Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Malignant infantile osteopetrosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.

Inclusion Criteria:

• Diagnosis of Osteopetrosis confirm by bone biopsy and radiographic imaging
• Age up to 5 year old

Exclusion Criteria:

• Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant
• Creatinine clearance ≤ 40ml/min/1.73m^2 or RTA
• Bilirubin ≥ 3mg/dL
• SGPT ≥ 500 U/L
• Current severe infection
• Evidence of CNS involvement
• Morbidity such as blindness or deafness