Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia

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 × 10⁹/L, platelet count <20 × 10⁹/L, or reticulocyte count <20 × 10⁹/L. vSAA is defined as SAA with an absolute neutrophil count <0.2 × 10⁹/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:

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 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 the study procedures, or any condition considered by the investigator to make the patient unsuitable for study participation.