Assessment of corpus callosum volume and attention deficits in patients with mucopolysaccharidosis

Takeaway

  • Attention and corpus callosum (CC) volumes were significantly reduced in patients (pts) with mucopolysaccharidosis type I (MPS I) compared to typically developing control pts.

Why this matters

  • MPS I is a metabolic disorder associated with attention difficulties

    • MPS IH: most severe form; early cognitive and physical decline and childhood mortality

    • MPS I attenuated (IATT): less severe form of MPS I with later onset and slower progression.

  • Smaller corpus callosum (CC) volumes have been found in a canine model of MPS I and are associated with attention in other patient populations.

  • Hematopoietic cell transplantation (HCT) is used for IH and can cause white matter (WM) disruptions; results indicate a less neurotoxic compound should be used for these pts.

  • Results also highlight the need for development of treatments to improve WM development.