Long‐term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real‐world setting
| Autor: | Katharina Versmold, Ferras Alashkar, Carina Raiser, Richard Ofori‐Asenso, Tao Xu, Yutong Liu, Pablo Katz, Aijing Shang, Alexander Röth |
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| Rok vydání: | 2023 |
| Předmět: | |
| Zdroj: | European Journal of Haematology. |
| ISSN: | 1600-0609 0902-4441 |
| DOI: | 10.1111/ejh.13970 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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