Long‐term medical outcomes in survivors of extra‐ocular retinoblastoma: The Memorial Sloan‐Kettering Cancer Center (MSKCC) experience1. Issue 4 (21st August 2012)

• Record Type: Journal Article
• Authors: Friedman, Danielle Novetsky; Sklar, Charles A.; Oeffinger, Kevin C.; Kernan, Nancy A.; Khakoo, Yasmin; Marr, Brian P.; Wolden, Suzanne L.; Abramson, David H.; Dunkel, Ira J.
• Is Part Of: Pediatric blood & cancer. Volume 60:Issue 4(2013:Apr.)
• Page Start: 694
• Page End: 699
• Publication Date: 2012-08-21
• Subjects: Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.24280
• Languages: English
• ISSNs: 1545-5009

Abstract

<abstract abstract-type="main" xml:lang="en"> <title>Abstract</title> <sec id="abs1-1" sec-type="section"> <title>Background</title> <p>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.</p> </sec> <sec id="abs1-2" sec-type="section"> <title>Procedure</title> <p>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%).</p> </sec> <sec id="abs1-3" sec-type="section"> <title>Results</title> <p>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.</p> </sec> <sec id="abs1-4" sec-type="section"> <title>Conclusion</title> <p>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. Pediatr Blood Cancer 2013; 60: 694–699. © 2012 Wiley Periodicals, Inc.</p> </sec> </abstract>