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• W3112908754 abstract "To derive equations to support projecting health outcomes to inform a health economic model being developed for chronic acid sphingomyelinase deficiency (ASMD). A patient-level simulation is being built to predict health outcomes for adults and children with chronic ASMD (types B and A/B) receiving the current standard of care. The model was conceptualized based on literature, treatment guidelines, and extensive consultation with clinical experts. The framework developed aims to capture respiratory decline over time (% predicted diffusion capacity [DLCO]) and associated risks of respiratory complications (exacerbations, infections), oxygen use, and lung transplant; increase spleen volume and risk of thrombocytopenia and bleeding; increase liver volume and complications (cirrhosis, liver failure, cancer) and transplant; and reduced weight in children. Data from a previously published prospective, multicenter (US, EU, Brazil), longitudinal study (59 patients; 6-11 years follow-up) were analyzed to explore deriving equations to support projecting outcomes (DLCO, spleen volume, platelet count, and weight for age z-score). Selected outcomes were modeled using repeated-measures mixed-effects (RMME) models with random intercepts, and random slope for DLCO. For children (median age 12) and adults (median age 32) at baseline, baseline mean DLCO values were 61.8% and 54.3% predicted, mean spleen volumes 12.9 and 10.1 multiples of normal (MN), mean liver volumes 2.2 and 1.9 MN, and mean platelet counts 160 and 158x109/L. DLCO decreased linearly with age by 0.9 per year (SD=1.4; p=0.003). Mean platelet count decreased with spleen volume by -2.3x109/L per MN in children (p=0.13) and by 6.4x109/L per MN in adults (p<0.001). Weight for age z-scores in children were U-shaped (p=0.001 for age, <0.001 for age2) with a minimum at 12.3 years. These functions will be used to project long-term outcomes and are a potential source of evidence to inform the development of the conceptual model for ASMD." @default.
• W3112908754 created "2020-12-21" @default.
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• W3112908754 date "2020-12-01" @default.
• W3112908754 modified "2023-09-30" @default.
• W3112908754 title "PRO110 A Framework for Developing a MODEL of Chronic Acid Sphingomyelinase Defficiency" @default.
• W3112908754 doi "https://doi.org/10.1016/j.jval.2020.08.1846" @default.
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