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

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.

Reversal of Acute Liver Failure Due to Wilson Disease by a Regimen of High‐Volume Plasma Exchange and Penicillamine

“…Plasma exchange in WD‐ALF has been reported as a bridge to OLT, as treatment removes ceruloplasmin‐bound and albumin‐bound copper, decreasing hemolysis and renal failure and perhaps also providing unsaturated ceruloplasmin and albumin to the patient. HVP improves survival in ALF of all causes .…”

Registry development for thrombotic microangiopathies: Biting off more than can be chewed?

“…To maximize the likelihood of achieving the National Heart Lung Blood Institute (NHLBI) sponsored Working Group in Therapeutic Apheresis recommendations and obtaining funding support for apheresis medicine studies, focused analyses of patient populations with specific disease processes will be necessary [8]. The ASFA Apheresis Registry Subcommittee has a successful history of developing registries to characterize patients with diseases treatable by apheresis medicine [9,10]. Thus, it is fitting that a national organi-zation such as ASFA, the premier apheresis society in North America, serves as the initial sponsor to answer focused and clinically relevant apheresis-centric questions about severely ADAMTS13 deficient acquired TTP.…”

Plasmapheresis; An Experience in Blood Bank of a Tertiary Care Hospital in Peshawar

“…3,4 There are many life style modifying disorders that require plasmapheresis as first-line therapy, or in conjunction with other treatment options (like thymectomy and use of steroids in many, or cytotoxic drugs) are as follows: GuillainBarre syndrome (GBS), Myasthenia gravis (MG), Thrombotic thrombocytopenic purpura (TTP) Hyperviscosity syndromes and Wilson disease etc. 5 Present study was conducted to determine the frequency of major indications require plasmapheresis in blood bank of Lady Reading …”