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Results of a prospective study for the treatment of retinoblastoma

BACKGROUND The objectives of this prospective study were to avoid adjuvant treatment for patients with intraocular disease and patients with postlaminar optic nerve invasion (PL‐ONI) without full choroidal or scleral invasion. Adjuvant chemotherapy (Regimen 1) was given to patients with scleral inva...

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Published in:Cancer 2004-02, Vol.100 (4), p.834-842
Main Authors: Chantada, Guillermo, Fandiño, Adriana, Dávila, María T. G., Manzitti, Julio, Raslawski, Elsa, Casak, Sandra, Schvartzman, Enrique
Format: Article
Language:English
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Summary:BACKGROUND The objectives of this prospective study were to avoid adjuvant treatment for patients with intraocular disease and patients with postlaminar optic nerve invasion (PL‐ONI) without full choroidal or scleral invasion. Adjuvant chemotherapy (Regimen 1) was given to patients with scleral invasion, PL‐ONI without cut section, and full choroidal and/or scleral invasion. A more intensive regimen of higher dose intravenous chemotherapy (Regimen 2) and local radiotherapy was given to patients with PL‐ONI and compromise at the cut end and to patients with overt extraocular disease. METHODS Six‐month intravenous chemotherapy included carboplatin plus etoposide alternating with cyclophosphamide plus vincristine (Regimen 1) and the same drugs at higher dosage plus idarubicin (Regimen 2). Chemoreduction with carboplatin and vincristine with or without etoposide was given to selected patients (n = 39 patients). RESULTS From 1994 to 2001, 169 patients were evaluable at the Hospital Garrahan (Buenos Aires, Argentina). One hundred eighteen patients with intraocular disease had a 5‐year disease free survival (DFS) rate of 0.98, including 54 patients with choroidal invasion. None of 22 patients with isolated PL‐ONI developed recurrent disease, whereas 2 of 8 patients with concomitant risk factors had tumor recurrences and died. Three of 5 patients with scleral invasion survived, and 7 of 10 patients with cut‐end ONI survived. The only patient with metastatic disease that survived (n = 6) had only lymph node invasion. CONCLUSIONS Adjuvant therapy can be avoided in patients with intraocular and isolated PL‐ONI. Patients with PL‐ONI who also had other risk factors required intensive adjuvant therapy, such as patients with cut‐end and overt extraocular disease. Metastatic disease was not found to be curable with this approach. Cancer 2004;100:834–42. © 2003 American Cancer Society. This prospective study of 169 patients with retinoblastoma showed that patients with intraocular disease and patients with isolated, postlaminar, optic nerve disease do not need adjuvant therapy. A new regimen of chemotherapy and radiation therapy for selected patients was effective for patients with regional disease, but patients with metastatic disease fared poorly.
ISSN:0008-543X
1097-0142
DOI:10.1002/cncr.11952