Background
Children with sickle cell disease (SCD) have an increased risk for gallstones due to chronic hyperbilirubinemia from hemolysis. Although gallstones are a known complication, there is ...variability in estimates of disease burden and uncertainty in the association between sex and gall bladder disease (GBD).
Methods
This was a retrospective cohort study of children with SCD using administrative claims data (January 1, 2014–December 31, 2018). Population‐averaged multivariable panel‐data logistic regression models were used to evaluate the association between GBD clinical encounters (outcome) and two exposures (age and sex). Annual GBD risk was calculated using predictive margins, adjusting for disease severity, transfusion frequency, and hydroxyurea exposure.
Results
A total of 13,745 individuals (of 21,487 possible) met inclusion criteria. The population was evenly split across sex (49.5% female) with predominantly Medicaid insurance (69%). A total of 946 individuals (6.9%) had GBD, 432 (3.1%) had a gallstone complication, and 487 (3.5%) underwent cholecystectomy. The annual risk of GBD rose nonlinearly from 1 to 5% between ages 1 and 19 years with no difference between males and females. Cholecystectomy occurred primarily in individuals with GBD (87%), and neither age nor sex was associated with cholecystectomy in this population. High disease severity (compared with low) more than doubled the annual risk of GBD at all ages.
Conclusions
GBD is associated with age but not sex in children with SCD. Neither age nor sex is associated with risk of cholecystectomy. High disease severity increases the rate of GBD at all ages.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Summary
There is an immediate need to address long‐standing questions about the reproductive health of girls and women with sickle cell disease (SCD). There are many SCD‐related reproductive risks ...and uncertainties across girls’ and women’s reproductive life span, with particularly outstanding concerns about menstruation, contraception, fertility and pregnancy. Extant literature addressing women’s reproductive health topics is mostly descriptive; there are few high‐quality interventional studies. In 2020, the Centers for Disease Control and Prevention and the Foundation for Women and Girls with Blood Disorders convened an expert panel to assess the knowledge gaps in women’s reproductive health in SCD. The panel identified significant limitations to clinical care due to the need for research. The panel also identified prominent barriers to research and care. In this report, we frame these issues, providing a roadmap for investigators, funding agencies, and policy makers to advance care for girls and women with SCD.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Objective
Asthma is a recognized comorbidity in children with sickle cell disease (SCD). It increases the risk of acute chest syndrome (ACS), vaso‐occlusive episodes, and early mortality. We aim to ...determine whether evaluation and management of children with SCD and asthma by a pulmonologist reduce rate of asthma exacerbation and ACS.
Methods
The study included 192 patients with SCD (0–21 years) followed at Children's Hospital of Philadelphia Hematology between January 1, 2015, and December 31, 2018, with a diagnosis of asthma, wheeze, or cough. Patients were placed in two groups: those evaluated by a pulmonologist (SCD‐A‐P) and those not (SCD‐A). Rates of emergency department (ED) visits and hospitalizations for asthma exacerbation and ACS were compared between groups and over time.
Results
SCD‐A‐P patients (n = 70) were predominantly SCD type SS with lower hemoglobin and hematocrit compared to SCD‐A patients (n = 122). SCD‐A‐P started with a higher average rate of hospital visits for asthma exacerbation and ACS per year (2.69 1.02–4.37) compared to SCD‐A (0.43 0.24–0.63), (p < 0.001). For SCD‐A‐P patients with at least one hospital visit (n = 48), the average rate decreased from 3.93 (1.57–6.29) to 0.85 (0.48–1.23) following pulmonary consultation (p = 0.014) and was comparable to the SCD‐A rate by study end.
Conclusion
SCD‐A‐P was mainly SCD type SS and had higher ED/hospitalization rates for asthma exacerbation and ACS compared to SCD‐A, but the rates significantly decreased following pulmonology consultation. These findings support the pulmonologist's role in the multidisciplinary care of SCD patients and highlight the need for evidence‐based asthma guidelines for children with SCD.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Despite disease‐modifying effects of hydroxyurea on sickle cell disease (SCD), poor adherence among affected youth commonly impedes treatment impact. Following our prior feasibility trial, the ...“Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT)” multi‐site randomized controlled efficacy trial aimed to increase hydroxyurea adherence for youth with SCD ages 10–18 years. Impaired adherence was identified primarily through flagging hydroxyurea‐induced fetal hemoglobin (HbF) levels compared to prior highest treatment‐related HbF. Eligible youth were enrolled as dyads with their primary caregivers for the 1‐year trial. This novel semi‐structured supportive, multidimensional dyad intervention led by community health workers (CHW), was augmented by daily tailored text message reminders, compared to standard care during a 6‐month intervention phase, followed by a 6‐month sustainability phase. Primary outcomes from the intervention phase were improved Month 6 HbF levels compared to enrollment and proportion of days covered (PDC) for hydroxyurea versus pre‐trial year. The secondary outcome was sustainability of changes up to Month 12. The 2020–2021 peak coronavirus disease 2019 (COVID‐19) pandemic disrupted enrollment and clinic‐based procedures; CHW in‐person visits shifted to virtual scheduled interactions. We enrolled 50 dyads, missing target enrollment. Compared to enrollment levels, both HbF level and PDC significantly ‐ but not sustainably ‐ improved within the intervention group (p = .03 and .01, respectively) with parallel increased mean corpuscular volume (MCV) (p = .05), but not within controls. No significant between‐group differences were found at Months 6 or 12. These findings suggest that our community‐based, multimodal support for youth–caregiver dyads had temporarily improved hydroxyurea usage. Durability of impact should be tested in a trial with longer duration of CHW‐led and mobile health support.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Background
Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as young as ...6 months of age, and pediatric patients are at risk for acute and life‐threatening complications. Early intervention with treatments that target the underlying pathophysiological mechanism of SCD, sickle hemoglobin (HbS) polymerization, are expected to slow disease progression and circumvent disease‐associated morbidity and mortality.
Procedure
The HOPE‐KIDS 1 trial (NCT02850406) is an ongoing four‐part, phase 2a, open‐label, single‐ and multiple‐dose study to evaluate the pharmacokinetics, efficacy, and safety of voxelotor—a first‐in‐class HbS polymerization inhibitor—in patients aged 6 months to 17 years with SCD. Initial findings from a cohort of 45 patients aged 4 to 11 years who received voxelotor treatment for up to 48 weeks are reported.
Results
Hemoglobin (Hb) response, defined as a >1.0 g/dl increase from baseline, was achieved at week 24 by 47% (n = 16/34) of patients with Hb measurements at baseline and week 24. At week 24, 35% (n = 12/34) and 21% (n = 7/34) of patients had a >1.5 g/dl increase and a >2.0 g/dl increase from baseline in Hb concentration, respectively. Concurrent improvements in hemolytic markers were observed. Voxelotor was well tolerated in this young cohort, with no newly emerging safety signals.
Conclusions
Based on its mechanism as an HbS polymerization inhibitor, voxelotor improves Hb levels and markers of hemolysis and has the potential to mitigate SCD‐related complications; these results support its use in patients aged ≥4 years.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Youth with sickle cell disease (SCD) and their caregivers are susceptible to stress and depression, perhaps exacerbated by pandemic‐associated health and economic concerns. Most of the 50 ...youth–caregiver dyads enrolled in the multisite trial, Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT), took an online survey of self‐reported mental health symptoms and food insecurity during the 2020 COVID‐19 pandemic. Compared to largely pre‐pandemic results, prevalence of mental health symptoms in dyad members appeared to have shifted: fewer youth and more caregivers were affected during the pandemic; many of both groups lacked optimism. Pandemic/post‐pandemic screening of youth with SCD for mental health symptoms and food insecurity appears warranted.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Abstract Background Suboptimal medication adherence is common across youth with chronic health conditions and may contribute to health disparities and adverse health outcomes, especially in ...underserved communities. Methods Using pharmacy prescription records and guided by the World Health Organization Multidimensional Adherence Model, we examined patient‐, treatment‐, and health system–related factors that may affect hydroxyurea adherence in 72 youth with sickle cell disease (SCD), 10‐18 years who had participated in the multisite “Hydroxyurea Adherence for Personal Best in SCD” (HABIT) feasibility (6 months) and efficacy (12 months) trials. Pharmacy data were collected from the year prior to study entry through the duration of each trial. We also examined hydroxyurea dose at baseline, prescribing patterns (hydroxyurea formulation and dose prescribed), quantity of hydroxyurea dispensed, and number of daily capsules/tablets prescribed. Data were analyzed using descriptive statistics. Results On average, youth were prescribed 1095 ± 402 mg hydroxyurea per day, requiring ingestion of 3 or more capsules for 39.4% of youth. Frequently identified potential barriers were complex medication regimens in which dose of hydroxyurea differed by day of week (47.2%); receipt of an inadequate (< 30 days) supply of hydroxyurea from the pharmacy ≥ 3 times during record collection period (29.2%); and prescription of hydroxyurea suspension suggesting problems swallowing capsules (22.2%). In this sample, most youth were exclusively prescribed 500 mg capsules (62.5%), which was associated with complex medication regimens (RR 3.0, 95% CI 1.4‐6.7). Potential barriers were common, occurred at all levels and are potentially modifiable with targeted interventions at the treatment‐ and health system–related levels.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Background
Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy associated with morbidity and mortality. We sought to understand family planning intentions of parents of young ...children with SCD including the awareness of three reproductive options (adoption, in vitro fertilization with egg/sperm donation IVFD, in vitro fertilization IVF with preimplantation genetic testing IVF/PGT) to decrease the risk of having a child with SCD.
Procedure
Qualitative, semistructured, one‐on‐one interviews with 18 female parents of young children with SCD at an urban, tertiary care pediatric hospital.
Results
Half of the parents knew their hemoglobinopathy status or their partner's status before pregnancy. Eight parents chose to have no further children because of fear of SCD in another child. Awareness of reproductive options prior to study enrollment was limited. After brief introduction, 7 parents would consider adoption, 2 IVFD, and 10 IVF/PGT. Desire for a biological child, fear of parental jealousy, ethical or religious considerations, and cost affected the acceptability of these options. Participants universally wanted information about reproductive options available to others prior to pregnancy.
Conclusions
There is limited awareness and variable acceptability of alternative reproductive options available to decrease the risk of a future child having SCD. Participants universally endorsed the need for education regarding hemoglobinopathy status, SCD, and reproductive options prior to pregnancy because for many participants having a child with SCD affected their reproductive intentions. Educational interventions to ensure informed reproductive decision making should be sensitive to desires for a biological child, and ethical and financial considerations.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
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