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Novel tool may better predict myelodysplastic syndrome outcomes
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A new tool that incorporates genetic and clinical variables may represent a convenient and effective method of predicting outcomes among patients with myelodysplastic syndrome, according to study results published in Mayo Clinic Proceedings.
“Myelodysplastic syndrome is one of the most frequent blood cancers affecting the elderly with annual incidence exceeding 50 cases per 100,000 in people [aged] 65 years or older,” Ayalew Tefferi, MD, hematologist in the division of hematology at Mayo Clinic in Rochester, Minnesota, said in a press release. ‘The only treatment that offers a chance for cure or prolonged survival is allogeneic hematopoietic stem cell transplant. Unfortunately, this procedure is associated with substantial risk of treatment-related death and morbidity, so an accurate and reliable prognostic tool is needed to select suitable patients for transplant.”
Tefferi and colleagues developed a novel risk model for myelodysplastic syndrome based upon mutations, karyotype and clinical variables.
The researchers assessed survival outcomes in a core cohort of 357 patients (median age, 74 years; 70% men) receiving care between 1994 and 2017 at Mayo Clinic for primary myelodysplastic syndrome. The analysis also included a validation cohort of 328 patients (median age, 66 years; 65% men) receiving care at National Taiwan University Hospital.
Results of multivariate analysis showed predictors for worse OS included monosomal karyotype (HR = 5.2; 95% CI, 3.1-8.6), non-monosomal karyotype abnormalities other than single/double del(5q) (HR = 1.8; 95% CI, 1.3-2.6), and mutations in RUNX1 (HR = 2.0; 95% CI, 1.2-3.1) and ASXL1 (HR =1.7; 95% CI, 1.2-2.3).
Further, absence of mutations in SF3B1 (HR =1.6; 95% CI, 1.1-2.4), age older than 70 years (HR = 2.2; 95% CI, 1.6-3.1), hemoglobin levels less than 8 g/dL in women or less than 9 g/dL in men (HR = 2.3; 95% CI, 1.7-3.1), platelet count less than 75 ×109/L (HR = 1.5; 95% CI, 1.1-2.1), and 10% or more bone marrow blasts (HR = 1.7; 95% CI, 1.1-2.8) also predicted poorer OS.
Based upon HR-weighted risk scores, the researchers developed a four-tiered Mayo Alliance Prognostic System for myelodysplastic syndrome. The tiers included low (n = 89), intermediate-1 (n = 104), intermediate-2 (n = 95) and high (n = 69).
Median 5-year OS was 73% among patients in the low tier, followed by 34% in intermediate-1, 7% in intermediate-2 and 0% in the high tier.
Researchers then used Akaike information criterion and area under the curve (AUC) estimates to assess their model’s performance compared with the revised International Prognostic Scoring System — the current standard for assessing myelodysplastic syndrome.
Analyses using the validation cohort showed the model demonstrated favorable Akaike information criterion (1,865 vs. 1,943) and AUC values (0.87 vs. 0.76) compared with the revised International Prognostic Scoring System.
“The new Mayo Alliance Prognostic System for myelodysplastic syndrome provides a simple to use and contemporary prognostication tool that was shown to perform equally well in an external patient cohort that is racially and geographically distinct from the core study cohort,” the researchers wrote. – by Jennifer Southall
Disclosures: The study was supported by grants from the Henry J. Predolin Foundation to Mayo Clinic, the Taiwan Ministry of Science and Technology, and the Taiwan Ministry of Health and Welfare to National Taiwan University Hospital. The researchers report no relevant financial disclosures.
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