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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