Appendix: Humanitarian use of misoprostol in severe refractory upper gastrointestinal disease

Abstract

Misoprostol was provided on a humanitarian basis to 157 patients with severe, often life-threatening, refractory upper gastrointestinal (UGI) disease not managed by available medical therapy (cimetidine, ranitidine, antacids, sucralfate, and/or prior surgery). A total of 162 separate clinical treatment courses were evaluated in the 157 patients for the period May 1, 1981 to May 6, 1986. Misoprostol was administered orally or via nasogastric tube, 800 μg to 1,200 μg daily in four to six divided doses, for periods of from one day to 17 months. Patients were considered to have a favorable clinical outcome if they achieved significant improvement in symptoms, hemorrhagic status, or appearance of their condition on endoscopy. Favorable clinical outcomes were observed in 52 of 83 treatment courses (63 percent) involving UGI hemorrhage and in 44 of 79 treatment courses (56 percent) for long-standing nonhemorrhagic UGI disease. A total of 116 treatment courses were for patients with a single UGI entry diagnosis; 28 treatment courses were for patients with two UGI entry diagnoses; four courses were for patients with three UGI entry diagnoses; and 14 treatment courses were for miscellaneous UGI entry diagnoses. Treatment outcomes were analyzed by the four most commonly treated UGI entry diagnoses; patients who had an initial diagnosis of refractory duodenal ulcer (n = 28), refractory gastric ulcer (n = 41), reflux esophagitis (n = 23), or hemorrhagic gastritis (n = 63) had favorable clinical outcomes of 71 percent, 58 percent, 61 percent, and 62 percent, respectively. Misoprostol was well tolerated, with the most common adverse experience being mild to moderate diarrhea. It is concluded that in humanitarian clinical trials, misoprostol was frequently associated with symptomatic relief, with improvement in UGI hemorrhage, or with endoscopic improvement in severe UGI disease that had proven refractory to available medical therapy.