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CASE REPORT article

Front. Pediatr.

Sec. Pediatric Hematology and Hematological Malignancies

Volume 13 - 2025 | doi: 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

Provisionally accepted
Sofia Maria Carlotta ArnaboldiSofia Maria Carlotta Arnaboldi1*Martha Caterina FaragunaMartha Caterina Faraguna2Antonella ColombiniAntonella Colombini2Alessandra SalaAlessandra Sala2Veronica LeoniVeronica Leoni2Marco SpinelliMarco Spinelli2Giacomo GottiGiacomo Gotti2Laura Rachele BettiniLaura Rachele Bettini2Viola CrescitelliViola Crescitelli2Anna CommoneAnna Commone2Serena GasperiniSerena Gasperini2Carmelo RizzariCarmelo Rizzari2
  • 1San Gerardo Hospital, Monza, Italy
  • 2Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy

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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.

Keywords: Acute Lymphoblastic Leukemia, Mucopolysaccaridosis, Morquio A Syndrome, B-lineage acute lymphoblastic leukemia, Enzyme replacement therapy (ERT), Chemo-radiotherapy

Received: 10 Jun 2025; Accepted: 11 Aug 2025.

Copyright: © 2025 Arnaboldi, Faraguna, Colombini, Sala, Leoni, Spinelli, Gotti, Bettini, Crescitelli, Commone, Gasperini and Rizzari. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Sofia Maria Carlotta Arnaboldi, San Gerardo Hospital, Monza, Italy

Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.

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