Haematopoietic stem cell transplantation in haemophagocytic lymphohistiocytosis
Haematopoietic stem cell transplantation in haemophagocytic lymphohistiocytosis
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DOI：
10.1111/j.1365-2141.2005.05501.x
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发表时间：
2005-06-01
影响因子：
6.5
通讯作者：
Henter, JI
中科院分区：
文献类型：
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作者：
Horne, A;Janka, G;Henter, JI
噬血细胞性淋巴组织细胞增生症（HLH）是一个主要的治疗挑战，其主要遗传形式家族性噬血细胞性淋巴组织细胞增生症（FHL）通常是致命的。我们评估了1995年至2000年间接受HLH-94治疗（依托泊苷、地塞米松、环孢素）后接受异基因干细胞移植（SCT）的86例儿童（29例家族性）的生存率，包括考克斯回归分析。SCT后总体估计3年生存率为64% [置信区间（CI）= +/- 10%]（n = 86）; 71 +/- 18%的患者有匹配的亲属供体（MRD，n 24），70 +/- 16%与匹配的无关供体（MUD，n = 33），50 +/- 24%为家族单倍体相合供体（单倍体相合，n = 16），54 +/- 27%为不匹配的无关供体（MMUD，n = 13）。校正潜在混杂因素后，与MRD相比，MUD、单倍相合和MMUD的死亡率估计比值比（OR）分别为1.93（CI = 0.61-6.19）、3.31（1.02-10.76）和3.01（0.91-9.97）。在治疗2个月后的活动性疾病儿童（n = 43）中，与非活动性疾病（n = 43）相比，OR为2.75（1.26-5.99）。在SCT时有活动性疾病的儿童（n = 37）中，校正2个月时的疾病活动性后，与非活动性疾病（n = 49）相比，OR为1.80（0.80-4.06）。死亡率主要与移植相关。大多数HLH患者在使用MRD或MUD的SCT中存活，部分不匹配供体的存活也是可以接受的。对初始移植前诱导治疗反应良好的患者表现最好，但一些持续的HLH活性不应自动排除进行SCT。
Haemophagocytic lymphohistiocytosis (HLH) poses major therapeutic challenges, and the primary inherited form, familial haemophagocytic lymphohistiocytosis (FHL), is usually fatal. We evaluated, including Cox regression analysis, survival in 86 children (29 familial) that received HLH-94-therapy (etoposide, dexamethasone, ciclosporin) followed by allogeneic stem cell transplantation (SCT) between 1995 and 2000. The overall estimated 3-year-survival post-SCT was 64% [confidence interval (CI) = +/- 10%] (n = 86); 71 +/- 18% in those patients with a matched related donor (MRD, n 24), 70 +/- 16% with a matched unrelated donor (MUD, n = 33), 50 +/- 24% with a family haploidentical donor (haploidentical, n = 16), and 54 +/- 27% with a mismatched unrelated donor (MMUD, n = 13). After adjustment for potential confounding factors, estimated odds ratios (OR) for mortality were 1.93 (CI = 0.61-6.19) for MUD, 3.31 (1.02-10.76) for haploidentical, and 3.01 (0.91-9.97) for MMUD, compared with MRD. In children with active disease after 2-months of therapy (n = 43) the OR was 2.75 (1.26-5.99), compared with inactive disease (n = 43). In children with active disease at SCT (n = 37), the OR was 1.80 (0.80-4.06) compared with inactive disease (n = 49), after adjustment for disease activity at 2-months. Mortality was predominantly transplant-related. Most HLH patients survived SCT using MRD or MUD, and survival with partially mismatched donors was also acceptable. Patients that responded well to initial pretransplant-induction therapy fared best, but some persisting HLH activity should not automatically preclude performing SCT.