AUTHOR=Arnaboldi Sofia Maria Carlotta , Faraguna Martha Caterina , Colombini Antonella , Sala Alessandra , Leoni Veronica , Spinelli Marco , Gotti Giacomo , Bettini Laura Rachele , Crescitelli Viola , Commone Anna , Gasperini Serena , Rizzari Carmelo 

TITLE=Case report: Successful treatment of a patient presenting with a very rare association of acute lymphoblastic leukemia and mucopolysaccharidosis type IVA

JOURNAL=Frontiers in Pediatrics

VOLUME=Volume 13 - 2025

YEAR=2025

URL=https://www.frontiersin.org/journals/pediatrics/articles/10.3389/fped.2025.1644720

DOI=10.3389/fped.2025.1644720

ISSN=2296-2360

ABSTRACT=Treating Acute Lymphoblastic Leukemia (ALL) in patients with genetic disorders poses significant challenges for onco-hematologists. Mucopolysaccharidosis type IVA (MPS-IVA) is a lysosomal storage disorder that clinically manifests with progressive and multi-systemic comorbidities, primarily affecting the bone, cartilage, spine, heart and lungs. We report a unique case of B-lineage ALL in a patient with MPS-IVA, who was successfully cured with a personalized chemo-radiotherapy approach. The treatment strategy aimed to balance a curative-intent chemotherapy attempt with the minimization of life-threatening complications. This case highlights the importance of individualized therapy in managing ALL in the context of complex comorbidities.