Haploidentical Hematopoietic Stem Cell Transplantation with Modified Post-transplantation Cyclophosphamide for Patients with Transfusion-dependent non-severe Aplastic Anemia/ refractory Aplastic Anemia: a prospective single-arm study

Haploidentical Hematopoietic Stem Cell Transplantation with Modified Post-transplantation Cyclophosphamide for Patients with Transfusion-dependent non-severe Aplastic Anemia/ refractory Aplastic Anemia:a prospective single-arm study

Status

Recruiting

Phases

Early Phase 1

Study type

Interventional

Source

ChiCTR

Registry ID

ChiCTR2300069540

Enrollment

Unknown

Registered

2023-03-20

Start date

2022-11-15

Completion date

Unknown

Last updated

2023-05-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transfusion-dependent non-severe Aplastic Anemia/ refractory severe Aplastic Anemia

Interventions

mPT-Cy Group:receive modified PT-Cy regimen

Eligibility

Sex/Gender

All

Age

18 Years to 70 Years

Inclusion criteria

Inclusion criteria: 1) Aged between 18 and 70 years; male or female; 2)Diagnosed with refractory/relapsed severe aplastic anemia or infusion-dependent non-severe aplastic anemia; 3)Need to receive allogeneic hematopoietic stem cell transplantation without HLA-matched sibling donors or unrelated donors 4) Normal liver and kidney function: AST / ALT = 3.0×ULN; serum bilirubin =3.0×ULN; serum creatinine = 130 µmol/L 5) General condition is acceptable, ECOG score = 2; 6) Normal cardiac function: LVEF = 50%; 7) The subjects were non-pregnant or had taken contraceptive measures during the treatment 8) Be able to understand and voluntarily provide informed consent.

Exclusion criteria

Exclusion criteria: 1) Hematopoietic stem cell transplantation complication index (HCT-CI) > 3 2) Active, uncontrolled severe infection 3) The subject had a history of other malignancies 4)Donor-specific antibody (DSA) titer =10000 5)Mental disorders that may hinder study participation 6)Pregnant or nursing women 7)Myelodysplastic syndrome (MDS), paroxysmal nocturnal hemoglobinuria (PNH) and other diseases 8)Any other condition in which the investigator considers the patient to be ineligible to participate in the study.

Design outcomes

Primary

Measure	Time frame
1-year failure-free survival rate;1-year overall survival rate;appraisal of life quality;	—

Secondary

Measure	Time frame
incidence of chronic Graft-Versus-Host Disease(cGVHD);1-year Gvhd-free relapse-free survival(GRFS);incidence of EB virus activation;incidence of cytomegalovirus(CMV) activation;Donor chimerism (STR) rate;Transplant-related mortality;Platelet implantation rate;Neutrophil implantation rate;	—

Countries

China

Contacts

Public ContactWB Liu

The First Affiliated Hospital of Zhejiang Chinese Medical University

szyyblood@163.com13454136838

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026