Evaluating the tradeoffs between patient survival and treatment toxicity has been, and always will be, a major challenge to clinicians. Treatment options that improve the Disease Free Survival (DFS) rate are often accompanied by increased side effects whereas options with lower DFS rates are usually less toxic. The administration of Cisplatin to pediatric germ cell tumor patients entails a tradeoff between mortality and ototoxicity. Increasing the dose of Cisplatin from 100mg to 200mg raises the DFS rate from 82% to 92%, yet also results in an increased incidence of permanent hearing loss, from 20% to 76%. The primary goal of this project is to evaluate whether the increase in cancer survival is worth the increased risk of suffering the functional impacts of Cisplatin ototoxicity. The project will proceed in four sequential aims: (1) To understand the hearing impairments due to alternative Cisplatin dosages. Data available from a recent (unpublished) CCG protocol will be used for finding a probability distribution of hearing deficits as a function of dosage. (2) To study the Quality of Life (QOL) consequences and construct health vignettes for decision analysis. Specific hearing deficits will be mapped onto their corresponding patient functional states. This summarizes the real-life difficulties as a result of hearing loss from Cisplatin treatment. Functional status of the child will be assessed by validated inventories from parental ratings and child self-assessments (N = 60). (3) To elicit parental utilities on hearing vignettes constructed from functional results. Using the Standard Gamble and an analog rating scale, five groups of parents will be asked to assign utilities to various hearing vignettes constructed from aim 2 (N = 20 per group). Group differences will be compared. (4) Finally, to perform decision and sensitivity analyses to determine the normative Cisplatin dosage. Preliminary recommendations will be made involving the use of high dose Cisplatin in pediatric germ cell patients. A Markov model is proposed, which extends the time horizon of the analysis and considers hearing deterioration, cancer relapse, second cancer, pediatric cancer, late effects in other organ systems, and additional functional deficits due to salvage regimes.

National Institute of Health (NIH)
National Institute on Deafness and Other Communication Disorders (NIDCD)
Small Research Grants (R03)
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Special Emphasis Panel (ZDC1-SRB-O (23))
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Freeman, Nancy
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Children's Hospital of Philadelphia
United States
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