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Abstract
To determine the effectiveness of enzyme replacement therapies (ERT) for children with Gaucher disease (GD). A 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. Consenting children (N = 25, aged 1.1 to 15.6years) 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, 5.57years; range 0-13.7years). Clinical outcomes chosen to reflect disease progression, included platelet count; haemoglobin and absence/presence of bone pain. Duration of ERT was associated with statistically significant improvements in platelet count (p < 0.001), haemoglobin (p < 0.001), and reported bone pain (p = 0.02). The magnitude of effect on haematological parameters was greater in children with GD3 than in those with GD1. These data provide further evidence of the long-term effectiveness of ERT in children with GD.
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