Thalassemia Major
Conditions
Interventions
single arm:Deferoxamine, 40-60mg/kg
Sponsors
The First Affiliated Hospital of Guangxi Medical University
Eligibility
Sex/Gender
All
Age
2 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1)Male or female patients with ß-thalassemia major; 2)Between 2 and 18 years of age; 3)Diagnosed as iron overload; 4)The peoples general condition is acceptable, the physical condition score (ECOG score) is <= 1, with follow-up conditions.
Exclusion criteria
Exclusion criteria: 1) Clinical evidence of cardiomyopathy as shown by CMR derived LV (left ventricular) Ejection Fraction 2 times upper limit of normal - entry may be delayed until return to normal); With severe renal insufficiency, renal function index (Cr or BUN) is more than 2 times the upper limit of normal value. 2) Patients unable to undergo study assessments including MRI, e.g. who are claustrophobic to MRI, have a cardiac pacemaker, ferromagnetic metal implants other than those approved as safe for use in MR scanners (Example: some types of aneurysm clips, shrapnel), and patients who are obese (exceeding the equipment limits). 3) Clinical evidence of active hepatitis B (positive HBsAg with negative HBsAb) or hepatitis C (positive HCV antibody and detectable HCV RNA with ALT above the normal range) (Note: Hepatitis B carriers will be allowed to enter the trial.). 4) Those who refuse to participate in the screening procedures or who are unable to participate in screening procedures or who are unable to comply with requirements of the protocol. 5) There are significant conditions that interfere with participation in this study (eg poorly controlled systemic hypertension, unstable heart disease that is not controlled after standard medical treatment, systemic diseases such as cardiovascular, kidney, liver, etc.). 6)Researchers evaluate other reasons for not being selected.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| iron level of liver;iron level of Myocardium;serum ferritin;Hepcidin;GDF15;transferin;Hepatic vein occlusive disease;GVHD incidence;2-year overall survival;2-year disease-free survival;Echocardiography; | — |
Secondary
| Measure | Time frame |
|---|---|
| Endocrine disease;adverse events; | — |
Countries
China
Contacts
Public ContactLai Yongrong
The First Affiliated Hospital of Guangxi Medical University
Outcome results
None listed