*This information was originally published as part of an abstract for the 66th ASH Annual Meeting*
Understanding PNH and CAD and the Unmet Patient Need
The management of paroxysmal nocturnal hemoglobinuria (PNH) and cold agglutinin disease (CAD) continues to evolve, as advances in complement inhibition and other targeted therapies reshape the treatment landscape. With new mechanisms of action emerging and updated clinical guidelines in development, health care providers face growing complexity in diagnosis, treatment selection, and patient monitoring for these rare, chronic hemolytic disorders.
Ensuring that health care providers have access to expert guidance and that patients and caregivers are equipped to participate in shared decision-making is essential to improving outcomes and advancing care for individuals living with PNH and CAD.
The Medlive Approach
To address gaps in clinical knowledge and strengthen both decision-making and patient engagement in PNH and CAD, Medlive launched a series of online continuing medical education (CME) initiatives developed in collaboration with the National Organization for Rare Disorders (NORD) and the Aplastic Anemia and MDS International Foundation (AAMDSIF).
Between 2020 and 2023, four accredited CME activities, two focused on PNH and two on CAD, were delivered through live and enduring digital formats, complemented by short micro-learning videos distributed via LinkedIn targeting NPI-verified oncologists. Each program was designed to enhance clinician competence, confidence, and readiness to apply evolving evidence-based strategies to practice.
PNH activities were a 30-minute endured CME activity launched April 2022, and a 90-minute live broadcast and subsequent endured CME activity launched May 2022. The CAD activities included a 60-minute live and endured CME activity launched October 2020, and a 30-minute endured CME activity launched April 2022. Across all sessions, expert hematologists guided discussions on diagnostic challenges, therapeutic advances, and strategies to optimize patient management.
Key Findings
- Meaningful knowledge gains: Across four accredited CME activities, average post-test scores rose from 38% to 78% for PNH and from 30% to 57% for CAD (p<0.05), demonstrating substantial gains in clinical knowledge and confidence.
- Identification of persistent practice barriers: 44% of learners cited lack of knowledge of evidence-based strategies as the top barrier to practice change, with “time to diagnosis” emerging as the leading management challenge across both PNH and CAD.
- Sustained practice impact: 77% of clinicians reported an intention to change practice following education, and more than 90% confirmed a positive impact on patient care at two-month follow-up.
Conclusion – Collaborating for Improved Patient Outcomes
The outcomes data revealed persistent challenges in recognizing and diagnosing PNH and CAD, emphasizing limited familiarity with clinical presentation and evidence-based management strategies. Findings underscore the ongoing need for targeted education to help clinicians apply evolving diagnostic criteria, interpret emerging therapeutic options, and navigate treatment sequencing in these rare disorders. By strengthening awareness, confidence, and clinical competence, education can help overcome barriers such as delayed diagnosis and uncertainty in treatment selection. Collectively, these results highlight the vital role of continuous learning in improving care coordination, optimizing management approaches, and enhancing outcomes for individuals living with PNH and CAD.
To learn more about partnering with Medlive to develop impactful CME programs, reach out via our Contact Us page.
These activities were supported by independent educational grants from Apellis Pharmaceuticals, Inc. and Sanofi Genzyme.
