These biomarkers are "widely available and easily accessible in routine diagnostic laboratories worldwide, and can be used for risk stratification, treatment selection, and patient management," they write in The Lancet Haematology.
In recent years, seven distinct risk stratification models have been proposed, some applicable to all patients with systemic mastocytosis, and some applicable only to given subgroups.
They are the Mayo Alliance Prognostic System (MAPS), the Spanish Network on Mastocytosis (REMA) scores (REMA-PFS and REMA-OS), the Clinical Risk Score (CRS), the Mutation-Adjusted Risk Score (MARS) for advanced systemic mastocytosis, and the International Prognostic Scoring Systems for non-advanced systemic mastocytosis (NonAdvSM-IPSM) and advanced systemic mastocytosis (AdvSM-IPSM).
These risk stratification models vary in their components and complexity and have not been directly compared, Dr. Alberto Orfao of the University of Salamanca in Spain and colleagues note in their paper.
They developed a global prognostic score for mastocytosis (GPSM) based on all currently available prognostic factors, and compared its ability to predict PFS (GPSM-PFS) and OS (GPSM-OS) with that of the other risk scores. They used retrospective data on 1,275 patients with systemic mastocytosis, with 422 in a discovery cohort and 853 in a validation cohort.
GPSM-PFS is based on platelet count and serum concentrations of tryptase and beta2-microglobulin while GPSM-OS is based on the integration of hemoglobin and serum alkaline phosphatase concentrations, and presence of at least one mutation in the SRSF2, ASXL1, RUNX1, or DNMT3A genes.
"Direct comparison of the GPSM scores and all pre-existing risk stratification models revealed that the GPSM-PFS and REMA-PFS models were the best predictors of PFS in systemic mastocytosis, particularly in non-advanced systemic mastocytosis," the authors report.
For OS, the AdvSM-IPSM and GPSM-OS scores were the most robust predictors of OS in the total study population and in advanced systemic mastocytosis, they say.
"Overall, our findings provide guidance on the performance of currently available prognostic scores for systemic mastocytosis, which should facilitate selection of the most informative risk models for adequate patient stratification, therapeutic decisions, and clinical management of patients, both in the real world and in future clinical trials," the authors conclude in their paper.
The author of a linked comment in the journal says the findings have "important practical implications for the routine management of patients with systemic mastocytosis. They reassure on the fact that prognostication can be accomplished in the absence of molecular information, even when only some routine laboratory parameters are available, using GPSM-PFS or AdvSM-IPSM for OS."
"Clinical research in the field of systemic mastocytosis is at a peak," writes Dr. Simona Soverini of the University of Bologna, Italy.
"Increasing treatment options is paralleled with increasing prognostication systems that have the potential to optimize treatment selection in patients with advanced systemic mastocytosis, and identify non-advanced patients at greater risk of progression and deserving of immediate therapeutic intervention. The range of prognostication systems now available is a cause for hope for patients with this neglected, under diagnosed, rare condition," she concludes.
The study had no commercial funding. Author disclosures are listed with the original article.
SOURCE: https://bit.ly/3oMYhNM and https://bit.ly/2YUrUm9 The Lancet Haematology, online January 25, 2021.
By Reuters Staff
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