scholarly journals Feasibility of Electronic Monitoring of Daily Oral Medications in Adolescents with Sickle Cell Disease

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4701-4701
Author(s):  
Lori E Crosby ◽  
Katherine M Kidwell ◽  
Aimee K Hildenbrand ◽  
Charles T. Quinn ◽  
Meghan E Mcgrady
Keyword(s):  
Medication Adherence ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Electronic Monitoring ◽  
Cell Disease ◽  
Oral Medications ◽  
Adherence Assessment ◽  
Monitoring Devices ◽  
Initial Uptake

Background: The field has made significant strides in understanding the mechanisms underlying pediatric sickle cell disease (SCD), and the coming years will likely see the approval of several new medications to treat SCD. The impact of these medications on clinical outcomes, however, will be dependent on patient adherence. Adolescents with SCD are at particular risk for non-adherence, and as disease management increasingly includes self-administration of oral medications, adherence assessment will become a critical component of research on medication effectiveness and clinical care. While electronic monitoring devices (bottles with computer chips that record date- and time-stamps of device openings) such as MEMS® bottles are considered the "gold standard" for adherence assessment in other populations, their feasibility among adolescents with SCD remains unknown. Objectives: The primary aims of this study were to examine data on MEMS® bottle use among adolescents (ages 13-21 years) with SCD to: 1) evaluate the feasibility of MEMS® bottle use; and 2) elicit barriers and facilitators to MEMS® bottle use. Methods: As part of a larger study of a self-management intervention, adolescents were asked to use a MEMS® bottle to store and administer their daily oral medication (hydroxyurea or deferasirox) for the 18-week study duration. The larger study included baseline, post-treatment, and follow-up assessments, at which adolescents were asked to provide their MEMS® bottle for data download. Descriptive statistics were calculated to assess multiple domains of MEMS® bottle feasibility including: initial uptake (% enrolled), MEMS® initiation (% initiated), and MEMS® sustained use (% completed; % provided bottles for download ±2 weeks of scheduled study visit = "on time"). Barriers and facilitators to MEMS® use were elicited via adolescent self-report. Results: In the larger study, 18 non-Hispanic African-American adolescents (M = 17.8 years, SD = 2.6; 61% male) with HbSS were asked to use a MEMS® bottle to store their hydroxyurea (n = 14) or deferasirox (n = 4). Initial uptake was 94.7%, with 18 of 19 eligible adolescents enrolling in the study. Of the 18 enrolled adolescents, all initiated MEMS® use (100%) and 11 sustained MEMS® use through the final study endpoint (61.1%). Eight (44.4%) and 2 (11.1%) adolescents provided their MEMS® bottle for download "on time" at post-treatment (42 days) and follow-up (126 days), respectively. Barriers to MEMS® use included medication changes (i.e., medication holds, dose timing changes) and transitioning from pediatric to adult care. Facilitators included tip sheets (e.g., places to store bottle, reminder to place refills in bottle) and reminder calls. Participants took a median of 26.2% of doses using the MEMS® across the 126 days (SD = 22.4; range 0.79% - 79.3%), illustrating that electronic monitoring devices are not a solution to medication adherence, but are one component of an adherence support strategy. See Figure 1 for adherence rates over time. Discussion: Data suggest that MEMS® are acceptable to adolescents with SCD. Ensuring sustained MEMS® use, however, will likely require additional supports. For example, electronic monitors which automatically transmit data in real-time via Bluetooth or cellular connections would eliminate the need for adolescents to bring bottles to study visits for data downloads. Researchers and clinicians interested in using electronic monitoring devices are encouraged to consider remote monitoring capabilities along with other device features when selecting a product. Larger studies are needed to evaluate concordance between rates of adherence obtained via electronic monitoring and other assessments and to validate electronically-monitored medication adherence with clinically-relevant outcomes for adolescents with SCD. Figure 1 Disclosures Quinn: Celgene: Membership on an entity's Board of Directors or advisory committees; Amgen: Other: Research Support.

scholarly journals Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?

Children ◽  
2021 ◽  
Vol 8 (2) ◽  
pp. 143
Author(s):  
Julie Sommet ◽  
Enora Le Roux ◽  
Bérengère Koehl ◽  
Zinedine Haouari ◽  
Damir Mohamed ◽  
...  
Keyword(s):  
Statistical Analysis ◽  
Sickle Cell Disease ◽  
Cohort Studies ◽  
Statistical Methods ◽  
Sickle Cell ◽  
Biological Parameters ◽  
Analysis Method ◽  
Cell Disease ◽  
Statistical Analysis Method

Background: Many pediatric studies describe the association between biological parameters (BP) and severity of sickle cell disease (SCD) using different methods to collect or to analyze BP. This article assesses the methods used for collection and subsequent statistical analysis of BP, and how these impact prognostic results in SCD children cohort studies. Methods: Firstly, we identified the collection and statistical methods used in published SCD cohort studies. Secondly, these methods were applied to our cohort of 375 SCD children, to evaluate the association of BP with cerebral vasculopathy (CV). Results: In 16 cohort studies, BP were collected either once or several times during follow-up. The identified methods in the statistical analysis were: (1) one baseline value per patient (2) last known value; (3) mean of all values; (4) modelling of all values in a two-stage approach. Applying these four different statistical methods to our cohort, the results and interpretation of the association between BP and CV were different depending on the method used. Conclusion: The BP prognostic value depends on the chosen statistical analysis method. Appropriate statistical analyses of prognostic factors in cohort studies should be considered and should enable valuable and reproducible conclusions.

scholarly journals Implementation of an Educational Intervention to Optimize Self-Management and Transition Readiness in Adolescents with Sickle Cell Disease

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3536-3536
Author(s):  
Cecelia Calhoun ◽  
Regina Abel ◽  
Hai Anh Pham ◽  
Shomari Thompson ◽  
Allison A King
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Psychosocial Development ◽  
Sexual Development ◽  
Independent Living ◽  
Self Management ◽  
Cell Disease ◽  
Adult Care ◽  
Transition Readiness

Abstract Background: The transition from the pediatric setting to adult care is a challenge for many adolescents with chronic disease. Patients with sickle cell disease (SCD) represent a unique cohort as the timing of psychosocial development of adolescence often coincides with worsening end organ damage. Previously, we used the Adolescent Autonomy Checklist (AAC) modified to include SCD specific tasks that patients with SCD need to practice in order to transition to adult healthcare and independent living. This study sought to use the AAC to measure the effects of skill based educational handouts on improving self-management and transition readiness in adolescents with SCD. Methods: This was a single center, retrospective study approved by the Washington University Institutional Review Board. Inclusion criteria were patients with SCD, age 13-21 years, and completion of pre and post assessments. As standard care, patients from a pediatric hematology clinic completed the AAC-SCD. The AAC-SCD assesses skill level in twelve domains (Table). The tool includes 100 items, and users check "can do already" or "needs practice" for each item. After review with the coordinator, participants were given skill-based handouts based on up to five noted deficits. Patients completed the AAC-SCD at the subsequent clinic visit. In addition to baseline and follow up AAC-SCD data, medical and demographic data were collected via chart abstraction. All data were entered into SPSS for statistical analysis, including descriptives, paired sample T-tests, and bivariate Pearson's correlations. Results: A total of 61 patients completed baseline and follow up. Of those participants, 49.2% were female. The mean age was 15.4 (+ 2.2) years. The genotypic distribution was as follows: 67.2% HbSS, 19.7% HbSC, 3.3% HbS-beta-thal+ and 9.8% HbS-beta-thal0. The majority of patients received healthcare coverage via Medicaid (52.5%), private insurance (45.6%) and 1.6% had no insurance coverage. Twenty-five patients (42.0 %) had a history of stroke or silent cerebral infarct and 34 (55.7%) were currently taking or were previously prescribed hydroxyurea. Formal academic support (IEP or 504 Plan) was reported for 20 (32.8%) of patients. At baseline, patients needed the most help with skills in the kitchen, housekeeping, personal care and leisure. Statistically significant improvements (p< 0.05) occurred in skills related to laundry, housekeeping, healthcare, sexual development and living arrangements. Modest sized and statistically significant correlation between the receipt of the educational handouts and decreased number of items marked "needs help" occurred in the areas of money management (r=-0.27, p=0.044), vocational skills (r=-0.27, p=0.046;) and laundry (r=0.32, p=0.015). A post hoc analysis by age groups 13-15 (n= 34),16-18 (n=24) and 19-21 (n=3) showed a decreased amount of items marked "needs help" in the areas of sexual development for both 13-15 year olds (r=0.42, p=0.024) and 16-18 year olds (r=0.93, p=0.001) as well. Conclusion: Transition skills improved over time among adolescents with SCD. While we cannot say for certain if gains in knowledge occur with age as development progresses or if a formal transition program can be credited, providing educational materials on transition related skills within a clinic setting was associated with significant improvements in three of the domains. Our preliminary data offers insight into what skill deficits may be most amenable to educational interventions based on age group. As is the case with medical management, the development of a multimodal intervention is needed to prepare adolescents with SCD to transition to adult care and independent living. Clinic based education is a simple intervention that could be one component of future approaches to transition. Disclosures No relevant conflicts of interest to declare.

scholarly journals Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 68-68 ◽  
Author(s):  
Janet L. Kwiatkowski ◽  
Julie Kanter ◽  
Heather J. Fullerton ◽  
Jenifer Voeks ◽  
Ellen Debenham ◽  
...  
Keyword(s):  
Ischemic Stroke ◽  
High Risk ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Stroke Prevention ◽  
Cell Disease ◽  
Clinical Series ◽  
The Mean

Abstract Background: The Stroke Prevention Trial in Sickle Cell Anemia (STOP) and Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) established routine transcranial Doppler ultrasound (TCD) screening with indefinite chronic red cell transfusions (CRCT) for children with abnormal TCD as standard of care. To identify children at high-risk of stroke, annual TCD screening is recommended from ages 2 to 16 years, with more frequent monitoring if the result is not normal. A reduction in stroke incidence in children with SCD has been reported in several clinical series and analyses utilizing large hospital databases when comparing rates before and after the publication of the STOP study in 1998. We sought to determine the rate of first ischemic stroke in a multicenter cohort of children who had previously participated in the STOP and/or STOP 2 trials and to determine whether these strokes were screening or treatment failures. Subjects and Methods: Between 1995 and 2005, STOP and STOP 2 (STOP/2) were conducted at 26 sites in the US and Canada. These studies included 3,835 children, ages 2 to 16 y with SCD type SS or S-beta-0-thalassemia. Participation in STOP/2 ranged from a single screening TCD to randomization. STOP 2 also had an observational arm for children on CRCT for abnormal TCD whose TCD had not reverted to normal. The Post-STOP study was designed to follow-up the outcomes of children who participated in one or both trials. 19 of the 26 original study sites participated in Post-STOP, contributing a total of 3,539 (92%) of the STOP/2 subjects. After exit from STOP/2, these children received TCD screening and treatment according to local practices. Data abstractors visited each clinical site and obtained retrospective data from STOP/2 study exit to 2012-2014 (depending on site) including follow-up TCD and brain imaging results, clinical information, and laboratory results. Two vascular neurologists, blinded to STOP/2 status and prior TCD and neuroimaging results, reviewed source records to confirm all ischemic strokes, defined as a symptomatic cerebral infarction; discordant opinions were resolved through discussion. For the first Post-STOP ischemic stroke, prior TCD result and treatment history subsequently were analyzed. Results: Of the 3,539 subjects, follow-up data were available for 2,850 (81%). Twelve children who had a stroke during STOP or STOP2 were excluded from these analyses resulting in data on 2,838 subjects. The mean age at the start of Post-STOP was 10.5 y and mean duration of follow-up after exiting STOP/2 was 9.1 y. A total of 69 first ischemic strokes occurred in the Post-STOP observation period (incidence 0.27 per 100 pt years). The mean age at time of stroke was 14.4±6.2 (median 13.8, range 3.5-28.9) y. Twenty-five of the 69 patients (36%) had documented abnormal TCD (STOP/2 or Post-STOP) prior to the stroke; 15 (60%) were receiving CRCT and 9 (36%) were not (treatment data not available for 1 subject). Among the 44 subjects without documented abnormal TCD, 29 (66%) had not had TCD re-screen in the Post-STOP period prior to the event; 7 of these 29 (24%) were 16 y or older at the start of Post-STOP, which is beyond the recommended screening age. Four of the 44 (9%) patients had inadequate TCD in Post-STOP (1 to 10.7 y prior to event). Six (14%) had normal TCD more than a year before the event (1.2 - 4 y); all but one of these children were younger than 16 y at the time of that TCD. Only 5 (11%) had a documented normal TCD less than 1 year prior to the event. Conclusions: In the Post-STOP era, the rate of first ischemic stroke was substantially lower than that reported in the Cooperative Study of Sickle Cell Disease, prior to implementation of TCD screening. Many (39%) of the Post-STOP ischemic strokes were associated with a failure to re-screen according to current guidelines, while only 11% occurred in children who had had recent low-risk TCD. Among those known to be at high risk prior to stroke, treatment refusal or inadequate treatment may have contributed. While TCD screening and treatment are effective at reducing ischemic stroke in clinical practice, significant gaps in screening and treatment, even at sites experienced in the STOP protocol, remain to be addressed. Closing these gaps should provide yet further reduction of ischemic stroke in SCD. Disclosures No relevant conflicts of interest to declare.

Perspective From the American Academy of Pediatrics

PEDIATRICS ◽  
1989 ◽  
Vol 83 (5) ◽  
pp. 913-914
Author(s):  
Neil A. Holtzman
Keyword(s):  
Sickle Cell Disease ◽  
American Academy ◽  
Sickle Cell ◽  
Second Phase ◽  
Pilot Program ◽  
Cooperative Study ◽  
Cell Disease ◽  
Pilot Studies ◽  
American Academy Of Pediatrics

Ten years ago, the initial report of the Committee on Genetics of the American Academy of Pediatrics was published, drawing heavily on the landmark report published 2 years earlier by the National Academy of Sciences. That report stated that new mass screening tests should not be implemented without pilot studies or facilities for follow-up. I would like to deal with the efficacy of screening as determined by pilot studies, the effectiveness of routine screening, and the importance of follow-up. The Cooperative Study of Sickle Cell Disease was essentially the first phase of a large pilot program that systematically demonstrated that sepsis, meningitis, and acute splenic sequestration occurred in young infants with sickle cell disease. Previous reports suggested, and the cooperative study corroborated, that without prior diagnosis many of these infants would die. The randomized trial of oral penicillin prophylaxis was the second phase of the pilot program, demonstrating that early treatment significantly reduced mortality. Thus, the results of this pilot program, conducted with support of the National Institutes of Health, documented that newborn screening for sickle cell disease satisfied a major criterion for screening: An intervention capable of reducing mortality would be efficacious if applied before the usual age of clinical diagnosis. The pilot program was conducted under ideal conditions. The findings are not enough to conclude that screening for sickle cell disease and other hemoglobinopathies will always be effective when performed routinely.

scholarly journals Barriers to Pediatric Sickle Cell Disease Guideline Recommendations

2019 ◽  
Vol 6 ◽  
pp. 2333794X1984702 ◽  
Author(s):  
Michael D. Cabana ◽  
Julie Kanter ◽  
Anne M. Marsh ◽  
Marsha J. Treadwell ◽  
Michael Rowland ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Support Staff ◽  
Cell Disease ◽  
National Guidelines ◽  
Convenience Sample ◽  
Different Types ◽  
Pediatric Sickle Cell Disease

National guidelines recommend that providers counsel all patients with sickle cell anemia about hydroxyurea (HU) therapy and screen children with sickle cell anemia annually for the risk of stroke with transcranial Doppler (TCD). We surveyed a national convenience sample of sickle cell disease clinicians to assess factors associated with low adherence. Adherence was 46% for TCD screening. Low adherence was associated with a lack of outcome expectancy (eg, a belief that there would be poor patient follow-up to TCD testing; P < .05). Adherence was 72% for HU counseling. Practice barriers (eg, lack of support staff or time) and a lack of agreement with HU recommendations were associated with low adherence ( P < .05). This study demonstrates that different types of strategies are needed to improve TCD screening (to address follow-up and access to testing) versus HU counseling (to address physician agreement and practice barriers).

scholarly journals A modified transfusion program for prevention of stroke in sickle cell disease

Blood ◽  
1992 ◽  
Vol 79 (7) ◽  
pp. 1657-1661 ◽  
Author(s):  
AR Cohen ◽  
MB Martin ◽  
JH Silber ◽  
HC Kim ◽  
K Ohene-Frempong ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cerebral Infarction ◽  
Sickle Cell ◽  
Recurrent Stroke ◽  
High Rate ◽  
First Year ◽  
Cell Disease ◽  
Red Blood Cell Transfusions ◽  
Major Reduction

Abstract Regular red blood cell transfusions reduce the rate of recurrent cerebral infarction in sickle cell disease but lead to accumulation of excessive iron. We studied the effect on the prevention of recurrent stroke and the volume of blood transfused of a modified transfusion program in which the pretransfusion percentage of hemoglobin S (HbS) was maintained at 50%, rather than the conventional 30%. Fifteen patients with sickle cell disease and cerebral infarction who had been free of recurrent stroke for at least 4 years during which the pretransfusion HbS was maintained below 30% were assigned to a transfusion program in which the HbS was allowed to increase to 50%. Transfusion regimens included simple transfusion and manual and automated partial exchange transfusion. The duration of follow-up was 14 to 130 months with a median duration of 84 months. None of the 15 patients had a recurrent cerebral infarction during 1,023 patient- months in which the target pretransfusion HbS was 50%. Analysis of this finding, using a binomial distribution, indicates that there is less than a 5% chance that the risk per patient of recurrent stroke in the first year of the modified transfusion program is greater than 18%. One 23-year-old patient had a fatal intraventricular hemorrhage when the HbS was 30% and a 21-year-old patient had a fatal subarachnoid hemorrhage in the 40th week of pregnancy when the HbS was 29%. Blood requirements with simple transfusions decreased by 17% to 48% (mean 31%) when the target pretransfusion HbS level was increased from 30% to 50% (P less than .001). Manual or automated partial exchange transfusions and a target HbS level of 50% in eight patients reduced blood requirements by 33% to 99% (mean 67%) in comparison with simple transfusion and a target HbS level of 30% (P less than .001). This study offers evidence that a target pretransfusion HbS level of 50% affords a continuing high rate of protection against recurrent cerebral infarction in sickle cell disease after 4 years of a conventional transfusion program. Increasing the target HbS level from 30% to 50% provides a major reduction in blood requirements and lowers the rate of iron accumulation.

scholarly journals Systematic and Meta-Analytic Review: Medication Adherence Among Pediatric Patients With Sickle Cell Disease

2015 ◽  
Vol 41 (4) ◽  
pp. 406-418 ◽  
Author(s):  
Kristin Loiselle ◽  
Jennifer L. Lee ◽  
Lauren Szulczewski ◽  
Sarah Drake ◽  
Lori E. Crosby ◽  
...  
Keyword(s):  
Medication Adherence ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Cell Disease ◽  
Analytic Review
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