Patient Case Presentation: Patient on ESA for First-Line Therapy and Not Receiving a Marked Response—What Do You Do Next, and Why?
Panelists discuss management strategies for a patient with low-risk myelodysplastic syndrome with erythropoiesis-stimulating agent (ESA) failure, weighing options between initiating luspatercept or escalating ESA dosage while emphasizing the need for thorough evaluation of reversible causes before advancing therapy.
EP: 1.Early vs Late Onset of ESA in LR-MDS: Results From Phase 3 EPO-PRETAR Trial
EP: 2.COMMANDS and ELEMENT-MDS Trials: Long-Term Analysis, Survival, Duration of Response, and Hemoglobin Levels
EP: 3.IMerge Trial
EP: 4.Debate: Optimal Initiation of ESAs, Luspatercept, and Imetelstat: Pretransfusion vs Posttransfusion
EP: 5.Debate: Inflection Point for LR-MDS Therapy: Serum EPO >200 U/L vs Serum EPO >500 U/L
EP: 6.Debate: Sequencing Therapies for LR-MDS: ESA First vs Luspatercept First
EP: 7.Patient Case Presentation: Patient Is RBC Transfusion Eligible—Would You Proceed to Transfusion or Initiate Long-Term Anemia Treatment Strategies?
EP: 8.Patient Case Presentation: Patient on ESA for First-Line Therapy and Not Receiving a Marked Response—What Do You Do Next, and Why?
The clinical scenario involved a 72-year-old woman with low-risk myelodysplastic syndrome (MDS) who had been treated with erythropoiesis-stimulating agents (ESA) at a dose of 150 µg every 2 weeks for 16 weeks without significant improvement in her anemia. Her hemoglobin remained low, dropping slightly from 8.5 to 8.3, while her neutrophil and platelet counts stayed normal. Despite the clear diagnosis of MDS, there was no mention of a full workup for common reversible causes such as iron, vitamin B12, or folic acid deficiencies. The key clinical question was how to manage the patient now that ESA therapy had failed.
One treatment option suggested was to initiate luspatercept, an agent shown to be effective in patients with lower-risk MDS who do not respond to ESA. This was considered reasonable regardless of certain missing risk markers in the case details. The alternative suggestion involved the possibility of increasing the ESA dose, as some protocols recommend escalating the dose before deeming the therapy a failure. Additionally, questions were raised about confirming the diagnosis of MDS and ensuring all reversible causes were excluded, including the rare but important copper deficiency. This highlighted the importance of a thorough diagnostic approach before moving forward with second-line therapies.
The discussion ended on a lighthearted note, emphasizing the camaraderie and collaborative spirit between the teams. Though a formal winner was declared based on some playful judging criteria, the overall consensus was that both teams performed admirably and that the real winner was the shared passion for advancing MDS care. The session closed with gratitude for the lively debate and anticipation for future discussions, underscoring the value of such exchanges in improving clinical decision-making.
