Valganciclovir Effective in Infants With Cytomegalovirus

Laird Harrison

October 10, 2013

SAN FRANCISCO — A 6-month course of oral valganciclovir improves hearing and nerve development in infants with cytomegalovirus, and works better than a 6-week course, report researchers.

This is the first major trial of oral valganciclovir for cytomegalovirus in newborns.

"This study is remarkable. It gives us confidence that we can offer therapy to babies born with symptomatic disease," coauthor Janet Englund, MD, from Seattle Children's Hospital in Washington, told Medscape Medical News.

Penelope Dennehy, MD, professor of pediatrics at Brown University in Providence, Rhode Island, presented the multicenter trial here at IDWeek 2013 after a power outage disrupted the originally scheduled presentation by David Kimberlin, MD.

Congenital cytomegalovirus is the leading nongenetic cause of deafness in the United States, and can cause delays in other neurologic developments.

A previous study found that 6 weeks of intravenous (IV) treatment with the antiviral medication ganciclovir was effective in preventing hearing loss, but the therapy has challenges (J Pediatric. 2003;143:6-25).

"To give 6 weeks of IV therapy to a baby is hard," said Dr. Englund.

To explore an alternative oral therapy, the National Institute of Allergy and Infectious Diseases' Collaborative Antiviral Study Group undertook a trial with valganciclovir, a prodrug form of ganciclovir.

Oral Form of Ganciclovir

A 16 mg/kg dose of oral valganciclovir provides the same systemic exposure to ganciclovir as 6 mg/kg of intravenous ganciclovir, said Dr. Dennehy.

The researchers enrolled 109 infants with symptomatic cytomegalovirus infections, with or without central nervous system involvement.

They randomly assigned the infants to 6 weeks of oral valganciclovir followed by placebo for the remainder of the 6-month treatment period, or to 6 months of oral valganciclovir.

The proportion of infants with abnormal hearing decreased slightly in those who got 6 weeks of the drug, but a greater decrease was seen in those who got 6 months of treatment. Improvements continued up to 24 months of age, and the differences between the groups were statistically significant at 12 and 24 months.

I think this is really, really important because we now know we can treat these kids.

The infants who got 6 months of treatment also scored higher on parts of the Bayley Scales of Infant and Toddler Development, Third Edition (Bayley III). The differences were statistically significant for language composite and receptive communication.

Also, after 6 weeks, white blood cell counts were significantly lower in the group that continued on valganciclovir.

In the first 6 weeks, the researchers interrupted the treatment for 3 subjects because of neutropenia. The neutropenia resolved in 1 week for 1 of these patients, 2 weeks for another, and 3.5 months for the third. The rate of neutropenia was significantly less in this trial than in the earlier IV trial of ganciclovir.

Asked to comment, Tina Tan, MD, a professor of pediatrics and infectious diseases at Northwestern University in Chicago, who was not involved in the study, told Medscape Medical News that the study's results are good news.

"I think this is really, really important because we now know we can treat these kids," she said.

The findings underscore the importance of testing the hearing of newborns, Dr. Tan added. Screening for cytomegalovirus in all newborns is still not helpful because the virus is pervasive, treatment can cause neutropenia, and the benefits of treatment are not known for asymptomatic newborns, she explained.

Instead, she stressed, oral valganciclovir should only be used in newborns with symptomatic infections.

Because of the interruption in the IDWeek schedule, there was no time for comments or questions from the audience after the presentation.

Dr. Englund reported receiving research support from Chimerix, Gilead, and Novartis, and a consulting fee from GlaxoSmithKline. Dr. Tan has disclosed no relevant financial relationships.

IDWeek 2013. Abstract LB-1. Presented October 5, 2013.


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