Single-dose pegylated G-CSF matches daily G-CSF for peripheral blood stem cell mobilization efficacy and safety in donors
Background
Mobilizing peripheral blood stem cells (PBSC) for transplantation typically requires daily injections of granulocyte colony-stimulating factor (G-CSF), which can be inconvenient and burdensome for healthy donors. This regimen poses a logistical challenge, potentially impacting donor recruitment and experience. Exploring alternative, less frequent dosing strategies, such as using pegylated forms of G-CSF, could significantly improve donor comfort and compliance while maintaining mobilization efficacy for hematopoietic stem cell transplantation.
Study Design
This multicenter, randomized controlled trial, conducted from May 2018 to April 2019, enrolled 192 eligible healthy donors. Donors were randomized to receive either a single injection of 12 mg Peg-G-CSF on day 1 or 10 µg/kg/d G-CSF administered daily from day 1. PBSC apheresis was performed on day 5 for all donors. The primary endpoint was the percentage of donors who successfully collected ≥4×10^6 CD34+cells/kg recipient weight after a single apheresis procedure. Safety profiles and recipient outcomes were also assessed.
Results
Single-dose Peg-G-CSF demonstrated comparable efficacy to daily G-CSF for PBSC mobilization. In the Peg-G-CSF group, 80 (83.3%) of 96 donors collected ≥4×10^6 CD34+cells/kg recipient weight, compared to 76 (79.2%) of 96 donors in the G-CSF group. The peak circulating CD34+ cell count occurred on day 5 in both treatment arms. The median yield of CD34+ cells collected via a single apheresis procedure was statistically comparable between the Peg-G-CSF and G-CSF groups. Importantly, the incidence of side effects was similar across both groups, with no significant differences observed.
Moreover, no significant differences were found in recipient engraftment,
graft-versus-host disease (GVHD), or overall survival between the two recipient cohorts. Multivariate analysis identified theCD34+ cellcount on day 5 as a significant variable influencingCD34+ cellyield.
Key Findings
- 83.3% of Peg-G-CSF donors collected
≥4×10^6 CD34+cells/kgvs. 79.2% for G-CSF. - Median
CD34+ cellyield after single apheresis was comparable between groups. - Incidence of side effects was similar, with no significant difference observed.
- No significant difference in recipient engraftment,
GVHD, or survival between groups.
Why It Matters
This study offers a significant practical improvement for peripheral blood stem cell donors, demonstrating that a single injection of Peg-G-CSF can effectively replace multiple daily injections of G-CSF. This simplified regimen could substantially enhance donor convenience and comfort, potentially increasing the pool of willing donors for hematopoietic stem cell transplantation. From a clinical translation perspective, this provides a ready-to-implement alternative protocol, as both agents are already approved and widely used. The comparable safety and efficacy profiles mean clinicians can adopt this approach with confidence, streamlining the PBSC mobilization process without compromising patient outcomes.
peg-g-csf
g-csf
stem-cell-mobilization
hematopoietic-stem-cells
clinical-trial
randomized-controlled-trial