Successful correction of hemophagocytic lymphohistiocytosis with related or unrelated bone marrow transplantation
Successful correction of hemophagocytic lymphohistiocytosis with related or unrelated bone marrow transplantation
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DOI：
10.1182/blood.v89.10.3857.3857_3857_3863
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发表时间：
1997-05-15
期刊：
影响因子：
20.3
通讯作者：
Filipovich, AH
中科院分区：
文献类型：
--
作者：
Baker, KS;DeLaat, CA;Filipovich, AH
噬血细胞性淋巴组织细胞增生症（HLH）是一种危及生命的免疫调节障碍，导致重要器官的广泛淋巴细胞和噬血细胞浸润。只有异基因骨髓移植（BMT）才能达到明显的治愈。本报告描述了20例连续患者，他们接受了匹配的同胞供体（n = 4）或无关供体（URD; n = 16）BMT。BMT时的年龄为0.4至5.3岁（中位数为0.8岁）。13例患者在诊断时存在中枢神经系统疾病。在BMT时，14例患者临床缓解，而6例患者有活动性HLH。所有患者均在白消安、环磷酰胺和依托泊苷细胞减灭后进行移植。所有患者发生II-III级急性移植物抗宿主病（GVHD）的概率为57%（95%置信限[CL]，0.28，0.86），URD患者为73%（95% CL，0.44，1.0）。当分别评价URD BMT时，3年生存率的总体概率为45%（95% CL，0.23，0.67）和44%（95% CL，0.19，0.68）。有利的BMT结果与BMT时的临床缓解状态相关。准备方案耐受性良好，在9例存活患者中，它提供了持久的植入，并有效根除了潜在的疾病。(C)1997年，美国血液学会。
Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening disorder of immune regulation leading to widespread lymphocytic and hemophagocytic infiltration of vital organs. Apparent cure has only been achieved with allogeneic bone marrow transplantation (BMT). This report describes 20 consecutive patients, who underwent either matched sibling donor (n = 4) or unrelated donor (URD; n = 16) BMT. Age at the time of BMT was 0.4 to 5.3 years (median, 0.8 years). Central nervous system disease was present at diagnosis in 13 patients. At BMT, 14 patients were in a clinical remission, whereas 6 patients had active HLH. All patients were engrafted after cytoreduction with busulfan, cyclophosphamide, and etoposide. The probability of grade II-III acute graft-versus-host disease (GVHD) for all patients was 57% (95% confidence limit [CL], 0.28, 0.86), and 73% (95% CL, 0.44, 1.0) in URD patients. The overall probability of survival at 3 years was 45% (95% CL, 0.23, 0.67) and 44% (95% CL, 0.19, 0.68) when URD BMT was evaluated separately. Favorable BMT outcome was associated with clinical remission status at the time of BMT. The preparative regimen was well tolerated, and in the 9 surviving patients it provided durable engraftment and was effective at eradicating the underlying disease. (C) 1997 by The American Society of Hematology.