thalasemia
Conditions
Interventions
Experimental group:Paternal donor haploid hematopoietic stem cell transplantation
Control group:Patients' donors were HLA identical non consanguineous or sibling donors
Sponsors
Zhongshan Hospital Xiamen University
Eligibility
Sex/Gender
All
Age
2 Years to 14 Years
Inclusion criteria
Inclusion criteria: 1. Frequency of genetic diagnosis or blood transfusion (>4 times/year) is consistent with thalassemia major; 2. Aged 2-14 years, weight <40kg; 3. Receiving haploid hematopoietic stem cell transplantation from father as donor; 4. Receive sibling or non-related HLA fully compatible hematopoietic stem cell transplantation; 5. Volunteer to take the test, and the guardian signs the informed consent.
Exclusion criteria
Exclusion criteria: 1. Patients with severe cardiac insufficiency and ejection fraction EF lower than 55%; Or severe arrhythmia, which the investigators evaluated as intolerant to preconditioning; 2. Patients with severe pulmonary insufficiency (obstructive and/or restrictive ventilation disorders) who could not tolerate preconditioning regimen as assessed by the investigators; 3. Patients with severe liver function impairment or liver fibrosis, liver function index (ALT) more than 3 times higher than the upper limit of normal value, who could not tolerate pretreatment regimen; 4. With severe renal insufficiency, renal function index (Cr) is more than 2 times the upper limit of normal value (ULN); Or creatinine clearance Ccr was less than 50ml/min, and the investigators evaluated that the patients could not tolerate the pretreatment regimen; 5. Serious infection existed before transplantation, and researchers evaluated patients who were difficult to control and unsuitable for transplantation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall survial;Thalasemia-free survial;Implantation rate;Incidence of GVHD; | — |
Secondary
| Measure | Time frame |
|---|---|
| Complication profile;Viral reactivation rates;Transplant related mortality; | — |
Countries
China
Contacts
Public ContactLu Quanyi
Zhongshan Hospital Xiamen University
Outcome results
None listed