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Original Article

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Blood Res 2017; 52(3):

Published online September 25, 2017

https://doi.org/10.5045/br.2017.52.3.207

© The Korean Society of Hematology

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

Chul Won Choi1, Jun Ho Jang2, Jin Seok Kim3, Deog-Yeon Jo4, Je-Hwan Lee5, Sung-Hyun Kim6, Yeo-Kyeoung Kim7, Jong-Ho Won8, Joo Seop Chung9, Hawk Kim10,11, Jae Hoon Lee11, Min Kyoung Kim12, Hyeon-Seok Eom13, Shin Young Hyun14, Jeong-A Kim15, and Jong Wook Lee16*

Author Info. +

1Department of Internal Medicine, Korea University Guro Hospital, Seoul, Korea.

2Department of Internal Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.

3Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea.

4Department of Internal Medicine, Chungnam National University, Daejeon, Korea.

5Department of Internal Medicine, Asan Medical Center, University of Ulsan, College of Medicine, Seoul, Korea.

6Department of Internal Medicine, Dong-A University Hospital, Busan, Korea.

7Department of Internal Medicine, Chonnam National University Hwasun Hospital, Hwasun, Korea.

8Department of Internal Medicine, SoonChunHyang University Hospital, Seoul, Korea.

9Department of Internal Medicine, Pusan National University Hospital, Busan, Korea.

10Department of Internal Medicine, Ulsan University Hospital, Ulsan, Korea.

11Department of Internal Medicine, Gachon University Gil Medical Center, Incheon, Korea.

12Department of Internal Medicine, Youngnam University Hospital, Daegu, Korea.

13Department of Internal Medicine, National Cancer Center, Goyang, Korea.

14Department of Internal Medicine, Yonsei University Wonju College of Medicine, Wonju, Korea.

15Department of Internal Medicine, St. Vincent Hospital, The Catholic University of Korea, Suwon, Korea.

16Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Korea.

Correspondence to : Jong Wook Lee, M.D., Ph.D. Department of Hematology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, 222 Banpo-daero, Seocho-gu, Seoul 06591, Korea. jwlee@catholic.ac.kr

Received: July 31, 2017; Revised: August 20, 2017; Accepted: August 29, 2017

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.

Keywords Paroxysmal nocturnal hemoglobinuria, Aplastic anemia, Complement C5 inhibitor, Eculizumab

Article

Original Article

Blood Res 2017; 52(3): 207-211

Published online September 25, 2017 https://doi.org/10.5045/br.2017.52.3.207

Copyright © The Korean Society of Hematology.

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

Chul Won Choi1, Jun Ho Jang2, Jin Seok Kim3, Deog-Yeon Jo4, Je-Hwan Lee5, Sung-Hyun Kim6, Yeo-Kyeoung Kim7, Jong-Ho Won8, Joo Seop Chung9, Hawk Kim10,11, Jae Hoon Lee11, Min Kyoung Kim12, Hyeon-Seok Eom13, Shin Young Hyun14, Jeong-A Kim15, and Jong Wook Lee16*

1Department of Internal Medicine, Korea University Guro Hospital, Seoul, Korea.

2Department of Internal Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.

3Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea.

4Department of Internal Medicine, Chungnam National University, Daejeon, Korea.

5Department of Internal Medicine, Asan Medical Center, University of Ulsan, College of Medicine, Seoul, Korea.

6Department of Internal Medicine, Dong-A University Hospital, Busan, Korea.

7Department of Internal Medicine, Chonnam National University Hwasun Hospital, Hwasun, Korea.

8Department of Internal Medicine, SoonChunHyang University Hospital, Seoul, Korea.

9Department of Internal Medicine, Pusan National University Hospital, Busan, Korea.

10Department of Internal Medicine, Ulsan University Hospital, Ulsan, Korea.

11Department of Internal Medicine, Gachon University Gil Medical Center, Incheon, Korea.

12Department of Internal Medicine, Youngnam University Hospital, Daegu, Korea.

13Department of Internal Medicine, National Cancer Center, Goyang, Korea.

14Department of Internal Medicine, Yonsei University Wonju College of Medicine, Wonju, Korea.

15Department of Internal Medicine, St. Vincent Hospital, The Catholic University of Korea, Suwon, Korea.

16Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Korea.

Correspondence to:Jong Wook Lee, M.D., Ph.D. Department of Hematology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, 222 Banpo-daero, Seocho-gu, Seoul 06591, Korea. jwlee@catholic.ac.kr

Received: July 31, 2017; Revised: August 20, 2017; Accepted: August 29, 2017

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.

Keywords: Paroxysmal nocturnal hemoglobinuria, Aplastic anemia, Complement C5 inhibitor, Eculizumab

Fig 1.

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Figure 1.

Reduction in hemolysis after eculizumab. Treatment with eculizumab induced rapid and consistent inhibition of hemolysis to near normal levels in all patients after 36 months of follow-up. Patients with both PNH/AA and classic PNH had significant median change of LDH level. P-values compared with baseline were assessed using Wilcoxon signed-rank test.

Abbreviations: AA, aplastic anemia; ecu, eculizumab; PNH, paroxysmal nocturnal hemoglobinuria; ULN, upper limit of normal.

Blood Research 2017; 52: 207-211https://doi.org/10.5045/br.2017.52.3.207

Fig 2.

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Figure 2.

Improvement of anemia after eculizumab. In both patients with PNH/AA and classic PNH, hemoglobin levels were significantly improved after the first 6 months of eculizumab treatment; the effect (hemoglobin above 10 g/dL) was sustained after 36 months. P-values compared with baseline were assessed by Wilcoxon signed-rank test.

Abbreviations: AA, aplastic anemia; ecu, eculizumab; PNH, paroxysmal nocturnal hemoglobinuria.

Blood Research 2017; 52: 207-211https://doi.org/10.5045/br.2017.52.3.207

Fig 3.

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Figure 3.

Reduction of transfusion requirement after eculizumab. The mean number of packed 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. P-values compared with baseline were assessed by Wilcoxon signed-rank test.

Abbreviations: AA, aplastic anemia; ecu, eculizumab; PNH, paroxysmal nocturnal hemoglobinuria; RBC, red blood cells.

Blood Research 2017; 52: 207-211https://doi.org/10.5045/br.2017.52.3.207

Table 1 . Patient characteristics and baseline laboratory findings..

a)Student t-test, b)chi-squared test, c)Mann–Whitney U test..

Abbreviations: AA, aplastic anemia; LDH, lactate dehydrogenase; PNH, paroxysmal nocturnal hemoglobinuria; RBC, red blood cells; ULN, upper limit of normal; WBC, white blood cells..

Table 2 . Baseline clinical signs and symptoms..

a)Chi-squared test, b)Mann–Whitney U test, c)male patients only..

Abbreviations: AA, aplastic anemia; PNH, paroxysmal nocturnal hemoglobinuria..

Table 3 . Anticoagulation use in patients with thromboembolism history..

a)One patient restarted anticoagulation treatment after recurring TE event at 36-month follow-up..
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