Evidence map›Paper›PMID 41257753›Full record

ArticleOrphanet journal of rare diseases2025

Allogeneic hematopoietic stem cell transplantation modulates neurodevelopmental trajectories in mucopolysaccharidosis: a longitudinal study of subtype-specific outcomes and age-dependent efficacy.

Yichao Xu, Xi Fang, Chengjuan Luo, Chen Zhou, Jianmin Wang, Yunhui Mi, Jing Xie, Min Shen

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In one paragraph

Article in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

8 authors.

Yichao Xu *Department of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xi Fang *Department of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Chengjuan LuoBlood and Marrow Transplantation Center, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Chen ZhouBlood and Marrow Transplantation Center, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Jianmin WangBlood and Marrow Transplantation Center, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yunhui MiDepartment of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Jing Xie *Department of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China. Xiejing@scmc.com.cn.ORCID 0009-0003-2142-4531
Min Shen *Department of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China. Shenmin@scmc.com.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundMucopolysaccharidosis (MPS) involves neurodevelopmental decline due to lysosomal dysfunction. Hematopoietic stem cell transplantation (HSCT) may modify disease progression, but subtype-specific outcomes remain unclear.

methodsFifty-seven MPS patients (aged 1-8 years) undergoing HSCT in Shanghai (2019-2024) were assessed longitudinally using Griffiths Mental Development Scales-Chinese (GDS-C) pre-HSCT and at 3, 12, and 24 months post-HSCT. Linear mixed models evaluated timepoint, age, and subtype effects.

resultsHSCT significantly improved locomotor function (F = 111.57, p < 0.001), with greatest gains in MPS III (β = 59.57) and age-dependent decline (β =  - 2.46/year). Personal-social function improved modestly (F = 4.44, p = 0.039), while language/eye-hand coordination showed progressive gains (p ≤ 0.001). Subtype influenced language (F = 3.75) and coordination (F = 2.89), with attenuated responses in MPS II. No Timepoint × Subtype interactions suggested uniform temporal effects.

conclusionsHSCT stabilizes/improves neurodevelopment in MPS, modulated by subtype and transplant age. Early intervention optimizes outcomes, particularly for MPS III. Culturally adapted GDS-C enables precise monitoring, guiding HSCT timing and rehabilitation.

Indexed as

Hematopoietic Stem Cell TransplantationMucopolysaccharidosesChildChild, PreschoolFemaleHumansInfantLongitudinal StudiesMaleTransplantation, HomologousAge-dependent efficacyHematopoietic stem cell transplantationMucopolysaccharidosisNeurodevelopmental trajectoriesSubtype-specific outcomes

Identifiers

PMID41257753
PMCPMC12629015

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