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

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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.

Original languageEnglish
Pages (from-to)207-211
Number of pages5
JournalBlood Research
Volume52
Issue number3
DOIs
Publication statusPublished - 2017 Jan 1

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Paroxysmal Hemoglobinuria
Aplastic Anemia
Prospective Studies
Hemolysis
eculizumab
Therapeutics
Registries
Flow Cytometry

All Science Journal Classification (ASJC) codes

  • Hematology

Cite this

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. / Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia : Prospective study of a Korean PNH cohort. In: Blood Research. 2017 ; Vol. 52, No. 3. pp. 207-211.
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title = "Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: Prospective study of a Korean PNH cohort",
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.",
author = "Choi, {Chul Won} and Jang, {Jun Ho} and Kim, {Jin Seok} and Jo, {Deog Yeon} and Lee, {Je Hwan} and Kim, {Sung Hyun} and Kim, {Yeo Kyeoung} and Won, {Jong Ho} and Chung, {Joo Seop} and Hawk Kim and Lee, {Jae Hoon} and Kim, {Min Kyoung} and Eom, {Hyeon Seok} and Hyun, {Shin Young} and Kim, {Jeong A.} and Lee, {Jong Wook}",
year = "2017",
month = "1",
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Choi, CW, Jang, JH, Kim, JS, Jo, DY, Lee, JH, Kim, SH, Kim, YK, Won, JH, Chung, JS, Kim, H, Lee, JH, Kim, MK, Eom, HS, Hyun, SY, Kim, JA & Lee, JW 2017, 'Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: Prospective study of a Korean PNH cohort', Blood Research, vol. 52, no. 3, pp. 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. / 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.

In: Blood Research, Vol. 52, No. 3, 01.01.2017, p. 207-211.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia

T2 - Prospective study of a Korean PNH cohort

AU - Choi, Chul Won

AU - Jang, Jun Ho

AU - Kim, Jin Seok

AU - Jo, Deog Yeon

AU - Lee, Je Hwan

AU - Kim, Sung Hyun

AU - Kim, Yeo Kyeoung

AU - Won, Jong Ho

AU - Chung, Joo Seop

AU - Kim, Hawk

AU - Lee, Jae Hoon

AU - Kim, Min Kyoung

AU - Eom, Hyeon Seok

AU - Hyun, Shin Young

AU - Kim, Jeong A.

AU - Lee, Jong Wook

PY - 2017/1/1

Y1 - 2017/1/1

N2 - 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.

AB - 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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DO - 10.5045/br.2017.52.3.207

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AN - SCOPUS:85029879529

VL - 52

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EP - 211

JO - Blood Research

JF - Blood Research

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