Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

Pediatric Research
Simon A JonesPaul Harmatz

Abstract

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 i...Continue Reading

References

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Citations

Aug 27, 2016·Orphanet Journal of Rare Diseases·Christian J HendrikszJeffrey I Gold
May 11, 2016·Molecular Genetics and Metabolism·Eriko YasudaShunji Tomatsu
Nov 20, 2016·Expert Review of Clinical Pharmacology·Christian J Hendriksz
Dec 17, 2016·Molecular Genetics and Metabolism·Shaukat KhanShunji Tomatsu
Aug 29, 2019·Journal of Human Genetics·Hui Hsuan ChenShunji Tomatsu
Feb 7, 2020·International Journal of Molecular Sciences·Akari Nakamura-UtsunomiyaSatoshi Okada
Apr 26, 2019·Journal of Human Genetics·Caitlin DohertyShunji Tomatsu
Nov 18, 2018·Italian Journal of Pediatrics·Daniela ConcolinoRossella Parini
Jun 15, 2019·Orphanet Journal of Rare Diseases·Mehmet Umut AkyolUNKNOWN MPS Consensus Programme Co-Chairs
Nov 20, 2019·The Journal of Clinical Investigation·Angela C SosaAdriana M Montaño
Dec 2, 2020·Orphanet Journal of Rare Diseases·Can FiciciogluAndrew J Degnan
Mar 24, 2021·Orphanet Journal of Rare Diseases·Sebile KılavuzHalise Neslihan Önenli Mungan
Sep 3, 2021·American Journal of Medical Genetics. Part a·Marta FrigeniHope Northrup

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Software Mentioned

MorCAP
SAS ( ) Proprietary

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