Late Effects in Children Treated with Intensive Multimodal Therapy for High-risk Neuroblastoma: High Incidence of Endocrine and Growth Problems

Overview

Journal Bone Marrow Transplant

Specialty General Surgery

Date 2014 Jan 21

PMID 24442245

Citations 31

Authors

L E Cohen

J H Gordon

E Y Popovsky

S Gunawardene

E Duffey-Lind

L E Lehmann

L R Diller

Affiliations

Soon will be listed here.

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.

Citing Articles

Endocrine Disorders in Childhood Brain Tumour Survivors: A Single-Centre Study.

Ramezan N, Hussain S, Mohamad N, Yaacob N J ASEAN Fed Endocr Soc. 2024; 39(1):12-17.

PMID: 38863912 PMC: 11166355. DOI: 10.15605/jafes.039.01.05.

Generation of Orthotopic and Subcutaneous Patient-Derived Xenograft Models from Diverse Clinical Tissue Samples of Pediatric Extracranial Solid Tumors.

Hanssen K, Fletcher J, Kamili A Methods Mol Biol. 2024; 2806:55-74.

PMID: 38676796 DOI: 10.1007/978-1-0716-3858-3_6.

Impact of risk-based therapy on late morbidity and mortality in neuroblastoma survivors: a report from the Childhood Cancer Survivor Study.

Friedman D, Goodman P, Leisenring W, Diller L, Cohn S, Howell R J Natl Cancer Inst. 2024; 116(6):885-894.

PMID: 38460547 PMC: 11160496. DOI: 10.1093/jnci/djae062.

Subtype-selective prenylated isoflavonoids disrupt regulatory drivers of MYCN-amplified cancers.

Stokes M, Vasciaveo A, Small J, Zask A, Reznik E, Smith N Cell Chem Biol. 2023; 31(4):805-819.e9.

PMID: 38061356 PMC: 11031350. DOI: 10.1016/j.chembiol.2023.11.007.

Survival of Patients With Neuroblastoma After Assignment to Reduced Therapy Because of the 12- to 18-Month Change in Age Cutoff in Children's Oncology Group Risk Stratification.

Bender H, Irwin M, Hogarty M, Castleberry R, Maris J, Kao P J Clin Oncol. 2023; 41(17):3149-3159.

PMID: 37098238 PMC: 10256433. DOI: 10.1200/JCO.22.01946.

References

George R, Li S, Medeiros-Nancarrow C, Neuberg D, Marcus K, Shamberger R . High-risk neuroblastoma treated with tandem autologous peripheral-blood stem cell-supported transplantation: long-term survival update. J Clin Oncol. 2006; 24(18):2891-6. DOI: 10.1200/JCO.2006.05.6986. View

Trahair T, Vowels M, Johnston K, Cohn R, Russell S, Neville K . Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation. Bone Marrow Transplant. 2007; 40(8):741-6. DOI: 10.1038/sj.bmt.1705809. View

Ishiguro H, Yasuda Y, Tomita Y, Shinagawa T, Shimizu T, Morimoto T . Long-term follow-up of thyroid function in patients who received bone marrow transplantation during childhood and adolescence. J Clin Endocrinol Metab. 2004; 89(12):5981-6. DOI: 10.1210/jc.2004-0836. View

Lorini R, Cortona L, Scaramuzza A, De Stefano P, Locatelli F, Bonetti F . Hyperinsulinemia in children and adolescents after bone marrow transplantation. Bone Marrow Transplant. 1995; 15(6):873-7. View

Meacham L, Sklar C, Li S, Liu Q, Gimpel N, Yasui Y . Diabetes mellitus in long-term survivors of childhood cancer. Increased risk associated with radiation therapy: a report for the childhood cancer survivor study. Arch Intern Med. 2009; 169(15):1381-8. PMC: 3529471. DOI: 10.1001/archinternmed.2009.209. View

Hoffmeister P, Storer B, Sanders J . Diabetes mellitus in long-term survivors of pediatric hematopoietic cell transplantation. J Pediatr Hematol Oncol. 2004; 26(2):81-90. DOI: 10.1097/00043426-200402000-00003. View

Laverdiere C, Cheung N, Kushner B, Kramer K, Modak S, LaQuaglia M . Long-term complications in survivors of advanced stage neuroblastoma. Pediatr Blood Cancer. 2005; 45(3):324-32. DOI: 10.1002/pbc.20331. View

Bowen K, Chung D . Recent advances in neuroblastoma. Curr Opin Pediatr. 2009; 21(3):350-6. DOI: 10.1097/MOP.0b013e32832b1240. View

. Diagnosis and classification of diabetes mellitus. Diabetes Care. 2010; 33 Suppl 1:S62-9. PMC: 2797383. DOI: 10.2337/dc10-S062. View

10.

Elmlinger M, Kuhnel W, Weber M, Ranke M . Reference ranges for two automated chemiluminescent assays for serum insulin-like growth factor I (IGF-I) and IGF-binding protein 3 (IGFBP-3). Clin Chem Lab Med. 2004; 42(6):654-64. DOI: 10.1515/CCLM.2004.112. View

11.

Matthay K, Villablanca J, Seeger R, Stram D, Harris R, Ramsay N . Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's Cancer Group. N Engl J Med. 1999; 341(16):1165-73. DOI: 10.1056/NEJM199910143411601. View

12.

Carpentieri S, Diller L . Neuropsychological resiliency after treatment for advanced stage neuroblastoma. Bone Marrow Transplant. 2005; 35(11):1117-22. DOI: 10.1038/sj.bmt.1704947. View

13.

Parsons S, Neault M, Lehmann L, Brennan L, Eickhoff C, Kretschmar C . Severe ototoxicity following carboplatin-containing conditioning regimen for autologous marrow transplantation for neuroblastoma. Bone Marrow Transplant. 1998; 22(7):669-74. DOI: 10.1038/sj.bmt.1701391. View

14.

Perwein T, Lackner H, Sovinz P, Benesch M, Schmidt S, Schwinger W . Survival and late effects in children with stage 4 neuroblastoma. Pediatr Blood Cancer. 2011; 57(4):629-35. DOI: 10.1002/pbc.23036. View

15.

Traggiai C, Stanhope R, Nussey S, Leiper A . Diabetes mellitus after bone marrow transplantation during childhood. Med Pediatr Oncol. 2002; 40(2):128-9. DOI: 10.1002/mpo.10098. View

16.

Hovi L, Saarinen-Pihkala U, Vettenranta K, Lipsanen M, Tapanainen P . Growth in children with poor-risk neuroblastoma after regimens with or without total body irradiation in preparation for autologous bone marrow transplantation. Bone Marrow Transplant. 1999; 24(10):1131-6. DOI: 10.1038/sj.bmt.1702021. View

17.

Kaste S, Goodman P, Leisenring W, Stovall M, Hayashi R, Yeazel M . Impact of radiation and chemotherapy on risk of dental abnormalities: a report from the Childhood Cancer Survivor Study. Cancer. 2009; 115(24):5817-27. PMC: 3754878. DOI: 10.1002/cncr.24670. View

18.

Sarafoglou K, Boulad F, Gillio A, Sklar C . Gonadal function after bone marrow transplantation for acute leukemia during childhood. J Pediatr. 1997; 130(2):210-6. DOI: 10.1016/s0022-3476(97)70345-7. View

19.

Shargorodsky J, Curhan S, Curhan G, Eavey R . Change in prevalence of hearing loss in US adolescents. JAMA. 2010; 304(7):772-8. DOI: 10.1001/jama.2010.1124. View

20.

van Santen H, de Kraker J, van Eck B, de Vijlder J, Vulsma T . High incidence of thyroid dysfunction despite prophylaxis with potassium iodide during (131)I-meta-iodobenzylguanidine treatment in children with neuroblastoma. Cancer. 2002; 94(7):2081-9. DOI: 10.1002/cncr.10447. View