P179 Lung-based assessment in Anderson Fabry Disease (AFD) demonstrates differential galactosidase a enzyme (GLA) activity at blood and tissue sites

N. T. Shafi, D. A. Hughes, R. Baker, C. Smith, J. Hurst, A. Mehta, M. Lipman
2010 Thorax  
from baseline to Week 12 (FP/FORM: +0.196 L; FP/SAL: +0.257 L). The LS mean difference in change in pre-dose FEV 1 was e0.061 L between treatments (95% CI e0.161 to 0.040). Non-inferiority of FP/FORM to FP/SAL was demonstrated (the lower limit of the 95% CI exceeded the acceptance limit of e0.2 L). In total, 72.3% (73/101) patients started on FP/FORM 250/10 mg and 75.2% (76/101) on FP/ SAL 250/50 mg. Eight patients (FP/FORM: n¼5; FP/SAL: n¼3) required an increase in dose. Similar numbers of
more » ... ents treated with FP/FORM and FP/SAL discontinued due to lack of efficacy (FP/ FORM: n¼1; FP/SAL: n¼2). Twenty-three patients (11.4%) experienced mild or moderate asthma exacerbations. Four patients (2.0%) experienced severe exacerbations (FP/FORM: n¼3; FP/SAL: n¼1; p¼0.621). Overall, 23.8% of patients experienced at least one AE. The rate was the same in both treatment groups (24/101). Most AEs were mild or moderate. No clinically important changes in laboratory results, vital signs or ECGs were observed. Conclusion FP/FORM and FP/SAL had similar efficacy and safety profiles. Abstract P177 Table 1 Pre-dose FEV 1 at Week 12eper protocol set n Pre-dose FEV 1 (L) Week 12 Difference FP/FORM L FP/SAL LS mean 95% CI LS mean 95% CI P value FP/FORM 96 2.402 2.324 to 2.481 À0.061 À0.161 to 0.040 0.007 FP/SAL 95 2.463 2.384 to 2.543
doi:10.1136/thx.2010.151043.30 fatcat:v7nepc744ng4rcmardelbuheni