Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis.

Autor: Parekh D; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Lin H; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Batajoo A; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Peckham-Gregory E; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Karri V; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Stanton W; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Scull B; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Fleishmann R; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., El-Mallawany N; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Eckstein OS; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Prudowsky ZD; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Gulati N; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.; Department of Pediatrics, Weill Cornell Medical College, New York, New York, USA., Agrusa JE; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.; Division of Pediatric Hematology/Oncology, C.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor, Michigan, USA., Ahmed AZ; Division of Hematology/Oncology, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, Michigan, USA., Chu R; Division of Hematology/Oncology/BMT, Central Michigan University College of Medicine, Children's Hospital of Michigan, Detroit, Michigan, USA., Dietz MS; Division of Pediatric Hematology and Oncology, University of Utah, Primary Children's Hospital, Salt Lake City, Utah, USA., Goldman SC; Pediatric Hematology/Oncology, Medical City Children's Hospital, Dallas, Texas, USA., Hogarty MD; Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Imran H; Department of Pediatrics, University of South Alabama, Mobile, Alberta, USA., Intzes S; Department of Pediatric Hematology/Oncology, Sacred Heart Children's Hospital, Spokane, Washington, USA., Kim JM; Department of Pediatrics, Division of Hematology/Oncology, University of California San Diego School of Medicine, Rady Children's Hospital, San Diego, California, USA., Kopp LM; Department of Pediatrics, University of Arizona, Mel and Enid Zuckerman College of Public Health, Tucson, Arizona, USA., Levy CF; Division of Pediatric Hematology/Oncology, Department of Pediatrics, Cohen Children's Medical Center, Zucker School of Medicine at Hofstra/Northwell, Hempstead, New York, USA., Neff P; Children's Blood and Cancer Center, The University of Texas at Austin, Dell Children's Medical Center of Central Texas, Austin, Texas, USA., Pillai PM; Jack Martin Division of Pediatric Hematology-Oncology, Jack and Lucy Clark Department of Pediatrics, Mount Sinai Kravis Children's Hospital, New York, New York, USA., Sisk BA; Division of Pediatric Hematology and Oncology, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA., Schiff DE; Department of Pediatrics, Division of Hematology/Oncology, University of California San Diego School of Medicine, Rady Children's Hospital, San Diego, California, USA., Trobaugh-Lotrario AD; Department of Pediatric Hematology/Oncology, Sacred Heart Children's Hospital, Spokane, Washington, USA., Walkovich K; Division of Pediatric Hematology/Oncology, C.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor, Michigan, USA., McClain KL; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA., Allen CE; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 May; Vol. 204 (5), pp. 1888-1893. Date of Electronic Publication: 2024 Mar 19.
DOI: 10.1111/bjh.19376
Abstrakt: Over 50% of patients with systemic LCH are not cured with front-line therapies, and data to guide salvage options are limited. We describe 58 patients with LCH who were treated with clofarabine. Clofarabine monotherapy was active against LCH in this cohort, including heavily pretreated patients with a systemic objective response rate of 92.6%, higher in children (93.8%) than adults (83.3%). BRAF V600E+ variant allele frequency in peripheral blood is correlated with clinical responses. Prospective multicentre trials are warranted to determine optimal dosing, long-term efficacy, late toxicities, relative cost and patient-reported outcomes of clofarabine compared to alternative LCH salvage therapy strategies.
(© 2024 British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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