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Long-term effectiveness of enzyme replacement therapy in children with Gaucher disease: results from the NCS-LSD cohort study |
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| Authors: | L. J. Anderson W. Henley K. M. Wyatt V. Nikolaou S. Waldek D. A. Hughes G. M. Pastores S. Logan |
| Affiliation: | 1. Institute of Health Research, University of Exeter Medical School, Veysey Building, Salmon Pool Lane, Exeter, Devon, UK, EX2 4SG 2. Salford Royal NHS Foundation Trust, Salford, UK 3. Department of Haematology, Royal Free Campus, University College, London, UK 4. Department of Medicine/National Centre for Inherited Metabolic Disorders, Mater Misericordiae University Hospital, Eccles Street, Dublin 7, Ireland |
| Abstract: | ObjectivesTo determine the effectiveness of enzyme replacement therapies (ERT) for children with Gaucher disease (GD).DesignA longitudinal cohort study including prospective and retrospective clinical data. Age- and gender-adjusted treatment effects were estimated using generalised linear mixed models. Children on treatment contributed data before and during treatment. Children not on treatment contributed natural history data.ParticipantsConsenting children (N?=?25, aged 1.1 to 15.6 years) with a diagnosis of GD (14 with GD1 and 11 with GD3) who attended a specialist treatment centre in England. At recruitment, 24 patients were receiving ERT (mean treatment duration, 5.57 years; range 0-13.7 years).Outcome measuresClinical outcomes chosen to reflect disease progression, included platelet count; haemoglobin and absence/presence of bone pain.ResultsDuration of ERT was associated with statistically significant improvements in platelet count (p?p?p?=?0.02). The magnitude of effect on haematological parameters was greater in children with GD3 than in those with GD1.ConclusionsThese data provide further evidence of the long-term effectiveness of ERT in children with GD. |
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