The didanosine Expanded Access Program: safety analysis.

Abstract

The didanosine Expanded Access Program was designed by the Bristol-Myers Squibb Company (Princeton, NJ) to provide didanosine for treatment of patients who could not be enrolled in clinical trials. This program consisted of two protocols: a treatment IND (investigational new drug) protocol for patients intolerant of zidovudine and an open-label protocol for patients whose clinical condition was deteriorating despite continued therapy with zidovudine. Information from the safety data base derived from study of the first 7,806 patients enrolled has shown that the major adverse events associated with didanosine therapy are pancreatitis (which can be life-threatening), peripheral neuropathy, and diarrhea. Pancreatitis was reported for 5% of the patients. Those with a history of pancreatitis were more likely to develop pancreatitis. In contrast to zidovudine, didanosine has been found to be minimally myelosuppressive. In general, hematologic parameters remained stable, especially for patients who entered the program with normal baseline values. The results of this program suggest that patients pretreated with zidovudine for prolonged periods are able to tolerate didanosine well.