HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS - EXPERIENCE AT 2 UK CENTERS
HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS - EXPERIENCE AT 2 UK CENTERS
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DOI：
10.1111/j.1365-2141.1994.tb05111.x
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发表时间：
1994-12-01
影响因子：
6.5
通讯作者：
PRITCHARD, J
中科院分区：
文献类型：
--
作者：
HIRST, WJR;LAYTON, DM;PRITCHARD, J
摘要噬血细胞性淋巴组织细胞增生症是一种罕见的巨噬细胞过度活化的疾病。家族性和散发性形式，这可能是感染相关的，是公认的。1985 ~ 1991年共收治HLH 23例，男12例，女11例。有8个家族性病例，定义为以前受影响的兄弟姐妹和/或历史的血缘关系，年龄3天至15个月的介绍。其余15例患者的年龄从1个月到9.5岁不等。在4例病例（EBV，2例;细小病毒B19，1例;埃可病毒II，1例）中确定了潜在的病毒触发因素，包括1例家族性病例。8例仅接受支持性治疗的患者中有6例（75%）在6个月内死亡，其中包括所有4例家族性病例。该组中的两名长期存活者年龄较大（7.5岁和8岁），并已证实或怀疑病毒相关HLH。15例患者接受足叶乙甙（150-250 mg/m2，第1-3天，每21 d）和甲泼尼龙治疗; 10例患者接受鞘内注射甲氨蝶呤。在这些病例中，有9例（60%）获得了完全（6例）或部分（3例）缓解，尽管有1例儿童出现了致命的“肿瘤溶解”综合征。治疗组的总死亡率为66.6%，在就诊时2岁以下患者中最高（75%），而在2岁以上患者中为33%。三个家族性病例中的两个和五个散发性病例中的一个复发，并在诊断后3天至20个月内死亡。仅1例家族性病例在随访2个月时存活。在剩下的5名幸存者中，2名接受了异基因骨髓移植（1名匹配相关，1名半相合），分别存活11个月和29个月。3例年龄分别为2.5岁、7.5岁和9.5岁的患者分别在11个月、20个月和25个月时保持缓解。HLH的高死亡率支持了同种异体BMT在选定病例中的作用，特别是那些有家族基础或发病时年龄小于2岁的病例。
Haemophagocytic lymphohistiocytosis (HLH) is a rare disorder of inappropriate macrophage activation. Both familial and sporadic forms, which may be infection-associated, are recognized. Between 1985 and 1991 we treated 23 cases of HLH (12 male, 11 female). There were eight familial cases, defined by a previously affected sibling and/or history of consanguinity, age 3 d to 15 months at presentation. The age of the remaining 15 cases varied from 1 month to 9.5 years. A potential viral trigger was identified in four cases (EBV, two; parvovirus B19, one; echovirus II, one) including one familial case. Six of eight (75%) patients who received supportive care alone, including all four familial cases, died within 6 months of presentation. Both long-term survivors in this group presented at an older age (7.5 and 8 years) and had proven or suspected virus-associated HLH. 15 patients were treated with etoposide (150-250 mg/m(2) days 1-3 every 21 d) and methylprednisolone; 10 patients received intrathecal methotrexate in addition. In nine (60%) of these cases a complete (six) or partial (three) response was achieved, though one child suffered a fatal 'tumour lysis' syndrome. Overall mortality in the treated group was 66.6%, being highest (75%) in patients under 2 years at presentation compared to 33% in those over 2 years. Two of three familial and one of five sporadic cases relapsed-and died 3 d to 20 months from diagnosis. Only one familial case survives at follow-up of II months. Of the five remaining survivors, two received allogeneic bone marrow transplantation (one matched related, one haploidentical) and are alive at 11 and 29 months. Three cases aged 2.5, 7.5 and 9.5 years remain in remission at 11, 20 and 25 months respectively. The high mortality of HLH supports a role for allogeneic BMT in selected cases, particularly those with a familial basis or under 2 years at presentation.