Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea
10.3346/jkms.2023.38.e328
- Author:
Jin Seok KIM
1
;
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
Author Information
1. Division of Hematology, Department of Internal Medicine, Yonsei University College of Medicine, Severance Hospital, Seoul, Korea
- Publication Type:Original Article
- From:Journal of Korean Medical Science
2023;38(41):e328-
- CountryRepublic of Korea
- Language:English
-
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. 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.
