Abstract
PURPOSETreatment abandonment because of enucleation refusal is a limitation of improving outcomes for children with retinoblastoma in countries with limited resources. Furthermore, many children present with buphthalmos and a high risk of globe rupture during enucleation. To address these unique circumstances, the AHOPCA II protocol introduced neoadjuvant chemotherapy with delayed enucleation.PATIENTS AND METHODSPatients with advanced unilateral intraocular disease (International Retinoblastoma Staging System [IRSS] stage I) were considered for upfront enucleation. Those with diffuse invasion of the choroid, postlaminar optic nerve, and/or anterior chamber invasion received six cycles of adjuvant chemotherapy (vincristine, carboplatin, and etoposide). Patients with buphthalmos and those with a perceived risk for enucleation refusal and/or abandonment were given two to three cycles of chemotherapy before scheduled enucleation followed by adjuvant chemotherapy to complete six cycles, regardless of pathology.RESULTSA total of 161 patients had unilateral IRSS stage I disease; 102 underwent upfront enucleation, and 59 had delayed enucleation. The estimated 5-year abandonment-sensitive event-free and overall survival rates for the group were 0.81 ± 0.03 and 0.86 ± 0.03, respectively. The 5-year estimated abandonment-sensitive event-free survival rates for patients undergoing upfront and delayed enucleation were 0.89 ± 0.03 and 0.68 ± 0.06, respectively (P = .001). Compared with AHOPCA I, abandonment for patients with IRSS stage I retinoblastoma decreased from 16% to 4%.CONCLUSIONAHOPCA describes the results of advanced intraocular retinoblastoma treated with neoadjuvant chemotherapy. In eyes with buphthalmos and patients with risk of abandonment, neoadjuvant chemotherapy can be effective when followed by enucleation and adjuvant chemotherapy. Our study suggests that this approach can save patients with buphthalmos from ocular rupture and might reduce refusal of enucleation and abandonment.
Highlights
Retinoblastoma is the most common eye tumor of childhood and represents 3% of childhood cancers.[1]
AHOPCA describes the results of advanced intraocular retinoblastoma treated with neoadjuvant chemotherapy
In eyes with buphthalmos and patients with risk of abandonment, neoadjuvant chemotherapy can be effective when followed by enucleation and adjuvant chemotherapy
Summary
Retinoblastoma is the most common eye tumor of childhood and represents 3% of childhood cancers.[1] In high-income countries (HICs), the majority of children are cured by enucleation only or with a combination of chemotherapy and focal treatments for ocular preservation; most present with limited intraocular disease, and orbital and metastatic disease are rare occurrences. In this scenario, children with advanced unilateral intraocular disease can be cured with enucleation followed by pathology risk–adapted adjuvant chemotherapy.[2,3,4,5,6]. Buphthalmos is associated with an increased risk of microscopic tumor in the cut end of the optic nerve (and often in the sclera), and upfront
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