Stem Cell Transplantation for Hurler
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring stem cell transplant, storage disease, errors of metabolism
Eligibility Criteria
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. Exclusion Criteria: Presence of major organ dysfunction (see above) Pregnancy Evidence of HIV infection or known HIV positive serology Patients or parents are psychologically incapable of undergoing BMT with associated strict isolation or documented history of medical non-compliance Patients >50 kg may be at risk for having cell doses below the goal of ≥ 10 x 106 CD 34 cells/kg and therefore will not be eligible to receive unrelated PBSCs.
Sites / Locations
- Masonic Cancer Center, University of Minnesota
Arms of the Study
Arm 1
Experimental
Treatment Arm
All patients treated with chemotherapy and transplantation.