Cited 2 times in
Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea
DC Field | Value | Language |
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dc.contributor.author | 김진석 | - |
dc.date.accessioned | 2024-01-03T01:07:32Z | - |
dc.date.available | 2024-01-03T01:07:32Z | - |
dc.date.issued | 2023-10 | - |
dc.identifier.issn | 1011-8934 | - |
dc.identifier.uri | https://ir.ymlib.yonsei.ac.kr/handle/22282913/197462 | - |
dc.description.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. Eighty patients who initiated eculizumab from 2009-2020 were enrolled. Results: At eculizumab initiation, the median age was 51.5 years, lactate dehydrogenase (LDH) 6.8 × 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. | - |
dc.description.statementOfResponsibility | open | - |
dc.language | English | - |
dc.publisher | 대한의학회(The Korean Academy of Medical Sciences) | - |
dc.relation.isPartOf | JOURNAL OF KOREAN MEDICAL SCIENCE | - |
dc.rights | CC BY-NC-ND 2.0 KR | - |
dc.subject.MESH | Cost of Illness | - |
dc.subject.MESH | Hemoglobinuria, Paroxysmal* / complications | - |
dc.subject.MESH | Hemoglobinuria, Paroxysmal* / drug therapy | - |
dc.subject.MESH | Hemoglobinuria, Paroxysmal* / epidemiology | - |
dc.subject.MESH | Hemolysis | - |
dc.subject.MESH | Humans | - |
dc.subject.MESH | Hypertension, Pulmonary* / complications | - |
dc.subject.MESH | Middle Aged | - |
dc.subject.MESH | Renal Insufficiency* / complications | - |
dc.subject.MESH | Republic of Korea | - |
dc.subject.MESH | Spasm / complications | - |
dc.subject.MESH | Thromboembolism* | - |
dc.title | Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea | - |
dc.type | Article | - |
dc.contributor.college | College of Medicine (의과대학) | - |
dc.contributor.department | Dept. of Internal Medicine (내과학교실) | - |
dc.contributor.googleauthor | Jin Seok Kim | - |
dc.contributor.googleauthor | Jun Ho Jang | - |
dc.contributor.googleauthor | Deog-Yeon Jo | - |
dc.contributor.googleauthor | Seo-Yeon Ahn | - |
dc.contributor.googleauthor | Sung-Soo Yoon | - |
dc.contributor.googleauthor | Je-Hwan Lee | - |
dc.contributor.googleauthor | Sung-Hyun Kim | - |
dc.contributor.googleauthor | Chul Won Choi | - |
dc.contributor.googleauthor | Ho-Jin Shin | - |
dc.contributor.googleauthor | Min-Kyoung Kim | - |
dc.contributor.googleauthor | Jae Hoon Lee | - |
dc.contributor.googleauthor | Yeung-Chul Mun | - |
dc.contributor.googleauthor | Jee Hyun Kong | - |
dc.contributor.googleauthor | BokJin Hyun | - |
dc.contributor.googleauthor | HyunSun Nam | - |
dc.contributor.googleauthor | Eunhye Kim | - |
dc.contributor.googleauthor | Min Joo Kwak | - |
dc.contributor.googleauthor | Yong Kyun Won | - |
dc.contributor.googleauthor | Jong Wook Lee | - |
dc.identifier.doi | 10.3346/jkms.2023.38.e328 | - |
dc.contributor.localId | A01017 | - |
dc.relation.journalcode | J01517 | - |
dc.identifier.eissn | 1598-6357 | - |
dc.identifier.pmid | 37873628 | - |
dc.subject.keyword | Eculizumab | - |
dc.subject.keyword | Overall Survival | - |
dc.subject.keyword | Paroxysmal Nocturnal Hemoglobinuria | - |
dc.subject.keyword | Real-World Evidence | - |
dc.contributor.alternativeName | Kim, Jin Seok | - |
dc.contributor.affiliatedAuthor | 김진석 | - |
dc.citation.volume | 38 | - |
dc.citation.number | 41 | - |
dc.citation.startPage | e328 | - |
dc.identifier.bibliographicCitation | JOURNAL OF KOREAN MEDICAL SCIENCE, Vol.38(41) : e328, 2023-10 | - |
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