Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study
Objective To evaluate the short-term efficacy and safety of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.Method Data were retrospectively collected from patients with PNH who received at least 3 months of full-dose eculizumab. Changes in clinical and laboratory...
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Taylor & Francis Group
2025-12-01
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| Series: | Hematology |
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| Online Access: | https://www.tandfonline.com/doi/10.1080/16078454.2025.2450575 |
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| author | Leyu Wang Ziwei Liu Chen Yang Miao Chen Bing Han |
| author_facet | Leyu Wang Ziwei Liu Chen Yang Miao Chen Bing Han |
| author_sort | Leyu Wang |
| collection | DOAJ |
| description | Objective To evaluate the short-term efficacy and safety of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.Method Data were retrospectively collected from patients with PNH who received at least 3 months of full-dose eculizumab. Changes in clinical and laboratory indicators after 1, 3, and 6 months of eculizumab therapy and at the end of follow-up were documented. The incidence rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and adverse events were recorded.Result A total of 48 patients, including 27 males, with a median age of 46 (12–78) years were included. Twenty-four (50%) patients had classic PNH and 24 (50%) had bone marrow failure (BMF)/PNH. Eighteen (37.5%) patients required blood transfusion. The median duration of follow-up was 6 (3–15) months. During the follow-up period, Lactate Dehydrogenase (LDH) levels were lower than those at baseline (<0.05) at all observation points. The patients showed a significant reduction in creatinine levels from baseline (P = 0.022 and P = 0.039, respectively) at 1 and 3 months. At the end of the follow-up, fifteen (83.3%) became transfusion-independent. No new thrombotic events were observed. The FACIT-Fatigue score significantly improved (P < 0.05). No significant differences were observed in the changes in hemoglobin or LDH levels between patients with classic PNH and those with BMF/PNH. BTH was observed in 17.4% of patients and EVH in 10.4%. Mild adverse events occurred in 22.9% of patients. No deaths or clonal evolution was observed.Conclusion Eculizumab can effectively control the hemolytic symptoms of PNH with good tolerance for Chinese patients. |
| format | Article |
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| institution | Kabale University |
| issn | 1607-8454 |
| language | English |
| publishDate | 2025-12-01 |
| publisher | Taylor & Francis Group |
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| series | Hematology |
| spelling | doaj-art-40c427dcf0fb4419b06632fe25b3d8952025-01-30T09:42:58ZengTaylor & Francis GroupHematology1607-84542025-12-0130110.1080/16078454.2025.2450575Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center studyLeyu Wang0Ziwei Liu1Chen Yang2Miao Chen3Bing Han4Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing, People’s Republic of ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing, People’s Republic of ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing, People’s Republic of ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing, People’s Republic of ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing, People’s Republic of ChinaObjective To evaluate the short-term efficacy and safety of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.Method Data were retrospectively collected from patients with PNH who received at least 3 months of full-dose eculizumab. Changes in clinical and laboratory indicators after 1, 3, and 6 months of eculizumab therapy and at the end of follow-up were documented. The incidence rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and adverse events were recorded.Result A total of 48 patients, including 27 males, with a median age of 46 (12–78) years were included. Twenty-four (50%) patients had classic PNH and 24 (50%) had bone marrow failure (BMF)/PNH. Eighteen (37.5%) patients required blood transfusion. The median duration of follow-up was 6 (3–15) months. During the follow-up period, Lactate Dehydrogenase (LDH) levels were lower than those at baseline (<0.05) at all observation points. The patients showed a significant reduction in creatinine levels from baseline (P = 0.022 and P = 0.039, respectively) at 1 and 3 months. At the end of the follow-up, fifteen (83.3%) became transfusion-independent. No new thrombotic events were observed. The FACIT-Fatigue score significantly improved (P < 0.05). No significant differences were observed in the changes in hemoglobin or LDH levels between patients with classic PNH and those with BMF/PNH. BTH was observed in 17.4% of patients and EVH in 10.4%. Mild adverse events occurred in 22.9% of patients. No deaths or clonal evolution was observed.Conclusion Eculizumab can effectively control the hemolytic symptoms of PNH with good tolerance for Chinese patients.https://www.tandfonline.com/doi/10.1080/16078454.2025.2450575Paroxysmal nocturnal hemoglobinuriahemolysiseculizumabefficacysafety |
| spellingShingle | Leyu Wang Ziwei Liu Chen Yang Miao Chen Bing Han Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study Hematology Paroxysmal nocturnal hemoglobinuria hemolysis eculizumab efficacy safety |
| title | Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study |
| title_full | Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study |
| title_fullStr | Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study |
| title_full_unstemmed | Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study |
| title_short | Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study |
| title_sort | eculizumab treatment for chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria pnh efficacy and safety a single center study |
| topic | Paroxysmal nocturnal hemoglobinuria hemolysis eculizumab efficacy safety |
| url | https://www.tandfonline.com/doi/10.1080/16078454.2025.2450575 |
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