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单克隆Ig沉积病患者的大剂量美法仑与自体造血干细胞移植

High-dose melphalan and auto-SCT in patients with monoclonal Ig deposition disease.

作者信息

Hassoun H, Flombaum C, D'Agati V D, Rafferty B T, Cohen A, Klimek V M, Boruchov A, Kewalramani T, Reich L, Nimer S D, Comenzo R L

机构信息

Division of Hematologic Oncology, Department of Medicine, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA.

出版信息

Bone Marrow Transplant. 2008 Sep;42(6):405-12. doi: 10.1038/bmt.2008.179. Epub 2008 Jun 23.

Abstract

The treatment of monoclonal Ig deposition disease (MIDD) is controversial and not standardized. We report our experience with high dose melphalan and auto-SCT (HDM/auto-SCT) in seven patients with MIDD associated with underlying Durie-Salmon stage IB multiple myeloma, including five with light chain deposition disease, one with light and heavy chain deposition disease and one with light chain crystal deposition disease. The median age of these patients was 50 years; six of them were male subjects. A monoclonal kappa-light chain was detected by Serum Free Light Chain Assay in all seven. The patients received melphalan 140 mg/m(2) followed by auto-SCT. All patients are alive and six remain in hematologic CR with a median follow up of 23.6 months (7.9-69.8 months). Renal function has improved compared to pre-HDSM/auto-SCT in five patients--two of whom had a renal transplant and became dialysis independent--remained stable in one and worsened in one leading to hemodialysis despite hematologic CR. Our results corroborate previous experience with HDM/auto-SCT in MIDD and argue in favor of kidney transplantation in patients who achieve hematologic CR after HDM/auto-SCT. Although this approach appears effective, multi-center studies are needed to define the optimal treatment for patients with MIDD.

摘要

单克隆免疫球蛋白沉积病(MIDD)的治疗存在争议且未标准化。我们报告了7例与潜在Durie-Salmon分期IB期多发性骨髓瘤相关的MIDD患者接受大剂量美法仑和自体造血干细胞移植(HDM/auto-SCT)的经验,其中包括5例轻链沉积病患者、1例轻链和重链沉积病患者以及1例轻链晶体沉积病患者。这些患者的中位年龄为50岁;其中6例为男性。通过血清游离轻链检测在所有7例患者中均检测到单克隆κ轻链。患者接受140 mg/m²的美法仑治疗,随后进行自体造血干细胞移植。所有患者均存活,6例仍处于血液学完全缓解状态,中位随访时间为23.6个月(7.9 - 69.8个月)。与HDM/auto-SCT治疗前相比,5例患者的肾功能有所改善,其中2例进行了肾移植并脱离透析,1例保持稳定,1例尽管血液学完全缓解但肾功能恶化并需要进行血液透析。我们的结果证实了先前HDM/auto-SCT治疗MIDD的经验,并支持在HDM/auto-SCT后达到血液学完全缓解的患者中进行肾移植。尽管这种方法似乎有效,但仍需要多中心研究来确定MIDD患者的最佳治疗方案。

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