Clinical Results of Chemotherapy based Treatment in Retinoblastoma Patients: A Single Center Experience

Abstract

Purpose: Retinoblastoma is the most common intraocular malignancy in children. Since the 1990s, chemotherapy was indicated for intraocluar disease to reduce the frequency of enucleation and spare the complications associated with external beam radiation. In this study, we analyzed treatment results of retinoblastoma in our institute. Materials and Methods:Datas from children diagnosed with retinoblastoma and treated at Seoul National University Children's Hospital between 1986 and 2008 were analyzed retrospectively. We utilized cyclophosphamide, vincristine, adriamycin, and methotrexate (CVAM) for OPD-based adjuvant chemotherapy. From 1990, primary chemotherapy was administered to patients with intraocular disease for eyeball-saving and patients received a combination of etoposide, vincristine, cisplatin (or ifosfamide) as a moderately intensive regimen, or a combination of cisplatin, doxorubicin, etoposide, and cycophosphamide (CDEC) as a highly intensive regimen.Results: One hundred eighteen children were analyzed. There were 68 unilateral and 50 bilateral diseases. The median age at diagnosis was 1 year and ReeseEllsworth stage V was the most common stage at the time of diagnosis. All patients were treated by chemotherapy-based multimodality methods, and primary chemotherapy was administered to 80 patients. The 10-year overall and event-free survival rate of all patients were 93.9% and 91.6%, respectively. Two patients who died were in the CDEC regimen group, but there was no significant statistical difference in survival rates by chemotherapy regimens. Fifty-six of 114 eyeballs were saved after primary chemotherapy-based treatment, and the eyeball-saving rate was 49.1%. Six patients relapsed after enucleation and 2 patients were treated successfully after autologous PBSCT. Osteosarcoma occurred in 2 patients as a secondary malignancy, and facial asymmetry after radiotherapy was the most common long-term sequelae.Conclusions: In this study, the overall and event-free survival rates of retinoblastoma were satisfactory and eye-saving was possible with primary chemotherapy.

Conservative management of retinoblastoma: Challenging orthodoxy without compromising the state of metastatic grace. “Alive, with good vision and no comorbidity”

“…During the irradiation era, 96% of bilaterally affected children had their worst eye enucleated at diagnosis, with primary enucleation of both eyes necessary in 3% and secondary enucleation of an only remaining eye due to treatment failure leading to total blindness in 25-29% (Abramson et al, 2004;Epstein et al, 2003). Despite the advent of systemic chemotherapy as first line treatment, 64-66% of children with bilateral disease still underwent primary enucleation of the more advanced eye and 2-6% required initial bilateral enucleation (Epstein et al, 2003;Kim et al, 2008), while advanced diseased eyes frequently needed external irradiation and/or secondary enucleation. The recent advances in targeted chemotherapy modalities allowed treatment of advanced disease and salvage of heavily pretreated eyes, resulting in a further decline of the primary enucleation rate to less than 8% and virtually no more bilateral ocular losses (Francis et al, 2018b).…”

High‐dose chemotherapy with autologous stem cell rescue for treatment of retinoblastoma: Report of five cases

“…in our small series of patients with either bilateral or unilateral disseminated/refractory RB. Although the use of ifosfamide for treatment of RB is not standard of care, several reports support its use . The treatment was very well tolerated by all patients with no major side effects or unexpected toxicities; at the last follow‐up evaluation, none of them showed any relevant organ toxicity.…”

“…With these caveats, the expected toxicity of HDCT–AuSCT is definitely inferior to that induced by EBRT in a very young child, and also less mutilating than enucleation. Yet, the role of this treatment modality for intraocular disease must be timely revisited in the light of wider use of intra‐arterial administration of chemotherapy .…”

Superselective ophthalmic artery infusion of melphalan for intraocular retinoblastoma: preliminary results from 140 treatments

“…2009), as well as from systemic multiagent chemotherapy, combined or not with other focal therapies (Gombos et al. 2007; Kim et al. 2008).…”

“…Permanent adverse ocular events occurred in four of the patients reported herein, including ptosis, strabismus ⁄ exotropia and chorioretinal dystrophy with pigment alteration. However, complications may also result from other focal treatments including periocular carboplatin injection (Schmack et al 2006), diode laser hyperthermia (Gombos et al 2006), external beam radiotherapy (Abramson et al 2004;Payne et al 2009), as well as from systemic multiagent chemotherapy, combined or not with other focal therapies (Gombos et al 2007;Kim et al 2008).…”