Prospective phase II study of children affected by bilateral intraocular retinoblastoma with macular involvement of both eyes or in the only preserved eye. Macular tumor control, eye preservation rate, and visual outcome.


Journal

Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624

Informations de publication

Date de publication:
01 2021
Historique:
received: 03 03 2020
revised: 19 08 2020
accepted: 04 09 2020
pubmed: 25 10 2020
medline: 28 4 2021
entrez: 24 10 2020
Statut: ppublish

Résumé

Conservative treatments of intraocular retinoblastoma often consist of chemotherapy and focal treatments. The protocols vary and currently may combine two or three drugs, with different number of cycles, associated to the ocular treatments. In case of macular/paramacular involvement, tumor location and retinal scars induced by focal treatments often have a major negative impact on final visual outcome. This study aimed to include children affected by bilateral intraocular macular/paramacular retinoblastoma in a prospective phase II study. The protocol consisted of six cycles of a three-drug combination (vincristine, etoposide, carboplatin), and the addition of macula-sparing transpupillary thermotherapy (TTT) to the third cycle. The primary endpoint was the local control rate without external beam radiotherapy (EBR) and/or enucleation. Nineteen patients (26 eyes) were included from July 2004 to November 2009. Thirteen eyes belonged to group V of the Reese-Ellsworth classification and 10 to group D of the International Intraocular Retinoblastoma Classification. Macular/paramacular tumors were treated with chemotherapy alone in nine eyes, and with chemotherapy associated with macula-sparing TTT in 17 eyes. Four eyes experienced macular relapse. At a median follow up of 77 months, 23 eyes (88.5%) were saved without EBR, two were enucleated and one received EBR. The median visual acuity of the 24 saved eyes was 20/50. No severe adverse effect was observed. Six cycles of a three-drug combination associated with macula-sparing TTT achieved good tumor control, improved eye preservation rates without EBR, and decreased macular damage, often providing satisfactory visual results with long-term follow up.

Sections du résumé

BACKGROUND
Conservative treatments of intraocular retinoblastoma often consist of chemotherapy and focal treatments. The protocols vary and currently may combine two or three drugs, with different number of cycles, associated to the ocular treatments. In case of macular/paramacular involvement, tumor location and retinal scars induced by focal treatments often have a major negative impact on final visual outcome.
METHODS
This study aimed to include children affected by bilateral intraocular macular/paramacular retinoblastoma in a prospective phase II study. The protocol consisted of six cycles of a three-drug combination (vincristine, etoposide, carboplatin), and the addition of macula-sparing transpupillary thermotherapy (TTT) to the third cycle. The primary endpoint was the local control rate without external beam radiotherapy (EBR) and/or enucleation.
RESULTS
Nineteen patients (26 eyes) were included from July 2004 to November 2009. Thirteen eyes belonged to group V of the Reese-Ellsworth classification and 10 to group D of the International Intraocular Retinoblastoma Classification. Macular/paramacular tumors were treated with chemotherapy alone in nine eyes, and with chemotherapy associated with macula-sparing TTT in 17 eyes. Four eyes experienced macular relapse. At a median follow up of 77 months, 23 eyes (88.5%) were saved without EBR, two were enucleated and one received EBR. The median visual acuity of the 24 saved eyes was 20/50. No severe adverse effect was observed.
CONCLUSION
Six cycles of a three-drug combination associated with macula-sparing TTT achieved good tumor control, improved eye preservation rates without EBR, and decreased macular damage, often providing satisfactory visual results with long-term follow up.

Identifiants

pubmed: 33098751
doi: 10.1002/pbc.28721
doi:

Substances chimiques

Vincristine 5J49Q6B70F
Etoposide 6PLQ3CP4P3
Carboplatin BG3F62OND5

Types de publication

Clinical Trial, Phase II Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

e28721

Informations de copyright

© 2020 Wiley Periodicals LLC.

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Auteurs

Ida Russo (I)

Oncology Center SIREDO, Institut Curie, PSL Research University, Paris, France.
Department of Onco-Hematology, Bambino Gesù Pediatric Hospital IRCCS, Rome, Italy.

Christine Levy-Gabriel (C)

Department of Ophthalmology, Institut Curie, Paris, France.

Axelle Dupont (A)

Department of Biostatistics, Institut Curie, Paris, France.

Livia Lumbroso-Le Rouic (L)

Department of Ophthalmology, Institut Curie, Paris, France.

Nathalie Cassoux (N)

Department of Ophthalmology, Institut Curie, Paris, France.
University of Paris, Paris, France.

Laurence Desjardins (L)

Department of Ophthalmology, Institut Curie, Paris, France.

Anne-Isabelle Bertozzi (AI)

Department of Pediatric Hematology-Oncology, Hôpital des Enfants, Toulouse, France.

Carole Coze (C)

Department of Pediatric Hematology-Oncology, Université Aix-Marseille/APHM, Hôpital d'Enfants de La Timone, Marseille, France.

François Doz (F)

Oncology Center SIREDO, Institut Curie, PSL Research University, Paris, France.
University of Paris, Paris, France.

Alexia Savignoni (A)

Department of Biostatistics, Institut Curie, Paris, France.

Isabelle Aerts (I)

Oncology Center SIREDO, Institut Curie, PSL Research University, Paris, France.

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