Retinoblastoma management in 13q deletion syndrome patients using super-selective chemotherapies and other cancer-directed interventions Journal Article
| Authors: | Cobbs, L. V.; Francis, J. H.; Dunkel, I. J.; Gobin, Y. P.; Brodie, S. E.; Abramson, D. H. |
| Article Title: | Retinoblastoma management in 13q deletion syndrome patients using super-selective chemotherapies and other cancer-directed interventions |
| Abstract: | Background: This study aimed to identify best practices for treating 13q deletion syndrome (13q−) patients with retinoblastoma in the era of super-selective ophthalmic artery chemosurgery (OAC) and intravitreal injection therapy (IVIT). Methods: Retrospective study of 21 eyes from 14 patients with retinoblastoma and 13q− who were treated at Memorial Sloan Kettering Cancer Center (MSKCC) between May 2006 and May 2020, with a mean follow up of 3.7 years. Ocular survival, patient survival, and treatment toxicities were assessed. Results: Nine of the 12 eyes that underwent OAC/IVIT at MSKCC have been progression free for at least 1 year since their last treatments. Fifteen out of 26 OAC cycles resulted in grade 3-4 hematologic toxicity. There was one death from sepsis in the setting of intravenous chemotherapy (IVC) for metastatic disease that occurred after OAC/IVIT therapy. The 2-year Kaplan-Meier ocular survival estimate for the whole cohort was 75% and for the eyes that received OAC or IVIT at MSKCC 83%. For OAC hematologic toxicities, one platelet transfusion and two filgrastim doses were administered, and one patient was hospitalized for neutropenic fevers. Conclusions: The majority of 13q− eyes treated with OAC/IVIT-based regimens can be cured, and there were no deaths related to complications from OAC or IVIT. 13q− Patients did have increased risk of systemic treatment complications, even from super-selective chemotherapies. Despite these toxicities, only one patient developed febrile neutropenia, one patient required a blood product transfusion, and two patients received filgrastim for both OAC and IVC complications. Précis: Children with 13q deletion syndrome with retinoblastoma managed with intra-arterial and intravitreal chemotherapy have excellent patient and ocular survival with acceptable toxicity. © 2020 Wiley Periodicals LLC |
| Keywords: | cancer chemotherapy; child; clinical article; controlled study; preschool child; school child; overall survival; neutropenia; cancer patient; topotecan; carboplatin; progression free survival; multiple cycle treatment; blood toxicity; leukopenia; thrombocytopenia; melphalan; retinoblastoma; retrospective study; febrile neutropenia; infant; eye enucleation; chemosurgery; cytopenia; data analysis software; eye toxicity; thrombocyte transfusion; cryotherapy; low level laser therapy; filgrastim; chromosome 13q; retina disease; chemotherapy toxicities; clinical genetics; chromosome deletion 13; retina surgery; human; male; female; priority journal; article |
| Journal Title: | Pediatric Blood and Cancer |
| Volume: | 68 |
| Issue: | 5 |
| ISSN: | 1545-5009 |
| Publisher: | Wiley Periodicals, Inc |
| Date Published: | 2021-05-01 |
| Start Page: | e28845 |
| Language: | English |
| DOI: | 10.1002/pbc.28845 |
| PUBMED: | 33355996 |
| PROVIDER: | scopus |
| PMCID: | PMC9316827 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 1 April 2021 -- Source: Scopus |
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