Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma Journal Article


Authors: Dunkel, I. J.; Khakoo, Y.; Kernan, N. A.; Gershon, T.; Gilheeney, S.; Lyden, D. C.; Wolden, S. L.; Orjuela, M.; Gardner, S. L.; Abramson, D. H.
Article Title: Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma
Abstract: Background. We previously reported promising pilot results treating patients with stage 4a metastatic retinoblastoma with combined intensive conventional chemotherapy, high-dose chemotherapy with autologous hematopoietic stem cell rescue, and radiation therapy and now present an expanded and updated series. Procedure. Fifteen patients with bone marrow (n=14), bone (n=10), orbit (n=9), and/or liver (n=4) disease were treated. Induction chemotherapy usually consisted of vincristine, cyclophosphamide, cisplatin, and etoposide. The high-dose chemotherapy regimen included carboplatin and thiotepa alone (n=1) or with etoposide (n=5) or topotecan (n=7). Results. Bone marrowcleared at first post-initiation of chemotherapy examination in all patients and stem cells were harvested after a median of 3.5 cycles of chemotherapy (range 3-6 cycles). Two patients progressed prior to high-dose chemotherapy and died. Thirteen received high-dose chemotherapy at a median of 6 months post-diagnosis of metastases (range 4-8 months). Ten are retinoblastoma-free in first remission at a median follow-up of 103 months (range 34-202 months) while three recurred (two in the CNS, one in the mandible) 14-20months post-diagnosis of metastases. Retinoblastoma-free and event-free survival at 5 years are 67% (95% confidence interval 38-85%) and 59% (95% confidence interval 31-79%). Six of the 10survivors received radiation therapy. Three patients developed secondary osteosarcoma 14, 4, and 9 years after diagnosis of metastatic disease. Conclusions. Intensive multimodality therapy including high-dose chemotherapy with autologous hematopoietic stem cell rescue was curative for the majority of patients with stage 4a metastatic retinoblastoma treated. The contribution of external beam radiation therapy is unclear. © 2010 Wiley-Liss, Inc.
Keywords: osteosarcoma; adolescent; adult; cancer survival; child; clinical article; event free survival; school child; child, preschool; survival analysis; retrospective studies; neutropenia; cancer recurrence; cisplatin; multimodality cancer therapy; combined modality therapy; chemotherapy; topotecan; cancer staging; drug megadose; follow-up studies; neoplasm staging; carboplatin; metastasis; etoposide; mucosa inflammation; antineoplastic combined chemotherapy protocols; radiotherapy; recurrence; cyclophosphamide; vincristine; hematopoietic stem cell transplantation; retinoblastoma; tumor regression; retinal neoplasms; thiotepa; fever; cancer regression; infant; hematopoietic cell; pancytopenia; external beam radiotherapy; transplantation, autologous; autologous hematopoietic stem cell transplantation; second malignancy
Journal Title: Pediatric Blood and Cancer
Volume: 55
Issue: 1
ISSN: 1545-5009
Publisher: Wiley Periodicals, Inc  
Date Published: 2010-07-15
Start Page: 55
End Page: 59
Language: English
DOI: 10.1002/pbc.22504
PUBMED: 20486171
PROVIDER: scopus
DOI/URL:
Notes: --- - "Export Date: 20 April 2011" - "CODEN: PBCEA" - "Source: Scopus"
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MSK Authors
  1. Nancy Kernan
    380 Kernan
  2. Ira J Dunkel
    255 Dunkel
  3. David C Lyden
    84 Lyden
  4. Yasmin Khakoo
    100 Khakoo
  5. Suzanne L Wolden
    424 Wolden
  6. David H Abramson
    254 Abramson
  7. Timothy Robin Gershon
    11 Gershon