Implementation of an Institutional Protocol for the Use of Intrathecal Dexamethasone for Treatment-Refractory Pediatric Neuroinflammatory Disease.

BACKGROUND

Multiple rare diagnoses in the neuroinflammatory disease spectrum are associated with a high morbidity and mortality and are refractory to systemic immunotherapies. Owing to disease rarity, there are often barriers or delays in introducing novel treatment protocols. The development of institutional treatment protocols helps to ensure consistent and improved efficiency to help ensure optimal patient outcomes.

METHODS

A multidisciplinary team with aligned interests was created and aided in identifying the need for a new treatment regimen for patients with treatment refractory neuroinflammatory disease. This work group was convened to determine a standardized template for the management of patients requiring intrathecal therapy. After review and assessment of existing clinical case reports, and other supporting documents, the workgroup reached a consensus regarding optimal treatment approaches, created methods for distribution, and education of providers to utilize the developed clinical template.

RESULTS

We document our process for creating and implementing a treatment protocol for intrathecal (IT) dexamethasone in children with treatment-refractory neuroinflammatory disease. The treatment protocol was approved by key stakeholders across multiple disciplines as part of a collaborative effort to improve outcomes. We describe eight patients with neuroinflammatory disease treated with IT dexamethasone without adverse events.

CONCLUSIONS

There are many barriers in the introduction of novel treatment protocols in rare disease. Utilization of a multidisciplinary team can help identify barriers and facilitate protocol development, leading to alternative treatment pathways in those with rare disease. The development of a standardized treatment approach can be an important way to ensure adequate and effective treatment of patients with rare disorders. The implementation process requires institutions to dedicate resources to ensure key stakeholder input to address barriers to implementation, and to provide ongoing input following implementation to assess patient outcomes.