Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea
- 주제(키워드) Paroxysmal Nocturnal Hemoglobinuria , Eculizumab , Real-World Evidence , Overall Survival
- 주제(기타) Medicine, General & Internal
- 설명문(일반) [Kim, Jin Seok] Yonsei Univ, Severance Hosp, Coll Med, Div Hematol,Dept Internal Med, Seoul, South Korea; [Jang, Jun Ho] Sungkyunkwan Univ, Sch Med, Samsung Med Ctr, Div Hematol Oncol,Dept Med, Seoul, South Korea; [Jo, Deog-Yeon] Chungnam Natl Univ, Coll Med, Dept Internal Med, Daejeon, South Korea; [Ahn, Seo-Yeon] Chonnam Natl Univ, Hwasun Hosp, Dept Hematol Oncol, Hwasun, South Korea; [Yoon, Sung-Soo] Seoul Natl Univ, Seoul Natl Univ Hosp, Dept Internal Med, Div Hematol Med Oncol,Coll Med, Seoul, South Korea; [Lee, Je-Hwan] Univ Ulsan, Coll Med, Asan Med Ctr, Dept Hematol, Seoul, South Korea; [Kim, Sung-Hyun] Dong A Univ, Dong A Univ Hosp, Dept Internal Med, Div Hematol & Oncol,Coll Med, Busan, South Korea; [Choi, Chul Won] Korea Univ, Guro Hosp, Dept Internal Med, Seoul, South Korea; [Shin, Ho-Jin] Pusan Natl Univ, Pusan Natl Univ Hosp, Sch Med, Dept Internal Med, Busan, South Korea; [Kim, Min-Kyoung] Yeungnam Univ, Coll Med, Dept Med, Div Hematol Oncol, Daegu, South Korea; [Lee, Jae Hoon] Gachon Univ, Gil Med Ctr, Dept Internal Med, Div Hematol, Incheon, South Korea; [Mun, Yeung-Chul] Ewha Womans Univ, Coll Med, Dept Internal Med, Seoul, South Korea; [Kong, Jee Hyun] Yonsei Coll Med, Wonju Severance Christian Hosp, Div Internal Med, Dept Hematol Oncol, Seoul, South Korea; [Kong, Jee Hyun] Yonsei Univ, Inst Convergence Sci, Ctr Evidence Based Med, Seoul, South Korea; [Hyun, BokJin; Nam, HyunSun; Kim, Eunhye] Handok Inc, Clin Res, Seoul, South Korea; [Kwak, Min Joo; Won, Yong Kyun] Handok Inc, Med Affairs, Seoul, South Korea; [Lee, Jong Wook] Catholic Univ Korea, Coll Med, Seoul St Marys Hosp, Div Hematol, 222 Banpo Daero, Seoul 06591, South Korea; [Kwak, Min Joo] AstraZeneca Korea, Seoul, South Korea; [Won, Yong Kyun] Soonchunhyang Univ, Coll Med, Dept Radiat Oncol, Cheonan, South Korea
- 등재 SCIE, SCOPUS, KCI등재
- OA유형 Green Published; Gold Open Access
- 발행기관 KOREAN ACAD MEDICAL SCIENCES
- 발행년도 2023
- 총서유형 Journal
- URI http://www.dcollection.net/handler/ewha/000000213468
- 본문언어 영어
- Published As https://doi.org/10.3346/jkms.2023.38.e328
- PubMed 37873628
초록/요약
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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