Children's Hospital Los Angeles Leads Development of First Machine Learning Tool to Predict Risk of Cisplatin-Induced Hearing Loss
LOS ANGELES--(BUSINESS WIRE)--An international research team led by Etan Orgel, MD at Children's Hospital Los Angeles (CHLA) has developed a novel machine learning model that can predict an individual child's risk of developing hearing loss from the chemotherapy drug cisplatin. The team, which includes researchers from the Keck School of Medicine of USC and other institutions across the U.S. and Canada, is the first to develop and validate a novel machine learning model for this purpose. The tool, called PedsHEAR, uses routine, readily available information to quickly predict this risk—with 95% confidence. Results were published in the Journal of Clinical Oncology , and the model is now available for public use.
Cisplatin has been used since the late 1970s to treat a variety of cancers and is highly effective against pediatric solid tumors in the brain and spinal cord, as well as neuroblastoma and rhabdomyosarcoma. Yet, cisplatin is well known to cause devastating side effects. In children, the most common side effect following therapy is debilitating, permanent hearing loss.
A decades-long journey to personalize care
The study grew out of two decades of efforts to prevent cisplatin-induced hearing loss in children. Investigators from CHLA led the pivotal phase 3 Children's Oncology Group trial of sodium thiosulfate (STS), and in 2022, the Food and Drug Administration approved STS as the first treatment to reduce the risk of hearing loss in children given cisplatin. But patients' treatment regimens are already highly complex, and some may not need STS to prevent hearing loss. For those who are not eligible for STS, it's critical for clinicians to understand each patient's risk and what options they have to protect that child's hearing.
'We want to give families and providers the tools they need to understand their child's risk and make an informed decision,' explains Dr. Orgel, who directs Quality and Patient Safety at CHLA's Cancer and Blood Disease Institute. 'This is the paradigm shift we're aiming for—speaking in certainties for each child versus speaking in generalities by regimen.'
This new predictive model is informed by a landmark study designed and led by Dr. Orgel in 2021. Researchers analyzed data from more than 1,400 cisplatin-treated patients across the United States and Canada to establish the first benchmarks for the prevalence of cisplatin-induced hearing loss in children and adolescents.
Researchers used the 1,400-person dataset as the foundation for the PedsHEAR model, training it to analyze risk factors and probabilities and accurately predict a child's risk level for hearing loss. The researchers also brought in two new, real-world data sets from the Children's Oncology Group and a children's hospital in Texas to validate the model in other populations. The now publicly available web model provides each patient with a percentage indicating the child's individual probability of hearing loss.
Creating a new treatment planning standard
'My goal is for this to become a routine clinical tool,' says Dr. Orgel. 'What's unique about this model is that it only uses routinely available data, so any doctor can use it from day one of diagnosis to plan treatment.' He adds, 'Ultimately, we aim to expand our approach to understand and predict risk for other common side effects of common chemotherapies.
'We want to equip all patients beginning their cancer journey with knowledge that supports meaningful discussions with their doctors on what to expect during and after treatment.'
The research team's next goal is to expand the model to young adults and adults up to 65 and to integrate genomics to make the model's output even more precise.
About Children's Hospital Los Angeles
Founded in 1901, Children's Hospital Los Angeles is at the forefront of pediatric medicine and is the largest provider of hospital care for children in California. Children's Hospital is home to renowned experts who work together across disciplines to deliver inclusive and compassionate care, and drive advances that set pediatric standards across the nation and around the globe. Children's Hospital Los Angeles delivers a level of care that is among the best in the world for a truly diverse population of children. The hospital is consistently ranked in the top 10 in the nation on U.S. News & World Report's Honor Roll of Best Children's Hospitals. CHLA is the top-ranked children's hospital in California and the Pacific U.S. region for 2024-25. Children's Hospital Los Angeles embraces the hospital's mission to create hope and build healthier futures. Children's Hospital Los Angeles is among the top 10 children's hospitals for National Institutes of Health funding. The Saban Research Institute of Children's Hospital Los Angeles supports the full continuum of research, allowing physicians and scientists to translate discoveries into treatments and bring answers to families faster. The pediatric academic medical center also is home to one of the largest training programs for pediatricians in the United States. And the hospital's commitment to building strong communities is evident in CHLA's efforts to fight food insecurity, enhance health education and literacy, and introduce more people to careers in health care. To learn more, follow CHLA on Facebook , Instagram, LinkedIn, YouTube, and X , formerly known as Twitter, and visit CHLA.org/blog.
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All statements regarding events or results to occur in the future contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the statements in Dr. Choueiri's and Dr. Rosen's quotes and statements regarding: the potency, efficacy or safety of casdatifan, including its potential for a best-in-class profile and potential as a combination therapy; and Arcus's development plans for the casdatifan program, including expected timing and design for new studies and cohorts and plans for generating data to support initiation of future studies. All forward-looking statements involve known and unknown risks and uncertainties and other important factors that may cause Arcus's actual results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to risks associated with: interim data not being replicated in future studies evaluating the same investigational molecules or regimen; the unexpected emergence of adverse events or other undesirable side effects with casdatifan; risks associated with manufacturing or supplying product for such clinical trials; uncertainties in timelines associated with the conduct of clinical studies and with respect to the regulatory application process; difficulties associated with the management of the collaboration activities with our strategic partners or expanded clinical programs; changes in the competitive landscape for Arcus's programs; and the inherent uncertainty associated with pharmaceutical product development and clinical trials. Risks and uncertainties facing Arcus are described more fully in the 'Risk Factors' section of Arcus's most recent periodic report filed with the U.S. Securities and Exchange Commission. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date of this press release. Arcus disclaims any obligation or undertaking to update, supplement or revise any forward-looking statements contained in this press release except to the extent required by law. The Arcus name and logo are trademarks of Arcus Biosciences, Inc. All other trademarks belong to their respective owners.
