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Background In recent years, reduced participation has been encountered across all epidemiological study designs, both in terms of non-response as well as refusal. A low response rate may reduce the statistical power but, more importantly, results may not be generalizable to the wider community. Methods In a telephone survey of 1413 randomly selected members of the Australian general population and of 690 participants sourced from previous studies, we examined factors affecting people’s stated willingness to participate in health research. Results The majority of participants (61 %) expressed willingness to participate in health research in general but the percentage increased when provided with more specific information about the research. People were more willing if they have personal experience of the disease under study, and if the study was funded by government or charity rather than pharmaceutical companies. Participants from previous studies, older people and women were the groups most willing to participate. Younger men preferred online surveys, older people a written questionnaire, and few participants in any age and sex groups preferred a telephone questionnaire. Conclusion Despite a trend toward reduced participation rates, most participants expressed their willingness to participate in health research. However, when seeking participants, researchers should be concrete and specific about the nature of the research they want to carry out. The preferred method of recommended contact varies with the demographic characteristics.

Background: Distinguishing between the anatomical variant of an anterior anus and mild forms of imperforate anus with rectoperineal fistula often requires inspection, calibration, and, in uncertain cases, electrical stimulation (ES) under general anesthesia. Anorectal manometry (AM), despite its ability to assess sphincter configuration and function, is rarely reported as a diagnostic tool. This study evaluated the utility of AM in such cases. Methods: A retrospective analysis of AM and clinical data from 38 patients (35 female, 3 male) with suspected anterior anus was conducted from October 2009 to September 2021. Water-perfused catheter probes with eight radial channels were used to perform pull-through maneuvers. Sphincter locations were identified through vector reconstruction, and pressure ratios of the anterior part to the circumference were recorded. Results were compared to clinical data, including ES findings. Statistical significance was assessed using Mann–Whitney U and Chi-Square tests. Results: Following AM, ES was unnecessary in 25 patients. Of the remaining patients, 83% showed abnormal sphincter configurations on ES, and seven underwent anoplasty. Patients with abnormal sphincter complexes demonstrated significantly lower mean anterior pressures (61.2 mmHg vs. 136.4 mmHg, U = 336.5, p = 0.001) and a trend toward lower anterior-to-circumferential pressure ratios (mean 0.42 vs. 0.85, U = 613, p = 0.270). Constipation was also more frequent in this group (X2(1,N = 38) = 4.1, p = 0.044). Average anterior pressures < 75 mmHg and ratios < 0.7 indicated an anus outside the sphincter complex (sensitivity 80%, specificity 90%). Conclusions: AM proves valuable for evaluating ambiguous anterior anus cases, potentially reducing reliance on ES under general anesthesia. 3D high-resolution AM may further increase diagnostic accuracy.

Restorative justice as a response to sexual violence continues to be subject to significant criticism. To assess the evidence, we sought to appraise and synthesize evaluations of restorative justice programs for sexual and family violence offences by conducting a systematic review of peer-reviewed literature. However, our rigorous search identified only one eligible study. This dearth of evidence leaves us unable to identify how best to achieve the aims for which programs were established and poses difficulties for policy makers determining whether it is justified to introduce restorative justice programs for sexual and family violence. We recommend that evaluations of restorative justice programs that accept sexual and family violence cases be conducted as a matter of urgency.

The adoptive transfer of CD19-specific chimeric antigen receptor engineered T cells (CAR T cells) resulted in encouraging clinical trials in indolent B-cell malignancies. However, they also show the limitations of this fascinating technology: CAR T cells can lead to even life-threatening off-tumor, on-target side effects if CAR T cells crossreact with healthy tissues. Here, we describe a novel modular universal CAR platform technology termed UniCAR that reduces the risk of on-target side effects by a rapid and reversible control of CAR T-cell reactivity. The UniCAR system consists of two components: (1) a CAR for an inert manipulation of T cells and (2) specific targeting modules (TMs) for redirecting UniCAR T cells in an individualized time- and target-dependent manner. UniCAR T cells can be armed against different tumor targets simply by replacement of the respective TM for (1) targeting more than one antigen simultaneously or subsequently to enhance efficacy and (2) reducing the risk for development of antigen-loss tumor variants under treatment. Here we provide ‘proof of concept’ for retargeting of UniCAR T cells to CD33- and/or CD123-positive acute myeloid leukemia blasts in vitro and in vivo .

Certain product development partnerships (PDPs) recognize that to promote the reduction of global health disparities they must create access to their products and strengthen research capacity in developing countries. We evaluated the contribution of 3 PDPs—Medicines for Malaria Venture, Drugs for Neglected Diseases Initiative, and Institute for One World Health—according to Frost and Reich’s access framework. We also evaluated PDPs’ capacity building in low- and middle-income countries at the individual, institutional, and system levels. We found that these PDPs advance public health by ensuring their products’ registration, distribution, and adoption into national treatment policies in disease-endemic countries. Nonetheless, ensuring broad, equitable access for these populations—high distribution coverage; affordability, particularly for the poor; and adoption at provider and end-user levels—remains a challenge.

Background/Objectives: Complex cases in pediatric surgery involving multiple congenital anomalies pose significant diagnostic and therapeutic challenges. These conditions require coordinated interdisciplinary care tailored to the individual patient. We present a case of syndromic congenital anomalies in a neonate, later diagnosed with CHARGE syndrome, to illustrate the importance of staged, multidisciplinary management. Methods: A 34-year-old woman in her third pregnancy developed significant polyhydramnios at 31 weeks of gestation, followed by preterm labor. The neonate presented with esophageal atresia with tracheoesophageal fistula (EA/TEF), intrathoracic stomach, aortic coarctation, patent ductus arteriosus, atrial septal defect, and bilateral choanal atresia. A structured treatment protocol was developed and implemented at Klinikum Stuttgart by an interdisciplinary team comprising gynecology, pediatric surgery, cardiology, ENT, neonatology, and genetics. Results: Initial pediatric surgical procedures included ligation of the tracheoesophageal fistula, repositioning of the intrathoracic stomach, and primary esophageal anastomosis. Cardiovascular anomalies were managed through staged interventions. Bilateral choanal atresia was surgically corrected. Genetic testing confirmed CHARGE syndrome. Postoperative care included respiratory support, enteral nutrition, and regular esophageal dilations. Due to persistent reflux esophagitis, antireflux surgery is planned. Conclusions: This case underscores the importance of a highly individualized and interdisciplinary approach in the management of syndromic congenital anomalies. The presence of CHARGE syndrome with multiple system involvement required careful staging of surgical interventions and long-term coordination of follow-up care. Early genetic diagnosis and integrated team planning were critical in optimizing outcomes in this complex neonatal case.

International collaborative health research is justifiably expected to help reduce global health inequities. Investment in health policy and systems research in developing countries is essential to this process but, currently, funding for international research is mainly channelled towards the development of new medical interventions. This imbalance is largely due to research legislation and policies used in high-income countries. These policies have increasingly led these countries to invest in health research aimed at boosting national economic competitiveness rather than reducing health inequities. In the United States of America and the United Kingdom of Great Britain and Northern Ireland, the regulation of research has encouraged a model that: leads to products that can be commercialized; targets health needs that can be met by profitable, high-technology products; has the licensing of new products as its endpoint; and does not entail significant research capacity strengthening in other countries. Accordingly, investment in international research is directed towards pharmaceutical trials and product development public-private partnerships for neglected diseases. This diverts funding away from research that is needed to implement existing interventions and to strengthen health systems, i.e. health policy and systems research. Governments must restructure their research laws and policies to increase this essential research in developing countries.

Background: Wound healing in deep dermal burn injuries continues to be a challenge in paediatrics. In the absence quick and spontaneous wound closure, split-thickness skin grafting is often necessary. Since the development of a new type of acellular fish matrix, which is very similar to the human skin matrix, skin closure and wound conditioning can be achieved without split-thickness skin grafting. Methods: The following study shows a case series of 20 children in whom a fish skin graft was used. The aim was to develop an algorithm for selecting and using fish skin and its long-term results after one and two years. Acellular fish skin worked as a granulation base for wound healing and also as a substitute for split-thickness skin grafts. Results: There was no evidence of infection. Skin transplants and, thus, additional operations could be avoided. The follow-up examinations showed an excellent result, both objectively by means of elasticity measurements (DermalabCombo®) and in the subjective assessment of the skin as part of the Patient and Observer Scar Assessment Scale (POSAS). Conclusion: Fish skin grafts are a good alternative to split-thickness skin grafts for deep dermal wounds. These results should be further analysed with a larger number of patients in future publications.

Sigmoid volvulus (SV) occurs rarely in children. After encountering two cases of recurrent SV, we reviewed the literature to define the recurrence risk, identify outcome predictors and to give treatment recommendations. We found 256 cases reported in children (mean age 10.2 years, gender ratio (m:f) 2.3:1). Associations exist with Hirschsprung disease (HD) in 10%, neurodevelopmental disorders in 10.9% and chronic constipation in 10.2%. Common symptoms and clinical signs were abdominal pain (85%), distension (85%), tenderness (54%) and vomiting (59%). Signs of peritonitis were present in 14% and indicated a gangrenous sigmoid (X2 = 45.33; p < 0.001). A total of 183 had abdominal radiographs, and 65% showed a positive ‘coffee-bean-sign’. Contrast enemas were positive in 90%. A total of 124 patients underwent laparotomy; in 41 cases, the sigmoid was gangrenous and associated with more complications (X2 = 15.68; p < 0.001). Non-operative treatment (NOT) like endoscopic, fluoroscopic or rectal tube decompression was performed in 135 patients and successful in 79% with a 38–57% recurrence rate. A total of 73 patients subjected to elective surgery: 50 underwent sigmoid resection; 17 had surgery for HD. Clinicians should consider SV in all children with abdominal pain, distension and vomiting. Gangrene carries a higher morbidity. After successful NOT we recommend counselling about the recurrence risk and definitive surgery should be advised. HD is frequent in newborns but sometimes found in older children.