Update on chemotherapy modalities for retinoblastoma: Progress and challenges.

Retinoblastoma stands as a paradigm of success in treating malignancies among pediatric patients. Over recent decades, the approach to managing retinoblastoma has evolved significantly, transitioning from the preservation of patients' lives to the preservation of eyes and vision while minimizing treatment-related complications. Chemotherapy, administered through diverse routes, has solidified its role as the cornerstone of retinoblastoma treatment.

Ceftriaxone exerts antitumor effects in MYCN-driven retinoblastoma and neuroblastoma by targeting DDX3X for translation repression.

MYCN proto-oncogene, bHLH transcription factor (MYCN) amplification is associated with aggressive retinoblastoma (RB) and neuroblastoma (NB) cancer recurrence that is resistant to chemotherapies. Therefore, there is an urgent need to identify new therapeutic tools. This study aimed to evaluate the potential repurposing of ceftriaxone for the treatment of MYCN-amplified RB and NB, based on the clinical observations that the drug was serendipitously found to decrease the volume of the MYCN-driven RB subtype.

Retinoblastoma in Asia: Clinical Presentation and Treatment Outcomes in 2112 Patients from 33 Countries.

Purpose: To describe the clinical presentation and treatment outcomes of children who received a diagnosis of retinoblastoma in 2017 throughout Asia.

Design: Multinational, prospective study including treatment-naïve patients in Asia who received a diagnosis of retinoblastoma in 2017 and were followed up thereafter.

Participants: A total of 2112 patients (2797 eyes) from 96 retinoblastoma treatment centers in 33 Asian countries.

Sunitinib efficacy with minimal toxicity in patient-derived retinoblastoma organoids.

Background: Recurrence of retinoblastoma (RB) following chemoreduction is common and is often managed with local (intra-arterial/intravitreal) chemotherapy. However, some tumors are resistant to even local administration of maximum feasible drug dosages, or effective tumor control and globe preservation may be achieved at the cost of vision loss due to drug-induced retinal toxicity. The aim of this study was to identify drugs with improved antitumor activity and more favorable retinal toxicity profiles via screening of potentially repurposable FDA-approved drugs in patient-derived tumor organoids.

Germline RB1 Mutation in Retinoblastoma Patients: Detection Methods and Implication in Tumor Focality.

Purpose: The study aimed to generate a stepwise method to reduce the workload of full-scale RB1 sequencing for germline mutation screening in retinoblastoma (RB) patients. The implication of germline mutation in tumor focality was also determined in this study.

Methods: A stepwise method was created on the basis of "hotspot" exons analyzed using data on germline RB1 mutation in the RB1-Leiden Open Variation Database and then tested for mutation screening in the blood DNA of 42 patients with RB.

Endogenous panophthalmitis in a patient with COVID-19 during hospitalization in an intensive care unit: A case report.

Purpose: To report a case of endogenous panophthalmitis in a patient with COVID-19 during treatment in an Intensive Care Unit.

Observation: A 64-year-old woman with COVID-19 and septicemia presented with decreased visual acuity, ocular pain, and proptosis in her right eye after treatment with favipiravir, intravenous dexamethasone, and ceftriaxone. An ocular examination of her right eye revealed periorbital tenderness, exophthalmos, and corneal haze.

Fatal Air Embolism after Choroidal Melanoma Endoresection without Air Infusion: A Case Report.

Background: The aim of this study was to report 2 cases of pulmonary air embolism developing several hours after choroidal melanoma endoresection without the use of air infusion during the surgery, with fatality in 1 patient.

Methods: The method of this study was case report.

Participants: Two patients with large choroidal melanomas who collapsed several hours after endoresection without air infusion as a result of pulmonary air embolism.

Sex, gender, and retinoblastoma: analysis of 4351 patients from 153 countries.

Objective: To investigate in a large global sample of patients with retinoblastoma whether sex predilection exists for this childhood eye cancer.

Methods: A cross-sectional analysis including 4351 treatment-naive retinoblastoma patients from 153 countries who presented to 278 treatment centers across the world in 2017. The sex ratio (male/female) in the sample was compared to the sex ratio at birth by means of a two-sided proportions test at global level, country economic grouping, continent, and for selected countries.

Silencing of the Long Noncoding RNA MYCNOS1 Suppresses Activity of MYCN-Amplified Retinoblastoma Without RB1 Mutation.

Purpose: MYCNOS (MYCN opposite strand) is co-amplified with MYCN in pediatric cancers, including retinoblastoma. MYCNOS encodes several RNA variants whose functions have not been elucidated in retinoblastoma. Thus, we attempted to identify MYCNOS variants in retinoblastoma and aimed to decipher the role of MYCNOS variant 1 (MYCNOS1) on the activity of MYCN-amplified retinoblastoma.

Clinical Presentations and Outcomes of Retinoblastoma Patients in relation to the Advent of New Multimodal Treatments: A 12-Year Report from Single Tertiary Referral Institute in Thailand.

Purpose: To investigate the clinical presentations and outcomes of retinoblastoma in relation to the advent of new multimodal treatments in Thailand. Retrospective case series. We evaluated the clinical presentation, staging, details of treatment, and treatment outcomes of retinoblastoma patients who were treated at Ramathibodi Hospital, Bangkok, Thailand, between January 1, 2007, and December 31, 2018.

Retinoblastoma: Etiology, Modeling, and Treatment.

Retinoblastoma is a retinal cancer that is initiated in response to biallelic loss of in almost all cases, together with other genetic/epigenetic changes culminating in the development of cancer. deficiency makes the retinoblastoma cell-of-origin extremely susceptible to cancerous transformation, and the tumor cell-of-origin appears to depend on the developmental stage and species. These are important to establish reliable preclinical models to study the disease and develop therapies.

Global Retinoblastoma Presentation and Analysis by National Income Level.

Importance: Early diagnosis of retinoblastoma, the most common intraocular cancer, can save both a child's life and vision. However, anecdotal evidence suggests that many children across the world are diagnosed late. To our knowledge, the clinical presentation of retinoblastoma has never been assessed on a global scale.

Intra-Arterial Chemotherapy for Retinoblastoma: 8-Year Experience from a Tertiary Referral Institute in Thailand.

Purpose: To study the safety and efficacy of intra-arterial chemotherapy (IAC) as a treatment for intraocular retinoblastoma in Thailand.

Design: Retrospective, interventional case series.

Methods: In this study, IAC was performed as primary or secondary treatment for patients with intraocular retinoblastoma using melphalan with or without additional topotecan or carboplatin.

Spectrum of germline mutations and clinical manifestations in retinoblastoma patients from Thailand.

Purpose: Retinoblastoma (RB) is a retinal tumor that most commonly occurs in children. Approximately 40% of RB patients carry germline mutations in the gene. RB survivors with germline mutations are at increased risk of passing on the disease to future offspring and of secondary cancer in adulthood.

Retinoblastoma in Asia.

Asia-Pacific region bears a significant global burden of retinoblastoma (RB), therefore understanding RB in Asia-Pacific region is important. Based on the year 2013 population estimates, 43% (3452 of 8099 children) of the global burden of RB lives in 6 countries of Asia-Pacific region: 1486 children in India, 1103 children in China, 277 children in Indonesia, 260 children in Pakistan, 184 children in Bangladesh, 142 children in Philippines. There exists a wide disparity, technological and socio-economical, within countries in this region resulting in a varied pattern of clinical presentation and survival varies.

A three-dimensional organoid model recapitulates tumorigenic aspects and drug responses of advanced human retinoblastoma.

Persistent or recurrent retinoblastoma (RB) is associated with the presence of vitreous or/and subretinal seeds in advanced RB and represents a major cause of therapeutic failure. This necessitates the development of novel therapies and thus requires a model of advanced RB for testing candidate therapeutics. To this aim, we established and characterized a three-dimensional, self-organizing organoid model derived from chemotherapy-naïve tumors.

INTRAVITREAL ANTI-VASCULAR ENDOTHELIAL GROWTH FACTOR INJECTIONS FOR EXUDATIVE RETINAL ARTERIAL MACROANEURYSMS.

Purpose: There is no established therapy for exudative-hemorrhagic complications in primary retinal arteriolar macroaneurysm (RAM).

Methods: Retrospective multicenter interventional study of anti-vascular endothelial growth factor in symptomatic RAMs. Central macular thickness in μm and best-corrected visual acuity in logMar were correlated with the RAM size and distance to the macula.

Idiopathic polypoidal choroidal vasculopathy in Thai patients with clinical and angiographic choroidal neovascularization.

Objective: This study aimed to study the prevalence and characteristics of idiopathic polypoidal choroidal vasculopathy (IPCV) in Thai patients with clinical and angiographic choroidal neovascularization (CNV).

Patients And Methods: A consecutive case study of 140 patients presenting with CNV was conducted in nine large referral eye centers throughout Thailand. The demographic data, fundus photographs, fundus fluorescein angiography and indocyanine green angiography of the patients were analyzed.

Epstein-Barr virus-associated iris smooth muscle tumor with epithelioid morphology in AIDS patients: a case report.

Importance: Report of an acquired immunodeficiency syndrome (AIDS) patient with Epstein-Barr virus (EBV)-associated iris smooth muscle tumor.

Observations: A 14-year-old African American female diagnosed with AIDS developed a painless iris mass in the right eye for 10 months. Iridocyclectomy was performed, and the pathology indicated EBV-associated iris smooth muscle tumor with epithelioid morphology.

Enhanced depth imaging optical coherence tomography of circumscribed choroidal hemangioma in 10 consecutive cases.

Purpose: The purpose was to evaluate the features of circumscribed choroidal hemangioma using spectral-domain enhanced depth imaging optical coherence tomography (EDI-OCT).

Design: Retrospective observational case series.

Participants: Ten patients with newly diagnosed circumscribed choroidal hemangioma.

Monthly intravitreal bevacizumab for macular edema after iodine-125 plaque radiotherapy of uveal melanoma.

Purpose: To examine the efficacy of monthly injections of intravitreal bevacizumab (IVB) for macular edema after iodine-125 plaque radiotherapy of uveal melanoma.

Methods: We studied 36 patients with macular edema after plaque radiotherapy of uveal melanoma in this noncomparative, interventional case series. All eyes were treated with 4 monthly injections of IVB.

Management of advanced retinoblastoma with intravenous chemotherapy then intra-arterial chemotherapy as alternative to enucleation.

Purpose: To determine the efficacy of primary intravenous chemotherapy (IVC) plus secondary intraarterial chemotherapy (IAC) for patients with advanced retinoblastoma.

Methods: Retrospective, nonrandomized interventional case series of 14 patients with retinoblastoma managed with primary systemic IVC (vincristine, etoposide, and carboplatin for 6 cycles) followed by secondary IAC (melphalan for 1-6 cycles).

Results: Fourteen patients with advanced retinoblastoma classified by the International Classification of Retinoblastoma as Group D (n = 6, 43%) or Group E (n = 8, 57%) were treated with IVC as primary treatment and subsequent secondary IAC as rescue or consolidation therapy.

Retinal vasoproliferative tumors: comparative clinical features of primary vs secondary tumors in 334 cases.

Objective: To compare the clinical features of primary vs secondary retinal vasoproliferative tumors (VPTs).

Methods: Retrospective case series of 334 tumors in 295 eyes of 275 patients.

Results: Of 275 patients with VPT, 41% (n = 113) were male and 59% (n = 162) were female, with a mean age of 44 years at presentation.

High-risk retinoblastoma based on international classification of retinoblastoma: analysis of 519 enucleated eyes.

Purpose: To determine the correlation between the International Classification of Retinoblastoma (ICRB) and histopathologic high-risk retinoblastoma.

Design: Retrospective study.

Participants: A total of 519 patients.