Work

Csf3r Mutations in Congenital Neutropenia, a Long Story: Data From the Italian Registry

Year: 2025

Type: article

Abstract: 50% h-ATG vs. 46% r-ATG) and at last follow-up (41% vs. 39%) were comparable between two groups.The median ANC at diagnosis was lower in non-responders (70/μl) than that of responders (218/ μl, p<0.01).HSCT was performed in 59% (h-ATG) and 55% (r-ATG) of patients.Clonal evolution occurred in three patients (all r-ATG).Five-year overall survival (OS) favored h-ATG (95% vs. 85%, p=0.07), while failure-free survival was similar between two groups.Responders at 6 months had superior OS (97% vs. 79%, p<0.01).Non-responders with an ANC of <200/μl at day 90 tended to have poorer OS compared to those with an ANC of >200/μl (75% vs. 90%, p = 0.09).Conclusions: Although the response rates and FFS were similar between the two ATG groups, OS was higher in patients receiving h-ATG, indicating a potential advantage of h-ATG.Patients with persistent severe neutropenia may benefit from early HSCT.

Source: EJC Paediatric Oncology

DOI: 10.1016/j.ejcped.2025.100399