Haploidentical stem cell transplantation for paediatric patients with thalassemia major

Not Applicable

• Conditions: Thalassemia major; Haematological Disorders; Beta thalassaemia

• Registration Number: ISRCTN54428419
• Lead Sponsor: Shenzhen Children's Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-08-10
• Last Update Posted: 22 February 2021
• Study Completion: 2023-08-31

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

1. Age 2 to 18 years
2. Diagnosed with thalassemia major
3. Indication of haematopoietic stem cell transplantation
4. A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function

Exclusion Criteria

1. Uncontrolled bacterial, viral, or fungal infections before transplant
2. Severe liver and heart overload: liver MR T2* value > 1.4 ms or heart MR T2* value > 10 ms
3. Any other restriction for transplant

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> 1. Overall survival (OS) using Kaplan-Meier estimator at 2 years<br> 2. Thalassemia-free survival (TFS) using R at 2 years<br>

Secondary Outcome Measures

Name	Time	Method
<br> 1. Engraftment: myeloid engraftment (absolute neutrophils count = 0.5×10^9/L for 3 consecutive days) at day +30<br> 2. Transplant-related mortality: using Kaplan-Meier estimator at 2 years<br> 3. Cumulative incidence of acute graft versus host disease (GVHD): acute GVHD by day +180. The classification of aGVHD is determined using either Glucksberg scoring system or IBMTR scoring system<br> 4. Cumulative incidence of chronic GVHD by 2 years. The classification of cGVHD is determined according to the overall severity chronic GVHD grading system<br> 5. Cumulative incidence of sinusoidal obstruction syndrome (SOS, also known as hepatic veno-occlusive disease, VOD): cumulative incidence of SOS/VOD at day +180<br> 6. Cumulative incidence of infectious complications: cumulative incidence of bacterial, fungal, and viral infections by 2 years<br>