1.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 ; HyunSun NAM ; Eunhye KIM ; Min Joo KWAK ; Yong Kyun WON ; Jong Wook LEE
Journal of Korean Medical Science 2023;38(41):e328-
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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