Online CME As a Tool to Increase Clinicians' Ability to Identify and Diagnose Paroxysmal Nocturnal Hemoglobinuria

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare clonal hematopoietic stem cell disorder characterized by episodes of hemolysis and a high risk of thrombosis [Hill A, et al. Nat Rev Dis Primers. 2017;3:17028.]. PNH has diverse clinical manifestations, which contribute to its particularly challenging diagnosis. As a result, diagnostic delays are common, and patients often experience complications of untreated PNH [Mancuso S, et al. Hematol Rep. 2018;10:7523.]. Due to the rarity and confounding presentation of PNH, many members of the healthcare team are challenged to accurately recognize signs and symptoms of the disease and implement appropriate diagnostic methods. The objective of this study was to determine if an online continuing medical education (CME) intervention could improve hematologists' and primary care physicians' (PCPs) ability in identifying and diagnosing patients with PNH.

Methods: The activity consisted of an approximately 2,000-word text-based interview between a moderator and a single expert faculty [Weitz I. https://www.medscape.org/viewarticle/943900. 2021.]. Educational effect was assessed with a repeated pairs pre-/post-assessment study including a 3-item, multiple choice, knowledge/competence questionnaire and 1 confidence assessment question, with each participant serving as his/her own control. Pre- and post-assessment scores were compared to determine relative changes in the proportion of correct responses to knowledge/competence questions. A paired samples t-test was conducted for significance testing on overall average number of correct responses and for confidence rating, and McNemar's test was conducted at the learning objective level (5% significance level, P <.05). Cohen's d with correction for paired samples estimated the effect size of the education on number of correct responses (<.20 modest, .20-.49 small, .59-.79 moderate, ≥.80 large). The activity launched 15 January 2021; data were collected until 3 May 2021.

Results: Overall, statistically significant improvements in knowledge/competence were seen after education consumption for hematologists (N=55, P <.001, Cohen's d=.59) and PCPs (N=332, P <.001, Cohen's d=.47). 5% of hematologists and 17% of PCPs improved (P <.317 and P <.001, respectively) and 78% and 57%, respectively, reinforced their knowledge regarding the clinical manifestations of PNH. 29% of hematologists and 40% of PCPs improved (P<.001) and 69% and 36%, respectively, reinforced their competence related to establishing a diagnosis of PNH. Following the activity, 38% of hematologists and 52% of PCPs had a measurable increase in confidence regarding their ability to evaluate patients with suspected PNH.

Conclusions: Participation in a text-based CME-certified activity resulted in statistically significant improvements in knowledge/competence and measurable increases in confidence of hematologists and PCPs regarding the diagnosis of PNH. These results have the ability to translate to improvements in clinical care. The need for additional educational activities was also identified to address residual gaps and further increase clinicians' ability in this rare clinical setting.