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A multidisciplinary treatment strategy that includes high-dose chemotherapy for metastatic retinoblastoma without CNS involvement

Bone Marrow Transplantation volume 35, pages 763–766 (2005)Cite this article

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.

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Acknowledgements

This research was supported by a Grant-in-Aid for Scientific Research from the Ministry of Health, Labor and Welfare.

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Authors and Affiliations

  1. Pediatric Oncology Division, National Cancer Center Hospital, Tokyo, Japan

    H Matsubara, A Makimoto, T Higa, H Kawamoto, S Sakiyama, A Hosono, J Takayama & M Ohira

  2. Hematopoietic Stem Cell Transplantation Division, National Cancer Center Hospital, Tokyo, Japan

    Y Takaue

  3. Radiation Oncology Division, National Cancer Center Hospital, Tokyo, Japan

    S Murayama & M Sumi

  4. Ophthalmology Division, National Cancer Center Hospital, Tokyo, Japan

    A Kaneko

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  1. H Matsubara
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Correspondence to H Matsubara.

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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

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