Materiały źródłowe

• #1 Retinoblastoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/retinoblastoma/hp/retinoblastoma-treatment-pdq

  Retinoblastoma is a pediatric cancer that requires careful integration of multidisciplinary care. Treatment of retinoblastoma aims to save the patient’s life and preserve useful vision. For patients presenting with extraocular retinoblastoma, treatment with systemic chemotherapy and radiation therapy is likely to be curative. […] However, extraorbital disease requires intensive chemotherapy and may include consolidation with high-dose chemotherapy and autologous hematopoietic stem cell rescue with or without radiation therapy. While a large proportion of patients with systemic extracentral nervous system (CNS) metastases can be cured, the prognosis for patients with intracranial disease is dismal. […] Screening children with a positive family history of retinoblastoma can improve their prognosis, in terms of globe sparing and use of less intensive, ocular-salvage treatments.

• #2 Prognosis and survival for retinoblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/retinoblastoma/prognosis-and-survival

  You may have questions about your childs prognosis for retinoblastoma. A prognosis is the doctors best estimate of how cancer will affect someone and how it will respond to treatment. Prognosis and survival depend on many factors. Only a doctor familiar with a childs medical history, the type and stage and other features of the cancer, the treatments chosen and the response to treatment can put all of this information together with survival statistics to arrive at a prognosis. […] The following are prognostic and predictive factors for retinoblastoma. […] Doctors use the stage of retinoblastoma to choose treatment and predict the outlook for a child. There are 3 staging systems that might be used for retinoblastoma. […] The lower the stage of retinoblastoma, the better the expected outcome.

• #3 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Retinoblastoma is a highly curable neoplasm in high-income countries, where patient survival exceeds 99%, making it the most curable of all pediatric cancers. In striking contrast, in many lower-income countries, most patients present with disseminated and metastatic disease, which is almost always fatal. […] Despite the changing paradigm of retinoblastoma treatment from systemic to OAC and IVi chemotherapy, eyes that relapse or that are initially refractory to conventional therapy are still difficult to treat with currently available drug options and most undergo enucleation. Thus, drug discovery in retinoblastoma is of paramount importance. […] The aim in developing OAC was to deliver the drug directly into the artery that irrigates the ocular tissues to increase local bioavailability while minimizing systemic exposure.

• #4 Retinoblastoma: Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/retinoblastoma

  The outlook for retinoblastoma depends strongly on how long it takes to diagnose and treat it, but the odds, in general, are very good. The overall survival rate for pediatric retinoblastoma is 95%. The odds of a good outcome including avoiding loss of vision are best with a diagnosis before age 2. […] People who survive retinoblastoma will need lifelong surveillance for new cancers. This usually involves yearly scans or other tests that can detect new tumors. Your provider can tell you what surveillance measures they recommend for your case.

• #5 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  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. […] Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. […] Poor outcome correlates with late diagnosis, difficulty accessing retinoblastoma-specific health care, and socio-economic issues leading to poor compliance, including family refusal to remove the affected eye (enucleation) and abandonment of therapy. […] Fortunately, with early diagnosis, many eyes can be safely treated and support a lifetime of good vision, pointing to key elements for global focus: awareness, collaboration, and affordable expert care.

• #6 Global, regional, and national burden of retinoblastoma in children aged under 10 years from 1990 to 2021 and projections for future disease burden | Scientific Reports

  https://www.nature.com/articles/s41598-025-91289-1

  Retinoblastoma, the most common intraocular malignancy in children, represents a significant global health challenge, particularly in low- and middle-income countries, where delayed diagnosis and limited access to care persist. […] Despite advances in diagnostic and therapeutic approaches, disparities in disease burden remain pronounced. […] Early diagnosis and timely intervention dramatically improve prognosis, with survival rates exceeding 95% in high-income countries. In contrast, survival rates in low- and middle-income countries remain approximately 50%. […] These disparities exacerbate survival inequalities and impose substantial psychosocial and financial burdens on affected families and healthcare systems in resource-limited settings. […] The findings highlight a dual trajectory: declining mortality and DALYs amid rising incidence and prevalence, signaling improved survival but an increasing burden on healthcare systems.

• #7 Retinoblastoma outcome at a single institution in South Africa

  http://www.scielo.org.za/scielo.php?script=sci_arttext&pid=S0256-95742014001200015

  Retinoblastoma (RB) is the most common eye cancer in children. Early detection is necessary for cure. […] Poor outcome is probably a result of late diagnosis. It is important to implement a strategy that will ensure early diagnosis and optimal management of RB in SA. […] Survival is excellent in developed countries, where the survival rate is 95%, but it is only ~50% in developing countries, mostly because of late diagnosis and extraocular disease at diagnosis. […] Late diagnosis and advanced disease are common in developing countries, however, and associated with an increased risk of extraocular disease and poor outcome. […] The major reason for poor outcome was progression of disease, followed by relapse in 5 patients in each group. […] Advanced disease in this study was associated with an overall survival rate of 70%. A strategy is necessary in SA to ensure that children with RB are diagnosed early and that 90% of them are cured.

• #8 Prognosis and survival for retinoblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/retinoblastoma/prognosis-and-survival

  You may have questions about your childs prognosis for retinoblastoma. A prognosis is the doctors best estimate of how cancer will affect someone and how it will respond to treatment. Prognosis and survival depend on many factors. Only a doctor familiar with a childs medical history, the type and stage and other features of the cancer, the treatments chosen and the response to treatment can put all of this information together with survival statistics to arrive at a prognosis. […] The following are prognostic and predictive factors for retinoblastoma. […] Doctors use the stage of retinoblastoma to choose treatment and predict the outlook for a child. There are 3 staging systems that might be used for retinoblastoma. […] The lower the stage of retinoblastoma, the better the expected outcome.

• #9 Prognosis and survival for retinoblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/retinoblastoma/prognosis-and-survival

  Children with retinoblastoma that has not spread outside the eye (intraocular retinoblastoma) have a better prognosis than children with retinoblastoma that has spread outside the eye (extraocular retinoblastoma) or to other parts of the body (metastatic retinoblastoma). […] Children with trilateral retinoblastoma often have a poorer prognosis. […] Survival statistics for retinoblastoma are very general estimates and must be interpreted very carefully. Because these statistics are based on the experience of groups of children, they cannot be used to predict a particular childs chances of survival.

• #10 Retinoblastoma: Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/retinoblastoma

  The outlook for retinoblastoma depends strongly on how long it takes to diagnose and treat it, but the odds, in general, are very good. The overall survival rate for pediatric retinoblastoma is 95%. The odds of a good outcome including avoiding loss of vision are best with a diagnosis before age 2. […] People who survive retinoblastoma will need lifelong surveillance for new cancers. This usually involves yearly scans or other tests that can detect new tumors. Your provider can tell you what surveillance measures they recommend for your case.

• #11 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Identifying high-risk features associated with tumor progression and metastasis by histopathological analysis of the enucleated eye is critical for selecting appropriate management. […] In summary, retinoblastoma treatment has evolved over the last century, resulting in a striking change in the treatment paradigm of this ocular tumor. Advances in the knowledge of its tumor biology and drug response and the development of new routes of drug delivery promise to lead to additional new, more effective, and less toxic therapies in retinoblastoma.

• #12 Retinoblastoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/retinoblastoma/hp/retinoblastoma-treatment-pdq

  Patients with suspected extraocular extension by imaging or high-risk pathology in the enucleated eye (i.e., massive choroidal invasion or involvement of the sclera or the optic nerve beyond the lamina cribrosa) may need to be evaluated for the presence of metastatic disease. Patients presenting with these pathological features in the enucleated eye are at high risk of developing metastases. […] The prognosis for patients with Group E eyes who are treated with systemic chemotherapy and local control measures is very poor without radiation therapy. […] The use of prolonged systemic chemotherapy for Group E eyes to avoid or delay enucleation has been associated with lower disease-specific survival.

• #13 Prognosis and survival for retinoblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/retinoblastoma/prognosis-and-survival

  Children with retinoblastoma that has not spread outside the eye (intraocular retinoblastoma) have a better prognosis than children with retinoblastoma that has spread outside the eye (extraocular retinoblastoma) or to other parts of the body (metastatic retinoblastoma). […] Children with trilateral retinoblastoma often have a poorer prognosis. […] Survival statistics for retinoblastoma are very general estimates and must be interpreted very carefully. Because these statistics are based on the experience of groups of children, they cannot be used to predict a particular childs chances of survival.

• #14 Retinoblastoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/retinoblastoma/hp/retinoblastoma-treatment-pdq

  Retinoblastoma is a pediatric cancer that requires careful integration of multidisciplinary care. Treatment of retinoblastoma aims to save the patient’s life and preserve useful vision. For patients presenting with extraocular retinoblastoma, treatment with systemic chemotherapy and radiation therapy is likely to be curative. […] However, extraorbital disease requires intensive chemotherapy and may include consolidation with high-dose chemotherapy and autologous hematopoietic stem cell rescue with or without radiation therapy. While a large proportion of patients with systemic extracentral nervous system (CNS) metastases can be cured, the prognosis for patients with intracranial disease is dismal. […] Screening children with a positive family history of retinoblastoma can improve their prognosis, in terms of globe sparing and use of less intensive, ocular-salvage treatments.

• #15 Global, regional, and national burden of retinoblastoma in children aged under 10 years from 1990 to 2021 and projections for future disease burden | Scientific Reports

  https://www.nature.com/articles/s41598-025-91289-1

  Retinoblastoma, the most common intraocular malignancy in children, represents a significant global health challenge, particularly in low- and middle-income countries, where delayed diagnosis and limited access to care persist. […] Despite advances in diagnostic and therapeutic approaches, disparities in disease burden remain pronounced. […] Early diagnosis and timely intervention dramatically improve prognosis, with survival rates exceeding 95% in high-income countries. In contrast, survival rates in low- and middle-income countries remain approximately 50%. […] These disparities exacerbate survival inequalities and impose substantial psychosocial and financial burdens on affected families and healthcare systems in resource-limited settings. […] The findings highlight a dual trajectory: declining mortality and DALYs amid rising incidence and prevalence, signaling improved survival but an increasing burden on healthcare systems.

• #16 Retinoblastoma | Children’s Cancer Web

  http://www.cancerindex.org/ccw/guide2r.htm

  Retinoblastoma is a rare tumour of the eye which develops in the cells of the retina, most patients are under 5 years old. […] The median onset age was 21 months. […] The most common presenting signs were leukocoria (73.6%) and strabismus (20.7%). […] The mean age at diagnosis was 23.5 months, and time to diagnosis was 5.4 months. […] In patients with clinical features of hereditary Rb, both onset of the first sign/symptom and diagnosis were at an earlier age than in patients without these features (12.3 vs 21.6 months [P=0.001] and 15.9 vs 28.0 months [P=0.001]). […] The current era of retinoblastoma management using intra-arterial chemotherapy plus additional intravitreal chemotherapy (as needed for vitreous seeding) has improved globe salvage in eyes with advanced retinoblastoma. […] Overall survival, eye salvage, and preservation of vision are largely dependent on the stage of disease at presentation. […] Early diagnosis, multidisciplinary treatment, and genetic counseling are all priorities in the management of this tumor.

• #17 Retinoblastoma | Children’s Cancer Web

  http://www.cancerindex.org/ccw/guide2r.htm

  Retinoblastoma is a rare tumour of the eye which develops in the cells of the retina, most patients are under 5 years old. […] The median onset age was 21 months. […] The most common presenting signs were leukocoria (73.6%) and strabismus (20.7%). […] The mean age at diagnosis was 23.5 months, and time to diagnosis was 5.4 months. […] In patients with clinical features of hereditary Rb, both onset of the first sign/symptom and diagnosis were at an earlier age than in patients without these features (12.3 vs 21.6 months [P=0.001] and 15.9 vs 28.0 months [P=0.001]). […] The current era of retinoblastoma management using intra-arterial chemotherapy plus additional intravitreal chemotherapy (as needed for vitreous seeding) has improved globe salvage in eyes with advanced retinoblastoma. […] Overall survival, eye salvage, and preservation of vision are largely dependent on the stage of disease at presentation. […] Early diagnosis, multidisciplinary treatment, and genetic counseling are all priorities in the management of this tumor.

• #18 Retinoblastoma outcome at a single institution in South Africa

  http://www.scielo.org.za/scielo.php?script=sci_arttext&pid=S0256-95742014001200015

  Retinoblastoma (RB) is the most common eye cancer in children. Early detection is necessary for cure. […] Poor outcome is probably a result of late diagnosis. It is important to implement a strategy that will ensure early diagnosis and optimal management of RB in SA. […] Survival is excellent in developed countries, where the survival rate is 95%, but it is only ~50% in developing countries, mostly because of late diagnosis and extraocular disease at diagnosis. […] Late diagnosis and advanced disease are common in developing countries, however, and associated with an increased risk of extraocular disease and poor outcome. […] The major reason for poor outcome was progression of disease, followed by relapse in 5 patients in each group. […] Advanced disease in this study was associated with an overall survival rate of 70%. A strategy is necessary in SA to ensure that children with RB are diagnosed early and that 90% of them are cured.

• #19 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Key outcomes of the widespread adoption of OAC include: Eliminating the use of EBRT, which has decreased the incidence of second, non-ocular tumors and thereby improved long-term survival. […] The introduction of ophthalmic artery chemosurgery (OAC) led to remarkable success in treating eyes with more advanced disease in which systemic chemotherapy had poorer results. […] Recent studies have used innovative multi-omics technology to identify deregulated pathways that could be targeted via novel treatment strategies in retinoblastoma. […] New drugs effective for both intraocular and disseminated retinoblastoma are urgently needed; these may include novel drugs and repurposed agents, i.e., those already used in the clinic for other cancers or diseases but still without an indication for retinoblastoma.

• #20 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Retinoblastoma is a highly curable neoplasm in high-income countries, where patient survival exceeds 99%, making it the most curable of all pediatric cancers. In striking contrast, in many lower-income countries, most patients present with disseminated and metastatic disease, which is almost always fatal. […] Despite the changing paradigm of retinoblastoma treatment from systemic to OAC and IVi chemotherapy, eyes that relapse or that are initially refractory to conventional therapy are still difficult to treat with currently available drug options and most undergo enucleation. Thus, drug discovery in retinoblastoma is of paramount importance. […] The aim in developing OAC was to deliver the drug directly into the artery that irrigates the ocular tissues to increase local bioavailability while minimizing systemic exposure.

• #21 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Key outcomes of the widespread adoption of OAC include: Eliminating the use of EBRT, which has decreased the incidence of second, non-ocular tumors and thereby improved long-term survival. […] The introduction of ophthalmic artery chemosurgery (OAC) led to remarkable success in treating eyes with more advanced disease in which systemic chemotherapy had poorer results. […] Recent studies have used innovative multi-omics technology to identify deregulated pathways that could be targeted via novel treatment strategies in retinoblastoma. […] New drugs effective for both intraocular and disseminated retinoblastoma are urgently needed; these may include novel drugs and repurposed agents, i.e., those already used in the clinic for other cancers or diseases but still without an indication for retinoblastoma.

• #22 Characterizing DNA methylation signatures of retinoblastoma using aqueous humor liquid biopsy | Nature Communications

  https://www.nature.com/articles/s41467-022-33248-2

  Retinoblastoma (RB) is a cancer that forms in the developing retina of babies and toddlers. The goal of therapy is to cure the tumor, save the eye and maximize vision. However, it is difficult to predict which eyes are likely to respond to therapy. Predictive molecular biomarkers are needed to guide prognosis and optimize treatment decisions. […] Finally, we use AH to characterize molecular subtypes that can potentially be used to predict the likelihood of treatment success for retinoblastoma patients. […] Our findings support accessible approaches of molecular-based RB diagnosis, and potential future clinical implications of epigenetic dysregulation in RB using this liquid biopsy. […] The distinguishable pattern between salvaged samples and enucleation samples in DNA from primary tumors or AH liquid biopsy suggests that DNA methylation analyses of AH cfDNA samples from RB eyes may be used to predict eye salvage. […] Taken together, these data suggested that a more aggressive RB tumor subtype could be predicted by cfDNA methylation profiles of AH liquid biopsies.

• #23 Characterizing DNA methylation signatures of retinoblastoma using aqueous humor liquid biopsy | Nature Communications

  https://www.nature.com/articles/s41467-022-33248-2

  Retinoblastoma (RB) is a cancer that forms in the developing retina of babies and toddlers. The goal of therapy is to cure the tumor, save the eye and maximize vision. However, it is difficult to predict which eyes are likely to respond to therapy. Predictive molecular biomarkers are needed to guide prognosis and optimize treatment decisions. […] Finally, we use AH to characterize molecular subtypes that can potentially be used to predict the likelihood of treatment success for retinoblastoma patients. […] Our findings support accessible approaches of molecular-based RB diagnosis, and potential future clinical implications of epigenetic dysregulation in RB using this liquid biopsy. […] The distinguishable pattern between salvaged samples and enucleation samples in DNA from primary tumors or AH liquid biopsy suggests that DNA methylation analyses of AH cfDNA samples from RB eyes may be used to predict eye salvage. […] Taken together, these data suggested that a more aggressive RB tumor subtype could be predicted by cfDNA methylation profiles of AH liquid biopsies.

• #24 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Key outcomes of the widespread adoption of OAC include: Eliminating the use of EBRT, which has decreased the incidence of second, non-ocular tumors and thereby improved long-term survival. […] The introduction of ophthalmic artery chemosurgery (OAC) led to remarkable success in treating eyes with more advanced disease in which systemic chemotherapy had poorer results. […] Recent studies have used innovative multi-omics technology to identify deregulated pathways that could be targeted via novel treatment strategies in retinoblastoma. […] New drugs effective for both intraocular and disseminated retinoblastoma are urgently needed; these may include novel drugs and repurposed agents, i.e., those already used in the clinic for other cancers or diseases but still without an indication for retinoblastoma.

• #25 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  Comprehensive analysis of the published literature on eye salvage by any strategy is confounded by the use of five different classification systems in different studies, preventing a clear-cut comparison of results between centres, especially for Group D and E eyes. […] The use of multiple classification schemes seeds confusion and undermines research in retinoblastoma. […] A priority to advance management strategies in retinoblastoma is to collect outcome evidence to develop one universally accepted classification based on evidence of the most important features at presentation that predict success for life and eye salvage, that will allow comparison between studies. […] Retinoblastoma is curable if diagnosed early. We propose that within 10 years retinoblastoma can be a ZERO-death cancer.

• #26 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  Comprehensive analysis of the published literature on eye salvage by any strategy is confounded by the use of five different classification systems in different studies, preventing a clear-cut comparison of results between centres, especially for Group D and E eyes. […] The use of multiple classification schemes seeds confusion and undermines research in retinoblastoma. […] A priority to advance management strategies in retinoblastoma is to collect outcome evidence to develop one universally accepted classification based on evidence of the most important features at presentation that predict success for life and eye salvage, that will allow comparison between studies. […] Retinoblastoma is curable if diagnosed early. We propose that within 10 years retinoblastoma can be a ZERO-death cancer.

• #27 Global, regional, and national burden of retinoblastoma in children aged under 10 years from 1990 to 2021 and projections for future disease burden | Scientific Reports

  https://www.nature.com/articles/s41598-025-91289-1

  These results underscore the urgency for equitable public health interventions to enhance early detection, treatment accessibility, and long-term care, particularly in high-burden regions. […] The global Bayesian age-period-cohort (BAPC) model forecasts a modest increase in ASIR, ASPR, and the age-standardized DALYs rate attributable to retinoblastoma over the next 14 years, while ASDR is projected to decline. […] The projected increase in the global burden of retinoblastoma by 2035, as indicated by rising ASIR, ASPR, and age-standardized DALYs rate, alongside a modest decline in ASDR, reflects both advancements in healthcare and ongoing challenges. […] The anticipated rise in incidence and prevalence is likely driven by global population growth and demographic shifts, particularly in regions with high birth rates. […] The modest decline in ASDR, despite rising incidence and prevalence, indicates that healthcare systems are improving in managing retinoblastoma, particularly in reducing mortality.

• #28 Global, regional, and national burden of retinoblastoma in children aged under 10 years from 1990 to 2021 and projections for future disease burden | Scientific Reports

  https://www.nature.com/articles/s41598-025-91289-1

  These results underscore the urgency for equitable public health interventions to enhance early detection, treatment accessibility, and long-term care, particularly in high-burden regions. […] The global Bayesian age-period-cohort (BAPC) model forecasts a modest increase in ASIR, ASPR, and the age-standardized DALYs rate attributable to retinoblastoma over the next 14 years, while ASDR is projected to decline. […] The projected increase in the global burden of retinoblastoma by 2035, as indicated by rising ASIR, ASPR, and age-standardized DALYs rate, alongside a modest decline in ASDR, reflects both advancements in healthcare and ongoing challenges. […] The anticipated rise in incidence and prevalence is likely driven by global population growth and demographic shifts, particularly in regions with high birth rates. […] The modest decline in ASDR, despite rising incidence and prevalence, indicates that healthcare systems are improving in managing retinoblastoma, particularly in reducing mortality.

• #29 Global, regional, and national burden of retinoblastoma in children aged under 10 years from 1990 to 2021 and projections for future disease burden | Scientific Reports

  https://www.nature.com/articles/s41598-025-91289-1

  These results underscore the urgency for equitable public health interventions to enhance early detection, treatment accessibility, and long-term care, particularly in high-burden regions. […] The global Bayesian age-period-cohort (BAPC) model forecasts a modest increase in ASIR, ASPR, and the age-standardized DALYs rate attributable to retinoblastoma over the next 14 years, while ASDR is projected to decline. […] The projected increase in the global burden of retinoblastoma by 2035, as indicated by rising ASIR, ASPR, and age-standardized DALYs rate, alongside a modest decline in ASDR, reflects both advancements in healthcare and ongoing challenges. […] The anticipated rise in incidence and prevalence is likely driven by global population growth and demographic shifts, particularly in regions with high birth rates. […] The modest decline in ASDR, despite rising incidence and prevalence, indicates that healthcare systems are improving in managing retinoblastoma, particularly in reducing mortality.

• #30 Retinoblastoma: Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/retinoblastoma

  The outlook for retinoblastoma depends strongly on how long it takes to diagnose and treat it, but the odds, in general, are very good. The overall survival rate for pediatric retinoblastoma is 95%. The odds of a good outcome including avoiding loss of vision are best with a diagnosis before age 2. […] People who survive retinoblastoma will need lifelong surveillance for new cancers. This usually involves yearly scans or other tests that can detect new tumors. Your provider can tell you what surveillance measures they recommend for your case.

• #31 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  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. […] Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. […] Poor outcome correlates with late diagnosis, difficulty accessing retinoblastoma-specific health care, and socio-economic issues leading to poor compliance, including family refusal to remove the affected eye (enucleation) and abandonment of therapy. […] Fortunately, with early diagnosis, many eyes can be safely treated and support a lifetime of good vision, pointing to key elements for global focus: awareness, collaboration, and affordable expert care.

• #32 Retinoblastoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/retinoblastoma/hp/retinoblastoma-treatment-pdq

  Retinoblastoma is a pediatric cancer that requires careful integration of multidisciplinary care. Treatment of retinoblastoma aims to save the patient’s life and preserve useful vision. For patients presenting with extraocular retinoblastoma, treatment with systemic chemotherapy and radiation therapy is likely to be curative. […] However, extraorbital disease requires intensive chemotherapy and may include consolidation with high-dose chemotherapy and autologous hematopoietic stem cell rescue with or without radiation therapy. While a large proportion of patients with systemic extracentral nervous system (CNS) metastases can be cured, the prognosis for patients with intracranial disease is dismal. […] Screening children with a positive family history of retinoblastoma can improve their prognosis, in terms of globe sparing and use of less intensive, ocular-salvage treatments.

• #33 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  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. […] Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. […] Poor outcome correlates with late diagnosis, difficulty accessing retinoblastoma-specific health care, and socio-economic issues leading to poor compliance, including family refusal to remove the affected eye (enucleation) and abandonment of therapy. […] Fortunately, with early diagnosis, many eyes can be safely treated and support a lifetime of good vision, pointing to key elements for global focus: awareness, collaboration, and affordable expert care.

• #34 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  Comprehensive analysis of the published literature on eye salvage by any strategy is confounded by the use of five different classification systems in different studies, preventing a clear-cut comparison of results between centres, especially for Group D and E eyes. […] The use of multiple classification schemes seeds confusion and undermines research in retinoblastoma. […] A priority to advance management strategies in retinoblastoma is to collect outcome evidence to develop one universally accepted classification based on evidence of the most important features at presentation that predict success for life and eye salvage, that will allow comparison between studies. […] Retinoblastoma is curable if diagnosed early. We propose that within 10 years retinoblastoma can be a ZERO-death cancer.

• #35 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  A number of initiatives address the inequality in retinoblastoma treatment between developing and developed countries. […] The KNRbS has supported the standardization of histopathologic examination of the enucleated eye to inform treatment decisions and discussion of prognosis with families. […] Adjuvant systemic chemotherapy after high-risk features are observed in an enucleated eye may reduce risk of metastatic relapse. […] The expected number of patients with retinoblastoma annually per country is most accurately calculated by multiplying the global retinoblastoma incidence (1 in 16,000-18,000 live births) by number of forecast surviving infants. […] Although prevalence is higher in middle- to low-income countries, most retinoblastoma treatment centres are in middle- and high-income countries, creating a gap in healthcare access.

• #36 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  A number of initiatives address the inequality in retinoblastoma treatment between developing and developed countries. […] The KNRbS has supported the standardization of histopathologic examination of the enucleated eye to inform treatment decisions and discussion of prognosis with families. […] Adjuvant systemic chemotherapy after high-risk features are observed in an enucleated eye may reduce risk of metastatic relapse. […] The expected number of patients with retinoblastoma annually per country is most accurately calculated by multiplying the global retinoblastoma incidence (1 in 16,000-18,000 live births) by number of forecast surviving infants. […] Although prevalence is higher in middle- to low-income countries, most retinoblastoma treatment centres are in middle- and high-income countries, creating a gap in healthcare access.

• #37 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Retinoblastoma is a highly curable neoplasm in high-income countries, where patient survival exceeds 99%, making it the most curable of all pediatric cancers. In striking contrast, in many lower-income countries, most patients present with disseminated and metastatic disease, which is almost always fatal. […] Despite the changing paradigm of retinoblastoma treatment from systemic to OAC and IVi chemotherapy, eyes that relapse or that are initially refractory to conventional therapy are still difficult to treat with currently available drug options and most undergo enucleation. Thus, drug discovery in retinoblastoma is of paramount importance. […] The aim in developing OAC was to deliver the drug directly into the artery that irrigates the ocular tissues to increase local bioavailability while minimizing systemic exposure.

• #38 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  A number of initiatives address the inequality in retinoblastoma treatment between developing and developed countries. […] The KNRbS has supported the standardization of histopathologic examination of the enucleated eye to inform treatment decisions and discussion of prognosis with families. […] Adjuvant systemic chemotherapy after high-risk features are observed in an enucleated eye may reduce risk of metastatic relapse. […] The expected number of patients with retinoblastoma annually per country is most accurately calculated by multiplying the global retinoblastoma incidence (1 in 16,000-18,000 live births) by number of forecast surviving infants. […] Although prevalence is higher in middle- to low-income countries, most retinoblastoma treatment centres are in middle- and high-income countries, creating a gap in healthcare access.

• #39 Retinoblastoma | Children’s Cancer Web

  http://www.cancerindex.org/ccw/guide2r.htm

  Retinoblastoma is a rare tumour of the eye which develops in the cells of the retina, most patients are under 5 years old. […] The median onset age was 21 months. […] The most common presenting signs were leukocoria (73.6%) and strabismus (20.7%). […] The mean age at diagnosis was 23.5 months, and time to diagnosis was 5.4 months. […] In patients with clinical features of hereditary Rb, both onset of the first sign/symptom and diagnosis were at an earlier age than in patients without these features (12.3 vs 21.6 months [P=0.001] and 15.9 vs 28.0 months [P=0.001]). […] The current era of retinoblastoma management using intra-arterial chemotherapy plus additional intravitreal chemotherapy (as needed for vitreous seeding) has improved globe salvage in eyes with advanced retinoblastoma. […] Overall survival, eye salvage, and preservation of vision are largely dependent on the stage of disease at presentation. […] Early diagnosis, multidisciplinary treatment, and genetic counseling are all priorities in the management of this tumor.

• #40 Treatment of Retinoblastoma: What Is the Latest and What Is the Future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9010858/

  Key outcomes of the widespread adoption of OAC include: Eliminating the use of EBRT, which has decreased the incidence of second, non-ocular tumors and thereby improved long-term survival. […] The introduction of ophthalmic artery chemosurgery (OAC) led to remarkable success in treating eyes with more advanced disease in which systemic chemotherapy had poorer results. […] Recent studies have used innovative multi-omics technology to identify deregulated pathways that could be targeted via novel treatment strategies in retinoblastoma. […] New drugs effective for both intraocular and disseminated retinoblastoma are urgently needed; these may include novel drugs and repurposed agents, i.e., those already used in the clinic for other cancers or diseases but still without an indication for retinoblastoma.

• #41 Retinoblastoma | Children’s Cancer Web

  http://www.cancerindex.org/ccw/guide2r.htm

  Retinoblastoma is a rare tumour of the eye which develops in the cells of the retina, most patients are under 5 years old. […] The median onset age was 21 months. […] The most common presenting signs were leukocoria (73.6%) and strabismus (20.7%). […] The mean age at diagnosis was 23.5 months, and time to diagnosis was 5.4 months. […] In patients with clinical features of hereditary Rb, both onset of the first sign/symptom and diagnosis were at an earlier age than in patients without these features (12.3 vs 21.6 months [P=0.001] and 15.9 vs 28.0 months [P=0.001]). […] The current era of retinoblastoma management using intra-arterial chemotherapy plus additional intravitreal chemotherapy (as needed for vitreous seeding) has improved globe salvage in eyes with advanced retinoblastoma. […] Overall survival, eye salvage, and preservation of vision are largely dependent on the stage of disease at presentation. […] Early diagnosis, multidisciplinary treatment, and genetic counseling are all priorities in the management of this tumor.

• #42 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  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. […] Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. […] Poor outcome correlates with late diagnosis, difficulty accessing retinoblastoma-specific health care, and socio-economic issues leading to poor compliance, including family refusal to remove the affected eye (enucleation) and abandonment of therapy. […] Fortunately, with early diagnosis, many eyes can be safely treated and support a lifetime of good vision, pointing to key elements for global focus: awareness, collaboration, and affordable expert care.

• #43 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  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. […] Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. […] Poor outcome correlates with late diagnosis, difficulty accessing retinoblastoma-specific health care, and socio-economic issues leading to poor compliance, including family refusal to remove the affected eye (enucleation) and abandonment of therapy. […] Fortunately, with early diagnosis, many eyes can be safely treated and support a lifetime of good vision, pointing to key elements for global focus: awareness, collaboration, and affordable expert care.

• #44 Retinoblastoma outcome at a single institution in South Africa

  http://www.scielo.org.za/scielo.php?script=sci_arttext&pid=S0256-95742014001200015

  Retinoblastoma (RB) is the most common eye cancer in children. Early detection is necessary for cure. […] Poor outcome is probably a result of late diagnosis. It is important to implement a strategy that will ensure early diagnosis and optimal management of RB in SA. […] Survival is excellent in developed countries, where the survival rate is 95%, but it is only ~50% in developing countries, mostly because of late diagnosis and extraocular disease at diagnosis. […] Late diagnosis and advanced disease are common in developing countries, however, and associated with an increased risk of extraocular disease and poor outcome. […] The major reason for poor outcome was progression of disease, followed by relapse in 5 patients in each group. […] Advanced disease in this study was associated with an overall survival rate of 70%. A strategy is necessary in SA to ensure that children with RB are diagnosed early and that 90% of them are cured.

• #45 Retinoblastoma

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5744255/

  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. […] Life-long follow-up is warranted since survivors of heritable retinoblastoma are at risk for developing second cancers. […] Poor outcome correlates with late diagnosis, difficulty accessing retinoblastoma-specific health care, and socio-economic issues leading to poor compliance, including family refusal to remove the affected eye (enucleation) and abandonment of therapy. […] Fortunately, with early diagnosis, many eyes can be safely treated and support a lifetime of good vision, pointing to key elements for global focus: awareness, collaboration, and affordable expert care.

• #46 Retinoblastoma | Children’s Cancer Web

  http://www.cancerindex.org/ccw/guide2r.htm

  Retinoblastoma is a rare tumour of the eye which develops in the cells of the retina, most patients are under 5 years old. […] The median onset age was 21 months. […] The most common presenting signs were leukocoria (73.6%) and strabismus (20.7%). […] The mean age at diagnosis was 23.5 months, and time to diagnosis was 5.4 months. […] In patients with clinical features of hereditary Rb, both onset of the first sign/symptom and diagnosis were at an earlier age than in patients without these features (12.3 vs 21.6 months [P=0.001] and 15.9 vs 28.0 months [P=0.001]). […] The current era of retinoblastoma management using intra-arterial chemotherapy plus additional intravitreal chemotherapy (as needed for vitreous seeding) has improved globe salvage in eyes with advanced retinoblastoma. […] Overall survival, eye salvage, and preservation of vision are largely dependent on the stage of disease at presentation. […] Early diagnosis, multidisciplinary treatment, and genetic counseling are all priorities in the management of this tumor.

Zobacz więcej