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. Objectives: To report the retinoblastoma stage at diagnosis in patients across the world during a single year, to investigate associations between clinical variables and national income level, and to investigate risk factors for advanced disease at diagnosis. Design, Setting, and Participants: A total of 278 retinoblastoma treatment centers were recruited from June 2017 through December 2018 to participate in a cross-sectional analysis of treatment-naive patients with retinoblastoma who were diagnosed in 2017. Main Outcomes and Measures: Age at presentation, proportion of familial history of retinoblastoma, and tumor stage and metastasis. Results: The cohort included 4351 new patients from 153 countries; the median age at diagnosis was 30.5 (interquartile range, 18.3-45.9) months, and 1976 patients (45.4%) were female. Most patients (n = 3685 [84.7%]) were from low- and middle-income countries (LMICs). Globally, the most common indication for referral was leukocoria (n = 2638 [62.8%]), followed by strabismus (n = 429 [10.2%]) and proptosis (n = 309 [7.4%]). Patients from high-income countries (HICs) were diagnosed at a median age of 14.1 months, with 656 of 666 (98.5%) patients having intraocular retinoblastoma and 2 (0.3%) having metastasis. Patients from low-income countries were diagnosed at a median age of 30.5 months, with 256 of 521 (49.1%) having extraocular retinoblastoma and 94 of 498 (18.9%) having metastasis. Lower national income level was associated with older presentation age, higher proportion of locally advanced disease and distant metastasis, and smaller proportion of familial history of retinoblastoma. Advanced disease at diagnosis was more common in LMICs even after adjusting for age (odds ratio for low-income countries vs upper-middle-income countries and HICs, 17.92 [95% CI, 12.94-24.80], and for lower-middle-income countries vs upper-middle-income countries and HICs, 5.74 [95% CI, 4.30-7.68]). Conclusions and Relevance: This study is estimated to have included more than half of all new retinoblastoma cases worldwide in 2017. Children from LMICs, where the main global retinoblastoma burden lies, presented at an older age with more advanced disease and demonstrated a smaller proportion of familial history of retinoblastoma, likely because many do not reach a childbearing age. Given that retinoblastoma is curable, these data are concerning and mandate intervention at national and international levels. Further studies are needed to investigate factors, other than age at presentation, that may be associated with advanced disease in LMICs

The global retinoblastoma outcome study : a prospective, cluster-based analysis of 4064 patients from 149 countries

DATA SHARING : The study data will become available online once all analyses are complete.BACKGROUND : Retinoblastoma is the most common intraocular cancer worldwide. There is some evidence to suggest that major differences exist in treatment outcomes for children with retinoblastoma from different regions, but these differences have not been assessed on a global scale. We aimed to report 3-year outcomes for children with retinoblastoma globally and to investigate factors associated with survival. METHODS : We did a prospective cluster-based analysis of treatment-naive patients with retinoblastoma who were diagnosed between Jan 1, 2017, and Dec 31, 2017, then treated and followed up for 3 years. Patients were recruited from 260 specialised treatment centres worldwide. Data were obtained from participating centres on primary and additional treatments, duration of follow-up, metastasis, eye globe salvage, and survival outcome. We analysed time to death and time to enucleation with Cox regression models. FINDINGS : The cohort included 4064 children from 149 countries. The median age at diagnosis was 23·2 months (IQR 11·0–36·5). Extraocular tumour spread (cT4 of the cTNMH classification) at diagnosis was reported in five (0·8%) of 636 children from high-income countries, 55 (5·4%) of 1027 children from upper-middle-income countries, 342 (19·7%) of 1738 children from lower-middle-income countries, and 196 (42·9%) of 457 children from low-income countries. Enucleation surgery was available for all children and intravenous chemotherapy was available for 4014 (98·8%) of 4064 children. The 3-year survival rate was 99·5% (95% CI 98·8–100·0) for children from high-income countries, 91·2% (89·5–93·0) for children from upper-middle-income countries, 80·3% (78·3–82·3) for children from lower-middle-income countries, and 57·3% (52·1-63·0) for children from low-income countries. On analysis, independent factors for worse survival were residence in low-income countries compared to high-income countries (hazard ratio 16·67; 95% CI 4·76–50·00), cT4 advanced tumour compared to cT1 (8·98; 4·44–18·18), and older age at diagnosis in children up to 3 years (1·38 per year; 1·23–1·56). For children aged 3–7 years, the mortality risk decreased slightly (p=0·0104 for the change in slope). INTERPRETATION : This study, estimated to include approximately half of all new retinoblastoma cases worldwide in 2017, shows profound inequity in survival of children depending on the national income level of their country of residence. In high-income countries, death from retinoblastoma is rare, whereas in low-income countries estimated 3-year survival is just over 50%. Although essential treatments are available in nearly all countries, early diagnosis and treatment in low-income countries are key to improving survival outcomes.The Queen Elizabeth Diamond Jubilee Trust and the Wellcome Trust.https://www.thelancet.com/journals/langlo/homeam2023Paediatrics and Child Healt

Case report: A Ten Year Old Boy with Unexplained Hemolytic Anemia

Babesiosis is a tick - borne zoonotic disease transmitted by the intracellular protozoan from genus Babesia. Infection with is uncommon and symptomatic disease is mostly confined to splenectomized patients. It may be transmitted by blood transfusion. In splenectomized patients, the disease has an acute onset and is often fatal. Unsplenectomized patients may experience a milder self - limiting disease, although intravascular hemolysis does occur. The diagnosis is made from the peripheral blood film, where the parasites, looking very similar to malaria, are seen inside the red cells. In this case report, we describe a child with unexplained hemolytic anemia with presumptive diagnosis of autoimmune hemolytic anemia treated with Steroids and immunosuppressive treatment with no response. After review of his investigation, surprisingly, his peripheral blood film revealed Babesiosis

Childhood cancer care in the Middle East, North Africa, and west/central Asia: A snapshot across five countries from the POEM network

Background: The Pediatric Oncology East and Mediterranean (POEM) network, through this report, provides a snapshot view of an expected child\u27s treatment journey in five countries in the region.Methods: Pediatric oncologists from cancer centers in Egypt, Lebanon, Iraq, Jordan, and Pakistan provided input on referral pathways, barriers to care, and patient outcomes, based on personal experience and published data. Outcome data were extracted from institutional registries. A literature review of articles and meeting abstracts was conducted, and results summarized.Results: Countries across the Middle Eastern, North African, and West Asian region face common difficulties relating to the provision of pediatric oncology care. National registries are largely lacking, with unavailability of outcome data. Economic barriers are a common theme, leading to delays in patient diagnosis, and interruptions and abandonment of therapy. Insufficient infrastructure and human resources, high rates of toxic deaths, and lack of common national protocols are common. The establishment of successful fundraising organizations linked to specific cancer hospitals showcase several success stories, enhancing services, improving patient access, and leading to outcomes comparable to those in developed countries. All identified published literature is institution-based and from only one or a few hospitals. Therefore, outcomes at a national level likely differ due to disparate cancer care capabilities.Conclusion: Well-designed national registries are essential for identifying gaps, and clear referral networks are needed to address delays to diagnosis and therapy. National and transversal programs to improve infrastructure, facilitate knowledge transfer, and promote advocacy, are needed to accelerate progress in the region

KIT gene deletion in a gastrointestinal stromal tumor of the small intestine.

[No abstract available

Chronic immune thrombocytopenic purpura in children overview of 60 patients

Background: A small percentage of children with Immune thrombocytopenic purpura (ITP) suffer from a clinically significant disease with severe thrombocytopenia that requires intervention. Treatment for these children presents a challenge as there are few known therapies that offer long-term remission, and all that are known have significant side effects and toxicities. Aim of the study: To evaluate the effects of a variety of treatment modalities on the clinical course, and long treatment outcomes in children with chronic ITP. Patients & methods: A study involved 60 children with chronic ITP who were referred to Hemato-Oncology unit/Children's Welfare Teaching Hospital/Medical City/Baghdad. Treatment of patients included steroid, Intravenous Immunoglobulins, Anti D immunoglobulin, 6-Mercaptopurine, Rituximab and splenectomy. The Period of data collection and analysis was from May 2009 to May 2011. Results: The most common presenting symptom was skin bleeding, seen in 42 (70%) patients. Thirty-four patients received one or more courses of steroids. Complete response was achieved in 7 (20.5%) patients while there was no response in 12 (35.2%) patients, Intravenous immunoglobulin was used for 5 patients, only one (16%) exhibited a good response. Anti D Immunoglobulin was used in six patients; only one (8.3%) patient got good response. Twelve patients received 6-mercaptopurine, only one (8.3%) patient had a partial response. Six patients received Rituximab; three (50%) had a partial response. Six patients underwent splenectomy; response was noted in 5/6 (83.3%) patients. At the end of the study; complete response was seen in 13 (22.4%) patients, partial in 19 (31.6%), no response in 28 (46.7%) patients. Conclusions: Splenectomy is the most effective treatment modality when treating children with chronic ITP whose symptoms are severe

Gastric Perforation as a Primary Manifestation of Lymphomatoid Granulomatosis

[No abstract available