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The Clinical and Cost-Effectiveness Analysis of Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation and Gene Editing Therapy for ß-Thalassemia Major

The Clinical and Cost-Effectiveness Analysis of Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation and Gene Editing Therapy for ß-Thalassemia Major

Status
Active, not recruiting
Phases
Unknown
Study type
Observational
Source
ChiCTR
Registry ID
ChiCTR2500100931
Enrollment
Unknown
Registered
2025-04-17
Start date
2025-05-01
Completion date
Unknown
Last updated
2025-04-21

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

Conditions

ß-Thalassemia Major

Interventions

Hematopoietic Stem Cell Transplantation Treatment Group:None
Gene Editing Therapy group:None

Sponsors

Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: 1.Diagnosed with major beta-thalassemia; 2.Underwent allogeneic hematopoietic stem cell transplantation (HSCT) for the first time (second transplants are not included in this statistical analysis) or received gene-editing therapy.

Exclusion criteria

Exclusion criteria: 1.Patients with pre-existing organ failure of the heart, liver, kidneys, etc. 2.Patients with severe infections at the time of treatment;

Design outcomes

Primary

MeasureTime frame
Overall cost;Quality-Adjusted Life Years, QALYs;—

Secondary

MeasureTime frame
Thalassaemia-free survival, TFS;Overall survival, OS;Number of hospitalizations;Outpatient follow-up;Total hospital expenses;Time allowance for dependents;—

Countries

China

Contacts

Public ContactFang Jianpei

Sun Yat-sen Memorial Hospital, Sun Yat-sen University

jpfang2005@163.com+86 20 81332009

Outcome results

None listed

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