Late effects in survivors of tandem peripheral blood stem cell transplant for high-risk neuroblastoma.
BACKGROUND
Increasing numbers of children with advanced neuroblastoma are achieving cure. We describe the clinical late effects specific to survivors of stage IV neuroblastoma all similarly treated using tandem autologous peripheral blood stem cell rescue with TBI.


METHODS
The medical records of 35 neuroblastoma patients treated at CHOP between 1997 and 2001 were examined. Eighteen of the 35 patients died of progressive disease, and 4 were lost to follow-up. Thirteen patients continue to follow-up in our Multidisciplinary Cancer Survivorship Clinic where they are evaluated and monitored by a consistent group of subspecialists that evaluate long-term sequelae. Data on treatment exposures including TBI and treatment related sequelae identified by clinician assessment and/or diagnostic testing were collected.


RESULTS
Results indicate late effects were present in all 13 subjects, 12 of whom suffered from multiple negative sequelae, including issues with growth hormone deficiency, dental problems, osteochondromas and hearing deficiencies, among others, most at higher rates than reported previously.


CONCLUSIONS
The findings in this small cohort indicate the need for future prospective studies of this intensive pediatric cancer treatment, and underscore the importance of medical intervention and long-term monitoring of these at-risk subjects to increase overall quality-of-life.
Hobbie
Wendy L
WL
Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Moshang
Thomas
T
Carlson
Claire A
CA
Goldmuntz
Elizabeth
E
Sacks
Nancy
N
Goldfarb
Samuel B
SB
Grupp
Stephan A
SA
Ginsberg
Jill P
JP
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007223:Infant; D008297:Male; D020360:Neoadjuvant Therapy / Q000009:adverse effects; D009447:Neuroblastoma / Q000628:therapy*; D017741:Survivors; D014916:Whole-Body Irradiation / Q000009:adverse effects*