Proposed global prognostic score for systemic mastocytosis: a retrospective prognostic modelling study.
Adult
Aged
Alkaline Phosphatase
/ blood
Core Binding Factor Alpha 2 Subunit
/ genetics
Female
Hemoglobins
/ analysis
Humans
Kaplan-Meier Estimate
Male
Mastocytosis, Systemic
/ diagnosis
Middle Aged
Polymorphism, Single Nucleotide
Prognosis
Progression-Free Survival
Repressor Proteins
/ genetics
Retrospective Studies
Risk Factors
Serine-Arginine Splicing Factors
/ genetics
Journal
The Lancet. Haematology
ISSN: 2352-3026
Titre abrégé: Lancet Haematol
Pays: England
ID NLM: 101643584
Informations de publication
Date de publication:
Mar 2021
Mar 2021
Historique:
received:
08
08
2020
revised:
20
11
2020
accepted:
26
11
2020
pubmed:
29
1
2021
medline:
10
3
2021
entrez:
28
1
2021
Statut:
ppublish
Résumé
Several risk stratification models have been proposed in recent years for systemic mastocytosis but have not been directly compared. Here we designed and validated a risk stratification model for progression-free survival (PFS) and overall survival (OS) in systemic mastocytosis on the basis of all currently available prognostic factors, and compared its predictive capacity for patient outcome with that of other risk scores. We did a retrospective prognostic modelling study based on patients diagnosed with systemic mastocytosis between March 1, 1983, and Oct 11, 2019. In a discovery cohort of 422 patients from centres of the Spanish Network on Mastocytosis (REMA), we evaluated previously identified, independent prognostic features for prognostic effect on PFS and OS by multivariable analysis, and designed a global prognostic score for mastocytosis (GPSM) aimed at predicting PFS (GPSM-PFS) and OS (GPSM-OS) by including only those variables that showed independent prognostic value (p<0·05). The GPSM scores were validated in an independent cohort of 853 patients from centres in Europe and the USA, and compared with pre-existing risk models in the total patient series (n=1275), with use of Harrells' concordance index (C-index) as a readout of the ability of each model to risk-stratify patients according to survival outcomes. Our GPSM-PFS and GPSM-OS models were based on unique combinations of independent prognostic factors for PFS (platelet count ≤100 × 10 All evaluated risk classifications predicted survival outcomes in systemic mastocytosis. The REMA-PFS and GPSM-PFS models for PFS, and the International Prognostic Scoring System for advanced systemic mastocytosis and GPSM-OS model for OS emerged as the most accurate models, indicating that robust prognostication might be prospectively achieved on the basis of biomarkers that are accessible in diagnostic laboratories worldwide. Carlos III Health Institute, European Regional Development Fund, Spanish Association of Mastocytosis and Related Diseases, Rare Diseases Strategy of the Spanish National Health System, Junta of Castile and León, Charles and Ann Johnson Foundation, Stanford Cancer Institute Innovation Fund, Austrian Science Fund.
Sections du résumé
BACKGROUND
BACKGROUND
Several risk stratification models have been proposed in recent years for systemic mastocytosis but have not been directly compared. Here we designed and validated a risk stratification model for progression-free survival (PFS) and overall survival (OS) in systemic mastocytosis on the basis of all currently available prognostic factors, and compared its predictive capacity for patient outcome with that of other risk scores.
METHODS
METHODS
We did a retrospective prognostic modelling study based on patients diagnosed with systemic mastocytosis between March 1, 1983, and Oct 11, 2019. In a discovery cohort of 422 patients from centres of the Spanish Network on Mastocytosis (REMA), we evaluated previously identified, independent prognostic features for prognostic effect on PFS and OS by multivariable analysis, and designed a global prognostic score for mastocytosis (GPSM) aimed at predicting PFS (GPSM-PFS) and OS (GPSM-OS) by including only those variables that showed independent prognostic value (p<0·05). The GPSM scores were validated in an independent cohort of 853 patients from centres in Europe and the USA, and compared with pre-existing risk models in the total patient series (n=1275), with use of Harrells' concordance index (C-index) as a readout of the ability of each model to risk-stratify patients according to survival outcomes.
FINDINGS
RESULTS
Our GPSM-PFS and GPSM-OS models were based on unique combinations of independent prognostic factors for PFS (platelet count ≤100 × 10
INTERPRETATION
CONCLUSIONS
All evaluated risk classifications predicted survival outcomes in systemic mastocytosis. The REMA-PFS and GPSM-PFS models for PFS, and the International Prognostic Scoring System for advanced systemic mastocytosis and GPSM-OS model for OS emerged as the most accurate models, indicating that robust prognostication might be prospectively achieved on the basis of biomarkers that are accessible in diagnostic laboratories worldwide.
FUNDING
BACKGROUND
Carlos III Health Institute, European Regional Development Fund, Spanish Association of Mastocytosis and Related Diseases, Rare Diseases Strategy of the Spanish National Health System, Junta of Castile and León, Charles and Ann Johnson Foundation, Stanford Cancer Institute Innovation Fund, Austrian Science Fund.
Identifiants
pubmed: 33508247
pii: S2352-3026(20)30400-2
doi: 10.1016/S2352-3026(20)30400-2
pii:
doi:
Substances chimiques
ASXL1 protein, human
0
Core Binding Factor Alpha 2 Subunit
0
Hemoglobins
0
RUNX1 protein, human
0
Repressor Proteins
0
SRSF2 protein, human
147153-65-9
Serine-Arginine Splicing Factors
170974-22-8
Alkaline Phosphatase
EC 3.1.3.1
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e194-e204Informations de copyright
Copyright © 2021 Elsevier Ltd. All rights reserved.