Browsing by Issue Date, starting with "2024-11"
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- Targeted Treatments for Myasthenia Gravis in Children and Adolescents.Publication . Ramdas, Sithara; Painho, Teresa; Vanegas, Maria I; Famili, Dennis T; Lim, Ming J; Jungbluth, HeinzMyasthenia gravis (MG) is an antibody-mediated disorder of the neuromuscular junction affecting children and adults. MG is a treatable condition with most patients requiring immunosuppression for disease control and/or remission. Juvenile myasthenia gravis (JMG) is rare in comparison with adult-onset MG but given the same underlying pathophysiology, treatment strategies are similar to those in adults. Until recently, there were only a few randomised controlled trials (RCTs) for MG treatments in adults and none in children, and management strategies were primarily based on expert consensus. In addition, treatment options for refractory MG cases have been severely limited, resulting in poor long-term quality of life in such patients due to the significant disease burden. Recently, there have been several RCTs focussing on novel therapeutic strategies with potentially promising outcomes, suggesting a change in MG management over the coming years and access to more effective and faster-acting drugs for MG patients. This paper will review current and new MG treatments including efgartigimod, eculizumab, rozanolixizumab, ravulizumab, and zilucoplan, with a focus on juvenile myasthenia gravis.
- The Effectiveness of Various CSF Diversion Surgeries in Idiopathic Normal Pressure Hydrocephalus: a Systematic Review and Meta-Analysis.Publication . Salih, Ahmed; Arif, Aksaan; Varadpande, Madhur; Fernandes, Rafael Tiza; Jankovic, Dragan; Kalasauskas, Darius; Ottenhausen, Malte; Kramer, Andreas; Ringel, Florian; Thavarajasingam, Santhosh GBackground: Idiopathic normal pressure hydrocephalus (iNPH) is commonly treated using cerebrospinal fluid (CSF) diversion procedures, most commonly ventriculoperitoneal (VP) but also lumboperitoneal (LP), ventriculoatrial (VA) shunting, and endoscopic third-ventriculostomy (ETV). Despite the prevalence of these interventions and recent advancements in iNPH diagnostic processes, there is limited up-to-date evidence regarding surgical outcomes. Methods: A systematic review and meta-analysis were conducted to analyse the effects of CSF diversion surgeries among iNPH patients. The primary outcome was efficacy of the CSF diversion procedure, defined as symptomatic improvement, and secondary outcomes included surgical complications. Several major databases were searched for original studies from inception up to June 4, 2024, which were evaluated using random-effects meta-analyses, meta-regression, and influence analyses. This study was registered with PROSPERO: CRD42023458526. Findings: Out of the 1963 studies screened, 54 were included in this review, and 4811 patients were pooled. Overall, more than 74% of patients experienced improvement after surgical treatment (95% CI: 70-78%). VP shunting demonstrated an efficacy of 75% (95% CI 70-79%), VA shunting at 75% (95% CI: 70-80%), and LP shunting at 70% (95% CI: 52-83%). ETV had a success rate of 69% (95% CI: 58-78%). Gait improvement was high at 72% (95% CI: 67-77%), while urinary and cognitive dysfunction each improved in approximately 50% of patients. The efficacy of surgery did not increase between 2005 and 2024 (p = 0.54). Complications occurred in 20.6% of cases, with a surgery revision rate of 15.1%. Interpretation: This meta-analysis found that the overall efficacy of CSF diversion procedures for iNPH remained unchanged from 2005 to 2024, with 74% of cases showing improvement. No procedure was found to be clearly superior, and only half of the patients saw improvements in urinary and cognitive dysfunction. The stagnant efficacy over time and frequent complications highlight the need for improved patient selection criteria to best identify those most likely to benefit from CSF shunting. Funding: None for this study.
- Proximal Humerus Epiphysiolysis as a Rare Cause of Fracture in Childbirth - A Case Report.Publication . Teles, Bárbara Noronha; Castro, João Carlos; Ovídeo, JoanaA epifisiólise proximal do úmero (EPU) é rara, presente em10,1/100.000 nascimentos e há poucos casos descritos na literatura. Apresentamos o caso de um recém-nascido com diagnóstico de EPU submetido a tratamento conservador. Em seis semanas apresentava mobilidade completa e extenso calo ósseo. Sendo uma situação muito rara, é imperativo um diagnóstico rápido, para o qual a ecografia é decisiva e a abordagem deve ser conservadora e expectante, dada uma evolução muito rápida e esperada no sentido da consolidação para a função normal. Este caso reforça o conhecimento prévio de que essas lesões normalmente evoluem favoravelmente e não são esperadas sequelas pós-traumáticas
- Severe Neonatal Hyperbilirubinaemia in European and Indian Subcontinent Descendent Newborns: a Retrospective Cohort StudyPublication . Simões, João Ferreira; Simão, Margarida; Rocha, Paula; Ferreira, Sara; da Câmara, Rosário Perry; Amaral, Diana; Costa, Beatriz; Coelho, MárioNeonatal hyperbilirubinaemia is more common in Asian-descendent populations, but differences in disease severity are poorly reported. Our study aimed to compare neonatal hyperbilirubinaemia severity between European and Indian subcontinent descendent newborns. We conducted a single-centre retrospective cohort study including newborns admitted with unconjugated hyperbilirubinaemia (January 2016 to December 2021). Patients were followed during admission, comparing those with European ancestry (control group) and Indian subcontinent ancestry (India, Pakistan, Bangladesh and Nepal) (study group). The primary outcome was severe hyperbilirubinemia (TSB > 25 mg/dL, phototherapy > 6 h or need for exchange transfusion [ET]), and the secondary was TSB levels. Adjusted analysis for potential confounding factors was performed using binary logistic regression models. Of 110 newborns included, 27 (24.5%) had Indian subcontinent ancestry. Occurrence of TSB > 25 mg/dL was significantly higher in the study group (22.2% vs. 4.8%, p = 0.006), while no differences were noted in exposure to phototherapy > 6 h and ET therapy. Logistic regression models for confounding factors adjustment showed Indian subcontinent ancestry as an independent risk factor for TSB > 25 mg/dL (OR 7.49, CI 95% [1.23-45.50]). The study group revealed also higher absolute values of TSB both at admission (22.0 mg/dL vs. 19.6 mg/dL, p = 0.013) and at discharge (13.6 mg/dL vs. 11.4 mg/dL, p = 0.005). Conclusion: Our findings suggest that newborns with Indian subcontinent ancestry might show a higher risk for the development of severe hyperbilirubinemia compared to European ancestry newborns. Implementing earlier treatment thresholds in this subset of patients may help prevent severe hyperbilirubinemia. What is Known: • Indian subcontinent descendent populations have high incidence of neonatal hyperbilirubinaemia but data regarding its severity are scarce. What is New: • This article shows that, compared to European descendent newborns, Indian subcontinent descendent newborns might be at higher risk for severe hyperbilirubinaemia.