Vulvar Langerhans cell histiocytosis and thalidomide: an effective treatment option

Beguerie, Julieta Ruíz; Fernández, Julia; Stringa, Matías; Anaya, Josefa

doi:10.1111/ijd.13377

Cited by 4 publications

(2 citation statements)

References 19 publications

(24 reference statements)

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“…52 The more severe skin manifestations have shown to respond well to phototherapy, 53 low-dose methotrexate, 54 and thalidomide or lenalidomide. 55 For patients requiring systemic therapy, vinblastinebased regimens remain quite effective in the adult population, with similar outcomes to children, provided that the dose of vinblastine is capped at 10 mg to avoid the development of peripheral neuropathy. 56 However, the diminished tolerance of adults to corticosteroid therapy and vinblastine limits treatment compliance; for this reason, treatment with cytarabine or cladribine is generally preferred, 52 although BRAF inhibitors are being increasingly used in this population.…”

Section: Treatment Of Adult Lchmentioning

confidence: 99%

“…Treatment of LCH in adults generally follows similar guidelines to those recommended for children, with some modifications 52 53 low‐dose methotrexate, 54 and thalidomide or lenalidomide 55 . For patients requiring systemic therapy, vinblastine‐based regimens remain quite effective in the adult population, with similar outcomes to children, provided that the dose of vinblastine is capped at 10 mg to avoid the development of peripheral neuropathy 56 .…”

Section: Treatment Of Lchmentioning

confidence: 99%

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Clinical features and treatment of Langerhans cell histiocytosis

Rodríguez‐Galindo

2021

Acta Paediatrica

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Langerhans cell histiocytosis (LCH) is caused by the expansion of CD1a+/CD207+ cells and is characterised by a wide spectrum of organ involvement and dysfunction, affecting all ages. While almost all organs and systems can be affected, only the involvement and dysfunction of liver, spleen, and haematopoietic system influence survival. The LCH pathogenic cells are defined by universal activation of the mitogen‐activated protein kinase (MAPK) signalling pathway. The most common alteration is a somatic BRAFV600E mutation, which is present in approximately two‐thirds of the cases, followed by MAP2K1 mutations. Treatment of LCH is risk‐adapted; patients with single lesions may respond well to local treatment, whereas patients with multi‐system disease require systemic chemotherapy. While survival for patients without organ dysfunction is excellent, mortality rates for patients with organ dysfunction may reach 20%. Despite progress made in the treatment of LCH, disease reactivation rates remain above 30%, and standard second‐line treatment has yet to be established. Long‐term effects, including neuroendocrine dysfunction and neurodegeneration, represent a major challenge for survivors. Treatment with BRAF or MEK inhibitors results in immediate responses, but reactivations are very common after discontinuation. Their role as single agents and in combination with chemotherapy is being explored.

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Section: Treatment Of Adult Lchmentioning

confidence: 99%

Section: Treatment Of Lchmentioning

confidence: 99%

Clinical features and treatment of Langerhans cell histiocytosis

Rodríguez‐Galindo

2021

Acta Paediatrica

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Thalidomide

2017

Reactions Weekly

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Langerhans cell histiocytosis

Rodríguez‐Galindo

Allen

2020

Blood

212

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Langerhans cell histiocytosis (LCH) is caused by clonal expansion of myeloid precursors that differentiate into CD1a+/CD207+ cells in lesions that leads to a spectrum of organ involvement and dysfunction. The pathogenic cells are defined by constitutive activation of the MAPK signaling pathway. Treatment of LCH is risk-adapted: patients with single lesions may respond well to local treatment, whereas patients with multisystem disease require systemic therapy. Although survival rates for patients without organ dysfunction is excellent, mortality rates for patients with organ dysfunction may reach 20%. Despite progress made in the treatment of LCH, disease reactivation rates remain above 30%, and standard second-line treatment is yet to be established. Treatment failure is associated with increased risks for death and long-term morbidity, including LCH-associated neurodegeneration. Early case series report promising clinical responses in patients with relapsed and refractory LCH treated with BRAF or MEK inhibitors, although potential for this strategy to achieve cure remains uncertain.

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Vulvar Langerhans cell histiocytosis and thalidomide: an effective treatment option

Cited by 4 publications

References 19 publications

Clinical features and treatment of Langerhans cell histiocytosis

Clinical features and treatment of Langerhans cell histiocytosis

Thalidomide

Langerhans cell histiocytosis

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