Sickle cell patients are characterized by a reduced glycocalyx volume

The glycocalyx is an important anti-inflammatory and anti-adhesive barrier at the luminal side of endothelial cells. Glycocalyx volume was significantly reduced in sickle cell patients (HbSS/HbSβ0-thalassemia median 0.47L, IQR 0.27-0.66, HbSC/HbSβ+-thalassemia 0.23L, 0.0-0.58) compared with controls (1×109L, 0.52-1.77) (p=0.03). Reduced glycocalyx may be a new factor in the pathophysiology of sickle cell disease

EVALUACIÓN DE RESULTADOS QUIRÚRGICOS DESDE LA PERSPECTIVA DEL PACIENTE

RESUMENMedir la calidad de vida relacionada a salud y los síntomas de los pacientes es un problema difícil. Las medición de los problemas de salud mediante escalas o cuestionarios se ha desarrollado para crear los Instrumentos de Evaluación desde la Perspectiva del Paciente o PRO's por su nombre en inglés: Patient-reported outcomes. Los PROs evalúan la calidad de vida en forma genérica o específica en un continuo, entregando instrumentos que pueden evaluar la gravedad de una enfermedad o el impacto de una intervención desde la perspectiva del paciente en frecuentes escenarios clínicos. El objetivo de la presente revisión es entregar a clínicos e investigadores una introducción hacia los PROs y resumir sus principales propiedades.SUMMARYQuantifying health-related quality of life and specific patient symptoms it is a difficult problem. Health measurement scales has developed to include rigorous techniques to develop patient-reported outcome measures (PROs). PROs assess objectively the QoL in a continuum, providing instruments to measure the severity of a given disease or the impact of a therapeutic intervention from patient perspective in different clinical problems. The following review aims to introduce the PROs to clinicians and researchers and summarize its main properties

Inhaled steroids reduce pain and sVCAM levels in individuals with sickle cell disease: A triple‐blind, randomized trial

Clinical and preclinical data demonstrate that altered pulmonary physiology (including increased inflammation, increased blood flow, airway resistance, and hyper‐reactivity) is an intrinsic component of Sickle Cell Disease (SCD) and may contribute to excess SCD morbidity and mortality. Inhaled corticosteroids (ICS), a safe and effective therapy for pulmonary inflammation in asthma, may ameliorate the altered pulmonary physiologic milieu in SCD. With this single‐center, longitudinal, randomized, triple‐blind, placebo controlled trial we studied the efficacy and feasibility of ICS in 54 nonasthmatic individuals with SCD. Participants received once daily mometasone furoate 220 mcg dry powder inhalation or placebo for 16 weeks. The primary outcome was feasibility (the number who complete the trial divided by the total number enrolled) with prespecified efficacy outcomes including daily pain score over time (patient reported) and change in soluble vascular cell adhesion molecule (sVCAM) levels between entry and 8‐weeks. For the primary outcome of feasibility, the result was 96% (52 of 54, 95% CI 87%‐99%) for the intent‐to‐treat analysis and 83% (45 of 54, 95% CI 71%‐91%) for the per‐protocol analysis. The adjusted treatment effect of mometasone was a reduction in daily pain score of 1.42 points (95%CI 0.61‐2.21, P = 0.001). Mometasone was associated with a reduction in sVCAM levels of 526.94 ng/mL more than placebo (95% CI 50.66‐1003.23, P = 0.03). These results support further study of ICS in SCD including multicenter trials and longer durations of treatment. www.clinicaltrials.gov (NCT02061202)Peer Reviewedhttps://deepblue.lib.umich.edu/bitstream/2027.42/137301/1/ajh24742.pdfhttps://deepblue.lib.umich.edu/bitstream/2027.42/137301/2/ajh24742_am.pd

N-acetylcysteine reduces oxidative stress in sickle cell patients

Oxidative stress is of importance in the pathophysiology of sickle cell disease (SCD). In this open label randomized pilot study the effects of oral N-acetylcysteine (NAC) on phosphatidylserine (PS) expression as marker of cellular oxidative damage (primary end point), and markers of hemolysis, coagulation and endothelial activation and NAC tolerability (secondary end points) were studied. Eleven consecutive patients (ten homozygous [HbSS] sickle cell patients, one HbSβ0-thalassemia patient) were randomly assigned to treatment with either 1,200 or 2,400 mg NAC daily during 6 weeks. The data indicate an increment in whole blood glutathione levels and a decrease in erythrocyte outer membrane phosphatidylserine exposure, plasma levels of advanced glycation end-products (AGEs) and cell-free hemoglobin after 6 weeks of NAC treatment in both dose groups. One patient did not tolerate the 2,400 mg dose and continued with the 1,200 mg dose. During the study period, none of the patients experienced painful crises or other significant SCD or NAC related complications. These data indicate that N-acetylcysteine treatment of sickle cell patients may reduce SCD related oxidative stress