Jordan M B, Filipovich A H
Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH 45229-3039, USA.
Bone Marrow Transplant. 2008 Oct;42(7):433-7. doi: 10.1038/bmt.2008.232. Epub 2008 Aug 4.
Hemophagocytic lymphohistiocytosis (HLH) is a rare, highly fatal disorder of uncontrolled inflammation, usually affecting infants. Significant progress in the treatment of this disorder has been achieved during the last decade, and outcomes for larger series of patients have been reported in recent years. Although medical therapy has advanced, hematopoietic cell transplantation remains the only curative therapy for patients with the familial form of this disorder. Unfortunately, these patients have demonstrated relatively poor post-transplant outcomes for a nonmalignant disorder, with approximately 30% mortality in the first 100 days. Early deaths were attributable to infection, GVHD, and unusually high rates of primary nonengraftment, venoocclusive disease and pneumonitis. In addition, a significant number of deaths were due to HLH reactivation, a unique complication seen in this patient group. In contrast, late complications were relatively infrequent and essentially all patients with durable engraftment remained in remission indefinitely. In this review, we will discuss recent progress in the transplant management of patients with HLH and potential future strategies, including the use of reduced intensity conditioning regimens.
噬血细胞性淋巴组织细胞增生症（HLH）是一种罕见的、高度致命的、炎症失控性疾病，通常影响婴儿。在过去十年中，这种疾病的治疗取得了重大进展，近年来已有更多患者系列的治疗结果报告。尽管药物治疗有所进步，但造血细胞移植仍然是家族性HLH患者的唯一治愈性疗法。不幸的是，对于一种非恶性疾病而言，这些患者移植后的结果相对较差，在移植后的前100天内死亡率约为30%。早期死亡归因于感染、移植物抗宿主病（GVHD）以及原发性植入失败、静脉闭塞性疾病和肺炎的异常高发生率。此外，相当数量的死亡是由于HLH复发，这是该患者群体中出现的一种独特并发症。相比之下，晚期并发症相对较少，基本上所有植入持久的患者都能无限期地保持缓解状态。在本综述中，我们将讨论HLH患者移植管理的最新进展以及潜在的未来策略，包括使用减低强度预处理方案。
