Histiocytosis X in children: Patterns of disease and results of treatment

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Abstract
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.

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