Stem cell transplantation for the management of primary systemic amyloidosis
Stem cell transplantation for the management of primary systemic amyloidosis
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DOI：
10.1016/s0002-9343(02)01208-1
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发表时间：
2002-11-01
影响因子：
5.9
通讯作者：
Litzow, MR
中科院分区：
文献类型：
--
作者：
Gertz, MA;Lacy, MQ;Litzow, MR
目得：回顾淀粉样变性患者的特点和结果治疗高剂量化疗和干细胞reconstitutions.SUBJECTS和METHODS：66例活检证实的淀粉样变性患者在1996年3月和2001年1月之间接受移植。所有患者都有克隆性浆细胞恶液质的证据，那些与非免疫球蛋白形式的淀粉样变性被排除在外，是那些谁没有淀粉样变性，紫癜，腕管综合征，或症状性多发性myeloma.RESULTS症状：淀粉样蛋白被认为是临床肾脏（n = 45例），心脏（n = 32），外周神经（n = 11），和肝脏（n = 11）。在46例患者的血清和57例患者的尿液中发现了单克隆蛋白。平均每日尿蛋白丢失量为4.1 g。超声心动图测量的间隔厚度范围为7 - 24 mm（中位数为12 mm）; 8例患者的间隔厚度大于或等于16 mm。10例患者在诊断后1年或更长时间接受了移植。所有患者均接受以美法仑为基础的化疗; 17例患者接受全身照射。9名患者需要透析，其中7人死亡。干细胞移植的治疗相关死亡率为14%（9/66）。移植后中位随访25个月后，一个器官受累的患者存活率为91%（31/34）;两个器官受累的患者存活率为82%（18/22）;三个器官受累的患者存活率为33%（3/9）;四个器官受累的患者存活率为0%（0/1）。在33例患者中观察到血液学反应，在32例患者中观察到器官反应。结论：干细胞移植治疗淀粉样变患者术前受累器官数是预测术后生存率的重要因素。干细胞移植应被视为淀粉样变性患者的一种治疗选择。(C)2002年，Excerpta Medica，Inc.
PURPOSE: To review the characteristics and outcomes of amyloidosis patients treated with high-dose chemotherapy and stem cell reconstitution.SUBJECTS AND METHODS: Sixty-six patients with biopsyproven amyloidosis received transplants between March 1996 and January 2001. All patients had evidence of a clonal plasma cell dyscrasia; those with nonimmunoglobulin forms of amyloidosis were excluded, as were those who had no symptoms of amyloidosis, purpura, carpal tunnel syndrome, or symptomatic multiple myeloma.RESULTS: Amyloid was seen clinically in the kidneys (n = 45 patients), heart (n = 32), peripheral nerves (n = 11), and liver (n = 11). A monoclonal protein was found in the serum in 46 patients and in the urine in 57 patients. The median daily urinary protein loss was 4.1 g. Septal thickness, measured by echo-cardiography, ranged from 7 to 24 mm (median, 12 mm); 8 patients had a septal thickness greater than or equal to16 mm. Ten patients received transplants 1 year or more after diagnosis. All patients received melphalan-based chemotherapy; 17 patients were conditioned with total body irradiation. Nine patients required dialysis, 7 of whom died. Treatment-related mortality for stem cell transplantation was 14% (9/66). After a median of 25 months of follow-up after transplantation, the percentage of patients alive with one organ involved was 91% (31 of 34); two organs, 82% (18 of 22); three organs, 33% (3 of 9); and four organs, 0% (0 of 1). Hematologic responses were seen in 33 patients and organ responses in 32 patients. The 2-year actuarial survival of all patients was 70%.CONCLUSION: The number of organs involved before stem cell transplantation for amyloidosis is the most important factor in predicting subsequent survival. Stem cell transplantation should be considered as a treatment option for selected patients with amyloidosis. (C) 2002 by Excerpta Medica, Inc.