Enzyme replacement therapy in the home setting for mucopolysaccharidosis VI: a survey of patient characteristics and physicians' early findings in the United States.
Galsulfase, a Food and Drug Administration-approved enzyme replacement therapy for mucopolysaccharidosis VI, is administered once weekly in a hospital setting as a 4-hour intravenous infusion. To improve convenience and alleviate family responsibilities associated with clinic visits, some physicians are transitioning appropriate patients to home infusion therapy. An online survey was conducted with 3 physicians treating 4 patients with mucopolysaccharidosis VI to better understand the factors motivating the transition to home infusion therapy, identify characteristics of appropriate candidates, and evaluate the potential impact on the lives of patients and their families. Survey results showed that home infusion may offer patients and their families increased flexibility of schedule and enhanced family life.
Tifft
Cynthia
C
Division of Genetics & Metabolism, Children's National Medical Center, Washington, District of Columbia 20010, USA. ctifft@cnmc.org
Proud
Virginia
V
Levy
Paul
P
DeMarco
Kristin
K
Nicely
Helen
H
Turbeville
Sean
S
D003625:Data Collection; D018718:Home Infusion Therapy; D006801:Humans; D007262:Infusions, Intravenous; D009087:Mucopolysaccharidosis VI / Q000188:drug therapy*; D020051:N-Acetylgalactosamine-4-Sulfatase / Q000008:administration & dosage* / Q000627:therapeutic use; D010820:Physicians; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D014481:United States
D011994:Recombinant Proteins; D020051:N-Acetylgalactosamine-4-Sulfatase; C508864:galsulfase