Late effects in children treated with intensive multimodal therapy for high-risk neuroblastoma: high incidence of endocrine and growth problems.
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.
1] Endocrinology, Boston Children's Hospital, Boston, MA, USA [2] Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.
L E
LE
Cohen
Endocrinology, Boston Children's Hospital, Boston, MA, USA.
J H
JH
Gordon
Endocrinology, Boston Children's Hospital, Boston, MA, USA.
E Y
EY
Popovsky
Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.
S
S
Gunawardene
Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.
E
E
Duffey-Lind
Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.
L E
LE
Lehmann
Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.
L R
LR
Diller
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003131:Combined Modality Therapy; D004700:Endocrine System Diseases / Q000209:etiology*; D005260:Female; D006130:Growth Disorders / Q000209:etiology*; D013006:Growth Hormone; D006801:Humans; D007037:Hypothyroidism / Q000209:etiology; D015994:Incidence; D007223:Infant; D007231:Infant, Newborn; D007333:Insulin Resistance; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000378:metabolism / Q000628:therapy; D009447:Neuroblastoma / Q000378:metabolism / Q000628:therapy*; D011379:Prognosis; D012189:Retrospective Studies; D017741:Survivors
D013006:Growth Hormone