OBJECTIVES: I. Determine the survival at 100 days, and then at 1 and 3 years of children with lysosomal and peroxisomal inborn errors of metabolism (storage diseases) after undergoing bone marrow or umbilical cord blood transplantation. II. Determine the change in neuropsychometric function in these patients at 100 days, and then at 1, 2, and 3 years after undergoing this treatment regimen. IV. Estimate the toxicity of this treatment regimen in these patients. V. Estimate the rate of hematological donor cell engraftment with this treatment regimen in these patients. VI. Estimate the incidence of graft versus host disease in these patients after undergoing this treatment regimen.

PROTOCOL OUTLINE: Patients are stratified according to donor type (related vs unrelated) and disease status (mucopolysaccharidosis or leukodystrophy with IQ at least 80 vs leukodystrophy with IQ less than 80). Patients receive cyclophosphamide IV over 2 hours on days -6 and -5; total body irradiation on days -3 to 0; methylprednisolone IV every 12 hours on days -3 to -1 and days 5 to 56; anti-thymocyte globulin IV over 4-6 hours every 12 hours on days -3 to -1; allogeneic T-cell depleted bone marrow or umbilical cord blood transplantation on day 0; cyclosporine IV or orally on days -3 to 180, and then tapering in the absence of graft versus host disease; and filgrastim (G-CSF) IV daily starting day 1 and continuing until blood counts recover. Patients are followed at days 100 and 180, and then at 1, 2, and 3 years.

Genders Eligible for Study:  Both

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

• Patients with either of the following inborn errors of metabolism (and developmental quotients within two standard deviations of normal): Mucopolysaccharidosis I (MPS I) (Hurler disease) Mucopolysaccharidosis VI (MPS VI) (Maroteaux-Lamy syndrome)

OR

• Patients with any of the following inborn errors of metabolism (and a performance IQ greater than 45): Adrenoleukodystrophy (co-A ligase deficiency) Metachromatic leukodystrophy (arylsulfatase A deficiency) Presymptomatic late infantile, juvenile, or adult form No symptomatic late infantile form Globoid cell leukodystrophy (Krabbe's disease) (galactocerebrosidase deficiency) No symptomatic infantile form Acid lipase deficiency (Wolman disease) No evidence of hepatic cirrhosis Alpha mannosidase deficiency (mannosidosis) Fucosidase deficiency (fucosidosis) Glucocerebrosidase deficiency (Gaucher's Disease, type III) I cell disease Niemann-Pick disease
• Must have an HLA identical, haplotype mismatched (at 1-3 antigens) related bone marrow or umbilical cord blood donor OR HLA 1 antigen mismatched unrelated bone marrow or umbilical cord blood donor

--Patient Characteristics--

• Hepatic: Bilirubin and AST less than 2 times normal
• Renal: Creatinine less than 2 times normal OR Creatinine clearance at least 60 mL/min
• Cardiac: Ejection fraction greater than 30%
• Other: Negative pregnancy test