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 Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
10
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 * Antithymocyte globulin (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
* Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in peripheral blood stem cell transplant (PBSCT) and +11 in Bone marrow transplantation or Umbilical Cord Blood (UCB) transplantation) 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)
Hematology-Oncology & SCT Research Center
Teharn, Tehran Province, Iran
RECRUITINGOverall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)
Time frame: 1 year
One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT)
Time frame: 1 year
One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT)
Time frame: 1 year
Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT)
Time frame: 1 year
Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT)
Time frame: 1 year
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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