Nabilone for the treatment of gastrointestinal dystonia for children and young people with severe neurodisabilities: a single-centre case series

Background For children and young people with severe neurodisabling conditions (CYPSND), the clinical constellation of pain behaviour, retching, bloating, abdominal distension and constipation/pseudo-obstruction can be referred to as gastrointestinal dystonia (GID). Nabilone, a synthetic analogue of the active component tetrahydrocannabinol found in cannabis, has a licence in paediatrics for the treatment of severe chemotherapy-induced nausea. We aim to describe our experience of using nabilone for CYPSND with GID. Methods Approval was sought on a named patient basis from the clinical directorate and lead pharmacist on the basis that; patients fulfilled our criteria for GID, patients had been trialled on medical therapies, jejunal feeding and blended diet, concomitant medication reviewed in multidisciplinary team including tone management and to review potential exacerbants to GID. Patients were admitted for 48 hours for monitoring of temperature, pulse, respiratory rate, oxygen saturations and symptoms using a modified paediatric pain score. Nabilone was commenced at 250 µg daily and then incremented in 250 µg doses to a dosage of <18 kg: 500 µg two times per day and 18–27 kg: 500 µg three times a day. Efficacy was assessed by parents’ and clinicians’ perception, PedsQL V.3.0 (gastrointestinal subset questions) and sleep diary, prior to treatment, 1 month and 6 months after stable dose, and median scores were analysed by paired Student’s t-test (p<0.05). Results In four of five suitable patients, the PedsQL score was higher after commencing treatment and there was a sustained improvement at 6 months. Patient median total PedsQL Quality of Life (QOL) scores were 46 (28–50) prior to treatment and 68.5 (58–74) 1 month after commencing treatment (p=0.032.) After 6 months, the median PedsQL QOL score was 73 (51–82) (p=0.017.) Conclusions Nabilone shows promise in treating GID, a poorly understood debilitating complication of severe neurodisability. In particular, symptoms of nausea, retching and pain on feeding were all reduced in our cohort.