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Pediatric Transplants

Tandem high-dose chemotherapy and autologous stem cell rescue in children with bilateral advanced retinoblastoma

Bone Marrow Transplantation volume 42, pages 385–391 (2008)Cite this article

Abstract

Although external-beam radiation therapy (EBRT) has been an effective treatment modality in patients with bilateral advanced retinoblastoma, it significantly increases the risk of second malignancies and facial deformities. This study aimed to evaluate the efficacy of tandem high-dose chemotherapy and autologous stem cell rescue (HDCT/ASCR) for treatment, instead of EBRT, in children with bilateral advanced retinoblastoma. Fourteen patients with bilateral retinoblastoma received chemotherapy, and local therapy was provided whenever possible. When at least one functional eye could not be saved by chemoreduction and local therapy, tandem HDCT/ASCR was provided to avoid EBRT. As a result, nine patients received tandem HDCT/ASCR. The toxicities were tolerable and there was no TRM. All nine patients who received tandem HDCT/ASCR had at least one functional eye without EBRT, and in two patients, both eyes were saved. No second malignancy has developed to date. HDCT/ASCR might be an effective treatment for bilateral advanced retinoblastoma, especially in cases in which at least one functional eye could not be preserved with chemoreduction and local therapy alone, and where EBRT was unavoidable. Long-term follow-up and further studies are needed to evaluate the efficacy and toxicity of HDCT/ASCR as an alternative treatment to EBRT.

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Acknowledgements

We thank the residents and nurses who cared for the patients, and without whom this study would not have been possible.

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Author notes

  1. K W Sung and D-I Ham: These authors contributed equally to this work.

Authors and Affiliations

  1. Pediatric Oncology Clinic, National Cancer Center, Goyang, Korea

    S H Lee

  2. Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea

    K H Yoo, K W Sung, J Y Kim, E J Cho & H H Koo

  3. Department of Ophthalmology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea

    S E Chung, S W Kang, S Y Oh, D-I Ham & Y-D Kim

Authors
  1. S H Lee
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  2. K H Yoo
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  5. E J Cho
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  10. D-I Ham
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Correspondence to K W Sung or D-I Ham.

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Lee, S., Yoo, K., Sung, K. et al. Tandem high-dose chemotherapy and autologous stem cell rescue in children with bilateral advanced retinoblastoma. Bone Marrow Transplant 42, 385–391 (2008). https://doi.org/10.1038/bmt.2008.181

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  • DOI: https://doi.org/10.1038/bmt.2008.181

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