Nocturnal enuresis is a prevalent and challenging problem in children and young adults with sickle cell disease (SCD). Limited progress has been made in elucidating etiology and pathophysiology of nocturnal enuresis in individuals with SCD. Among adults with SCD ages 18-20 years, approximately 9% report nocturnal enuresis. Nocturnal enuresis contributes to decreased health related quality of life in people with SCD, resulting in low self-esteem and sometimes social isolation. Postulated non-mutually exclusive causes of nocturnal enuresis in individuals with SCD include hyposthenuria leading to nocturnal polyuria, decreased bladder capacity or nocturnal bladder overactivity, increased arousal thresholds, and sleep disordered breathing. No evidence-based therapy for nocturnal enuresis in SCD exists. This review is focused on describing the natural history, postulated causes and a rational approach to the evaluation and management of nocturnal enuresis in children and adults with SCD.
Individuals with sickle cell anemia (SCA) exhibit delayed growth trajectories and lower blood pressure (BP) measurements than individuals without SCA. We evaluated factors associated with height, weight, and BP and established reference growth curves and BP tables using data from the Silent Cerebral Infarct MultiCenter Clinical (SIT) Trial (NCT00072761). Quantile regression models were used to determine the percentiles of growth and BP measurements. Multivariable quantile regression was used to test associations of baseline variables with height, weight, and BP measurements. Height and weight measurements were collected from a total of 949 participants with median age of 10.5 years [Interquartile range (IQR) 8.2-12.9] and median follow-up time of 3.2 years (IQR 1.8-4.7, range 0-12.9). Serial BP measurements were collected from a total of 944 and 943 participants, respectively, with median age of 10.6 years (IQR 5 8.3-12.9 years), and median follow-up time of 3.3 years (IQR 5 1.7-4.8). Multivariable quantile regression analysis revealed that higher hemoglobin measurements at baseline were associated with greater height (P < 0.001), weight (P 5 0.000), systolic BP (P < 0.001), and diastolic BP (P 5 0.003) measurements. We now provide new reference values for height, weight, and BP measurements that are now readily available for medical management.
We evaluated the performance of Time to Clinical Stability (TCS), a longitudinal outcome measure using four physiologic parameters (temperature, heart rate, respiratory rate, and use of supplemental oxygen), among children enrolled in a prospective study of pneumonia hospitalizations. We calculated the time from admission to normalization for each of the four parameters individually and various combinations of these parameters (>2 parameters). We assessed for agreement between the combined TCS measures and both hospital length of stay and an ordinal severity scale (non-severe, severe, and very severe). Overall, 323 (96.7%) of 334 included children had ≥1 parameter abnormal on admission; 70 (21%) children had ≥1 parameter abnormal at discharge. For the four combined measures, median TCS decreased with increasing age. Increasing TCS was associated with both longer length of stay and increasing disease severity. The simplest combined measure incorporating only respiratory rate and need for supplemental oxygen performed similarly to more complex measures including additional parameters. Our study demonstrates that longitudinal TCS measures may be useful in children with pneumonia, both in clinical settings to assess recovery and readiness for discharge, and as an outcome measure in research and quality assessments. Additional study is needed to further validate our findings.
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