Health TrialsbyHealthPanel

Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study

ConditionAplastic Anemias
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age6-75
SponsorShanxi Bethune Hospital

About this trial

Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

A patient age of 6-75 years

Patients diagnosed with Severe Aplastic Anemia (SAA) , Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) or Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and suitable for allo-HSCT

Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study

Disqualifiers

Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)

Patients with any conditions not suitable for the trial (investigators' decision)

Trial design

Design model

Single group

Treatments tested in this trial

  • Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.

    Procedure/Surgery

    Conditioning regimen consisted of the following1)Severe aplastic anemia(SAA):FC-ATG:Flu 30 mg/m\^2/d ×4d (- 7d- -4d), CTX 22.5 -25mg/kg/d × 4d (-7d- -4d), ATG 7.5mg in total（- 5d- -2d).2)Transfusion-dependent non-severe aplastic anemia（TD-NSAA) and Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA):BU/FC-ATG:Bu 0.8mg/kg(q6h -8d）+FC-ATG. The GVHD prophylaxis program was Application of Anti-CD25 Humanized Monoclonal Antibody 50mg (+1d,+4d);cyclosporine A + mycophenolate mofetil (MMF)+short course methotrexate (MTX).The infusion time of umbilical cord blood (UCB) stem cells in the UCB-assisted haploidentical hematopoietic stem cell transplantation (UCB-Haplo-HSCT) group: 4 hours before the infusion of peripheral blood stem cells.In the follow-up phase, outcome evaluation indicators must be collected according to the follow-up plan, ensuring data quality.

Treatment groups

110 Participants
are divided into 1 treatment group
Group A: Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment StudyExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Time to neutrophil engraftment

Neutrophil engraftment was defined as the first of three consecutive days on which the patient's absolute neutrophil count was 500/uL.

Time frame
60 days

Secondary outcomes

1

Time to platelet engraftment

It was defined as independence from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10\^9/L.

Time frame
60 days
2

Incidence of infections (including CMV and EBV incidence)

CMV DNAemia was defined as positive CMV-DNA in the blood when the copies exceeded 500 copies/ml.EBV DNAemia was defined as positive EBV-DNA in the blood when the copies exceeded 500 copies/ml.

Time frame
100 days,6 months,12 months,24 months
3

Overall survival rate (OS)

Overall survival is a secondary endpoint that will be measured as time from the start of treatment until death from any cause, or the last date the patient was known to be alive.

Time frame
24 months
4

Event-free survival (EFS)

Considering as event aGVHD, SOS, relapse and death

Time frame
24 months

Other outcomes

Sponsors and contacts

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

Shanxi Bethune Hospital

Lead sponsor

Source ClinicalTrials.gov
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