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Jul 17, 2019

Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia — Part 1: Our interview with Dr Komrokji highlights the following topics and cases from his practice.

  • Approved and emerging therapies for patients with transfusion-dependent myelodysplastic syndromes (MDS); mechanism of action of the investigational erythroid maturation agent (EMA) luspatercept (00:00)
  • Design, entry criteria and outcomes of the Phase III MEDALIST trial of luspatercept for the treatment of anemia in patients with very low-, low- or intermediate-risk MDS with ring sideroblasts who require red blood cell (RBC) transfusions (2:10)
  • Clinical experience with the EMAs luspatercept and sotatercept (6:03)
  • Potential FDA approval of luspatercept for the management of low-risk MDS (9:11)
  • Ongoing investigation of luspatercept-based strategies for MDS (12:25)
  • Investigation of venetoclax in combination with a hypomethylating agent for higher-risk MDS (16:00)
  • Role of luspatercept in myelofibrosis; predictors of benefit from luspatercept (18:24)
  • Case: A man in his mid-70s presents with fatigue and dyspnea and is diagnosed with lower-risk MDS with ring sideroblasts (21:17)
  • Initial workup and diagnosis for patients with MDS (23:58)
  • Risk stratification in MDS and therapeutic options for patients at lower versus higher risk (25:52)
  • Clinical experience with EMAs for lower-risk, RBC transfusion-dependent MDS (29:42)
  • Monitoring and management of iron overload in patients with lower-risk, RBC transfusion-dependent MDS (32:30)
  • Case: A man in his early 60s with lenalidomide-refractory, lower-risk MDS and a del(5q) mutation receives the telomerase inhibitor imetelstat on a clinical trial (36:09)
  • Case: A man in his mid-70s with postpolycythemia vera myelofibrosis with anemia and splenomegaly receives initial ruxolitinib therapy (40:49)
  • Activity and tolerability of JAK1/2 inhibitors in myelofibrosis (45:09)
  • Case: A woman in her mid-70s with myelofibrosis and a JAK2 mutation presents with progressive splenomegaly and anemia and receives ruxolitinib (48:31)
  • Novel agents and strategies under investigation for myeloproliferative neoplasms (MPNs) (50:40)
  • Incidence of IDH1/2 mutations in patients with MPNs; responses to IDH1/2 inhibitors and ongoing clinical investigations (53:13)
  • Activity of the antifibrotic immunomodulator PRM-151 in patients with myelofibrosis (56:04)
  • Common misconceptions about the use of erythropoietin-stimulating agents (57:45)
  • Perspective on the appropriate choice of therapy for patients with intermediate-2 or high-risk myelofibrosis (1:01:41)
  • Lack of correlation between JAK2 mutation status and response to ruxolitinib (1:03:19)

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