complete response (sCR), two patients achieved complete remission (CR), and the rest of the patients had either partial response (PR) or progressive disease. Most of the 11 patients were pretreated with melphalan 120-140 mg/m and TMI 12 Gy. The intravenous infusion median mononuclear cell count (MNC) was 8.13 (4.16-11.84) × 10/kg, and the median CD34 count was 4.74 (2.51-21.98) × 10/kg. The minimal residual disease (MRD) in the grafts as determined by flow cytometry (FCM) and fluorescence in situ hybridization (FISH) were negative in 10 patients but positive in the progressive patient. All patients stopped maintenance therapy after transplantation, and further observation focused on the efficacy and tolerability of the transplantation. The neutropenic and thrombocytopenia durations were 11 (7-28) and 14 (8-70) days, respectively. The primary acute non-hematological toxicities were mild oral and gastrointestinal mucositis; there were no transplant-related deaths or serious complications. Of the eight patients who did not achieve sCR before transplantation, seven converted to sCR and one converted to VGPR after transplantation. The median follow-up period was 24 (10-57.5) months. Only one patient relapsed, and the progression-free survival (PFS) was 90.9%, while the overall survival (OS) was 100%. Our preliminary results suggest that melphalan 120-140 mg/m plus TMI 12 Gy/6f as a conditioning regimen is safe and efficient for patients with MM.
评估了全身骨髓照射（TMI）联合降低剂量的美法仑作为多发性骨髓瘤（MM）患者自体干细胞移植（ASCT）预处理方案的疗效和安全性。11例MM患者的中位年龄为57岁（范围：46 - 75岁）；根据梅奥骨髓瘤分层和风险适应性治疗（mSMART）标准危险因素，其中6例为标准风险，5例为高风险。在ASCT之前，3例患者达到严格完全缓解（sCR），2例患者达到完全缓解（CR），其余患者为部分缓解（PR）或疾病进展。11例患者中的大多数之前接受过120 - 140 mg/m的美法仑和12 Gy的TMI治疗。静脉输注的单核细胞计数（MNC）中位数为8.13（4.16 - 11.84）×10/kg，CD34计数中位数为4.74（2.51 - 21.98）×10/kg。通过流式细胞术（FCM）和荧光原位杂交（FISH）测定，10例患者移植物中的微小残留病（MRD）为阴性，但疾病进展的患者为阳性。所有患者移植后均停止维持治疗，进一步观察聚焦于移植的疗效和耐受性。中性粒细胞减少和血小板减少持续时间分别为11天（7 - 28天）和14天（8 - 70天）。主要的急性非血液学毒性为轻度口腔和胃肠道黏膜炎；无移植相关死亡或严重并发症。在移植前未达到sCR的8例患者中，7例移植后转为sCR，1例转为非常好的部分缓解（VGPR）。中位随访期为24个月（10 - 57.5个月）。仅1例患者复发，无进展生存期（PFS）为90.9%，总生存期（OS）为100%。我们的初步结果表明，120 - 140 mg/m的美法仑联合12 Gy/6f的TMI作为预处理方案对MM患者是安全有效的。
