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Open research questions in Ocular Oncology and Treatments

121 gap statements mined from Ocular Oncology and Treatments papers in our 4.5M-paper local library, which holds 725 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

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What the literature leaves open

  • Current treatment options for uveal melanoma hepatic metastases have limited effectiveness. There is a need for alternative treatment options that can improve patient outcomes. Radioembolization is a promising treatment option that has not been fully explored.

    Yttrium-90 Radioembolization as a Safe and Effective Treatment Option for Hepatic Uveal Melanoma Metastases · 2026 · DOI

  • The low response rates to immune checkpoint inhibitor therapies in UM patients, - The lack of effective treatments for uveal melanoma, - The need for patient stratification and personalized therapies to improve treatment outcomes.

    Current Treatment of Uveal Melanoma · 2025 · DOI

  • Nivolumab plus ipilimumab has demonstrated activity after anti-PD-1 failure in advanced melanoma, but its effectiveness in later lines and as rechallenge remains unclear.

    Real‐World Outcomes of Nivolumab and Ipilimumab in Metastatic Melanoma as Third Line and Beyond · 2026 · DOI

  • To further evaluate this therapeutic approach, prospective controlled studies are warranted to assess efficacy and optimize patient safety.

    Tolerability and efficacy of chemosaturation in combination with systemic therapy for metastatic uveal melanoma · 2026 · DOI

  • BAP1 loss and PRAME expression define the dominant prognostic axes in UM, yet how they promote malignant progression remains unclear.

    BAP1 loss and PRAME expression converge to remodel the tumor-immune ecosystem during uveal melanoma progression · 2026 · DOI

  • There is no standard of care treatment for PVRL and survival data is limited.

    Combination of Ocular Radiation With Systemic Chemotherapy for Treatment of Primary Vitreoretinal Lymphoma: A Prospective Single Center Cohort With Long Follow‐Up · 2026 · DOI

  • We discuss the hypothesis that noncanonical Gαi-associated spindle-orientation machinery may represent a future therapeutic vulnerability in uveal melanoma, while emphasizing that this concept remains to be validated in disease-specific models.

    Canonical and noncanonical heterotrimeric G-protein signaling in uveal melanoma: Established mechanisms and emerging spindle-orientation hypotheses · 2026 · DOI

  • Research exploring biomarkers for retinoblastoma (RB) diagnosis exists; however, their specific impact on RB has not been thoroughly investigated through systematic quantitative analysis.

    Global research landscape of retinoblastoma biomarkers: a multidisciplinary bibliometric analysis based on multiple databases (2005–2025) · 2025 · DOI

  • While long-term survival data in cutaneous melanoma (CM) are promising, such data are lacking in UM and long-term survivors are rare.

    Characterization of long‐term survivors with liver metastases from uveal melanoma diagnosed between 2005 and 2021 · 2025 · DOI

  • Peritoneal metastases (PM) occur rarely, and there is limited knowledge about this subgroup´s clinical course and biology.

    Characterizing uveal melanoma patients with peritoneal metastases: A retrospective single-center analysis · 2025 · DOI

  • FISH (fluorescence in situ hybridization) represents a fast and reliable method to measure gene copy numbers in various tumors but has not yet been systematically evaluated in retinoblastoma.

    Definition of parameters and thresholds to detect MYCN amplification in retinoblastomas · 2025 · DOI

  • Genetic and cytogenetic alterations of intraocular leiomyomas, as well as their association with various clinical and histopathologic features, have not been previously studied.

    Genomic Profiling of Intraocular Leiomyomas Reveals Recurrent Copy Number Alterations · 2025 · DOI

  • These findings support pursuing other methods for prediction that should be validated before use in clinical practice.

    Malignant Transformation of Choroidal Indeterminate Melanocytic Tumors · 2025 · DOI

  • However, while RB1 mosaicism has been reported in retinoblastoma, its clinical implications and potential impact on cfDNA testing remain unclear.

    Detection and Characterization of RB1 Mosaicism in Patients With Retinoblastoma Receiving cfDNA Test · 2025 · DOI

  • UM key gene signature and prognosis predictive model might provide insights for further investigation of the pathogenesis and development of UM at the molecular level, and provide theoretical basis for determining new prognostic markers of UM and immunotherapy.

    Immunogenic profiling of metastatic uveal melanoma discerns a potential signature related to prognosis · 2024 · DOI

  • Studies are lacking on long-term effects among retinoblastoma patients in low- and middle-income countries.

    Overall survival and cause‐specific mortality in a hospital‐based cohort of retinoblastoma patients in São Paulo, Brazil · 2024 · DOI

  • However, their mechanisms for preferential inhibition of tumour tissue and constituent malignant cells are not fully elucidated.

    Systematic drug screening and target analysis identify digitoxin as a potential therapy for uveal melanoma · 2024 · DOI

  • Nevertheless, the crucial molecular mechanisms underlying VM in the progression of UM remain unclear.

    UBE2G2 inhibits vasculogenic mimicry and metastasis of uveal melanoma by promoting ubiquitination of LGALS3BP · 2024 · DOI

  • This observation is limited by the retrospective nature of the study and merits further investigation in prospective clinical trials.

    Treatment sequence with tebentafusp and immune checkpoint inhibitors in patients with metastatic uveal melanoma and metastatic GNA11/GNAQ mutant melanocytic tumors · 2024 · DOI

  • Despite advances in molecular profiling and metastatic stratification of uveal melanoma tumors, little is known regarding their underlying biology of metastasis.

    Detection of neoplastic-immune hybrid cells with metastatic properties in uveal melanoma · 2024 · DOI

  • Few additional somatic short nucleotide variants (SNVs) indels, or structural variants with deleterious potential were detected, suggesting that somatic changes are rare but not limited to the VHL gene region.

    Whole-Genome Landscape of Retinal Hemangioblastomas · 2026 · DOI

  • Preoperative aqueous humor cytokine analysis lacked specificity. The study has a small sample size (one patient). The follow-up period may not be sufficient to assess long-term outcomes.

    A rare case of adenoma of the nonpigmented ciliary epithelium managed with a triple procedure: a case report · 2026 · DOI

  • Small sample size of 25 patients, - Retrospective study design, - Limited follow-up…

    Yttrium-90 Radioembolization as a Safe and Effective Treatment Option for Hepatic Uveal Melanoma Metastases · 2026 · DOI

  • The study was conducted retrospectively, - The sample size was limited to 74 patients, - The data were split randomly on the patient level, - The study adheres to the Declaration of Helsinki, - The slides and patient information…

    Direct Prediction of 48 Month Survival Status in Patients with Uveal Melanoma Using Deep Learning and Digital Cytopathology Images · 2025 · DOI

  • Validation of the model prospectively, - Comparison of the model with the current gold standard for UM prognostication, - Exploration of the model's ability to provide personalized predictions, - Investigation of the model's potential to be accessed anywhere in the world with an internet…

    Direct Prediction of 48 Month Survival Status in Patients with Uveal Melanoma Using Deep Learning and Digital Cytopathology Images · 2025 · DOI

Most-cited papers in Ocular Oncology and Treatments

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Open questions raised by papers listed on this page include: (1) There is a need for alternative treatment options that can improve patient outcomes (“Yttrium-90 Radioembolization as a Safe and Effective Treatment Option for…”, 2026); (2) Nivolumab plus ipilimumab has demonstrated activity after anti-PD-1 failure in advanced melanoma, but its effectiveness in later lines and as rechallenge remains unclear (“Real‐World Outcomes of Nivolumab and Ipilimumab in Metastatic Melanoma as Third…”, 2026); (3) To further evaluate this therapeutic approach, prospective controlled studies are warranted to assess efficacy and optimize patient safety (“Tolerability and efficacy of chemosaturation in combination with systemic…”, 2026); (4) BAP1 loss and PRAME expression define the dominant prognostic axes in UM, yet how they promote malignant progression remains unclear (“BAP1 loss and PRAME expression converge to remodel the tumor-immune ecosystem…”, 2026); (5) There is no standard of care treatment for PVRL and survival data is limited (“Combination of Ocular Radiation With Systemic Chemotherapy for Treatment of…”, 2026). 121 gap statements have been mined from Ocular Oncology and Treatments papers in our 4.5M-paper local library, which holds 725 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

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