Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort
Year: 2017
Type: article
Study design: Observational Study
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.
Source: Blood Research
Authors: Chul Won Choi, Jun Ho Jang, Jin Seok Kim, Deog-Yeon Jo, Je-Hwan Lee, Sung-Hyun Kim, Yeo-Kyeoung Kim, Jong-Ho Won, Joo Seop Chung, Hawk Kim, Jae Hoon Lee, Min Kyoung Kim, Hyeon-Seok Eom, Shin Young Hyun, Jeong-A Kim, Jong Wook Lee
Institutions: Korea University Guro Hospital, Samsung Medical Center, Sungkyunkwan University, Yonsei University, The Catholic University of Korea St. Vincent's Hospital, Yonsei University Health System, Catholic University of Korea, Chungnam National University, Asan Medical Center, University of Ulsan, Gachon University Gil Medical Center, The Catholic University of Korea Seoul St. Mary's Hospital, Dong-A University Hospital, Dong-A University, Chonnam National University Hwasun Hospital, Soonchunhyang University Hospital Seoul, Pusan National University Hospital, Ulsan University Hospital, Yeungnam University Medical Center, Yeungnam University, National Cancer Center, National Cancer Center Hospital, Wonju Severance Christian Hospital, Yonsei University Mirae Campus
Language: English
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $2,290
OpenAlex ID: W2760674400