Placebo controlled double-blind study of pentoxifylline in sickle cell disease patients.

Sixty patients (37 males, 23 females, age range 7 to 34 years) suffering from sickle cell disease were treated with pentoxifylline (1200 mg/day per os, Trental 400 t.i.d.) or placebo in a double-blind randomized study of six week duration. Observation of pain frequency, intensity and duration of pain events as well as determination of various laboratory and hematologic parameters were maintained on weekly or biweekly basis, respectively, throughout the study. At the end of the trial the number of patients (14) with pain periods and the number of pain events (57) and the mean duration of pain events (4.1 days) in the drug group were significantly better than in the placebo group with 25 patients with pain, 219 pain events and 8.8 days mean pain duration. Hematocrit, red cell count, hemoglobin and platelet aggregation showed slight but significant positive changes with no corresponding alteration with placebo. The over all assessment of the therapeutic response showed 21 clearly improved patients with pentoxifylline compared to 10 such patients with placebo (p less than 0.05). Two patients on pentoxifylline and four on placebo experienced transient side effects of nausea and gastric intolerance.