scholarly journals Relationship of Hydroxyurea Adherence to Depression and Fatigue Among Children and Adolescents with Sickle Cell Disease: A Longitudinal Cohort Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3104-3104
Author(s):  
Sherif M. Badawy ◽  
Kathryn King ◽  
Olivia E. Atherton ◽  
Daniel Mroczek ◽  
Alexis A. Thompson ◽  
...  
Keyword(s):  
Cohort Study ◽  
Sickle Cell Disease ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Research Funding ◽  
Self Report ◽  
Cell Disease ◽  
Laboratory Markers ◽  
Relationship Of ◽  
Over Time

Abstract Background: Sickle cell disease (SCD) is the most common genetic disorder in the United States, seen in 100,000 Americans. SCD complications include pain episodes, chronic anemia and long-term end organ damage, leading to significant impairment in health-related quality of life (HRQOL) across the lifespan. Hydroxyurea (HU) reduces morbidity and mortality, improves HRQOL and lowers healthcare utilization, yet adherence remains suboptimal. Limited evidence from cross-sectional studies demonstrates an association between lower HU adherence and worse HRQOL scores. Objective: To assess the longitudinal relationship of HU adherence to HRQOL domains, including fatigue and depression. We hypothesized that higher HU adherence over time would be associated with improvement in HRQOL domain scores, especially depression and fatigue. Methods: In this longitudinal cohort study (NCT04675645), patients were enrolled from the comprehensive sickle cell clinic at Lurie Children's Hospital of Chicago. Patients were eligible if they were ³8 years old, had SCD (any genotype), and on HU with a stable dose for ³2 months. Study assessments included PROMIS ® measures for depression and fatigue, self-report of adherence using visual analogue scale (VAS), and patient demographics. Assessments were completed at baseline and every 3 months with a total of 5 visits (0, 3, 6, 9 and 12 months). Laboratory markers of adherence collected from chart review, including fetal hemoglobin (HbF%) and mean corpuscular volume (MCV). We conducted bivariate correlations among demographic variables, adherence markers and HRQOL scores as well as among adherence variables (VAS, HbF, MCV) at each visit. We conducted different multilevel models (MLMs), fixed and random effects, to understand the extent to which between- and within-person variation in adherence was associated with HRQOL scores over the 12-month period. We report unstandardized betas (B) and 95% Confidence Intervals (CI) from the MLMs. Results: Twenty-three patients have been enrolled (96% HbSS, 65% females, 100% Black, median age 15 [range 9-22] years old). At baseline, participants had a median Hb level of 9.5 (IQR 8.3-10.3 g/dl) with a HbF of 16.4% (IQR 13.1-28.7%) and MCV of 106.5 fl (IQR 91.6-113.9 fl). Participants' MCV levels significantly correlated with HbF% and VAS at visit 1 (r=0.58, P <0.01; r=0.6, P <0.01), visit 2 (r=0.66, P <0.01; r=0.63, P <0.01), visit 4 (r=0.76, P <0.01; r=0.72, P <0.01) and visit 5 (r=0.71, P <0.01; r=0.59, P <0.05), respectively. Participants' VAS adherence levels significantly increased from visit 1 to visit 5 (median 72 [IQR 60-92] vs. 88 [IQR 75-95], P=0.04, respectively) along with significant improvement in their fatigue scores (median 52.8 [IQR 35.1-70.5] vs. 30.8 [IQR 13.2-48.4], P=0.001, respectively). Variation in fatigue and depression scores across the study period was due to between-person differences (38% and 71%, respectively) or within-person fluctuations (62% and 29%, respectively). Using fixed and random effect MLMs, between-person differences in HU adherence over 12 months using VAS and HbF% were significantly related to participants' reported depression (B -0.43, 95% CI -0.69 to -0.17, P <0.01; B -0.58, 95% CI -1 to -0.15, P <0.05, respectively) (Figure 1) and fatigue scores (B -0.42, 95% CI -0.68 to -0.16, P <0.01; B -0.43, 95% CI -0.78. to -0.06, P <0.05, respectively) (Figure 2). In contrast, we found no statistically significant effects of within-person variation in adherence, using VAS and HbF, on participants' reported fatigue and depression scores over 12 months, which could be due our small sample size. Conclusions: Children and adolescents who were more adherent to HU across the entire study period were less likely to experience fatigue and depression, compared to those who were less adherent. Participants' self-report and laboratory markers of adherence were significantly correlated across study visits. Within-person fluctuations in adherence were not associated with changes in fatigue and depression scores across the study period. Future multi-institutional studies with a larger sample size are needed to better understand the within-person effects of variation in HU adherence on HRQOL scores over time. Behavioral interventions, such as mHealth apps, that are focused on improving HU adherence among children and adolescents with SCD has the potential to improve HRQOL and other important health outcomes. Figure 1 Figure 1. Disclosures Badawy: Bluebird Bio Inc: Consultancy; Vertex Pharmaceuticals Inc: Consultancy; Sanofi Genzyme: Consultancy. Thompson: Biomarin: Research Funding; Baxalta: Research Funding; bluebird bio, Inc.: Consultancy, Research Funding; Celgene/BMS: Consultancy, Research Funding; CRISPR Therapeutics: Research Funding; Vertex: Research Funding; Editas: Research Funding; Graphite Bio: Research Funding; Novartis: Research Funding; Agios: Consultancy; Beam: Consultancy; Global Blood Therapeutics: Current equity holder in publicly-traded company. Cella: FACIT: Membership on an entity's Board of Directors or advisory committees.

scholarly journals Height-corrected low bone density associates with severe outcomes in sickle cell disease: SCCRIP cohort study results

2019 ◽  
Vol 3 (9) ◽  
pp. 1476-1488 ◽  
Author(s):  
Oyebimpe O. Adesina ◽  
James G. Gurney ◽  
Guolian Kang ◽  
Martha Villavicencio ◽  
Jason R. Hodges ◽  
...  
Keyword(s):  
Bone Mineral Density ◽  
African American ◽  
Cohort Study ◽  
Sickle Cell Disease ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Chronological Age ◽  
Cell Disease ◽  
Mineral Density ◽  
Z Scores

AbstractLow bone mineral density (BMD) disproportionately affects people with sickle cell disease (SCD). Growth faltering is common in SCD, but most BMD studies in pediatric SCD cohorts fail to adjust for short stature. We examined low BMD prevalence in 6- to 18-year-olds enrolled in the Sickle Cell Clinical Research and Intervention Program (SCCRIP), an ongoing multicenter life span SCD cohort study initiated in 2014. We calculated areal BMD for chronological age and height-adjusted areal BMD (Ht-aBMD) z scores for the SCCRIP cohort, using reference data from healthy African American children and adolescents enrolled in the Bone Mineral Density in Childhood Study. We defined low BMD as Ht-aBMD z scores less than or equal to –2 and evaluated its associations with demographic and clinical characteristics by using logistic regression analyses. Of the 306 children and adolescents in our study cohort (mean age, 12.5 years; 50% female; 64% HbSS/Sβ0-thalassemia genotype; 99% African American), 31% had low areal BMD for chronological age z scores and 18% had low Ht-aBMD z scores. In multivariate analyses, low Ht-aBMD z scores associated with adolescence (odds ratio [OR], 7.7; 95% confidence interval [CI], 1.94-30.20), hip osteonecrosis (OR, 4.0; 95% CI, 1.02-15.63), chronic pain (OR, 10.4; 95% CI, 1.51-71.24), and hemoglobin (OR, 0.74; 95% CI, 0.57-0.96). Despite adjusting for height, nearly 20% of this pediatric SCD cohort still had very low BMD. As the SCCRIP cohort matures, we plan to prospectively evaluate the longitudinal relationship between Ht-aBMD z scores and markers of SCD severity and morbidity.

scholarly journals Improved survival of children and adolescents with sickle cell disease

Blood ◽  
2010 ◽  
Vol 115 (17) ◽  
pp. 3447-3452 ◽  
Author(s):  
Charles T. Quinn ◽  
Zora R. Rogers ◽  
Timothy L. McCavit ◽  
George R. Buchanan
Keyword(s):  
Sickle Cell Disease ◽  
Young Children ◽  
Medical Care ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Care Quality ◽  
Transition To Adult Care ◽  
Cell Disease ◽  
Over Time

Abstract The survival of young children with sickle cell disease (SCD) has improved, but less is known about older children and adolescents. We studied the Dallas Newborn Cohort (DNC) to estimate contemporary 18-year survival for newborns with SCD and document changes in the causes and ages of death over time. We also explored whether improvements in the quality of medical care were temporally associated with survival. The DNC now includes 940 subjects with 8857 patient-years of follow-up. Most children with sickle cell anemia (93.9%) and nearly all children with milder forms of SCD (98.4%) now live to become adults. The incidence of death and the pattern of mortality changed over the duration of the cohort. Sepsis is no longer the leading cause of death. All the recent deaths in the cohort occurred in patients 18 years or older, most shortly after the transition to adult care. Quality of care in the DNC has improved over time, with significantly more timely initial visits and preventive interventions for young children. In summary, most children with SCD now survive the childhood years, but young adults who transition to adult medical care are at high risk for early death.

Sickle Cell Disease Pain in Children and Adolescents: Change in Pain Frequency and Coping Strategies Over Time

1993 ◽  
Vol 18 (5) ◽  
pp. 621-637 ◽  
Author(s):  
Karen M. Gil ◽  
Robert J. Thompson ◽  
Barbara R. Keith ◽  
Mary Tota-Faucette ◽  
Stephanie Noll ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Coping Strategies ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Cell Disease ◽  
Pain Frequency ◽  
And Coping ◽  
Over Time

Episodic Patterns of High Emergency Department Utilization Among Sickle Cell Disease Patients

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 316-316
Author(s):  
Susan Paulukonis ◽  
Lisa Feuchtbaum ◽  
Elliott Vichinsky ◽  
Mary Hulihan
Keyword(s):  
Emergency Department ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Surveillance System ◽  
Research Funding ◽  
Cell Disease ◽  
Ed Visits ◽  
High Utilization ◽  
The Mean ◽  
Over Time

Abstract Background: High utilization of emergency department (ED) services among those with sickle cell disease (SCD) compared to the general population and compared to those with other chronic diseases is well documented in the literature. Some reports note that high utilization is episodic. Most analyses address the problem as a consistent one within patients, rather than consistent over time across the patient population but sporadic for patients. Reducing the high rate of ED utilization among patients with SCD requires an understanding of temporal patterns of ED utilization, the consistency of ED utilization over time by patients and the proportion of the population affected at any given time. Methods: CDC has developed the Sickle Cell Data Collection program (SCDC) to conduct state level surveillance in this disease, and to continue and improve upon work begun through the Registry and Surveillance System in Hemoglobinopathies (RuSH). Through SCDC, California has collected ED and hospitalization data for years 2005-2014 on 4,325 people with SCD. A period of high ED utilization among this cohort was defined as three or more ED encounters (either treat and release or admission to the hospital from the ED) for any diagnosis (not only SCD diagnoses) each fewer than 30 days from the prior visit. The start of an episode of high utilization is the date of the first ED encounter; the end is the date of the last eligible ED encounter. All cohort members were divided into categories of utilization using the proportion of time spent in periods of high utilization divided by the total time in cohort. Total time is cohort is defined as the length of time from the earliest appearance in the ED or hospital data 2005-2014 to the latest appearance. The five categories were defined as no episodes of high ED utilization, and approximate quartile groups for those with high ED utilization: 1.1 to 3.0%, 3.1-10.0%, 10.1% or greater. Age categories (pediatric is < 21 years, and adult is 21 years or older) are defined as patient age at close of study (end of 2014) or at death if prior. Patient ID beginning with P is a pediatric, A is adult in the figures. Results: There were 4,325 individuals with 27,694 person years in the cohort (mean 6.4 person years, median 7.6 person years). Sixty-three percent (n=2,715) of the cohort were aged 21 years and older. Forty-five percent, (n=1,955, 513 pediatric and 1,442 adults) had at least one episode of high utilization during the 10 year study for a total of 7,866 episodes of high utilization. Forty-three percent of patients with one or more high utilization episodes were male, and 63% were between the ages of 20 and 50. Nine percent of these high utilizing patients' total time in the cohort was made up of episodes of high utilization. The mean time span from start of episodes of high utilization to end of the episodes was 63.3 days, median 35 days; mean number of ED visits per episode was 9.0, median 4.0. Most episodes of high utilization were brief: 42.2% included just three visits, and 70.7% included five or fewer ED visits. Among these individuals with episodes of high utilization, the mean number of such episodes was 4.0 over the 10 years study period, and the median was 2.0. Most (76.4%) had five or fewer high utilization episodes, and 35.5% had just one (n = 693). Sample utilization patterns, including hospital admissions, are shown in Figure 1. Conclusions: We demonstrate that among individuals with SCD seen in a population-based, statewide surveillance system, periods of high ED utilization are common, but most SCD patients have only a limited number of short episodes of such utilization. We found that high ED utilization is episodic rather than consistent within individuals, and that while the range of time spent in episodes of high utilization varies, few patients are high utilizers of ED services over a long period of time. Statewide surveillance that follows individual patients over time and in different hospital settings and includes ED utilization (including visits not coded as being related to SCD), provides high quality public health information to inform clinicians and healthcare systems in their development of efforts to reduce ED utilization among those living with SCD. Figure 1 Figure 1. Disclosures Paulukonis: Pfizer: Research Funding; Biogen: Research Funding.

Population-Based Analysis Of High Hospital Utilization (HHU) Among Children and Adolescents With Sickle Cell Disease (SCD)

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 1740-1740
Author(s):  
Rodney S Theodore ◽  
Maa-Ohui Quarmyne ◽  
James Bost ◽  
Carlton Dampier ◽  
Peter A. Lane
Keyword(s):  
Health Care ◽  
Sickle Cell Disease ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Insurance Coverage ◽  
Population Based ◽  
Hospital Utilization ◽  
Cell Disease ◽  
Relationship Of ◽  
The Relationship

Abstract Background Sickle cell disease (SCD) is characterized by marked heterogeneity in clinical manifestations, severity and utilization of health care services. This heterogeneity is particularly evident with regards to utilization of inpatient services, which account for a large percentage of total health care costs and significant absence from school and work. Previous studies of utilization, based on analysis of administrative datasets, have been limited by inaccuracies in ICD-coding, inability to differentiate among SCD genotypes, and/or inability to capture individuals never hospitalized. Single center studies have been limited by potential disease severity referral bias, inability to capture service utilization at outside facilities, and relatively small numbers. Objectives We sought to identify children and adolescents with SCD with high hospital utilization (HHU) and to determine the relationship of HHU with age, SCD genotype, and insurance coverage. We also sought to examine trends over a three year period (2010-2012) and explore the relationship of any trends in hospital utilization over time. Methods The clinical database of the SCD Program at Children's Healthcare of Atlanta (CHOA) was used to identify all patients with SCD, ages ≥ 5 years, who received comprehensive outpatient, acute care, and inpatient services at three CHOA facilities in metro Atlanta during 2010-2012. SCD genotype was confirmed for each patient by review of hematologic and clinical data, including results of diagnostic hemoglobin electrophoresis. All outpatient clinic, emergency department, and inpatient utilization was captured as well as insurance (payer) status at each encounter. HHU was defined as ≥3 admissions during a calendar year. Data were analyzed for significance by Chi-square. The extent to which the data were population-based was determined from the Georgia Hospital Association database with includes DRG-level data for all inpatient hospitalizations in Georgia. Results Hospitalizations at CHOA accounted for 96.1% of all SCD admissions for children within the 28-county greater metro Atlanta area. During 2010-2012, 1348 individual children and adolescents ≥ 5yr of age with SCD received comprehensive care at CHOA (1076 in 2010, 1112 in 2011, 1159 in 2012): 928 (68.8%) had SS/S β°thal, 334 (24.8%) SC, 86 (6.4%) S β+thal. Of 1159 active patients in 2012, 441 (38.1%) were 5-9 yr of age, 419 (36.2%) 10-14 yr, 299 (25.8%) ≥ 15 yr; 474 (40.9%) had traditional medicaid (MC), 205 (17.7%) managed-care medicaid (MMC), 374 (32.3%) private payer (PP), and 105 (9.1%) multiple payers (MP). HHU (≥ 3 admissions/yr) decreased from 119 of 1076 (11.1%) in 2010 to 96 of 1112 (8.6%) in 2011 and 95 of 1159 (8.2%) in 2012 (p <.01). The Table shows percentage HHU for each year by age, genotype, and payer. Conclusions For children and adolescents with SCD, HHU decreased during the three year period. HHU was significantly associated with older age and with MC and MP insurance coverage during all three years. HHU was significantly associated with the SS/S β°thal genotype in 2010, but not in 2011-12. The extent to which the decline of HHU among patients with SS/S β°thal genotype was related to increasing use of hydroxyurea is being investigated. Disclosures: Off Label Use: Hydroxyurea is not FDA approved for use in children.

Retrospective Cohort Study Comparing the Effect of Obesity in Children with Sickle Cell Disease

PEDIATRICS ◽  
2016 ◽  
Vol 137 (Supplement 3) ◽  
pp. 264A-264A
Author(s):  
Parth J. Bhatt ◽  
Dinesh Singh ◽  
Akingbola Olubenga ◽  
Devraj Chavda ◽  
Achint Patel
Keyword(s):  
Cohort Study ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Cell Disease ◽  
Obesity In Children

Comprehensive Handbook of Childhood Cancer and Sickle Cell Disease

2006 ◽  
Keyword(s):  
Quality Of Life ◽  
Sickle Cell Disease ◽  
Childhood Cancer ◽  
Children And Adolescents ◽  
Peer Relationships ◽  
Sickle Cell ◽  
Cell Disease ◽  
Life Issues ◽  
Art Research

Over recent decades, tremendous advances in the prevention, medical treatment, and quality of life issues in children and adolescents surviving cancer have spawned a host of research on pediatric psychosocial oncology. This important volume fulfills the clear need for an up-to-date, comprehensive handbook for practitioners that delineates the most recent research in the field--the first of its kind in over a decade. Over 60 renowned authors have been assembled to provide a thorough presentation of the state-of-the art research and literature, with topics including: -Neuropsychological effects of chemotherapy and radiation therapy -Bone marrow transplantation -Important issues about quality of life during and following treatment -Collaborative research among child-focused psychologists -Standards of psychological care for children and adolescents -Stress and coping in the pediatric cancer experience -The role of family and peer relationships The Comprehensive Handbook of Childhood Cancer and Sickle Cell Disease represents both multidisciplinary and international efforts, an alliance between physicians and parents, and a combination of research and service. With a wealth of information of great interest to patients and their families, this volume will also be a welcome resource to the psychologists, psychiatrists, pediatricians, oncologists, nurses, and social workers who confront these issues as they help children and their families through the treatment, recovery, and grieving processes.

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