Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia


Interview with Rami Komrokji, MD

Track 1: Approved and emerging therapies for patients with transfusion-dependent myelodysplastic syndromes (MDS); mechanism of action of the investigational erythroid maturation agent (EMA) luspatercept
Track 2: 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
Track 3: Clinical experience with the EMAs luspatercept and sotatercept
Track 4: Potential FDA approval of luspatercept for the management of low-risk MDS
Track 5: Ongoing investigation of luspatercept-based strategies for MDS
Track 6: Investigation of venetoclax in combination with a hypomethylating agent for higher-risk MDS
Track 7: Role of luspatercept in myelofibrosis; predictors of benefit from luspatercept
Track 8: Case: A man in his mid-70s presents with fatigue and dyspnea and is diagnosed with lower-risk MDS with ring sideroblasts
Track 9: Initial workup and diagnosis for patients with MDS
Track 10: Risk stratification in MDS and therapeutic options for patients at lower versus higher risk
Track 11: Clinical experience with EMAs for lower-risk, RBC transfusion-dependent MDS
Track 12: Monitoring and management of iron overload in patients with lower-risk, RBC transfusion-dependent MDS
Track 13: 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
Track 14: Case: A man in his mid-70s with postpolycythemia vera myelofibrosis with anemia and splenomegaly receives initial ruxolitinib therapy
Track 15: Activity and tolerability of JAK1/2 inhibitors in myelofibrosis
Track 16: Case: A woman in her mid-70s with myelofibrosis and a JAK2 mutation presents with progressive splenomegaly and anemia and receives ruxolitinib
Track 17: Novel agents and strategies under investigation for myeloproliferative neoplasms (MPNs)
Track 18: Incidence of IDH1/2 mutations in patients with MPNs; responses to IDH1/2 inhibitors and ongoing clinical investigations
Track 19: Activity of the antifibrotic immunomodulator PRM-151 in patients with myelofibrosis
Track 20: Common misconceptions about the use of erythropoietin-stimulating agents
Track 21: Perspective on the appropriate choice of therapy for patients with intermediate-2 or high-risk myelofibrosis
Track 22: Lack of correlation between JAK2 mutation status and response to ruxolitinib

Interview with Maria-Domenica Cappellini, MD

Track 1: Pathophysiology of thalassemia
Track 2: Evolution of therapies for thalassemia
Track 3: Prevalence and incidence of thalassemia
Track 4: Classification and genetic inheritance of thalassemia
Track 5: Novel treatments under investigation for thalassemia; role of gene therapy and EMAs
Track 6: Emerging data with luspatercept and sotatercept for patients with thalassemia
Track 7: Results of the Phase III BELIEVE trial evaluating luspatercept versus placebo for adult patients with beta thalassemia who require regular RBC transfusions
Track 8: Tolerability and side effects associated with luspatercept
Track 9: Ongoing Phase II BEYOND study evaluating the efficacy and safety of luspatercept in adults with beta thalassemia who are not transfusion dependent
Track 10: Activity of luspatercept in patients with anemia of chronic disease and MDS
Track 11: Effect of luspatercept on quality of life for patients with thalassemia
Track 12: Optimal selection of patients with thalassemia who would benefit from luspatercept
Track 13: Gene therapy for transfusion-dependent beta thalassemia
Track 14: Gene therapy process for patients with thalassemia
Track 15: Perspective on novel approaches to the treatment of thalassemia
 
FACULTY
 
Rami Komrokji, MD
Senior Member
Section Head, Leukemia and MDS
Vice Chair, Department of Malignant Hematology
H Lee Moffitt Cancer Center
Professor of Oncologic Sciences
University of South Florida
Tampa, Florida
 
Maria-Domenica Cappellini, MD
Professor of Internal Medicine
University of Milan
Fondazione IRCCS Ca’ Granda Ospedale Policlinico
Milan, Italy
 
EDITOR
 
Neil Love, MD
Research To Practice
Miami, Florida