Background: Respiratory failure is the leading cause of mortality in spinal muscular atrophy type 1 (SMA1) children. The current study aims to evaluate the effect of nusinersen treatment on respiratory outcome of the patients with SMA1.
Methods: In this retrospective, single-center study, 52 SMA1 patients treated with nusinersen were included in the analysis. Patients were divided into two groups based on their age at the time of their first nusinersen treatment (Group 1: ≤6 months, Group 2: >6 months). Respiratory outcome on the 180th day of treatment is defined as the type of ventilation support (spontaneous breathing, noninvasive ventilation (NIV), and tracheostomized or intubated on invasive mechanical ventilation). Demographic data, respiratory outcome, and Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores were obtained from medical records.
Results: On the 180th day of treatment, 46 of the 52 (88.4%) children were alive. Prevalence of the mortality was similar in both groups (P = 0.65). The comparison of respiratory outcome in patients between group 1 and group 2 was as follows: spontaneous breathing, 7 (43.7%) versus 4 (13.3%) (P = 0.03); NIV <16 h/day, 3 (18.7%) versus 4 (13.3%) (P = 0.68); invasive mechanical ventilation, 6 (37.5%) versus 22 (73.3%) (P = 0.01). There were no patients using NIV ≥16 h/day. There were significant improvements in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores of the patients at day 180 in comparison with the baseline (P < 0.001).
Conclusions: Early initiation of nusinersen treatment in SMA1 patients may alter the disease's natural course.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1111/ped.15175 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Effects of two different dexamethasone dosing regimens on ventilator-free days and long-term mortality in COVID-19 patients with moderate-to-severe ARDS: the REMED randomized clinical trial.
Eur J Med Res
December 2024
Bloomsbury Institute of Intensive Care Medicine, Division of Medicine, University College London, London, UK.
Background: Dexamethasone 6 mg in patients with severe COVID-19 has been shown to decrease mortality and morbidity. The effects of higher doses of corticosteroid, that would further increase anti-inflammatory effects, are uncertain. The objective of our study was to assess the effect of 20 mg dexamethasone vs.
View Article and Find Full Text PDFECMO for Adult Respiratory Failure: A Rapid Review of Clinical and Service Delivery Evidence to Guide Policy in Wales.
Semin Cardiothorac Vasc Anesth
December 2024
NHS Wales Joint Commissioning Committee, Pontypridd, UK.
Background: While several studies have summarised the clinical effectiveness evidence for extracorporeal membrane oxygenation (ECMO), there are no evidence syntheses of the impact of centres' ECMO patient volume on patient outcomes or the impact of bedside ECMO care being delivered by either a perfusionist or a nurse. There is also limited information on the cost-effectiveness of ECMO.
Purpose: This review was carried out to evaluate the clinical effectiveness and cost of different service delivery models of pulmonary ECMO to inform NHS Wales commissioning policy.
Background: Optimizing outcomes of hospitalized patients anchors on standardizing processes in medical management, interventions to reduce the risk of decompensation, and prompt intervention when a patient decompensates.
Methods: A quality improvement initiative (optimized sepsis and respiratory compromise management, reducing health care-associated infection and medication risk, swift management of the deteriorating patient, feedback on performance, and accountability) was implemented in a multistate health system. The primary outcome was risk-adjusted in-hospital mortality.
[A preliminary study on the short-term effectiveness and safety of sublingual immunotherapy-spray for patients with respiratory allergy].
Zhonghua Yu Fang Yi Xue Za Zhi
December 2024
Department of Otorhinolaryngology & Clinical Allergy Center, The First Affiliated Hospital, Nanjing Medical University, Nanjing210029, China International Centre for Allergy Research, Nanjing Medical University, Nanjing210029, China.
To investigate the short-term effectiveness and safety of sublingual allergen immunotherapy with allergen sprays (SLIT-sprays) in Chinese patients with allergic rhinitis (AR) with or without asthma using real-world data. The retrospective cohort study included 100 patients who received SLIT-sprays in the ENT departments in Hainan Shulan (Boao) Hospital and Boao Super Hospital between October 2023 and August 2024. A questionnaire survey was conducted to collect clinical data on the effectiveness and safety of SLIT-sprays, examining the types and incidence of adverse events (AEs) during treatment, treatments after the occurrence of AEs, and changes in Visual Analog Scale (VAS) scores before and after SLIT-sprays.
View Article and Find Full Text PDFState-of-the-Art Narrative Review: Mounier-Kuhn Syndrome and Tracheobronchomegaly.
Respir Med
December 2024
New York City Health & Hospitals, Woodhull; NYU Grossman School of Medicine, Division of Pulmonary Medicine, New York City Health and Hospitals, Woodhull, 760 Broadway, 8(th) Floor, Brooklyn, NY 11206, USA. Electronic address:
Mounier-Kuhn syndrome (MKS) or tracheobronchomegaly is an uncommon disease of the central airways. It is characterized by pathological dilatation of the trachea and main bronchi and inevitably leads to recurrent respiratory infections, bronchiectasis, hospitalizations, and results in considerable morbidity and mortality. Despite numerous case reports, there is a shortage of evidence on clinical outcomes and limited data on interventions, thus presenting a significant gap in the literature.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!