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KMID : 0191120230380410328
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Journal of Korean Medical Science
2023 Volume.38 No. 41 p.328 ~ p.328
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Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea
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Kim Jin-Seok
Kim Soon-Joon
Jo Deog-Yeon
Ahn Seo-Yeon
Yoon Sung-Soo
Lee Je-Hwan
Kim Sung-Hyun
Shin Na-Kyoung
Shin Ho-Jin
Kim Min-Kyoung
Wie Jin-Hong
Mun Yeung-Chul
Kong Jee-Hyun
Hyun Bok-Jin
Nam Hyun-Sun
Dan Xu
Kwak Min-Joo
Won Yong-Kyun
Lee Jong-Wook
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Abstract
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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.
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KEYWORD
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Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, Real-World Evidence, Overall Survival
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