RESUMO
In May 2003, Madrid established the universal newborn screening (NBS) for sickle cell disease (SCD). However, there are no studies resembling the evolution of a SCD neonate cohort followed according to national guidelines in Spain. The aim of this study is to describe the morbimortality and the stroke prevention programme in patients diagnosed by SCD NBS in Madrid. This is a multicentre, observational, prospective cohort study between 2003 and 2018; 187 patients diagnosed with SCD were included (151 HbSS, 6 HbSß0, 27 HbSC, 3 HbSß +), and median follow-up was 5.2 years (0.03-14.9). There were 5 deaths: 2 related to SCD in patients with severe genotype (HbSS/HbSß0). Overall survival reached 95% and SCD-related survival 96.8%. The most frequent events were fever without focus, vaso-occlusive crises and acute chest syndromes. Eight strokes occurred in 5 patients which led to a 90.7% stroke-free survival in severe genotype patients (first stroke rate, 0.54 per 100 patient-years). Transcranial Doppler (TCD) was performed in 95% of eligible patients; 75% of children with pathological TCD remained stroke-free. Regarding HbSS/HbSß0 patients, 50.1% received hydroxyurea and 9.5% haematopoietic stem cell transplantation. This study reflects the evolution of Madrid SCD cohort and provides morbimortality data similar to other developed countries.
Assuntos
Síndrome Torácica Aguda , Anemia Falciforme , Acidente Vascular Cerebral , Criança , Humanos , Recém-Nascido , Anemia Falciforme/terapia , Anemia Falciforme/tratamento farmacológico , Hemoglobina Falciforme , Hidroxiureia/uso terapêutico , Estudos Prospectivos , Acidente Vascular Cerebral/epidemiologia , Acidente Vascular Cerebral/etiologia , Acidente Vascular Cerebral/prevenção & controle , Lactente , Pré-Escolar , AdolescenteRESUMO
BACKGROUND: There is no consensus on which right ventricle (RV) strain parameter should be used in the clinical practice: four chamber RV longitudinal strain (4CH RV-LS) or free wall longitudinal strain (FWLS). The aim of this study was to analyze which RV strain parameter better predicts prognosis in patients with left heart disease. METHODS: One hundred and three outpatients with several degrees of functional tricuspid regurgitation severity secondary to left heart disease were prospectively included. 4CH RV-LS and FWLS were assessed using speckle tracking. Left ventricular (LV) systolic function was determined using LV ejection fraction and RV systolic function using tricuspid annular plane systolic excursion (TAPSE). Patients were followed up for 23.1 ± 12.4 months for an endpoint of cardiac hospitalization due to heart failure. RESULTS: The cutoff value related to RV dysfunction (TAPSE < 17 mm) was lower, in absolute value, for 4CH RV-LS (4CH RV-LS = -17.3%; FWLS = -19.5%). There were 33 adverse events during the follow-up. Patients with 4CH RV-LS > -17.3% (log rank [LR] = 22.033; p < 0.001); FWLS > -19.5% (LR = 12.2; p < 0.001), TAPSE < 17 mm (LR = 17.4; p < 0.001) and LV systolic dysfunction (LR = 13.3; p < 0.001) had lower event-free survival (Kaplan Meier). In Cox multivariate analysis, 4CH RV-LS > -17.3% (hazard ratio [HR] = 3.593; p < 0.002), TAPSE < 17 (HR = 2.093; p < 0.055) and LV systolic dysfunction (HR = 2.087; p < 0,054) had prognostic value, whereas FWLS did not reach significance. CONCLUSIONS: Although both 4CH RV-LS and FWLS have prognostic value, 4CH RV-LS is a better predictor of episodes of heart failure in patients with left heart disease, providing additional information to that obtained by TAPSE.