Myelodysplastic syndromes: 2021 update on diagnosis, risk stratification and management.
Guillermo Garcia-ManeroKelly Sharon ChienGuillermo Montalban BravoPublished in: American journal of hematology (2020)
Therapy is selected based on risk, transfusion needs, percent of bone marrow blasts, cytogenetic and mutational profiles, comorbidities, potential for allogeneic stem cell transplantation (alloSCT) and prior exposure to hypomethylating agents (HMA). Goals of therapy are different in lower-risk patients than in higher-risk individuals and in those with HMA failure. In lower-risk MDS, the goal is to decrease transfusion needs and transformation to higher risk disease or AML, as well as to improve survival. In higher-risk disease, the goal is to prolong survival. In 2020, we witnessed an explosion of new agents and investigational approaches. Current available therapies include growth factor support, lenalidomide, HMAs, intensive chemotherapy and alloSCT. Novel therapeutics approved in 2020 are luspatercept and the oral HMA ASTX727. At the present time, there are no approved interventions for patients with progressive or refractory disease particularly after HMA-based therapy. Options include participation in a clinical trial, cytarabine-based therapy or alloSCT.
Keyphrases
- stem cell transplantation
- growth factor
- bone marrow
- clinical trial
- high dose
- stem cells
- newly diagnosed
- randomized controlled trial
- acute myeloid leukemia
- physical activity
- squamous cell carcinoma
- mesenchymal stem cells
- cardiac surgery
- multiple sclerosis
- low dose
- radiation therapy
- small molecule
- free survival
- open label
- prognostic factors
- study protocol
- cell therapy
- double blind