An investigational new drug treatment program for patients with gemcitabine: results for over 3000 patients with pancreatic carcinoma.

摘要: BACKGROUND An Investigational New Drug (IND) treatment program allows patients access to a drug that has shown activity against serious or life-threatening disease prior full Food and Administration (FDA) review approval. This IND program, in which with locally advanced metastatic pancreatic carcinoma were treated gemcitabine, began 1995. METHODS Eligibility criteria ≤1 chemotherapy regimen; Karnofsky performance status (KPS) of ≥50; adequate bone marrow, liver, renal function. Gemcitabine was given at dose 1000 mg/m2 weekly × 7 followed by week rest, then 3 every 4 weeks thereafter. In this disease-related symptom improvement (DRSI) defined retrospectively as 1) pain (on 7-point scale) and/or analgesic class (e.g., morphine improving codeine) KPS (≥20 points), 2) stability these three parameters 7% increase weight from baseline. RESULTS A total 3023 enrolled. At baseline, 80% them had Stage IV disease, 84% baseline ≥ 70. The median age 65 years, 56% the male. cumulative DRSI response rate after fourth cycle 18.4%. Of 982 tumor data, there 14 complete 104 partial response, for an overall 12.0% (95% confidence interval [CI], 10.0–14.0%). For 2380 survival 4.8 months CI, 4.5–5.1 months) 12-month 15%. well tolerated; only 4.6% discontinuations due adverse events. CONCLUSIONS Notable seen gemcitabine large, compassionate-use setting, findings agreement those earlier registration trials. Cancer 1999;85:1261–8. © 1999 American Society.