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. 2014 Nov;165(5):990-6.
doi: 10.1016/j.jpeds.2014.07.063. Epub 2014 Oct 21.

Differentiating Skin-Limited and Multisystem Langerhans Cell Histiocytosis

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Free PMC article

Differentiating Skin-Limited and Multisystem Langerhans Cell Histiocytosis

Stephen J Simko et al. J Pediatr. .
Free PMC article

Abstract

Objective: To identify features associated with multisystem involvement and therapeutic failure in patients with skin Langerhans cell histiocytosis (LCH).

Study design: We reviewed medical records of 71 consecutive patients with LCH with skin involvement evaluated at Texas Children's Hospital and analyzed clinical features, laboratory results, and the presence of circulating cells with the BRAF-V600E mutation with respect to initial staging and clinical outcomes.

Results: Skin disease in patients older than 18 months of age at diagnosis was associated with the presence of multisystem disease (OR, 9.65; 95% CI, 1.17-79.4). Forty percent of patients referred for presumed skin-limited LCH had underlying multisystem involvement, one-half of these with risk-organ involvement. Patients with skin-limited LCH had a 3-year progression-free survival of 89% after initial therapy, and none developed multisystem disease. Patients with skin/multisystem involvement had a 3-year progression-free survival of 44% with vinblastine/prednisone therapy, and risk-organ involvement did not correlate with failure to achieve nonactive disease. Circulating cells with BRAF-V600E were detected at higher frequency in patients with multisystem involvement (8 of 11 skin/multisystem vs 1 of 13 skin-limited; P = .002).

Conclusion: Skin-limited LCH necessitates infrequent therapeutic intervention and has a lower risk of progression relative to skin plus multisystem LCH. The less-aggressive clinical course and lack of circulating cells with the BRAF-V600E mutation in skin-limited LCH suggest a different mechanism of disease origin compared with multisystem or risk-organ disease.

Conflict of interest statement

The authors declare no conflicts of ineterset.

Figures

Figure 1
Figure 1
A) Distribution of patient age at diagnosis. *: P<0.05 B) Onset of symptoms at birth was not significantly associated with absence of multisystem disease.
Figure 2
Figure 2
A) OS and PFS of patients with skin-limited LCH. B) Progression in skin-limited LCH versus skin plus multisystem disease. C–D) Progression for risk-organ involvement in the entire series (C) and in patients receiving uniform initial therapy (D). E) Progression requiring need for second therapy in skin plus multisystem disease versus skin-limited disease. F) BRAF-V600E mutation was detected in circulating peripheral blood cells in 8/11 patients with active skin plus multisystem LCH, but only 1/13 patients with active skin-limited LCH, and only in a low percentage of cells (P = 0.0009).
Figure 3
Figure 3
Skin LCH has variable appearance, including A) eczematous dermatitis, B) hypopigmented, eroded papules, C) hypopigmented macules, or D) and E) crusted papulonodules. Presentation does not reflect presence or absence of multisystem disease; despite similar appearance, the patient in D) had a single lesion, but the patient in E) had risk-organ involvement.

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