During OTO EXPO, panelists discussed advancements in pediatric hearing loss treatment, focusing on early CMV detection, cochlear implants, and emerging gene therapy.
Key Highlights:
- CMV and Hearing Loss: Early detection of congenital CMV is crucial, as antiviral treatment like Valganciclovir can prevent hearing deterioration if administered within the first few weeks of life.
- Cochlear Implants: Currently the most effective treatment for pediatric hearing loss, especially for progressive or sudden cases.
- Gene Therapy: Emerging as a promising future treatment, with early clinical trials showing success in restoring hearing, but it is not yet a replacement for cochlear implants.
- Advice to Parents: Physicians recommend not delaying current treatments like cochlear implants while waiting for gene therapy, which is still in experimental stages.
Advances Unveiled at Annual Otolaryngology Meeting in Miami
The American Academy of Otolaryngology-Head and Neck Surgery Foundation’s annual meeting and expo, in stunning (and humid) Miami, Florida, was a significant gathering of surgeons, physicians, medical students, and otolaryngology product and service exhibitors.
Otolaryngology physicians from across the United States and internationally converged in the Miami Beach Convention Center for the conference, which covered clinical subjects around cancer immunology, gender-affirming facial feminization surgery, chronic migraines, and other consequential topics in the field. One presentation—given at every AAO-HNSF meeting since 2007 by Dr. Margaret Kenna and Dr. Daniel Choo, joined this year by Dr. John Grienwald—discussed revelatory advancements for the specialty of pediatric hearing-related surgical interventions.
The talk, “Pediatric Hearing Loss: What’s New in Genetics and Gene Therapy, Congenital CMV, and Imaging,” is adjusted yearly following emerging research and centers on cutting-edge knowledge in new treatments and diagnostic practices in pediatric hearing loss.
CMV in Congenital and Acquired Child Hearing Loss
The presentation opened with a staggering statistic on childhood hearing loss: 1 to 3 out of 1,000 children in the United States will experience congenital (pre-birth) or acquired (post-birth) hearing loss. While presenters later delineated the relationship between later onset hearing loss and genetic factors, they initially spoke about the relation between CMV (Cytomegalovirus) and pediatric hearing loss. CMV, a relatively benign cold-like virus for most, is dangerous for infants, pregnant women, and those with compromised immune systems.
According to estimates provided at the talk, one-third of all American children will have contracted CMV by the age of 5. Despite its status as a common virus, when a pregnant woman becomes infected with CMV, there is a 40% chance that the fetus will contract the virus. This leads to around 1 out of every 200 children being born with CMV. For these children infected in vivo, there is a higher likelihood of hearing impairment.
When detected very early, particularly within the first month after birth, a child can be inculcated with an antiviral medication, Valganciclovir. A recent controlled study on the use of Valganciclovir, published in The Journal of Pediatrics, found that “[h]earing deterioration was more likely in the control group compared with the treatment group.” Overall, the researchers found that:
“6 weeks of valganciclovir, initiated in the first 3 months of life, has a beneficial effect on hearing outcome in children with hearing loss and clinically inapparent cCMV.”
While antiviral medication is one modality of addressing pediatric hearing impairment, it requires an ideal diagnostic situation: The child needs to be recognized as being infected with CMV upon birth and usually tested within the first three weeks to be treated as quickly as possible. If not, the subsequent option has become standard for treating progressive and even sudden hearing loss: Cochlear implants.
Pediatric Hearing Treatment: Cochlear Implants and Gene Therapy
Cochlear implants have been a profound restorative practice for hearing loss since it was successfully implanted in the first human in 1961. Currently, it is the best available intervention for most cases of hearing loss in children.
However, very recent research has found promising results for gene therapy as an intervention in pediatric hearing loss. Dr. Grienwald discussed the findings of a 2024 peer-reviewed article published in Advanced Science that found:
“Gene therapy has the potential to restore the hearing function of deafness patients.”
The study used two pediatric cases, a five-year-old and an eight-year-old, who were both given gene therapy in one ear. Researchers discovered that, after being administered injections of gene therapy, the children could hear only out of the ear that had received the injections.
These findings are corroborated by a well-known piece published in the New York Times that reported the case of a formerly deaf 11-year-old boy in the United States who, after being given gene therapy, was able to hear for the first time.
Do Not Delay Treatment of Hearing Loss
Although these advancements are exceedingly promising and optimism-inducing for parents of children experiencing deafness or progressive hearing loss, all three presenters emphasized the temporal limitations of gene therapy. When responding to an audience question on how a physician should respond to a parent considering waiting for gene therapy, the consensus among the presenting physicians was that, due to gene therapy being in the early phases of clinical trials, parents should not delay treatment of hearing loss. Instead, they insisted parents should expeditiously employ the currently available and crucially effective treatment of pediatric cochlear implants.
The only exception the presenters noted were children experiencing hearing loss attributable to mutations in the otoferlin (OTOF) gene—since those cases are being targeted in clinical trials. However, the presentation ended on a hopeful future-oriented note: The presenters commented that, in five years, once gene therapy for pediatric hearing loss has undergone the requisite clinical trials, the response to parents may be entirely different.
