Pitfalls in placebo-controlled trials in palliative care: dexamethasone for the palliation of malignant bowel obstruction

• Published in: Palliative medicine 1998-11, Vol.12 (6), p.437-442
• Main Authors: Hardy, Janet, Ling, Julie, Mansi, Janine, Isaacs, Richard, Bliss, Judith, A'Hern, Roger, Blake, Peter, Gore, Martin, Shepherd, John, Hanks, Geoff
• Format: Article
• Language: English
• Subjects: Abdomen; Adult; Aged; Aged, 80 and over; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Anti-Inflammatory Agents - therapeutic use; Biological and medical sciences; Bowel cancer; Cancer; Clinical death. Palliative care. Organ gift and preservation; Cross-Over Studies; Dexamethasone - therapeutic use; Double-Blind Method; Female; Gastroenterology. Liver. Pancreas. Abdomen; Humans; Intestinal obstruction; Intestinal Obstruction - drug therapy; Medical sciences; Middle Aged; Other diseases. Semiology; Palliative Care; Patients; Placebos; Randomized controlled trials; Steroids; Stomach. Duodenum. Small intestine. Colon. Rectum. Anus; Treatment
• ISSN: 0269-2163; 1477-030X
• DOI: 10.1191/026921698666334766

To determine the effect of dexamethasone when treating malignant bowel obstruction, 35 patients were randomized to receive intravenous dexamethasone or a placebo, crossing over to the alternate treatment arm if there had been no resolution of obstruction by day 5. This was done in two consecutive studies. Patients were stratified according to whether or not they had received specific anticancer therapy within 28 days of study. In trial 1, 15 out of 22 patients ‘responded’ (resolution of obstruction by day 5; 10 on dexamethasone, five on placebo). Eleven out of 15 patients were ‘on treatment’. In trial 2, six out of 13 responded (three on dexamethasone, three on placebo); three out of six were ‘on treatment’. When both studies are combined, 60% (21/35) patients responded (13 on dexamethasone, eight on placebo). Poor patient accrual terminated both studies. Numbers are too small to allow a combination of studies or formal statistical analysis. We are unable to make any conclusion as to the effectiveness of dexamethasone in the palliation of malignant bowel disease.