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Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort

 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 
 BLOOD RESEARCH, Vol.52(3) : 207-211, 2017 
Journal Title
Issue Date
Aplastic anemia ; Complement C5 inhibitor ; Eculizumab ; Paroxysmal nocturnal hemoglobinuria
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.
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1. College of Medicine (의과대학) > Dept. of Internal Medicine (내과학교실) > 1. Journal Papers
Yonsei Authors
Kim, Jin Seok(김진석) ORCID logo https://orcid.org/0000-0001-8986-8436
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