Report of the ASFA apheresis registry study on Wilson's disease

Huy P. Pham, Joseph Schwartz, Laura Cooling, Jan C. Hofmann, Haewon C. Kim, Shanna Morgan, Monica B. Pagano, Jennifer Schneiderman, Jeffrey L. Winters, Chisa Yamada, Edward C.C. Wong, Yanyun Wu*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

13 Scopus citations

Abstract

Purpose Wilson's disease is a rare autosomal recessive genetic disorder that results in accumulation of copper in the liver, brain, cornea and kidney. Therapeutic plasma exchange (TPE) has been used to remove copper and provide a bridge to liver transplantation. We report here the collective experiences through the ASFA apheresis registry on Wilson's disease. Methods The ASFA apheresis registry is a multi-center registry study. Both prospective and retrospective data, with the latter involving data collection back to January 2000 are entered in the registry. The registry includes patient demographics, apheresis procedural information, treatment schedules, and treatment outcomes and complications. Results A total of 10 patients (3 males and 7 females) with Wilson's disease treated between 2005 and 2013 were included. Median age of first diagnosis and first TPE were 16 and 17 years, respectively. Via central venous access, these patients underwent a total of 43 TPEs; the median number of TPE procedures per patient was 3.5. All of the TPEs used ACD-A as anticoagulation, 42/43 TPEs targeted 1-1.25 plasma volumes, and 41/43 TPEs were performed with 100% fluid balance. Post TPE procedures, 9 patients underwent liver transplantation; all 10 patients had at least a 6-month survival. Conclusions All 10 patients with Wilson's disease who underwent TPE had a positive outcome in terms of 6-month survival. In this first report of the ASFA apheresis registry study, we have demonstrated the value of using this registry to collect apheresis-related patient outcomes from multiple centers.

Original languageEnglish (US)
Pages (from-to)11-15
Number of pages5
JournalJournal of clinical apheresis
Volume31
Issue number1
DOIs
StatePublished - Feb 1 2016

Keywords

  • ASFA registry
  • Wilson's disease
  • copper
  • plasma exchange
  • plasmapheresis

ASJC Scopus subject areas

  • Hematology

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