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

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

An urgent call to raise the bar in oncology

Important breakthroughs in medical treatments have improved outcomes for patients suffering from several types of cancer. However, many oncological treatments approved by regulatory agencies are of low value and do not contribute significantly to cancer mortality reduction, but lead to unrealistic patient expectations and push even affluent societies to unsustainable health care costs. Several factors that contribute to approvals of low-value oncology treatments are addressed, including issues with clinical trials, bias in reporting, regulatory agency shortcomings and drug pricing. With the COVID-19 pandemic enforcing the elimination of low-value interventions in all fields of medicine, efforts should urgently be made by all involved in cancer care to select only high-value and sustainable interventions. Transformation of medical education, improvement in clinical trial design, quality, conduct and reporting, strict adherence to scientific norms by regulatory agencies and use of value-based scales can all contribute to raising the bar for oncology drug approvals and influence drug pricing and availability

Plasma concentrations of asymmetric dimethylarginine, an endogenous nitric oxide synthase inhibitor, are elevated in sickle cell patients but do not increase further during painful crisis

Plasma concentrations of asymmetric dimethylarginine (ADMA), an endogenous nitric oxide synthase inhibitor, are elevated in the clinically asymptomatic state of sickle cell disease (SCD). However, the role of ADMA during vaso-occlusive complications has not been defined. ADMA concentrations were determined in HbSS (n = 43) and HbSC (n = 25) patients with healthy blood donors (HbAA) as controls. In the clinically asymptornatic state ADMA concentrations were elevated in sickle cell patients as compared to healthy controls (HbSS 0.70 mu mol/L, HbSC 0.54 mu mol/L, HbAA 0.39 mu mol/L) (P <0.001). Yet plasma ADMA concentrations did not increase further at presentation with a painful crisis implicating no role of primary importance during vaso-occlusive crises

Pain rate and social circumstances rather than cumulative organ damage determine the quality of life in adults with sickle cell disease

International audienceBackground: Due to the significant morbidity associated with sickle cell disease (SCD) sickle cell patients have a reduced quality of life (QoL). Even though pain is considered an important determinant of QoL in sickle cell patients, factors such as chronic organ damage and socioeconomic circumstances may also be important. Therefore we determined the contribution of chronic organ damage and sickle cell related complications to QoL and also analysed the effect of vaso-occlusive crises and socio-economic circumstances on QoL in our patients. Methods: Consecutive adult sickle cell patients were included. QoL was represented in a physical component scale (PCS) and a mental component scale (MCS) and was assessed with SF-36 forms. We studied these QoL scores in relation to SCD severity (organ damage and pain rate) and socioeconomic factors (patients occupation and educational level). Results: Higher pain rates were related to lower QoL scores. Pain rate was significantly associated with MCS but not with the PCS. Both occupation and the level of education were significantly related to PCS while no relation with MCS or pain rate was found. Thirty-five percent patients were unemployed as compared to 6% of the general population and 16% of immigrants without SCD. Neither genotype nor the presence of chronic organ damage were significantly related to QoL. Conclusion: Sickle cell patients in Amsterdam, the Netherlands, have a significantly reduced QoL mainly determined by pain rate, occupation and educational level. Chronic organ damage, although a major factor determining life expectancy in SCD, was not a determinant of QoL