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

June 1, 2026 updated by: 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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

180

Phase

  • Not Applicable

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Procedure: G-CSF-Primed Peripheral Blood Stem Cell Graft
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.
Experimental: 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.
Procedure: G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation
Time Frame: 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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Peking University People's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 15, 2026

Primary Completion (Estimated)

April 15, 2027

Study Completion (Estimated)

December 31, 2029

Study Registration Dates

First Submitted

June 1, 2026

First Submitted That Met QC Criteria

June 1, 2026

First Posted (Actual)

June 5, 2026

Study Record Updates

Last Update Posted (Actual)

June 5, 2026

Last Update Submitted That Met QC Criteria

June 1, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2026PHB428-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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