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Journal of Sport and Health Science May 2022Soccer match-play is typically contested over 90 min; however, in some cup and tournament scenarios, when matches are tied, they proceed to an additional 30 min, which... (Review)
Review
OBJECTIVE
Soccer match-play is typically contested over 90 min; however, in some cup and tournament scenarios, when matches are tied, they proceed to an additional 30 min, which is termed "extra-time" (ET). This systematic review sought to appraise the literature available on 120-min of soccer-specific exercise, with a view to identifying practical recommendations and future research opportunities.
METHODS
The review was conducted according to the PRISMA guidelines. Independent researchers performed a systematic search of PubMed, CINAHL, and PsycINFO in May 2019, with the following keywords entered in various combinations: "soccer", "football", "extra-time", "extra time", "extratime", "120 minutes", "120 min", "additional 30 minutes", and "additional 30 min".
RESULTS
The search yielded an initial 73 articles. Following the screening process, 11 articles were accepted for analyses. Articles were subsequently organized into the following 5 categories: movement demands of ET, performance responses to ET, physiological and neuromuscular response during ET, nutritional interventions, and recovery and ET. The results highlighted that during competitive match-play, players cover 5%-12% less distance relative to match duration (i.e., meters per minute) during ET compared to the preceding 90 min. Reductions in technical performance (i.e., shot speed, number of passes and dribbles) were also observed during ET. Additionally, carbohydrate provision may attenuate and improve dribbling performance during ET. Moreover, objective and subjective measures of recovery may be further compromised following ET when compared to 90 min.
CONCLUSION
Additional investigations are warranted to further substantiate these findings and identify interventions to improve performance during ET.
Topics: Athletic Performance; Movement; Soccer; Humans
PubMed: 32445903
DOI: 10.1016/j.jshs.2020.03.008 -
Sleep Feb 2015The relationship between nasal surgery and its effect on continuous positive airway pressure (CPAP) device therapeutic treatment pressures and CPAP device use has not... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
The relationship between nasal surgery and its effect on continuous positive airway pressure (CPAP) device therapeutic treatment pressures and CPAP device use has not been previously systematically examined.
STUDY OBJECTIVES
To conduct a systematic review and meta-analysis evaluating the effect of isolated nasal surgery on therapeutic CPAP device pressures and use in adults with obstructive sleep apnea (OSA).
METHODS
MEDLINE, Scopus, Web of Science, and The Cochrane Library were searched through July 15, 2014. The MOOSE consensus statement and PRISMA statement were followed.
RESULTS
Eighteen studies (279 patients) reported CPAP data after isolated nasal surgery. Seven studies (82 patients) reported preoperative and postoperative mean therapeutic CPAP device pressures and standard deviations (SD), which reduced from 11.6 ± 2.2 to 9.5 ± 2.0 centimeters of water pressure (cwp) after nasal surgery. Pooled random effects analysis demonstrated a statistically significant pressure reduction, with a mean difference (MD) of -2.66 cwp (95% confidence interval (CI), -3.65 to -1.67); P < 0.00001. Eleven studies (153 patients) reported subjective, self-reported data for CPAP use; and a subgroup analysis demonstrated that 89.1% (57 of 64 patients) who were not using CPAP prior to nasal surgery subsequently accepted, adhered to, or tolerated it after nasal surgery. Objective, device meter-based hours of use increased in 33 patients from 3.0 ± 3.1 to 5.5 ± 2.0 h in the short term (<6 mo of follow-up).
CONCLUSION
Isolated nasal surgery in patients with OSA and nasal obstruction reduces therapeutic CPAP device pressures and the currently published literature's objective and subjective data consistently suggest that it also increases CPAP use in select patients.
Topics: Continuous Positive Airway Pressure; Humans; Nasal Surgical Procedures; Postoperative Period; Preoperative Period; Pressure; Sleep Apnea, Obstructive; Treatment Outcome
PubMed: 25325439
DOI: 10.5665/sleep.4414 -
JAMA Network Open Dec 2020Controversy remains regarding the transmission routes of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
IMPORTANCE
Controversy remains regarding the transmission routes of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
OBJECTIVE
To review current evidence on air contamination with SARS-CoV-2 in hospital settings and the factors associated with contamination, including viral load and particle size.
EVIDENCE REVIEW
The MEDLINE, Embase, and Web of Science databases were systematically queried for original English-language articles detailing SARS-CoV-2 air contamination in hospital settings between January 1 and October 27, 2020. This study was conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR) guidelines. The positivity rate of SARS-CoV-2 viral RNA and culture were described and compared according to the setting, clinical context, air ventilation system, and distance from patients. The SARS-CoV-2 RNA concentrations in copies per meter cubed of air were pooled, and their distribution was described by hospital areas. Particle sizes and SARS-CoV-2 RNA concentrations in copies or median tissue culture infectious dose (TCID50) per meter cubed were analyzed after categorization as less than 1 μm, from 1 to 4 μm, and greater than 4 μm.
FINDINGS
Among 2284 records identified, 24 cross-sectional observational studies were included in the review. Overall, 82 of 471 air samples (17.4%) from close patient environments were positive for SARS-CoV-2 RNA, with a significantly higher positivity rate in intensive care unit settings (intensive care unit, 27 of 107 [25.2%] vs non-intensive care unit, 39 of 364 [10.7%]; P < .001). There was no difference according to the distance from patients (≤1 m, 3 of 118 [2.5%] vs >1-5 m, 13 of 236 [5.5%]; P = .22). The positivity rate was 5 of 21 air samples (23.8%) in toilets, 20 of 242 (8.3%) in clinical areas, 15 of 122 (12.3%) in staff areas, and 14 of 42 (33.3%) in public areas. A total of 81 viral cultures were performed across 5 studies, and 7 (8.6%) from 2 studies were positive, all from close patient environments. The median (interquartile range) SARS-CoV-2 RNA concentrations varied from 1.0 × 103 copies/m3 (0.4 × 103 to 3.1 × 103 copies/m3) in clinical areas to 9.7 × 103 copies/m3 (5.1 × 103 to 14.3 × 103 copies/m3) in the air of toilets or bathrooms. Protective equipment removal and patient rooms had high concentrations per titer of SARS-CoV-2 (varying from 0.9 × 103 to 40 × 103 copies/m3 and 3.8 × 103 to 7.2 × 103 TCID50/m3), with aerosol size distributions that showed peaks in the region of particle size less than 1 μm; staff offices had peaks in the region of particle size greater than 4 μm.
CONCLUSIONS AND RELEVANCE
In this systematic review, the air close to and distant from patients with coronavirus disease 2019 was frequently contaminated with SARS-CoV-2 RNA; however, few of these samples contained viable viruses. High viral loads found in toilets and bathrooms, staff areas, and public hallways suggest that these areas should be carefully considered.
Topics: Air Microbiology; COVID-19; Hospitals; Humans; Microbial Viability; Particle Size; RNA, Viral; SARS-CoV-2
PubMed: 33355679
DOI: 10.1001/jamanetworkopen.2020.33232 -
American Journal of Physical Medicine &... Mar 2016The purpose of this systematic review was to examine studies that examined the effectiveness of aerobic exercise interventions on mobility in long-term stroke survivors. (Review)
Review
OBJECTIVE
The purpose of this systematic review was to examine studies that examined the effectiveness of aerobic exercise interventions on mobility in long-term stroke survivors.
DESIGN
The authors searched electronic databases for randomized control trials between January 1995 and December 2014 investigating aerobic exercise interventions and mobility in stroke survivors after the subacute phase (>6 mos). Mobility was measured using objective functional fitness tests: 6-minute walk, 10-meter walk, and up-n-go.
RESULTS
Nine randomized control trials that compared aerobic exercise with a control group among stroke survivors (mean age, 56.95-68 yrs) were identified. Aerobic interventions lasted between 2 and 6 mos and primarily involved walking. Using the Comprehensive Meta-analysis software, it was found that two of the three mobility outcomes showed small to moderate effect sizes favoring the aerobic exercise group: 6-minute walk (g = 0.366, P < 0.001) and 10-meter walk (g = 0.411, P = 0.002), while the up-n-go test was not significant (g = -0.150, P = 0.330).
CONCLUSION
These findings demonstrate that stroke survivors may continue to benefit from aerobic exercise after the subacute phase. Future research needs to examine the precise dose and recommendation for aerobic exercise, test other exercise modalities, and use larger samples to thoroughly determine long-term exercise effects on mobility in this population.
Topics: Exercise; Humans; Stroke; Stroke Rehabilitation; Walking
PubMed: 26544857
DOI: 10.1097/PHM.0000000000000416 -
Environment International Sep 2021The World Health Organization (WHO) and the International Labour Organization (ILO) are developing joint estimates of the work-related burden of disease and injury... (Meta-Analysis)
Meta-Analysis
The effect of occupational exposure to noise on ischaemic heart disease, stroke and hypertension: A systematic review and meta-analysis from the WHO/ILO Joint Estimates of the Work-Related Burden of Disease and Injury.
BACKGROUND
The World Health Organization (WHO) and the International Labour Organization (ILO) are developing joint estimates of the work-related burden of disease and injury (WHO/ILO Joint Estimates), with contributions from a large number of individual experts. Evidence from mechanistic data suggests that occupational exposure to noise may cause cardiovascular disease (CVD). In this paper, we present a systematic review and meta-analysis of parameters for estimating the number of deaths and disability-adjusted life years from CVD that are attributable to occupational exposure to noise, for the development of the WHO/ILO Joint Estimates.
OBJECTIVES
We aimed to systematically review and meta-analyse estimates of the effect of any (high) occupational exposure to noise (≥85 dBA), compared with no (low) occupational exposure to noise (<85 dBA), on the prevalence, incidence and mortality of ischaemic heart disease (IHD), stroke, and hypertension.
DATA SOURCES
A protocol was developed and published, applying the Navigation Guide as an organizing systematic review framework where feasible. We searched electronic academic databases for potentially relevant records from published and unpublished studies up to 1 April 2019, including International Trials Register, Ovid MEDLINE, PubMed, Embase, Lilacs, Scopus, Web of Science, and CISDOC. The MEDLINE and Pubmed searches were updated on 31 January 2020. We also searched grey literature databases, Internet search engines and organizational websites; hand-searched reference lists of previous systematic reviews and included study records; and consulted additional experts.
STUDY ELIGIBILITY AND CRITERIA
We included working-age (≥15 years) workers in the formal and informal economy in any WHO and/or ILO Member State but excluded children (<15 years) and unpaid domestic workers. We included randomized controlled trials, cohort studies, case-control studies and other non-randomized intervention studies with an estimate of the effect of any occupational exposure to noise on CVD prevalence, incidence or mortality, compared with the theoretical minimum risk exposure level (<85 dBA).
STUDY APPRAISAL AND SYNTHESIS METHODS
At least two review authors independently screened titles and abstracts against the eligibility criteria at a first stage and full texts of potentially eligible records at a second stage, followed by extraction of data from qualifying studies. We prioritized evidence from cohort studies and combined relative risk estimates using random-effect meta-analysis. To assess the robustness of findings, we conducted sensitivity analyses (leave-one-out meta-analysis and used as alternative fixed effects and inverse-variance heterogeneity estimators). At least two review authors assessed the risk of bias, quality of evidence and strength of evidence, using Navigation Guide tools and approaches adapted to this project.
RESULTS
Seventeen studies (11 cohort studies, six case-control studies) met the inclusion criteria, comprising a total of 534,688 participants (39,947 or 7.47% females) in 11 countries in three WHO regions (the Americas, Europe, and the Western Pacific). The exposure was generally assessed with dosimetry, sound level meter and/or official or company records. The outcome was most commonly assessed using health records. We are very uncertain (low quality of evidence) about the effect of occupational exposure to noise (≥85 dBA), compared with no occupational exposure to noise (<85 dBA), on: having IHD (0 studies); acquiring IHD (relative risk (RR) 1.29, 95% confidence interval (95% CI) 1.15 to 1.43, two studies, 11,758 participants, I 0%); dying from IHD (RR 1.03, 95% CI 0.93-1.14, four studies, 198,926 participants, I 26%); having stroke (0 studies); acquiring stroke (RR 1.11, 95% CI 0.82-1.65, two studies, 170,000 participants, I 0%); dying from stroke (RR 1.02, 95% CI 0.93-1.12, three studies, 195,539 participants, I 0%); having hypertension (0 studies); acquiring hypertension (RR 1.07, 95% CI 0.90-1.28, three studies, four estimates, 147,820 participants, I 52%); and dying from hypertension (0 studies). Data for subgroup analyses were missing. Sensitivity analyses supported the main analyses.
CONCLUSIONS
For acquiring IHD, we judged the existing body of evidence from human data to provide "limited evidence of harmfulness"; a positive relationship is observed between exposure and outcome where chance, bias, and confounding cannot be ruled out with reasonable confidence. For all other included outcomes, the bodies of evidence were judged as "inadequate evidence of harmfulness". Producing estimates for the burden of CVD attributable to occupational exposure to noise appears to not be evidence-based at this time.
PROTOCOL IDENTIFIER
10.1016/j.envint.2018.09.040.
PROSPERO REGISTRATION NUMBER
CRD42018092272.
Topics: Adolescent; Cost of Illness; Europe; Female; Humans; Hypertension; Male; Myocardial Ischemia; Noise, Occupational; Occupational Diseases; Occupational Exposure; Stroke; World Health Organization
PubMed: 33612311
DOI: 10.1016/j.envint.2021.106387 -
A Systematic Review of the Methods of Assessment of Gastro-Oesophageal Reflux in Anaesthetized Dogs.Animals : An Open Access Journal From... Mar 2021We reviewed the definitions and methods of assessment of gastro-oesophageal reflux (GOR) in anaesthetized dogs. Three databases were used. Titles and abstracts were... (Review)
Review
We reviewed the definitions and methods of assessment of gastro-oesophageal reflux (GOR) in anaesthetized dogs. Three databases were used. Titles and abstracts were screened by two of the authors independently. A total of 22 studies was included in the analysis. The definition of GOR implied the presence of fluids not reaching the mouth or nose in the oesophagus in all studies. Most studies considered a change in pH using oesophageal pH meters as the sole method of assessment. Calibration of the pH probe was inconsistently reported. The position of the tip of the oesophageal probe was inconsistent and not always precisely described. The correct positioning in the intended location was verified in a limited number of studies. Some studies considered that GOR had happened for changes in pH below 4.0 or above 7.5 while others considered that GOR had happened when the pH dropped below 4.0 only. Some studies stated that the pH change had to be sustained for a minimum period of time (20 or 30 s) whereas others did not mention any duration. The variability of definitions and methods of assessment of GOR in anaesthetized dogs precludes meaningful comparison of the findings. Re-evaluation and uniformization of the methods appear necessary.
PubMed: 33803871
DOI: 10.3390/ani11030852 -
Journal of the American Heart... Aug 2014Peripheral artery disease (PAD) is associated with impaired mobility and a high rate of mortality. The aim of this systematic review was to investigate whether reduced... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Peripheral artery disease (PAD) is associated with impaired mobility and a high rate of mortality. The aim of this systematic review was to investigate whether reduced lower extremity performance was associated with an increased incidence of cardiovascular and all-cause mortality in people with PAD.
METHODS AND RESULTS
A systematic search of the MEDLINE, EMBASE, SCOPUS, Web of Science, and Cochrane Library databases was conducted. Studies assessing the association between measures of lower extremity performance and cardiovascular or all-cause mortality in PAD patients were included. A meta-analysis was conducted combining data from commonly assessed performance tests. The 10 identified studies assessed lower extremity performance by strength tests, treadmill walking performance, 6-minute walk, walking velocity, and walking impairment questionnaire (WIQ). A meta-analysis revealed that shorter maximum walking distance was associated with increased 5-year cardiovascular (unadjusted RR=2.54, 95% CI 1.86 to 3.47, P<10(-5), n=1577, fixed effects) and all-cause mortality (unadjusted RR=2.23 95% CI 1.85 to 2.69, P<10(-5), n=1710, fixed effects). Slower 4-metre walking velocity, a lower WIQ stair-climbing score, and poor hip extension, knee flexion, and plantar flexion strength were also associated with increased mortality. No significant associations were found for hip flexion strength, WIQ distance score, or WIQ speed score with mortality.
CONCLUSIONS
A number of lower extremity performance measures are prognostic markers for mortality in PAD and may be useful clinical tools for identifying patients at higher risk of death. Further studies are needed to determine whether interventions that improve measures of lower extremity performance reduce mortality.
Topics: Cardiovascular Diseases; Exercise Test; Humans; Lower Extremity; Mortality; Peripheral Arterial Disease; Prognosis; Walking
PubMed: 25122666
DOI: 10.1161/JAHA.114.001105 -
British Journal of Haematology Aug 2015Many studies report estimated pulmonary artery systolic pressure (ePASP) in patients with sickle cell disease (SCD) screened by echocardiography. To better understand... (Meta-Analysis)
Meta-Analysis Review
Many studies report estimated pulmonary artery systolic pressure (ePASP) in patients with sickle cell disease (SCD) screened by echocardiography. To better understand the prevalence and outcomes of elevated ePASP in clinically stable SCD patients, we conducted a random-effects meta-analysis. A total of 45 studies, representing 15 countries and including 6109 individuals, met our inclusion criteria. In most (70%) studies, elevated ePASP was defined by a tricuspid regurgitant velocity of 2.5 m/s. The prevalence of elevated ePASP was 21% (17-26%) in children and 30% (26-35%) in adults. After adjustment for sex, SCD genotype, haemoglobin, hydroxycarbamide (hydroxyurea) treatment, country and publication year, age remained associated with elevated ePASP, yielding a 12% (0.4-23%) higher adjusted prevalence in adults. Few studies reported 6-min walk tests or mortality outcomes, and estimates were highly heterogeneous. In random effects meta-analyses, patients with elevated ePASP walked an estimated 30.4 (6.9-53.9) metres less than those without elevated ePASP and had an associated mortality hazard ratio of 4.9 (2.4-9.7).
Topics: Adult; Anemia, Sickle Cell; Antisickling Agents; Arterial Pressure; Echocardiography; Female; Humans; Hydroxyurea; Male; Prevalence; Pulmonary Artery
PubMed: 25854714
DOI: 10.1111/bjh.13447 -
The Cochrane Database of Systematic... Jun 2017Circuit class therapy (CCT) offers a supervised group forum for people after stroke to practise tasks, enabling increased practice time without increasing staffing. This... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Circuit class therapy (CCT) offers a supervised group forum for people after stroke to practise tasks, enabling increased practice time without increasing staffing. This is an update of the original review published in 2010.
OBJECTIVES
To examine the effectiveness and safety of CCT on mobility in adults with stroke.
SEARCH METHODS
We searched the Cochrane Stroke Group Trials Register (last searched January 2017), CENTRAL (the Cochrane Library, Issue 12, 2016), MEDLINE (1950 to January 2017), Embase (1980 to January 2017), CINAHL (1982 to January 2017), and 14 other electronic databases (to January 2017). We also searched proceedings from relevant conferences, reference lists, and unpublished theses; contacted authors of published trials and other experts in the field; and searched relevant clinical trials and research registers.
SELECTION CRITERIA
Randomised controlled trials (RCTs) including people over 18 years old, diagnosed with stroke of any severity, at any stage, or in any setting, receiving CCT.
DATA COLLECTION AND ANALYSIS
Review authors independently selected trials for inclusion, assessed risk of bias in all included studies, and extracted data.
MAIN RESULTS
We included 17 RCTs involving 1297 participants. Participants were stroke survivors living in the community or receiving inpatient rehabilitation. Most could walk 10 metres without assistance. Ten studies (835 participants) measured walking capacity (measuring how far the participant could walk in six minutes) demonstrating that CCT was superior to the comparison intervention (Six-Minute Walk Test: mean difference (MD), fixed-effect, 60.86 m, 95% confidence interval (CI) 44.55 to 77.17, GRADE: moderate). Eight studies (744 participants) measured gait speed, again finding in favour of CCT compared with other interventions (MD 0.15 m/s, 95% CI 0.10 to 0.19, GRADE: moderate). Both of these effects are considered clinically meaningful. We were able to pool other measures to demonstrate the superior effects of CCT for aspects of walking and balance (Timed Up and Go: five studies, 488 participants, MD -3.62 seconds, 95% CI -6.09 to -1.16; Activities of Balance Confidence scale: two studies, 103 participants, MD 7.76, 95% CI 0.66 to 14.87). Two other pooled balance measures failed to demonstrate superior effects (Berg Blance Scale and Step Test). Independent mobility, as measured by the Stroke Impact Scale, Functional Ambulation Classification and the Rivermead Mobility Index, also improved more in CCT interventions compared with others. Length of stay showed a non-significant effect in favour of CCT (two trials, 217 participants, MD -16.35, 95% CI -37.69 to 4.99). Eight trials (815 participants) measured adverse events (falls during therapy): there was a non-significant effect of greater risk of falls in the CCT groups (RD 0.03, 95% CI -0.02 to 0.08, GRADE: very low). Time after stroke did not make a difference to the positive outcomes, nor did the quality or size of the trials. Heterogeneity was generally low; risk of bias was variable across the studies with poor reporting of study conduct in several of the trials.
AUTHORS' CONCLUSIONS
There is moderate evidence that CCT is effective in improving mobility for people after stroke - they may be able to walk further, faster, with more independence and confidence in their balance. The effects may be greater later after the stroke, and are of clinical significance. Further high-quality research is required, investigating quality of life, participation and cost-benefits, that compares CCT with standard care and that also investigates the influence of factors such as stroke severity and age. The potential risk of increased falls during CCT needs to be monitored.
Topics: Adult; Arm; Exercise Therapy; Gait; Humans; Postural Balance; Randomized Controlled Trials as Topic; Recovery of Function; Stroke Rehabilitation; Walk Test; Walking Speed
PubMed: 28573757
DOI: 10.1002/14651858.CD007513.pub3 -
The Cochrane Database of Systematic... Apr 2016Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome, with the scale of severity being... (Review)
Review
BACKGROUND
Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome, with the scale of severity being such that Hurler syndrome is the most severe and Scheie syndrome the least severe. It is a rare, autosomal recessive disorder caused by a deficiency of alpha-L-iduronidase. Deficiency of this enzyme results in the accumulation of glycosaminoglycans within the tissues. The clinical manifestations are facial dysmorphism, hepatosplenomegaly, upper airway obstruction, skeletal deformity and cardiomyopathy. If Hurler syndrome is left untreated, death ensues by adolescence. There are more attenuated variants termed Hurler-Scheie or Scheie syndrome, with those affected potentially not presenting until adulthood. Enzyme replacement therapy has been used for a number of years in the treatment of Hurler syndrome, although the current gold standard would be a haemopoietic stem cell transplant in those diagnosed by 2.5 years of age. This is an updated version of the original Cochrane review published in 2013.
OBJECTIVES
To evaluate the effectiveness and safety of treating mucopolysaccharidosis type I with laronidase enzyme replacement therapy as compared to placebo.
SEARCH METHODS
We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register, MEDLINE via OVID and Embase.Date of most recent search: 05 October 2015.
SELECTION CRITERIA
Randomised and quasi-randomised controlled studies of laronidase enzyme replacement therapy compared to placebo.
DATA COLLECTION AND ANALYSIS
Two authors independently screened the identified studies. The authors then appraised and extracted data.
MAIN RESULTS
One study of 45 patients met the inclusion criteria. This double-blind, placebo-controlled, randomised, multinational study looked at laronidase at a dose of 0.58 mg/kg/week versus placebo in patients with mucopolysaccharidosis type I. All primary outcomes listed in this review were studied in this study. The laronidase group achieved statistically significant improvements in per cent predicted forced vital capacity compared to placebo, MD 5.60 (95% confidence intervals 1.24 to 9.96) and in the six-minute-walk test (mean improvement of 38.1 metres in the laronidase group; P = 0.039, when using a prospectively planned analysis of covariance). The levels of urinary glycoaminoglycans were also significantly reduced. In addition, there were improvements in hepatomegaly, sleep apnoea and hypopnoea. Laronidase antibodies were detected in nearly all patients in the treatment group with no apparent clinical effect and titres were reducing by the end of the study. Infusion-related adverse reactions occurred in both groups but all were mild and none necessitated medical intervention or infusion cessation.
AUTHORS' CONCLUSIONS
The current evidence demonstrates that laronidase is effective when compared to placebo in the treatment of mucopolysaccharidosis type I. The included study was comprehensive and of good quality, although there were few participants. The study included all of the key outcome measures we wished to look at. It demonstrated that laronidase is efficacious in relation to reducing biochemical parameters (reduced urine glycosaminoglycan excretion) and improved functional capacity as assessed by forced vital capacity and the six-minute-walk test. In addition glycosaminoglycan storage was reduced as ascertained by a reduction in liver volume. Laronidase appeared to be safe and, while antibodies were generated, these titres were reducing by the end of the study. More studies are required to determine long-term effectiveness and safety and to assess the impact upon quality of life. Enzyme replacement therapy with laronidase can be used pre- and peri-haemopoietic stem cell transplant, which is now the gold standard treatment in those patients diagnosed under 2.5 years of age.
Topics: Adolescent; Adult; Antibodies; Child; Enzyme Replacement Therapy; Female; Humans; Iduronidase; Male; Mucopolysaccharidosis I; Randomized Controlled Trials as Topic; Rare Diseases; Severity of Illness Index; Young Adult
PubMed: 27033167
DOI: 10.1002/14651858.CD009354.pub4