Ferras Alashkar

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.

Introduction Patients with hematologic disease are at high risk of morbidity and mortality from COVID‐19 due to disease‐inherent and therapy‐related immunodeficiency. Whether infection with the SARS‐CoV2 omicron variant leads to attenuated disease severity in these patients is currently unknown. Methods We assessed clinical and laboratory parameters in 61 patients with underlying hematologic conditions with a SARS‐CoV2 omicron variant infection confirmed by nucleic acid amplification testing. Results Fifty patients reported symptoms of COVID‐19, most commonly fatigue (37 patients, 60.66%) and cough (32 patients, 52.46%). 39.34% of patients reported fever. Dyspnea was reported by 10 patients and 7 patients (11.48%) required oxygen therapy. Anosmia and ageusia were relatively rare, occurring in less than 10% of patients. Severity of SARS‐CoV2 infection could be assessed in 60 patients. Five cases of critical illness leading to ICU admission occurred during the observation period. Overall mortality was 9.84% in this patient cohort, with heterogeneous causes of death. The majority of omicron‐infected hematologic patients experienced mild symptoms or remained asymptomatic. Discussion In this study, symptoms of COVID‐19 tended to be milder than described for previous SARS‐CoV2 variants. However, the extent to which attenuated severity of omicron‐variant SARS‐CoV2 infection is caused by altered viral pathogenicity or pre‐existing host immunity cannot be inferred from our data and should be investigated in larger prospective studies.