Association of hydroxyurea adherence with transcranial Doppler screenings in children with sickle cell disease.

BACKGROUND: National sickle cell disease (SCD) guidelines recommend oral hydroxyurea (HU) starting at 9 months of age, and annual transcranial Doppler (TCD) screenings to identify stroke risk in children aged 2-16 years. We examined prevalence and proportion of TCD screenings in North Carolina Medicaid enrollees to identify associations with sociodemographic factors and HU adherence over 3 years. STUDY DESIGN: We conducted a longitudinal study with children ages 2-16 years with SCD enrolled in NC Medicaid from years 2016-2019. Prevalence of TCD screening claims was calculated for 3 years, and proportion was calculated for 12, 24, and 36 months of Medicaid enrollment. Enrollee HU adherence was categorized using HU proportion of days covered. Multivariable Poisson regression assessed for TCD screening rates by HU adherence, controlling for age, sex, and rurality. RESULTS: The prevalence of annual TCD screening was between 39.5% and 40.1%. Of those with 12-month enrollment, 77.8% had no TCD claims, compared to 22.2% who had one or higher TCD claims. Inversely, in children with 36 months of enrollment, 36.7% had no TCD claims compared to 63.3% who had one or higher TCD claims. The proportion of children with two or higher TCD claims increased with longer enrollment (10.5% at 12 months, 33.7% at 24 months, and 52.6% at 36 months). Children with good HU adherence were 2.48 (p < .0001) times more likely to have TCD claims than children with poor HU adherence. CONCLUSION: While overall TCD screening prevalence was low, children with better HU adherence and longer Medicaid enrollment had more TCD screenings. Multilevel interventions are needed to engage healthcare providers and families to improve both evidence-based care and annual TCD screenings in children with SCD.

Associations between Medicaid enrollment and diagnosis stage and survival among pediatric cancer patients.

BACKGROUND: Medicaid-associated disparities in childhood and adolescent (pediatric) cancer diagnosis stage and survival have been reported. However, a key limitation of prior studies is the assessment of health insurance at a single time point. To evaluate Medicaid-associated disparities more robustly, we used Surveillance, Epidemiology, and End Results (SEER)-Medicaid linked data to examine diagnosis stage and survival disparities in those (i) Medicaid-enrolled and (ii) with discontinuous and continuous Medicaid enrollment. METHODS: SEER-Medicaid linked data from 2006 to 2013 were obtained on cases diagnosed from 0 to 19 years. Medicaid enrollment was classified as enrolled versus not enrolled, with further classifications as continuous when enrolled 6 months before through 6 months after diagnosis, and discontinuous when not enrolled continuously for this period. We used multinomial logistic and Cox proportional hazards regression models to determine associations between enrollment measures, diagnosis stage, and cancer death adjusted for covariates. RESULTS: Among 21,502 cases, a higher odds of distant stage diagnoses were observed in association with Medicaid enrollment (odds ratio [OR] = 1.56, 95% confidence interval [CI]: 1.48-1.65), with the highest odds for discontinuous enrollment (OR = 2.0, 95% CI: 1.86-2.15). Among 30,654 cases, any Medicaid enrollment, continuous enrollment, and discontinuous enrollment were associated with 1.68 (95% CI: 1.35-2.10), 1.66 (95% CI: 1.35-2.05), and 1.89 (95% CI: 1.54-2.33) times higher hazards of cancer death versus no enrollment, respectively. CONCLUSIONS: Medicaid enrollment, particularly discontinuous enrollment, is associated with a higher  distant stage diagnosis odds and risk of death. This study supports the critical need for consistent health insurance coverage in children and adolescents.

The Moderating Role of Motivation in the Real-Time Associations of Fatigue, Cognitive Complaints, and Pain With Depressed Mood Among Stroke Survivors: An Ecological Momentary Assessment Study.

OBJECTIVE: Stroke symptoms fluctuate during the day as stroke survivors participate in daily activities. Understanding the real-time associations among stroke symptoms and depressed mood, as well as the role of motivation for daily activities, informs, and post-stroke symptom management in the context of everyday living. This study aimed to (1) investigate the real-time associations of fatigue, cognitive complaints, and pain with depressed mood and (2) examine the role of motivation for daily activity participation as a potential moderator of these associations in stroke survivors. DESIGN: A prospective cohort study involving 7 days of ecological momentary assessment (EMA), during which participants completed 8 EMA surveys per day. Multilevel modeling was used to analyze data. SETTING: Community. PARTICIPANTS: Forty community-dwelling stroke survivors (N=40). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: EMA measures of depressed mood, stroke symptoms (physical and mental fatigue, cognitive complaints, and pain), and motivation (autonomous motivation, controlled motivation). RESULTS: Higher levels of within- and between-person physical fatigue, mental fatigue, cognitive complaints, and pain were momentarily associated with greater depressed mood (Ps<.001). Within-person autonomous motivation significantly buffered the momentary associations of physical fatigue (B=-0.06, P<.001), mental fatigue (B=-0.04, P=.032), and pain (B=-0.21, P<.001) with depressed mood. CONCLUSIONS: Findings indicate the momentary associations of fatigue, cognitive complaints, and pain with depressed mood in stroke survivors. Autonomous motivation underpinning daily activity participation was found to buffer the associations of fatigue and pain with depressed mood. Promoting autonomous motivation for daily activity participation may be viable for preventing and mitigating poststroke depression.

Multimodal Ambulatory Monitoring of Daily Activity and Health-Related Symptoms in Community-Dwelling Survivors of Stroke: Feasibility, Acceptability, and Validity.

OBJECTIVE: To examine the feasibility, acceptability, and validity of multimodal ambulatory monitoring, which combines accelerometry with ecological momentary assessment (EMA), to assess daily activity and health-related symptoms among survivors of stroke. DESIGN: Prospective cohort study involving 7 days of ambulatory monitoring; participants completed 8 daily EMA surveys about daily activity and symptoms (mood, cognitive complaints, fatigue, pain) while wearing an accelerometer. Participants also completed retrospective assessments and an acceptability questionnaire. SETTING: Community. PARTICIPANTS: Forty survivors of stroke (N=40). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Feasibility was determined using attrition rate and compliance. Acceptability was reported using the acceptability questionnaire. Convergent and discriminant validity were determined by the correlations between ambulatory monitoring and retrospective self-reports. Criterion validity was determined by the concordance between accelerometer-measured and EMA-reported daily activity. RESULTS: All participants completed the study (attrition rate=0%). EMA and accelerometer compliance were 93.6 % and 99.7%, respectively. Participants rated their experience with multimodal ambulatory monitoring positively. They were highly satisfied (mean, 4.8/5) and confident (mean, 4.7/5) in using ambulatory monitoring and preferred it over traditional retrospective assessments (mean, 4.7/5). Multimodal ambulatory monitoring estimates correlated with retrospective self-reports of the same and opposing constructs in the predicted directions (r=-0.66 to 0.72, P<.05). More intense accelerometer-measured physical activity was observed when participants reported doing more physically demanding activities and vice versa. CONCLUSIONS: Findings support the feasibility, acceptability, and validity of multimodal ambulatory monitoring in survivors of mild stroke. Multimodal ambulatory monitoring has potential to provide a more complete understanding of survivors' daily activity in the context of everyday life.

Limitations of Current Rehabilitation Practices in Pediatric Oncology: Implications for Improving Comprehensive Clinical Care.

OBJECTIVE: To identify the proportion of hospitals/clinics in the United States (US) that have a comprehensive pediatric oncology rehabilitation program and characterize current practices. DESIGN: Cross-sectional survey of rehabilitation providers in the US and internationally. SETTING: Electronic or telephone survey. PARTICIPANTS: Rehabilitation or supportive care practitioners employed at a hospital, outpatient clinic, or medical university (N=231). INTERVENTIONS: Electronic and telephone survey. The full electronic survey contained 39 questions, provided opportunities for open-ended responses, and covered 3 main categories specific to pediatric cancer rehabilitation: service delivery, rehabilitation program practices, and education/training. The short telephone survey included 4 questions from the full survey and was designed to answer the primary study objective. MAIN OUTCOME MEASURES: Proportion of hospitals/clinics with a comprehensive pediatric oncology rehabilitation program. RESULTS: This cohort includes rehabilitation providers from 191 hospitals/clinics, 49 states within the US, and 5 countries outside of the US. Of hospitals/clinics represented from the full and short survey, 145 (76%) do not have an established pediatric oncology rehabilitation program. Nearly half of full survey respondents reported no knowledge of the prospective surveillance model, and 65% reported no education was provided to them regarding pediatric cancer rehabilitation. Qualitative survey responses fell into 3 major themes: variability in approach to rehabilitation service delivery, program gaps, and need for additional educational opportunities. CONCLUSIONS: There is evidence of limited comprehensive rehabilitation programming for children with cancer as demonstrated by the lack of programs with coordinated interdisciplinary care, variability in long-term follow-up, and absence of education and training. Research is needed to support the development and implementation of comprehensive pediatric oncology rehabilitation programs.

Association of Medicaid Expansion With Insurance Coverage Among Children With Cancer.

Importance: Despite evidence of improved insurance coverage under the Affordable Care Act and Medicaid expansion among adults with cancer, little is known regarding the association of these policies with coverage among children with cancer. Objective: To assess the association of early Medicaid expansion with rates of Medicaid coverage, private coverage, and no uninsurance among children with cancer. Design, Setting, and Participants: This cross-sectional study used data from the Surveillance, Epidemiology, and End Results (SEER) database from January 1, 2007, to December 31, 2015, to identify children diagnosed with cancer at ages 0 to 14 years in the United States. Data were analyzed from July 27, 2017, to October 7, 2019. Exposures: Changes in insurance status at diagnosis after early Medicaid expansion in California, Connecticut, Washington, and New Jersey (EXP states) were compared with changes in nonexpansion (NEXP) states (Arkansas, Georgia, Hawaii, Iowa, Kentucky, Louisiana, Michigan, New Mexico, and Utah). Main Outcomes and Measures: Difference-in-differences (DID) analyses were used to compare absolute changes in insurance status (uninsured, Medicaid, private/other) at diagnosis before (2007 to 2009) and after (2011 to 2015) expansion in EXP relative to NEXP states. Results: A total of 21 069 children (11 265 [53.5%] male; mean [SD] age, 6.18 [4.57] years) were included. A 5.25% increase (95% CI, 2.61%-7.89%; P < .001) in Medicaid coverage in children with cancer was observed in EXP vs NEXP states, with larger increases among children of counties with middle to high (adjusted DID estimates, 10.18%; 95% CI, 4.22%-16.14%; P = .005) and high (adjusted DID estimates, 6.13%; 95% CI, 1.10%-11.15%; P = .05) poverty levels (P = .04 for interaction). Expansion-associated reductions of children reported as uninsured (-0.73%; 95% CI, -1.49% to 0.03%; P = .06) and with private or other insurance (-4.52%; 95% CI, -7.16% to -1.88%; P < .001) were observed. For the latter, the decrease was greater for children from counties with middle to high poverty (-9.00%; 95% CI, -14.98% to -3.02%) and high poverty (-6.38%; 95% CI, -11.36% to -1.40%) (P = .04 for interaction). Conclusions and Relevance: In this study, state Medicaid expansions were associated with increased Medicaid coverage in children with cancer overall and in some subgroups primarily owing to switching from private coverage, particularly in counties with higher levels of poverty but also through reductions in the uninsured.

Impact of the affordable care act dependent coverage provision on young adult cancer patient insurance coverage by sociodemographic and economic characteristics.

PURPOSE: To evaluate the impact of the Affordable Care Act Dependent Care Provision by sociodemographic and economic characteristics in young adult cancer patients. METHODS: The National Cancer Database (NCDB) and the Surveillance, Epidemiology, and End Results (SEER) 18 database were queried for young adult cancer cases diagnosed during 2007-2014. Using a difference-in-differences approach, we examined insurance coverage in different subgroups of policy-eligible 19-25 year-olds versus policy-ineligible 27-29 year-olds from the pre- (2007-2009) to post- (2011-2014) Dependent Care Provision period. RESULTS: Across subgroups and study populations, insurance coverage increased significantly following the Provision enactment in the policy-eligible versus policy-ineligible group across most subgroups (range in NCDB: 1.83 to 6.38% for low and mid-low education areas, respectively; range in SEER: 1.43 to 6.18 for Non-Hispanic Others and Hispanics, respectively). Heterogenous impacts were observed by sex with a larger impact in males (NCDB: 5.14%, 95% CI 3.59-6.69; SEER: 4.46, 2.12-6.8) than females (NCDB: 2.51%, 95% CI 1.39-3.62; SEER: 2.50, 0.82-4.18). We observed no other statistical evidence for Dependent Care Provision subgroup heterogeneity except for a smaller impact in individuals from low education areas in NCDB. CONCLUSIONS: Our results indicate a positive Dependent Care Provision impact on insurance coverage in young adults with cancer across subgroups, with evidence for a smaller impact in females relative to males and in low relative to high education areas.

Functional and Radiologic Assessment of the Brain after Reduced-Intensity Unrelated Donor Transplantation for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network Study 0601.

Stroke and cognitive decline are hallmarks of sickle cell disease (SCD). The natural history of SCD predicts progressive loss of 1 IQ point per year attributable to disease-related pathology. Hematopoietic cell transplantation (HCT) is curative by reverting to donor-derived erythropoiesis, but evidence that HCT can positively influence disease-induced cognitive decline is lacking. The Sickle Cell Unrelated Transplant Trial prospectively evaluated cognition and brain magnetic resonance imaging (MRI) findings at 2 years after reduced-intensity conditioning followed by unrelated donor HCT. Thirteen study participants completed pre-HCT and post-HCT assessments of intelligence. The mean age of participants was 12.5 ± 3.3 years (range, 6.7 to 17.4 years). Eleven of the 13 recipients completed imaging studies at baseline and post-HCT. Seven had overt stroke pre-HCT, and 1 had an elevated transcranial Doppler velocity with abnormal MRI. The mean Full-Scale IQ was stable: 90.9 ± 13 at baseline and 91.2 ± 13 post-HCT. The mean Performance IQ was 89.9 ± 13 at baseline versus 90.9 ± 13 post-HCT, and mean Verbal IQ was 93.4 ± 13 at baseline versus 93.2 ± 13 post-HCT, respectively. Six recipients had stable MRI; 2 showed resolution of all areas of infarction. Three had additional infarcts post-HCT noted at the 2-year time point. This is the first report describing stabilization of IQ and central nervous system outcomes after unrelated donor HCT despite previous central nervous system morbidity and post-HCT posterior reversible encephalopathy syndrome. These preliminary results post-HCT suggest that HCT may stabilize the cognitive decline of SCD and should continue to be followed over the long term.

Assessment of the treatment approach and survival outcomes in a modern cohort of patients with atypical teratoid rhabdoid tumors using the National Cancer Database.

BACKGROUND: Atypical teratoid rhabdoid tumors (ATRTs) are rare brain tumors that occur primarily in children under the age of 3 years. This report evaluates the treatment approach and survival outcomes in a large cohort of patients treated in the United States. METHODS: Using the National Cancer Database, the analysis included all ATRT patients aged 0 to 18 years who were diagnosed between 2004 and 2012 and had complete treatment data. RESULTS: Three hundred sixty-one ATRT patients were evaluated. The 5-year overall survival (OS) rate was 29.9%, and it was significantly lower for children who were less than 3 years old (5-year OS, 27.7%) versus those who were 3 years old or older (5-year OS, 37.5%; P < .001). The best outcome was seen for patients with localized disease who received trimodality therapy (surgery, chemotherapy, and radiation therapy [RT]) with a 5-year OS rate of 46.8%. The utilization of trimodality therapy significantly increased during the study period (27.7% in 2004-2008 vs 45.1% in 2009-2012; P < .01), largely because of the increased use of RT. In a multivariate analysis, treatment that did not utilize trimodality therapy was associated with significantly worse OS (hazard ratio, 2.52; 95% confidence interval (1.82-3.51). Children aged 0 to 2 years were significantly less likely to receive trimodality therapy because of decreased utilization of RT in this age group. CONCLUSIONS: The use of trimodality therapy significantly increased during the study period and was associated with improved outcomes. For patients with localized disease who received trimodality therapy, the OS rate at 5 years approached 50%. However, further research into the optimal management of children less than 3 years old is needed because of their significantly worse OS in comparison with older children. Cancer 2017;123:682-687. © 2016 American Cancer Society.

Prevention of central nervous system sequelae in sickle cell disease without evidence from randomized controlled trials: the case for a team-based learning collaborative.

Since 1998, the National Institutes of Health has funded 5 randomized controlled trials (RCTs) for primary and secondary prevention of strokes in children with sickle cell anemia (SCA). In a systematic fashion, these trials have significantly advanced the care of children with SCA. In the absence of an RCT, clinicians are often compelled to make decisions at the bedside, based on experience, observational studies, and principles of hematology. We will provide an initial example that describes how a team-based, learning collaborative developed a multisite standard care protocol with a low budget (<$10 000 per year) to overcome the intrinsic limitations of advancing the care of neurologic complications in sickle cell disease (SCD). The critical components of this approach include: (1) regular meetings with the multidisciplinary team from multiple sites; (2) consensus regarding the best evidence-based neurologic management in multiple SCD centers; (3) an Institutional Review Board-approved protocol based on consensus standard care; (4) minimizing and ensuring accurate data collection; and most importantly, (5) a spirit of collaboration to improve the care of individuals with SCD. Four common neurologic problems and strategies for management in children and adults with SCD will be discussed: (1) secondary stroke prevention in high-income countries; (2) primary stroke prevention in low- and middle-income countries (LMICs); (3) poor academic performance in students; and (4) cognitive disability in adults. With a commitment to a team-based learning collaborative, incremental advances are possible for the neurologic care of children and adults with SCD.

Youth with Sickle Cell Disease: Genetic and Sexual Health Education Needs.

OBJECTIVES: Assess the need for and interest in a sexual health and sickle cell disease (SCD) inheritance educational program for youth with SCD. METHODS: Using a cross-sectional approach, qualitative data were collected during interviews of youth with SCD between ages 11-19 years from an urban hospital. Inductive and emergent coding was used to identify themes. Frequencies were recorded and analyzed. RESULTS: Thirty-five youth patients were approached; 20 (57%) consented and completed interviews. Half of the participants were adolescent girls. The mean age was 16.9±1.8 years (range 13-19). Of the 20 participants, 100% expressed interest in SCD genetic counseling and/or posed questions regarding SCD inheritance. Nineteen (95%) demonstrated deficits in sexual health knowledge and/or requested sexual health education. Seventeen (85%) actively engaged with educational materials. Twelve (60%) reported engaging in sexual activity, 7 (35%) in risky behaviors, and 3 (15%) reported past sexually transmitted infection (STI) diagnosis. CONCLUSIONS: Youth with SCD demonstrated limited knowledge regarding sexual health and inheritance of SCD. Topics like information-seeking, limited knowledge, and need for social skills training provide evidence for the creation of an educational intervention for this vulnerable population. This program shows initial feasibility for youth with SCD.

Transition Needs of Adolescents With Sickle Cell Disease.

OBJECTIVE: This article describes how adolescents with sickle cell disease (SCD) perceive their ability to perform everyday tasks required for transition to adult health care and independent living. METHOD: The Adolescent Autonomy Checklist (AAC) was adapted to include skills associated with managing SCD (AAC-SCD) and was administered to adolescents during clinic visits. Participants indicated "can do already" or "needs practice" for 100 activities in 12 categories. RESULTS: Of 122 patients, the percentage of adolescents who needed practice was greatest in living arrangements (38.7%), money management (35.8%), vocational skills (29.6%), and health care skills (25.5%). We found a significant effect of age and of cerebrovascular injury on the percentage of those who reported "needs practice" in multiple categories. We found no effect of gender and limited effect of hemoglobin phenotype on any skill category. CONCLUSION: Findings support the need for educational intervention to improve transition skills in adolescents with SCD.

Development and feasibility of a home-based education model for families of children with sickle cell disease.

BACKGROUND: Children with sickle cell disease (SCD) commonly have cognitive deficits, even among toddlers. Much medical literature emphasizes disease-based factors to account for these deficits. However, the social environment plays a large role in child development. To address the specific needs of early childhood, a monthly hospital-based education program was initiated to educate parents about child development. Education sessions were poorly attended (20-25%) and deemed unsuccessful. This study describes the development and implementation of a home-based education service to teach parents about SCD, developmental milestones and positive parenting techniques. METHODS: This was a prospective, single-arm intervention to study the feasibility of a home-based caregiver education program for families with infants and toddlers with SCD. Parents of children aged 0-3 years with SCD from one Midwestern hospital were approached to participate in a home-based program. The program followed the Born to Learn™ curriculum provided through the Parents as Teachers™ National Center. Reminder calls or texts were provided the day before each visit. Results of the first twenty-six months of the program are presented. RESULTS: A total of 62% (56 of 91) of families approached agreed to participate; all were African American. The majority of caregivers were single mothers with a high school education or less and whose children had Medicaid for health coverage. The phenotypes of SCD represented in this sample were similar to those in the general SCD population. Over 26 months, 39 families received at least one home visit. Parents of infants (younger than 8 months) were more likely to participate in the home-based education program than parents of older children, (Fisher's exact test, p < .001). CONCLUSIONS: For participating families, home-based visits were a feasible method for reinforcing clinic education. About 43% of eligible families participated in the education, a two-fold increase in the poor attendance (20%) for a previous hospital-based program. A home visitation program for parents of infants with SCD could offer an effective approach to helping these children overcome adverse environmental conditions that are compounded by the complexities of a chronic health condition.