Reappraisal of prognostic factors used in the European Pediatric Soft Tissue Sarcoma Study Group RMS 2005 study for localized rhabdomyosarcoma to optimize risk stratification and generate a prognostic nomogram

© 2024 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society..

BACKGROUND: The objective of this study was to investigate the role of clinical factors together with FOXO1 fusion status in patients with nonmetastatic rhabdomyosarcoma (RMS) to develop a predictive model for event-free survival and provide a rationale for risk stratification in future trials.

METHODS: The authors used data from patients enrolled in the European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) RMS 2005 study (EpSSG RMS 2005; EudraCT number 2005-000217-35). The following baseline variables were considered for the multivariable model: age at diagnosis, sex, histology, primary tumor site, Intergroup Rhabdomyosarcoma Studies group, tumor size, nodal status, and FOXO1 fusion status. Main effects and significant second-order interactions of candidate predictors were included in a multiple Cox proportional hazards regression model. A nomogram was generated for predicting 5-year event-free survival (EFS) probabilities.

RESULTS: The EFS and overall survival rates at 5 years were 70.9% (95% confidence interval, 68.6%-73.1%) and 81.0% (95% confidence interval, 78.9%-82.8%), respectively. The multivariable model retained five prognostic factors, including age at diagnosis interacting with tumor size, tumor primary site, Intergroup Rhabdomyosarcoma Studies clinical group, and FOXO1 fusion status. Based on each patient's total score in the nomogram, patients were stratified into four groups. The 5-year EFS rates were 94.1%, 78.4%, 65.2%, and 52.1% in the low-risk, intermediate-risk, high-risk, and very-high-risk groups, respectively, and the corresponding 5-year overall survival rates were 97.2%, 91.5%, 74.3%, and 60.8%, respectively.

CONCLUSIONS: The results presented here provide the rationale to modify the EpSSG stratification, with the most significant change represented by the replacement of histology with fusion status. This classification was adopted in the new international trial launched by the EpSSG.

Medienart:

E-Artikel

Erscheinungsjahr:

2024

Erschienen:

2024

Enthalten in:

Zur Gesamtaufnahme - year:2024

Enthalten in:

Cancer - (2024) vom: 24. Feb.

Sprache:

Englisch

Beteiligte Personen:

De Salvo, Gian Luca [VerfasserIn]
Del Bianco, Paola [VerfasserIn]
Minard-Colin, Veronique [VerfasserIn]
Chisholm, Julia [VerfasserIn]
Jenney, Meriel [VerfasserIn]
Guillen, Gabriela [VerfasserIn]
Devalck, Christine [VerfasserIn]
Van Rijn, Rick [VerfasserIn]
Shipley, Janet [VerfasserIn]
Orbach, Daniel [VerfasserIn]
Kelsey, Anna [VerfasserIn]
Rogers, Timothy [VerfasserIn]
Guerin, Florent [VerfasserIn]
Scarzello, Giovanni [VerfasserIn]
Ferrari, Andrea [VerfasserIn]
Cesen Mazic, Maja [VerfasserIn]
Merks, Johannes H M [VerfasserIn]
Bisogno, Gianni [VerfasserIn]
European Pediatric Soft Tissue Sarcoma Study Group [VerfasserIn]

Links:

Volltext

Themen:

FOXO1 protein, human
Journal Article
Nomograms
Pediatrics
Proportional hazards models
Rhabdomyosarcoma
Survival analysis

Anmerkungen:

Date Revised 24.02.2024

published: Print-Electronic

Citation Status Publisher

doi:

10.1002/cncr.35258

funding:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

NLM368908089