Challenges in PNH and CAD Management Revealed through Educational Interventions over Three Years
Authors: Ackbarali Sumera , Ackbarali Tariqa , Kowalski Katie , D'Sa Shirley , de Castro Carlos M , Chonat Satheesh , Broome Catherine M. 
Published: December 2024

BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) and cold agglutinin disease (CAD) are rare, hemolytic anemias that present challenges in clinical practice, particularly in diagnosis and the utilization of an evolving therapeutic armamentarium. To improve clinical decision-making and understand practice barriers among clinicians who care for patients with these non-malignant hematologic disorders, educational initiatives in PNH and CAD were designed in collaboration with the National Organization for Rare Disorders (NORD).
METHODS: Between 2020 and 2023, PlatformQ Health delivered four educational activities in PNH and CAD; 3 of which were part of the first and second Annual Rare Cancers and Blood Disorders series. PNH activities: 1) 30-minute on-demand activity broadcast on OMedLive between April 2022 and April 2023, 2) 90-minute live-online activity broadcast on OMedLive between May 2022 and May 2023. CAD activities: 1) 60-minute live-online activity broadcast on OMedLive between October 2022 and October 2021, 2) 30-minute on-demand activity broadcast on OMedLive between April 2022 and April 2023. Expert hematologists led the faculty panel of each activity. Changes on knowledge/competence pre-/post-test questions were assessed. Aggregate outcomes data on clinician-reported challenges and intent to modify treatment plans post-program (5-point Likert scales) were also analyzed.
RESULTS: By the end of the enduring periods of all four activities, 1,976 clinicians participated in the education (PNH: 45% physicians, 36% nurses/advanced practitioners; CAD: 40% physicians, 39% nurses/advanced practitioners). Across programs, 21% of learners were hematology/oncology specialists while 30% were primary care clinicians, and the majority (74%) reported seeing up to 5 patients with PNH or CAD on a weekly basis. These educational activities motivated three-quarters of learners to modify management approaches or patients' treatment plans. Additionally, 78% anticipated improvements in patients' clinical outcomes. However, approximately 44% of treaters identified “lack of knowledge regarding evidence-based strategies” as the top barrier to change, indicating the ongoing need for clinician education about new data and best practices in PNH and CAD management. The second greatest barrier to change differed between PNH and CAD; “insurance, reimbursement or legal issues” for PNH treaters, “lack of time/resources to consider change” for CAD treaters. The top management challenge in PNH was “time to diagnosis” of patients (27%). However, the top management challenge in CAD changed from “lack of novel therapies” (26%) in 2020 to “time to diagnosis” (27%) in 2022. This could be explained by the paucity of targeted therapies in 2020, prior to the approval of the first CAD therapy (sutimlimab) in early 2022. Additionally, healthcare interruptions during the COVID-19 pandemic may explain diagnostic delays experienced in 2022 across all disorders. Among PNH and CAD treaters, the major diagnostic roadblock was lack of familiarity with the clinical presentation of each disorder. Most learners perceived “adhering to treatment schedules” as the greatest challenge faced by patients. Data on knowledge/competence pre-/post-test questions and qualitative responses will be shared.
CONCLUSIONS: Online PNH and CAD education, delivered between 2020 and 2023, improved clinician motivation to improve patient outcomes through the incorporation of new knowledge into clinical practice. However, outcomes from these educational initiatives also uncovered important challenges and barriers in practice. These insights necessitate ongoing clinician education and inform the clinical strategies included in future initiatives.
These activities were supported by independent educational grants from Apellis Pharmaceuticals and Sanofi.