Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Koreaopen access
- Authors
- Kim, Jin Seok; Jang, Jun Ho; Jo, Deog-Yeon; Ahn, Seo-Yeon; Yoon, Sung-Soo; Lee, Je-Hwan; Kim, Sung-Hyun; Choi, Chul Won; Shin, Ho-Jin; Kim, Min-Kyoung; Lee, Jae Hoon; Mun, Yeung-Chul; Kong, Jee Hyun; Hyun, BokJin; Nam, HyunSun; Kim, Eunhye; Kwak, Min Joo; Won, Yong Kyun; Lee, Jong Wook
- Issue Date
- Oct-2023
- Publisher
- KOREAN ACAD MEDICAL SCIENCES
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria; Eculizumab; Real-World Evidence; Overall Survival
- Citation
- JOURNAL OF KOREAN MEDICAL SCIENCE, v.38, no.41, pp 1 - 12
- Pages
- 12
- Journal Title
- JOURNAL OF KOREAN MEDICAL SCIENCE
- Volume
- 38
- Number
- 41
- Start Page
- 1
- End Page
- 12
- URI
- https://scholarworks.bwise.kr/gachon/handle/2020.sw.gachon/89630
- DOI
- 10.3346/jkms.2023.38.e328
- ISSN
- 1011-8934
1598-6357
- Abstract
- Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by uncontrolled terminal complement activation. Eculizumab, a monoclonal antibody C5 inhibitor was introduced in Korea in 2009 and has been the standard treatment option for PNH.Methods: This study assessed the long-term efficacy/safety of eculizumab in PNH using real-world data from the Korean Health Insurance Review and Assessment Service. Eightypatients who initiated eculizumab from 2009-2020 were enrolled. Results: At eculizumab initiation, the median age was 51.5 years, lactate dehydrogenase (LDH) 6.8 x upper limit of normal, and granulocyte clone size 93.0%. All patients had at least one PNH-related complication before eculizumab initiation, including renal failure (n = 36), smooth muscle spasm (n = 24), thromboembolism (n = 20), and pulmonary hypertension (n = 15). The median (range) duration of eculizumab treatment was 52.7 (1.0, 127.3) months (338.6 total treated patient-years). Despite high disease activity in the study population before treatment initiation, overall survival was 96.2% and LDH levels were stabilized in most patients during treatment. PNH-related complications at treatment initiation were resolved in 44.4% of patients with renal failure, 95.8% with smooth muscle spasm, 70.0% with thromboembolism, and 26.7% with pulmonary hypertension. Extravascular hemolysis occurred in 28.8% of patients (n = 23; 0.09 per patient-year) and breakthrough hemolysis in 18.8% (n = 15; 0.06 per patient-year). No treatment discontinuation cases related to eculizumab were observed. Conclusion: These data provided evidence for the long-term efficacy and safety of eculizumab in Korean PNH patients with high disease burdens.
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