476 Background: Sickle cell disease (SCD) is an inherited chronic disease characterized by complications such as recurrent painful vaso-occlusive events that can require hospitalizations and contribute to early and increased mortality. Prior work using generic health-related quality of life (HRQL) instruments has demonstrated that patients with SCD experience significantly impaired HRQL in their baseline state of health that worsens during acute complications of the disease. To better understand differences in health status in children with SCD, we developed the PedsQL™ SCD Module to measure SCD-specific HRQL. The goal of this study was to determine the measurement properties for the child self- and parent-proxy reports for the newly developed PedsQL™ SCD Module. We hypothesized that the PedsQL™ SCD Module would be feasible and reliable and that children with more severe SCD would have worse HRQL than those with mild disease as measured by the PedsQL™ SCD Module. Methodology: This was a cross-sectional study conducted at 5 sites across the United States. Study participants were children with SCD ages 2–18 years who presented to clinic for a routine visit. HRQL was the main outcome measured with the newly developed 43-item PedsQL™ SCD Module which includes nine scales: Pain/Hurt (PH, 9 items), Pain Impact (PI, 10 items), Pain Management/Control (PMC, 2 items), Worry I (WO1, 5 items), Worry II (WO2, 2 items), Emotions (EM, 2 items), Treatment (TR, 7 items), Communication I (CO1, 3 items), Communication II (CO2, 3 items). Higher scores indicate better HRQL and lower SCD symptoms. Missing items were used to determine feasibility and Cronbach's alpha was used to determine reliability. HRQL of children with mild and severe disease were compared using an independent t-test to determine construct validity. Severe disease was defined as patients with 3 or more hospitalizations for pain in the 3 years prior, history of stroke and/or prior acute chest syndrome. Results: A total of 321 families (313 parents, 243 children ages 5–18 years) completed questionnaires. The average age of the children (46.7% boys) was 9.62 years (SD = 4.88). Feasibility was established, with 3% or less missing data for the module. The PedsQL™ SCD Module was reliable and distinguished between children with mild and severe SCD (Table 1). Conclusions: The PedsQL™ SCD Module performed well and demonstrated strong measurement properties in patients with SCD. Overall, both the parent-proxy report and child self-report differentiated between patients with severe and mild SCD supporting construct validity of the module. Although these are the first results using the PedsQL™ SCD Module, it has shown great potential as being a strong measure of HRQL for patients with SCD. Future studies incorporating the PedsQL™ SCD Module will benefit from the measure's disease-specific scales and overall ability to distinguish between mild and severe symptoms. Continually, these studies will help further define its' measurement properties and advance our knowledge of the HRQL in patients with SCD. Disclosures: Off Label Use: Hydroxyurea is approved for use in sickle cell disease in adults but not children. Varni:Mapi Research Trust: Dr. Varni holds the copyright and the trademark for the PedsQL™ and receives financial compensation from the Mapi Research Trust, which is a nonprofit research institute that charges distribution fees to for-profit companies that use the Pediatric Quality Other, PedsQL™, PedsQL™ Patents & Royalties.

Health-related quality of life (HRQL) measures provide information about disease assessment; however, healthcare 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 to the Pain and Hurt and Pain Impact scale scores to determine levels representing high and low HRQL. These scores were compared to disease severity and the PedsQL™ Generic Core Scales. Scores of 60 or below 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 or higher 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 or lower are cause for concern and suggest areas of HRQL impairment in SCD.

This study explored the kinds of relationship experiences associated with earned-security, i.e., the extent to which mothers who report early negative relationship histories with their parents are later able to form a secure working model of attachment (indicated by the ability to speak clearly and coherently about these histories). Mothers from a low-risk sample (N = 121) expecting their first child completed the Adult Attachment Interview (AAI), which was used to assess earned-security retrospectively using the stringent definition recommended by Main and Hesse (Hesse, 2008 ; Main, Goldwyn, & Hesse, 2002 ), as well as to identify alternative support figures. Participants also completed self-report measures of depressive symptomatology, questionnaires concerning their experiences in therapy, and later, when their babies were 12 to 15 months old, the Strange Situation procedure. Sixteen mothers were classified as earned-secure (25% of those classified as secure-autonomous and 13% of the whole sample). Women who were earned-secure (vs. insecure and continuous-secure) reported significantly higher levels of emotional support, but not instrumental support, from alternative support figures. They also spent more time in therapy than did insecure and continuous-secure women and were more likely to form secure attachments with their infants than insecure women. These findings were obtained even after controlling for depressive symptoms.

The purpose of the present study was to identify rates of adherence for three outpatient quality indicators noted by Wang and colleagues (2011): (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.

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.

The few studies found through this systematic review demonstrate that there is little empirical evidence on which to judge the use of direct preference elicitation methods with children regarding health states.

Despite the benefits of patient reported outcomes (PROs), the systematic use of PRO assessment in clinical care is rare. The goal of this pilot project was to examine the feasibility of PRO assessment via the Patient Reported Outcomes Measurement Information System-Computerized Adaptive Testing (PROMIS-CAT) within pediatric hematology, oncology, and bone marrow transplant (BMT) clinic settings. Fifty-one patients (ages 5 to 26 years), 53 parents, and 13 medical providers were recruited successfully. After patients and parents completed the PROMIS-CAT on electronic tablets, patients, parents, and medical providers completed questionnaires regarding acceptability, burden, and potential barriers with electronic PRO assessment within the clinic setting. The majority of patients (72%) and parents (70%) endorsed positive acceptability with completing the PROMIS-CAT. Patients and parents found the assessment easy to use and the questions understandable relative to the enjoyableness and helpfulness of the measure. Positive acceptability findings from the medical providers (25% satisfied) were lower than expected. Completion of electronic PRO assessment did not appear to add significant burden to patients, families, or the clinic workflow. Technological issues, comprehension of questions, and medical provider confusion with interpretation of the original score report of the PROMIS-CAT were identified as potential barriers with capturing PROs in the clinics. Overall, this study supports the feasibility of collecting PROs in real time in a clinic setting as patients, parents, and medical providers found the assessment acceptable and of low burden, and minimal barriers with the integration of PRO assessment within a clinic setting were identified.

Using historical cohorts of healthy, acutely ill, and chronically ill infants for comparison, we sought to determine whether infants with sickle cell disease (SCD) have impaired health-related quality of life (HRQL). We conducted a cross-sectional study at 2 sites: the Medical College of Wisconsin/Children's of Wisconsin and the University of Alabama School of Medicine/Children's of Alabama. Parents of 90 infants with SCD completed the PedsQL Infant Module corresponding to their infant's age (1 to 12 mo or 13 to 24 mo) during a regular clinic visit. At 1 to 12 months, infants with SCD displayed lower Physical HRQL than healthy infants, but better HRQL than chronically ill infants. By 13 to 24 months, infants with SCD had worse HRQL in all areas than healthy infants and worse Physical and Total HRQL than acutely ill infants. Compared with chronically ill infants in this age group, infants with SCD had similar Physical HRQL and better Psychosocial and Total HRQL. By 13 to 24 months, a greater proportion of infants with SCD had impaired Physical and Total HRQL compared with infants aged 1 to 12 months. All differences were significant at the (P<0.05) level. Impaired HRQL can be detected in infants with SCD.