Results of a prospective study for the treatment of retinoblastoma

Abstract

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.

Strategies to manage retinoblastoma in developing countries

“…Pathologic evaluation of enucleated eyes is seldom done, and relatively intensive chemotherapy is not usually available [19][20][21]. Survival in many middle-income countries (MICs) is over 70%, and some upper MICs that have established multidisciplinary teams and adequate treatment facilities have survival rates near 90% (Table I) [22][23][24][25].…”

“…In contrast, in most MICs, metastatic disease is present at diagnosis in less than 10% of the cases [7,22,23]. Treatment with neoadjuvant chemotherapy, followed by secondary enucleation and adjuvant therapy is the current approach in most centers [13,40,41].…”

“…Encouraging results have been reported for the treatment of retinoblastoma that is disseminated to the orbit or the preauricular lymph nodes has resulted in disease-free survival rates over 70% [41]. Different chemotherapy combinations have been used to treat overt extraocular retinoblastoma; most regimens include a platinum derivative; an alkylating agent, such as cyclophosphamide or ifosfamide, etoposide, and anthracyclines [22,41,42]. A low intensity regimen using these agents led to 56% survival at 30 months in Mali [13].…”

Successful treatment of metastatic retinoblastoma with high-dose chemotherapy and autologous stem cell rescue in South America

“…[3][4][5][6] Results from the most recent Latin-American series reporting patients treated with conventional chemo-and radiotherapy regimens were discouraging with survival figures from 0 to 20%. 3,7,8 Only occasional patients with distant metastatic disease survived and the few survivors reported with conventional chemotherapy included those who had metastasis limited to the lymph nodes, 9 which are now considered as regionally disseminated rather than a distant metastatic site. 10 High-dose chemotherapy followed by ASCR was not available in our setting until recently and, to our knowledge, there are no reported results of this treatment modality for retinoblastoma from less developed countries.…”

Retinoblastoma: Review of Current Management

“…There are reports that claim that choroidal invasion by tumor does not contribute to a poor outcome, while others claim that choroidal invasion can result in mortality in the range of 11%-81% [32,[87][88][89][90][91][92][93][94][95][96][97][98][99][100][101][102]. The combination of choroidal invasion along with any degree of optic nerve invasion has also been recognized as a risk factor with an adverse influence on outcome by some groups [48,103].…”