Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Overview
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Study Type
- Study Type: Interventional
- Study Design
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: May 31, 2022
Detailed Description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Interventions
- Drug: Busulfan
- Busulfan(4 mg/kg/day,4 days)
- Drug: Cyclophosphamide
- Cyclophosphamide(50 mg/kg/day,4 days)
- Drug: Fludarabine
- Fludarabine(50 mg/m2/day,3 days)
- Drug: Thymoglobulin
- Thymoglobulin(2.5 mg/kg/day,4 days)
- Drug: cyclosporine A
- cyclosporine A
- Drug: Mycophenolate mofetil
- Mycophenolate mofetil(0.25g/day)
- Drug: Tacrolimus
- Tacrolimus
- Drug: Methotrexate
- Methotrexate
- Drug: Basiliximab
- Basiliximab
- Drug: Ruxolitinib
- Ruxolitinib
Arms, Groups and Cohorts
- Experimental: MSD-HSCT
- matched sibling donors hematopoietic stem cell transplantation
- Experimental: URD-HSCT
- unrelated donor hematopoietic stem cell transplantation
- Experimental: haplo-HSCT
- haplo-identical hematopoietic stem cell transplantation
Clinical Trial Outcome Measures
Primary Measures
- Overall survival
- Time Frame: 2 years
- 2-years overall survival
- Thalassemia-free survival
- Time Frame: 2 years
- 2-years thalassemia-free survival
Secondary Measures
- Engraftment
- Time Frame: 30 days
- Myeloid engraftment at day +30
- Transplant Related Mortality
- Time Frame: 1 year
- Transplant-related mortality by 1 year
- Cumulative Incidence of acute Graft Versus Host Disease
- Time Frame: 180 days
- Acute graft versus host disease at day +180
- Cumulative Incidence of chronic Graft Versus Host Disease
- Time Frame: 2 years
- Chronic graft versus host disease by 2 years
- Cumulative Incidence of Infectious Complications
- Time Frame: 2 years
- Cumulative incidence of bacterial, fungal and viral infections by 2 years
Participating in This Clinical Trial
Inclusion Criteria
1. Diagnosed with thalassemia major. 2. Indication of hematopoietic stem cell transplantation. 3. A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function. Exclusion Criteria:
1. Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria; 2. Uncontrolled bacterial, viral or fungal infections; 3. Any other restriction for transplantation.
Gender Eligibility: All
Minimum Age: 2 Years
Maximum Age: 20 Years
Are Healthy Volunteers Accepted: No
Investigator Details
- Lead Sponsor
- First Affiliated Hospital of Guangxi Medical University
- Collaborator
- Peking University People’s Hospital
- Provider of Information About this Clinical Study
- Principal Investigator: Yongrong Lai, Director of the Hematology Department – First Affiliated Hospital of Guangxi Medical University
- Overall Official(s)
- Yongrong Lai, MD, Principal Investigator, First Affiliated Hospital of Guangxi Medical University
- Overall Contact(s)
- Yongrong Lai, MD, +86(0771)5356510, laiyongrong@hotmail.com
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