Haploidentical stem cell transplantation for paediatric patients with thalassemia major

Combined haematopoietic stem cell transplantation with haploidentical graft and cord blood for paediatric patients with thalassemia major: a single centre, prospective study (SZTM2020)

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN54428419

Enrollment

200

Registered

2020-08-10

Start date

2020-11-01

Completion date

Unknown

Last updated

2021-02-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Thalassemia major Haematological Disorders Beta thalassaemia

Interventions

Conditioning chemotherapy: 1. Cyclophosphamide (CY, 50 mg/kg/day x 2 days, day -8 to -7) 2. Busulfan (BU, 2.8-4.4 mg/kg/day x 3 days, day -6 to -4) *The

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
1. Overall survival (OS) using Kaplan-Meier estimator at 2 years 2. Thalassemia-free survival (TFS) using R at 2 years	—

Secondary

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

Measure

Time frame

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

—

Countries

China

Contacts

Public ContactUet Yu

cloveringo69@hotmail.com+86 (0)13622311888

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: Feb 4, 2026