Pyridostigmine for the treatment of gastrointestinal symptoms in systemic sclerosis.

BACKGROUND/PURPOSE: Symptoms of gastrointestinal dysmotility are common among patients with systemic sclerosis (SSc), and the management of severe cases is often limited by a relative lack of effective interventions. The objective of this case series was to review our experience with pyridostigmine as a treatment for patients with SSc and symptomatic gastrointestinal disease.

METHODS: This study evaluated rates of symptom improvement, side effects, medication adherence, and dose ranges for SSc patients prescribed pyridostigmine for refractory gastrointestinal symptoms over a 10-year period at a quaternary referral center. Patients were defined as responders if they remained on pyridostigmine for at least 4 weeks and clinical benefit was documented by the recorded response of the patient or by the treating physician RESULTS: Of 31 patients treated with pyridostigmine for at least 4 weeks, 51.6% reported symptomatic improvement. Constipation was the most commonly improved symptom based on prevalence prior to therapy (noted by 6/20 patients suffering with constipation). Fifteen of 31 patients reported adverse effects, most commonly diarrhea. Throughout the duration of follow-up (median 126 days, range: 28-506 days), pyridostigmine was continued by 81.3% of patients who reported symptomatic benefit and 58.1% of patients overall.

CONCLUSIONS: Pyridostigmine holds promise for the treatment of various gastrointestinal symptoms in SSc patients, particularly in patients with refractory constipation. Though side effects may limit its use, most patients who experienced benefit chose to continue therapy.