Children's Hospital of Illinois, Peoria, IL
Beth Speckhart , Reuben Antony , Karen S. Fernandez
Background: Neuroblastoma (NBL) is the most common extra-cranial solid organ malignancy in children. Although low and intermediate-risk patients have a survival of close to 90%, the same cannot be said for patients with high risk (HR) disease. In the last decade multi-modality treatment of HR NBL patients has been intensified to include chemotherapy, surgery, radiation, bone marrow transplantation and immunotherapy and has resulted in improved survival (Yu, 2009). Data regarding the medium to long-term side effects of this intensive multi-modality therapy is now being collected as the population of HR neuroblastoma survivors continues to grow. Methods: We retrospectively reviewed the clinical data of survivors of HR NBL treated at the Children’s Hospital of Illinois diagnosed since 2009 and evaluated the long-term side effects of survivors through 2015. Results: We found 14 NBL patients of whom 10 had HR disease. Four patients died of progressive disease. Therapy-related, long-term side effects occurred in 6 patients: hearing loss (n = 6), adrenal insufficiency (n = 2), focal nodular hyperplasia of the liver (n = 2), linear growth retardation (n = 1). No patient in our cohort developed thyroid or cardiac problems and no patient was diagnosed with a second malignancy in the 5 years of observation. Conclusions: While the improved outcomes seen in HR NBL patients is encouraging, we found in our small cohort hat survivors of multimodality therapy for high-risk neuroblastoma experience significant long-term effects of treatment which impact their quality of life as well as growth and development. As the number of NBL survivors increases our understanding of the long-term therapy related side effects will continue to improve. Larger longitudinal studies are needed to monitor for other possible side effects that may manifest overtime such as cardiomyopathy, disturbances in sexual development, fertility, intellectual function, learning problems and therapy related second malignancies later in life.
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