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Pediatric Transplants

Late effects in children treated with intensive multimodal therapy for high-risk neuroblastoma: High incidence of endocrine and growth problems

Abstract

Due to the poor prognosis of high-risk (HR) neuroblastoma (NBL), scant data exist on late effects after treatment. Recently, protocols utilizing intense multimodal treatment have resulted in improved long-term survival. The objective of this study was to determine the prevalence of late effects in survivors of HR NBL. A retrospective review of clinical data for serial patients completing treatment between September 1994 and October 2007 and surviving for at least 1 year was performed. Therapy included aggressive chemotherapy, surgery, radiation and single or tandem SCT. Oncology follow-up was standard; clinical criteria were utilized for referrals to endocrinology and other services. Fifty-one eligible patients were identified. Median follow-up was 6.1 years (range 1.0–15.2). Height was significantly impacted (ΔZ-score −1.91 in those treated with TBI and −0.77 in those without). Pre-diabetes or diabetes, hypothyroidism and ovarian insufficiency were observed in 50, 59 and 75% of at-risk survivors, respectively. Hearing loss and dental issues were common. Nine patients had relapse of NBL; seven died of progressive disease. As there is a high prevalence of late effects in long-term survivors of HR NBL, close monitoring and further studies after treatment are indicated, and in particular after more modern, non-TBI regimens.

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Acknowledgements

We would like to thank Henry Feldman, PhD for his statistical advice; and Terence Law in the Department of Laboratory Medicine, Boston Children’s Hospital for his help with assay methods and correlations. This work was supported in part by the Friends for Life Foundation.

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Correspondence to L E Cohen.

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Cohen, L., Gordon, J., Popovsky, E. et al. Late effects in children treated with intensive multimodal therapy for high-risk neuroblastoma: High incidence of endocrine and growth problems. Bone Marrow Transplant 49, 502–508 (2014). https://doi.org/10.1038/bmt.2013.218

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  • DOI: https://doi.org/10.1038/bmt.2013.218

Keywords

  • neuroblastoma
  • late effects
  • growth hormone
  • insulin resistance
  • hypothyroidism
  • ovary

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