Radically different treatment recommendations for newly diagnosed neuroblastoma: Pitfalls in assessment of risk Journal Article


Authors: Kushner, B. H.; LaQuaglia, M. P.; Kramer, K.; Cheung, N. K. V.
Article Title: Radically different treatment recommendations for newly diagnosed neuroblastoma: Pitfalls in assessment of risk
Abstract: Neuroblastoma risk stratification is based on stage, age, and biology and prescribes surgery for low-risk disease, moderate-dose chemotherapy for intermediate-risk disease, and maximal therapy (including myeloablative treatment with stem cell transplantation) for high-risk disease. Four cases are described that depict pitfalls in risk assessment with potentially far-reaching consequences. This report focuses on a subset of four patients referred for second opinions. Stage was defined by the International Neuroblastoma Staging System. The first recommendations were for maximal therapy, but second opinions were radically different die, surgery alone). Ages at diagnosis were 15 to 25 months. Shimada histopathology was unfavorable in three of the four patients, but chromosomal, serum, and urine prognostic markers were favorable. All four patients did well without cytotoxic therapy (follow-up: 2 years 10 months plus to 4 years 8 months plus). Patient 1 had abdominal and upper thoracic/supraclavicular masses (stage 4); the former was resected and the latter spontaneously regressed. Patient 2 had retroperitoneal disease, without bone marrow involvement, but imaging studies showed lesions in vertebral bodies. Biopsies of the latter showed no neuroblastoma and the primary tumor (with regional lymph nodes) was resected, changing stage from 4 to 2B. Patient 3 had a retroperitoneal mass but no distant disease. Though initially deemed to be unresectable, the abdominal tumor was excised, changing the classification from high risk (stage 3 with unfavorable histopathology) to low risk (stage 1). Patient 4 had a pelvic mass, with unfavorable histopathology, and bilateral inguinal lymph node involvement (stage 3); all soft tissue disease was resected. The absence of cortical bone and extensive bone marrow metastatic involvement in a young neuroblastoma patient should cause a shift in attention to biologic prognostic markers. Some patients classified as having high-risk neuroblastoma might actually do well with no cytotoxic therapy.
Keywords: adolescent; adult; child; human tissue; treatment outcome; child, preschool; major clinical study; cytotoxic agent; cancer risk; cancer staging; follow up; staging; antineoplastic agent; neoplasm staging; technetium 99m; biological markers; bone marrow; practice guideline; stem cell transplantation; tumor biopsy; diagnostic imaging; risk assessment; infant; neuroblastoma; health planning guidelines; lymph node; newborn; prognostic factors; age distribution; (3 iodobenzyl)guanidine i 131; vertebra body; (3 iodobenzyl)guanidine i 125; humans; prognosis; human; female; priority journal; article
Journal Title: Journal of Pediatric Hematology/Oncology
Volume: 26
Issue: 1
ISSN: 1077-4114
Publisher: Lippincott Williams & Wilkins  
Date Published: 2004-01-01
Start Page: 35
End Page: 39
Language: English
DOI: 10.1097/00043426-200401000-00012
PROVIDER: scopus
PUBMED: 14707711
DOI/URL:
Notes: J. Pediatr. Hematol. Oncol. -- Cited By (since 1996):11 -- Export Date: 16 June 2014 -- CODEN: JPHOF -- Source: Scopus
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  1. Brian Kushner
    311 Kushner
  2. Nai-Kong Cheung
    648 Cheung
  3. Kim Kramer
    236 Kramer