Case report: Successful treatment of a patient presenting with a very rare association of acute lymphoblastic leukemia and mucopolysaccharidosis type IVA
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.
Authors
- Anna Commone
- Martha Caterina Faraguna (ORCID: https://orcid.org/0000-0003-3426-2749)
- Laura Rachele Bettini (ORCID: https://orcid.org/0000-0002-0280-1704)
- Sofia Maria Carlotta Arnaboldi
- Alessandra Sala (ORCID: https://orcid.org/0000-0003-2966-1518)
- Antonella Colombini
- Carmelo Rizzari (ORCID: https://orcid.org/0000-0002-4828-3893)
- Serena Gasperini (ORCID: https://orcid.org/0000-0001-8743-4407)
- Veronica Leoni (ORCID: https://orcid.org/0000-0001-8419-4241)
- Giacomo Gotti (ORCID: https://orcid.org/0000-0001-5024-7894)
- Viola Crescitelli
- Marco Spinelli
Institutions
- Fondazione Matilde Tettamanti Menotti De Marchi (IT)
- Azienda Ospedaliera San Gerardo (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- University of Milano-Bicocca (IT)
Publication Details
- Journal
- Frontiers in Pediatrics
- Published
- 2025-09-04
- DOI
- https://doi.org/10.3389/fped.2025.1644720
- Primary Topic
- Acute Lymphoblastic Leukemia research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ministero della Salute
- Fondazione Pierfranco e Luisa Mariani