Intravitreal Melphalan for Persistent or Recurrent Retinoblastoma Vitreous Seeds

Abstract

IMPORTANCE Recurrent or persistent vitreous seeds following treatment of retinoblastoma poses difficult management and often leads to enucleation. OBJECTIVE To describe the technique and evaluate the efficacy and complications of intravitreal melphalan for vitreous seeding from retinoblastoma. DESIGN, SETTING, AND PARTICIPANTS This retrospective noncomparative analysis was conducted at a tertiary referral center. The study included 11 consecutive eyes of 11 patients with viable persistent or recurrent vitreous seeds following treatment of retinoblastoma. INTERVENTIONS All eyes received intravitreal melphalan injection (20-30 μg) by transconjunctival pars plana route with concomitant triple-freeze cryotherapy at the injection site during needle withdrawal for prevention of extraocular seeding. Each patient was offered planned 6 monthly cycles. MAIN OUTCOMES AND MEASURES Vitreous seed control and complications of therapy. RESULTS The mean patient age at vitreous injection was 37 months (median, 27 months; range, 16-82 months). Viable vitreous seeds involved 2 (n = 1), 3 (n = 4), or 4 (n = 6) quadrants. The solid intraretinal retinoblastoma and subretinal seeds showed regression in all eyes following intravenous chemotherapy (n = 6) or intra-arterial chemotherapy (n = 5). There were a total of 55 injections, with a mean number per patient of 5 (median, 6; range, 2-6). Fewer than 6 injections (n = 5) were delivered owing to complete vitreous seed control and parental desire to avoid more injections. By a mean of 9 months' follow-up (median, 9 months; range, 6-16 months), therapeutic success with complete vitreous seed regression was achieved in all 11 cases (100%). Globe salvage was attained in all cases (100%). Further vitreous seed development did not occur in any case. Complications included focal retinal pigment epithelial mottling near the site of chemotherapy injection (2 eyes) and nonaxial posterior lens opacity (2 eyes). There was no case of extraocular tumor extension, hypotony, or phthisis bulbi. CONCLUSIONS AND RELEVANCE These preliminary short-term results suggest that intravitreal melphalan injection for persistent or recurrent vitreous retinoblastoma seeding can provide tumor control with minimal toxicity and complications.

Atypical Retinal Pigment Epithelial Hyperplasia and Glial Proliferation Masquerading as Progressive Recurrent Retinoblastoma: A Case Report Review and Clinicopathologic Correlation

“…Although RPE hyperplasia, hypertrophy, and proliferation following laser therapy and chemotherapy in retinoblastoma patients have been documented, this case report demonstrates that, associated with glial proliferation, RPE hyperplasia can present as a progressive opaque white growth, thus mimicking the clinical presentation of active retinoblastoma, both on clinical exam and OCT imaging [3,4,5,6]. The knowledge of the phenomenon is especially important in patients with increasing retinal whitening after having received multiple rounds of laser consolidation therapy, which is known to induce RPE and glial proliferation.…”

“…It can occur as a result of RPE toxicity from intra-arterial and/or intravitreal chemotherapy, as well as from local laser therapy [3,4,5,6]. Indeed, laser photocoagulation, a common method used for the consolidative treatment of retinoblastoma, has been proven to induce RPE hyperplasia in and around the laser site on the retina using a mouse model [7,8].…”

Retinoblastom Tedavisinde Güncel Yaklaşımlar

“…Bunun önüne geçebilmek amacı ile vitreusa en yaygın olarak 8-50 µg melfalan enjekte edilmektedir. 51 Teknik olarak limbustan 3 mm mesafede pars planadan 31 G iğne ile girilerek ilacın verilmesinin ardından giriş bölgesine kriyoterapi uygulanmaktadır. Retinaya toksik olmayan, hipotoni ve fitizis yaratmayan melfalan doz aralığının ise 20-30 µg/0,1 cc olduğu düşünülmektedir.…”

“…Retinaya toksik olmayan, hipotoni ve fitizis yaratmayan melfalan doz aralığının ise 20-30 µg/0,1 cc olduğu düşünülmektedir. 51,52 Bu yöntem ile %87-%100 gözde vitreus tohumlanması ortadan kaldırılmaktadır. Doz 8 µg/0,1 cc tutulduğu takdirde tümör kontrol oranı %43'e düşmektedir.…”

“…Doz 8 µg/0,1 cc tutulduğu takdirde tümör kontrol oranı %43'e düşmektedir. 51 İntravitreal melfalan enjeksiyonu sonrası komplikasyon ve yan etkiler nadirdir ancak fokal iris atrofileri, koryoretinal atrofi, vitreus hemorajileri ve retina dekolmanı bildirilmiştir. 53 İntravitreal olarak tiotepa, karboplatin ve metotreksat çok daha az sıklıkta kullanılmaktadır.…”