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

First Affiliated Hospital of Guangxi Medical University823 enrolled

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.

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

823

Conditions

Thalassemia Major

Interventions

BusulfanDRUG

Busulfan（4 mg/kg/day,4 days）

CyclophosphamideDRUG

Cyclophosphamide（50 mg/kg/day,4 days）

FludarabineDRUG

Fludarabine（50 mg/m2/day,3 days）

Thymoglobulin

Eligibility

Sex: ALLMin age: 2 YearsMax age: 20 Years

Medical Language ↔ Plain English

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.

Locations (1)

First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Outcomes

Primary Outcomes

Overall survival

2-years overall survival

Time frame: 2 years

Thalassemia-free survival

2-years thalassemia-free survival

Time frame: 2 years

Secondary Outcomes

Engraftment

Myeloid engraftment at day +30

Time frame: 30 days

Transplant Related Mortality

Transplant-related mortality by 2 year

Time frame: 2 year

Cumulative Incidence of acute Graft Versus Host Disease

Acute graft versus host disease at day +100

Time frame: 100 days

Cumulative Incidence of chronic Graft Versus Host Disease

Chronic graft versus host disease by 2 years

Time frame: 2 years

Cumulative Incidence of Infectious Complications

Cumulative incidence of bacterial, fungal and viral infections by 2 years

Time frame: 2 years

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.