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Clinical Trials/NCT07338422
NCT07338422
Not yet recruiting
Not Applicable

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

Institute of Hematology & Blood Diseases Hospital, China0 sites116 target enrollmentStarted: January 14, 2026Last updated:
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InterventionsIST (ATG + CsA+TPORA)tranplantation

Overview

Phase
Not Applicable
Status
Not yet recruiting
Enrollment
116
Primary Endpoint
Failure Free Survival, FFS
OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Overview

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.

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
14 Years to 70 Years (Child, Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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

  • •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;

Arms & Interventions

IST

Other

Intervention: IST (ATG + CsA+TPORA) (Procedure)

HID-HSCT

Experimental

Intervention: tranplantation (Procedure)

Outcomes

Primary Outcomes

Failure Free Survival, FFS

Time Frame: 2 year

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

Secondary Outcomes

  • Overall Survival, OS(2 year)
  • Treatment Related Mortality, TRM(2 year)
  • hematology remission(2 year)
  • Infection incidence(2 year)
  • GVHD incidence(2 year)
  • QoL evaluation(2 year)

Investigators

Sponsor Class
Other
Responsible Party
Sponsor

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