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Find video protocols related to scientific articles indexed in Pubmed.
Health-related quality of life in children with sickle cell anemia: Impact of blood transfusion therapy.
Am. J. Hematol.
PUBLISHED: 09-16-2014
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The completion of the Multicenter Silent Infarct Transfusion Trial demonstrated that children with pre-existing silent cerebral infarct and sickle cell anemia (SCA) who received regular blood transfusion therapy had a 58% relative risk reduction of infarct recurrence when compared to observation. However, the total benefit of blood transfusion therapy, as assessed by the parents, was not measured against the burden of monthly blood transfusion therapy. In this planned ancillary study, we tested the hypothesis that a patient centered outcome, health-related quality of life (HRQL), would be greater in participants randomly assigned to the blood transfusion therapy group than the observation group. A total of 89% (175 of 196) of the randomly allocated participants had evaluable entry and exit HRQL evaluations. The increase in Change in Health, measured as the child's health being better, was significantly greater for the transfusion group than the observation group (Difference Estimate = -0.54, p ? 0.001). This study provides the first evidence that children with SCA who received regular blood transfusion therapy felt better and had better overall HRQL than those who did not receive transfusion therapy.
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The Use of Neuropathic Pain Drugs in Children With Sickle Cell Disease is Associated With Older Age, Female Sex, and Longer Length of Hospital Stay.
J. Pediatr. Hematol. Oncol.
PUBLISHED: 09-16-2014
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Although neuropathic pain is increasingly recognized in sickle cell disease (SCD), it is unknown how neuropathic pain drugs are used in children with SCD. Thus, we investigated use of these drugs and hypothesized older age and female sex are associated with increased neuropathic drug use and the use of these drugs is associated with longer length of stay. We analyzed the Pediatric Health Information System (2004 to 2009) including all inpatient visits aged 0 to 18 years with any SCD-related (all genotypes) discharge diagnosis. To limit confounding we excluded psychiatric and seizure visits. Antiepileptics, tricyclic antidepressants, and selective serotonin reuptake inhibitors were drugs of interest. Generalized Estimating Equations determined the impact of age and sex on neuropathic drug use and the impact of neuropathic drug use on length of stay. We analyzed 53,557 visits; 2.9% received?1 neuropathic drugs. The odds of receiving a neuropathic drug increased significantly with age (reference group, 0 to 4 y: 5 to 10, odds ratio [OR], 5.7; 11 to 14: OR, 12.5; 15 to 18: OR, 22.8; all P<0.0001] and female sex (OR, 1.5; P=0.001). Neuropathic drug use was associated with longer length of stay (risk ratio, 8.3; P<0.0001). Neuropathic drug use in children with SCD was associated with older age, female sex, and longer length of stay.
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Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia.
N. Engl. J. Med.
PUBLISHED: 08-21-2014
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Silent cerebral infarcts are the most common neurologic injury in children with sickle cell anemia and are associated with the recurrence of an infarct (stroke or silent cerebral infarct). We tested the hypothesis that the incidence of the recurrence of an infarct would be lower among children who underwent regular blood-transfusion therapy than among those who received standard care.
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Silent cerebral infarction, income, and grade retention among students with sickle cell anemia.
Am. J. Hematol.
PUBLISHED: 07-08-2014
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Children with sickle cell anemia have a higher-than-expected prevalence of poor educational attainment. We test two key hypotheses about educational attainment among students with sickle cell anemia, as measured by grade retention and use of special education services: (1) lower household per capita income is associated with lower educational attainment; (2) the presence of a silent cerebral infarct is associated with lower educational attainment. We conducted a multicenter, cross-sectional study of cases from 22 U.S. sites included in the Silent Infarct Transfusion Trial. During screening, parents completed a questionnaire that included sociodemographic information and details of their child's academic status. Of 835 students, 670 were evaluable; 536 had data on all covariates and were used for analysis. The students' mean age was 9.4 years (range: 5-15) with 52.2% male; 17.5% of students were retained one grade level and 18.3% received special education services. A multiple variable logistic regression model identified that lower household per capita income (odds ratio [OR] of quartile 1 = 6.36, OR of quartile 2 = 4.7, OR of quartile 3 = 3.87; P = 0.001 for linear trend), age (OR = 1.3; P < 0.001), and male gender (OR, 2.2; P = 0.001) were associated with grade retention; silent cerebral infarct (P = 0.31) and painful episodes (P = 0.60) were not. Among students with sickle cell anemia, household per capita income is associated with grade retention, whereas the presence of a silent cerebral infarct is not. Future educational interventions will need to address both the medical and socioeconomic issues that affect students with sickle cell anemia.
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Clinically Meaningful Interpretation of Pediatric Health-related Quality of Life in Sickle Cell Disease.
J. Pediatr. Hematol. Oncol.
PUBLISHED: 06-20-2014
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Health-related quality of life (HRQL) measures provide information about disease assessment; however, health care providers may be reluctant to use HRQL assessments as scores can be difficult to interpret. We sought to identify levels for impaired pain-related HRQL in children with sickle cell disease (SCD). Children (n=251) completed the PedsQL Generic Core Scales and PedsQL SCD Module in a multisite study. Using children's item scores on the Pain and Hurt and Pain Impact scales of the PedsQL SCD Module, High, Intermediate, and Low functioning groups were created. We compared functioning groups with the Pain and Hurt and Pain Impact scale scores to determine levels representing high and low HRQL. These scores were compared with disease severity and the PedsQL Generic Core Scales. Scores of ?60 on the PedsQL SCD Pain and Hurt and Pain Impact scales were associated with severe disease and met requirements for impaired functioning on the PedsQL Generic Core Scales. Scores of ?81 on the Pain and Hurt and the Pain Impact scales can be considered consistent with good HRQL in those domains in SCD. Alternately, scores of ?60 are cause for concern and suggest areas of HRQL impairment in SCD.
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Ambulatory quality indicators to prevent infection in sickle cell disease.
Am. J. Hematol.
PUBLISHED: 04-30-2014
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The purpose of this study was to identify rates of adherence for three outpatient quality indicators noted by Wang et al. (Pediatrics 2011;128:484–493): (1) influenza vaccine, (2) pneumococcal immunizations, and (3)penicillin prophylaxis in patients with sickle cell disease (SCD) in a Medicaid sample. These variables were chosen based on Wang and colleagues' suggestion that these variables are important for the assessment of the quality of care of children with SCD. We hypothesized that the overall rate of adherence would be poor with adults having worse rates of adherence than children. We conducted a retrospective cohort study using the Wisconsin State Medicaid database over a 5-year period to assess the preventative medication adherence of individuals with SCD. Preventative medication variables in this study included influenza vaccination,pneumococcal immunizations (PCV7, PPV23), and penicillin prophylaxis. As predicted, the 2003–2007 Wisconsin State Medicaid database showed patients with SCD had low adherence in terms of recommended influenza vaccinations (21.58% adherent), PPV23 pneumococcal immunizations (43.47% adherent), and penicillin prophylaxis (18.18% adherent). Pneumococcal immunizations for PCV7 were higher than expected (77.27%adherent). Although children tended to adhere to recommended preventative medications more than adults,overall adherence was low. Although we cannot explain why adherence is low, it is likely due to multiple factors at the patient- and provider-level. We encourage patients and providers to create a partnership to meet adherence recommendations, and we describe our strategies for increasing adherence.
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Population pharmacokinetics of hydroxyurea for children and adolescents with sickle cell disease.
J Clin Pharmacol
PUBLISHED: 03-31-2014
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The objective of this study was to develop a population pharmacokinetic (PK) model sufficient to describe hydroxyurea (HU) concentrations in serum and urine following oral drug administration in pediatric patients with sickle cell disease. Additionally, the measured hydroxyurea concentrations for particular sampling time were correlated with exposure measures (AUC) to find the most predictive relationship. Hydroxyurea concentrations were determined in 21 subjects. Using a population nonlinear mixed-effect modeling, the HU PK was best described by a one-compartment model with two elimination pathways (metabolic and renal) and a transit compartment absorption. The typical mean absorption time was 0.222?hour. The typical apparent volume of distribution was 21.8?L and the apparent systemic clearance was 6.88?L/h for an average weight patient of 30.7?kg. The 50% of the HU dose was renally excreted. Linear correlations were apparent between the plasma HU concentration at 1, 1.5, 2, 4, and 6?hours post-dose and AUC with the most significant (R(2) ?=?0.71) observed at 1.5?hours. A population PK model was successful in describing HU disposition in plasma and urine. Data from the model also demonstrated that HU plasma concentrations at 1.5?hours after an oral dose of the drug were highly predictive of systemic drug exposure.
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The Proportion of Potentially Preventable Emergency Department Visits by Patients With Sickle Cell Disease.
J. Pediatr. Hematol. Oncol.
PUBLISHED: 02-13-2014
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Emergency department (ED) visits by children with sickle cell disease (SCD) are often classified as urgent based on resource utilization. This classification may not accurately reflect the potentially preventable nature of SCD visits. We sought to determine the proportion of SCD crisis-related pediatric ED visits that are possibly preventable.
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Intravenous magnesium for pediatric sickle cell vaso-occlusive crisis: methodological issues of a randomized controlled trial.
Pediatr Blood Cancer
PUBLISHED: 01-17-2014
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Multiple recent Sickle Cell Disease studies have been terminated due to poor enrollment. We developed methods to overcome past barriers and utilized these to study the efficacy and safety of intravenous magnesium for vaso-occlusive crisis (VOC). We describe the methods of the Intravenous Magnesium in Sickle Vaso-occlusive Crisis (MAGiC) trial and discuss methods used to overcome past barriers. MAGiC was a multi-center randomized double-blind placebo-controlled trial of intravenous magnesium versus normal saline for treatment of VOC. The study was a collaboration between Pediatric Hematologists and Emergency Physicians in the Pediatric Emergency Care Applied Research Network (PECARN). Eligible patients were randomized within 12 hours of receiving intravenous opioids in the Emergency Department (ED) and administered study medication every 8 hours. The primary outcome was hospital length of stay. Associated plasma studies elucidated magnesium's mechanism of action and the pathophysiology of VOC. Health-related quality of life was measured. Site-, protocol-, and patient-related barriers from prior studies were identified and addressed. Limited study staff availability, lack of collaboration with the ED, and difficulty obtaining consent were previously identified barriers. Leveraging PECARN resources, forging close collaborations between Sickle Cell Centers and EDs of participating sites, and approaching eligible patients for prior consent helped overcome these barriers. Participation in the PECARN network and establishment of collaborative arrangements between Sickle Cell Centers and their affiliated EDs are major innovative features of the MAGiC study that allowed improved subject capture. These methods could serve as a model for future studies of VOCs.
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Neuropathic pain in patients with sickle cell disease.
Pediatr Blood Cancer
PUBLISHED: 08-08-2013
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Despite the suggestion of a neuropathic component to sickle cell disease (SCD) pain, there are minimal data on the systematic assessment of neuropathic pain in patients with SCD. Neuropathic pain is defined as pain primarily initiated by dysfunction of the peripheral or central nervous system.
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Vitamin B12 deficiency: The great masquerader.
Pediatr Blood Cancer
PUBLISHED: 07-30-2013
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Vitamin B12 deficiency is rare in children, with nonspecific symptoms including failure to thrive, vomiting, anorexia, and neurologic changes with or without hematologic disturbances. The neuropathy can be severe and irreversible. We report four cases of children with B12 deficiency secondary to adult type pernicious anemia, a presumed transport protein abnormality, and a metabolic defect. All demonstrated neurologic compromise that improved after initiation of B12 therapy. Hematologic manifestations may be preceded by constitutional, gastrointestinal, or neurologic changes, and must raise concern for B12 deficiency. Therapy should be initiated promptly in this setting to prevent irreversible neuropathy. Pediatr Blood Cancer 2013;9999:XX-XX. © 2013 Wiley Periodicals, Inc.
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Academic attainment findings in children with sickle cell disease.
J Sch Health
PUBLISHED: 07-10-2013
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Children with sickle cell disease (SCD) demonstrate deficits in cognitive and academic functioning. This study compared the academic attainment of children with SCD relative to national, state, and local school district rates for African American students.
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PedsQL™ Multidimensional Fatigue Scale in sickle cell disease: feasibility, reliability, and validity.
Pediatr Blood Cancer
PUBLISHED: 03-27-2013
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Sickle cell disease (SCD) is an inherited blood disorder characterized by a chronic hemolytic anemia that can contribute to fatigue and global cognitive impairment in patients. The study objective was to report on the feasibility, reliability, and validity of the PedsQL™ Multidimensional Fatigue Scale in SCD for pediatric patient self-report ages 5-18 years and parent proxy-report for ages 2-18 years.
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Clinically meaningful measurement of pain in children with sickle cell disease.
Pediatr Blood Cancer
PUBLISHED: 03-15-2013
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Limited understanding of the interpretability of patient-reported pain scores may impact pain management. The current study assessed the minimal clinically significant improvement in pain and pain scores signifying patient-reported need for medication and treatment satisfaction in patients with sickle cell disease (SCD).
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Severe thrombocytopenia in a child secondary to passive platelet antibody transfer from a plasma transfusion.
J. Pediatr. Hematol. Oncol.
PUBLISHED: 03-06-2013
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Although rare, passive transfer of platelet antibodies through blood products can result in thrombocytopenia, acute transfusion reactions, and death. We report a case of severe alloimmune thrombocytopenia from a plasma transfusion. A postliver transplant patient with a normal platelet count received fresh frozen plasma before liver biopsy. Postbiopsy, she developed cardiorespiratory distress, petechiae, and severe thrombocytopenia (platelet count 2000/?L). Her platelet count recovered to normal after 1 week. This diagnosis should be considered whenever an unexpected drop in the platelet count occurs after a plasma-rich transfusion. Conservative transfusion practices and more targeted donor screening may prevent similar events.
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PedsQL™ sickle cell disease module: feasibility, reliability, and validity.
Pediatr Blood Cancer
PUBLISHED: 01-15-2013
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Sickle cell disease (SCD) is an inherited chronic disease that is characterized by complications such as recurrent painful vaso-occlusive events that require frequent hospitalizations and contribute to early mortality. The objective of the study was to report on the initial measurement properties of the new PedsQL™ SCD Module for pediatric patient self-report ages 5-18 years and parent proxy-report for ages 2-18 years.
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Improving communication between doctors and parents after newborn screening.
WMJ
PUBLISHED: 12-15-2011
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Newborn screening (NBS) enables early treatment, and some consider it a natural vehicle for genetic screening. Bioethicists argue for caution since families of infants with carrier status can develop psychosocial complications. This paper describes the methods and feasibility of Wisconsins statewide project for quality improvement of communication and psychosocial outcomes after NBS.
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Associated risk factors for silent cerebral infarcts in sickle cell anemia: low baseline hemoglobin, sex, and relative high systolic blood pressure.
Blood
PUBLISHED: 11-17-2011
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The most common form of neurologic injury in sickle cell anemia (SCA) is silent cerebral infarction (SCI). In the Silent Cerebral Infarct Multi-Center Clinical Trial, we sought to identify risk factors associated with SCI. In this cross-sectional study, we evaluated the clinical history and baseline laboratory values and performed magnetic resonance imaging of the brain in participants with SCA (HbSS or HbS?° thalassemia) between the ages of 5 and 15 years with no history of overt stroke or seizures. Neuroradiology and neurology committees adjudicated the presence of SCI. SCIs were diagnosed in 30.8% (251 of 814) participants who completed all evaluations and had valid data on all prespecified demographic and clinical covariates. The mean age of the participants was 9.1 years, with 413 males (50.7%). In a multivariable logistic regression analysis, lower baseline hemoglobin concentration (P < .001), higher baseline systolic blood pressure (P = .018), and male sex (P = .030) were statistically significantly associated with an increased risk of an SCI. Hemoglobin concentration and systolic blood pressure are risk factors for SCI in children with SCA and may be therapeutic targets for decreasing the risk of SCI. This study is registered at www.clinicaltrials.gov as #NCT00072761.
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Development of the PedsQL™ Sickle Cell Disease Module items: qualitative methods.
Qual Life Res
PUBLISHED: 05-19-2011
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The objective of this qualitative study was to develop the items and support the content validity of the PedsQL™ Sickle Cell Disease Module for pediatric patients with sickle cell disease (SCD).
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Differences in health-related quality of life in children with sickle cell disease receiving hydroxyurea.
J. Pediatr. Hematol. Oncol.
PUBLISHED: 04-26-2011
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Hydroxyurea is a safe and efficacious medication for children with sickle cell disease (SCD). Our objective was to compare health-related quality of life (HRQL) between children taking hydroxyurea and those not taking hydroxyurea. We conducted a retrospective cohort study of children with SCD who had completed the PedsQL 4.0 at Duke University Medical Center or the Midwest Sickle Cell Center. Our primary outcome was HRQL in children receiving hydroxyurea therapy compared with those not receiving hydroxyurea. One hundred and ninety-one children with SCD were included in the study. Children in the hydroxyurea group had higher self-reported Total PedsQL median scores than children in the no hydroxyurea group (P=0.04). Child self-reported physical functioning scores were significantly higher for children in the hydroxyurea group (P=0.01). In conclusion, children with SCD who received hydroxyurea therapy reported better overall HRQL and better physical HRQL than children who did not receive this therapy despite disease severity. Further research assessing the impact of hydroxyurea therapy on HRQL, such as prospective assessment over time, would aid in our understanding of the effectiveness of hydroxyurea for individual children. Ultimately, this may aid in decreasing the barriers to the use of hydroxyurea.
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Outpatient follow-up and rehospitalizations for sickle cell disease patients.
Pediatr Blood Cancer
PUBLISHED: 03-02-2011
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Rehospitalization rates are increasingly used as quality indicators for a variety of illnesses, including sickle cell disease. While one small, single center study suggested outpatient follow-up with a pediatric hematologist was associated with fewer rehospitalizations, no study has examined the effect of post-discharge outpatient follow-up on rehospitalization rates across ages and beyond a single site.
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Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease.
Blood
PUBLISHED: 10-12-2010
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Children with sickle cell disease (SCD) and strokes receive blood transfusion therapy for secondary stroke prevention; despite this, approximately 20% experience second overt strokes. Given this rate of second overt strokes and the clinical significance of silent cerebral infarcts, we tested the hypothesis that silent cerebral infarcts occur among children with SCD being transfused for secondary stroke prevention. A prospective cohort enrolled children with SCD and overt strokes at 7 academic centers. Magnetic resonance imaging and magnetic resonance angiography of the brain were scheduled approximately every 1 to 2 years; studies were reviewed by a panel of neuroradiologists. Eligibility criteria included regularly scheduled blood transfusion therapy. Forty children were included; mean pretransfusion hemoglobin S concentration was 29%. Progressive cerebral infarcts occurred in 45% (18 of 40 children) while receiving chronic blood transfusion therapy; 7 had second overt strokes and 11 had new silent cerebral infarcts. Worsening cerebral vasculopathy was associated with new cerebral infarction (overt or silent; relative risk = 12.7; 95% confidence interval, 2.65-60.5, P = .001). Children with SCD and overt strokes receiving regular blood transfusion therapy experience silent cerebral infarcts at a higher rate than previously recognized. Additional therapies are needed for secondary stroke prevention in children with SCD.
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The effect of parental mental health on proxy reports of health-related quality of life in children with sickle cell disease.
Pediatr Blood Cancer
PUBLISHED: 07-01-2010
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The objectives of this study were to evaluate factors that influence agreement between parent-proxy and child self-report of health-related quality of life (HRQL) in sickle cell disease. We hypothesized that the mental health of the parent, parental HRQL and child characteristics would affect agreement.
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The impact of a multidisciplinary pain management model on sickle cell disease pain hospitalizations.
Pediatr Blood Cancer
PUBLISHED: 05-24-2010
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Sickle cell disease (SCD) pain is acute or chronic, leads to school absenteeism, impaired health-related quality of life and early mortality. Given that little is known about children with SCD and chronic pain, we (1) described characteristics of these children and (2) evaluated the impact of a multidisciplinary pain management model on health care utilization.
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Acute care utilization and rehospitalizations for sickle cell disease.
JAMA
PUBLISHED: 04-08-2010
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Published rates of health care utilization and rehospitalization by people with sickle cell disease have had limited generalizability and are not population based.
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Impact of family income and sickle cell disease on the health-related quality of life of children.
Qual Life Res
PUBLISHED: 08-29-2009
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The objective of this study was to determine the impact of family income and sickle cell disease on the health-related quality of life (HRQL) of children.
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Postdischarge pain, functional limitations and impact on caregivers of children with sickle cell disease treated for painful events.
Br. J. Haematol.
PUBLISHED: 05-15-2009
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This study aimed to describe the outcomes of children with sickle cell disease (SCD) after discharge from medical care for vaso-occlusive painful events and to test the hypothesis that older age, longer length of hospital stay, and a history of frequent vaso-occlusive painful events will be associated with poor outcomes. Children aged 2-18 years with SCD treated in the emergency department or inpatient unit for a painful event were contacted after discharge to assess: days of pain, days of functional limitations for the child, and days of work/school absenteeism for the caregiver. Descriptive statistics were applied and multivariate logistic regression examined the association between the predictors and outcomes. Fifty-eight children were enrolled (mean age 10.8 +/- 4.8 years, 53.5% female). Postdischarge, 46.5% of children reported three or more days of pain, 54.3% had two or more days of functional limitations, and 24.3% of caregivers missed two or more days of work/school. Children with three or more prior painful events had increased odds of a poor outcome postdischarge (OR 1.79; 95% CI = 1.026, 3.096). In conclusion, acute vaso-occlusive painful events impact the lives of children with SCD and their caregivers, even after discharge to home.
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A psychometric evaluation of the PedsQL Family Impact Module in parents of children with sickle cell disease.
Health Qual Life Outcomes
PUBLISHED: 04-16-2009
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Caring for a child with a chronic condition, such as sickle cell disease, can have a significant impact on parents and families. In order to provide comprehensive care and support to these families, psychometrically sound instruments are needed as an initial step in measuring the impact of chronic diseases on parents and families. We sought to evaluate the psychometric properties of the PedsQL Family Impact Module in populations of children with and without sickle cell disease. In addition, we sought to determine the correlation between parents well being and their proxy report of their childs health-related quality of life (HRQL).
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Dissatisfaction with hospital care for children with sickle cell disease not due only to race and chronic disease.
Pediatr Blood Cancer
PUBLISHED: 04-08-2009
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A previous study reported increased dissatisfaction with hospital care for children with sickle cell disease (SCD); however, its small size excluded determining whether race and chronic disease explained the difference.
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What is the evidence that hydroxyurea improves health-related quality of life in patients with sickle cell disease?
Hematology Am Soc Hematol Educ Program
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A 10-year-old male patient with homozygous sickle cell disease presents for a follow-up clinic visit after a recent hospitalization for a painful vasoocclusive event. His parents mention that in the past year he has had 4 hospitalizations for vasoocclusive events, 2 of which were complicated by the development of acute chest syndrome that resulted in transfer to the intensive care unit. He has missed many school days and may be retained a grade this year. He feels particularly sad about missing the school field trip that occurred during his last hospitalization. He also reports that he is not able to keep up with his friends when participating in physical activities at school. The childs parents are worried that he may be depressed. You as the provider discuss the option of hydroxyurea therapy. His parents ask if hydroxyurea would improve his overall well-being and functioning.
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Health-related quality of life in patients with hemoglobinopathies.
Hematology Am Soc Hematol Educ Program
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The use of patient-reported outcomes to measure the health and well-being of patients from their perspective has become an acceptable method to determine the impact of a disease and its treatment on patients. In patients with hemoglobinopathies, prior work has demonstrated that patients experience significant impairment in health-related quality of life (HRQL, a type of patient-reported outcome). This work has provided a better understanding of the burden that these patients experience and the factors that are associated with worse HRQL. The recent development of disease-specific HRQL instruments in sickle cell disease heralds new opportunities to explore the impact of the disease and its treatment on patients. The standards necessary to incorporate the measurement of HRQL into clinical trials are now well outlined by regulatory agencies. Measuring HRQL within a clinical practice setting and outside of the healthcare setting while the patient is at home are now possible and present new opportunities to understand the health and well-being of patients with hemoglobinopathies.
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Mental health disorders influence admission rates for pain in children with sickle cell disease.
Pediatr Blood Cancer
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Patients with sickle cell disease (SCD) experience a broad range of mental health disorders placing them at risk for more complicated hospitalizations for pain. The current study examined the impact of mental health disorders on admission rates and hospital length of stay (LOS) for vaso-occlusive pain events (VOE) in pediatric patients with SCD.
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Patients with sickle cell disease have increased sensitivity to cold and heat.
Am. J. Hematol.
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Sickle cell disease (SCD) pain is associated with colder temperatures and touch and described as "cold," "hot," and "shooting" suggesting hypersensitivity to tactile stimuli. Sickle mice exhibit hypersensitivity to thermal (cold, heat) and mechanical stimuli compared to controls. It is unknown whether humans experience this same hypersensitivity. Thus, we quantified thermal and mechanical sensitivity differences between patients with SCD and controls. Our primary hypothesis was that patients with SCD will exhibit hypersensitivity to thermal and mechanical stimuli compared to race-matched controls. Our secondary hypothesis was this hypersensitivity will be associated with older and female subjects, and with frequent pain and hemolysis in patients with SCD. A total of 55 patients and 57 controls ?7 years completed quantitative sensory testing. Patients with SCD detected the sensation of cold and warm temperatures sooner as seen in their significantly lower median cold and heat detection thresholds [29.5°C vs. 28.6°C, P = 0.012 and 34.5°C vs. 35.3°C, P = 0.02] and experienced cold and heat pain sooner as seen in their significantly lower median cold and heat pain thresholds [21.1°C vs. 14.8°C, P = 0.01 and 42.7°C vs. 45.2°C, P = 0.04]. We found no mechanical threshold differences. Older age was associated with lower cold, heat, and mechanical pain thresholds in both groups. No association with pain, gender, or hemolysis was found. Patients with SCD exhibit hypersensitivity to thermal stimuli suggesting peripheral or central sensitization may exist and could contribute to SCD pain.
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Health-related quality of life in sickle cell disease: past, present, and future.
Pediatr Blood Cancer
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Health-related quality of life (HRQL) is defined as the patients appraisal of how his/her well being and level of functioning, compared to the perceived ideal, are affected by individual health. The study of HRQL in children and adults with sickle cell disease (SCD) has begun to flourish. Given the devastating complications of the disease and other co-morbid factors patients experience that influence HRQL, it is increasingly important to understand HRQL. The focus of this critical review was to examine past and current research in HRQL in SCD where a validated instrument was used. In addition, future directions for HRQL in SCD are explored.
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Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).
Biol. Blood Marrow Transplant.
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The Sickle Cell Unrelated Donor Transplant Trial (SCURT trial) of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) is a phase II study of the toxicity and efficacy of unrelated donor hematopoietic cell transplantation in children with severe sickle cell disease (SCD) using a reduced-intensity conditioning regimen. Here we report the results for the cord blood cohort of this trial. Eight children with severe SCD underwent unrelated donor cord blood transplantation (CBT) following alemtuzumab, fludarabine, and melphalan. Cyclosporine or tacrolimus and mycophenolate mofetil were administered for graft-versus-host disease (GVHD) prophylaxis. Donor/recipient HLA match status was 6 of 6 (n = 1) or 5 of 6 (n = 7), based on low/intermediate-resolution molecular typing at HLA -A, -B, and high-resolution typing at -DRB1. Median recipient age was 13.7 years (range: 7.4-16.2 years), and median weight was 35.0 kg (range: 25.2-90.2 kg). The median pre-cryopreservation total nucleated cell dose was 6.4 × 10(7) /kg (range: 3.1-7.6), and the median postthaw infused CD34 cell dose was 1.5 × 10(5) /kg (range: 0.2-2.3). All patients achieved neutrophil recovery (absolute neutrophil count >500/mm(3)) by day 33 (median: 22 days). Three patients who engrafted had 100% donor cells by day 100, which was sustained, and 5 patients had autologous hematopoietic recovery. Six of 8 patients had a platelet recovery to >50,000/mm(3) by day 100. Two patients developed grade II acute GVHD. Of these, 1 developed extensive chronic GVHD and died of respiratory failure 14 months posttransplantation. With a median follow-up of 1.8 years (range: 1-2.6), 7 patients are alive with a 1-year survival of 100%, and 3 of 8 are alive without graft failure or disease recurrence. Based upon the high incidence of graft rejection after unrelated donor CBT, enrollment onto the cord blood arm of the SCURT trial was suspended. However, because this reduced-intensity regimen has demonstrated a favorable safety profile, this trial remains open to enrollment for unrelated marrow donor transplants. Novel approaches aimed at improving engraftment will be needed before unrelated CBT can be widely adopted for transplanting patients with severe SCD.
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