Summary:
The prognosis of patients with metastatic retinoblastoma is poor with conventional chemotherapy and radiation. Since retinoblastoma is highly chemosensitive, dose-escalation of chemotherapeutic agents with stem cell support should be promising. We report our experience with high-dose chemotherapy (HDC) and autologous stem cell transplantation (SCT) in patients with metastatic retinoblastoma. Five patients with metastatic retinoblastoma underwent HDC with autologous SCT following conventional chemotherapy and local radiation therapy. Stem cells (bone marrow in four and peripheral blood stem cells in one) were collected after marrow involvement was cleared. Melphalan was a key drug in all patients, and was administered in combination with other agents such as cisplatin, cyclophosphamide, carboplatin or thiotepa. Three patients are currently alive disease-free at 113, 107 and 38 months, respectively, from the time of SCT. They had no central nervous system (CNS) involvement. The two patients who died of disease had CNS involvement. No long-term sequelae of HDC have been noted. Our treatment strategy using HDC appears to be effective for treating metastatic retinoblastoma without CNS involvement.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Sugano K, Yoshida T, Izumi H et al. Outpatient clinic for genetic counseling and gene testing of retinoblastoma. Int J Clin Oncol 2004; 9: 25–30.
Doz F, Neuenschwander S, Plantaz D et al. Etoposide and carboplatin in extraocular retinoblastoma: a study by the Societe Francaise d'Oncologie Pediatrique. J Clin Oncol 1995; 13: 902–909.
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: 31–34.
Kaneko A . Japanese contributions to ocular oncology. Int J Clin Oncol 1999; 4: 321–326.
Higa T, Makimoto A, Matsubara H et al. Successful preservation of eye ball by application of multidisciplinary local ophthalmic treatments (LOT) for unilateral retinoblastoma. ASCO 2001 (abstract no. 1493).
Makimoto A . Results of treatment of retinoblastoma that has infiltrated the optic nerve, is recurrent, or has metastasized outside the eyeball. Int J Clin Oncol 2004; 9: 7–12.
Grabowski EF, Abramson DH . Intraocular and extraocular retinoblastoma. Hematol Oncol Clin N Am 1987; 1: 721–735.
Donaldson SS, Egbert PR, Newsham I et al. Retinoblastoma. In: Pizzo PA, Poplack DG (eds). Principles and Practice of Pediatric Oncology, 4th edn. Lippincott-Raven: Philadelphia, 2002, pp 825–846.
Honavar SG, Singh AD, Shields CL et al. Postenucleation adjuvant therapy in high-risk retinoblastoma. Arch Ophthalmol 2002; 120: 923–931.
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: 722–732.
Schvartzman E, Chantada G, Fandino A et al. Results of a stage-based protocol for the treatment of retinoblastoma. J Clin Oncol 1996; 14: 1532–1536.
Saleh RA, Gross S, Cassano W, Gee A . Metastatic retinoblastoma successfully treated with immunomagnetic purged autologous bone marrow transplantation. Cancer 1988; 62: 2301–2303.
Saarinen UM, Sariola H, Hovi L . Recurrent disseminated retinoblastoma treated by high-dose chemotherapy, total body irradiation, and autologous bone marrow rescue. Am J Pediatr Hematol Oncol 1991; 13: 315–319.
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: 2368–2375.
Yamane S, Shirai C, Arimoto A et al. Disseminated retinoblastoma successfully treated with myeloablative chemotherapy – implication for molecular detection of minimal residual disease. Bone Marrow Transplant 1999; 23: 971–974.
Dunkel IJ, Aledo A, Kernan NA et al. Successful treatment of metastatic retinoblastoma. Cancer 2000; 89: 2117–2121.
Hertzberg H, Kremens B, Velten I et al. Recurrent disseminated retinoblastoma in a 7-year-old girl treated successfully by high-dose chemotherapy and CD34-selected autologous peripheral blood stem cell transplantation. Bone Marrow Transplant 2001; 27: 653–655.
Yamashita N, Nishiuchi R, Oda M et al. Molecular detection of metastatic retinoblastoma cells by reverse transcription polymerase reaction for interphotoreceptor retinoid-binding protein mRNA. Cancer 2001; 91: 1568–1573.
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: 281–284.
Rodriguez-Galindo C, Wilson MW, Haik BG et al. Treatment of metastatic retinoblastoma. Ophthalmology 2003; 110: 1237–1240.
Rodenhuis S, Bontenbal M, Beex LV et al. High-dose chemotherapy with hematopoietic stem-cell rescue for high-risk breast cancer. N Engl J Med 2003; 349: 7–16.
Cheng T, Forsyth P, Chaudhry A et al. High-dose thiotepa, busulfan, cyclophosphamide and ASCT without whole-brain radiotherapy for poor prognosis primary CNS lymphoma. Bone Marrow Transplant 2003; 31: 679–685.
Matthay KK, Villablanca JG, Seeger RC et al. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's Cancer Group. N Engl J Med 1999; 14: 1165–1173.
Carli M, Colombatti R, Oberlin O et al. High-dose melphalan with autologous stem-cell rescue in metastatic rhabdomyosarcoma. J Clin Oncol 1999; 17: 2796–2803.
Matsubara H, Makimoto A, Higa T et al. Possible benefits of high-dose chemotherapy as intensive consolidation in patients with high-risk rhabdomyosarcoma who achieve complete remission with conventional chemotherapy. Pediatr Hematol Oncol 2003; 20: 201–210.
Kushner BH, Meyers PA . How effective is dose-intensive/myeloablative therapy against Ewing's sarcoma/primitive neuroectodermal tumor metastatic to bone or bone marrow? The Memorial Sloan-Kettering experience and a literature review. J Clin Oncol 2001; 19: 870–880.
Inomata M, Kaneko A . Chemosensitivity profiles of primary and cultured retinoblastoma cells in a human tumor clonogenic assay. Jpn J Cancer Res 1987; 78: 858–868.
Ishii E, Matsuzaki A, Ohnishi Y et al. Successful treatment with ranimustine and carboplatin for recurrent intraocular retinoblastoma with vitreous seeding. Am J Clin Oncol 1996; 19: 562–565.
White L . Chemotherapy for retinoblastoma: where do we go from here? Ophthalmic Paediatr Genet 1991; 12: 115–130.
Acknowledgements
This research was supported by a Grant-in-Aid for Scientific Research from the Ministry of Health, Labor and Welfare.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
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 35, 763–766 (2005). https://doi.org/10.1038/sj.bmt.1704882
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1704882
Keywords
This article is cited by
-
High-dose chemotherapy for high-risk retinoblastoma: clinical course and outcome of 14 cases in the National Cancer Center, Japan
Bone Marrow Transplantation (2015)