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

ConditionAplastic Anaemia
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age14-70
SponsorInstitute of Hematology & Blood Diseases Hospital, China

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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;

Trial design

Design model

Parallel

Treatments tested in this trial

  • IST (ATG + CsA+TPORA)

    Procedure/Surgery

    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).

  • tranplantation

    Procedure/Surgery

    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).

Treatment groups

116 Participants
are divided into 2 treatment groups
Group A: HID-HSCTExperimental treatment 1 intervention
Group B: ISTOther 1 intervention

Trial outcomes

Primary outcomes

1

Failure Free Survival, FFS

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

Time frame
2 year

Secondary outcomes

1

Overall Survival, OS

Time frame
2 year
2

Treatment Related Mortality, TRM

Transplantation-related mortality (TRM) was defined as death without graft failure

Time frame
2 year
3

hematology remission

hematology recovery, including WBC, Hb and PLT

Time frame
2 year
4

Infection incidence

Time frame
2 year

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Institute of Hematology & Blood Diseases Hospital, China

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: