Hallahan A R, Carpenter P A, O'Gorman-Hughes D W, Vowels M R, Marshall G M
Department of Haematology and Oncology, Sydney Children's Hospital, Randwick, New South Wales, Australia.
J Paediatr Child Health. 1999 Feb;35(1):55-9. doi: 10.1046/j.1440-1754.1999.t01-1-00354.x.
To evaluate the clinical and diagnostic features of children presenting with haemophagocytic lymphohistiocytosis (HLH), evolution of the disease and outcomes in response to treatment.
The medical records of 12 children, aged 5 weeks to 13 years at diagnosis, with HLH managed at a single institution were reviewed.
Presenting features were fever, hepatosplenomegaly, pancytopenia and hypertriglyceridemia or hypofibrinogenemia. Nine patients (75%) developed central nervous system (CNS) disease. Only one child with CNS disease survived. Five children had complete responses to therapy (42%), but all relapsed at a median of 1.5 months after starting treatment (range 2 weeks to 5 months). Two of the children treated are long-term survivors (17%), both after allogeneic bone marrow transplantation. All deaths occurred in the context of active disease.
Haemophagocytic lymphohistiocytosis is a disease with a poor prognosis. Central nervous system complications are common and response to treatment usually is transient. This study provides support for the use of immunomodulatory therapy for remission introduction followed by consideration of allogeneic bone marrow transplantation.
评估噬血细胞性淋巴组织细胞增生症（HLH）患儿的临床及诊断特征、疾病演变过程及治疗反应。
回顾了一家机构收治的12例诊断时年龄在5周龄至13岁的HLH患儿的病历。
主要表现为发热、肝脾肿大、全血细胞减少及高甘油三酯血症或低纤维蛋白原血症。9例患者（75%）出现中枢神经系统（CNS）疾病。仅1例CNS疾病患儿存活。5例患儿治疗后完全缓解（42%），但均在开始治疗后中位1.5个月复发（范围2周-5个月）。接受治疗的患儿中有2例长期存活（17%），均在接受异基因骨髓移植后。所有死亡均发生在疾病活动期。
噬血细胞性淋巴组织细胞增生症预后不良。中枢神经系统并发症常见，治疗反应通常是短暂的。本研究支持在诱导缓解时使用免疫调节治疗，随后考虑异基因骨髓移植。
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