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Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia (PB-Graft Haplo)

15 september 2026 uppdaterad av: Xiao-Jun Huang, Peking University People's Hospital

A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia

Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.

Traditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.

This multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.

Studieöversikt

Studietyp

Interventionell

Inskrivning (Beräknad)

180

Fas

  • Inte tillämpbar

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

    • Beijing Municipality
      • Beijing, Beijing Municipality, Kina, 100033
        • Rekrytering
        • Peking University People's Hospital
        • Kontakt:

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen

Tar emot friska volontärer

Nej

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: G-CSF-Primed Peripheral Blood Stem Cell Graft
Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, and peripheral blood stem cells will be collected by apheresis. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis.
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by peripheral blood stem cell collection by apheresis.
Experimentell: G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis.
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection.

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation
Tidsram: Up to 100 Days After Transplantation
The cumulative incidence of newly diagnosed or persistent grade II-IV acute graft-versus-host disease within 100 days after haploidentical hematopoietic stem cell transplantation. Acute graft-versus-host disease will be graded according to the MAGIC criteria and assessed based on skin, gastrointestinal, and liver manifestations. Endpoint adjudication will be performed by an independent blinded endpoint review committee using medical records, photographs, endoscopy reports, and/or biopsy reports, as applicable.
Up to 100 Days After Transplantation

Samarbetspartners och utredare

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Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

15 juni 2026

Primärt slutförande (Beräknad)

15 april 2027

Avslutad studie (Beräknad)

31 december 2029

Studieregistreringsdatum

Först inskickad

1 juni 2026

Först inskickad som uppfyllde QC-kriterierna

1 juni 2026

Första postat (Faktisk)

5 juni 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

16 september 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

15 september 2026

Senast verifierad

1 maj 2026

Mer information

Termer relaterade till denna studie

Läkemedels- och apparatinformation, studiedokument

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