Although larger validation studies are required, RH-PAT is a promising non-invasive technique to assess endothelial function in children with T1D. Non-invasive measures of endothelial dysfunction may provide the additional risk stratification data needed to justify more aggressive primary prevention of cardiovascular disease in children with T1D.
Fifty-one children with type 1 diabetes mellitus (DM1) participated in a double blinded, randomized, cross-over pilot study to determine whether 12 weeks of daily atorvastatin (20 mg daily) would reduce arterial stiffness and improve endothelial function. Secondary analysis demonstrated potential reduction of arterial stiffness following atorvastatin therapy (p = 0.06). Additional long-term prospective studies with larger numbers of patients are needed.
Background
Recent studies suggest that cerebral revascularization surgery may be a safe and effective therapy to reduce stroke risk in patients with sickle cell disease and moyamoya syndrome (SCD–MMS).
Methods
We performed a multicenter, retrospective study of children with SCD–MMS treated with conservative management alone (conservative group)—chronic blood transfusion and/or hydroxyurea—versus conservative management plus surgical revascularization (surgery group). We monitored cerebrovascular event (CVE) rates—a composite of strokes and transient ischemic attacks. Multivariable logistic regression was used to compare CVE occurrence and multivariable Poisson regression was used to compare incidence rates between groups. Covariates in multivariable models included age at treatment start, age at moyamoya diagnosis, antiplatelet use, CVE history, and the risk period length.
Results
We identified 141 patients with SCD–MMS, 78 (55.3%) in the surgery group and 63 (44.7%) in the conservative group. Compared with the conservative group, preoperatively the surgery group had a younger age at moyamoya diagnosis, worse baseline modified Rankin scale scores, and increased prevalence of CVEs. Despite more severe pretreatment disease, the surgery group had reduced odds of new CVEs after surgery (odds ratio = 0.27, 95% confidence interval [CI] = 0.08–0.94, p = .040). Furthermore, comparing surgery group patients during presurgical versus postsurgical periods, CVEs odds were significantly reduced after surgery (odds ratio = 0.22, 95% CI = 0.08–0.58, p = .002).
Conclusions
When added to conservative management, cerebral revascularization surgery appears to reduce the risk of CVEs in patients with SCD–MMS. A prospective study will be needed to validate these findings.
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