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 diseaseinduced 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.

Sickle cell disease (SCD) is a genetic disorder predominantly affecting people of African descent and is associated with significant morbidity and mortality. To improve SCD outcomes, the National Heart Lung and Blood Institute funded eight centers to participate in the SCD Implementation Consortium. Sites were required to each recruit 300 individuals with SCD, over 20 months. We aim to describe recruitment strategies and challenges encountered. Participants aged 15–45 years with confirmed diagnosis of SCD were eligible. Descriptive statistics were used to analyze the effectiveness of each recruitment strategy. A total of 2432 participants were recruited. Majority (95.3%) were African American. Successful strategies were recruitment from clinics (68.1%) and affiliated sites (15.6%). Recruitment at community events, emergency departments and pain centers had the lowest yield. Challenges included saturation of strategies and time constraints. Effective recruitment of participants in multi-site studies requires multiple strategies to achieve adequate sample sizes.

Objective Caregivers of young children with chronic illnesses are at high risk for elevated levels of stress and mental health symptoms. This study examined stress and mental health symptoms as well as the socioeconomic status (SES) and home environments of a cohort of caregivers of infants and toddlers with sickle cell disease (SCD). Methods Forty-two caregivers of infants and toddlers (aged 1–34 months) with SCD completed the Brief Symptom Inventory (BSI) and Parent Stress Index (PSI). The Home Observation for Measurement of the Environment (HOME) was used to assess family living environments. Results Compared to test norms, caregivers reported high levels of situational/demographic life stress [mean difference (MD) 5.7, p = .003] and child distractibility/hyperactivity (MD 3.62, p = .001) on the PSI. However, no significant differences in psychological symptoms of distress were noted on the BSI. Caregivers scored significantly lower than norms on PSI subdomains of acceptability (MD −1.88, p = .03), competence (MD −3.11, p = .002), depression (MD −3.94, p < .001), and the overall parent domain (MD −12.55, p = .005). Significant correlations were found between PSI scores and the HOME and between SES and the HOME. Conclusion Caregivers of infants and toddlers with SCD experience elevated levels of life stress but, in turn, endorse high acceptance of their child and self-competence in parenting. Although life stress may be high in this population, symptoms of psychological distress were not identified. Caregivers reporting elevated life and illness-specific stressors may benefit from environmental supports and interventions.

Pediatric central nervous system (CNS) tumor survivors are at risk for experiencing cognitive late effects (CLEs). Caregivers of survivors may be unaware of these changes or receive untimely information regarding CLEs. Conversely, health care providers (HCPs) may face barriers to providing education. This study aims to (a) understand the knowledge and resource gap for caregivers regarding CLEs and (b) explore how HCPs currently provide education. Caregivers and HCPs were both interviewed. Qualitative analysis was performed using emergent coding. Fifteen caregivers and eight HCPs participated. Caregivers generally felt confident in assisting their survivor but experienced "information overload" during initial diagnosis and treatment. HCPs reported difficulties in determining appropriate timing for education and perceived that caregivers typically lack understanding of CLEs. Caregivers should be aware of and understand a survivor's risk for CLEs to help survivors manage changes. With increasing survival rates of pediatric CNS tumor patients, HCPs must be prepared to provide appropriate education and referrals regarding CLEs for long-term care.

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