Long-term medical outcomes in survivors of extra-ocular retinoblastoma: the Memorial Sloan-Kettering Cancer Center (MSKCC) experience.
BACKGROUND
Data on long-term outcomes of survivors of extra-ocular retinoblastoma are lacking. The authors sought to provide the first report characterizing long-term outcomes among survivors of extra-ocular retinoblastoma.


METHODS
Retrospective analysis of long-term medical outcomes in 19 survivors of extra-ocular retinoblastoma treated between 1992 and 2009. Severity of outcomes was graded using Common Terminology Criteria for Adverse Events. All patients received intensive multimodality therapy for their extra-ocular disease after management of their primary intra-ocular disease, including conventional chemotherapy (n = 19, 100%), radiotherapy (n = 15, 69%), and/or high-dose chemotherapy with autologous stem cell transplant (n = 17, 89%).


RESULTS
The median follow-up was 7.8 years from diagnosis of extra-ocular retinoblastoma (range 2-17.8 years). The most common long-term non-visual outcomes were hearing loss (n = 15, 79%), short stature (n = 7, 37%), and secondary malignancies [SMN] (n = 6, 31%). Sixty-eight percent of survivors exhibited ≥2 non-visual long-term outcomes of any grade. Except short stature, which was not graded for severity, Grade 3-4 outcomes were limited to: ototoxicity (n = 8; n = 4 require hearing aids), SMNs (n = 6), and unequal limb length (n = 1). Five patients who developed SMNs carried a known RB1 mutation. SMNs developed at a median of 11.1 years after initial diagnosis; two patients died of their SMN. Long-term cardiac, pulmonary, hepatobiliary, or renal conditions were not identified in any survivors.


CONCLUSIONS
Long-term outcomes are commonly seen in extra-ocular retinoblastoma survivors but the majority are mild-moderate in their severity. Longer comprehensive follow-up is needed to fully assess treatment-related outcomes but the information collected to date may affect management decisions for children with extra-ocular disease.
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, New York 10065, USA. friedmad@mskcc.org
Danielle Novetsky
DN
Friedman
Charles A
CA
Sklar
Kevin C
KC
Oeffinger
Nancy A
NA
Kernan
Yasmin
Y
Khakoo
Brian P
BP
Marr
Suzanne L
SL
Wolden
David H
DH
Abramson
Ira J
IJ
Dunkel
D000970:Antineoplastic Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003072:Cognition Disorders / Q000453:epidemiology / Q000209:etiology; D004700:Endocrine System Diseases / Q000453:epidemiology / Q000209:etiology; D005260:Female; D034381:Hearing Loss / Q000453:epidemiology / Q000209:etiology; D006801:Humans; D007223:Infant; D008297:Male; D016609:Neoplasms, Second Primary / Q000453:epidemiology / Q000209:etiology; D011878:Radiotherapy; D019572:Retinal Neoplasms / Q000150:complications* / Q000628:therapy; D012175:Retinoblastoma / Q000150:complications* / Q000628:therapy; D012189:Retrospective Studies; D017741:Survivors / Q000706:statistics & numerical data*; D016896:Treatment Outcome; D014786:Vision Disorders / Q000453:epidemiology / Q000209:etiology
D000970:Antineoplastic Agents