Journal of investigative medicine : the official publication of the American Federation for Clinical Research
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Patients with sickle cell disease (SCD) experience a range of clinical symptoms, including acute and chronic pain, fatigue, and respiratory problems, as well as chronic organ complications that can lead to disability and accelerated mortality. Voxelotor is a first-in-class therapy that targets sickle hemoglobin polymerization, the root cause of SCD. It is approved by the US Food and Drug Administration for treatment of SCD in patients aged 4 years and older and in the European Union and United Arab Emirates for the treatment of SCD in patients aged 12 years and older. ⋯ As observed in previous clinical studies, hemoglobin concentrations and markers of hemolysis were improved in patients treated with voxelotor. Most patients reported marked improvement in disease symptoms, which correlated well with the clinicians' assessments. Although limited by the retrospective open-label study design, these findings suggest that voxelotor use has a positive impact on outcomes in patients with SCD.
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This study aims to evaluate the efficacy and safety of endovascular therapy versus neurosurgical clipping carried out for patients with unruptured middle cerebral artery bifurcation aneurysms (MCABAs). Patients diagnosed with MCABAs were enrolled in this prospective study according to the inclusion and exclusion standard. Enrolled patients were divided into a study group (endovascular therapy) and a control group (neurosurgical clipping), with 65 cases in each group. ⋯ The postoperative brain injury indicators neuron-specific enolase and S100β in the study group were significantly lower than in the control group (p<0.001), and the postoperative life activity score of patients in the study group was significantly higher than in the control group (p<0.001). In terms of safety, the postoperative hospital stay of patients in the study group was significantly shorter than in the control group (p<0.001), and the incidence rate of postoperative pulmonary and intracranial infections in the study group was significantly lower than in the control group (p<0.05). Endovascular therapy for patients with unruptured MCABAs may be effective in improving outcomes and has better safety profile compared with neurosurgical clipping, but may increase the risk of postoperative recurrence.