Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea

Jin Seok Kim, Jun Ho Jang, Deog Yeon Jo, Seo Yeon Ahn, Sung Soo Yoon, Je Hwan Lee, Sung Hyun Kim, Chul Won Choi, Ho Jin Shin, Min Kyoung Kim, Jae Hoon Lee, Yeung Chul Mun, Jee Hyun Kong, Bokjin Hyun, Hyun Sun Nam, Eunhye Kim, Min Joo Kwak, Yong Kyun Won, Jong Wook Lee

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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.

Original languageEnglish
Pages (from-to)1-12
Number of pages12
JournalJournal of Korean Medical Science
Issue number41
StatePublished - 2023

Bibliographical note

Publisher Copyright:
© 2023 The Korean Academy of Medical Sciences.


  • Eculizumab
  • Overall Survival
  • Paroxysmal Nocturnal Hemoglobinuria
  • Real-World Evidence


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