Clinical, radiological and molecular responses to combination chemotherapy with MAPK pathway inhibition in relapsed and refractory Langerhans cell histiocytosis.

Autor: Karri V; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., Lin H; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; 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.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., Batajoo A; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; 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.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., Stanton W; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; 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.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., El-Mallawany NK; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; 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.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., Eckstein O; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., Gulati N; Department of Pediatrics, Weill Cornell Medical College, New York, New York, USA., Schwartz J; Studer Family Children's Hospital Pediatric Blood and Cancer, Pensacola, Florida, USA., Woods-Swafford W; Unity Point Health, Blank Children's Hospital Cancer and Blood Disorders Clinic, Des Moines, Iowa, USA., Boyd J; Pediatra Hematólogo y Oncólogo at Consultorios Royal Center, Panamá City, Panama., Saha A; Prisma Health Cancer Institute, Greenville, South Carolina, USA., Allen CE; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.; 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.; Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 May; Vol. 204 (5), pp. 1882-1887. Date of Electronic Publication: 2024 Mar 19.
DOI: 10.1111/bjh.19380
Abstrakt: 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.
(© 2024 British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
Externí odkaz: