Selective ophthalmic intra-arterial melphalan therapy for advanced retinoblastoma: Implementation and outcomes of a new chemotherapy protocol

Abstract

Retinoblastoma is a relatively uncommon childhood tumor. If untreated, RB grows to fill the eye and destroys the ocular globe's internal architecture. Metastatic spread usually begins after the first 6 months, and death occurs within a matter of years. When treated, overall survival rounds 97%, the alkylating drug melphalan being the most extensively used chemotherapeutic agent in localized treatment. In our hospital, pediatric oncologists asked the Pharmacy Department for assessment in order to implement a new chemotherapy protocol for the treatment of advanced intraocular elegible retinoblastoma cases using melphalan administered directly through the ophthalmic artery. In this paper, we describe the protocol implementation carried out by our collaborative interdisciplinary team as well as the clinical outcomes of five cases treated with ophthalmic intra-arterial melphalan therapy. Oncology pharmacists can contribute with their knowledge to the implementation process of new collaborative practice protocols recommending doses, predicting possible adverse effects and assessing about drug stability and elaboration, packaging and administration methods.

Intra‐arterial chemotherapy for retinoblastoma. Challenges of a prospective study

“…Even among them, to our knowledge, only two groups outside Japan have reported more than 20 patients (Gobin et al 2011;Venturi et al 2013). Case reports or shorter series, comparable to our experience, were reported by other groups (Vajzovic et al 2010;Shields et al 2011b;Valero Garcia et al 2013;Materin et al 2013;Muen et al 2012;Schaiquevich et al 2013). Because the safety and efficacy of this treatment outside the major referral centres was unknown, we decided to implement a strategy under a formal phase II national-wide study to obtain rigorous data.…”

“…Even though the study was open to participants referred from other institutions from our country, it failed to recruit the target number of patients. Changes in the referral patterns caused a barrier for patients from other regions to be enrolled in this national protocol and other smaller groups launched their own programmes of intra-arterial chemotherapy without a formal study (Valero Garcia et al 2013). Very few prospective studies have been undertaken for IACT in retinoblastoma despite they are perceived as the best way of generating solid evidence (Levin et al 2011).…”

Intravitreal melphalan therapy for vitreous seeds in retinoblastoma: Implementation and outcomes of a new chemotherapy protocol

“…RB can be classified as unilateral if it only affects one eye or as bilateral if it affects both eyes. 2 According to the number of lesions that appear in the retina, RB can be classified as a single focal or multifocal disease. Bilateral RB is always hereditary, unlike unilateral and unifocal diseases, which are estimated to present a hereditary component in only 15% of cases.…”

“…Bilateral RB is always hereditary, unlike unilateral and unifocal diseases, which are estimated to present a hereditary component in only 15% of cases. [2][3][4][5][6] Another classification system of intraocular RB was developed by Reese and Ellsworth. This classification has already demonstrated prognostic value in vision conservation and the ability to control local disease.…”

“…The optimal treatment choice depends on the visual prognosis, the size, the number of tumours, their location, the presence or absence of metastatic disease, the patient age, and whether it has a hereditary origin or not. 2 Historically, RB treatment has been based on the enucleation of the eye involved, and EBRT is performed in locally advanced cases. This treatment has considerable complications, including facial disfigurement and increased risk of secondary tumours.…”