Excellence in PNH in Canada (EPIC): A Single Centre Pilot Project Evaluating Disease Trajectory for PNH Patients Receiving Eculizumab

Conclusion:PNH is rare and can present in heterogeneous ways. Outside hematology, HCPs may rarely encounter patients, if ever. Time interval between onset of symptoms to formal diagnosis in our cohort had the greatest duration and variation. Once diagnosed, all patients in our cohort were initiated on therapy quite rapidly save for one who had a nine-year delay between diagnosis and availability of eculizumab in Canada. The greatest delay in this cohort was the time from symptom onset to diagnosis, suggesting that a focus on increasing awareness of PNH may be the area where most efforts should be placed.DisclosuresPatriquin: Octapharma: Honoraria; Alexion Pharmaceuticals, Inc.: Consultancy, Honoraria, Other: Travel Support and is site investigator for clinical trials with the company; Ra Pharmaceuticals: Consultancy, Research Funding.
Source: Blood - Category: Hematology Authors: Tags: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron Source Type: research