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Choi CW, Jang JH, Kim JS, et al. Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort. Blood Res. 2017;52(3):207-211doi: 10.5045/br.2017.52.3.207.
Choi, C. W., Jang, J. H., Kim, J. S., Jo, D. Y., Lee, J. H., Kim, S. H., Kim, Y. K., Won, J. H., Chung, J. S., Kim, H., Lee, J. H., Kim, M. K., Eom, H. S., Hyun, S. Y., Kim, J. A., & Lee, J. W. (2017). Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort. Blood research, 52(3), 207-211. https://doi.org/10.5045/br.2017.52.3.207
Choi, Chul Won, et al. "Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort." Blood research vol. 52,3 (2017): 207-211. doi: https://doi.org/10.5045/br.2017.52.3.207
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. Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort. Blood Res. 2017 Sep;52(3):207-211. doi: 10.5045/br.2017.52.3.207. Epub 2017 Sep 25. PMID: 29043236; PMCID: PMC5641513.
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Blood Res. 2017 Sep;52(3):207-211. doi: 10.5045/br.2017.52.3.207. Epub 2017 Sep 25.

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

Blood research

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

Affiliations

  1. Department of Internal Medicine, Korea University Guro Hospital, Seoul, Korea.
  2. Department of Internal Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
  3. Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea.
  4. Department of Internal Medicine, Chungnam National University, Daejeon, Korea.
  5. Department of Internal Medicine, Asan Medical Center, University of Ulsan, College of Medicine, Seoul, Korea.
  6. Department of Internal Medicine, Dong-A University Hospital, Busan, Korea.
  7. Department of Internal Medicine, Chonnam National University Hwasun Hospital, Hwasun, Korea.
  8. Department of Internal Medicine, SoonChunHyang University Hospital, Seoul, Korea.
  9. Department of Internal Medicine, Pusan National University Hospital, Busan, Korea.
  10. Department of Internal Medicine, Ulsan University Hospital, Ulsan, Korea.
  11. Department of Internal Medicine, Gachon University Gil Medical Center, Incheon, Korea.
  12. Department of Internal Medicine, Youngnam University Hospital, Daegu, Korea.
  13. Department of Internal Medicine, National Cancer Center, Goyang, Korea.
  14. Department of Internal Medicine, Yonsei University Wonju College of Medicine, Wonju, Korea.
  15. Department of Internal Medicine, St. Vincent Hospital, The Catholic University of Korea, Suwon, Korea.
  16. Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Korea.

PMID: 29043236 PMCID: PMC5641513 DOI: 10.5045/br.2017.52.3.207

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: Aplastic anemia; Complement C5 inhibitor; Eculizumab; Paroxysmal nocturnal hemoglobinuria

Conflict of interest statement

Authors' Disclosures of Potential Conflicts of Interest: No potential conflicts of interest relevant to this article were reported.

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