Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis-Reference-Cited by-同舟云学术

Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis

Published:2024-03-19 Issue:5 Volume:204 Page:1888-1893
ISSN:0007-1048
Container-title:British Journal of Haematology
language:en
Short-container-title:Br J Haematol

Author:

Parekh Deevyashali¹²,Lin Howard¹²,Batajoo Akanksha¹²,Peckham‐Gregory Erin¹²,Karri Vivekanudeep¹²,Stanton Whitney¹²,Scull Brooks¹²,Fleishmann Ryan¹²,El‐Mallawany Nader¹²,Eckstein Olive S.¹²^ORCID,Prudowsky Zachary D.¹²,Gulati Nitya¹²³^ORCID,Agrusa Jennifer E.¹²⁴,Ahmed Asra Z.⁵,Chu Roland⁶,Dietz Matthew S.⁷,Goldman Stanton C.⁸,Hogarty Michael D.⁹,Imran Hamayun¹⁰,Intzes Stefanos¹¹,Kim Jenny M.¹²,Kopp Lisa M.¹³,Levy Carolyn Fein¹⁴,Neff Philip¹⁵,Pillai Pallavi M.¹⁶,Sisk Bryan A.¹⁷,Schiff Deborah E.¹²,Trobaugh‐Lotrario Angela D.¹¹,Walkovich Kelly⁴,McClain Kenneth L.¹²^ORCID,Allen Carl E.¹²^ORCID

Affiliation:

1. Texas Children's Cancer Center Texas Children's Hospital Houston Texas USA

2. Department of Pediatrics, Division of Pediatric Hematology and Oncology Baylor College of Medicine Houston Texas USA

3. Department of Pediatrics Weill Cornell Medical College New York New York USA

4. Division of Pediatric Hematology/Oncology, C.S. Mott Children's Hospital University of Michigan Medical School Ann Arbor Michigan USA

5. Division of Hematology/Oncology, Department of Internal Medicine University of Michigan Medical School Ann Arbor Michigan USA

6. Division of Hematology/Oncology/BMT Central Michigan University College of Medicine, Children's Hospital of Michigan Detroit Michigan USA

7. Division of Pediatric Hematology and Oncology University of Utah, Primary Children's Hospital Salt Lake City Utah USA

8. Pediatric Hematology/Oncology Medical City Children's Hospital Dallas Texas USA

9. Division of Oncology, Children's Hospital of Philadelphia Perelman School of Medicine at the University of Pennsylvania Philadelphia Pennsylvania USA

10. Department of Pediatrics University of South Alabama Mobile Alberta USA

11. Department of Pediatric Hematology/Oncology Sacred Heart Children's Hospital Spokane Washington USA

12. Department of Pediatrics, Division of Hematology/Oncology University of California San Diego School of Medicine, Rady Children's Hospital San Diego California USA

13. Department of Pediatrics University of Arizona, Mel and Enid Zuckerman College of Public Health Tucson Arizona USA

14. Division of Pediatric Hematology/Oncology, Department of Pediatrics, Cohen Children's Medical Center Zucker School of Medicine at Hofstra/Northwell Hempstead New York USA

15. Children's Blood and Cancer Center The University of Texas at Austin, Dell Children's Medical Center of Central Texas Austin Texas USA

16. 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

17. Division of Pediatric Hematology and Oncology Washington University in St. Louis School of Medicine St. Louis Missouri USA

Abstract

SummaryOver 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.

Funder

National Institutes of Health

St. Baldrick's Foundation

Leukemia and Lymphoma Society

Publisher

Wiley

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bjh.19376

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