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Tailoring treatment for retinoblastoma

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Primary chemotherapy is more effective in retinoblastoma tumours in the macula and if children are over 2 months old, according to a study by at the Ocular Oncology Service at St Bartholomew's and Moorfields Hospitals, London, UK.

Noting that primary chemotherapy produced varying outcomes in their patients, Gombos et al examined individual tumour features—basal size and location, previously rather overlooked—and patient age at diagnosis to see whether they predicted outcome. Their retrospective review was confined to 36 children receiving chemotherapy alone and followed up for one year minimum (range 12–44 months). Half were boys, 10 had familial retinoblastoma, and eight sporadic retinoblastoma. Within 42 eyes were 78 tumour foci: 31 (40%) in the macula, 38 (50%) in the equatorial region, and eight (10%) in the anterior ora region; 56 (72%) responded to chemotherapy alone; 22 (28%) needed other treatment.

Chemotherapy was most successful for macular tumours, tumours >2 mm across, sporadic tumours, and for children over 2 years at diagnosis, logistic regression showed. This was true for macular tumours when age—a confounder linked with tumour size and inheritance—was accounted for. Further analysis confirmed age >2 months and macular site as independently linked to favourable treatment outcome, but the relation with tumour size was less clear.

These insights allow treatment to be better tailored at diagnosis. Macular foci probably do not need adjuvant thermotherapy; tumours <2 mm across demand careful regular checks. In view of the small numbers, poor outcome in very young children deserves further study.

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