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Drug usage and adverse drug reactions in paediatric patients.

The development of epidemiological methods for the study of adverse drug reactions is reviewed in connection with the presentation of data obtained by intensive monitoring of 1 000 admissions to a medical paediatric unit. Compared with adults and American children, the patients received fewer drugs and experienced fewer reactions while in hospital. The drug usage pattern was different from that of American paediatric practice and general practice in the United Kingdom. Fifty-one (6%) patients experienced 119 adverse drug reactions. These occurred more frequently in children suffering from serious disorders and in the majority of cases the basic therapy was continued regardless of the severity of the drug side-effects. Treatment was required for the effects of 66 (55%) adverse reactions. It appears that drug monitoring in paediatric practice may be of greater value if surveillance programmes are designed to provide a "therapeutic audit" and extended to include children receiving drugs in the community.

Child

Biallelic pathogenic variants in FLNB are associated with paediatric steroid-resistant nephrotic syndrome via podocyte cytoskeletal dysfunction.

BACKGROUND: Steroid-resistant nephrotic syndrome (SRNS) is a severe paediatric kidney disease and a leading cause of end-stage kidney disease in children, with a high genetic contribution. While over 80 monogenic causes of SRNS have been identified, a significant proportion of affected patients still lack a clear genetic diagnosis, indicating that additional causative genes remain to be discovered. METHODS: Through whole-exome sequencing of a paediatric SRNS cohort, we identified three probands carrying biallelic FLNB pathogenic variants. Sanger sequencing was performed for familial cosegregation verification and ACMG classification. Expression of Filamin B, Nephrin and Synaptopodin in renal tissues was assessed by immunohistochemistry/immunofluorescence. Wild-type and patient-derived variant FLNB plasmids were constructed and transfected into HEK293T cells and immortalised human podocytes (HPCs). The effects of these variants on protein expression, localisation and cytoskeletal organisation were assessed by western blotting and immunofluorescence. FLNB expression in HPCs was silenced using shRNA to evaluate the impact on podocyte marker proteins, cytoskeletal integrity and migratory capacity. A zebrafish flnb knockdown model was employed to validate its effects on renal development. RESULTS: All three probands presented with isolated SRNS without skeletal developmental abnormalities, and renal tissues showed significantly reduced Filamin B protein expression. In vitro, p.L117P and p.M1803L variants led to markedly reduced protein expression, while p.R470L and p.K2586R induced perinuclear aggregation of Filamin B accompanied by F-actin rearrangement. FLNB silencing led to downregulation of Nephrin and Synaptopodin, cytoskeletal disorganisation and impaired cell migration. Zebrafish flnb knockdown exhibited pericardial oedema, defective nephron development and abnormal podocyte foot processes. CONCLUSION: We report for the first time that biallelic FLNB pathogenic variants are associated with paediatric SRNS by disrupting Filamin B expression, cytoskeletal integrity and podocyte function, providing evidence that FLNB is a novel monogenic cause of SRNS.

Humans

Postoperative death in paediatric urology.

In the combined experience of two paediatric urologists over a 25 year period only 4 fatalities relating directly to the surgical procedure could be identified. Two other deaths are presented and retrospectively most of the fatalities could have been avoided by present day management but not at the time they occured. Operative mortality in relation to paediatric urology is discussed fully and it is suggested that death related to the immediate surgery should be fully avoidable in relation to paediatric urology. Death from renal failure is a different problem and is unrelated to this paper.

Child, Preschool

Effectiveness of a digi-physical tool and working method for paediatric obesity treatment in Abu Dhabi: a non-inferiority intervention study using an external historical comparator.

BACKGROUND: Effective paediatric obesity treatment requires high intensity, scalable interventions. A digi-physical tool for paediatric obesity treatment has shown positive results in Stockholm, Sweden. This study evaluates whether the same treatment method is effective in a different cultural setting. METHODS: This non-inferiority intervention study, using an external historical comparator, included 60 consecutively recruited children aged 6-15.9 years with obesity who initiated treatment at Sheikh Shakhbout Medical City in Abu Dhabi between June and December 2023. Patients were treated with Evira, a digi-physical tool and working method enabling high intensity individualized care, real-time monitoring, and interactive patient-clinician communication. The primary outcome was BMI z-score change at 26 weeks. Non-inferiority was assessed using a predefined margin of 0.10 BMI z-score, with outcomes compared to a prior published trial in Stockholm (n = 107). RESULTS: A total of 112 children were included in the analysis (Abu Dhabi cohort, n = 35; Stockholm cohort, n = 77). The adjusted mean change in BMI z-score was - 0.20 (95% CI: - 0.28, - 0.12) in the Abu Dhabi cohort and - 0.20 (- 0.26, - 0.14) in the Stockholm cohort (p = 0.88). Non-inferiority was confirmed, (predefined margin 0.10 was not exceeded). A clinically significant BMI z-score reduction (≥ 0.20 units) was achieved by 45.7% of participants in Abu Dhabi and 36.4% in Stockholm (p = 0.35). Non-retention rates at 26 weeks were 41.7% vs. 28.0%, respectively (p = 0.07). CONCLUSIONS: The findings provide promising evidence that treatment outcomes achieved with the digi-physical treatment tool were comparable in the Abu Dhabi and Stockholm cohorts, supporting its feasibility in a second cultural and healthcare setting.

Humans

Speculations on the relevance of developmental psychology to paediatrics.

Many paediatricians are now interested in applying the concepts, theories and methods of developmental psychology to clinical practice. The new understanding so obtained should enable abnormalities of clinical importance to be detected earlier; it should also provide means for evaluating the outcome of some disease processes and medical procedures more fully than was hitherto possible. The genesis of the nurturing failure or deficiency which underlies many paediatric problems becomes explicable as the nature of the maternal bond is unravelled. New ways may thus be opened for the more effective prevention and treatment of child abuse, neglect and deprivation. Developmental psychology also promises to provide for the first time a framework on which child-rearing methods which will positively promote healthy psychological development can be based. However, some caution and critical interpretation, as well as much collaborative investigation, will be needed before the full relevance of developmental psychology to paediatrics is established with certainty.

Child Abuse

Electronic platelet counting with capillary blood samples--experiences in a paediatric clinic.

For several reasons, electronic platelet counts have not been generally adopted in paediatric routine laboratories. A method of platelet counting in capillary blood samples using an electronic counter is described. This is based on brief centrifugation of the blood sample after the addition of a diluent containing heparin/dextran and with a density of 1.050. The thrombocytes are uniformly suspended in the supernatant after 5 min of centrifugation at 250 g, so that the counting procedure in the electric unit can follow immediately. This simplified, dependable and reproducible method is also suitable for venous blood, and as a result of several years of successful trial, it can be recommended for routine use, especially in clinics with paediatric patients.

Blood

A trial of enflurane for paediatric out-patient anaesthesia.

Enflurane was compared with halothane for anaesthesia for short surgical procedures in paediatric out-patients. Induction of anaesthesia was more prolonged with enflurane and recovery times were similar with both agents. Coughing and laryngospasm during induction occurred more frequently with enflurane. The incidence of post-operative complications was essentially similar in both groups, but there was no evidence that the use of enflurane was followed by rapid recovery at home. Enflurane has no advantages over halothane in anaesthesia for short procedures for paediatric out-patients.

Ambulatory Care

The development and usability of 'The Genetics Navigator': a digital solution for adult and paediatric clinical genetics services.

Clinical genetic services address diverse genetic testing needs, but there is no comprehensive digital solution to meet this variety. We aimed to develop and test the usability of the Genetics Navigator (GN), a platform designed to enhance genetic services for paediatric and adult patients. The GN prototype was created with input from a patient and clinician advisory board, informed by prior research. Usability testing involved genetics patients (N = 14), parents of paediatric patients (N = 4), and the general public (N = 10). Participants provided feedback using the 'think aloud' method when using the platform. We used the System Usability Scale (SUS) for quantitative evaluation. Qualitative data were coded by platform section, item, and identified key areas for improvement. Building on the Genetics Adviser platform, we added video and written content for various genetic conditions and patient groups, including pre-test education, counselling, decision support, history collection, post-test result disclosure, and management. Key feedback during rounds of usability testing emphasized the need for a supportive design, seamless workflow, and engaging experience of the tool. The tool was modified to reflect the feedback, and the GN achieved an average SUS score of 87.7 ± 10.9 (N = 28), indicating above-average usability. Future research will evaluate its clinical and cost-effectiveness in a randomized trial.

Humans

Challenges in identifying paediatric cancer predisposition syndromes: international SCOPE survey and SIOPE expert consensus recommendations.

Cancer Predisposition Syndromes (CPS) are heritable genetic conditions associated with an increased risk of developing various cancers throughout life. While early identification and tumour surveillance can improve outcomes, CPS are often underdiagnosed in clinical practice. To evaluate clinicians' perspectives and identify barriers to CPS identification and care across Europe, we conducted the SCOPE study: a three-part cross-sectional survey of paediatric haematology/oncology professionals, followed by a modified Delphi consensus process with members of the SIOP Europe Host Genome Working Group. A total of 185 paediatric oncologists from 22 countries participated in the survey. More than 40% of participants reported low or uncertain confidence across different CPS-related tasks, particularly in counselling families (64.3%) and interpreting germline genetic findings (57.3%). Access to clinical geneticists and dedicated CPS clinics were predictors of higher confidence for some domains, while individual experience and institutional patient volume had limited influence. Regular use of universal CPS screening tools was low (42.3%), with most clinicians relying on personal judgement rather than structured criteria. The most cited barriers were lack of screening guidelines (57%) and difficulties in interpreting results (35.1%). Regular training and workshops, availability of genetic counsellors or educators for patient support, and patient-friendly education material were most cited as areas of improvement. The Delphi process led to three recommendations: (1) improve clinician training and communication strategies, (2) integrate CPS screening into standard treatment plans, and (3) develop accessible, patient-centred educational materials. These recommendations highlight opportunities to enhance CPS care through structured support, interdisciplinary collaboration, and systematic screening approaches.

Humans

A postgraduate course in community paediatrics.

Community paediatrics is assuming increasing importance but is rarely taught as an entity. We have recently organized a course in community paediatrics for general practitioners, doctors working in the area child health services and hospital paediatricians. Features of this day release course are self-instruction at home with specially prepared material, discussion between doctors from different disciplines, discussion with non-medical professionals and the undertaking and presentation of a project. The course is flexible, and once set up can be run without extensive facilities and resources.

Child Health Services

The use of double renal transplants from paediatric cadaver donors.

In 224 consecutive kidney transplantations, 3 patients, aged 44, 37 and 13 years received double renal transplants from paediatric cadaver donors aged 1 day, 23 and 19 months respectively. Both adult recipients developed severe hypertension during postoperative rejection crises leading to multiple graft ruptures in the recipient of the neonatal kidneys. 1 patient died from massive gastrointestinal bleeding 4 weeks after transplantation while kidney function was satisfactory. Excellent graft function was achieved in the 3rd patient. The techniques of removal, preservation and transplantation of double renal transplants are described. The significance of increasing the potential number of donor kidneys by the use of paediatric cadaver donors is stressed.

Adolescent

Chromosome studies at the paediatric necropsy.

The results of chromosome studies from 1193 consecutive paediatric necropsies in Edinburgh and 331 in Adelaide are given. In the Edinburgh series 51 major chromosome abnormalities were detected in 780 (6.5%) necropsies where chromosome studies were successful and in Adelaide 16 in 295 (5.4%) were found. It is suggested that chromosome studies should become an integral part of the paediatric necropsy except for deaths due to primary central nervous system lesions and trauma.

Australia

Paediatric services within the primary health care system in suburban areas.

Results from a few studies on community paediatrics in suburbs of Stockholm are presented. Pattern of diagnoses, utilization of care and social and psychological team work are discussed. Goals of the paediatric service in the primary care are set up and a distribution of duties between different professionals is suggested.

Child

Students' assessment of paediatric teaching and their opinions 7 years later.

A prospective study of the long-term validity of student assessment of paediatric teaching began during the period 1968--70. The students gave scores to the different parts of the course in a standard questionnaire. Between 6 and 8 years later the same students were given the same questionnaire to complete. 76 of the original 92 students responded. By this time most of them were either principals in general practice, or senior registrars or consultants in hospital medicine. The 18 items in the paediatric course were given remarkably similar scores in the two questionnaires, and the popularity ratings of only 2 items had altered significantly during the 7-year interval. The doctor 6--8 years after the course seems more likely to appreciate the need for teaching about communication skills.

Education, Medical, Undergraduate

The training and evaluation of clinical nurse, with special reference to paediatrics and child health.

There is an urgent need to train nurses to assist with the provision of health services for children in the Republic at a clinical level as opposed to their traditional nursing role. Aspects of paediatric training and evaluation of the clinical nurse are discussed. The need for flexibility in training programmes and in their evaluation, without in any way affecting the minimum standards required by the Nursing Council and the South African Medical and Dental Council, is stressed. Steps should be taken to give recognition to the advanced and primary paediatric clinical nurse by defining her function, training and evaluation, designation and status within the health services and the community.

Child Health Services

[Intravenous fluid therapy in paediatric surgery].

From 1956 to 1969 we adopted a generous approach pertaining to the quantity of parenteral fluid required for paediatric surgery. Since 1970 we have changed our policy, and we now give smaller quantities during surgery and postoperatively. This article presents the calculated parenteral fluid regimen as implemented for general paediatric surgery in the Tygerberg Hospital. Our reasons for this change are indicated.

Age Factors

Hot vs cold knife for endoscopic ablation of posterior urethral valves: a systematic review by the EAU-YAU paediatric urology working group.

INTRODUCTION: Posterior urethral valves (PUV) are the most frequent cause of congenital lower urinary tract obstruction in males. Despite early surgical ablation, up to 22% of patients develop chronic kidney disease and 11% progress to end-stage renal disease. Multiple endoscopic modalities have been described for valve ablation but the optimal technique remains uncertain. This systematic review aims to determine whether cold or hot knife ablation provides superior effectiveness for primary endoscopic treatment of PUV in a single surgical session. MATERIAL AND METHODS: A systematic search of PubMed and Embase databases was conducted to identify studies comparing cold and hot knife techniques for endoscopic ablation of PUV in children, covering all publications up to December 2025. The review was performed in accordance with PRISMA 2020 guidelines and was prospectively registered in PROSPERO (ID CRD420251180556). Original studies including patients <18 years who underwent primary valve ablation with postoperative cystoscopy or VCUG and &#x2265;6 months of follow-up were included. Quality assessment was done using RoB 2.0 for randomized trials and MINORS for observational studies. RESULTS: A total of 1581 studies were identified, of which 26 met the inclusion criteria, comprising one randomized controlled trial, five prospective, and 20 retrospective studies constituting a sum of 1725 paediatric patients. The overall methodological quality of included studies was moderate, with marked heterogeneity in design, follow-up duration, and outcome reporting, limiting direct comparisons across series. Thus statistical analysis was not possible. Of these, 829 (48.1%) underwent cold valve ablation and 896 (51.9%) underwent hot ablation techniques. Within the cold group, most patients were treated with a cold knife (80.2%), followed by balloon dilatation (7%), the Mohan valvotome (6.5%), cold hook (5%), and, rarely, a modified venous valvulotome (1.3%). Among hot techniques, 32.8% of procedures were performed by electro-fulguration with a resectoscope, 29.4% using a Bugbee electrode, 23.8% with a hook electrode and 14% with laser-based systems. Follow-up ranged from 6 months to 22 years across studies. Single-session success rates for valve ablation ranged from 22% to 100% in the cold resection group and from 71.4% to 100% in the hot resection group. Reintervention for residual valves was reported in 0%-78% of cold cases and in 0%-28.6% of hot resections. Urethral stricture rates ranged from 0% to 11.1% after cold incision and from 0% to 23.8% after hot techniques. Reporting of postoperative outcomes such as urinary tract infection, incontinence, bladder dysfunction, vesicoureteral reflux (VUR) resolution, hydronephrosis improvement, and renal function varied widely among studies and was assessed using different methodologies. CONCLUSIONS: Both cold and hot ablation techniques for PUV achieved high single-session success rates and low complication rates. Cold resection appeared slightly safer, although this finding should be interpreted cautiously given the heterogeneity and observational nature of the available data.

Humans

Immunobiology of paediatric intracranial tumours. A preliminary report.

Preliminary findings in the evaluation of the immune response of children with primary neoplasms of the CNS, mainly medulloblastomas, are reported and discussed. A broad scheme for the monitoring of B- and T-cell-dependent immunity and of delayed hypersensitivity reactions in this type of patient is presented. The most important immunobiological findings are discussed. Special attention is given to the striking failure of the T-cell-dependent pool (currently identified by "active" RFC and blastigenesis tests) and to the remarkable decrease of hypersensitivity reactions (depressed skin-test response), both of which seem to be related to the degree of malignancy of the tumour. A very peculiar feature, i.e., the appearance of cells with natural cytotoxic activity, is dealt with in some detail. Our present knowledge concerning the immunobiology of primary CNS neoplasms is still very incomplete, but seems to suggest a possible role for immunotherapy in paediatric neurosurgery.

B-Lymphocytes