Functioning and well-being in older children and adolescents with achondroplasia: A qualitative study.

The study aimed to explore how having achondroplasia affects older children and adolescents' day-to-day functioning and well-being. Individual/focus group interviews were conducted with older children/adolescents between the ages of 9 to <18 years and diagnosed with achondroplasia to elicit key concepts. An adapted grounded theory approach informed the qualitative analysis of interview data. Thirty-two children and adolescents completed interviews. Study results revealed five impact domains, including physical health, functioning, school impacts, emotional well-being, and social well-being. Frequently reported impacts on physical health included low stamina/tiring easily (81%) and back pain (69%). Key impacts in the functioning domain were difficulty with reaching objects or high places (84%) and walking long distances (75%). Emotional impacts included feeling different (63%), worried/scared (47%), and embarrassed/self-conscious (47%). Impacts on social well-being included difficulty with sports or physical play (81%) and others treating child as younger than their actual age (75%). The most frequent school impact was trouble participating in physical education (81%). A preliminary theoretical model depicting the experiences of older children/adolescents with achondroplasia was constructed based on the analysis. The preliminary theoretical model of older children and adolescents' experiences of living with achondroplasia may be used to inform future research and clinical practice.

A qualitative study of the impacts of having an infant or young child with achondroplasia on parent well-being.

BACKGROUND: Currently, there is limited research on how having a child diagnosed with achondroplasia affects parents' lives. The purpose of the study was to investigate the experiences of parents of infants and young children less than two years of age with achondroplasia. METHODS: Concept elicitation interviews were conducted with parents of children less than 2 years of age with achondroplasia in the United States and Spain. Using grounded theory methods modified for health outcomes research, a qualitative analysis of interview transcripts was conducted. Based on the qualitative analysis, a preliminary theoretical model of the experiences of parents of infants and young children with achondroplasia was developed. RESULTS: Fifteen parents, including 14 mothers and 1 father from 15 unique families, participated in individual or focus group concept elicitation interviews in the US (n = 9) and Spain (n = 6). The qualitative analysis identified four key parent impact domains, which included caretaking responsibilities, impacts on emotional well-being, having worries and concerns about their child, and impacts on daily well-being. Frequently discussed caretaking responsibilities among parents were managing child's medical care/treatment (93%), obtaining adaptations/items for child (73%), and monitoring child to avoid complications of achondroplasia (67%). Emotional impacts included feeling stressed/overwhelmed (67%), depressed/sad (40%), and anxious/nervous (33%). Worries and concerns included worry/concern about the future (100%), concerns regarding child's physical health (87%), worry about child's social well-being (80%), concern for child's emotional well-being (73%), and worry about child being able to function independently (67%). Daily well-being impacts included family strain (60%), missed work time (47%), and missed/limited social activities (33%). Based on the qualitative findings, a preliminary theoretical model depicting the experiences of parents of infants and young children with achondroplasia was created. CONCLUSIONS: The study sheds light on the range of impacts that parents of infants and young children with achondroplasia may experience, including caretaking responsibilities, impacts on emotional well-being, worries/concerns regarding their child, and impacts on daily well-being. The theoretical model of parent experiences may provide a helpful framework for informing future research and clinical practice.

Assessing physical symptoms, daily functioning, and well-being in children with achondroplasia.

This study's purpose was to provide qualitative evidence to support the development of two observer-reported outcome measures assessing the physical symptoms/complications of achondroplasia in children and impacts on children's quality of life. Individual/focus group concept elicitation interviews were conducted with parents of children aged 2 to <12 years with achondroplasia and experts. Qualitative analysis of transcripts, based on an adapted grounded theory approach, informed item generation and measure development. Cognitive debriefing (CD) interviews were conducted with parents to confirm relevance and understanding. Thirty-six parents participated in concept elicitation interviews. The analysis identified major physical symptoms/complications and impacts of achondroplasia, which informed the development of the Achondroplasia Child Experience Measures (ACEMs): ACEM-Symptom and ACEM-Impact. ACEM-Symptom was comprised of eight major symptoms/complications including pain (58%), ear infections/fluid in ear (56%), and low stamina/tiring easily (56%). ACEM-Impact consisted of 31 major impacts in the domains of daily functioning, emotional well-being, social well-being, and need for assistance/adaptive devices. Impacts on functioning included difficulty reaching objects/high places (89%) and toileting (67%). Emotional impacts included feeling different (53%) and feeling frustrated/annoyed (47%). Social impacts included difficulty participating in sports/physical play (86%) and being treated as younger than age (83%). Following CD interviews with 16 additional parents, validation-ready ACEM measures were generated. The study improves our understanding of the experiences of children with achondroplasia and provides evidence supporting the content validity of the ACEMs. Validated ACEMs may be used to assess potential benefits of future treatments for comorbidities of achondroplasia.

Assessing the impacts of having a child with achondroplasia on parent well-being.

PURPOSE: This study's purpose was to develop a better understanding of the experiences of parents of children with achondroplasia and to provide qualitative evidence to support the development of a patient-reported outcome (PRO) measure of parent impacts. METHODS: Concept elicitation (CE) individual/focus group interviews were conducted with parents of children aged 2 to < 12 years with achondroplasia in the United States and Spain. The qualitative analysis informed the PRO measure development. Cognitive debriefing (CD) interviews were conducted to ensure parent understanding and item relevance. RESULTS: Thirty-six parents participated in individual/focus group CE interviews. The analysis identified parent impacts in four domains, including caretaking responsibilities, emotional well-being, family, and work, and results informed the development of the Achondroplasia Parent Experience Measure (APEM). Caretaking responsibilities included managing child's medical care (92%), helping child with self-care (67%), advocating for child (64%), assisting child (56%), and observing/monitoring child (e.g., to ensure safety; 47%). Impacts on parents' emotional well-being included worry about the future (75%), worry about child's physical health (67%), safety concerns (50%), feeling stressed/overwhelmed (44%), and worry about child's social relationships (42%). Impacts on family and work included family strain (56%), limiting/adapting family activities (42%), and missed work time (50%). CD interviews with an additional 16 parents of children with achondroplasia confirmed understanding and item relevance. CONCLUSION: The results improve our understanding of the experiences of parents of children with achondroplasia and provide qualitative evidence to support the content validity of the APEM. A psychometric study is needed to validate the measure.

Post-Basal Insulin Intensification and Healthcare Resource Use in Type 2 Diabetes: A Web-Based Physician Survey in the United States and United Kingdom.

INTRODUCTION: Currently, there is limited knowledge of the healthcare resources and time needed to intensify patients with type 2 diabetes (T2D) treated with basal insulin to more complex treatment regimens. The purpose of the study was to investigate physicians' perspectives on the time and healthcare resources required for post-basal insulin intensification to basal-bolus and to basal in combination with a glucagon-like peptide-1 receptor agonist (GLP-1) regimens. The study also examined referrals to specialists for intensification and patient challenges with intensification. METHODS: A web-based survey of physicians was conducted in the United Kingdom (UK) and the United States (USA). RESULTS: A total of 458 physicians completed the survey, including general practitioners (58.5%) and specialists (endocrinologists/diabetologists; 41.5%). On average, 7.0 healthcare provider (HCP) visits (SD 3.7) over 30.1 weeks (SD 17.4) were required to intensify to a basal-bolus regimen, while 5.7 HCP visits (SD 3.8) over 23.5 weeks (SD 15.2) were needed to intensify to basal insulin in combination with GLP-1. Referral to a specialist for intensification required on average an additional 8 weeks of wait time before intensification. Physicians reported that the complexity of the basal-bolus regimen and frequent injections were key challenges for T2D patients intensifying to basal-bolus, while frequent injections and side effects were key challenges for those intensifying with GLP-1. CONCLUSION: Less complex regimens for intensification following basal insulin may help reduce the time and healthcare resources required for intensification and address some of the challenges T2D patients face when intensifying to basal-bolus or basal with GLP-1. FUNDING: Novo Nordisk, A/S.

Diabetes Management and Healthcare Resource Use When Intensifying from Basal Insulin to Basal-Bolus: A Survey of Type 2 Diabetes Patients.

INTRODUCTION: Currently, there is limited knowledge about the experiences and challenges type 2 diabetes (T2D) patients face when intensifying from basal insulin to more complex regimens. The purpose of this study was to examine the experiences of adults with T2D who have been intensified to a basal-bolus insulin regimen, including challenges related to intensification, medication adherence issues, non-persistence, and healthcare resource use related to intensification. METHODS: A web-based survey of adults diagnosed with T2D and currently treated with basal insulin was conducted in the UK and the USA. The analysis sample was restricted to respondents with current/recent basal-bolus treatment (n = 398) and divided into three analysis groups: (1) "basal-bolus adherent" (current basal-bolus treatment with at least 90% adherence); (2) "basal-bolus non-adherent" (current basal-bolus treatment with less than 80% adherence); and (3) "stopped bolus" (discontinued bolus in past 12 months). RESULTS: Basal-bolus non-adherent respondents reported fewer discussions with their healthcare providers (HCPs) before starting bolus and more frequent difficulties with and worries about taking bolus insulin compared to basal-bolus adherent and stopped bolus groups. The most frequently reported reasons for discontinuing bolus were related to the complicated nature of regimen, including too complicated to calculate bolus doses (25.7%), too complicated to regulate food in relation to bolus (20.7%), and too complicated to keep track of taking two different insulins (18.6%). Respondents who stopped bolus reported more frequent HCP visits related to diabetes compared to the basal-bolus adherent and basal-bolus non-adherent groups. CONCLUSION: Results suggest that the complicated nature of basal-bolus therapy contributes to the difficulties that T2D patients have with the regimen and to non-persistence. Physician and patient education may help patients address these treatment challenges to improve basal-bolus adherence and persistence, which could reduce healthcare resource use and costs. Less complex regimens may be appropriate for patients with persistent treatment difficulties. FUNDING: Novo Nordisk A/S.

Postprandial glucose and healthcare resource use: a cross-sectional survey of adults with diabetes treated with basal-bolus insulin.

AIMS: Postprandial glucose (PPG) control is a well-known challenge for diabetes management, yet there is limited research on how PPG affects healthcare resource utilization. This study investigated the association between PPG levels and healthcare resource utilization among adults with diabetes treated with basal-bolus insulin. METHODS: A web-based, cross-sectional survey (n = 940) of adults with type 1 or type 2 diabetes and treated with basal and bolus insulins was conducted in Italy, the UK, and the US. Descriptive analyses included frequencies, cross-tabulations, and comparison of means. Incidence-rate ratios (IRR) were calculated using negative binomial regression analysis to investigate the relationship between elevated PPG and healthcare resource utilization. Models controlled for demographic characteristics, duration of diabetes, and diabetes-related complications. RESULTS: Among respondents who measured PPG regularly and reported their two highest PPG values in the past week (n = 691), the mean average highest PPG value was 11.9 mmol/L (SD = 4.0). On average, holding other variables constant at their means, a 1 mmol/L increase in PPG was associated with an increase in healthcare resource utilization related to diabetes and elevated PPG, including the expected number of healthcare provider (HCP) visits in the past 6 months (IRR [95% CI] = 1.14 [1.08-1.21], p < .001), the expected number of calls/emails to HCPs in the past 6 months (IRR [95% CI] = 1.12 [1.06-1.19], p < .001), and the expected number of overnight hospitalizations in the past year (IRR [95% CI] = 1.14 [1.07-1.23], p < .001). LIMITATIONS: The study relied on self-reported data, which may be subject to recall bias. Given the cross-sectional nature of the study, results should be interpreted with caution. CONCLUSIONS: Increased PPG levels were significantly associated with increased healthcare resource utilization among adults with diabetes. Additional education regarding PPG monitoring may help improve the day-to-day management of diabetes and reduce healthcare costs.

Development of the Weight-Related Sign and Symptom Measure.

BACKGROUND: Overweight and obesity have been associated with physical and emotional signs & symptoms. Research has shown that modest weight loss can mitigate some symptoms in individuals with overweight or obesity. This study's purpose was to conduct concept elicitation (CE) interviews to provide documented qualitative support for the development of the Weight-Related Sign and Symptom Measure (WRSSM) to assess weight-related signs/symptoms in U.S. adults with overweight or obesity, with or without type 2 diabetes (T2DM).Eight focus groups were conducted in the U.S. with adults with overweight or obesity to understand weight-related sign/symptom impact from the patient perspective. Individual interviews were conducted with clinical experts to understand the impact of overweight or obesity on patient signs and symptoms. Transcripts were analyzed to identify symptoms and observable signs. A clinical challenge was conducted with clinical experts to confirm the signs/symptoms were clinically relevant, important to patients, and would improve with modest weight loss. Cognitive debriefing (CD) was conducted with individuals with overweight or obesity to confirm readability and symptom relevance. RESULTS: CE interviews were conducted with four clinical experts, and 61 people, 32% of whom had T2DM, participated in the focus groups. Analyses identified two major areas of obesity impacts: weight-related physical signs/symptoms, and emotional impacts. The most frequently reported physical signs/symptoms were feeling tired (74%), shortness of breath (69%), and joint pain (64%). The most often reported emotional impacts included poor self-image (72%) and depression (51%). Twelve signs/symptoms were identified during item generation and included on the preliminary measure. Twelve adults with overweight/obesity, who were not part of the focus groups, participated in CD. After the CD, a validation-ready, 10-item WRSSM measure was generated. CONCLUSIONS: Findings provide evidence of content validity for the validation-ready WRSSM in U.S. adults with overweight or obesity, including people with and without T2DM.

Understanding bolus insulin dose timing: the characteristics and experiences of people with diabetes who take bolus insulin.

OBJECTIVE: Despite the increased popularity of newer, fast-acting bolus insulin treatment options that allow for more flexibility in the timing of bolus insulin dosing in recent years, relatively little is known about people with diabetes who administer bolus insulin at differing times in relation to their meals. The purpose of this study was to investigate bolus insulin dose timing in relation to meals among people with type 1 (T1D) and type 2 (T2D) diabetes, as well as to better understand the characteristics and experiences of people who bolus dose at differing times. METHODS: A web-based survey of adults with T1D and T2D treated with bolus insulin therapy in Germany, the UK, and USA was conducted. RESULTS: A total of 906 respondents completed the survey (39% T1D; 61% T2D). A majority of respondents reported bolus dosing before meals in the previous week (57.0%), followed by after meals (18.9%), with meals (12.7%), and at varying times (11.5%). Compared to respondents who dosed with or after meals, those who dosed before meals were significantly less likely to experience hypoglycemia (before, 55.7%; with, 72.8%; after, 68.7%; p < .001) in the previous week. Respondents who bolus dosed before meals were significantly more likely to perceive bolus dose timing as flexible (45.5%) compared to those who dosed with (27.8%) or after (35.7%) meals (p < .001). CONCLUSION: Results show that many people with T1D and T2D dose their bolus insulin with or after meals. Key limitations of all self-report surveys include potential bias in responses and generalizability of findings. However, the study was designed to help mitigate these limitations. The findings have implications for clinicians and suggest opportunities for improving diabetes education and care.

Perceptions of diabetes control among people with type 2 diabetes treated with basal insulin in Sweden, Switzerland, and the United Kingdom.

OBJECTIVE: To investigate perceptions of control among people with uncontrolled and well controlled type 2 diabetes (T2D) treated with basal insulin, as well as differences in perceptions and diabetes management practices between the two groups. RESEARCH DESIGN AND METHODS: Web surveys of 1012 people with uncontrolled T2D (HbA1c >8.0% or 64 mmol/mol) on basal insulin in Sweden, Switzerland, and the UK and 295 people with well controlled T2D (HbA1c <7.5% or 58 mmol/mol) on basal insulin in the UK were conducted. RESULTS: People with uncontrolled T2D perceived a wide range of factors as very/extremely important for deciding whether they are well controlled, including diet (80.7%), HbA1c value (78.9%), times per day insulin taken (78.8%), insulin units taken per day (77.6%), and energy levels (74.5%). Fifty-one percent of uncontrolled respondents considered the past week or more recently when thinking about control. Perceived major obstacles to control included stress (75.4%), other health issues (70.8%), medicine side effects (69.9%), food cravings (69.8%), doctor not understanding individual situation (67.6%), and life crises (66.9%). Many uncontrolled respondents reported that diabetes was very/extremely interfering with their lives, including energy level (71.0%), performance at work (70.0%), general health (69.9%), and doing what one wants (69.3%). Analyses showed significant differences between well controlled and uncontrolled UK respondents. Compared to the uncontrolled, people with well controlled T2D were significantly more likely to consider the last 24 hours/current time when thinking about control (50% vs. 21%, p < 0.001) and reported greater healthcare contact related to diabetes and more frequent glucose measurement. Study limits include potential selection bias of web surveys and possible recall bias in patient-reported data. CONCLUSIONS: The results illuminate how people with T2D treated with basal insulin perceive control and show important differences between the well controlled and uncontrolled. Findings may have implications for improving patient and physician education and diabetes management.

Understanding Post-Prandial Hyperglycemia in Patients with Type 1 and Type 2 Diabetes: A Web-based Survey in Germany, the UK, and USA.

INTRODUCTION: To explore how patients with diabetes experience post-prandial hyperglycemia (PPH) or elevated blood glucose (BG) following a meal. METHODS: A web-based survey of patients with type 1 or type 2 diabetes using bolus insulin in Germany, the USA, and the UK was conducted. RESULTS: A total of 906 respondents completed the survey. PPH was a frequent occurrence among patients with type 1 and type 2 diabetes; 61.9% of respondents had experienced PPH in the past week, and differences by diabetes type were not significant. More than half of the respondents reported that they knew they were experiencing PPH because they had measured their BG (64.8%) and/or because they "just didn't feel right" (51.9%). The most frequently reported reasons given for PPH were eating more fat/sugar than estimated (31.2%) and over-eating in terms of their calculated bolus insulin dose (30.4%). The most common situations/factors contributing to PPH were stress (27.4%), eating at a restaurant (24.9%), being busy (21.1%), and/or feeling tired (19.2%). The most frequent corrective actions respondents took following PPH were testing BG and taking bolus insulin based on the reading (62.0%), and/or eating less/more carefully at their next meal or snack (18.8%). Additionally, significant differences in the reasons and contributing factors given for PPH and corrective actions following PPH, as well as emotions experienced when taking bolus insulin, were found by diabetes type. CONCLUSION: These findings shed light on how patients with diabetes experience and manage PPH on a day-to-day basis and have implications for improving diabetes self-management. Clinicians and diabetes educators should help patients address eating habits and lifestyle issues that may contribute to PPH. FUNDING: This study was sponsored by Novo Nordisk.

Perceptions of diabetes control among physicians and people with type 2 diabetes uncontrolled on basal insulin in Sweden, Switzerland, and the United Kingdom.

Objective A large proportion of people with type 2 diabetes (T2D) remain uncontrolled on basal insulin. Yet, there is limited understanding of how people with uncontrolled type 2 diabetes (PWUD) perceive control and insulin intensification and whether their perceptions differ from those of physicians. The purpose of the study was to investigate perceptions of control and views on insulin intensification among physicians and PWUD. Research design and methods Web surveys of 1012 PWUD on basal insulin and 300 physicians were conducted in Sweden, Switzerland, and the United Kingdom. Results Analyses revealed significant differences between physicians and PWUD. Physicians were significantly more likely than PWUD to indicate that HbA1c (85.0% vs. 78.9%, p < 0.05), complications from diabetes (89.3% vs. 75.3%, p < 0.001), and frequency/severity of hypoglycemia (93.3% vs. 68.6%, p < 0.001) were very/extremely important for deciding whether or not diabetes is well controlled. In contrast PWUD were significantly more likely to place importance on a variety of factors, including energy levels (74.5% vs. 33.0%, p < 0.001), insulin units/day (77.6% vs. 29.0%, p < 0.001) and how predictable life is (72.1% vs. 29.3%, p < 0.001). PWUD also perceived significantly greater obstacles to control and viewed uncontrolled T2D as more interfering in their lives compared to physicians. Physicians were most reluctant to intensify insulin when there is a lack of patient agreement. Worries about weight gain and feelings of 'getting sicker' were the most frequently reported reasons why PWUD on basal insulin were reluctant to intensify insulin. Conclusions Results revealed a significant disconnect between physicians and PWUD in their perceptions of diabetes control. While physicians generally expressed a more focused and clinical view of diabetes control, patients had a broader view. Results also provide insights into PWUD and physicians' reluctance to intensify insulin. The findings suggest that physician and patient education on differing perceptions could benefit communication and improve diabetes management.

The Economic Burden of Post-prandial Hyperglycemia (PPH) Among People with Type 1 and Type 2 Diabetes in Three Countries.

INTRODUCTION: Post-prandial hyperglycemia (PPH) among people with diabetes is a well-known clinical challenge to diabetes management. While the economic burden of diabetes is well studied, little is known about economic costs specific to PPH. The purpose of this study was to investigate costs of PPH related to work, diabetes management, and use of healthcare resources among people with diabetes taking bolus insulin. METHODS: Data were collected in a web survey of 906 adults with type 1 (39%) and type 2 (61%) diabetes taking bolus insulin in Germany (34%), the UK (26%), and the USA (40%). RESULTS: Sixty-two percent of respondents experienced PPH in the past week, and respondents averaged 1.7 episodes per week. Working respondents indicated that PPH affected their work productivity: 27% missed work time and 71% experienced work productivity issues while at work due to a recent episode of PPH. In terms of diabetes management, respondents with PPH in the past week measured their blood glucose (BG) more frequently than those without PPH (3.7 vs. 2.5 times/day, P < 0.001). PPH was also significantly associated with greater use of healthcare resources. Compared to those without PPH, respondents with PPH reported greater contact with healthcare professionals related to diabetes in the past year (5.5 vs. 4.4 visits, P < 0.001; 2.7 vs. 1.4 calls/emails, P < 0.001) and were more likely to report medical complications related to diabetes (72% vs. 55%, P < 0.001). Average annual costs associated with PPH due to missed work time, additional BG test strips, and physician visits were estimated to be $1239 USD per employed person in the USA. CONCLUSION: Results indicate that PPH is associated with greater economic costs and that reducing the incidence of PPH would help mitigate such costs. Additional research is needed to better understand costs associated with PPH that may be more difficult to measure, as well as more long-term impacts and costs. FUNDING: Novo Nordisk.

The black-white test score gap and early home environment.

Based on panel data for three age cohorts of children from the Panel Study of Income Dynamics, we examine how early home environment contributes to black-white achievement gaps at different developmental stages and the extent to which early gaps contribute to later racial achievement gaps. We find large black-white test score differences among children of all ages even before children start formal schooling. Except for the oldest cohort, the gaps for all tests widened when children's cognitive skills were assessed six years later. Racial achievement gaps in applied problem scores by grade three and letter-word scores by grade six, can be accounted for by child's characteristics, family socioeconomic background, and mother's cognitive skills. However, these covariates explain an increasingly smaller proportion of the black-white achievement gap as children advance to higher grades. Gaps in early cognitive skills are highly predictive of gaps at later ages, setting off a trajectory of cumulative disadvantage for black children over time. Our results underscore the key role of early home environment and the intergenerational roots of the persistent black-white achievement gap.