1,721,052 research outputs found
Overnight respiratory support for prevention of morbidity in Sickle Cell Disease (POMS 2a) - parent and child preferences
No full textPURPOSE: Obstructive sleep apnea and nocturnal hypoxemia are common in children with sickle cell disease (SCD) and some degree of improvement has been reported following adenotonsillectomy(Finch et al., 2013). NOT and continuous positive airways pressure (CPAP) have shown some benefits in children and adolescent patients(Hargrave, 2003). While NOT and CPAP are offered to children with or without co-morbidities, there are concerns about acceptability and safety(Makani et al., 2009). The data on patient preference is limited in childhood and more so for those with SCD. To achieve optimal adherence to therapy in SCD, Auto-Adjusting Positive Airways Pressure (APAP), where airway pressure is delivered only when obstruction occurs, may be a more acceptable option than CPAP(Marshall et al., 2009). Therefore prior to designing Phase II and III trials that are adequately powered, this study sought to: i) identify parent/caregiver and child treatment preference; ii) assess whether parent/caregiver or child should manage the electronic pain diary; iii) assess any treatment effect on Health Related Quality of Life (HRQOL) in children; and iv) explore the relationship of treatment adherence to: a) experience of pain, and b) overall clinical benefit from the intervention
The effects of hydroxycarbamide on the plasma proteome of children with sickle cell anaemia
No full textWe have investigated changes in the plasma proteome of children with sickle cell anaemia (SCA) associated with hydroxycarbamide (HC) use, to further characterize the actions of HC.
51 children with SCA consented to take part in this study. 18 were taking HC at a median dose of 22mg/kg, and 33 were not on HC. Plasma was analyzed using an unbiased proteomic approach and a panel of 92 neurological biomarkers. HC was associated with increased haemoglobin (Hb) (89.8g/l vs 81.4g/l, P=0.007) and HbF (15.3 vs 6.7%, P<0.001). 17 proteins were decreased on HC compared to controls by a factor of <0.77, and six proteins showed >1.3 fold increased concentration. HC use was associated with reduced haemolysis (lower globin chains, haptoglobin related protein, complement C9; higher hemopexin), reduced inflammation (lower -1-acid glycoprotein, CD5 antigen-like protein, ceruloplasmin, factor XII, immunoglobulins, cysteine-rich secretory protein 3, vitamin D-binding protein) and decreased activation of coagulation (lower factor XII, carboxypeptidase B2, platelet basic protein). There was a significant correlation between the increase in HbF% on HC and hemopexin levels (R=0.603, P=0.023). This study demonstrated three ways in which HC may be beneficial in SCA, and identified novel proteins, which may be useful to monitor therapeutic response
Prevention of morbidity in Sickle Cell Disease phase II (Improvement of pain and quality of life in children with Sickle Cell Disease with auto-adjusting Continuous Positive Airways Pressure: Phase II) (POMS 2b pediatric cohort)
No full textReliability of different estimated glomerular filtration rate as measures of renal function in children with sickle cell disease
No full textThere is no reliable marker for detecting early renal disease in early children with sickle cell disease (SCD). Estimation of glomerular filtration rate (eGFR) as derived from the height/plasma creatinine formula is dependent on the accuracy of the creatinine analytical method used. The aim of this study was to evaluate different equations for eGFR
Lung function impairment in pediatric patients with sickle cell anemia from Nigeria is associated with low steady state hemoglobin
No full textNocturnal oxygen desaturation and disordered sleep as a potential factor in executive dysfunction in sickle cell anemia
No full textPrevious research has identified cognitive impairment in children with sickle cell anemia (SCA, Hemoglobin SS) compared with controls, partly accounted for by overt neuropathology after clinical stroke, covert (silent) infarction, and severity of anemia. However, cognitive deficits have also been identified in children with SCA with no history of stroke and a normal T2-weighted magnetic resonance imaging (MRI) scan. Our aim was to investigate whether nocturnal hemoglobin oxygen desaturation and sleep fragmentation could be associated with cognitive impairment in children with SCA. We assessed 10 children with SCA (9 with normal MRI) using neuropsychological measures of executive function. Cognitive assessment was immediately followed by overnight polysomnography to record nocturnal hemoglobin oxygen saturation and sleep arousals. Decreases in hemoglobin oxygen saturation and/or increased sleep arousals were associated with reduced performance on cognitive assessment. Nocturnal hemoglobin oxygen desaturation and sleep fragmentation may be a contributing factor to executive dysfunction in SCA.</p
Lung clearance index may detect early peripheral lung disease in sickle cell anemia
No full textRationale: chronic lung injury is common in sickle cell anemia (SCA) and worsens outcomes. Sensitive lung function tests might predict reversible disease that might benefit from therapeutic interventions. Objectives: to evaluate whether lung clearance index (LCI) (measuring global ventilation inhomogeneity), intraacinar ventilation inhomogeneity (S acin), and conductive ventilation inhomogeneity (S cond) are more frequently abnormal than lung volumes in young people with SCA. Methods: nitrogen multiple-breath washout, spirometry, and body plethysmography were cross-sectionally evaluated at steady state in subjects with SCA (hemoglobin SS) and healthy control subjects aged 8–21 years from London, United Kingdom. Results: thirty-five patients (51% boys, mean 6 SD age, 16.4 6 3.5 yr) and 31 control subjects (48% boys; 16.2 6 3.2 yr) were tested. There were significant differences between the study and control groups in mean LCI (mean difference, 0.42 units; 95% confidence interval [CI], 0.22 to 0.63; P = 0.0001), S acin (mean difference, 0.014 units; 95% CI, 0.001 to 0.026; P = 0.04), forced expiratory volume in 1 second (FEV 1) (mean difference, 20.79 z-scores; 95% CI, 21.28 to 20.30; P = 0.002), forced vital capacity (FVC) (mean difference, 20.80 z-scores; 95% CI, 21.28 to 20.31, P = 0.002), and total lung capacity (mean difference, 20.76 z-scores; 95% CI, 21.25 to 20.29, P = 0.002), but not in S cond and FEV 1-to-FVC ratio. Whereas 29% (10 of 35) of patients had LCI . 95th percentile of control subjects, 23% (8 of 35) had abnormal FEV 1 (,5th percentile of the reference population). Conclusions: LCI detected slightly more abnormalities than lung volumes in young people with SCA. Significant differences from control subjects in LCI and S acin but not in S cond and FEV 1to-FVC ratio suggest that the lung function changes were most likely owing to patchy peripheral lung disease
Comparison of Lung Function in Healthy Nigerian Children Living in Nigeria and in the United Kingdom
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Limited access to transcranial Doppler screening and stroke prevention for children with sickle cell disease in Europe: Results of a multinational EuroBloodNet survey
Full text linkBackgroundEnsuring equitable access to adequate standard of care for patients with rare hematological disease is one of the aims of the European Reference Network (ERN) EuroBloodNet. Stroke is one of the most devastating complications for children with sickle cell disease (SCD). For effective prevention of stroke risk, annual transcranial Doppler (TCD) according to a defined protocol is recommended for patients aged 2-16 years, with red blood cell transfusion therapy for those at risk. There is no information regarding screening for stroke risk and stroke prevention programs in Europe.MethodsSeven SCD experts of five healthcare providers (HCPs) of ERN EuroBloodNet developed an online survey to assess the access to TCD screening and stroke prevention programs for children with SCD in Europe.ResultsEighty-one experts in 77 HCPs from 16 European countries responded to 16 online questions. Thirty-two of 77 (51%) HCPs were EuroBloodNet reference centers, and 36% physicians reported not having a dedicated TCD/TCD imaging service for children with SCD. Only 30% of physicians provided estimates that all their patients received annual TCD according to the standard protocol due to lack of trained staff (43%), lack of TCD instruments (11%), refusal of patients due to logistical difficulties (22%), and lack of funds for dedicated staff or equipment (11%).ConclusionsThis multinational European survey provides the first comprehensive picture of access to TCD screening and stroke prevention in European countries. Identifying the potential underlying causes of the lack of effective standardized screening, this survey also addresses possible dedicated actions to cover these needs
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