Peripheral Blood Stem Cells Alone vs Bone Marrow Plus Peripheral Blood for Haploidentical Transplants in Severe Aplastic Anemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, randomized, open-label trial compares G-CSF-primed peripheral blood stem cell grafts to the combination of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells in haploidentical transplants for severe aplastic anemia. It is designed as a non-inferiority study to determine if the simpler peripheral blood-only approach provides similar outcomes with potentially lower donor burden. Eligible adults aged 18 to 40 with newly diagnosed or relapsed/severe disease and a haploidentical donor are enrolled, with safety and efficacy endpoints assessed over the follow-up period. The trial addresses whether a less invasive graft source can maintain transplant success while reducing procedural burden on donors.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Patients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria.
* SAA is defined as bone marrow cellularity \<25% and at least two of the following peripheral blood criteria: absolute neutrophil count \<0.5 × 10\^9/L, platelet count \<20 × 10\^9/L, or reticulocyte count \<20 × 10\^9/L.
* vSAA is defined as SAA with an absolute neutrophil count \<0.2 × 10\^9/L.
* Age 18 to 40 years.
* Availability of a haploidentical related donor.
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
* Adequate organ function, defined as follows:
* Cardiac function: left ventricular ejection fraction ≥50% and no severe arrhythmia.
* Hepatic function: total bilirubin ≤2 times the upper limit of normal and alanine aminotransferase/aspartate aminotransferase ≤3 times the upper limit of normal.
* Renal function: creatinine clearance ≥60 mL/min.
* No active infection.
* For women of childbearing potential, a negative pregnancy test is required.
* All patients must agree to use effective contraception during the study period.
* Written informed consent provided by the patient or legal representative.

Exclusion Criteria:

* Presence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).
* Prior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.
* Availability of an HLA-matched sibling donor.
* Active uncontrolled infection, including unresolved invasive fungal disease, active tuberculosis, sepsis, or other uncontrolled infections.
* Severe comorbidities, defined as a Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥4, or uncontrolled autoimmune disease.
* Donor-related contraindications, including donor age \<12 years or \>65 years, donor body mass index \<18 or \>35 kg/m², contraindications to anesthesia, or contraindications to bone marrow harvest.
* Psychiatric illness that prevents cooperation with study procedures, or any condition considered by the investigator to make the patient unsuitable for study participation.

Where it's running

• Peking university people's hospital
  Beijing, Beijing Municipality, China Recruiting

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.