Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation
This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this
is a new protocol to take the place of several older protocols. While survival has been
very good on the prior protocols over the past decade, incomplete engraftment has remained
somewhat problematic. Therefore, we have modified the preparative regimen somewhat to
increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that
is more immune suppressive. In addition, we have modified the supportive care regimen.
Based on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and
biomarkers of inflammation and oxidative stress for the families that consent to these
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Patients Achieving Engraftment
Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.
Paul Orchard, MD
University of Minnesota Medical Center
United States: Institutional Review Board
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