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

Abstract

The pathologic materials and clinical courses of 36 children aged 1 month-22 years, with histiocytosis X (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 (3 patients). These 20 patients were treated with surgery alone (1 patient), RT (4 patients), 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 presented with peripheral blood count depression.

Langerhans cell histiocytosis: An exploratory epidemiologic study of 177 cases

“…No statistically significant association was found be- tween LCH and the use of antibiotics, tranquilizers, anti-is similar to that of other reported case series [21,22]. hypertensives, anti-inflammatory agents, hormones, al-Moreover, as in other reports [4][5][6], LCH cases demonlergy medication, oral contraceptives, and recreational strated a slight male predominance, and appeared to be drugs.…”

Response to initial treatment of multisystem langerhans cell histiocytosis: An important prognostic indicator

“…These factors have been successfully applied with some variations in different institutions [1][2][3][4]9,14,20]. They generally correlated with disease outcome, but failed to clearly discriminate patients with very poor prognosis.…”

“…The Histiocyte Society defined two categories for a uniform description of the disease extent, namely ''single system'' LCH (SS-LCH) and ''multisystem'' LCH (MS-LCH) [12]. SS-LCH is usually associated with a favorable prognosis [1,[13][14][15]. MS-LCH, however, has an extremely variable clinical course and unpredictable prognosis.…”

Langerhans' cell histiocytosis (Histiocytosis X): Experience at the children's hospital of philadelphia, 1970–1984

“…tients were excluded from this review because of inadequate specimens or incorrect diagnosis, as previously reported [2]. Five additional patients were excluded from analysis of treatment results because of inadequate initial radiographic studies of the bony skeleton (three patients) or lack of any follow-up information (two patients).…”