Transplant Outcomes in Mucopolysaccharidoses
Section snippets
HSCT for Hurler Syndrome (MPS IH)
Continuing improvement in the overall outcomes following HSCT and 25 years of experience in HSCT for Hurler syndrome points to greater familiarity and acceptance of this form of therapy.24, 26 Meanwhile transplant complications and consequent morbidity and mortality have decreased over the years. In addition, the use of cord blood as a donor source has improved access, allowed faster transplantation, and increased donor chimerism and enzyme levels. While these gains are encouraging, many
HSCT for Other Mucopolysaccharidoses
Compared to Hurler syndrome, the overall experience in the use of HSCT for treatment of other MPSs (II, III, IV, VI, and VII) is very limited (Table 1). Subtypes of MPS have many similarities yet significant clinicopathologic differences making them distinct enough to have differential outcomes after HSCT. Undoubtedly, the scientific basis for effectiveness of HSCT across MPS spectrum remains the same. However, there may be individual differences in the kinetics of cellular migration,
Conclusions
Despite their inherent limitations, HSCT and ERT are the only therapies that have been shown to provide benefit in MPS patients. HSCT involves early mortality risks and later morbidity but offers a chance for a life-time supply of donor-derived enzymes to the various organs and tissues, including, most importantly, the CNS. Thus HSCT alone is able to provide neurological and cognitive benefits. There is general consensus that the earlier a patient is diagnosed and treated, the better their
Transplant Guidelines
- 1
Patients should be evaluated expeditiously at a center familiar with transplantation for inherited metabolic disorders. HLA typing of the patient and family should be performed. Those patients found to be suitable candidates for HSCT should be evaluated and transplanted quickly to derive maximum benefit.
- 2
All patients, regardless of therapy utilized, need comprehensive evaluations for increased intracranial pressure, hearing loss, cardiac disease, neurocognitive and neurodevelopmental status, and
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