Prognosis

The type and number of disease sites, degree of organ dysfunction, and response to treatment are the main prognostic factors. Pulmonary LCH in adolescents and adults often regresses after smoking cessation.[115]

Age ≤3 years at presentation, hepatosplenomegaly, thrombocytopenia, polyostotic disease (≥3 bones involved), risk organ involvement, and lack of response to chemotherapy within the first 6 weeks (if risk organs are involved) are associated with increased mortality.[25][26][116] Hepatosplenomegaly and thrombocytopenia are associated with bone marrow involvement at diagnosis, which carries a poorer prognosis.[43][44]

Five-year survival for patients with multisystem disease is 90% in children and 70% in adults.[11][117]

Patients with polyostotic disease are at higher risk of reactivation.[44] Reactivation of disease with skeletal recurrence and/or new bone lesions is strongly associated with presence of diabetes insipidus in children, but not in adults.[26] Presence of BRAF V600E mutation is associated with a 2-fold increased risk of disease reactivation.[16]

In one cross-sectional study, the majority of adult survivors of childhood LCH reported long-term morbidity and adverse health-related quality of life. Hypothalamic pituitary dysfunction affected 50%, cognitive dysfunction 20%, and cerebellar symptoms 17.5%.[118] Patients with LCH have often been known to experience chronic pain, fatigue, and mood disorders, which should be sought and treated.

Patients treated with BRAF inhibitors require close monitoring for the development of secondary cancers, which are associated with late mortality. These agents are established in the management of metastatic melanoma, and late sequelae of melanoma and nonmelanoma skin cancers have been reported.[119]

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