Effects of enzyme replacement therapy on a Fabry disease patient receiving hemodialysis: Pharmacokinetics of agalsidase alpha in a hemodialysis patient

• Okada Shioko

  Department of Internal Medicine, Ohno Memorial Hospital

• Inariba Hiromi

  Department of Internal Medicine, Ohno Memorial Hospital

• Sai Yoshinori

  Department of Internal Medicine, Ohno Memorial Hospital

• Kawano Hitomi

  Department of Internal Medicine, Ohno Memorial Hospital

• Teragaki Masakazu

  Department of Internal Medicine, Ohno Memorial Hospital

• Okamura Mikio

  Department of Internal Medicine, Ohno Memorial Hospital

• Yoshimoto Mitsuru

  Department of Urology, Ohno Memorial Hospital

• Tanaka Akemi

  Department of Pediatrics, Osaka City University Graduate School of Medicine

• Negoro Nobuo

  Department of Cardiology, Osaka City University Graduate School of Medicine

• Yoshiyama Minoru

  Department of Cardiology, Osaka City University Graduate School of Medicine

Bibliographic Information

Other Title

• ファブリー病透析患者における酵素補充療法の治療効果と，アガルシダーゼアルファの体内動態について
• 症例報告 ファブリー病透析患者における酵素補充療法の治療効果と,アガルシダーゼアルファの体内動態について
• ショウレイ ホウコク ファブリービョウ トウセキ カンジャ ニ オケル コウソ ホジュウ リョウホウ ノ チリョウ コウカ ト アガルシダーゼアルファ ノ タイナイ ドウタイ ニ ツイテ

Abstract

Fabry disease is an X-linked inherited lysosomal storage disease caused by deficiency of α-galactosidase A (AGA). Enzyme-replacement therapy (ERT) has been used to treat Fabry disease. Two preparations of α-galactosidase A, agalsidase α (Replagal^®) and agalsidase β (Fabrazyme^®), are available. Agalsidase β can be administered during hemodialysis without any decrease in efficacy, but there are no data on the efficacy of agalsidase α administration during hemodialysis. We investigated the pharmacokinetics of an agalsidase α preparation by assessing the AGA activity in the plasma. [Case] The patient was a 46-year-old man who showed cerebral infarction and was diagnosed with Fabry disease at the age of 26 years. His renal function diminished, and hemodialysis was started in August 2006. In November 2006, ERT with agalsidase β was initiated ; however, because of an infusion reaction, the therapeutic agent was changed to agalsidase α in April 2007. We measured AGA activity in plasma when the patient was receiving agalsidase α intravenously ; these assessments were serially performed during hemodialysis and again during the interval between dialysis sessions. There was no difference between AGA activity during hemodialysis and that during the interval between sessions. [Discussion] There was no reduction in the agalsidase α activity in the patient's plasma during hemodialysis. The 2 preparations showed equal efficacy as ERT for hemodialysis patients with Fabry disease. The clinical course of our case demonstrated the importance of continuing ERT in patients undergoing hemodialysis.

Journal

• Nihon Toseki Igakkai Zasshi

  Nihon Toseki Igakkai Zasshi 43 (11), 945-951, 2010

  The Japanese Society for Dialysis Therapy

Keywords

• Fabry disease
• agalsidase
• enzyme replacement therapy
• hemodialysis

Details

• CRID: 1390001204680290432
• NII Article ID: 10027893550
• NII Book ID: AN10432053
• DOI: 10.4009/jsdt.43.945
• ISSN: 1883082X
• ISSN: 13403451
• NDL BIB ID: 10921610
• Web Site: https://ndlsearch.ndl.go.jp/books/R000000004-I10921610
• Web Site: https://search.jamas.or.jp/link/ui/2011084389
• Text Lang: ja
• Data Source:
  • JaLC
  • NDL
  • Crossref
  • CiNii Articles
  • KAKEN
• Abstract License Flag: Disallowed