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Background <p>Mucopolysaccharidosis (MPS) involves neurodevelopmental decline due to lysosomal dysfunction. Hematopoietic stem cell transplantation (HSCT) may modify disease progression, but subtype-specific outcomes remain unclear.</p> Methods <p>Fifty-seven MPS patients (aged 1–8&#xa0;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.</p> Results <p>HSCT significantly improved locomotor function (<i>F</i> = 111.57,&#xa0;<i>p</i> &lt; 0.001), with greatest gains in MPS III (β = 59.57) and age-dependent decline (β =  − 2.46/year). Personal-social function improved modestly (<i>F</i> = 4.44,&#xa0;<i>p</i> = 0.039), while language/eye-hand coordination showed progressive gains (<i>p</i> ≤ 0.001). Subtype influenced language (<i>F</i> = 3.75) and coordination (<i>F</i> = 2.89), with attenuated responses in MPS II. No Timepoint × Subtype interactions suggested uniform temporal effects.</p> Conclusions <p>HSCT 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.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年11月19日

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

摘要

Background

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

Methods

Fifty-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.

Results

HSCT 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.

Conclusions

HSCT 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.