Dendritic Cell Disorders: Matters of Lineage and Clinical Drug Testing in Rare Diseases

Langerhans Cell Histiocytosis with Good Response to Low-Dose Imatinib: Case Report and Literature Review

“…Multiple systems (MS-LCH) and SS-LCH with multifocal involvement or involvement of critical anatomic sites are treated with systemic therapy, including vinblastine, methotrexate, and prednisone. However, there is no consensus on the optimal treatment for adult patients [ 11 ]. Response is best assessed with positron emission tomography (PET).…”

A phase IIa study of afuresertib, an oral pan‐AKT inhibitor, in patients with Langerhans cell histiocytosis

“…Since the majority of lesions from patients with LCH harbor BRAF V600E mutations, 6,7 an attempt was made to correlate response rate with this mutation. In this study, two of the 13 patients with confirmed BRAF genotypes had detectable V600E-mutated BRAF.…”

“…Median duration on study was 7 months (range 1. [5][6][7][8][9][10][11][12][13][14]. The most frequent reasons given for treatment discontinuation were progressive disease (9), investigator decision (5), and AE (2).…”

“…6 Clinical manifestations of LCH are related to the pattern of infiltration or organ involvement. [7][8][9][10] Patients with localized (single-system) disease in "low-risk" organs typically have a good prognosis; however, multisystem disease involving "high-risk" organs (bone marrow, spleen, liver), increases the likelihood of poor outcomes and disease reactivation following initial therapy. 9 BRAF V600E activating mutations occur frequently in LCH.…”