Investigating home-based opioid use among youth with sickle cell disease using ecological momentary assessment.

BACKGROUND: Opioids are a common and essential treatment for acute sickle cell disease (SCD) pain. However, opioids carry well-known adverse side effects, including potential development of hyperalgesia and nociplastic pain. We characterized opioid use in youth with SCD using ecological momentary assessment (EMA) data, and investigated the relationships between home-based opioid use, pain, and a range of biopsychosocial factors. METHOD: Eighty-eight youth with SCD (aged 8-17 years) completed EMAs assessing home-based opioid use, pain, and related factors. Analyses consisted of descriptive and multilevel logistic regression to predict daily home opioid use. RESULTS: Youth averaged 3.64 weeks of EMAs. Approximately 35% of the sample (n = 31) took an opioid during the EMA period, and used them on only 24% of reported pain days. Youth who took opioids reported a higher percentage of pain days (t = -2.67, p < .05) and mean pain severity scores (t = -2.30, p < .05) than youth who did not take opioids. Multilevel logistic regression analyses indicated that high daily pain severity (odds ratio [OR] = 1.02, p < .01), older age (OR = 1.324, p < .01), and low positive affect (OR = 0.91, p < .01) were each related to an increased likelihood of opioid use. CONCLUSION: Youth with SCD take opioids appropriately in response to their pain, based on daily self-report. Beyond daily pain severity, age, and daily variation in positive affect were related to home-based opioid use. This suggests that behavioral interventions that enhance positive affect may promote reduced opioid use among youth with SCD.

Intraindividual pain variability metrics for youth with sickle cell disease: Relations to health outcomes.

BACKGROUND: While the majority of pediatric sickle cell disease (SCD) research has used mean pain intensity as the only pain metric, recent evidence suggests this metric alone is inadequate in describing the intraindividual variability in SCD pain experiences and subsequent impact. There is limited information on other intraindividual pain metrics in youth with SCD, or how they relate to health outcomes in this population. The aims of this study were to describe differing patterns of intraindividual pain metrics derived from ecological momentary assessments (EMAs) of youth with SCD and to characterize the unique relationships between these metrics and health outcomes. METHODS: Eighty-eight youth with SCD, aged 8-17 (mean age = 11.6), were recruited from three regional pediatric SCD clinics in the United States. At baseline, youth and their guardians reported on demographic and disease information. Then youth completed twice daily EMAs for up to 4 weeks. Pain metrics derived from EMA data were calculated including mean daily pain intensity (DP), SD-DP (standard deviation of DP), proportion of pain days (PPD), and 90th percentile of DP (p90). Pearson correlations were calculated between pain metrics and health outcomes. RESULTS: High DP and SD-DP were correlated with more anxiety symptoms, while high SD-DP and p90 were correlated with more depression symptoms. High SD-DP was correlated with low self-esteem, and high DP and PPD were correlated with low sickle cell self-efficacy. For healthcare utilization due to pain, high p90 was correlated with more emergency department visits, while high DP, p90, and PPD were correlated with more healthcare contacts. CONCLUSION: There are distinct associations between pain variability metrics beyond DP and health outcomes. Collectively, the patterns of associations suggest the utility of these pain metrics for determining risk in relation to specific health outcomes for youth with SCD.

Structural Racism and Impact on Sickle Cell Disease: Sickle Cell Lives Matter.

Wealth begets health: the health care system in the United States is run by and benefits the groups that have traditionally held power. Systems of structural racism and health care disparities persist. Patients with sickle cell disease (SCD) remain particularly vulnerable to disparities. They suffer from stigmas, lack of well-trained providers, and from misalignment of their needs with the priorities of their health care teams. These critically important burdens may actually be worsening rather than improving mortality for individuals living with SCD. Changes must be made at the federal, state, and local levels in order to address these systems of inequity and save vulnerable lives.

Satisfaction and access to care for adults and adolescents with sickle cell disease: ASCQ-Me quality of care and the SHIP-HU study.

A lack of adult sickle cell providers has long been blamed for poor satisfaction and access to specialty care for adults with sickle cell disease (SCD). We were interested in comparing how adolescent and adult patients already in established SCD centers perceived access and quality of care. Hydroxyurea-eligible patients aged 15 years and older were enrolled in the Start Healing in Patients with Hydroxyurea trial, which required them to be affiliated with a SCD specialist. Patients were seen in one of three adult-oriented specialty clinic sites or one of three pediatric-oriented sites. At baseline, patients completed the Adult Sickle Cell Quality of Life Measurement Information System measure as part of a survey battery. Patients treated at adult clinic sites reported being less able to get timely ambulatory appointments (p = .004). They reported emergency department (ED) wait times of >1 h far more often (47.7 vs. 19.3%, p = .0048). They reported less overall satisfaction with care (7.47 vs. 8.77, p < .0001), and less satisfaction with care in the ED (2.88 vs. 3.4, p = .0068. Ambulatory satisfaction was no different between pediatric site versus adult site patients. Poorer systems of care appeared to underlie reported differences, rather than differences in biopsychosocial determinants. Even among specialty-care-affiliated SCD patients, those seen in adult clinics reported worse access to care and lower satisfaction with care than patients seen in pediatric clinics. In addition to increasing the number of adult SCD providers and better preparing pediatric SCD patients to transfer to adult programs, SCD clinical caregivers must also improve aspects of adult care quality to meet reasonable patient expectations of timeliness and interpersonal aspects of care quality.

Feasibility of a Pilot Home Phlebotomy Program for Pediatric Hematology/Oncology Patients During the COVID-19 Pandemic.

Medical care during the Coronavirus 2019 global pandemic required significant shifts in health care delivery systems. Telehealth was widely deployed but was of limited utility for patient populations who rely heavily on laboratory monitoring. This includes pediatric hematology and oncology patients. We report on the feasibility and successful implementation of a home phlebotomy program that has minimized disruption in care for this high-risk patient population. During the initial months of the COVID-19 outbreak, we completed 189 home phlebotomy visits for pediatric hematology and oncology patients. Patient and staff satisfaction with the program were high, and potential exposures to COVID were avoided.

Aggressive vertebral hemangioma masquerading as neurological disease in a pediatric patient.

Aggressive hemangioma is a rare vertebral lesion in pediatric patients which can present with deteriorating neurological function. It can mimic malignancy on imaging, particularly as it regularly has an extrasosseous soft tissue component. We present a case of a 13-year-old male who presented with a three month history of lower extremity weakness that was found to have an infiltrative mass at T10 with associated cord compression from epidural extension of the lesion. In this report we review the characteristic imaging findings associated with aggressive hemangioma, including its appearance on read-out segmented diffusion-weighted images. It is imperative that radiologists who interpret studies of children be aware that this lesion exists and what it looks like, as it can be associated with massive hemorrhage if encountered unexpectedly during surgery.

Exercise Testing of Adolescents and Young Adults With Sickle Cell Disease: Perceptual Responses and the Gas Exchange Threshold.

For individuals with sickle cell disease (SCD), mild to moderate exercise is advised, but self-regulation of these intensities is difficult. To regulate intensity, one SCD recommendation is to stop exercising at the first perception of fatigue. However, perceived effort and affect (how one feels) are perceptual cues that are commonly used to guide exercise intensity. This study (a) examined perceived effort, affect, and fatigue in relation to metabolic state (gas exchange) in adolescents and young adults (AYAs) with SCD, (b) explored guidelines AYAs use to self-regulate exercise, and (c) compared perceived effort and affect at gas exchange threshold (GET) with healthy counterparts. Twenty-two AYAs with SCD completed an incremental cycle test. Perceived effort, affect, and fatigue were assessed every 2 minutes. A mixed-effects linear model was conducted to model changes in effort, affect, and fatigue across time. Mean scores of effort and affect at GET were compared with published data of healthy counterparts. Participants were queried about self-regulation exercise strategies. Findings indicated that both perceived fatigue and effort at GET was lower than expected. Perceived effort was lower (p < .0001), and perceived affect was significantly higher (p = .0009) than healthy counterparts. Interviews revealed that most participants (95%) do not stop exercising until fatigue is moderate to severe, and many (73%) do not stop until symptoms are severe (chest tightness, blurry vision). Nurses should review guidelines for safe exercise with AYAs with SCD. Exercise training may be beneficial to AYAs with SCD for learning how to interpret bodily responses to exercise to improve self-regulation.

A novel approach to reducing admissions for children with sickle cell disease in pain crisis through individualization and standardization in the emergency department.

BACKGROUND: Vaso-occlusive crisis (VOC) is frequent in children with sickle cell disease (SCD) creating significant burden on patients, families, and emergency departments (ED). The objective of the project was to reduce the admission rate for children with SCD presenting to our ED with VOC by >20% within 6 months of initiating individualized pain plans (IPP). METHODS: A multi-disciplinary quality improvement team was assembled. A Plan-Do-Study-Act (PDSA) format was employed. The IPP document was created in a unique folder within the electronic medical record. IPPs were created through retrospective chart review for our 80 highest resource users. Pediatric residents, ED residents, and ED attending physicians were instructed on use of the IPPs. Our study measured the presence of an IPP, adherence to the IPP, and time to opiate administration. Our primary outcome was admission rate. Length of stay and 72-hr return to the ED were assessed as balancing measures. RESULTS: Overall, admission rate decreased by 24% following implementation compared with the previous 5 years (P = 0.046). IPPs were created for 78% of patients and followed by ED staff in 86% of visits. Admission rate was significantly lower for patients receiving a second opiate dose within 45 min of the first dose (P < 0.01). There was no difference in readmission rate or 72-hr return rate to ED. CONCLUSIONS: This study presents an effective strategy to reduce admission rate for children with SCD presenting with VOC. Shorter time to second opiate dosing was also associated with reduced risk of admission.

Health literacy and disease-specific knowledge of caregivers for children with sickle cell disease.

This study was conducted to measure the health literacy (HL) and disease-specific knowledge (DSK) of caregivers for children with sickle cell disease (SCD) and relate them to their child's health care utilization. The authors conducted a cross-sectional study of caregiver-child dyads attending an urban pediatric sickle cell clinic. Caregivers were administered the Shortened Test of Functional Health Literacy (S-TOFHLA) and a locally developed DSK questionnaire. Retrospective review of the child's electronic medical record (EMR) was performed to determine annual emergency department (ED) visits and hospitalizations. A total of 142 caregiver-child dyads were recruited for the study. Less than 5% of caregivers had limited HL, with less education (P =.03) and primary language other than English (P =.04) being the only risk factors. Although caregiver HL was not associated with ED visits or hospitalizations, surprisingly DSK was. Caregivers with higher DSK scores had children with higher annual rates of ED utilization (P =.002) and hospitalizations (P =.001), and these children were also more likely to be classified as high ED utilizers (≥4 visits per year; P =.01). Further, caregiver adherence to medication and clinic visits was associated with their child's age (P =.01). Although HL and DSK are both constructs that measure basic health understanding, they differently affect caregivers' ability to navigate and understand the health care system of children with chronic illnesses. This study suggests that the DSK/health care utilization relationship may be a more important measure than HL for programs following children with sickle cell disease and could also have applications in other pediatric chronic diseases.

Sorafenib treatment following hematopoietic stem cell transplant in pediatric FLT3/ITD acute myeloid leukemia.

BACKGROUND: FLT3/ITD is associated with poor outcomes in adult and pediatric acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation (HSCT) can improve cure rates, however relapse is still common. Recent studies demonstrate the activity of FLT3 inhibitors, including sorafenib, in targeting the underlying mutation. PROCEDURE: We conducted a retrospective study of 15 pediatric patients with FLT3/ITD+ AML treated with sorafenib within 18 months after receiving HSCT. Sorafenib was administered either as prophylaxis in patients considered at very high risk for relapse (n = 6) or at the time of disease recurrence (n = 9). RESULTS: Sorafenib was initiated at a median of 100 days post HSCT. Overall, 11/15 (73%) of patients experienced medically significant toxicities. Among patients who experienced toxicity, 6/11 (55%) received treatment at doses above what was later determined to be the maximum tolerated dose of sorafenib for pediatric leukemia. Importantly, sorafenib did not appear to exacerbate graft versus host disease. Our findings suggest that sorafenib may be of particular efficacy in patients with minimal residual disease (MRD); all patients who received sorafenib for MRD immediately prior to transplant or with emergence post-HSCT are alive and remain in complete remission at a median of 48 months post HSCT. CONCLUSIONS: Our case series suggests that sorafenib administration is feasible and tolerable in pediatric FLT3/ITD+ AML patients early post HSCT. Ongoing prospective controlled studies are needed to further define the dosing of sorafenib in the post-HSCT period and to determine the optimal context for this treatment approach.

Impact of conditioning regimen in allogeneic hematopoetic stem cell transplantation for children with acute myelogenous leukemia beyond first complete remission: a pediatric blood and marrow transplant consortium (PBMTC) study.

Total body irradiation (TBI)-based conditioning regimens for pediatric patients with acute myelogenous leukemia (AML) beyond first complete remission (CR1) are controversial. Because the long-term morbidity of busulfan (Bu)-based regimens appears to be lower, determining efficacy is critical. We retrospectively evaluated 151 pediatric patients with AML beyond CR1, comparing outcomes in 90 patients who received a TBI-based conditioning regimen and 61 patients who received a Bu-based conditioning regimen. There were no differences between the 2 groups with respect to age, sex, duration of CR1, time from most recent remission to transplantation, or donor source. The probability of relapse at 2 years also did not differ between the 2 groups (26% and 27%, respectively; P=.93). No significant difference in event-free survival (EFS) (P=.29) or overall survival (OS) (P=.11) was noted between the 2 groups. These findings were supported by a multivariate analysis in which TBI was not associated with improved EFS (hazard ratio [HR]=1.17; 95% confidence interval [CI]=0.66-2.10; P=.58) or OS (HR=1.42; 95% CI=0.76-2.64; P=.27). Shorter CR1 and receiving an HLA-mismatched transplant adversely affected EFS and OS in this cohort. Our study provides no evidence of an advantage to using TBI in children with AML beyond CR1. A prospective, randomized study is needed to confirm these results.