ABSTRACT
Context: Somatrogon is a long-acting recombinant human growth hormone treatment developed as a once-weekly treatment for pediatric patients with growth hormone deficiency (GHD). Objective: Evaluate patient and caregiver perceptions of the treatment burden associated with the once-weekly somatrogon injection regimen vs a once-daily Somatropin injection regimen. Methods: Pediatric patients (≥3 to <18 years) with GHD receiving once-daily somatropin at enrollment were randomized 1:1 to Sequence 1 (12 weeks of once-daily Somatropin, then 12 weeks of once-weekly somatrogon) or Sequence 2 (12 weeks of once-weekly somatrogon, then 12 weeks of once-daily Somatropin). Treatment burden was assessed using validated questionnaires completed by patients and caregivers. The primary endpoint was the difference in mean overall life interference (LI) total scores after each 12-week treatment period (somatrogon vs Somatropin), as assessed by questionnaires. Results: Of 87 patients randomized to Sequence 1 (nâ =â 43) or 2 (nâ =â 44), 85 completed the study. Once-weekly somatrogon had a significantly lower treatment burden than once-daily Somatropin, based on mean overall LI total scores after somatrogon (8.63) vs Somatropin (24.13) treatment (mean difference -15.49; 2-sided 95% CI -19.71, -11.27; Pâ <â .0001). Once-weekly somatrogon was associated with greater convenience, higher satisfaction with treatment experience, and less LI. The incidence of treatment-emergent adverse events (TEAEs) for Somatropin and somatrogon was 44.2% and 54.0%, respectively. No severe or serious AEs were reported. Conclusion: In pediatric patients with GHD, once-weekly somatrogon had a lower treatment burden and was associated with a more favorable treatment experience than once-daily Somatropin.
ABSTRACT
CONTEXT: X-linked hypophosphatemia (XLH) is an inherited skeletal disorder that can lead to lifelong deleterious musculoskeletal and functional consequences. Although often perceived as a childhood condition, children and adults both experience the negative effects of XLH. Adolescents and young adults (AYAs) benefit from effective health care transition (HCT) preparation to support the transfer from pediatric- to adult-focused care. Whereas transition timelines, milestones, and educational tools exist for some chronic conditions, they do not meet the unique needs of patients with XLH. EVIDENCE ACQUISITION: To produce the first expert recommendations on HCT preparation for AYAs with XLH developed by clinical care investigators and transition experts, a formal literature search was conducted and discussed in an advisory board meeting in July 2020. A modified Delphi method was used to refine expert opinion and facilitate a consensus position. EVIDENCE SYNTHESIS: We identified the need for psychosocial and access-related resources for disease education, genetic counseling, family planning, and AYA emancipation from caregiver-directed care. Additionally, we recognized that it is necessary to facilitate communication with patients through channels familiar and accessible to AYAs and teach patients to advocate for their health care/access to specialists. CONCLUSION: Clear HCT preparation guidelines and treatment-related goals are defined. Individualized timelines and practical strategies for HCT preparation are proposed to optimize health outcomes resulting from continuous clinical care throughout the patient lifecycle. We provide an expert consensus statement describing a tailored HCT preparation program specifically for AYAs with XLH to aid in the effective transfer from pediatric- to adult-focused health care.
Subject(s)
Familial Hypophosphatemic Rickets/therapy , Practice Guidelines as Topic , Transition to Adult Care/standards , Adolescent , Child , Consensus , Familial Hypophosphatemic Rickets/diagnosis , Familial Hypophosphatemic Rickets/epidemiology , Humans , Young AdultSubject(s)
Blood Glucose Self-Monitoring/methods , Caregivers/psychology , Diabetes Mellitus, Type 1/blood , Hypoglycemia/prevention & control , Smartphone , Adolescent , Adult , Blood Glucose Self-Monitoring/psychology , Child , Child, Preschool , Fear , Female , Humans , Hypoglycemia/psychology , Hypoglycemic Agents/administration & dosage , Infant , Infant, Newborn , Insulin/administration & dosage , Insulin Infusion Systems , Male , Pilot ProjectsABSTRACT
BACKGROUND: The mySentry system (Medtronic Inc.) is the first to amplify and relay continuous glucose monitoring (CGM) and insulin pump data to a remote site within the house. Its usability and acceptability were evaluated in families having a child with type 1 diabetes. METHODS: Each enrolled family included a child (age 7-17 years) who used a Paradigm REAL-Time Revel sensor-augmented insulin pump (Medtronic). After a 1-week run-in phase, families set up and used the mySentry system for a 3-week study phase. Opinion surveys were completed by parents, and pump and CGM data were collected and analyzed retrospectively. No formal hypothesis testing was performed, and the study was not powered to detect changes in nocturnal glycemia. RESULTS: Thirty-five families completed the study. Enrolled children (61.1% female) had a mean (± standard deviation) age of 11.9 ± 2.70 years and a mean age at initiation of pump therapy of 7.1 ± 3.19 years. Baseline survey results indicated that most parents were fearful of their unawareness of their children's nocturnal glucose excursions. The mySentry system met the predefined acceptability criteria for general experience, product usability, and training materials. There were no unanticipated device-related adverse effects. Among children who experienced nocturnal hypo- or hyperglycemic episodes in both phases of the study, there was a trend toward less frequent and less prolonged episodes during mySentry use. CONCLUSION: The mySentry system met all predefined criteria for acceptability and did not demonstrate safety issues. Alerting parents to abnormal glucose values or trends may attenuate nocturnal hypoglycemia and hyperglycemia by prompting appropriate and timely intervention.
