Two U.S. Food and Drug Administration (FDA)–approved studies underway at NYU Langone could provide vital new treatment options for young children with profound hearing loss who do not have functioning cochlear nerves, potentially paving the way for improved lifelong communication and social connection.

Measuring the Subjective Benefits of Auditory Brainstem Implants

In one first-of-its-kind prospective study, investigators evaluated 12 children with cochlear nerve deficiency who received an auditory brainstem implant (ABI). All but one of the children experienced a measurable degree of auditory benefit from their implant, says lead investigator Susan B. Waltzman, PhD, the Marica F. Vilcek Professor of Otolaryngology—Head and Neck Surgery and co-director of the Cochlear Implant Center. Many parents also reported improvement in their child’s overall quality of life, including increased self-esteem and enhanced social relationships.

“While other studies have looked at speech and hearing outcomes in pediatric ABI patients, ours is the first to examine how it might impact patients’ overall mental and physical wellbeing,” says Dr. Waltzman. “Parental feedback suggests that these children may be getting more benefit from ABIs than what we have been able to measure objectively.”

Currently, ABIs for teens and adults are FDA approved only for those with profound hearing loss, usually found in patients with neurofibromatosis type 2. NYU Langone is among a small group of specialized centers approved for off-label use of the devices in children who do not qualify for cochlear implants due to absent or deficient cochlear nerves. The FDA has limited their approval in these patients due to their relatively incremental improvement in hearing and quality of life and the higher associated surgical risk compared with cochlear implantation.

In the trial, a cochlear implant was suggested when there was evidence of a cochlear nerve and implantation was possible. This was a first step to rule out whether these patients experienced notable benefits before they received an ABI as their only potentially effective treatment option. The NYU Langone study aims to enhance the understanding of the more subjective benefits of ABIs for this specific patient population.

Extending Cochlear Implantation to the Youngest Patients

A separate, retrospective multicenter study underway at NYU Langone could support the extension of FDA approval for cochlear implantation in children with hearing loss under one year of age. The investigation will focus on safety data from more than 160 children who received implants at NYU Langone since 2012. Based on these results, the FDA could consider lowering the approved age for implantation from one year to nine months, says Dr. Waltzman, who is leading the effort at NYU Langone.

Previous on-site studies showed no increase in adverse events among the youngest implant patients compared with older children or adults, suggesting that the procedure is safe and beneficial for younger children. Lowering the approved age to nine months would make a significant difference for many families who currently must wait longer in order to qualify for insurance coverage, adds Dr. Waltzman. It also allows them to realize the cumulative benefits that begin with early communication when hearing is restored.

“Earlier implantation gives children a leg up in language and speech development because they experience hearing at a time when their brain is still developing,” she says. “Children who received an implant at five or six months of age are doing so well that you often can’t tell they were born with hearing loss at all.”