High-dose chemotherapy followed by stem cell transplantation in the management of retinoblastoma: a systematic review

BACKGROUND AND OBJECTIVESIn recent years, there has been an increasing role for stem cell transplantation in the management of retinoblastoma. the aim of this study was to systematically review the role high-dose chemotherapy followed by stem cell transplantation in the treatment of patients with metastatic or relapsed, tri lateral or bilateral advanced retinoblastoma, and in patients with tumor at the surgical margin of the optic nerve and/or extrascleral extension.DESIGNSystematic literature review.METHODSWe performed an extensive Pubmed database search on 25 february 2012 for studies describing the use of high-dose chemotherapy followed by stem cell transplantation in the management of patients with retinoblastoma.RESULTSWe located 15 studies that met the inclusion criteria and that included 101 patients. following treatment for metastatic and relapsed disease, 44 of 77 patients (57.1 %) were alive with no evidence of disease at the time of follow-up. however, a higher rate of local relapse developed in patients with CNs metastases (73.1%), which dropped to 47.1 % in patients who received thiotepa. in patients with trilateral or bilateral advanced retinoblastoma, 5 of 7 (71.4%) with reported outcome data were alive with no evidence of disease at the time of follow-up. in patients with tumor at the surgical margin of the optic nerve and/or extrascleral extension, 6 of 7 patients (85.7%) were alive with no evidence of disease at the time of follow-up.CONCLUSIONSDurable tumor control is possible in patients with non-Cns metastases, trilateral or bilateral advanced retinoblastoma, and in patients with tumor at the surgical margin of the optic nerve and/or extrascleral extension. Patients with CNS metastases require thiotepa to improve tumor control.