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Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease
- Source :
- Molecular Genetics and Metabolism. 120:111-115
- Publication Year :
- 2017
- Publisher :
- Elsevier BV, 2017.
-
Abstract
- The introduction of a home therapy option during clinical trials of velaglucerase alfa in patients with type 1 Gaucher disease marked the first time that home infusions have been permitted during a clinical trial for an investigational drug for Gaucher disease. Home infusions were an available option in 4 open-label velaglucerase alfa clinical studies to eligible patients who received their initial infusions at a clinic. Patients who participated in the home therapy option and received at least 10% of their infusions at home (n=100) received a range of 11.6%-100% of their scheduled infusions at home (median 87.5%), excluding infusions received at the clinic during protocol-mandated visits. The length of time over which individual patients received home therapy ranged from 13days to 4.56years (median 0.57years). During the time that home therapy was available, 2904 of 3572 (81.3%) infusions were administered at home. Ten patients experienced 62 infusion-related adverse events (IRAEs) during 38 home infusions, with malaise, pain, hypertension, fatigue, and headache being reported most frequently. No notable differences were found between the type and severity of IRAEs experienced at home and those experienced at the clinic. Home infusions administered by trained and qualified medical personnel were successfully introduced into the velaglucerase alfa clinical development program, and fewer than 10% of patients experienced IRAEs in the home setting. Local labeling and practice guidelines should be consulted for administration of velaglucerase alfa infusions at home.
- Subjects :
- Male
medicine.medical_specialty
Pediatrics
Home therapy
Endocrinology, Diabetes and Metabolism
Biochemistry
Malaise
03 medical and health sciences
0302 clinical medicine
Endocrinology
Genetics
Humans
Medicine
Enzyme Replacement Therapy
In patient
Prospective Studies
Infusions, Intravenous
Adverse effect
Molecular Biology
Gaucher Disease
business.industry
Velaglucerase alfa
Type 1 Gaucher Disease
Enzyme replacement therapy
Surgery
Clinical trial
Treatment Outcome
030220 oncology & carcinogenesis
Glucosylceramidase
Female
medicine.symptom
business
030215 immunology
medicine.drug
Subjects
Details
- ISSN :
- 10967192
- Volume :
- 120
- Database :
- OpenAIRE
- Journal :
- Molecular Genetics and Metabolism
- Accession number :
- edsair.doi.dedup.....24a9300e0b2eecc4ad6bc0999d51c0c0