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Clinical, radiological and molecular responses to combination chemotherapy with MAPK pathway inhibition in relapsed and refractory Langerhans cell histiocytosis.
Karri, Vivekanudeep; Lin, Howard; Velazquez, Jessica; Batajoo, Akanksha; Parekh, Deevyashali; Stanton, Whitney; Abhyankar, Harshal; El-Mallawany, Nader K; Agrusa, Jennifer; Eckstein, Olive; Gulati, Nitya; Schwartz, Jeffrey; Woods-Swafford, Wendy; Boyd, Jaime; Saha, Anikit; Allen, Carl E; McClain, Kenneth L.
Afiliación
  • Karri V; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Lin H; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Velazquez J; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Batajoo A; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Parekh D; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Stanton W; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Abhyankar H; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • El-Mallawany NK; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Agrusa J; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Eckstein O; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Gulati N; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Schwartz J; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Woods-Swafford W; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Boyd J; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • Saha A; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
  • Allen CE; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
  • McClain KL; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.
Br J Haematol ; 204(5): 1882-1887, 2024 May.
Article en En | MEDLINE | ID: mdl-38501390
ABSTRACT
Optimal therapeutic approaches for advanced Langerhans cell histiocytosis (LCH) are not known. We assessed the safety and efficacy of combined chemotherapy with MAPK pathway inhibition in 10 patients with refractory systemic disease and/or LCH-associated neurodegeneration. Overall response rate was 9/10 (90%) for the entire cohort 5/5 (100%) for patients with systemic disease and 6/7 (86%) for patients with central nervous system disease. BRAFV600E+ peripheral blood fraction decreased in 5/6 (83%). Toxicities included fever, skin rash, myalgias, neuropathy, cytopenias and hypocalcaemia. Prospective trials are required to optimize combination strategies, determine potential to achieve cure and compare outcomes to chemotherapy or MAPK inhibitor monotherapy.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Histiocitosis de Células de Langerhans / Sistema de Señalización de MAP Quinasas / Inhibidores de Proteínas Quinasas Límite: Female / Humans / Male Idioma: En Revista: Br J Haematol Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Imprimir
XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Histiocitosis de Células de Langerhans / Sistema de Señalización de MAP Quinasas / Inhibidores de Proteínas Quinasas Límite: Female / Humans / Male Idioma: En Revista: Br J Haematol Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido