50 CHAPTER 2 Demographics were closely matched (table 1) and there were no significant differences between the treatment arms at baseline. Four children were previously treated with anticholinergic medication prescribed by clinicians in other (some of them foreign) institutions. Among these four patients, three children were treated with glycopyrrolate and one patient was treated with scopolamine patches. Reasons for discontinuation and referral were lack of effect and side effects. Follow-up attendance at 8 weeks was 100% and 98.1% at 32 weeks. Missing data were limited: the DQ was missing for 2 patients at 8 weeks and for 4 patients at 32 weeks. One patient was not able to attend for 32 weeks visit due to personal circumstances. For this patient the subjective measurements were obtained by telephone. For two patients the DQ at 8, and 32 weeks was unreliable due to spitting of saliva during the assessment. For one patient the DQ at 32 weeks was omitted because the patient kept her hands in her mouth during the measurement. For another patient the “DQ during activity” at 32 weeks was substituted with “DQ in rest” due to unreliable measurements of DQ during activity. There were 10 (18,9%) and 16 (30,2%) missing values for the masked DQ at 8 weeks and 32 weeks respectively. Primary outcome Sixty-three percent of children showed a clinically significant response (≥50% reduction in the DQ or caregivers VAS) to 2-DL after 32 weeks, versus 26.9% for BoNT-A (difference 36.1%, 95 % CI 18.1-54.1, NNT 3, 95% CI 2-6). After 8 weeks, this was 88.9% for 2-DL and 53.8% for BoNT-A (difference 35.1%, 95% CI 23.6-46.6, NNT 3, 95% CI 2-4). When substituting the DQ with the (video-evaluated) masked DQ at 32 weeks, the response to 2-DL was 72.0% versus 26.9% to BoNT-A (difference 45.1%, 95% CI 32.9-57.4, NNT 2, 95% CI 2-3). After 8 weeks, this was 92.6% versus 57.7% respectively (difference 34.9, 95% CI 24.0-45.8, NNT 3, 95% CI 2-4). There was a significant association between VAS and DQ at baseline (r = 0.29, p = 0.039), 8 weeks (r = 0.52, p < 0.001), and 32 weeks (r = 0.39, p = 0.006). VAS for severity of drooling The VAS at follow-up was significantly lower after 2-DL when compared to BoNT-A (figure 2, table 2) using mixed model analyses. For both BoNT-A and 2-DL VAS was significantly (difference 19.4, p < 0.001, 95% CI 10.2 – 28.5) higher at 32 weeks
RkJQdWJsaXNoZXIy MjY0ODMw