Results of a prospective study for the treatment of unilateral retinoblastoma.
BACKGROUND
Few prospective studies about the management of unilateral retinoblastoma with pathology risk factors (PRFs) have been published.


METHODS
Patients (n = 114) were divided into four groups: Group 1 (initial chemoreduction) (n = 17). Groups 2 and 3, included patients initially enucleated with no, or lower risk PRFs: (n = 65) and with higher risk PRFs (n = 30), respectively. The later included postlaminar optic nerve involvement (PLONI) (n = 23), tumor at resection margin of optic nerve (n = 5) or isolated scleral invasion (n = 2). Group 3 received adjuvant chemotherapy including a total eight cycles of carboplatin and etoposide, alternating with cyclophosphamide, idarubicin, and vincristine. Orbital radiotherapy (45 Gy) was given to patients with invasion to the resection margin. Group 4 included patients with metastatic disease (n = 2). They were given neoadjuvant therapy followed by surgery and high-dose chemotherapy and autologous stem cell rescue.


RESULTS
Five-year event-free survival is 0.94 (1 for Group 1, 0.94 for Group 2, 0.96 for Group 3, and 0 for Group 4). Events included. Group 2: Systemic relapse (n = 2) and combined orbital and CNS relapse (n = 1). Relapsing patients had PLONI (n = 2) and isolated focal choroidal invasion (n = 1). Group 3: CNS relapse (n = 1) in a patient with tumor at the resection margin of optic nerve. Group 4: CNS relapse (n = 2). Only one relapsed patient survived. Eight of 17 eyes treated conservatively were preserved.


CONCLUSIONS
The survival of patients with unilateral retinoblastoma was excellent and 60% were spared from adjuvant treatment. Our intensive regimen was likely to be effective for prevention of metastasis in patients with higher risk PRFs.
Chantada
Guillermo L
GL
Hemato-Oncology Department, Hospital JP Garrahan, Buenos Aires, Argentina. gchantada@yahoo.com
Fandiño
Adriana C
AC
Guitter
Myriam R
MR
Raslawski
Elsa C
EC
Dominguez
Julieta L
JL
Manzitti
Julio
J
de Dávila
María T G
MT
Zubizarreta
Pedro
P
Scopinaro
Marcelo
M
D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D009367:Neoplasm Staging; D011446:Prospective Studies; D012008:Recurrence; D015203:Reproducibility of Results; D019572:Retinal Neoplasms / Q000175:diagnosis / Q000556:secondary / Q000628:therapy*; D012175:Retinoblastoma / Q000175:diagnosis / Q000556:secondary / Q000628:therapy*; D012307:Risk Factors; D016019:Survival Analysis; D014182:Transplantation, Autologous; D016896:Treatment Outcome