Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort.
- Author:
Chul Won CHOI
1
;
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
Author Information
- Publication Type:Original Article
- Keywords: Paroxysmal nocturnal hemoglobinuria; Aplastic anemia; Complement C5 inhibitor; Eculizumab
- MeSH: Anemia, Aplastic*; Cohort Studies*; Flow Cytometry; Follow-Up Studies; Hemoglobinuria, Paroxysmal*; Hemolysis; Humans; Prospective Studies*
- From:Blood Research 2017;52(3):207-211
- CountryRepublic of Korea
- Language:English
- 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.
