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Not yet recruiting NCT07338422

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

No phase Interventional Aplastic Anaemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: IST (ATG + CsA+TPORA), tranplantation.
Who it may be relevant to
Registry conditions: Aplastic Anaemia. Basic parameters: 14 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

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.

Interventions

  • 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).
  • 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).

Primary outcome measures

  • Failure Free Survival, FFS [Time frame: 2 year]
Secondary outcome measures (6)
  • Overall Survival, OS [Time frame: 2 year]
  • Treatment Related Mortality, TRM [Time frame: 2 year]
  • hematology remission [Time frame: 2 year]
  • Infection incidence [Time frame: 2 year]
  • GVHD incidence [Time frame: 2 year]
  • QoL evaluation [Time frame: 2 year]

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

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07338422 · IIT2025089

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗