Updates on risk stratification and management of lower-risk myelodysplastic syndromes/neoplasms

The majority of lower-risk myelodysplastic syndromes/neoplasms patients present with anemia. Historically, these patients were treated with erythropoiesis-stimulating agents (ESA), with modest responses. A subset of these patients with del(5q) may do better with lenalidomide. Recently, in randomized trials, luspatercept has shown better responses compared with ESAs in treatment-naive patients and imetelstat in patients refractory to ESAs. Other evaluated novel compounds (fostamatinib, H3B-880, roxadustat, pyruvate kinase receptor activator) have not yet shown meaningful efficacy. More needs to be done to improve outcomes; in pursuance of this, participation in clinical trials evaluating novel therapies should be encouraged. While lower-risk myelodysplastic syndromes/neoplasms tend to have an indolent course, a subset of them has a dismal prognosis. Improving prognostication and serial monitoring will help in identifying high-risk patients for appropriate management.

Automated summary

The majority of lower-risk myelodysplastic syndromes/neoplasms patients present with anemia. Historically, they were treated with erythropoiesis-stimulating agents (ESA) but with modest responses. Recently, luspatercept has shown better responses compared with ESAs in treatment-naive patients and imetelstat in patients refractory to ESAs. Other evaluated novel compounds have not yet shown meaningful efficacy. More needs to be done to improve outcomes, and participation in clinical trials evaluating novel therapies should be encouraged. Improving prognostication and serial monitoring will help identify high-risk patients for appropriate management.