CSF3R MUTATIONS IN CONGENITAL NEUTROPENIA, A LONG STORY: DATA FROM THE ITALIAN REGISTRY

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/l) than that of responders (218/ l, 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/l at day 90 tended to have poorer OS compared to those with an ANC of >200/l (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.

Authors

Institutions

Publication Details

Journal
EJC Paediatric Oncology
Published
2025-11-05
DOI
https://doi.org/10.1016/j.ejcped.2025.100399
Primary Topic
Cerebrospinal fluid and hydrocephalus
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

CSF3R MUTATIONS IN CONGENITAL NEUTROPENIA, A LONG STORY: DATA FROM THE ITALIAN REGISTRY

Francesco Pegoraro, Angelica Barone, Marinella Veltroni, Giorgio Costagliola et al.
EJC Paediatric Oncology
Cerebrospinal fluid and hydrocephalus
article

CSF3R MUTATIONS IN CONGENITAL NEUTROPENIA, A LONG STORY: DATA FROM THE ITALIAN REGISTRY

Francesco Pegoraro, Angelica Barone, Marinella Veltroni, Giorgio Costagliola, Francesco Saettini, Antonio Marzollo, Giovanna Giagnuolo, Laura Giunti, Maria Carla Giarratana, Marta Pillon, Maria Fay Cortella, Francesca Fioredda, Baldassarre Martire, Carlo Dufour, Elena Mastrodicasa, Agnese Roveta
article en

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/l) than that of responders (218/ l, 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/l at day 90 tended to have poorer OS compared to those with an ANC of >200/l (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.

EJC Paediatric OncologyVol. 6
University of Parma (IT), University of Padua (IT), Istituto Giannina Gaslini (IT), Ospedale di Rivoli (IT), Fondazione Matilde Tettamanti Menotti De Marchi (IT), Meyer Children's Hospital (IT), Santobono Children's Hospital (IT), Azienda Ospedaliera Universitaria Pisana (IT), University of Florence (IT), Misericordia University (US)
Openalex Percentile: Top 65%
Cerebrospinal fluid and hydrocephalus
DOIOpen Access PDFOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.