chemotherapy-induced hearing loss in pediatrics
“This was hands-down the most empowering oncology-related event I have ever participated in. My daughter felt heard, appreciated and validated in a way that she, as a survivor struggling with serious late effects, has not felt at other events.”
In September of 2018, Children’s Cancer Cause co-hosted an Externally-Led Patient Focused Drug Development (EL-PFDD) workshop on chemotherapy-induced hearing loss in pediatrics, along with the Children’s Brain Tumor Foundation, Mattie Miracle Cancer Foundation, and Momcology.
The pediatric patient population with chemotherapy-induced hearing loss represents a segment of the cancer survivor community with a severe burden of disease with great unmet medical and psychosocial need. In the U.S., approximately 5,000 children with cancer receive platinum-based chemotherapy such as cisplatin and carboplatin as their first or second line treatment annually. Approximately 50-60% of children treated with cisplatin and 5-20% of those treated with carboplatin acquire some degree of irreversible hearing loss.
The half-day meeting followed the FDA model for externally-led PFDD meetings and featured a series of facilitated panel and audience discussions, and a webinar with live polling focused on providing the FDA and other stakeholders with patient views on burden of the condition, current and potential future treatments, a risk-benefit assessment, and clinical trials.
This EL-PFDD was an incredibly empowering day for childhood cancer survivors and family members, giving them a long-deserved spotlight to share their very personal stories about the severe impact that hearing loss has had on their lives and their families.
Approximately 250 stakeholders participated, either in person or via webcast, and we heard over and over again about the impactful and emotional power of this event.
This workshop was made possible through generous sponsorships from Decibel Therapeutics, Fennec Pharma, Frequency Therapeutics, Novartis, Otonomy, Sensorion, and Spiral Therapeutics.
Among the key lessons learned:
“I feel left out and isolated which makes me feel like I’m not part of this world. I’m sad about that. The same drugs that saved my life in some way also took it away. I’m alive, but I don’t feel alive because I feel alone and isolated in this quiet road I live on.”
Survivors with hearing loss report severe social isolation and anxiety as their single most challenging quality-of-life issue, describing difficulties in trying to keep up at school or interact with colleagues at work.
Although patients use a variety of devices, therapies, and adaptation strategies to manage the consequences of hearing loss, the effectiveness is limited and each modality has significant downsides, with 37% of respondents indicating that these interventions did not help at all.
Hearing aids are the most widely-used intervention but can have significant disadvantages, including discomfort, expense, reliance on battery life, and poor performance in noisy environments.
The number one issue that worries caregivers and patients the most about hearing loss is that its severity will worsen, impacting related issues like tinnitus and balance.
