HID-HSCT Versus IST as First-line Treatment for SAA

NCT ID: NCT07338422

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 116 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-14
• Study Completion Date: 2030-09-14

Brief Summary

This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.

Conditions

Aplastic Anaemia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HID-HSCT

Group Type: EXPERIMENTAL

tranplantation

Intervention Type: PROCEDURE

The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.

Patients in the transplantation group undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).

IST

Group Type: OTHER

IST (ATG + CsA+TPORA)

Intervention Type: PROCEDURE

The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.

Patients in the immunosuppressive therapy group receive a regimen comprising anti-thymocyte globulin (ATG) + cyclosporine A (CSA) + thrombopoietin receptor agonist (TPO-RA).

Interventions

IST (ATG + CsA+TPORA)

The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.

Patients in the immunosuppressive therapy group receive a regimen comprising anti-thymocyte globulin (ATG) + cyclosporine A (CSA) + thrombopoietin receptor agonist (TPO-RA).

Intervention Type: PROCEDURE

tranplantation

The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.

Patients in the transplantation group undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

• (1) Confirmed diagnosis of severe aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, and hepatitis-associated severe aplastic anemia.

(2) Age 14-70 years. (3) No HLA-matched related donor available. (4) HIV negative, HBV negative, HCV negative. (5) No absolute contraindications to transplantation or immunosuppressive therapy.

(6) Signed informed consent form must be obtained before study procedures begin; for subjects aged 18 years or older, informed consent shall be signed by the patient themselves or direct family members. Considering the patient's medical condition, if the patient's own signature is medically inadvisable, the informed consent shall be signed by a legal guardian or the patient's direct family member.

Exclusion Criteria

1. Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;

2. Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;

3. Known severe allergy to ATG;

4. Previous allogeneic or autologous hematopoietic stem cell transplantation;

5. Previous solid organ transplantation;

6. Uncontrolled infection at enrollment, or requiring mechanical ventilation or hemodynamic instability;

7. Active HIV replication at enrollment, detectable HCV antibody positivity and HCV-RNA positivity within 90 days prior to enrollment, or HBsAg positivity; known seropositivity for HIV or active hepatitis C virus;

8. History of malignant tumors (except resected basal cell carcinoma or treated cervical carcinoma in situ);

9. Psychiatric disorders or other conditions that prevent compliance with study treatment and monitoring requirements;

10. Inability or unwillingness to sign the consent form;

11. Other special circumstances deemed ineligible by the investigator.

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Xiaoyu Zhang

Role: CONTACT

zhangxiaoyu@ihcams.ac.cn

022-23608569

Other Identifiers

IIT2025089

Identifier Type: -

Identifier Source: org_study_id