title: Intravenous enzyme replacement therapy: hospital vs home.
abstract: Two surveys were carried out to establish the status of enzyme replacement therapy (ERT) for lysosomal storage diseases in Italy. The first was a national survey covering the regional reference centres (RRCs) for these diseases; replies disclosed that 57.7% of patients are on ERT, administered almost exclusively in hospital settings (local hospital 60.7%, RRC 34.8%, home 2.6%); Italian health service procedures do not support ERT at home. The second survey was a regional survey in Lombardy, involving 48 patients (six of whom were on ERT at home). According to 40% of the patients, hospital-based ERT is disruptive, causing loss of days at school/work, stress and family issues. The patients on home therapy did not have these problems. However, 93% of patients receiving ERT in hospital perceived the advantages of greater safety, closer monitoring and more support from health professionals and experts. A total of 55% were willing to receive ERT at home, but 33% were against it. This may be the result of a lack of experience with ERT at home in Italy, or because of different opinions between family members and physicians. As international experience shows that ERT at home saves healthcare resources and improves quality of life, the issue should be raised with Italian healthcare policy makers, who should ensure nursing support for home-based ERT.
authors.affiliation: Rare Metabolic Diseases Unit, Department of Paediatrics, San Gerardo Hospital, Monza, Italy.
authors.forename: Rossella
authors.initials: R
authors.lastname: Parini
authors.forename: Katia
authors.initials: K
authors.lastname: Pozzi
authors.forename: Stefania
authors.initials: S
authors.lastname: Di Mauro
authors.forename: Francesca
authors.initials: F
authors.lastname: Furlan
authors.forename: Miriam
authors.initials: M
authors.lastname: Rigoldi
mesh_terms: D000041:Absenteeism; D000328:Adult; D001294:Attitude to Health; D002648:Child; D056947:Enzyme Replacement Therapy / Q000009:adverse effects / Q000451:nursing / Q000706:statistics & numerical data*; D019538:Health Care Surveys; D018718:Home Infusion Therapy / Q000009:adverse effects / Q000451:nursing / Q000706:statistics & numerical data*; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D007558:Italy / Q000453:epidemiology; D016464:Lysosomal Storage Diseases / Q000188:drug therapy* / Q000453:epidemiology / Q000235:genetics; D009313:National Health Programs / Q000458:organization & administration; D020380:Needs Assessment; D010818:Practice Patterns, Physicians' / Q000458:organization & administration; D017751:Safety Management