Stem Cell Transplantation for Hurler

Inclusion Criteria

• •Patients with Mucopolysaccharidosis, type I (e.g., Hurler syndrome), Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or mismatched (at 1 antigen) related marrow, PBSC, or cord blood donor.
• •Patients with Mucopolysaccharidosis, type I, Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or HLA-1 antigen mismatched unrelated marrow, PBSC, or HLA-0-2 antigen mismatched umbilical cord blood donor.
• •Patients with MPS type I, Maroteaux Lamy Syndrome (MPS VI), or mucolipidosis type II (I-cell disease) will have a mental developmental index within two standard deviations of the normal mean, as best as can be determined using Bayley scales of infant development or other standardized testing, recognizing that these may be affected by speech and/or hearing impairment.
• •Adequate organ function:
• •Cardiac: ejection fraction \>40%; no decompensated congestive heart failure or uncontrolled arrhythmia
• •Renal: serum creatinine \<2.0 mg/dl
• •Hepatic: total bilirubin \<3x Upper limits of normal transaminases \< 5.0 x Upper limits of normal
• •Signed consent.