Background: Neuroblastoma (NB) is the most common extracranial solid tumor in pediatric. In highrisk NB patients, the 5-year overall survival rate (OS) remains a stark < 50 % with conventional therapies. Autologous hematopoietic stem cell transplantation with high dose chemotherapies was used in poor prognosis and high-risk patients.Today, Plerixafor is used to increase stem cells mobilization in patients who are candidates for autologous transplantation.

Objective: This study examined safety and efficacy Plerixafor is administered as a subcutaneous injection in pediatric NB patients for stem cell mobilization STUDY DESIGN: A cohort of 19 pediatric neuroblastoma (NB) patients underwent autologous hematopoietic stem cell transplantation (HSCT) between February 2017 and April 2019, receiving G-CSF mobilization only. Subsequently, 37 NB patients underwent HSCT between December 2019 and October 2023, receiving both G-CSF and plerixafor for mobilization (auto-HSCT).

Results: The final product CD34 cell dose /kg was evidently higher in combination group at 5.363 ± 4.243 vs. G-CSF group at 2.827 ± 3.586 × 106(P value= 0.001). Neutrophils and platelet engraftment were occurred sooner in combination group compared with G-CSF group. The 1-year overall survival (OS) rate for the G-CSF and G-CSF-and-plerixafor combination group was 70.8 % and 63.3 %, respectively (P = 0.874). No statistically significant difference in OS or disease-free survival (DFS) was observed between the two treatment groups.

Conclusion: The results show that plerixafor may be safe and effective in NB pediatric patients in routine clinical practice. It was well tolerated in NB patients and no specific side effects were observed. It was not associated with improved survival.

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http://dx.doi.org/10.1016/j.transci.2025.104067DOI Listing

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