ORAL BECLOMETHASONE DIPROPIONATE FOR TREATMENT OF HUMAN INTESTINAL GRAFT-VERSUS-HOST DISEASE

Intestinal graft-versus-host disease (GVHD) causes anorexia, vomiting, abdominal pain, and diarrhea. We investigated oral beclomethasone dip ropionate (BDP), a potent, topically active corticosteroid, as therapy for this disease. Forty-two allogeneic marrow-graft recipients with b iopsy-proven intestinal graft-versus-host disease of mild-to-moderate severity received BDP (8 mg daily) for up to 28 days. Weekly symptom s cores, oral intake, and surveillance throat and stool cultures were co mpared with baseline values. Adrenal testing was performed serially in patients not receiving concurrent prednisone. Improvement was seen in appetite (P<0.001), oral intake (P<0.001), nausea (P=0.013), and diar rhea (P=0.02) over the course of therapy, and an overall beneficial re sponse was observed in 72% of 40 evaluable patients, Surveillance cult ures of throat and stool showed no increase in bacterial or fungal col onization over time. The adrenal axis became suppressed in 11 of 20 ev aluable patients (55%) but suppression was not a prerequisite for clin ical response, as 6 of 9 patients who retained normal adrenal function improved clinically. We conclude that oral BDP is a safe and effectiv e treatment for mild-to-moderate intestinal graft-versus-host disease, Systemic absorption probably occurs, but adrenal suppression is not a prerequisite for clinical efficacy, suggesting that the biological ef fect is primarily topical. BDP should be further investigated as a top ical therapy for intestinal GVHD.