FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2897645750> ?p ?o ?g. }

• W2897645750 abstract "The dystrophinopathies include Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and X-linked dilated cardiomyopathy (XLDCM). In recent years, co-ordinated multidisciplinary management for these diseases has improved the quality of care, with early corticosteroid use prolonging independent ambulation, and the routine use of non-invasive ventilation signficantly increasing survival. The next target to improve outcomes is optimising treatments to delay the onset or slow the progression of cardiac involvement and so prolong survival further.To assess the effects of interventions for preventing or treating cardiac involvement in DMD, BMD, and XLDCM, using measures of change in cardiac function over six months.On 16 October 2017 we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE and Embase, and on 12 December 2017, we searched two clinical trials registries. We also searched conference proceedings and bibliographies.We considered only randomised controlled trials (RCTs), quasi-RCTs and randomised cross-over trials for inclusion. In the Discussion, we reviewed open studies, longitudinal observational studies and individual case reports but only discussed studies that adequately described the diagnosis, intervention, pretreatment, and post-treatment states and in which follow-up lasted for at least six months.Two authors independently reviewed the titles and abstracts identified from the search and performed data extraction. All three authors assessed risk of bias independently, compared results, and decided which trials met the inclusion criteria. They assessed the certainty of evidence using GRADE criteria.We included five studies (N = 205) in the review; four studies included participants with DMD only, and one study included participants with DMD or BMD. All studied different interventions, and meta-analysis was not possible. We found no studies for XLDCM. None of the trials reported cardiac function as improved or stable cardiac versus deteriorated.The randomised first part of a two-part study of perindopril (N = 28) versus placebo (N = 27) in boys with DMD with normal heart function at baseline showed no difference in the number of participants with a left ventricular ejection fraction (LVEF%) of less than 45% after three years of therapy (n = 1 in each group; risk ratio (RR) 1.04, 95% confidence interval (CI) 0.07 to 15.77). This result is uncertain because of study limitations, indirectness and imprecision. In a non-randomised follow-up study, after 10 years, more participants who had received placebo from the beginning had reduced LVEF% (less than 45%). Adverse event rates were similar between the placebo and treatment groups (low-certainty evidence).A study comparing treatment with lisinopril versus losartan in 23 boys newly diagnosed with Duchenne cardiomyopathy showed that after 12 months, both were equally effective in preserving or improving LVEF% (lisinopril 54.6% (standard deviation (SD) 5.19), losartan 55.2% (SD 7.19); mean difference (MD) -0.60% CI -6.67 to 5.47: N = 16). The certainty of evidence was very low because of very serious imprecision and study limitations (risk of bias). Two participants in the losartan group were withdrawn due to adverse events: one participant developed an allergic reaction, and a second exceeded the safety standard with a fall in ejection fraction greater than 10%. Authors reported no other adverse events related to the medication (N = 22; very low-certainty evidence).A study comparing idebenone versus placebo in 21 boys with DMD showed little or no difference in mean change in cardiac function between the two groups from baseline to 12 months; for fractional shortening the mean change was 1.4% (SD 4.1) in the idebenone group and 1.6% (SD 2.6) in the placebo group (MD -0.20%, 95% CI -3.07 to 2.67, N = 21), and for ejection fraction the mean change was -1.9% (SD 9.8) in the idebenone group and 0.4% (SD 5.5) in the placebo group (MD -2.30%, 95% CI -9.18 to 4.58, N = 21). The certainty of evidence was very low because of study limitations and very serious imprecision. Reported adverse events were similar between the treatment and placebo groups (low-certainty evidence).A multicentre controlled study added eplerenone or placebo to 42 patients with DMD with early cardiomyopathy but preserved left ventricular function already established on ACEI or ARB therapy. Results showed that eplerenone slowed the rate of decline of magnetic resonance (MR)-assessed left ventricular circumferential strain at 12 months (eplerenone group median 1.0%, interquartile range (IQR) 0.3 to -2.2; placebo group median 2.2%, IQR 1.3 to -3.1%; P = 0.020). The median decline in LVEF over the same period was also less in the eplerenone group (-1.8%, IQR -2.9 to 6.0) than in the placebo group (-3.7%, IQR -10.8 to 1.0; P = 0.032). We downgraded the certainty of evidence to very low for study limitations and serious imprecision. Serious adverse events were reported in two patients given placebo but none in the treatment group (very low-certainty evidence).A randomised placebo-controlled study of subcutaneous growth hormone in 16 participants with DMD or BMD showed an increase in left ventricular mass after three months' treatment but no significant improvement in cardiac function. The evidence was of very low certainty due to imprecision, indirectness, and study limitations. There were no clinically significant adverse events (very low-certainty evidence).Some studies were at risk of bias, and all were small. Therefore, although there is some evidence from non-randomised data to support the prophylactic use of perindopril for cardioprotection ahead of detectable cardiomyopathy, and for lisinopril or losartan plus eplerenone once cardiomyopathy is detectable, this must be considered of very low certainty. Findings from non-randomised studies, some of which have been long term, have led to the use of these drugs in daily clinical practice.Based on the available evidence from RCTs, early treatment with ACE inhibitors or ARBs may be comparably beneficial for people with a dystrophinopathy; however, the certainty of evidence is very low. Very low-certainty evidence indicates that adding eplerenone might give additional benefit when early cardiomyopathy is detected. No clinically meaningful effect was seen for growth hormone or idebenone, although the certainty of the evidence is also very low." @default.
• W2897645750 created "2018-10-26" @default.
• W2897645750 creator A5005870558 @default.
• W2897645750 creator A5053619828 @default.
• W2897645750 creator A5077009027 @default.
• W2897645750 date "2018-10-16" @default.
• W2897645750 modified "2023-10-13" @default.
• W2897645750 title "Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy" @default.
• W2897645750 cites W1134129585 @default.
• W2897645750 cites W137715681 @default.
• W2897645750 cites W1484824605 @default.
• W2897645750 cites W1490803713 @default.
• W2897645750 cites W1508659364 @default.
• W2897645750 cites W1521539618 @default.
• W2897645750 cites W1533447420 @default.
• W2897645750 cites W1537782350 @default.
• W2897645750 cites W1623572179 @default.
• W2897645750 cites W1786058540 @default.
• W2897645750 cites W1926031445 @default.
• W2897645750 cites W1964560232 @default.
• W2897645750 cites W1965227811 @default.
• W2897645750 cites W1967569266 @default.
• W2897645750 cites W1968131392 @default.
• W2897645750 cites W1968243270 @default.
• W2897645750 cites W1973803913 @default.
• W2897645750 cites W1977978675 @default.
• W2897645750 cites W1983606052 @default.
• W2897645750 cites W1985454225 @default.
• W2897645750 cites W1986001051 @default.
• W2897645750 cites W1986703506 @default.
• W2897645750 cites W1990558552 @default.
• W2897645750 cites W1992180113 @default.
• W2897645750 cites W1993044043 @default.
• W2897645750 cites W1995474990 @default.
• W2897645750 cites W1996618016 @default.
• W2897645750 cites W1998483343 @default.
• W2897645750 cites W1998917643 @default.
• W2897645750 cites W2000887179 @default.
• W2897645750 cites W2001638362 @default.
• W2897645750 cites W2002426309 @default.
• W2897645750 cites W2007367321 @default.
• W2897645750 cites W2008403762 @default.
• W2897645750 cites W2011097105 @default.
• W2897645750 cites W2013711331 @default.
• W2897645750 cites W2015000540 @default.
• W2897645750 cites W2015115844 @default.
• W2897645750 cites W2018358939 @default.
• W2897645750 cites W2020685420 @default.
• W2897645750 cites W2023408841 @default.
• W2897645750 cites W2030266786 @default.
• W2897645750 cites W2031789342 @default.
• W2897645750 cites W2034872844 @default.
• W2897645750 cites W2036005426 @default.
• W2897645750 cites W2037598698 @default.
• W2897645750 cites W2037638857 @default.
• W2897645750 cites W2037928660 @default.
• W2897645750 cites W2038352688 @default.
• W2897645750 cites W2039302465 @default.
• W2897645750 cites W2041892008 @default.
• W2897645750 cites W2042941794 @default.
• W2897645750 cites W2044530341 @default.
• W2897645750 cites W2045045738 @default.
• W2897645750 cites W2046329168 @default.
• W2897645750 cites W2049986040 @default.
• W2897645750 cites W2050423231 @default.
• W2897645750 cites W2051741101 @default.
• W2897645750 cites W2054116442 @default.
• W2897645750 cites W2057977641 @default.
• W2897645750 cites W2059571229 @default.
• W2897645750 cites W2062665453 @default.
• W2897645750 cites W2067635871 @default.
• W2897645750 cites W2068435782 @default.
• W2897645750 cites W2069770791 @default.
• W2897645750 cites W2080338828 @default.
• W2897645750 cites W2087760263 @default.
• W2897645750 cites W2088488094 @default.
• W2897645750 cites W2088880023 @default.
• W2897645750 cites W2090112049 @default.
• W2897645750 cites W2094763401 @default.
• W2897645750 cites W2094778815 @default.
• W2897645750 cites W2098941422 @default.
• W2897645750 cites W2106493153 @default.
• W2897645750 cites W2107774728 @default.
• W2897645750 cites W2107878321 @default.
• W2897645750 cites W2109584088 @default.
• W2897645750 cites W2110974433 @default.
• W2897645750 cites W2112090184 @default.
• W2897645750 cites W2113013062 @default.
• W2897645750 cites W2115297798 @default.
• W2897645750 cites W2116484895 @default.
• W2897645750 cites W2118122636 @default.
• W2897645750 cites W2120307618 @default.
• W2897645750 cites W2121775242 @default.
• W2897645750 cites W2124458633 @default.
• W2897645750 cites W2128448322 @default.
• W2897645750 cites W2130927534 @default.
• W2897645750 cites W2136126591 @default.
• W2897645750 cites W2136642260 @default.
• W2897645750 cites W2141526082 @default.
• W2897645750 cites W2141695132 @default.

• next