Paroxysmal nocturnal hemoglobinuria treated with eculizumab in Wakayama, Japan : a retrospective analysis
Currently, the humanized anti-C5 monoclonal antibody, eculizumab, is widely used for treating paroxysmal nocturnal hemoglobinuria (PNH) due to its effects on suppression of intravascular hemolysis and resulting improvement in quality of life. However, in some cases, this treatment is refractory or is associated with meningococcal meningitis. No region-specific analyses have been published, and currently, information on region specificity and genetic factors is limited. We present here the results of a retrospective study involving eight patients with PNH who were treated with eculizumab in our hospital in Wakayama, Japan. The median age of these patients was 77 (range 23-88) years. Six patients had a complication of aplastic anemia, four patients had a history of thrombosis, and two experienced hemolytic episodes. Before initiating eculizumab treatment, the median serum LDH level was 1,192 IU/l (range 755-1,525 IU/l). Serum LDH levels normalized in five patients within a month of initiating therapy and PNH-related symptoms disappeared. C5 gene mutations were identified in the three patients who did not respond to eculizumab.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2020 |
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| Erschienen: |
2020 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:61 |
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| Enthalten in: |
Rinsho ketsueki] The Japanese journal of clinical hematology - 61(2020), 6 vom: 04., Seite 605-611 |
| Sprache: |
Japanisch |
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| Beteiligte Personen: |
Tamura, Shinobu [VerfasserIn] |
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| Links: |
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| Themen: |
A3ULP0F556 |
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| Anmerkungen: |
Date Completed 15.07.2020 Date Revised 15.07.2020 published: Print Citation Status MEDLINE |
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| doi: |
10.11406/rinketsu.61.605 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM312022301 |
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| 245 | 1 | 0 | |a Paroxysmal nocturnal hemoglobinuria treated with eculizumab in Wakayama, Japan |b a retrospective analysis |
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| 500 | |a Date Completed 15.07.2020 | ||
| 500 | |a Date Revised 15.07.2020 | ||
| 500 | |a published: Print | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Currently, the humanized anti-C5 monoclonal antibody, eculizumab, is widely used for treating paroxysmal nocturnal hemoglobinuria (PNH) due to its effects on suppression of intravascular hemolysis and resulting improvement in quality of life. However, in some cases, this treatment is refractory or is associated with meningococcal meningitis. No region-specific analyses have been published, and currently, information on region specificity and genetic factors is limited. We present here the results of a retrospective study involving eight patients with PNH who were treated with eculizumab in our hospital in Wakayama, Japan. The median age of these patients was 77 (range 23-88) years. Six patients had a complication of aplastic anemia, four patients had a history of thrombosis, and two experienced hemolytic episodes. Before initiating eculizumab treatment, the median serum LDH level was 1,192 IU/l (range 755-1,525 IU/l). Serum LDH levels normalized in five patients within a month of initiating therapy and PNH-related symptoms disappeared. C5 gene mutations were identified in the three patients who did not respond to eculizumab | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a C3-mediated extravascular hemolysis | |
| 650 | 4 | |a C5 genetic variants | |
| 650 | 4 | |a Eculizumab | |
| 650 | 4 | |a Paroxysmal nocturnal hemoglobinuria | |
| 650 | 7 | |a Antibodies, Monoclonal, Humanized |2 NLM | |
| 650 | 7 | |a eculizumab |2 NLM | |
| 650 | 7 | |a A3ULP0F556 |2 NLM | |
| 700 | 1 | |a Furuya, Yoshiaki |e verfasserin |4 aut | |
| 700 | 1 | |a Hori, Yoshikazu |e verfasserin |4 aut | |
| 700 | 1 | |a Hiroi, Takayuki |e verfasserin |4 aut | |
| 700 | 1 | |a Yamashita, Yusuke |e verfasserin |4 aut | |
| 700 | 1 | |a Oiwa, Takehiro |e verfasserin |4 aut | |
| 700 | 1 | |a Murata, Shogo |e verfasserin |4 aut | |
| 700 | 1 | |a Mushino, Toshiki |e verfasserin |4 aut | |
| 700 | 1 | |a Nishikawa, Akinori |e verfasserin |4 aut | |
| 700 | 1 | |a Hanaoka, Nobuyoshi |e verfasserin |4 aut | |
| 700 | 1 | |a Sonoki, Takashi |e verfasserin |4 aut | |
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