Agalsidase beta treatment is associated with improved quality of life in patients with Fabry disease: findings from the Fabry Registry.
OBJECTIVE
To evaluate the effect of agalsidase beta on longitudinal health-related quality of life in patients with Fabry disease.


METHODS
The SF-36® Health Survey was used to measure health-related quality of life in Fabry Registry patients. Seventy-one men and 59 women who were treated with agalsidase beta (median dose: 1.0 mg/kg/² weeks) and who had baseline and at least 2 yearly posttreatment health-related quality of life measurements were included in these analyses. A repeated measures model was used to analyze change in score from baseline.


RESULTS
Men improved in the physical component summary and in all eight scales of the SF-36 after 1 and 2 years and in the mental component summary after 1 year of agalsidase beta treatment (P < 0.05). Women improved in the mental component summary and in six of the eight scales after 1 and/or 2 years of treatment. Patients whose baseline SF-36 scores were below the median showed the greatest improvements. These responses were comparable with or greater than the published effects of various treatments for multiple sclerosis, rheumatoid arthritis, central neuropathic pain, and Gaucher disease.


CONCLUSIONS
Long-term treatment with agalsidase beta resulted in substantial improvements in health-related quality of life in both men and women; the effect was more pronounced in men.
Watt
Torquil
T
Department of Endocrinology, National University Hospital, Copenhagen, Denmark.
Burlina
Alessandro P
AP
Cazzorla
Chiara
C
Schönfeld
Dorothee
D
Banikazemi
Maryam
M
Hopkin
Robert J
RJ
Martins
Ana Maria
AM
Sims
Katherine
K
Beitner-Johnson
Dana
D
O'Brien
Fanny
F
Feldt-Rasmussen
Ulla
U
D000328:Adult; D000368:Aged; D000795:Fabry Disease / Q000188:drug therapy*; D005260:Female; D006306:Health Surveys; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D012042:Registries; D016896:Treatment Outcome; D055815:Young Adult; D000519:alpha-Galactosidase / Q000627:therapeutic use*
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta