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Introduction: Patients with high-risk or metastatic Ewing sarcoma (ES) and rhabdomyosarcoma (RMS) have a guarded prognosis. High-dose chemotherapy (HDT) with autologous stem cell transplant (ASCT) has been evaluated as a treatment option to improve outcomes. However, survival benefits remain unclear, and treatment is associated with severe toxicities., Methods: A systematic review was conducted, using the population, intervention, comparison outcome (PICO) model, to evaluate whether utilization of HDT/ASCT impacts the outcome of patients with ES and RMS compared to standard chemotherapy alone, as part of first line treatment or in the relapse setting. Medline, Embase and Cochrane Central were queried for publications from 1990 to October 2022 that evaluated event-free survival (EFS), overall survival (OS), and toxicities. Each study was screened by two independent reviewers for suitability. A qualitative synthesis of the results was performed., Results: Of 1,172 unique studies screened, 41 studies were eligible for inclusion with 29 studies considering ES, 10 studies considering RMS and 2 studies considering both. In ES patients with high-risk localised disease who received HDT/ASCT after VIDE chemotherapy, consolidation with melphalan-based HDT/ASCT as first line therapy conveyed an EFS and OS benefit over standard chemotherapy consolidation. Efficacy of HDT/ASCT using a VDC/IE backbone, which is now standard care, has not been established. Survival benefits are not confirmed for ES patients with metastatic disease at initial diagnosis. For relapsed/refractory ES, four retrospective studies report improvement in outcomes with HDT/ASCT with the greatest evidence in patients who demonstrate a treatment response before HDT, and in patients under the age of 14. In RMS, there is no proven survival benefit of HDT/ASCT in primary localised, metastatic or relapsed disease., Conclusion: Prospective randomised trials are required to determine the utility of HDT/ASCT in ES and RMS. Selected patients with relapsed ES could be considered for HDT/ASCT., Competing Interests: Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Angela M Hong: advisory board payment from Telix, other authors: None., (Copyright © 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved.)

A diagnosis of cancer impacts the person's physical and mental health and the psychosocial and financial health of their caregivers. While data on the experience of living with cancer is available, there is a dearth of data from persons in low- and middle-income countries (LMICs). The perspectives of other impacted individuals also remain understudied (e.g., bereaved family members), as well as the impact on survivors and their families over time. The objective of this study is to describe the psychosocial and financial impact of cancer on people diagnosed with cancer as a child, adolescent or adult, their families/caregivers, and the family members of those who have died from cancer, in high-income countries (HICs) and LMICs. This study is an observational, descriptive, quantitative study. Data will be collected anonymously via a digital online cross-sectional survey distributed globally by the World Health Organization (WHO) via the LimeSurvey software. Participants will include (a) adults aged 18+ who have been diagnosed with cancer at any age, who are currently undergoing cancer treatment or who have completed cancer treatment; (b) adult family members of individuals of any age with a cancer diagnosis, who are currently undergoing cancer treatment or who have completed cancer treatment; and (c) bereaved family members. Participants will be anonymously recruited via convenience and snowball sampling through networks of organisations related to cancer. Survey results will be analysed quantitatively per respondent group, per time from diagnosis, per disease and country. Results will be disseminated in peer-reviewed journals and at scientific conferences; a summary of results will be available on the WHO website. This study will suggest public health interventions and policy responses to support people affected by cancer and may also lead to subsequent research focusing on the needs of people affected by cancer., Competing Interests: The authors have declared that no competing interests exist., (Copyright: This is an open access article, free of all copyright, and may be freely reproduced, distributed, transmitted, modified, built upon, or otherwise used by anyone for any lawful purpose. The work is made available under the Creative Commons CC0 public domain dedication.)

Background: An estimated 400,000 children develop cancer worldwide. Of those, 90% occur in low- and middle-income countries, where survival rates can be as low as 30%. To reduce the childhood cancer survival gap between high- and low- and middle-income countries (LMIC), the World Health Organization launched the Global Initiative for Childhood Cancer in 2018, to support governments in building sustainable childhood cancer programs, with the aim to increase access and quality of care for children with cancer. Developing a high-quality and trained workforce is key to the success of childhood cancer services, but more information is needed on the interventions used to develop and train a workforce. The objective of this review is to understand the key factors described in the literature in relation to the development and training of a workforce in childhood cancer (defined here as ages 0-19) in LMIC, including challenges, interventions and their outcomes., Methods: We will include sources of evidence that describe the development or training of a childhood cancer workforce in health services that diagnose, refer or treat children and adolescents with cancer, in low- and middle-income countries as defined by the World Bank. The following databases will be searched: OVID Medline, Embase and Pubmed from 2001 to present with no restriction of language. Grey literature searches will also be performed in Proquest Dissertation and Theses, as well as relevant organizations' websites, and conference proceedings will be searched in conference websites. In addition, references lists will be reviewed manually. Two people will screen abstracts and full-texts and extract data. Data will be presented in a table or chart, with an accompanying narrative summary responding to the review questions. A framework synthesis will be conducted: data will be charted against a framework adapted from the 2016 WHO Global Strategy for Human Resources for Heath: Workforce 2030., Discussion: This scoping review will allow to map the existing literature on workforce development in LMIC, identify potential interventions and highlight data and knowledge gaps. This constitutes a first step towards adopting successful strategies more broadly, formulating research priorities and developing effective policies and interventions., Systematic Review Registration: Open Science Framework osf.io/3mp7n., (© 2022. The Author(s).)

With the World Health Organization (WHO) Global Initiative for Childhood Cancer, there is renewed interest in sustainable interventions to improve childhood cancer care in low-/middle-income countries (LMICs). Practitioners in LMICs have traditionally practiced "twinning," i.e., targeted international pediatric oncology partnerships (TIPPs) between one or more institutions in a high-income country (HIC) and an LMIC, to improve care for children with cancer in the latter. The International Society of Paediatric Oncology Committee for Paediatric Oncology in Developing Countries Working Group on Twinning, Collaboration, and Support reviewed guidelines from https://cancerpointe.com and the current literature, gathered input from practitioners in LMICs, and in this article discuss the role of TIPPs in the WHO initiative., (© 2020 Wiley Periodicals LLC.)

Background: The 9q22.3 syndrome is an autosomal dominant microdeletion syndrome with similarities to Gorlin syndrome (GS). It encompasses the PTCH1 gene locus that harbors mutations for GS. Although the 9q22.3 syndrome is associated with Wilms tumor (WT), WT is not a GS-associated tumor, implying a different mechanism involving PTCH1, or a different locus in the 9q22.3 region. The goal of this study is to report the association between WT and 9q22.3 syndrome and review the outcome of treatment., Observations: We report 2 new cases of WT with 9q22.3 deletion and review the literature. Among the 44 described patients with 9q22.3 deletion, 7 developed WT (16%) at a mean age of 45 months (range, 4 to 84 mo). All patients had dysmorphic features, macrocephaly, and developmental delay, and there was an association with overgrowth (4/7). One patient had bilateral WT, another had a synchronous rhabdomyosarcoma. The outcome was excellent with all cases reported to be in complete remission., Conclusions: The 9q22.3 microdeletion syndrome should be considered at diagnosis of WT in children with dysmorphic features. Conversely, patients with a known 9q22.3 deletion syndrome should be considered for a WT predisposition surveillance program, especially those with overgrowth. The management should be individualized and given the excellent prognosis, and the unknown future risk of metachronous disease or other malignancy, the surgical approach should be carefully considered.

Background: Reduction in neonatal deaths has been a major challenge globally. To prevent neonatal deaths, improvements in newborn care have been promoted worldwide. The World Health Organization Western Pacific Regional Office has been promoting the Early Essential Newborn Care (EENC), a package of specific simple and cost-effective interventions, in their region. However, mere introduction of EENC cannot reduce neonatal deaths unless quality of care is ensured. In Lao PDR, the government introduced self-managed continuous monitoring as a sustainable way to improve the quality of care described in the EENC., Methods: A clustered randomized controlled trial was designed to compare the effectiveness of self-managed continuous monitoring with external supervisory visits to monitor health workers' satisfactory EENC performance and their knowledge and skills related to the EENC in Lao PDR. Determinants of EENC performance will be measured with a structured questionnaire developed based on the Theory of Planned Behaviour, which predicts future behaviour. During self-managed continuous monitoring activities, health workers in each district hospital will conduct periodical peer reviews and feedback sessions. Fifteen district hospitals will be randomly allocated into the self-managed continuous monitoring (intervention) and the supervision (control) groups. Fifteen health workers routinely involved in maternity and newborn care including physicians, midwives and other health staff will be recruited from each hospital (effect size 0.6, intra-cluster correlation coefficient 0.06, 5% alpha error and 80% power). We will compare the change in the mean score of the determinants before and one year after randomisation between the two groups. We will also compare the retention of knowledge and skills related to the EENC between the two groups. The expected enrolment period is July 20th, 2017 to July 20th, 2018., Discussion: This is the first cluster randomized trial to evaluate a self-managed continuous monitoring system for quality maintenance of newborn care in a resource-limited country. This research is conducted in collaboration with the Ministry of Health and international organizations; therefore, if effective, this intervention would be applied in larger areas of the country and the region., Trial Registration: This trial was registered at UMIN-CTR on 15th of June, 2017. Registration number is UMIN000027794 .

Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of lymphocyte apoptosis. Children present with chronic nonmalignant lymphadenopathy, hepatosplenomegaly, and autoimmune cytopenias. Recent advances show efficacy of treatment with immunosuppressive drugs. Sirolimus, an mammalian target of rapamycin inhibitor, improves autoimmune cytopenias and lymphoproliferation, with a safe profile. We present 2 patients, a 5-year-old girl and 15-year-old boy, diagnosed with ALPS with initial partial response to steroid treatment. Autoimmune cytopenias and lymphoproliferation then became refractory to treatment, with recurrence of symptoms. In both cases, treatment with sirolimus was started, with a rapid response, complete remission of cytopenias, and resolution of lymphoproliferation, with no significant adverse effects., Conclusion: sirolimus is an effective and safe drug for controlling children with cytopenias and lymphoproliferation linked to ALPS.

Unlabelled: The objective was to determine the uselfulness of D Dimer (DD) as a diagnostic or prognostic marker in acute appendicitis (AA) in children using a prospective observational study in the pediatric emergency department of a tertiary hospital. We enrolled 135 patients aged 1-16 years presenting with abdominal pain consistent with AA, who required laboratory studies. We analyzed clinical, analytical variables and histopathology findings (when they underwent surgery). Statistical analysis was conducted using SPSS. 38.5% of the children were clinically diagnosed with AA (n = 52), confirmed by pathology in 51 patients. 55.8% were gangrenous appendicitis. Leucocyte count, C-reactive protein (CRP), and DD were higher in the AA group and in the gangrenous appendicitis group (p < 0.05), with highest values of DD in the gangrenous group. The area under the receiving operating characteristics (ROC) curve for DD in the diagnosis of AA is 0.66 (95% CI 0.56-0.75). For DD cut-off point of 230 ng/mL, sensitivity (Se) was 0.40, specificity (Sp) 0.80, positive predictive value (PPV) 0.57, and negative predictive value (NPV) 0.66. The area under the ROC curve for DD in children with gangrenous appendicitis is 0.93 (95% CI 0.87-1). A DD cut-off point of 230 ng/mL exhibited: Se = 0.69, Sp = 1, PPV = 1 and NPV = 0.72., Conclusion: DD levels increase in patients with AA. Although it does not constitute a useful diagnostic marker, it could be a good prognostic marker.