Report of the ASFA apheresis registry study on Wilson's disease.
OBJECTIVE
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.
Department of Pathology, University of Alabama at Birmingham, Birmingham, Alabama.
Huy P
HP
Pham
Department of Pathology and Cell Biology, New York-Presbyterian Hospital and Columbia University, New York, New York.
Joseph
J
Schwartz
Department of Pathology, University of Michigan, Ann Arbor, Michigan.
Laura
L
Cooling
Department of Medicine, California Pacific Medical Center, San Francisco, California.
Jan C
JC
Hofmann
Department of Pathology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.|Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Haewon C
HC
Kim
Department of Laboratory Medicine and Pathology, University of Minnesota, Minneapolis, Minnesota.
Shanna
S
Morgan
Medical Division, Bloodworks Northwest, Seattle, Washington.
Monica B
MB
Pagano
Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
Jennifer
J
Schneiderman
Division of Transfusion Medicine, Mayo Clinic, Rochester, Minnesota.
Jeffrey L
JL
Winters
Department of Pathology, University of Michigan, Ann Arbor, Michigan.
Chisa
C
Yamada
Department of Laboratory Medicine, Children's National Health System, Washington, District Columbia.
Edward C C
EC
Wong
Medical Division, Bloodworks Northwest, Seattle, Washington.|Department of Laboratory Medicine, Yale University School of Medicine, New Haven, Connecticut.
Yanyun
Y
Wu
D000293:Adolescent; D000328:Adult; D001781:Blood Component Removal; D002648:Child; D005260:Female; D006527:Hepatolenticular Degeneration / Q000150:complications / Q000628:therapy*; D006801:Humans; D017114:Liver Failure, Acute / Q000209:etiology / Q000628:therapy; D016031:Liver Transplantation; D008297:Male; D008875:Middle Aged; D010951:Plasma Exchange; D011446:Prospective Studies; D012042:Registries; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
ASFA registry; Wilson's disease; copper; plasma exchange; plasmapheresis