For the first time, NICE has recommended an innovative new medicine that can help prevent babies, children, and young people undergoing cancer treatment from losing their hearing.
How cisplatin chemotherapy can impact hearing
Cisplatin is a powerful chemotherapy medicine, which is routinely used to treat many cancers in children. Over time, cisplatin builds up in the inner ear and can cause inflammation and damage, known as ototoxicity, leading to permanent hearing loss.
Anhydrous sodium thiosulfate, also known as Pedmarqsi and made by Norgine, is recommended in final draft guidance for preventing hearing loss caused by cisplatin chemotherapy in children from the ages of one month to 17-years-old with solid tumours that have not spread to other parts of the body.
About 60% of children having cisplatin-based treatment develop irreversible hearing loss, and 283 new cases of ototoxic hearing loss were diagnosed in people under 18 in England in 2022 to 2023.
How does Pedmarqsi work?
The medicine, which is given as an infusion by a nurse or doctor, works by binding to and blocking the action of cisplatin that has not been taken up by cells and preventing damage to cells in the ear.
Evidence shows a large reduction in occurrence of hearing loss
Evidence from two clinical trials showed the treatment almost halves the incidence of hearing loss in children receiving cisplatin chemotherapy.
- In one clinical trial it was found that hearing loss occurred in 63% of children being treated with cisplatin, compared to 32.7% of children who received anhydrous sodium thiosulfate following their cisplatin treatment.
- In another trial, 56.4% of children treated with cisplatin developed hearing loss, compared with 28.6% who were given anhydrous sodium thiosulfate following cisplatin.
The trials also showed that, if children did develop hearing loss, it was less severe overall in children who had anhydrous sodium thiosulfate.
Speech and language development, functioning at school and at home, can all be affected if hearing is impaired through cisplatin chemotherapy, parents told the NICE independent committee.
Ralph Holme, Director of Research at RNID, said; “We are thrilled by the announcement that this groundbreaking drug will be made available to young patients undergoing cancer treatment to prevent hearing loss as a side effect of their cisplatin chemotherapy.
We look forward to seeing it being rolled out in hospitals across the country, and we are hopeful that all children who may benefit will soon have access to this vital treatment.
Our sincere thanks go to our supporters whose contributions have enabled RNID to provide essential insight and evidence to NICE helping to make this treatment widely accessible in England.
It is the first drug specifically developed to prevent hearing loss to be recommended for use in the NHS. This is an important milestone that will give confidence to those investing in and developing treatments for hearing loss that they can by successfully brought to market.”
Treatment to be available on the NHS
The treatment should be available on the NHS in England within three months of NICE’s final guidance being published.
Helen Knight, director of medicines evaluation at NICE, said: “Hearing loss due to cancer treatment is devastating for children and their families so I’m pleased we are able to recommend this ground-breaking treatment.
“This is the first drug shown to prevent and reduce the impact of hearing loss, and it will have a life changing effect on the lives of children and young people.
“Our recommendation for this innovative treatment demonstrates NICE’s commitment to focussing on what matters most by getting the best care to patients fast and ensuring value for the taxpayer.”
Read the full final draft guidance for anhydrous sodium thiosulfate for preventing ototoxicity caused by cisplatin chemotherapy in people aged one month to 17 years with localised solid tumours.