Baker K S, DeLaat C A, Steinbuch M, Gross T G, Shapiro R S, Loechelt B, Harris R, Filipovich A H
Section of Pediatric Hematology/Oncology and Bone Marrow Transplant, University of Nebraska Medical Center, Omaha 68198-2168, USA.
Blood. 1997 May 15;89(10):3857-63.
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.
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紊乱疾病，可导致重要器官广泛的淋巴细胞和噬血细胞浸润。只有通过异基因骨髓移植（BMT）才能实现明显治愈。本报告描述了连续20例接受同胞匹配供体（n = 4）或无关供体（URD；n = 16）BMT的患者。BMT时的年龄为0.4至5.3岁（中位数，0.8岁）。13例患者在诊断时存在中枢神经系统疾病。在进行BMT时，14例患者处于临床缓解期，而6例患者患有活动性HLH。所有患者在接受白消安、环磷酰胺和依托泊苷进行细胞减灭术后均实现了植入。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%置信区间[CL]，0.28，0.86），URD患者为73%（95% CL，0.44，1.0）。单独评估URD BMT时，3年总体生存概率为45%（95% CL，0.23，0.67）和44%（95% CL，0.19，0.68）。BMT的良好结局与BMT时的临床缓解状态相关。预处理方案耐受性良好，在9例存活患者中，它提供了持久的植入，并有效根除了潜在疾病。
