Skip to main content
Top

05-09-2014 | Ophthalmologic cancers | Article

21. Children’s Oncology Group (COG) Trials for Retinoblastoma

Authors: Dan S. Gombos, MD, FACS, Anna T. Meadows, MD, Murali Chintagumpala, MD, Ira J. Dunkel, MD, Debra Friedman, MD, Julie Ann Stoner, PhD, Rima Jubran, MD, Judith Grob Villablanca, MD

Publisher: Springer Berlin Heidelberg

Abstract

For the past 30 years, retinoblastoma, a tumor that occurs in only 3 % of children with cancer, has been the subject of extensive molecular biologic research. However, apart from a short period in the 1970s, retinoblastoma has not been studied by any of the pediatric cooperative groups. The past decade has witnessed significant multidisciplinary prospective clinical and biologic studies of this rare pediatric neoplasm. The Children’s Oncology Group (COG) has successfully opened four clinical trials with a fifth recently approved. This chapter will review these ongoing prospective multicenter trials.
Literature
1.
Dyer MA, Bremner R. The search for the retinoblastoma cell of origin. Nat Rev Cancer. 2005;5:91–101.PubMedCrossRef
2.
Friedman DL, Himelstein B, Shields CL, et al. Chemoreduction and local ophthalmic therapy for intraocular retinoblastoma. J Clin Oncol. 2000;18:12–7.PubMed
3.
Murphree AL, Villablanca JG, Deegan 3rd WF, et al. Chemotherapy plus local treatment in the management of intraocular retinoblastoma. Arch Ophthalmol. 1996;114:1348–56.PubMedCrossRef
4.
Shields CL, Honavar SG, Meadows AT, et al. Chemoreduction plus focal therapy for retinoblastoma: factors predictive of need for treatment with external beam radiotherapy or enucleation. Am J Ophthalmol. 2002;133:657–64.PubMedCrossRef
5.
Knudson AG. The role of retinoblastoma in understanding human cancer. In: Singh AD, Damato BE, Pe’er J, Murphree AL, Perry JD, editors. Clinical ophthalmic oncology. Philadelphia: Saunders-Elsevier; 2007, Chapter 65.
6.
Murphree AL. Intraocular retinoblastoma: the case for a new group classification. Ophthalmol Clin N Am. 2005;18:41–53.CrossRef
7.
Schvartzman E, Chantada G, Fandino A, et al. Results of a stage-based protocol for the treatment of retinoblastoma. J Clin Oncol. 1996;14:1532–6.PubMed
8.
Messmer EP, Heinrich T, Hopping W, et al. Risk factors for metastases in patients with retinoblastoma. Ophthalmology. 1991;98:136–41.PubMedCrossRef
9.
Khelfaoui F, Validire P, Auperin A, et al. Histopathologic risk factors in retinoblastoma: a retrospective study of 172 patients treated in a single institution. Cancer. 1996;77:1206–13.PubMedCrossRef
10.
Shields CL, Shields JA, Baez KA, et al. Choroidal invasion of retinoblastoma: metastatic potential and clinical risk factors. Br J Ophthalmol. 1993;77:544–8.PubMedCrossRefPubMedCentral
11.
Uusitalo MS, Van Quill KR, Scott IU, et al. Evaluation of chemoprophylaxis in patients with unilateral retinoblastoma with high-risk features on histopathologic examination. Arch Ophthalmol. 2001;119:41–8.PubMedCrossRef
12.
Honavar SG, Singh AD, Shields CL, et al. Postenucleation adjuvant therapy in high-risk retinoblastoma. Arch Ophthalmol. 2002;120:923–31.PubMedCrossRef
13.
Chantada GL, Dunkel IJ, de Davila MT, et al. Retinoblastoma patients with high risk ocular pathological features: who needs adjuvant therapy? Br J Ophthalmol. 2004;88:1069–73.PubMedCrossRefPubMedCentral
14.
Woolson RF. Rank tests and a one-sample logrank test for comparing observed survival data to a standard population. Biometrics. 1981;37:687–96.CrossRef
15.
Jennison C, Turnbull BW. Group sequential methods with application to clinical trials. Boca Raton: Chapman and Hall/CRC; 2000.
16.
Anteby I, Ramu N, Gradstein L, et al. Ocular and orbital complications following the treatment of retinoblastoma. Eur J Ophthalmol. 1998;8:106–11.PubMed
17.
Kaste SC, Chen G, Fontanesi J, et al. Orbital development in long-term survivors of retinoblastoma. J Clin Oncol. 1997;15:1183–9.PubMed
18.
Kleinerman RA, Tucker MA, Tarone RE, et al. Risk of new cancers after radiotherapy in long-term survivors of retinoblastoma: an extended follow-up. J Clin Oncol. 2005;23:2272–9.PubMedCrossRef
19.
Fletcher O, Easton D, Anderson K, et al. Lifetime risks of common cancers among retinoblastoma survivors. J Natl Cancer Inst. 2004;96:357–63.PubMedCrossRef
20.
Rodriguez-Galindo C, Wilson MW, Haik BG, et al. Treatment of intraocular retinoblastoma with vincristine and carboplatin. J Clin Oncol. 2003;21:2019–25.PubMedCrossRef
21.
Smith MA, Rubinstein L, Anderson JR, et al. Secondary leukemia or myelodysplastic syndrome after treatment with epipodophyllotoxins. J Clin Oncol. 1999;17:569–77.PubMed
22.
Gallie BL, Budning A, DeBoer G, et al. Chemotherapy with focal therapy can cure intraocular retinoblastoma without radiotherapy. Arch Ophthalmol. 1996;114:1321–8.PubMedCrossRef
23.
Chan HSL, Heon E, Budning A, et al. Improvement of the cure rate of intraocular retinoblastoma without significantly increasing toxicity with higher dose carboplatin-teniposide in a cyclosporine multidrug reversal regimen. In: Proceedings 10th International Symposium on Retinoblastoma. 2001.
24.
Villablanca JG, Atchaneeyasakul L, Murphree AL. Clinical outcome of Group V eyes treated with cyclosporin A/carboplatin/etoposide/vincristine. In: Proceedings 10th International Symposium on Retinoblastoma. 2001.
25.
Villablanca JG, Jubran RF, Murphree LA. Phase I study of subtenon carboplatin with systemic high dose carboplatin/etoposide/vincristine for eyes with disseminated intraocular retinoblastoma. In: Proceedings 10th International Symposium on Retinoblastoma. 2001.
26.
Abramson DH, Frank CM, Dunkel IJ. A phase I/II study of subconjunctival carboplatin for intraocular retinoblastoma. Ophthalmology. 1999;106:1947–50.PubMedCrossRef
27.
Chantada G, Fandino A, Casak S, et al. Treatment of overt extraocular retinoblastoma. Med Pediatr Oncol. 2003;40:15–161.CrossRef
28.
Antoneli CB, Steinhorst F, de Cassia Braga Ribeiro K, et al. Extraocular retinoblastoma: a 13-year experience. Cancer. 2003;98:1292–8.PubMedCrossRef
29.
Namouni F, Doz F, Tanguy ML, et al. High-dose chemotherapy with carboplatin, etoposide and cyclophosphamide followed by a haematopoietic stem cell rescue in patients with high-risk retinoblastoma: a SFOP and SFGM study. Eur J Cancer. 1997;33:2368–75.PubMedCrossRef
30.
Dunkel IJ, Aledo A, Kernan NA, et al. Successful treatment of metastatic retinoblastoma. Cancer. 2000;89:2117–21.PubMedCrossRef
31.
Kremens B, Wieland R, Reinhard H, et al. High-dose chemotherapy with autologous stem cell rescue in children with retinoblastoma. Bone Marrow Transplant. 2003;31:281–4.PubMedCrossRef
32.
Rodriguez-Galindo C, Wilson MW, Haik BG, et al. Treatment of metastatic retinoblastoma. Ophthalmology. 2003;110:1237–40.PubMedCrossRef
33.
Jubran RF, Erdreich-Epstein A, Butturini A, et al. Approaches to treatment for extraocular retinoblastoma: Children’s Hospital Los Angeles experience. J Pediatr Hematol Oncol. 2004;26:31–4.PubMedCrossRef
34.
Matsubara H, Makimoto A, Higa T, et al. A multidisciplinary treatment strategy that includes high-dose chemotherapy for metastatic retinoblastoma without CNS involvement. Bone Marrow Transplant. 2005;35:763–6.PubMedCrossRef
35.
Doz F, Khelfaoui F, Mosseri V, et al. The role of chemotherapy in orbital involvement of retinoblastoma: the experience of a single institution with 33 patients. Cancer. 1994;74:722–32.PubMedCrossRef
36.
Kivela T. Trilateral retinoblastoma: a meta-analysis of hereditary retinoblastoma associated with primary ectopic intracranial retinoblastoma. J Clin Oncol. 1999;17:1829–37.PubMed
37.
Yamane T, Kaneko A, Mohri M. The technique of ophthalmic arterial infusion therapy for patients with intraocular retinoblastoma. Int J Clin Oncol. 2004;9:69–73.PubMedCrossRef
38.
Abramson DH, Dunkel IJ, Brodie SE, et al. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008;115:1398–404. 1404.e1.PubMedCrossRef
39.
Gobin YP, Dunkel IJ, Marr BP, et al. Intra-arterial chemotherapy for the management of retinoblastoma: four-year experience. Arch Ophthalmol. 2011;129:732–7.PubMedCrossRef