Use of complementary and alternative medicine by patients with lysosomal storage diseases.
OBJECTIVE
To evaluate the extent of complementary and alternative medicine use and perceived effectiveness in patients with lysosomal storage diseases.


METHODS
A 26-item survey was distributed to 495 patients with type 1 Gaucher, Fabry, and type B Niemann-Pick diseases who were seen at the Lysosomal Storage Disease Program at the Mount Sinai School of Medicine. Survey responses were entered into an access database and analyzed using descriptive statistics.


RESULTS
Surveys were completed by 167 respondents with an overall response rate of 34%. Complementary and alternative medicines were used by 45% of patients with type 1 Gaucher disease, 41% of patients with Fabry disease, and 47% of patients with type B Niemann-Pick for symptoms related to their disease. Complementary and alternative medicines were used most frequently by adult females (55%), in patients who reported having one or more invasive procedures due to their disease, patients who use one or more conventional medical therapies, or those with depression and/or anxiety. Overall perceived effectiveness of complementary and alternative medicine supplements was low; however, complementary and alternative medicine therapies were perceived as effective.


CONCLUSIONS
Complementary and alternative medicines are commonly used among patients with lysosomal storage diseases. Assessment of the effectiveness of these approaches in the lysosomal storage diseases is needed, and physicians should be aware of complementary and alternative medicine therapies used by patients to evaluate safety and possible drug interactions.
Balwani
Manisha
M
Department of Genetics and Genomic Sciences, Mount Sinai School of Medicine, New York City, NY, USA. manisha.balwani@mssm.edu
Fuerstman
Laura
L
Desnick
Robert J
RJ
Buckley
Brian
B
McGovern
Margaret M
MM
D000293:Adolescent; D000328:Adult; D002648:Child; D000529:Complementary Therapies / Q000706:statistics & numerical data*; D005260:Female; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000453:epidemiology / Q000523:psychology / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D017060:Patient Satisfaction; D010465:Perception / Q000502:physiology; D010821:Physicians, Family / Q000706:statistics & numerical data; D016896:Treatment Outcome; D055815:Young Adult