Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

• Status: Recruiting
• Trial end date: 2023-05-31
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: First Affiliated Hospital of Guangxi Medical University
• Collaborators: Fourth Affiliated Hospital of Guangxi Medical University; Hainan General Hospital; Liuzhou General Hospital; Peking University People's Hospital; Ruijin Hospital; The 923th Hospital of People's Liberation Army; The Affiliated Hospital Of Guizhou Medical University; The First People's Hospital of Yunnan
• Treatments: Basiliximab; Busulfan; Cyclophosphamide; Cyclosporine; Cyclosporins; Fludarabine; Fludarabine phosphate; Methotrexate; Mycophenolic Acid; Tacrolimus; Thymoglobulin

Criteria

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.