Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y
Copyright policy )Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y
Previous studies have shown that elosulfase alfa has a favorable efficacy/safety profile in Morquio A patients aged ≥5 y. This study evaluated safety and impact on urine keratan sulfate (uKS) levels and growth velocity in younger patients.Fifteen Morquio A patients aged <5 y received elosulfase alfa 2.0 mg/kg/week for 52 wk during the primary treatment phase of a phase II, open-label, multinational study. Primary endpoint was safety and tolerability; secondary endpoints were change in uKS and growth velocity over 52 wk.All 15 patients completed the primary treatment phase. Six of 743 infusions (0.8%) administered led to adverse events (AEs) requiring infusion interruption and medical intervention. Eleven patients (73.3%) had ≥1 study drug-related AE, mostly infusion-associated reactions. Mean z-score growth rate per year numerically improved from -0.6 at baseline to -0.4 at week 52. Comparison to untreated subjects of similar age in the Morquio A Clinical Assessment Program study showed a smaller decrease in height z-scores for treated than for untreated patients. Mean percent change from baseline in uKS was -30.2% at 2 wk and -43.5% at 52 wk.Early intervention with elosulfase alfa is well-tolerated and produces a decrease in uKS and a trend toward improvement in growth.
- University of Manchester United Kingdom
- BioMarin (United States) United States
- University of Manchester United Kingdom
- University of California, San Francisco United States
- St Mary's Hospital United Kingdom
Male, Time Factors, 1117 Public Health and Health Services (for), Europe (mesh), Pediatrics, Chondroitinsulfatases (mesh), Clinical Research (rcdc), Enzyme Replacement Therapy (mesh), Mucopolysaccharidosis IV (mesh), GALNS protein, Preschool (mesh), Child Development, 32 Biomedical and Clinical Sciences (for-2020), Child, Male (mesh), Infusions, Intravenous, 6.2 Cellular and gene therapies, Pediatric, Humans (mesh), Time Factors (mesh), Child Development (mesh), Treatment Outcome (mesh), steroid, Body Height (mesh), Age Factors, Mucopolysaccharidosis IV, biological marker, Early Medical Intervention (mesh), Chondroitinsulfatases, Recombinant Proteins, Europe, Biomarkers (mesh), Treatment Outcome, 6.1 Pharmaceuticals, United Kingdom (mesh), 3213 Paediatrics (for-2020), Child, Preschool, Public Health and Health Services, Female, Patient Safety, Intravenous, Infusions, Clinical Sciences, 610, 1114 Paediatrics and Reproductive Medicine (for), Drug Administration Schedule, Paediatrics and Reproductive Medicine, 6.2 Cellular and gene therapies (hrcs-rac), Clinical Research, GALNS protein, human, Early Medical Intervention, 616, Recombinant Proteins (mesh), n acetylgalactosamine 4 sulfatase, Humans, Enzyme Replacement Therapy, Pediatrics (science-metrix), human, Clinical Investigation, Preschool, Pediatric (rcdc), 3202 Clinical Sciences (for-2020), Keratan Sulfate (mesh), 6.1 Pharmaceuticals (hrcs-rac), Biomedical and Clinical Sciences, Patient Safety (rcdc), keratan sulfate, Infant, Paediatrics, Infant (mesh), Drug Administration Schedule (mesh), United Kingdom, Body Height, elosulfase alfa, antihistaminic agent, Keratan Sulfate, Female (mesh), Intravenous (mesh), Age Factors (mesh), recombinant protein, Biomarkers
Male, Time Factors, 1117 Public Health and Health Services (for), Europe (mesh), Pediatrics, Chondroitinsulfatases (mesh), Clinical Research (rcdc), Enzyme Replacement Therapy (mesh), Mucopolysaccharidosis IV (mesh), GALNS protein, Preschool (mesh), Child Development, 32 Biomedical and Clinical Sciences (for-2020), Child, Male (mesh), Infusions, Intravenous, 6.2 Cellular and gene therapies, Pediatric, Humans (mesh), Time Factors (mesh), Child Development (mesh), Treatment Outcome (mesh), steroid, Body Height (mesh), Age Factors, Mucopolysaccharidosis IV, biological marker, Early Medical Intervention (mesh), Chondroitinsulfatases, Recombinant Proteins, Europe, Biomarkers (mesh), Treatment Outcome, 6.1 Pharmaceuticals, United Kingdom (mesh), 3213 Paediatrics (for-2020), Child, Preschool, Public Health and Health Services, Female, Patient Safety, Intravenous, Infusions, Clinical Sciences, 610, 1114 Paediatrics and Reproductive Medicine (for), Drug Administration Schedule, Paediatrics and Reproductive Medicine, 6.2 Cellular and gene therapies (hrcs-rac), Clinical Research, GALNS protein, human, Early Medical Intervention, 616, Recombinant Proteins (mesh), n acetylgalactosamine 4 sulfatase, Humans, Enzyme Replacement Therapy, Pediatrics (science-metrix), human, Clinical Investigation, Preschool, Pediatric (rcdc), 3202 Clinical Sciences (for-2020), Keratan Sulfate (mesh), 6.1 Pharmaceuticals (hrcs-rac), Biomedical and Clinical Sciences, Patient Safety (rcdc), keratan sulfate, Infant, Paediatrics, Infant (mesh), Drug Administration Schedule (mesh), United Kingdom, Body Height, elosulfase alfa, antihistaminic agent, Keratan Sulfate, Female (mesh), Intravenous (mesh), Age Factors (mesh), recombinant protein, Biomarkers
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