Korean J Hematol 2010; 45(4):
Published online December 31, 2010
https://doi.org/10.5045/kjh.2010.45.4.269
© The Korean Society of Hematology
1Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea.
2Department of Internal Medicine, The Catholic University of Korea, Seoul St. Mary's Hospital, Seoul, Korea.
3Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea.
4Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
5Department of Hematology-Oncology, Pusan National University Medical School, Pusan National University Hospital, Busan, Korea.
Correspondence to : Correspondence to Jong Wook Lee, M.D., Ph.D. Division of Hematology, Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, 505, Banpo-dong, Seocho-gu, Seoul 137-701, Korea. Tel: +82-2-2258-6050, Fax: +82-2-780-1283, jwlee@catholic.ac.kr
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder characterized by chronic complement-mediated hemolysis. Eculizumab, a humanized monoclonal antibody against the terminal complement protein C5, potently reduces chronic intravascular hemolysis. We tested the clinical efficacy and safety of a 24-week treatment with eculizumab in 6 Korean patients with PNH.
We enrolled 6 patients with PNH who had clinically significant hemolysis. Eculizumab was administered intravenously at 600 mg/week for the first 4 weeks followed by 900 mg at week 5 and 2nd weekly thereafter.
Three men and 3 women with a median age of 39.5 years (24-61 years) were enrolled. The median duration of PNH was 11 years (6-25 years). Hemolysis occurred in all patients [median lactate dehydrogenase (LDH) level, 7.95 times the upper limit of the reference range of LDH]. All patients treated with eculizumab had a rapid and sustained reduction in the degree of hemolysis. RBC transfusion requirements for 3 months were decreased from 0-12 units (median requirement, 1.5 units) to 0-6 units (median requirement, 0 units). Improvement in fatigue was noted in 4 patients. Further, 5 patients who had been receiving corticosteroids either reduced the dose or discontinued therapy. No significant adverse events related to eculizumab therapy were observed.
These results show that eculizumab reduces the degree of intravascular hemolysis, reduces or eliminates the requirement of RBC transfusion, and improves anemia and fatigue in patients with PNH. Eculizumab is an effective and safe option for treating Korean patients with PNH.
Keywords Paroxysmal nocturnal hemoglobinuria, Eculizumab, Efficacy, Safety
Korean J Hematol 2010; 45(4): 269-274
Published online December 31, 2010 https://doi.org/10.5045/kjh.2010.45.4.269
Copyright © The Korean Society of Hematology.
Jin Seok Kim1, Jong Wook Lee2*, Byoung Kook Kim3, Je-Hwan Lee4, and Jooseop Chung5
1Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea.
2Department of Internal Medicine, The Catholic University of Korea, Seoul St. Mary's Hospital, Seoul, Korea.
3Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea.
4Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
5Department of Hematology-Oncology, Pusan National University Medical School, Pusan National University Hospital, Busan, Korea.
Correspondence to: Correspondence to Jong Wook Lee, M.D., Ph.D. Division of Hematology, Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, 505, Banpo-dong, Seocho-gu, Seoul 137-701, Korea. Tel: +82-2-2258-6050, Fax: +82-2-780-1283, jwlee@catholic.ac.kr
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder characterized by chronic complement-mediated hemolysis. Eculizumab, a humanized monoclonal antibody against the terminal complement protein C5, potently reduces chronic intravascular hemolysis. We tested the clinical efficacy and safety of a 24-week treatment with eculizumab in 6 Korean patients with PNH.
We enrolled 6 patients with PNH who had clinically significant hemolysis. Eculizumab was administered intravenously at 600 mg/week for the first 4 weeks followed by 900 mg at week 5 and 2nd weekly thereafter.
Three men and 3 women with a median age of 39.5 years (24-61 years) were enrolled. The median duration of PNH was 11 years (6-25 years). Hemolysis occurred in all patients [median lactate dehydrogenase (LDH) level, 7.95 times the upper limit of the reference range of LDH]. All patients treated with eculizumab had a rapid and sustained reduction in the degree of hemolysis. RBC transfusion requirements for 3 months were decreased from 0-12 units (median requirement, 1.5 units) to 0-6 units (median requirement, 0 units). Improvement in fatigue was noted in 4 patients. Further, 5 patients who had been receiving corticosteroids either reduced the dose or discontinued therapy. No significant adverse events related to eculizumab therapy were observed.
These results show that eculizumab reduces the degree of intravascular hemolysis, reduces or eliminates the requirement of RBC transfusion, and improves anemia and fatigue in patients with PNH. Eculizumab is an effective and safe option for treating Korean patients with PNH.
Keywords: Paroxysmal nocturnal hemoglobinuria, Eculizumab, Efficacy, Safety
Changes in the levels of lactate dehydrogenase during treatment with eculizumab. Abbreviations: LDH, lactate dehydrogenase; ULN, upper limit of the normal reference range.
Reduction in the number of units of red blood cell transfused during treatment with eculizumab.
Table 1 . Summary of PNH patients..
a)Clone size was measured by determining the PNH granulocyte proportion by using flow cytometric analysis (CD59), b)LDH concentrations are expressed as multiples of the upper limit of the normal reference range of LDH..
Abbreviations: LDH, lactate dehydrogenase; ANC, absolute neutrophil count..
Table 2 . Symptoms related to PNH during the eculizumab therapy..
a)This patient received eculizumab therapy for only 12 weeks..
Abbreviations: Pre-Tx, pretreatment of with eculizumab; Mod, moderate..
Table 3 . Clinical effectiveness of eculizumab..
a)This patient received eculizumab therapy for only 12 weeks, b)LDH concentrations are expressed as multiples of the upper limit of the normal reference range of LDH..
Abbreviations: LDH, lactate dehydrogenase; FACIT-Fatigue, Functional Assessment of Chronic Illness Therapy-Fatigue; Hb, hemoglobin..
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Changes in the levels of lactate dehydrogenase during treatment with eculizumab. Abbreviations: LDH, lactate dehydrogenase; ULN, upper limit of the normal reference range.
|@|~(^,^)~|@|Reduction in the number of units of red blood cell transfused during treatment with eculizumab.