Long-term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real-world setting.

OBJECTIVE: Describe the real-world clinical profile of eculizumab-treated patients by characterizing their short- and long-term clinical and laboratory outcomes. METHODS: This retrospective study used preexisting medical records of eculizumab-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) at the University Hospital Essen. Hematologic response, breakthrough hemolysis, transfusion dependence, and other outcomes were assessed. RESULTS: Of 85 patients with PNH, 76 received eculizumab for ≥24 weeks (mean follow-up: 5.59 years; total: 425 person-years). At 24 weeks (n = 57 patients with data), 7% and 9% had complete and major hematologic response, respectively. Breakthrough hemolysis occurred in 8%, and 38% required a blood transfusion. Over long-term follow-up (25-264 weeks), 70%-82% of patients did not achieve complete or major hematologic response in any 24-week period. Breakthrough symptoms, breakthrough hemolysis, and transfusion dependence occurred in 63%, 43%, and 63% of patients, respectively, at any point during follow-up. The majority (79%-89%) of patients did not achieve normalized hemoglobin, with 76%-93% having elevated bilirubin or absolute reticulocyte count in any 24-week window. Mean percentage reduction in lactate dehydrogenase (baseline to end of follow-up) was 80.3% (95% CI, 64.0-96.6). CONCLUSIONS: A considerable proportion of patients with PNH receiving eculizumab did not achieve optimal clinical outcomes and had an ongoing disease burden.

Experience with an OSCE anamnesis station via Zoom: Feasibility, acceptance and challenges from the perspective of students, simulated patients and examiners during the COVID-19 pandemic.

Aim: Assessments of practical clinical competencies pose a challenge during the COVID-19 pandemic. Reports about OSCE stations administered online show that, despite technical feasibility and acceptance, there is a lingering desire for in-person assessments. Barriers and challenges must therefore also be identified in regard to the future integration of digital competencies into the curriculum. Based on a study investigating the feasibility and acceptance of an online OSCE anamnesis station and the descriptions given by students, simulated patients and examiners of the challenges and limitations, we make recommendations for necessary future adaptations to anamnesis training and testing in the context of telemedicine. Method: We surveyed students after completion of an OSCE anamnesis station, adapted to the telemedical setting, that was administered as an alternative assessment to 149 students via Zoom®. Using semi-structured interviews, we analyzed the resulting challenges and limitations as seen by all of the participants. Results: We confirm the existence of good technical and organizational feasibility, positive learning experiences through feedback, the acquisition of clinical competencies, and a high acceptance of this format as an alternative assessment during the pandemic. Using the semi-structured interviews, it was also possible to analyze additional categories that identify necessary adaptations of this type of format. Conclusion: Adaptation of the content-based training for all of the participants and a targeted revision of the checklists, e.g., regarding communication techniques in a telemedicine setting, is required due to the effects of the online format on communication and interactions between students and simulated patients.