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Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis.
Parekh, Deevyashali; Lin, Howard; Batajoo, Akanksha; Peckham-Gregory, Erin; Karri, Vivekanudeep; Stanton, Whitney; Scull, Brooks; Fleishmann, Ryan; El-Mallawany, Nader; Eckstein, Olive S; Prudowsky, Zachary D; Gulati, Nitya; Agrusa, Jennifer E; Ahmed, Asra Z; Chu, Roland; Dietz, Matthew S; Goldman, Stanton C; Hogarty, Michael D; Imran, Hamayun; Intzes, Stefanos; Kim, Jenny M; Kopp, Lisa M; Levy, Carolyn Fein; Neff, Philip; Pillai, Pallavi M; Sisk, Bryan A; Schiff, Deborah E; Trobaugh-Lotrario, Angela D; Walkovich, Kelly; McClain, Kenneth L; Allen, Carl E.
Afiliación
  • Parekh D; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Lin H; 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.
  • Peckham-Gregory E; 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.
  • Stanton W; 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.
  • Fleishmann R; 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.
  • Eckstein OS; 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.
  • Gulati N; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Agrusa JE; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Ahmed AZ; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Chu R; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Dietz MS; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Goldman SC; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Hogarty MD; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Imran H; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Intzes S; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Kim JM; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Kopp LM; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Levy CF; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Neff P; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Pillai PM; Department of Pediatrics, Weill Cornell Medical College, New York, New York, USA.
  • Sisk BA; Texas Children's Cancer Center, Texas Children's Hospital, Houston, Texas, USA.
  • Schiff DE; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, Texas, USA.
  • Trobaugh-Lotrario AD; Division of Pediatric Hematology/Oncology, C.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor, Michigan, USA.
  • Walkovich K; Division of Hematology/Oncology, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, Michigan, USA.
  • McClain KL; Division of Hematology/Oncology/BMT, Central Michigan University College of Medicine, Children's Hospital of Michigan, Detroit, Michigan, USA.
  • Allen CE; Division of Pediatric Hematology and Oncology, University of Utah, Primary Children's Hospital, Salt Lake City, Utah, USA.
Br J Haematol ; 204(5): 1888-1893, 2024 May.
Article en En | MEDLINE | ID: mdl-38501389
ABSTRACT
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%). BRAFV600E+ 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.
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Histiocitosis de Células de Langerhans / Clofarabina Límite: Adolescent / Adult / Aged / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged Idioma: En Revista: Br J Haematol Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Histiocitosis de Células de Langerhans / Clofarabina Límite: Adolescent / Adult / Aged / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged Idioma: En Revista: Br J Haematol Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos