14800-DvRappard

70 Chapter 4 DISCUSSION In this study, we showed that ITB is a feasible therapy in MLD patients with either predominant spastic or dyskinetic motor impairment, with a treatment course comparable to SCP. The complication rate in MLD was comparable to SCP patients and similar to rates reported in previous studies. 21,22 The dosing in the first sixmonths of ITB after pump implantationwas mostly comparable in SMLD and SCP. Only the bolus number administered in flexible programming mode was higher in the SMLD group compared to SCP, indicating that more flexible programming may be more effective in improving care and comfort in SMLD than static administration of baclofen. As relevant spasticity in MLD usually occurs rather rapidly after wheelchair dependency, especially in younger patients, ITB should be considered early in the disease course. Goals of treatment were mainly to improve care, improve sitting position and reduce pain. In this study, we did not formally evaluatewhether goalswere reached; still, families reported clear improvements on follow-up visits. A previous study on the effect of ITB on activities of daily life in SCP, DCP and progressive neurological disorders showed that caregivers were generally satisfied with the improvement in comfort and daily care. However, the group with progressive neurological disorders showed less improvement in comfort during ITB treatment than the two CP groups. This study counted only two MLD patients. 4 A limitation of this study is the small number of patients and the short follow-up time. To obtain more evidence on optimal treatment programs and efficacy of ITB in MLD, future research should address treatment course and outcomes in larger cohorts. This could likely be achieved by studying larger clinical cohorts with systematic gathering of data in multicenter studies, for example by centers of expertise participating in the European Reference Network for Rare Neurological Diseases (ERN-RND). It is important to also address goal achievement and outcomes at the level of activities as well as body structure and function, at the same time taking into account that MLD is a progressive disorder with inherent decline of function despite treatment.