Intra‐arterial chemotherapy is more effective than sequential periocular and intravenous chemotherapy as salvage treatment for relapsed retinoblastoma

Schaiquevich, Paula; Ceciliano, Alejandro; Millan, Natalia; Taich, Paula Juliana; Villasante, Francisco; Fandiño, Adriana; Domínguez, Julieta; Chantada, Guillermo

doi:10.1002/pbc.24356

Cited by 51 publications

(42 citation statements)

References 29 publications

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“…Second line treatment options include focal therapies, repeated systemic chemotherapy, 117,118 IAC, 114,119 brachytherapy (internal radiotherapy), 120,121 , EBRT 122–124 and whole-eye 121 or proton beam 125 radiation. Criteria for secondary enucleation are not well defined but are dominated by refractory subretinal and vitreous seeding, vitreous haemorrhage and secondary neovascular glaucoma, 117,126 and socio-economic and psychological fatigue to save an eye with poor vision.…”

Section: Managementmentioning

confidence: 99%

“…133–135 The efficacy of intravitreal and IAC chemotherapy may ultimately eliminate the need for EBRT, even as second line therapy. 136 A combination of repeat IAC and intravitreal chemotherapy may play an important part in saving eyes that have failed IVC, radiation or IAC 119,130,131 However, in general, extensive treatments over a long time to save an eye may increase family stress, decrease family resources, 108 and risk metastases and death. 114,116,129 …”

Section: Managementmentioning

confidence: 99%

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Retinoblastoma

Dimaras

Corson

Cobrinik

et al. 2015

Nat Rev Dis Primers

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464

390

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Retinoblastoma is a rare cancer of the infant retina, which forms when both RB1 alleles mutate in a susceptible retinal cell, likely a cone photoreceptor precursor. Loss of the tumour suppressor functions of the retinoblastoma protein, pRB, leads to uncontrolled cell division and recurrent genomic changes during tumour progression. Although pRB is expressed in virtually all tissues, cone precursors have biochemical and molecular features that may sensitize to RB1 loss to enable tumourigenesis. Retinoblastoma is diagnosed in ~8,000 children each year worldwide. Patient survival is >95% in high-income countries, but <30% globally. However, outcomes are improving through increasing awareness for earlier diagnosis, new guidelines and sharing of expertise. Intra-arterial and intravitreal chemotherapy have emerged as promising methods to salvage eyes. Ongoing international collaborations will replace the multiple different classifications of eye involvement with standardized definitions to consistently assess eligibility, efficacy and safety of treatment options. Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. Defining the molecular consequences of RB1 loss in diverse tissues may open new avenues for treatment and prevention of retinoblastoma as well as second cancers in patients with germline RB1 mutations.

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Section: Managementmentioning

confidence: 99%

Section: Managementmentioning

confidence: 99%

Retinoblastoma

Dimaras

Corson

Cobrinik

et al. 2015

Nat Rev Dis Primers

Self Cite

464

390

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“…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.…”

Section: Discussionmentioning

confidence: 99%

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

Parareda

Català

Carcaboso

et al. 2013

Acta Ophthalmologica

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ABSTRACT.Purpose: To report the efficacy and complications of intra-ophthalmic artery melphalan (IAM) for treatment of patients with advanced intra-ocular retinoblastoma. Methods: Patients with newly diagnosed, unilateral, group D retinoblastoma were included in a phase II protocol. Children with relapsed-refractory disease after systemic chemoreduction were later treated under the same guidelines. Melphalan (3-5 mg/procedure) was injected through a 1.2 F microcatheter placed into the ophthalmic artery every 21 days. Results: Eleven patients (12 eyes, eight as primary treatment) received 33 IAM procedures. The phase II protocol closed prematurely because of low accrual. The IAM technique was overall safe and could be performed successfully in 31 of 33 (94%) attempts. After the second administration of IAM, very good partial response was achieved in all treated eyes. With a median follow-up time of 29.5 months (range 6-57), ocular salvage was achieved in 7 of 12 (58%) eyes. No systemic adverse events were observed. Two patients developed diffuse arteriolar sclerosis, hyperpigmentation of the retinal pigment epithelium and partial retinal atrophy after the second IAM. Both eyes were preserved with no tumour activity, good motility and perception of light, 56 and 30 months after the last IAM treatment. Multinucleated macrophages with intracytoplasmic foreign material were found in the choroid and the retina in 2 of 5 enucleated eyes. Conclusion: Our study reports the activity and reproducibility of IAM in advanced retinoblastoma but also underlines the challenges of performing prospective studies on this treatment modality. Toxicity was limited to only ocular vascular events.

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“…Shields et al [47] [49] showed excellent control of macular retinoblastoma. Thampi et al [50] noted 86% response rate in groups A, B, and C but only 38% response in groups D and E. In a small cohort of 18 eyes, Schaiquevich et al [51] found that IAC was more effective than POC or IVC for treatment of relapsed retinoblastoma. A two-drug IAC regimen was explored by Francis et al [52], and a three-drug regiment by Marr et al [53].…”

Section: When To Use Intra-arterial Chemotherapymentioning

confidence: 97%