Osteopetrosis
Conditions
Keywords
Osteopetrosis, HSCT
Brief summary
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.
Interventions
DRUGBusulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
For sibling full match: * Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po * Cyclophosphamide 200 mg/kg iv For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood: * Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po * Cyclophosphamide 200 mg/kg iv * ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg For haploidentical: * Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po * Cyclophosphamide 200 mg/kg iv * Fludarabine 160 mg/m\^2
PROCEDUREStem Cell Transplantation
Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source: 1. Sibling full match 2. Other related full match 3. Sibling or other related with 1 mismatch antigen 4. Cord Blood 5. Haploidentical
DRUGCyclosporin, Methotrexate (GVHD prophylaxis)
* Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po * 10 mg/m\^2 iv day +1 then 6 mg/m\^ iv day +3 and +6 (Not for UCBT)
Sponsors
Tehran University of Medical Sciences
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 5 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT | 1 year |
Secondary
| Measure | Time frame |
|---|---|
| One year overall survival after allogeneic HSCT | 1 year |
| One year Progressive Free Survival after allogeneic HSCT | 1 year |
| Transplantation Related Mortality (TRM) after allogeneic HSCT | 1 year |
| Acute and chronic GVHD rate after allogeneic HSCT | 1 year |
Countries
Iran
Contacts
Primary ContactAmir Ali Hamidieh, MD
Outcome results
None listed