Effect of ganciclovir therapy on hearing in symptomatic congenital cytomegalovirus disease involving the central nervous system:: A randomized, controlled trial

Objective To evaluate the efficacy and safety of gancielovir therapy in neonates with congenital cytomegalovirus (CMV) disease. Study design Neonates with symptomatic CMV disease involving the central nervous system were randomly assigned to receive 6 weeks of intravenous gancielovir versus no treatment. The primary end point was improved brainstem-evoked response (BSER) between baseline and 6-month follow-up (or, for patients with normal baseline hearing, normal BSER at both time points). Results From 1991 to 1999, 100 patients were enrolled. Of these, 42 patients had both a baseline and 6-month follow-up BSER audiometric examination and thus were evaluable for the primary end point. Twenty-one (84%) of 25 gancielovir recipients had improved hearing or maintained normal hearing between baseline and 6 months versus 10 (59%) of 17 control patients (P = .06). None (0%) of 25 gancielovir recipients bad worsening in hearing between baseline and 6 months versus 7 (41%) of 17 control patients (P < .01). A total of 43 patients had a BSER at both baseline and at I year or beyond. Five (21%) of 24 gancielovir recipients had worsening of hearing between baseline and greater than or equal to 1 year versus 13 (68%) of 19 control patients (P <.01). A total of 89 patients had absolute neutrophil counts determined during the course of the study; 29 (63%) of 46 ganciclovir-treated patients had grade 3 or 4 neutropenia during treatment versus 9 (21%) of 43 control patients (P <.01). Conclusions Ganciclovir therapy begun in the neonatal period in symptomatically infected infants with CMV infection involving the central nervous system prevents hearing deterioration at 6 months and may prevent hearing deterioration at ! I year. Almost two thirds of treated infants have significant neutropenia during therapy.