Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage

Summary

For selected cases with advanced Retinoblastoma (RTB) intraocular involvement(stage V of the Reese-Ellsworth classification) in which enucleation would usually be the standard therapeutic approach, in this project the investigators propose an alternative conservative treatment using intra-arterial chemotherapy with melphalan, via direct administration by catheterization of the ophthalmic artery.

Timeline

Start

2009-11

Primary completion

2012-12

Completion

2013-05

Outcome

Outcome not reported

Stopped (Enrollment): “Only 5 subjects could be enrolled. Sample of 25 pat. not be achieved (rare disease).”

Publications

• Background Abramson DH, Dunkel IJ, Brodie SE, Kim JW, Gobin YP. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008 Aug;115(8):1398-404, 1404.e1. doi: 10.1016/j.ophtha.2007.12.014. Epub 2008 Mar 14.
• Background Aerts I, Pacquement H, Doz F, Mosseri V, Desjardins L, Sastre X, Michon J, Rodriguez J, Schlienger P, Zucker JM, Quintana E. Outcome of second malignancies after retinoblastoma: a retrospective analysis of 25 patients treated at the Institut Curie. Eur J Cancer. 2004 Jul;40(10):1522-9. doi: 10.1016/j.ejca.2004.03.023.
• Background Apushkin MA, Apushkin MA, Shapiro MJ, Mafee MF. Retinoblastoma and simulating lesions: role of imaging. Neuroimaging Clin N Am. 2005 Feb;15(1):49-67. doi: 10.1016/j.nic.2005.02.003.
• Background Brisse HJ, Lumbroso L, Freneaux PC, Validire P, Doz FP, Quintana EJ, Berges O, Desjardins LC, Neuenschwander SG. Sonographic, CT, and MR imaging findings in diffuse infiltrative retinoblastoma: report of two cases with histologic comparison. AJNR Am J Neuroradiol. 2001 Mar;22(3):499-504.
• Background Chantada G, Doz F, Antoneli CB, Grundy R, Clare Stannard FF, Dunkel IJ, Grabowski E, Leal-Leal C, Rodriguez-Galindo C, Schvartzman E, Popovic MB, Kremens B, Meadows AT, Zucker JM. A proposal for an international retinoblastoma staging system. Pediatr Blood Cancer. 2006 Nov;47(6):801-5. doi: 10.1002/pbc.20606.
• Background Chantada G, Fandino A, Manzitti J, Urrutia L, Schvartzman E. Late diagnosis of retinoblastoma in a developing country. Arch Dis Child. 1999 Feb;80(2):171-4. doi: 10.1136/adc.80.2.171.
• Background Chantada GL, Dunkel IJ, de Davila MT, Abramson DH. Retinoblastoma patients with high risk ocular pathological features: who needs adjuvant therapy? Br J Ophthalmol. 2004 Aug;88(8):1069-73. doi: 10.1136/bjo.2003.037044.
• Background Chantada GL, Fandino A, Mato G, Casak S. Phase II window of idarubicin in children with extraocular retinoblastoma. J Clin Oncol. 1999 Jun;17(6):1847-50. doi: 10.1200/JCO.1999.17.6.1847.
• Background Chantada GL, Rossi J, Casco F, Fandino A, Scopinaro M, de Davila MT, Abramson DH. An aggressive bone marrow evaluation including immunocytology with GD2 for advanced retinoblastoma. J Pediatr Hematol Oncol. 2006 Jun;28(6):369-73. doi: 10.1097/00043426-200606000-00009.
• Background Dunkel IJ, Aledo A, Kernan NA, Kushner B, Bayer L, Gollamudi SV, Finlay JL, Abramson DH. Successful treatment of metastatic retinoblastoma. Cancer. 2000 Nov 15;89(10):2117-21. doi: 10.1002/1097-0142(20001115)89:103.0.co;2-9.
• Background Dunkel IJ, Gerald WL, Rosenfield NS, Strong EW, Abramson DH, Ghavimi F. Outcome of patients with a history of bilateral retinoblastoma treated for a second malignancy: the Memorial Sloan-Kettering experience. Med Pediatr Oncol. 1998 Jan;30(1):59-62. doi: 10.1002/(sici)1096-911x(199801)30:13.0.co;2-3.
• Background Kaneko A, Suzuki S. Eye-preservation treatment of retinoblastoma with vitreous seeding. Jpn J Clin Oncol. 2003 Dec;33(12):601-7.
• Background Kiribuchi M. [Retrograde infusion of anti-cancer drugs to ophthalmic artery for intraocular malignant tumors]. Nippon Ganka Gakkai Zasshi. 1966 Nov;70(11):1829-33. No abstract available. Japanese.
• Background Moll AC, Imhof SM, Bouter LM, Kuik DJ, Den Otter W, Bezemer PD, Koten JW, Tan KE. Second primary tumors in patients with hereditary retinoblastoma: a register-based follow-up study, 1945-1994. Int J Cancer. 1996 Aug 7;67(4):515-9. doi: 10.1002/(SICI)1097-0215(19960807)67:43.0.CO;2-V.
• Background Linn Murphree A. Intraocular retinoblastoma: the case for a new group classification. Ophthalmol Clin North Am. 2005 Mar;18(1):41-53, viii. doi: 10.1016/j.ohc.2004.11.003.
• Background REESE AB, ELLSWORTH RM. The evaluation and current concept of retinoblastoma therapy. Trans Am Acad Ophthalmol Otolaryngol. 1963 Mar-Apr;67:164-72. No abstract available.
• Background Rodriguez-Galindo C, Wilson MW, Haik BG, Merchant TE, Billups CA, Shah N, Cain A, Langston J, Lipson M, Kun LE, Pratt CB. Treatment of intraocular retinoblastoma with vincristine and carboplatin. J Clin Oncol. 2003 May 15;21(10):2019-25. doi: 10.1200/JCO.2003.09.103.
• Background Shields CL, Shields JA, Baez K, Cater JR, De Potter P. Optic nerve invasion of retinoblastoma. Metastatic potential and clinical risk factors. Cancer. 1994 Feb 1;73(3):692-8. doi: 10.1002/1097-0142(19940201)73:33.0.co;2-8.
• Background Shields CL, Shields JA, Minelli S, De Potter P, Hernandez C, Cater J, Brady L. Regression of retinoblastoma after plaque radiotherapy. Am J Ophthalmol. 1993 Feb 15;115(2):181-7. doi: 10.1016/s0002-9394(14)73922-4.
• Background Shields JA, Parsons H, Shields CL, Giblin ME. The role of cryotherapy in the management of retinoblastoma. Am J Ophthalmol. 1989 Sep 15;108(3):260-4. doi: 10.1016/0002-9394(89)90116-5.
• Background Muen WJ, Kingston JE, Robertson F, Brew S, Sagoo MS, Reddy MA. Efficacy and complications of super-selective intra-ophthalmic artery melphalan for the treatment of refractory retinoblastoma. Ophthalmology. 2012 Mar;119(3):611-6. doi: 10.1016/j.ophtha.2011.08.045. Epub 2011 Dec 22.

Drugs

Indications