The impact of hematopoietic stem cell transplantation (HSCT) on growth in patients diagnosed with mucopolysaccharidosis I Hurler (MPS-IH) has been historically regarded as unsatisfactory. Nevertheless, the growth patterns recorded in transplanted patients have always been compared to those of healthy children.

The objective of this study was to verify the impact of HSCT on MPS-IH long term growth achievements. The auxological data of 15 patients were assessed longitudinally and compared both to the WHO growth centiles for healthy individuals and to recently published curves of untreated MPS-IH children. Despite a progressive decrease after HSCT when estimated with reference to the WHO growth charts, median height SDS showed a progressive and statistically significant increase when comparing the stature recorded at each timepoint in our population to the curves of untreated MPS-IH individuals (from ‐0.39 SDS at t₀ to +1.35 SDS 5 years after HSCT, p value < 0.001 and to +3.67 SDS at the age of 9 years, p value < 0.0001).

In conclusion, though not efficient enough to restore a normal growth pattern in MPS-IH patients, we hereby demonstrate that HSCT positively affects growth and provides transplanted patients with a remarkable height gain compared to untreated gender- and age- matched individuals.

造血干细胞移植 (HSCT) 对诊断为粘多糖贮积症 I Hurler (MPS-IH) 的患者生长的影响历来被认为是不令人满意的。尽管如此，移植患者记录的生长模式总是与健康儿童的生长模式进行比较。

本研究的目的是验证 HSCT 对 MPS-IH 长期增长成果的影响。纵向评估了 15 名患者的生长学数据，并将其与 WHO 健康个体的生长百分位数和最近公布的未经治疗的 MPS-IH 儿童的曲线进行比较。尽管参考 WHO 生长图表估计 HSCT 后逐渐下降，但将我们人群中每个时间点记录的身高与未经治疗的 MPS-IH 个体的曲线（从 ‐0.39 SDS在t ₀ HSCT至1.35 SDS 5年后，p值<  0.001和3.67 SDS在9岁，p值<  0.0001）。

总之，虽然不足以恢复 MPS-IH 患者的正常生长模式，但我们在此证明，与未经治疗的性别和年龄匹配的个体相比，HSCT 对生长有积极影响，并为移植患者提供显着的身高增长。