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 | Kim, Jin Seok | - |
dc.contributor.author | Jang, Jun Ho | - |
dc.contributor.author | Jo, Deog-Yeon | - |
dc.contributor.author | Ahn, Seo-Yeon | - |
dc.contributor.author | Yoon, Sung-Soo | - |
dc.contributor.author | Lee, Je-Hwan | - |
dc.contributor.author | Kim, Sung-Hyun | - |
dc.contributor.author | Choi, Chul Won | - |
dc.contributor.author | Shin, Ho-Jin | - |
dc.contributor.author | Kim, Min-Kyoung | - |
dc.contributor.author | Lee, Jae Hoon | - |
dc.contributor.author | Mun, Yeung-Chul | - |
dc.contributor.author | Kong, Jee Hyun | - |
dc.contributor.author | Hyun, BokJin | - |
dc.contributor.author | Nam, HyunSun | - |
dc.contributor.author | Kim, Eunhye | - |
dc.contributor.author | Kwak, Min Joo | - |
dc.contributor.author | Won, Yong Kyun | - |
dc.contributor.author | Lee, Jong Wook | - |
dc.date.accessioned | 2023-12-15T15:10:52Z | - |
dc.date.available | 2023-12-15T15:10:52Z | - |
dc.date.issued | 2023-10 | - |
dc.identifier.issn | 1011-8934 | - |
dc.identifier.issn | 1598-6357 | - |
dc.identifier.uri | https://scholarworks.bwise.kr/gachon/handle/2020.sw.gachon/89630 | - |
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. 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. | - |
dc.format.extent | 12 | - |
dc.language | 영어 | - |
dc.language.iso | ENG | - |
dc.publisher | KOREAN ACAD MEDICAL SCIENCES | - |
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.identifier.wosid | 001100305900006 | - |
dc.identifier.doi | 10.3346/jkms.2023.38.e328 | - |
dc.identifier.bibliographicCitation | JOURNAL OF KOREAN MEDICAL SCIENCE, v.38, no.41, pp 1 - 12 | - |
dc.identifier.kciid | ART003008294 | - |
dc.description.isOpenAccess | Y | - |
dc.identifier.scopusid | 2-s2.0-85175324024 | - |
dc.citation.endPage | 12 | - |
dc.citation.startPage | 1 | - |
dc.citation.title | JOURNAL OF KOREAN MEDICAL SCIENCE | - |
dc.citation.volume | 38 | - |
dc.citation.number | 41 | - |
dc.type.docType | Article | - |
dc.publisher.location | 대한민국 | - |
dc.subject.keywordAuthor | Paroxysmal Nocturnal Hemoglobinuria | - |
dc.subject.keywordAuthor | Eculizumab | - |
dc.subject.keywordAuthor | Real-World Evidence | - |
dc.subject.keywordAuthor | Overall Survival | - |
dc.relation.journalResearchArea | General & Internal Medicine | - |
dc.relation.journalWebOfScienceCategory | Medicine, General & Internal | - |
dc.description.journalRegisteredClass | scie | - |
dc.description.journalRegisteredClass | scopus | - |
dc.description.journalRegisteredClass | kci | - |
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