New Paths for Targeting the Complement System: Addressing Unmet Needs in Paroxysmal Nocturnal Hemoglobinuria
PNH is a rare hematopoietic stem cell disorder that is largely undiagnosed, partially because patients experience a variety of nonspecific clinical symptoms, including fatigue, abdominal pain, and dyspnea. However, the disease is also characterized by hemolytic anemia, hemoglobinuria, thrombosis, and bone marrow failure, and it has a significant impact on patient quality of life due to transfusion dependence and hemolysis. In addition, until the advent of specific therapies for PNH, approximately 50% of patients died within 10 years, but now the survival rate is over 75%, and the therapeutic landscape is rapidly evolving. This activity, designed around group discussion led by a PNH expert, will help clinicians improve the identification and treatment of PNH through an examination of diagnostic recommendations, appropriate lab testing, and clinical trial evidence for therapeutic options within the context of patient cases.
Would you be interested in hosting a complimentary virtual Visiting Professorship Program (VPP)? Easy-to-administer one-hour independent medical education is available to your clinical team. Let us bring the expert to you virtually, we will coordinate a day and time that works for you.These interactive group discussions offer a forum to hear from an expert, interact with colleagues, share real-world experiences, review cases, have a collaborative discussion, and address key challenges clinicians face diagnosing and treating PNH.