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[Eculizumab in patients with paroxysmal nocturnal hemoglobinuria: a real-world study in China].
- Source :
-
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi [Zhonghua Xue Ye Xue Za Zhi] 2024 Feb 14; Vol. 45 (2), pp. 184-189. - Publication Year :
- 2024
-
Abstract
- Objective: To evaluate the efficacy and safety of eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China. Methods: Data from PNH patients who received at least 3 months of full-dose eculizumab and were followed for at least 3 months between December 2022 and July 2023 were retrospectively collected. We evaluated changes in clinical and laboratory parameters after 1, 2, 3, and 6 months of eculizumab treatment. The rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and the occurrence of adverse reactions were also monitored. Results: The study included nine patients, six males and three females, with a median age of 54 (28-69) years. 5 of the patients had classic PNH, while 4 had PNH/AA. The number of episodes of hemoglobinuria was 5 (1-25) per month before eculizumab. 4 patients required blood transfusion, 5 had thrombosis and one had renal impairment before eculizumab. The median time to eculizumab was 6 (3-7) months and the followup period was 3 (3-6) months after treatment. The number of episodes of hemoglobinuria following eculizumab was 0 (0-1). During the followup period, no additional thrombotic events occurred. LDH at any time after eculizumab was lower than at baseline, and some patients' HGB increased. All transfused patients became transfusion-independent after receiving eculizumab. The FACIT-Fatigue score improved by an average of 17.3 points following treatment. 2 patients developed BTH and improved with symptomatic treatment. There were three adverse events that caused mild symptoms. There are no serious adverse events or deaths. Conclusion: Eculizumab can effectively control the hemolytic-related symptoms of PNH in China, reducing the need for blood transfusions to some extent, while also demonstrating a higher safety profile.
Details
- Language :
- Chinese
- ISSN :
- 0253-2727
- Volume :
- 45
- Issue :
- 2
- Database :
- MEDLINE
- Journal :
- Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi
- Publication Type :
- Academic Journal
- Accession number :
- 38604796
- Full Text :
- https://doi.org/10.3760/cma.j.cn121090-20231106-00250