- Randomized, controlled trial conducted in 100 patients with urine-confirmed CMV, evidence of CNS disease, =32 weeks gestations, =1200 g BW, and =1 month at baseline. Exclusion criteria included imminent death, treatment with other antiretrovirals or IVIG, creatinine >1.5 mg/dL, HIV infection, or hydranencephaly. Patients received either IV ganciclovir 6 mg/kg BID x 6 weeks or no-treatment.
- Both functional and biological assessment of hearing was performed using brainstem auditory evoked response (BAER) were done at baseline, 6 months, 1 year, and 2 years. These were all interpreted by a single audiologist who was blinded to the randomization.
- No patients treated with ganciclovir had worsening of hearing by both functional and biological assessments versus 41-42% in the no-treatment control at the 6 month follow-up (p<.001). This effect persisted after multivariate analysis; OR 21.11 (p<.01)
- Only 21% of patients treated with ganciclovir had worsening of hearing by functional or biological assessments versus 61-68% in the no-treatment control after at least 1 year of follow-up (p=.002). This effect also persisted after multivariate analysis; OR 4.38 (p=.03)
- There was a large proportion (58%) of non-evaluable patients raising the possibility of follow-up bias. These patients were also more likely to be black and premature. Premature infants may be at higher risk of adverse outcome. This represents a selection bias which the multivariate analysis attempted to correct for.
- Power analysis from the phase II trial calculated n=90 to be sufficient. This was not achieved as the trial was terminated prematurely when the interim analysis showed significant benefit.
- It is unclear whether functional and biological assessment of hearing via BAER is a sufficient surrogate measure of neurodevelopmental outcome.