RESUMO
ABSTRACT: We assessed the feasibility to estimate illness burden in adults with SCD, investigated factors associated with health-related quality of life (HRQoL), and estimated societal burden. We recruited 32 participants and collected data on fatigue, HRQoL, and work productivity and activity impairment via patient survey. Health care utilization was abstracted for the 12 months before enrollment using medical chart review. Mean age was 36.7 years; 84.4% of participants had hemoglobin SS or Sßthal0 disease, and 81.3% reported chronic pain (experiencing pain on ≥3 days per week in the past 6 months). Mean EQ-5D-3L visual analogue scale score was 63.4 and the index score was 0.79. The mean fatigue score was 57.9. Higher fatigue score was correlated with lower EQ-5D index score (correlation coefficient r = -0.35; P = .049) and Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) scores, including pain (r = -0.47; P = .006), sleep (r = -0.38; P = .03), and emotion scores (r = -0.79; P < .0001). The number of hospitalizations was negatively correlated with HRQoL (all P < .05). Patients who reported chronic pain had significantly lower mean ASCQ-Me sleep scores (48.3 vs 57.1; P = .04) and EQ-5D index scores (0.72 vs 0.89; P = .002) than those without chronic pain. Mean estimated annual per person costs were $51 779 (median, $36 366) for total costs, $7619 ($0) for indirect costs (estimated from lost earnings of participants), and $44 160 ($31 873) for medical costs. Fatigue, SCD complications, hospitalization, and chronic pain negatively affected HRQoL. This sample experienced a high economic burden, largely from outpatient doctor visits.
Assuntos
Anemia Falciforme , Efeitos Psicossociais da Doença , Qualidade de Vida , Humanos , Anemia Falciforme/complicações , Anemia Falciforme/economia , Adulto , Projetos Piloto , Masculino , Feminino , Pessoa de Meia-Idade , Fadiga/etiologiaRESUMO
Many people with sickle cell disease (SCD) or other anemias require chronic blood transfusions, which often causes iron overload that requires chelation therapy. The iron chelator deferiprone is frequently used in individuals with thalassemia syndromes, but data in patients with SCD are limited. This open-label study assessed the efficacy and safety of deferiprone in patients with SCD or other anemias receiving chronic transfusion therapy. A total of 228 patients (mean age: 16.9 [range, 3-59] years; 46.9% female) were randomized to receive either oral deferiprone (n = 152) or subcutaneous deferoxamine (n = 76). The primary endpoint was change from baseline at 12 months in liver iron concentration (LIC), assessed by R2* magnetic resonance imaging (MRI). The least squares mean (standard error) change in LIC was -4.04 (0.48) mg/g dry weight for deferiprone vs -4.45 (0.57) mg/g dry weight for deferoxamine, with noninferiority of deferiprone to deferoxamine demonstrated by analysis of covariance (least squares mean difference 0.40 [0.56]; 96.01% confidence interval, -0.76 to 1.57). Noninferiority of deferiprone was also shown for both cardiac T2* MRI and serum ferritin. Rates of overall adverse events (AEs), treatment-related AEs, serious AEs, and AEs leading to withdrawal did not differ significantly between the groups. AEs related to deferiprone treatment included abdominal pain (17.1% of patients), vomiting (14.5%), pyrexia (9.2%), increased alanine transferase (9.2%) and aspartate transferase levels (9.2%), neutropenia (2.6%), and agranulocytosis (0.7%). The efficacy and safety profiles of deferiprone were acceptable and consistent with those seen in patients with transfusion-dependent thalassemia. This trial study was registered at www://clinicaltrials.gov as #NCT02041299.
Assuntos
Anemia Falciforme , Sobrecarga de Ferro , Talassemia , Adolescente , Anemia Falciforme/complicações , Anemia Falciforme/tratamento farmacológico , Transfusão de Sangue , Deferiprona/uso terapêutico , Desferroxamina/efeitos adversos , Feminino , Humanos , Quelantes de Ferro/efeitos adversos , Sobrecarga de Ferro/tratamento farmacológico , Sobrecarga de Ferro/etiologia , Masculino , Piridonas/efeitos adversos , Talassemia/complicações , Talassemia/tratamento farmacológico , TransferasesRESUMO
INTRODUCTION: sickle cell disease (SCD) is a chronic illness. Individuals affected by this disease are at risk for lifelong complications including episodes of acute pain, chronic pain and multi-organ injury that leads to reduced quality of life and a shortened life span. There is a wealth of data on acute care utilization for SCD in the United States. However, data from the Caribbean region is limited. The objective of this study is to explore Emergency Department (ED) utilization for SCD in St. Vincent and the Grenadines by describing: i) the characteristics of SCD related ED encounters; ii) the urgency of these encounters as defined by resource utilization; iii) the disposition for these ED encounters. METHODS: the study was a cross-sectional study utilizing data from the ED log books at the Milton Cato Memorial Hospital (MCMH) during non-consecutive time periods between January 1st, 2012 - December 31st, 2016. RESULTS: there were 666 SCD-related ED encounters during the study period. Thirty-four percent of encounters resulted in hospitalization and 66% of encounters met criteria for an urgent visit. The most commonly reported diagnosis was vaso-occlusive crisis and accounted for 84% of all encounters. The most frequently documented age group was the 18-30 age category at 43%. CONCLUSION: although SCD comprised less than 2% of all ED visits, the majority of these visits could be classified as urgent visits based on resource utilization. This study adds to the emerging data on the burden of this disease in this St. Vincent and the Grenadines.