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2092 Eltrombopag Plus G-CSF for Peripheral Blood Stem Cell Mobilization: Interim Analysis of a Single Arm, Phase 2 Trial

Program: Oral and Poster Abstracts
Session: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Poster I
Hematology Disease Topics & Pathways:
Clinical Practice (Health Services and Quality), Treatment Considerations, Biological therapies, Transplantation (Allogeneic and Autologous)
Saturday, December 7, 2024, 5:30 PM-7:30 PM

Ta-Chuan Yu1*, Tai-Chung Huang, MD2 and Shang-Ju Wu2*

1Department of internal medicine, National Taiwan University Hospital Yunlin Branch, Yunlin, Taiwan
2Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan

Background

Autologous hematopoietic stem cell transplantation (auto-HSCT) is the standard treatment for relapsed/refractory lymphoma. However, 9% of patients in the plerixafor era could still not obtain adequate peripheral blood stem cells (PBSC) for auto-HSCT. Thrombopoietin (TPO) receptor agonists have been shown to stimulate stem cell proliferation. This study aimed to address whether add-on eltrombopag could improve the successful rate of autologous PBSC harvest.

Methods & Materials

In this phase 2, single-arm IIT, East-Asian patients with lymphomas scheduled to receive ESHAP chemo-mobilization for PBSC harvest are enrolled. Subjects receive eltrombopag at a dose of 75 mg daily from day 6 of ESHAP until the end of PBSC harvest. The primary endpoint is the proportion of patients obtaining more than 2.0 x 106/kg CD34+ cells on the first apheresis day. Based on Simon’s two-stage design, an interim analysis was performed when 13 patients received the intervention. A total of 46 patients will be enrolled for final analysis.

Results

The interim analysis completed in May 2024, is reported here. The median age was 55 (range 22-72); DLBCL is the most common diagnosis (n=7). Twelve patients (92.3%) obtained more than 2.0 x 106/kg CD34+ cells on the first apheresis day with the add-on eltrombopag. The median CD34+ cells obtained at the first apheresis was 9.34 x 106/kg. Both results were significantly higher than those derived from the historical control data. No significant eltrombopag-related side effect was observed.

Conclusions

This interim analysis showed that adding eltrombopag to stem cell mobilization is safe and can potentially increase the amount of harvested PBSC. The efficacy and safety will be verified in the final analyses.

Disclosures: Yu: Novartis: Research Funding; Takeda: Honoraria, Speakers Bureau. Huang: Novartis: Honoraria, Speakers Bureau; Roche: Consultancy, Honoraria, Speakers Bureau; AbbVie: Consultancy, Speakers Bureau; Antengene: Consultancy, Speakers Bureau; Astra-Zeneca: Consultancy; Beigene: Consultancy; AbbVie, Antengene, Bristol-Myers Squibb, Celltrion, Hematology: Speakers Bureau; HOPE Foundation for Cancer Care, Janssen, MundiPharma, Novartis, Roche: Honoraria; NTUH Hematology Clinical Research Grant, Yonglin Foundation: Research Funding; AbbVie, Antengene, AstraZeneca, Beigene, Roche, Takeda: Consultancy; MundiPharma: Honoraria, Speakers Bureau; Takeda: Consultancy, Honoraria, Speakers Bureau; Janssen: Honoraria.

OffLabel Disclosure: This trial investigated the effect of eltrombopag on the peripheral blood stem cell mobilization and collection (off-label use)

*signifies non-member of ASH