OBJECTIVE: To evaluate the efficacy and safety of ganciclovir therapy for congenital cytomegalovirus (CMV) infection in newborn infants. METHODS: The randomized controlled trials (RCTs) and quasi-RCTs on ganciclovir therapy for congenital CMV were reviewed in the following electronic databases: PubMed (January 1988 to January 2009), EMbase (January 1988 to January 2009), the Cochrane library (Issue 3, 2003 and Issue 1, 2009), the Chinese Journals Full-text Database (January 1994 to January 2009), the Chinese Biological Medical Disc (January 1994 to January 2009) and the Chinese Medical Current Contents (January 1994 to January 2009). Quality assessment, data extraction, and meta analysis were performed. RESULTS: Ten papers were included. Meta analysis showed that the ganciclovir therapy increased the improvement rate (91.4% vs 34.0%; P<0.01) and led CMV infection indexes to become negative in more patients (87.6% vs 15.3%; P<0.01) and decreased incidence of hearing disturbance (4.7% vs 37.2%; P<0.01) as compared with the non-ganciclovir therapy control group. The incidence of the ganciclovir-therapy-related side effects was low. CONCLUSIONS: Ganciclovir treatment may increase the improvement rate and the rate of CMV infection indexes becoming negative, and decrease incidence of hearing disturbance, with few side effects, in newborn infants with CMV infection. However the supporting evidence is not strong due to few trials and more high-quality research is needed.［Chin J Contemp Pediatr, 2010, 12 (1):35-39］