Efficacy and safety of long-term phenobarbital therapy of familial cholestasis.

Two brothers with progressive familial intrahepatic cholestasis were followed during four years of phenobarbital therapy to assess its long-term safety and efficacy. Effects attributable to phenobarbital included sustained relief of pruritus, lowering of serum bilirubin levels, and enhanced hepatic excretion of 131I-rose bengal; serum bile acid levels were only transiently decreased. Two brief interruptions in therapy resulted in symptomatic deterioration. Phenobarbital did not retard growth nor impede the response to vitamin D therapy of concomitant rickets.