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Transitioning from pediatric to adult health care with familial hypercholesterolemia: Listening to young adult and parent voices.

ABSTRACT: BACKGROUND:Young adults with familial hypercholesterolemia (FH) are at a critical period for establishing behaviors to promote future cardiovascular health. OBJECTIVE:To examine challenges transitioning to adult care for young adults with FH and parents of FH-affected young adults in the context of 2 developmental tasks, transitioning from childhood to early adulthood and assuming responsibility for self-management of a chronic disorder. METHODS:Semistructured, qualitative interviews were conducted with 12 young adults with FH and 12 parents of affected young adults from a pediatric subspecialty preventive cardiology program in a northeastern academic medical center. Analyses were conducted using a modified grounded theory framework. RESULTS:Respondents identified 5 challenges: (1) recognizing oneself as a decision maker, (2) navigating emerging independence, (3) prioritizing treatment for a chronic disorder with limited signs and symptoms, (4) managing social implications of FH, and (5) finding credible resources for guidance. Both young adults and parents proposed similar recommendations for addressing these challenges, including the need for family and peer involvement to establish and maintain diet and exercise routines and to provide medication reminders. Systems-level recommendations included early engagement of adolescents in shared decision-making with health care team; providing credible, educational resources regarding FH; and using blood tests to track treatment efficacy. CONCLUSION:Young adults with FH transitioning to adult care may benefit from explicit interventions to address challenges to establishing healthy lifestyle behaviors and medication adherence as they move toward being responsible for their medical care. Further research should explore the efficacy of recommended interventions.

SUBMITTER: Sliwinski SK

PROVIDER: S-EPMC6547361 | biostudies-literature | 2017 Jan - Feb

REPOSITORIES: biostudies-literature

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Transitioning from pediatric to adult health care with familial hypercholesterolemia: Listening to young adult and parent voices.

Sliwinski Samantha K SK Gooding Holly H de Ferranti Sarah S Mackie Thomas I TI Shah Supriya S Saunders Tully T Leslie Laurel K LK

Journal of clinical lipidology 20161109 1

<h4>Background</h4>Young adults with familial hypercholesterolemia (FH) are at a critical period for establishing behaviors to promote future cardiovascular health.<h4>Objective</h4>To examine challenges transitioning to adult care for young adults with FH and parents of FH-affected young adults in the context of 2 developmental tasks, transitioning from childhood to early adulthood and assuming responsibility for self-management of a chronic disorder.<h4>Methods</h4>Semistructured, qualitative ...[more]

PMID: 28391881

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Project description:BackgroundHomozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) levels due to profoundly defective LDL receptor (LDLR) function. Given that severely elevated LDL-C starts in utero, atherosclerosis often presents during childhood or adolescence, creating a largely unmet need for aggressive LDLR-independent lipid-lowering therapies in young patients with HoFH. Here we present the first evaluation of the efficacy and safety of evinacumab, a novel LDLR-independent lipid-lowering therapy, in pediatric patients with HoFH from parts A and B of a 3-part study.MethodsThe phase 3, part B, open-label study treated 14 patients 5 to 11 years of age with genetically proven HoFH (true homozygotes and compound heterozygotes) with LDL-C >130 mg/dL, despite optimized lipid-lowering therapy (including LDLR-independent apheresis and lomitapide), with intravenous evinacumab 15 mg/kg every 4 weeks.ResultsEvinacumab treatment rapidly and durably (through week 24) decreased LDL-C with profound reduction in the first week, with a mean (SE) LDL-C reduction of -48.3% (10.4%) from baseline to week 24. ApoB (mean [SE], -41.3% [9.0%]), non-high-density lipoprotein cholesterol (-48.9% [9.8%]), and total cholesterol (-49.1% [8.1%]) were similarly decreased. Treatment-emergent adverse events were reported in 10 (71.4%) patients; however, only 2 (14.3%) reported events that were considered to be treatment-related (nausea and abdominal pain). One serious treatment-emergent adverse event of tonsillitis occurred (n=1), but this was not considered treatment-related.ConclusionsEvinacumab constitutes a new treatment for pediatric patients with HoFH and inadequately controlled LDL-C despite optimized lipid-lowering therapy, lowering LDL-C levels by nearly half in these extremely high-risk and difficult-to-treat individuals.RegistrationURL: https://www.clinicaltrials.gov; Unique identifier: NCT04233918.

Project description:BACKGROUND:Hospital charges and lengths of stay may be greater when adults with chronic conditions are admitted to children's hospitals. Despite multiple efforts to improve pediatric-adult healthcare transitions, little guidance exists for transitioning inpatient care. OBJECTIVE:This study sought to characterize pediatricadult inpatient care transitions across general pediatric services at US children's hospitals. DESIGN, SETTING AND PARTICIPANTS:National survey of inpatient general pediatric service leaders at US children's hospitals from January 2016 to July 2016. MEASUREMENTS:Questionnaires assessed institutional characteristics, presence of inpatient transition initiatives (having specific process and/or leader), and 22 inpatient transition activities. Scales of highly correlated activities were created using exploratory factor analysis. Logistic regression identified associations between institutional characteristics, transition activities, and presence of an inpatient transition initiative. RESULTS:Ninety-six of 195 children's hospitals responded (49.2% response rate). Transition initiatives were present at 38% of children's hospitals, more often when there were dual-trained internal medicine-pediatrics providers or outpatient transition processes. Specific activities were infrequent and varied widely from 2.1% (systems to track youth in transition) to 40.5% (addressing potential insurance problems). Institutions with initiatives more often consistently performed the majority of activities, including using checklists and creating patient-centered transition care plans. Of remaining activities, half involved transition planning, the essential step between readiness and transfer. CONCLUSIONS:Relatively few inpatient general pediatric services at US children's hospitals have leaders or dedicated processes to shepherd transitions to adultoriented inpatient care. Across institutions, there is a wide variability in performance of activities to facilitate this transition. Feasible process and outcome measures are needed.

Project description:ImportanceMany pediatric patients with heterozygous familial hypercholesterolemia (HeFH) cannot reach recommended low-density lipoprotein cholesterol (LDL-C) concentrations on statins alone and require adjunct lipid-lowering therapy (LLT); the use of alirocumab in pediatric patients requires evaluation.ObjectiveTo assess the efficacy of alirocumab in pediatric patients with inadequately controlled HeFH.Design, setting, and participantsThis was a phase 3, randomized clinical trial conducted between May 2018 and August 2022 at 43 centers in 24 countries. Pediatric patients aged 8 to 17 years with HeFH, LDL-C 130 mg/dL or greater, and receiving statins or other LLTs were included. Following consecutive enrollment into dosing cohorts, 25 of 99 patients screened for dosing every 2 weeks (Q2W) failed screening; 25 of 104 patients screened for dosing every 4 weeks (Q4W) failed screening. A total of 70 of 74 Q2W patients (95%) and 75 of 79 Q4W patients (95%) completed the double-blind period.InterventionsPatients were randomized 2:1 to subcutaneous alirocumab or placebo and Q2W or Q4W. Dosage was based on weight (40 mg for Q2W or 150 mg for Q4W if <50 kg; 75 mg for Q2W or 300 mg for Q4W if ≥50 kg) and adjusted at week 12 if LDL-C was 110 mg/dL or greater at week 8. After the 24-week double-blind period, patients could receive alirocumab in an 80-week open-label period.Main outcomes and measuresThe primary end point was percent change in LDL-C from baseline to week 24 in each cohort.ResultsAmong 153 patients randomized to receive alirocumab or placebo (mean [range] age, 12.9 [8-17] years; 87 [56.9%] female), alirocumab showed statistically significant reductions in LDL-C vs placebo in both cohorts at week 24. Least squares mean difference in percentage change from baseline was -43.3% (97.5% CI, -56.0 to -30.7; P < .001) Q2W and -33.8% (97.5% CI, -46.4 to -21.2; P < .001) Q4W. Hierarchical analysis of secondary efficacy end points demonstrated significant improvements in other lipid parameters at weeks 12 and 24 with alirocumab. Two patients receiving alirocumab Q4W experienced adverse events leading to discontinuation. No significant difference in adverse event incidence was observed between treatment groups. Open-label period findings were consistent with the double-blind period.Conclusions and relevanceThe findings in this study indicate that alirocumab Q2W or Q4W significantly may be useful for reducing LDL-C and other lipid parameters and be well tolerated in pediatric patients with HeFH inadequately controlled with statins.Trial registrationClinicalTrials.gov Identifier: NCT03510884.

Dataset Information

Transitioning from pediatric to adult health care with familial hypercholesterolemia: Listening to young adult and parent voices.

Publications

Transitioning from pediatric to adult health care with familial hypercholesterolemia: Listening to young adult and parent voices.

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