Systemic therapy for the treatment of adult patients with lower-risk myelodysplastic syndromes

Guideline Objective

To provide guidance for the management of lower-risk myelodysplastic syndromes (MDS) using systemic therapy. Therapies include, but are not limited to:

• Hematopoiesis growth factors (i.e., erythropoiesis-stimulating agents [ESA] such as erythropoietin [EPO], granulocyte colony-stimulating factors [G-CSF], romiplostim, and eltrombopag)
• Lenalidomide in deletion 5q (del[5q]) MDS
• Lenalidomide in non-del(5q) MDS
• Hypomethylating agents (5-azacytidine [AZA] and decitabine [DAC])
• Iron chelation therapy
• Immunosuppressive therapy (i.e., cyclosporine [CsA] and anti-thymocyte globulin [ATG])

Patient Population

Adult patients (age ≥18 years) with lower-risk MDS, (i.e., International Prognostic Scoring System [IPSS] risk score ≤1.0, and IPSS (revised) score ≤3.5)

Intended Guideline Users

Clinicians involved in the care of patients with MDS: hematologists, medical oncologists, oncology nurses, and oncology pharmacists.

Research Questions

1. In patients with low-risk MDS, what is the efficacy of hematopoiesis-stimulating agents, thrombopoietin receptor agonists, immunomodulatory agents, hypomethylating agents, iron chelation, immunosuppressive agents, and other/novel agents?
2. What adverse events are associated with the use of hematopoiesis-stimulating agents, thrombopoietin receptor agonists, immunomodulatory agents, hypomethylating agents, iron chelation, immunosuppressive agents, and other/novel agents?
3. Which patients are more or less likely to benefit from treatment with hematopoiesis-stimulating agents, thrombopoietin receptor agonists, immunomodulatory agents, hypomethylating agents, iron chelation, immunosuppressive agents, and other/novel agents?
4. What are the optimal dose and schedule, and treatment duration for the aforementioned treatments?