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.

Arnaboldi, S., Faraguna, M., Colombini, A., Sala, A., Leoni, V., Spinelli, M., et al. (2025). Case report: Successful treatment of a patient presenting with a very rare association of acute lymphoblastic leukemia and mucopolysaccharidosis type IVA. FRONTIERS IN PEDIATRICS, 13 [10.3389/fped.2025.1644720].

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

Faraguna M. C.;Bettini L. R.;Rizzari C.
2025

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.
Articolo in rivista - Articolo scientifico
acute lymphoblastic leukemia; B-lineage acute lymphoblastic leukemia; chemo-radiotherapy; enzyme replacement therapy (ERT); Morquio a syndrome; mucopolysaccaridosis;
English
4-set-2025
2025
13
1644720
open
Arnaboldi, S., Faraguna, M., Colombini, A., Sala, A., Leoni, V., Spinelli, M., et al. (2025). Case report: Successful treatment of a patient presenting with a very rare association of acute lymphoblastic leukemia and mucopolysaccharidosis type IVA. FRONTIERS IN PEDIATRICS, 13 [10.3389/fped.2025.1644720].
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/579200
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