Blood Res 2017; 52(3): 207-211  https://doi.org/10.5045/br.2017.52.3.207
Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort
Chul Won Choi1, Jun Ho Jang2, Jin Seok Kim3, Deog-Yeon Jo4, Je-Hwan Lee5, Sung-Hyun Kim6, Yeo-Kyeoung Kim7, Jong-Ho Won8, Joo Seop Chung9, Hawk Kim10,11, Jae Hoon Lee11, Min Kyoung Kim12, Hyeon-Seok Eom13, Shin Young Hyun14, Jeong-A Kim15, Jong Wook Lee16
Department of Internal Medicine, 1Korea University Guro Hospital, 2Samsung Medical Center, Sungkyunkwan University School of Medicine, 3Yonsei University College of Medicine, Seoul, 4Chungnam National University, Daejeon, 5Asan Medical Center, University of Ulsan, College of Medicine, Seoul, 6Dong-A University Hospital, Busan, 7Chonnam National University Hwasun Hospital, Hwasun, 8SoonChunHyang University Hospital, Seoul, 9Pusan National University Hospital, Busan, 10Ulsan University Hospital, Ulsan, 11Gachon University Gil Medical Center, Incheon, 12Youngnam University Hospital, Daegu, 13National Cancer Center, Goyang, 14Yonsei University Wonju College of Medicine, Wonju, 15St. Vincent Hospital, The Catholic University of Korea, Suwon, 16Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Korea
Correspondence to: Jong Wook Lee, M.D., Ph.D.Department of Hematology, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, 222 Banpo- daero, Seocho-gu, Seoul 06591, KoreaE-mail: jwlee@catholic.ac.kr
Received: July 31, 2017; Revised: August 20, 2017; Accepted: August 29, 2017; Published online: September 30, 2017.
© The Korean Journal of Hematology. All rights reserved.

Abstract
Background 
Patients with paroxysmal nocturnal hemoglobinuria (PNH) often have concurrent aplastic anemia (AA). This study aimed to determine whether eculizumab-treated patients show clinical benefit regardless of concurrent AA.
Methods 
We analyzed 46 PNH patients ≥18 years of age who were diagnosed by flow cytometry and treated with eculizumab for more than 6 months in the prospective Korean PNH registry. Patients were categorized into two groups: PNH patients with concurrent AA (PNH/AA, N=27) and without AA (classic PNH, N=19). Biochemical indicators of intravascular hemolysis, hematological laboratory values, transfusion requirement, and PNH-associated complications were assessed at baseline and every 6 months after initiation of eculizumab treatment.
Results
The median patient age was 46 years and median duration of eculizumab treatment was 34 months. Treatment with eculizumab induced rapid inhibition of hemolysis. At 6-month follow-up, LDH decreased to near normal levels in all patients; this effect was maintained until the 36-month follow-up regardless of concurrent AA. Transfusion independence was achieved by 53.3% of patients within the first 6 months of treatment and by 90.9% after 36 months of treatment. The mean number of RBC units transfused was significantly reduced, from 8.5 units during the 6 months prior to initiation of eculizumab to 1.6 units in the first 6 months of treatment, for the total study population; this effect was similar in both PNH/AA and classic PNH.
Conclusion
This study demonstrated that eculizumab is beneficial in the management of patients with PNH/AA, similar to classic PNH.
Keywords: Paroxysmal nocturnal hemoglobinuria, Aplastic anemia, Complement C5 inhibitor, Eculizumab 


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