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

NCT07338422 | Not Yet Recruiting DMC

• 1 other identifier: IIT2025089
• Study Type: interventional
• Target: 116
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

• Failure Free Survival, FFS

  survival with complete response whereas death, graft failure and relapse are considered treatment failures

  2 year

Secondary Outcomes (6)

• Overall Survival, OS

  2 year

• Treatment Related Mortality, TRM

  2 year

• hematology remission

  2 year

• Infection incidence

  2 year

• GVHD incidence

  2 year

Study Arms (2)

HID-HSCT

EXPERIMENTAL

Procedure: tranplantation

IST

OTHER

Procedure: IST (ATG + CsA+TPORA)

Interventions

IST (ATG + CsA+TPORA) 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).

IST

tranplantation 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).

HID-HSCT

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• (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.

You may not qualify if:

• Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;
• Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;
• Known severe allergy to ATG;
• Previous allogeneic or autologous hematopoietic stem cell transplantation;
• Previous solid organ transplantation;
• Uncontrolled infection at enrollment, or requiring mechanical ventilation or hemodynamic instability;
• 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;
• History of malignant tumors (except resected basal cell carcinoma or treated cervical carcinoma in situ);
• Psychiatric disorders or other conditions that prevent compliance with study treatment and monitoring requirements;
• Inability or unwillingness to sign the consent form;
• Other special circumstances deemed ineligible by the investigator.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Central Study Contacts

Xiaoyu Zhang

CONTACT

022-23608569; zhangxiaoyu@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2025
• First Posted: January 13, 2026
• Study Start: January 14, 2026
• Primary Completion (Estimated): September 14, 2030
• Study Completion (Estimated): September 14, 2030
• Last Updated: January 13, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share