Efficacy of the <scp>MEK</scp> inhibitor cobimetinib for wild‐type <i><scp>BRAF</scp></i> Erdheim‐Chester disease

Aubart, F. Cohen; Emile, Jean François; Maksud, Philippe; Galanaud, Damien; Cluzel, Philippe; Benameur, Neïla; Aumaître, O.; Amoura, Zahir; Haroche, Julien

doi:10.1111/bjh.14284

Cited by 64 publications

(30 citation statements)

References 10 publications

(20 reference statements)

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“…Cohen-Aubart et al also reported their retrospective results of cobimetinib used in monotherapy for three BRAF -wildtype patients with ECD. The three patients had a sustained metabolic response, and response was assessed with PET scan and also confirmed by decrease of creatinine and C-reactive protein levels and/or magnetic resonance imaging [46]. Although 2 of the 3 patients in this study had a MAP2K1 mutation, the exact mutation was not noted however.…”

Section: Therapeutic Efficacy Of Kinase Inhibitor Therapy In Histiocymentioning

confidence: 99%

Identification and Targeting of Kinase Alterations in Histiocytic Neoplasms

Özkaya

Doğan

Abdel‐Wahab

2017

Hematology/Oncology Clinics of North America

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Section: Therapeutic Efficacy Of Kinase Inhibitor Therapy In Histiocymentioning

confidence: 99%

Identification and Targeting of Kinase Alterations in Histiocytic Neoplasms

Özkaya

Doğan

Abdel‐Wahab

2017

Hematology/Oncology Clinics of North America

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“…Cobimetinib, a MEK inhibitor, was associated with clinical improvement in one case series. This case series described treatment response in three patients after clinical progression of disease despite initial therapy, including vemurafenib [15].…”

Section: Discussionmentioning

confidence: 95%

Erdheim-Chester disease, moving away from the orphan diseases: A case report

Stempel

Alvarez

Carpio

et al. 2017

Respiratory Medicine Case Reports

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With approximately 750 cases reported, Erdheim-Chester disease is an exceedingly rare histiocyte cell disorder. Affected sites typically include long bones, large vessels and central nervous system. However, cutaneous and pulmonary involvement can also occur. The diagnosis is ascertained by identification of foamy histiocytes positive for CD68, CD163, and factor XIIIa on immunoperoxidase staining. Recently published literature have described an association between Erdheim-Chester disease and BRAF V600E mutation. This finding prompted the investigation of therapeutic possibilities with BRAF inhibitors, successful agents against other BRAF mutation-positive diseases. Vemurafenib, a BRAF kinase inhibitor, has been shown to be effective in BRAF V600E mutation-positive malignancies, such as NSCLC and melanoma, as well as in several case reports of Erdheim-Chester disease. We report a case of Erdheim-Chester disease diagnosed at our institution, treated with vemurafenib.

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“…A case series evaluated cobimetinib in histologically proven BRAF V600E wild‐type ECD disease in patients with renal and cardiac involvement. Radiographic and laboratory remissions were achieved, with no major toxicities reported …”

Section: Introductionmentioning

confidence: 88%

“…Radiographic and laboratory remissions were achieved, with no major toxicities reported. 8 Here-in, we describe our experience with a BRAF wild type, KRAS G12S-positive ECD patient treated with cobimetinib that developed "dropped head" syndrome (DHS).…”

Section: (B) April 2018 (3 Months After Attenuated Cobimetinib Schedumentioning

confidence: 99%