denotes an abstract that is clinically relevant.
denotes that this is a recommended PHD Trainee Session.
denotes that this is a ticketed session.Hematology Disease Topics & Pathways:
Hemoglobinopathies, Diseases, Treatment Considerations, Metabolic Disorders
Description:
This session will provide an overview on the current evidence of different novel therapies for the management of Classical Heme Disorders including β-Thalassemia, Acute Intermittent Porphyria (AIP) and anemia in chronic kidney disease (CKD).
Dr. Volker Haase will outline mechanisms of action and pharmacologic properties of HIF-prolyl hydroxylase inhibitors (HIF-PHIs), a new class of oral anemia drugs approved for the use in patients with chronic kidney disease (CKD). He will discuss nondesirable on-target and off-target effects, cardiovascular and other safety concerns, and provide a benefit/risk-based perspective on how this new class of oral drugs might impact current management of anemia in CKD. A clinical case is presented that highlights the clinical complexities and therapeutic challenges in the management of anemia in patients with CKD.
Prof. Taher will highlight persisting unmet needs in patients with non-transfusion-dependent and transfusion-dependent β-thalassemia. His presentation will overview the current evidence base for luspatercept as a novel disease-modifying agent targeting ineffective erythropoiesis in β-thalassemia. He will also provide guidance on best-practices for practical application of luspatercept in the real world setting and identify emerging data gaps.
For Dr. Dickey’s presentation, she will review the givosiran clinical trial data and the available clinical literature on givosiran, as well as highlight important unanswered questions.
Dr. Volker Haase will outline mechanisms of action and pharmacologic properties of HIF-prolyl hydroxylase inhibitors (HIF-PHIs), a new class of oral anemia drugs approved for the use in patients with chronic kidney disease (CKD). He will discuss nondesirable on-target and off-target effects, cardiovascular and other safety concerns, and provide a benefit/risk-based perspective on how this new class of oral drugs might impact current management of anemia in CKD. A clinical case is presented that highlights the clinical complexities and therapeutic challenges in the management of anemia in patients with CKD.
Prof. Taher will highlight persisting unmet needs in patients with non-transfusion-dependent and transfusion-dependent β-thalassemia. His presentation will overview the current evidence base for luspatercept as a novel disease-modifying agent targeting ineffective erythropoiesis in β-thalassemia. He will also provide guidance on best-practices for practical application of luspatercept in the real world setting and identify emerging data gaps.
For Dr. Dickey’s presentation, she will review the givosiran clinical trial data and the available clinical literature on givosiran, as well as highlight important unanswered questions.