RESUMEN
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.
Asunto(s)
Anemia de Células Falciformes , Sobrecarga de Hierro , Talasemia , Adolescente , Anemia de Células Falciformes/complicaciones , Anemia de Células Falciformes/tratamiento farmacológico , Transfusión Sanguínea , Deferiprona/uso terapéutico , Deferoxamina/efectos adversos , Femenino , Humanos , Quelantes del Hierro/efectos adversos , Sobrecarga de Hierro/tratamiento farmacológico , Sobrecarga de Hierro/etiología , Masculino , Piridonas/efectos adversos , Talasemia/complicaciones , Talasemia/tratamiento farmacológico , TransferasasRESUMEN
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.