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KMID : 0366220170520030207
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Korean Journal of Hematology
2017 Volume.52 No. 3 p.207 ~ p.211
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Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort
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Choi Chul-Won
Jang Jun-Ho
Kim Jin-Seok
Jo Deog-Yeon
Lee Je-Hwan
Kim Sung-Hyun
Kim Yeo-Kyeoung
Won Jong-Ho
Chung Joo-Seop
Kim Hawk
Lee Jae-Hoon
Kim Min-Kyoung
Eom Hyeon-Seok
Hyun Shin-Young
Kim Jeong-A
Lee Jong-Wook
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Abstract
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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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KEYWORD
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Paroxysmal nocturnal hemoglobinuria, Aplastic anemia, Complement C5 inhibitor, Eculizumab
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