Histiocytosis X in children: patterns of disease and results of treatment.
Histiocytosis X in children: patterns of disease and results of treatment.
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儿童组织细胞增多症 X：疾病模式和治疗结果。
DOI：
10.1002/mpo.2950110206
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发表时间：
1983
期刊：
影响因子：
--
通讯作者：
Meadows,AT
中科院分区：
文献类型：
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作者：
Matus-Ridley,M;RaneyJr,RB;Thawerani,H;Meadows,AT
本文回顾了1970 ~ 1979年费城儿童癌症研究中心收治的36例1个月~ 22岁组织细胞增生症×（H-X）患儿的病理资料和临床经过。H-X的病理亚型在31例患者中是有利的（II型），在1例患者中是不利的（I型），在4例患者中未分类，其标本仅限于皮肤活检。16例患者有局部H-X，涉及骨（14例患者）、软组织（1例患者）或仅皮肤（1例患者）;所有患者在单独手术（12例患者）、放疗（RT）（3例患者）或观察（1例患者）治疗后均存活良好; 16例患者中仅1例复发H-X。其他20例患者表现为多灶性H-X，仅累及骨骼（3例患者）;骨骼和肝脏以外的软组织（7例患者）;肝脏以外的软组织（3例患者）;包括肝脏在内的软组织（4例患者）;或软组织、骨骼和肝脏或多种药物± RT（15例患者）。20例患者中有7例在诊断后中位4年内存活且无复发。20例患者中有9例（包括3例肝功能障碍患者）对初始治疗完全有反应，但出现复发;每例患者均接受药物治疗，中位生存期为4年。其余4例患者在诊断时存在广泛疾病伴肝脏和/或造血系统功能障碍，无应答，死亡。我们的结论是：（1）多发性骨病变伴或不伴相关软组织疾病或皮肤受累的患者具有良好的前景，不需要全身化疗;（2）由于复发罕见，因此对于局限性H-X患者也不需要全身治疗;（3）药物可以使多灶性H-X患者受益，尽管最佳治疗时间尚不清楚;和（4）对治疗的良好反应表明无病生存的可能性很高。然而，诊断时的器官功能障碍是不祥的：7名肝功能障碍患者中有4名死亡，3名外周血细胞计数下降的患者也都死亡。
The pathologic materials and clinical courses of 36 children aged 1 month‐22 years, with histiocytosis × (H‐X) seen at the Philadelphia Children's Cancer Research Center from 1970 to 1979 were reviewed. The pathologic subtype of H‐X was favorable (type II) in 31 patients, unfavorable (type I) in one patient, and unclassified in four patients whose specimens were limited to a skin biopsy. Sixteen patients had localized H‐X involving bone (14 patients), soft tissue (1 patient), or skin only (1 patient); all are alive and well after treatment with surgery alone (12 patients), radiation therapy (RT) (3 patients), or observation (1 patient); only 1 of the 16 developed recurrent H‐X. The other 20 patients presented with multifocal H‐X involving the skeleton alone (3 patients); the skeleton and soft tissues other than liver (7 patients); soft tissue exclusive of the liver (3 patients); soft tissue including the liver (4 patients); or soft tissues, skeleton, and liver or multiple drugs ± RT (15 patients). Seven of the 20 patients are alive and well without recurrence at a median of 4 years after diagnosis. Nine of the 20 patients, including 3 with liver dysfunction, responded completely to initial therapy but developed recurrence; each was retreated with drugs and is alive and well at a median of 4 years. The remaining 4 patients had widespread disease with dysfunction of the liver and/or hematopoietic system at diagnosis, failed to respond, and died. We conclude that (1) patients with multiple bony lesions with or without associated soft tissue disease or skin involvement have a favorable outlook and do not require systemic chemotherapy; (2) systemic treatment also is unnecessary for patients with localized H‐X since recurrence is rare; (3) drugs can benefit patients with multifocal H‐X, although the optimal duration of therapy is unclear; and (4) favorable response to treatment indicates high probability of disease‐free survival. However, organ dysfunction at diagnosis is ominous: four of seven patients with liver dysfunction are dead, as are all three patients who prsented with peripheral blood count depression.