-
Nefrologia Aug 2021To assess the effects of pharmacological interventions in patients with idiopathic hypercalciuria. (Review)
Review
OBJECTIVE
To assess the effects of pharmacological interventions in patients with idiopathic hypercalciuria.
METHODS
We performed a search of multiple databases, trial registries, grey literature and conference proceedings up to October 2019. We included randomized and quasi-randomized controlled trials that examined any pharmacological intervention for preventing complications of idiopathic hypercalciuria (given for at least four months and six of follow-up). The primary outcomes were stone-free patients, urinary symptoms and severe adverse events.
RESULTS
We included five RCTs (n=446 patients, all adults, 4 in individuals with kidney stones and 1 in postmenopausal women with osteoporosis). Diuretics were likely to increase the number of stone-free patients (RR 1.61, 95% CI 1.33-1.96, moderate quality of evidence (QoE)); 274 more stone-free patients/1000 patients treated (95% CI: 148-432) and produced a slight decrease in the stone formation rate (mean difference -0.18, 95% CI -0.30 to -0.06, low QoE); 180 fewer stones/year/1000 patients treated (95% CI: 300 r to 60). No data on urinary symptoms were reported. The association between diuretic use and severe adverse events was uncertain (RR 5.00, 95% CI 0.60-41.88, very low QoE); 4 more severe adverse events/1000 patients treated (95% CI: 0 fewer to 39 more).
CONCLUSIONS
The addition of diuretics to a normal or modified diet probably reduces the number of stone recurrences and may decrease the stone formation rate. It is uncertain whether diuretics increase the occurrence of severe adverse events. There were no studies investigating other outcomes or in children.
PubMed: 34393000
DOI: 10.1016/j.nefro.2021.04.007 -
Neurosurgical Review Apr 2021Brainstem hemangioblastomas are benign, highly vascular tumors located in the mesencephalon, pons, and medulla oblongata. Although surgical resection is currently... (Meta-Analysis)
Meta-Analysis
Brainstem hemangioblastomas are benign, highly vascular tumors located in the mesencephalon, pons, and medulla oblongata. Although surgical resection is currently considered the main therapeutic option for symptomatic lesions, evidence supporting the application of microsurgery has not been systematically assessed. This meta-analysis aims to evaluate the safety and efficacy of surgical treatment for brainstem hemangioblastomas. A comprehensive search of the PubMed, Embase, and Web of Science databases was performed to identify all English language publications reporting the outcomes of surgical treatment for brainstem hemangioblastomas. Studies from January 1990 to July 2019 with ≥ 10 cases were included. We analyzed the surgical outcomes, including gross total resection, mortality, neurological morbidity, and functional outcome according to the McCormick Scale or Karnofsky Performance Scale. Thirteen studies with 473 cases were included. The pooled proportion of gross total resection was 98% (95% confidence interval (CI), 94-100%). Overall mortality and neurological morbidity were 4 (95% CI, 2-6%) and 13% (95% CI, 7-20%), respectively. Favorable functional outcomes at the last follow-up were achieved in 85% (95% CI, 78-92%) of all patients. Improved or stable functional outcomes at long-term follow-up were achieved in 94% (95% CI, 89-97%) of patients. This meta-analysis revealed that surgical treatment for brainstem hemangioblastomas is technically feasible and effective with lasting patient benefits and cure.
Topics: Adult; Brain Stem Neoplasms; Female; Hemangioblastoma; Humans; Karnofsky Performance Status; Male; Microsurgery; Middle Aged; Neurosurgical Procedures; Observational Studies as Topic; Retrospective Studies; Treatment Outcome
PubMed: 32356022
DOI: 10.1007/s10143-020-01305-3 -
The Cochrane Database of Systematic... Jan 2009Acetylcysteine and carbocysteine are the most commonly prescribed mucolytic drugs in many European countries. To our knowledge, no systematic review has been published... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Acetylcysteine and carbocysteine are the most commonly prescribed mucolytic drugs in many European countries. To our knowledge, no systematic review has been published on their efficacy and safety for acute upper and lower respiratory tract infections (ARTIs) in children without chronic broncho-pulmonary disease.
OBJECTIVES
The objective was to assess the efficacy and safety and to establish a benefit-risk ratio of acetylcysteine and carbocysteine as symptomatic treatments for ARTIs in children without chronic broncho-pulmonary disease.
SEARCH STRATEGY
We searched the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library 2007, issue 4) which contains the Acute Respiratory Infections (ARI) Group's Specialized Register, MEDLINE (1966 to 2008), EMBASE (1980 to 2008); Micromedex (2008), Pascal (1987 to 2004), and Science Citation Index (1974 to 2008).
SELECTION CRITERIA
To study efficacy, we used randomised controlled trials (RCTs) comparing the use of acetylcysteine or carbocysteine versus placebo either alone or as an add-on therapy.To study safety, we also used trials comparing the use of acetylcysteine or carbocysteine versus active treatment or no treatment and case reports.
DATA COLLECTION AND ANALYSIS
At least two review authors extracted data and assessed trial quality. We performed a subgroup analysis of children younger than two years of age.
MAIN RESULTS
Six trials involving 497 participants were included to study efficacy. They showed some benefit from mucolytic agents, although differences were of little clinical relevance. No conclusion was drawn about the subgroup of infants younger than two years because the data were unavailable. Thirty-four studies including the previous six trials involving 2064 children were eligible to study safety. Overall safety was good but very few data were available to evaluate safety in infants younger than two years. However, 48 cases of paradoxically increased bronchorrhoea observed in infants were reported to the French pharmacovigilance system.
AUTHORS' CONCLUSIONS
The results of this review have to be interpreted with caution because it was based on a limited number of participants included in studies whose methodological quality is questionable. Acetylcysteine and carbocysteine seem to have a limited efficacy and appear to be safe in children older than two years. These results should take into consideration the fact that acetylcysteine and carbocysteine are prescribed for self-limiting diseases (for example, acute cough, bronchitis). Regarding children younger than two years, given concerns about safety, these drugs should only be used for ARTIs in the context of an RCT.
Topics: Acetylcysteine; Carbocysteine; Child; Child, Preschool; Expectorants; Humans; Infant; Randomized Controlled Trials as Topic; Respiratory Tract Infections
PubMed: 19160217
DOI: 10.1002/14651858.CD003124.pub3 -
Archives of Physical Medicine and... Jun 2018To report current evidence regarding the safety of intramuscular botulinum toxin injection (BTI) in children with orthopedic- and neurologic-related musculoskeletal...
OBJECTIVE
To report current evidence regarding the safety of intramuscular botulinum toxin injection (BTI) in children with orthopedic- and neurologic-related musculoskeletal disorders >2 years of age.
DATA SOURCES
PubMed, Cochrane Library, and ScienceDirect, Google Scholar, and Web of Science.
STUDY SELECTION
Two reviewers independently selected studies based on predetermined inclusion criteria.
DATA EXTRACTION
Data relating to the aim were extracted. Methodologic quality was graded independently by 2 reviewers using the Physiotherapy Evidence Database scale for randomized controlled trials (RCTs) and the Downs and Black evaluation tool for non-RCTs. Level of evidence was determined using the modified Sackett scale.
DATA SYNTHESIS
Data of 473 infants were analyzed. Fifty-five infants had cerebral palsy, 112 had obstetric brachial plexus palsy, 257 had clubfoot, and 44 had congenital torticollis. No studies reported any severe adverse event that could be attributed to the BTI. The rate of mild to moderate adverse events reported varied from 5% to 25%. Results regarding efficacy were preliminary, dependent on the pathology, and limited by the small number of studies and their low levels of evidence.
CONCLUSIONS
BTI is already widely used as an early treatment for this age group. The safety profile of BTI in infants appears similar to that of older children and risks appear more related to the severity of the pathology and the location of the injections than to the toxin itself. Regarding effectiveness, other studies with higher levels of evidence should be carried out for each specific pathology.
Topics: Botulinum Toxins, Type A; Brachial Plexus Neuropathies; Cerebral Palsy; Clubfoot; Humans; Infant; Injections, Intramuscular; Musculoskeletal Diseases; Neuromuscular Agents; Randomized Controlled Trials as Topic; Torticollis
PubMed: 29288113
DOI: 10.1016/j.apmr.2017.11.013 -
Cerebellum (London, England) Apr 2024The term Pontocerebellar Hypoplasia (PCH) was initially used to designate a heterogeneous group of fetal-onset genetic neurodegenerative disorders. As a descriptive...
The term Pontocerebellar Hypoplasia (PCH) was initially used to designate a heterogeneous group of fetal-onset genetic neurodegenerative disorders. As a descriptive term, PCH refers to pons and cerebellum of reduced volume. In addition to the classic PCH types described in OMIM, many other disorders can result in a similar imaging appearance. This study aims to review imaging, clinical and genetic features and underlying etiologies of a cohort of children with PCH on imaging. We systematically reviewed brain images and clinical charts of 38 patients with radiologic evidence of PCH. Our cohort included 21 males and 17 females, with ages ranging between 8 days to 15 years. All individuals had pons and cerebellar vermis hypoplasia, and 63% had cerebellar hemisphere hypoplasia. Supratentorial anomalies were found in 71%. An underlying etiology was identified in 68% and included chromosomal (21%), monogenic (34%) and acquired (13%) causes. Only one patient had pathogenic variants in an OMIM listed PCH gene. Outcomes were poor regardless of etiology, though no one had regression. Approximately one third of patients deceased at a median age of 8 months. All individuals had global developmental delay, 50% were non-verbal, 64% were non-ambulatory and 45% required gastrostomy feeding. This cohort demonstrates that radiologic PCH has heterogenous etiologies and the "classic" OMIM-listed PCH genes underlie only a minority of cases. Broad genetic testing, including chromosomal microarray and exome or multigene panels, is recommended in individuals with PCH-like imaging appearance. Our results strongly suggest that the term PCH should be used to designate radiologic findings, and not to imply neurogenerative disorders.
Topics: Male; Child; Female; Humans; Infant; Cerebellar Diseases; Cerebellum; Pons; Magnetic Resonance Imaging; Developmental Disabilities; Nervous System Malformations
PubMed: 36971923
DOI: 10.1007/s12311-023-01544-2 -
Journal of Neuromuscular Diseases 2023Recent pharmaceutical breakthroughs in neuromuscular diseases may considerably change the prognosis and natural history these diseases. The ability to measure clinically...
BACKGROUND
Recent pharmaceutical breakthroughs in neuromuscular diseases may considerably change the prognosis and natural history these diseases. The ability to measure clinically relevant outcomes such as motor function is critical for the assessment of therapeutics and the follow up of individuals. The Motor Function Measure (MFM) is a quantitative scale designed to measure motor function in adult and children with neuromuscular disease (NMD).
OBJECTIVE
The objective of this study is to assess the quality and level of evidence of the MFM's published measurement properties by completing a systematic review of the validation and responsiveness studies of the MFM20 (a 20-item version of MFM adapted for children 2 to 6 years of age) and the MFM32 (the original 32 item version), in all NMDs and in specific diseases.
METHODS
A search for MFM responsiveness and MFM validation studies was completed in February 2023 in EMBASE, MEDLINE, SCOPUS and Web of Science databases. The PRISMA guidelines and the COSMIN manual for systematic reviews were followed for databases searches, articles screening and selection, study quality and measurement properties evaluation.
RESULTS
49 studies were included in analysis. In studies including individuals with all NMDs, MFM's internal consistency, reliability, convergent validity, construct validity and responsiveness were rated as sufficient with a high quality of evidence. Structural validity was rated sufficient with a moderate quality of evidence In SMA in particular, MFM's reliability, internal consistency, convergent validity, discriminant validity and responsiveness are sufficient with a high quality of evidence. More studies would be required to assess specific measurement properties in different diseases. MFM32's minimal clinically relevant difference has been defined between 2 and 6%.
CONCLUSION
MFM's structural validity, internal consistency, reliability, construct validity, convergent validity and responsiveness have been verified with moderate to high level of evidence.
Topics: Adult; Child; Humans; Psychometrics; Reproducibility of Results; Neuromuscular Diseases
PubMed: 37125561
DOI: 10.3233/JND-230001 -
AJNR. American Journal of Neuroradiology Aug 2016Polyglutamine expansion spinocerebellar ataxias are autosomal dominant slowly progressive neurodegenerative diseases with no current treatment. MR imaging is the...
BACKGROUND AND PURPOSE
Polyglutamine expansion spinocerebellar ataxias are autosomal dominant slowly progressive neurodegenerative diseases with no current treatment. MR imaging is the best-studied surrogate biomarker candidate for polyglutamine expansion spinocerebellar ataxias, though with conflicting results. We aimed to review quantitative central nervous system MR imaging technique findings in patients with polyglutamine expansion spinocerebellar ataxias and correlations with well-established clinical and molecular disease markers.
MATERIALS AND METHODS
We searched MEDLINE, LILACS, and Cochrane data bases of clinical trials between January 1995 and January 2016, for quantitative MR imaging volumetric approaches, MR spectroscopy, diffusion tensor imaging, or other quantitative techniques, comparing patients with polyglutamine expansion spinocerebellar ataxias (SCAs) with controls. Pertinent details for each study regarding participants, imaging methods, and results were extracted.
RESULTS
After reviewing the 706 results, 18 studies were suitable for inclusion: 2 studies in SCA1, 1 in SCA2, 15 in SCA3, 1 in SCA7, 1 in SCA1 and SCA6 presymptomatic carriers, and none in SCA17 and dentatorubropallidoluysian atrophy. Cerebellar hemispheres and vermis, whole brain stem, midbrain, pons, medulla oblongata, cervical spine, striatum, and thalamus presented significant atrophy in SCA3. The caudate, putamen and whole brain stem presented similar sensitivity to change compared with ataxia scales after 2 years of follow-up in a single prospective study in SCA3. MR spectroscopy and DTI showed abnormalities only in cross-sectional studies in SCA3. Results from single studies in other polyglutamine expansion spinocerebellar ataxias should be replicated in different cohorts.
CONCLUSIONS
Additional cross-sectional and prospective volumetric analysis, MR spectroscopy, and DTI studies are necessary in polyglutamine expansion spinocerebellar ataxias. The properties of preclinical disease biomarkers (presymptomatic) of MR imaging should be targeted in future studies.
Topics: Adult; Female; Humans; Male; Neuroimaging; Spinocerebellar Ataxias
PubMed: 27173364
DOI: 10.3174/ajnr.A4760 -
Revue de Laryngologie - Otologie -... 2013Hyposmia is a common cause of functional complaint in patients with nasal polyposis. The aim of the current study was to report the olfactory functional results after... (Review)
Review
INTRODUCTION
Hyposmia is a common cause of functional complaint in patients with nasal polyposis. The aim of the current study was to report the olfactory functional results after sinus surgery.
MATERIALS AND METHODS
A systematic review of the scientific literature was achieved in the Pubmed database.
RESULTS
Overall, 10 series published between 1989 and 2013, involving 959 patients, were selected. The surgery for nasal polyposis, adjuvant medical treatment, may allow olfactory improvement. The results are even better than surgery is as wide as possible and the evolutionary stage is low.
Topics: Humans; Nasal Polyps; Olfaction Disorders; Otorhinolaryngologic Surgical Procedures; Smell; Treatment Outcome
PubMed: 24974407
DOI: No ID Found -
European Radiology Jun 2022
Do you dare with a health-economy methodology issue? An editorial comment on "A systematic review of cost-effectiveness analyses on endovascular thrombectomy in ischemic stroke patients".
Topics: Brain Ischemia; Cost-Benefit Analysis; Endovascular Procedures; Humans; Ischemic Stroke; Stroke; Thrombectomy; Treatment Outcome
PubMed: 35357539
DOI: 10.1007/s00330-022-08734-2 -
Clinical & Translational Oncology :... Aug 2022Daily, moderate hypofractionation has become standard treatment for breast cancer following breast-conserving surgery, although substantial variation exists in its use....
INTRODUCTION
Daily, moderate hypofractionation has become standard treatment for breast cancer following breast-conserving surgery, although substantial variation exists in its use. This paper describes the generation of consensus-based recommendations for the utilisation of this therapy at the healthcare system level and compares these to American Society for Radiation Oncology (ASTRO) guidelines.
MATERIALS AND METHODS
Consensus-based guidelines were developed in three steps, including a systematic literature review and involvement of radiation oncologists specialising in breast cancer in Catalonia: (a) creation of a working group and evidence review; (b) consideration of the levels of evidence and agreement on the formulation of survey questions; and (c) performance of survey and development of consensus-based recommendations. Results were compared to the ASTRO recommendations.
RESULTS
Consensus was above 80% for 10 of the 14 survey items. Experts supported hypofractionated radiotherapy for all breast cancer patients aged 40 years or more; with invasive carcinoma and breast-conserving surgery; without radiation of lymph nodes; and regardless of the tumour size, histological grade, molecular subtype, breast size, laterality, other treatment characteristics, or need for a boost. Over half favoured its use in all situations, even where available scientific evidence is insufficient. The resulting recommendations and the quality of the evidence are comparable to those from ASTRO, despite some differences in the degree of consensus.
CONCLUSION
Specialists agree that hypofractionation is the standard treatment for breast cancer following breast-conserving surgery, but some specific areas require a higher level of evidence before unequivocally extending indications.
Topics: Breast Neoplasms; Carcinoma; Female; Humans; Mastectomy, Segmental; Radiation Dose Hypofractionation; Radiation Oncology; Radiotherapy, Adjuvant
PubMed: 35190961
DOI: 10.1007/s12094-022-02798-8