Clinical, radiological and molecular responses to combination chemotherapy with <scp>MAPK</scp> pathway inhibition in relapsed and refractory Langerhans cell histiocytosis-Reference-Cited by-同舟云学术

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

Published:2024-03-19 Issue:5 Volume:204 Page:1882-1887
ISSN:0007-1048
Container-title:British Journal of Haematology
language:en
Short-container-title:Br J Haematol

Author:

Karri Vivekanudeep¹²,Lin Howard¹²,Velazquez Jessica¹²,Batajoo Akanksha¹²,Parekh Deevyashali¹²,Stanton Whitney¹²,Abhyankar Harshal¹²,El‐Mallawany Nader K.¹²,Agrusa Jennifer¹²,Eckstein Olive¹²^ORCID,Gulati Nitya³^ORCID,Schwartz Jeffrey⁴,Woods‐Swafford Wendy⁵,Boyd Jaime⁶,Saha Anikit⁷,Allen Carl E.¹²^ORCID,McClain Kenneth L.¹²^ORCID

Affiliation:

1. Texas Children's Cancer and Hematology Centers, Texas Children's Hospital Houston Texas USA

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

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

4. Studer Family Children's Hospital Pediatric Blood and Cancer Pensacola Florida USA

5. Unity Point Health, Blank Children's Hospital Cancer and Blood Disorders Clinic Des Moines Iowa USA

6. Pediatra Hematólogo y Oncólogo at Consultorios Royal Center Panamá City Panama

7. Prisma Health Cancer Institute Greenville South Carolina USA

Abstract

SummaryOptimal 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.

Funder

Leukemia and Lymphoma Society

National Institutes of Health

Publisher

Wiley

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bjh.19380

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