An interventional study of IST (ATG + CsA+TPORA) and tranplantation in Aplastic Anaemia, sponsored by Institute of Hematology & Blood Diseases Hospital, China. Not yet recruiting. Open to participants aged 14 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-01-13.
Sponsored by Institute of Hematology & Blood Diseases Hospital, China · Not applicable, Interventional, and Treatment
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.
343 studies on the registry are indexed under Anemia, Aplastic; 99 are open to participants now.
This study's planned enrollment of 116 is above the median of 39 across 268 interventional studies indexed under Anemia, Aplastic.
Browse Anemia, Aplastic studies →Institute of Hematology & Blood Diseases Hospital, China is the lead sponsor of 398 studies on the registry; 293 are open to participants now.
Counted across the registry records on this site, refreshed daily.
(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:
Procedure: tranplantation
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).
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).
Failure Free Survival, FFS
survival with complete response whereas death, graft failure and relapse are considered treatment failures
Time frame: 2 year
Overall Survival, OS
Time frame: 2 year
Treatment Related Mortality, TRM
Transplantation-related mortality (TRM) was defined as death without graft failure
Time frame: 2 year
hematology remission
hematology recovery, including WBC, Hb and PLT
Time frame: 2 year
Infection incidence
Time frame: 2 year
GVHD incidence
Time frame: 2 year
QoL evaluation
life quality assessment
Time frame: 2 year
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Nov 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Institute of Hematology & Blood Diseases Hospital, China