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

Nonrandomized Controlled Study of HLA-Haploidentical Hematopoietic Stem Cell Transplantation Versus Immunosuppressive Therapy as First-Line Treatment for Severe Aplastic Anemia

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Aplastic Anaemia
• Intervention / Treatment: Procedure: tranplantation; Procedure: IST (ATG + CsA+TPORA)

• Study Type: Interventional
• Enrollment (Estimated): 116
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Xiaoyu Zhang; Phone Number: 022-23608569; Email: zhangxiaoyu@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HID-HSCT	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).
Other: IST	Procedure: 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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Failure Free Survival, FFS	survival with complete response whereas death, graft failure and relapse are considered treatment failures	2 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival, OS		2 year
Treatment Related Mortality, TRM	Transplantation-related mortality (TRM) was defined as death without graft failure	2 year
hematology remission	hematology recovery, including WBC, Hb and PLT	2 year
Infection incidence		2 year
GVHD incidence		2 year
QoL evaluation	life quality assessment	2 year

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Estimated): January 14, 2026
• Primary Completion (Estimated): September 14, 2030
• Study Completion (Estimated): September 14, 2030

Study Registration Dates

• First Submitted: September 25, 2025
• First Submitted That Met QC Criteria: January 5, 2026
• First Posted (Actual): January 13, 2026

Study Record Updates

• Last Update Posted (Actual): January 13, 2026
• Last Update Submitted That Met QC Criteria: January 5, 2026
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Hemic and Lymphatic Diseases; Anemia, Aplastic
• Other Study ID Numbers: IIT2025089

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No