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Beyond body structure in intrathecal baclofen therapy

Over the past two decades there have been three systematic reviews published on intrathecal baclofen (ITB) treatment for children with cerebral palsy (CP). A separate systematic review focused specifically on the effect of ITB for ambulant children with spasticity or dystonia of cerebral origin but found only low-level evidence for those with spasticity and no evidence for those with dystonia. The authors concluded there was no evidence to support the clinical use of ITB in ambulant individuals with hypertonicity without further rigorous longitudinal studies. Buizer et al. have now published a systematic review of ITB therapy for all children, with important additional information on the effect of ITB for children with neurological conditions other than CP. Table IV(a) of their paper shows effect sizes on different domains of the International Classification of Functioning, Disability and Health: Children and Youth Version (ICF-CY) for those with CP, and it remains evident that the majority of research is still within the domain of body structure and function. For those with other neurological conditions, there is no evidence of the effectiveness of ITB beyond this domain. Whilst most of the research evidence for ITB supports an effective reduction in spasticity, patients and their carers typically do no seek ITB for that specific purpose. Goalsetting using standardized methods such as the Canadian Occupational Performance Measure or Goal Attainment Scaling allow for patient relevant goals, with studies finding most patients with CP undergoing ITB treatment seek improvements related to dressing, positioning and transfers, pain, and comfort. What patients and clinicians want to know is: will ITB be effective for my (or my patient’s) specific goals and condition, and what are the risks of treatment? To date we have insufficient information to answer this question reliably, particularly for those with conditions other than CP. The ITB test dose, either by ITB bolus injection or intrathecal infusion, may help point towards whether the patient’s goals will be achieved after pump implantation. However, some clinicians believe little is gained from the test dose other than temporary spasticity reduction. They proceed straight to a pump implant, counselling the patient that the full effect of treatment may not be evident for several weeks or months and only after careful dose titration. A previous commentary in this journal noted that a better understanding of patient characteristics and how they influence goal achievement may help answer the above question. Buizer et al. also state the need for ITB researchers to address all ICF-CY components and collaborate to pool data to determine what patient characteristics predict optimal outcome. In Australia there are now two such initiatives, one for ITB and the other for selective dorsal rhizotomy (protocol submitted for publication by SDR-AUS registry group). Both these Australian research groups welcome the opportunity to pool data, allowing for more robust statistical analyses. Harmonizing outcome data sets for these procedures between different centres and countries may also allow for comparison of different surgical techniques and dosage regimens, as well as helping to predict which patients are most at risk for adverse events.