Background To capture the broad range of treatment burden issues experienced by adolescent and ad... more Background To capture the broad range of treatment burden issues experienced by adolescent and adult people with hemophilia (PWH), the Hemophilia Treatment Experience Measure (Hemo-TEM) was developed. We describe the development of this new hemophilia-specific patient-reported outcome (PRO) measure including concept elicitation, cognitive debriefing, and psychometric validation. Results Concept elicitation interviews were conducted with 5 clinical experts and 30 adult PWH in the United States (US). The qualitative analysis of these interviews and a review of the literature informed the PRO measure development. The project team reviewed concept endorsement rates and generated a 27-item preliminary version of the Hemo-TEM. Cognitive debriefing interviews were conducted to ensure participant understanding and item relevance in samples of (adolescent (n = 20) and adult (n = 14)) PWH in the US. The refined, validation-ready version of the Hemo-TEM included 30 items. Lastly, data from 3 c...
Purpose The aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child... more Purpose The aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child Treatment Burden Measure-Child (GHD-CTB-Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency-Child Treatment Burden Measure-Observer (GHD-CTB-Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years. Methods A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis. The child sample consisted of 59 treatment-naïve children (no prior exposure to growth hormone [GH] therapy; were starting GH treatment at study start per standard of care) and 184 children already maintained on treatment for at least 6 months. At baseline, all study participants completed a paper validation battery including all measures needed to conduct the validation analyses. Follow-up assessments with children in the maintenance group and their caregiver/parent were conducted approximately 2 weeks post-baseline to evaluate test-retest reproducibility. To evaluate sensitivity to change and meaningful change thresholds, treatment-naïve participants in both child and parent/guardian populations were assessed within 1 week of report of minimal improvement between week 3 and week 11 and at week 12. Psychometric analyses were implemented following an a priori statistical analysis plan. Results Factor analyses confirmed the a priori conceptual domains and Overall score for each measure (GHD-CTB-Child and GHD-CTB-Observer domains: Physical, Emotional Well-being, and Interference; GHD-PTB domains: Emotional Wellbeing and Interference). Internal consistency was acceptable for all measures (Cronbach's alpha > 0.70). Test-retest reliability was acceptable for the Physical, Emotional, and Overall domains of the GHD-CTB versions, and the Emotional and Overall domains of the GHD-PTB (intraclass correlation coefficient above 0.70). All but one of the convergent validity hypotheses for the GHD-CTB versions and all hypotheses for the GHD-PTB were proven (r > 0.40). Known-groups validity hypotheses were significant for length of time to administer the injections in the GHD-CTB versions (p < 0.001 for Physical, Emotional, and Overall, and p < 0.01 for Interference) and whether parents/guardians versus child gave the injections more often for the Emotional domain of the GHD-PTB (p < 0.05). Associated effect sizes ranged from −0.27 to −0.57 for GHD-CTB versions and from −0.74 to −0.69 for the GHD-PTB, indicating that the measures are sensitive to change. Anchor-based patient and parent/guardian ratings of severity suggest preliminary meaningful change thresholds (GHD-CTB: 6 points for Physical score, 9 for Emotional, and 6 for Interference; GHD-PTB: 10 points for Emotional and 6 for Interference scores). Conclusions The psychometric properties of the GHD-CTB-Child, GHD-CTB-Observer, and GHD-PTB support the validity of their use as PRO and ObsRO measures to capture the experiences associated with treatment burden for children with GHD and their parents/guardians in both clinical and research settings. The Clinicaltrials.gov registration number NCT02580032 was first posted October 20, 2015. Extended author information available on the last page of the article M. Brod et al.
Purpose The purpose of this study was to conduct cognitive debriefing (CD) interviews with adults... more Purpose The purpose of this study was to conduct cognitive debriefing (CD) interviews with adults diagnosed with chronic hypoparathyroidism (HP) to assess the content validity of the SF-36v2® Health Survey Acute (SF-36v2) measure in this population. Methods CD interviews were conducted with adults with HP in the United States (US). Interviews were conducted by a trained moderator using a semi-structured interview guide, employing a think-aloud method in conjunction with verbal probing. Participants were asked whether each item was understandable, relevant, important, and sensitive to change in relation to HP. Additionally, comprehension of instructions, response options, and the appropriateness of a 1-week recall period was assessed. Results Sixteen adults with HP participated in individual CD telephone interviews. All items in the SF-36v2 were reported to be understood, relevant, important, and sensitive to change by at least half, and in most cases, by a strong majority of study p...
cohort with out-of-pocket costs. Retention was measured in both cohorts between January 2016 and ... more cohort with out-of-pocket costs. Retention was measured in both cohorts between January 2016 and January 2017 using a Kaplan-Meier approach. A sensitivity analysis was performed to investigate the cost of retention for increase incremental increase in out-of-pocket cost. ReSultS: Brand 1 Co-pay cohort are retained on average 27 days longer than the Cash cohort (p< 0.05), whereas the Brand 2 Co-pay cohort are retained on average 51 days longer than the Cash cohort. The sensitivity analysis of retention for Brand 1 Cash cohort showed that patients with higher out-of-pocket cost have significantly lower retention than patients with lower out-of-pocket cost (p< 0.001). ConCluSionS: Co-pay cards have the potential to improve patient retention for patients experiencing access barriers due to high out-of-pocket costs. We recommended channeling the Co-pay card patient assistant program to patients with high out-of-pocket costs to achieve the biggest impact on retention.
Clinical Assessment and Comorbidities of Sleep Disorders, 2019
Background: Obstructive sleep apnea (OSA) with excessive daytime sleepiness (EDS) can impair qual... more Background: Obstructive sleep apnea (OSA) with excessive daytime sleepiness (EDS) can impair quality of life (QOL) and may go undiagnosed. Objectives: To examine the diagnosis experience and impacts of EDS with OSA on QOL in the US. Methods: Focus groups in 3 US cities with 42 patients experiencing EDS with OSA; coded transcripts qualitatively analysed using adapted grounded theory approach. Results: Prior to diagnosis, 40% (n=17) of patients were aware of their EDS and 74% (n=31) were informed of their other OSA symptoms by a spouse/partner, family or friends. About half (n=22, 52%) waited an average of 11.4 (range 1-37) years to seek medical care for OSA; 32% (n=7/22) had thought their symptoms were normal rather than a sign of OSA. Reasons for seeking care were: input from a loved one (n=21, 50%); self-concern about symptoms (n=7, 17%); and impaired driving (n=5, 12%). Twenty-seven patients discussed referral pathways: 15/27 (56%) first saw a primary care physician and 12/27 (44%...
total, intensification required 7.0 HCP visits [95% CI, 6.6-7.3] and 30.1 weeks [95% CI, 28.4-31.... more total, intensification required 7.0 HCP visits [95% CI, 6.6-7.3] and 30.1 weeks [95% CI, 28.4-31.7], on average, excluding referral time. Among intensified patients, physicians reported that, on average, 57.0% reached their HbA1c target after 2.6 HCP visits [95% CI, 2.5-2.8] and 37.8% discontinued basal-bolus. ConCluSionS: Intensifying T2D patients to basal-bolus requires use of expensive healthcare resources and is time-consuming. Study limitations include possible overlap in the intensification training and titration process. Similar findings have been reported for the costs of intensifying T2D patients on basal insulin to basal with GLP-1.
Conclusions The burden of GHD in children and adolescents is considerable and not limited to shor... more Conclusions The burden of GHD in children and adolescents is considerable and not limited to short stature. The severity of GHD impact on children and adolescents appears to be variable and individualized, but these data indicate that early identification and growth hormone treatment may lead to fewer impacts.
Introduction: Post-prandial hyperglycemia (PPH) among people with diabetes is a well-known clinic... more 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 Electronic supplementary material The online version of this article (
The objective of this study was to develop and psychometrically evaluate a general measure of pat... more The objective of this study was to develop and psychometrically evaluate a general measure of patients' satisfaction with medication, the Treatment Satisfaction Questionnaire for Medication (TSQM). The content and format of 55 initial questions were based on a formal conceptual framework, an extensive literature review, and the input from three patient focus groups. Patient interviews were used to select the most relevant questions for further evaluation (n = 31). The psychometric performance of items and resulting TSQM scales were examined using eight diverse patient groups (arthritis, asthma, major depression, type I diabetes, high cholesterol, hypertension, migraine, and psoriasis) recruited from a national longitudinal panel study of chronic illness (n = 567). Participants were then randomized to complete the test items using one of two alternate scaling methods (Visual Analogue vs. Likert-type). A factor analysis (principal component extraction with varimax rotation) of spe...
Content validity of patient-reported outcome measures (PROs) has been a focus of debate since the... more Content validity of patient-reported outcome measures (PROs) has been a focus of debate since the 2006 publication of the U.S.
Background Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappr... more Background Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone with associated significant physical and cognitive symptoms. This study evaluated the psychometric properties of the Hypoparathyroidism Patient Experience Scales (HPES), which were developed as disease-specific, patient-reported outcome (PRO) measures to assess the symptoms and impacts associated with HP in adults. Methods Data from a non-interventional, observational study ( N = 300) and a Phase 2 clinical trial ( N = 59) were used in the psychometric evaluation. Observational and trial assessments included: an online validation battery (baseline or screening) and retest (approximately 2 weeks after baseline or screening). In the trial, the primary efficacy endpoint was assessed at week 4 through re-administration of the HPES and validation battery subset. The observational study’s larger sample size allowed for evaluation of the...
The aim of this study was to perform psychometric testing of the Growth Hormone Deficiency-Child ... more The aim of this study was to perform psychometric testing of the Growth Hormone Deficiency-Child Impact Measure (GHD-CIM): a patient-reported outcome (PRO) for children with GHD aged 9 to < 13 years and an observer-reported outcome (ObsRO) for parents/guardians of children who are unable to answer for themselves. A non-interventional, multicenter, clinic-based study was conducted in 30 private-practice and large institutional sites in the US and the UK. Psychometric analyses were conducted following an a priori validation statistical analysis plan. A preliminary examination of the data determined a PRO version for children aged 9 to < 13 years was not psychometrically sound and therefore the decision was made to have only an ObsRO measure of the GHD-CIM, which would be suitable for children aged 4 to < 13 years. The GHD-CIM ObsRO validity analyses included 98 parents/guardians. Factor analyses identified three domains: Physical Functioning (PHYS), Social Well-Being (SWB), a...
Background Currently, there is limited research on how having a child diagnosed with achondroplas... more 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 includ...
Background To capture the broad range of treatment burden issues experienced by adolescent and ad... more Background To capture the broad range of treatment burden issues experienced by adolescent and adult people with hemophilia (PWH), the Hemophilia Treatment Experience Measure (Hemo-TEM) was developed. We describe the development of this new hemophilia-specific patient-reported outcome (PRO) measure including concept elicitation, cognitive debriefing, and psychometric validation. Results Concept elicitation interviews were conducted with 5 clinical experts and 30 adult PWH in the United States (US). The qualitative analysis of these interviews and a review of the literature informed the PRO measure development. The project team reviewed concept endorsement rates and generated a 27-item preliminary version of the Hemo-TEM. Cognitive debriefing interviews were conducted to ensure participant understanding and item relevance in samples of (adolescent (n = 20) and adult (n = 14)) PWH in the US. The refined, validation-ready version of the Hemo-TEM included 30 items. Lastly, data from 3 c...
Purpose The aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child... more Purpose The aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child Treatment Burden Measure-Child (GHD-CTB-Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency-Child Treatment Burden Measure-Observer (GHD-CTB-Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years. Methods A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis. The child sample consisted of 59 treatment-naïve children (no prior exposure to growth hormone [GH] therapy; were starting GH treatment at study start per standard of care) and 184 children already maintained on treatment for at least 6 months. At baseline, all study participants completed a paper validation battery including all measures needed to conduct the validation analyses. Follow-up assessments with children in the maintenance group and their caregiver/parent were conducted approximately 2 weeks post-baseline to evaluate test-retest reproducibility. To evaluate sensitivity to change and meaningful change thresholds, treatment-naïve participants in both child and parent/guardian populations were assessed within 1 week of report of minimal improvement between week 3 and week 11 and at week 12. Psychometric analyses were implemented following an a priori statistical analysis plan. Results Factor analyses confirmed the a priori conceptual domains and Overall score for each measure (GHD-CTB-Child and GHD-CTB-Observer domains: Physical, Emotional Well-being, and Interference; GHD-PTB domains: Emotional Wellbeing and Interference). Internal consistency was acceptable for all measures (Cronbach's alpha > 0.70). Test-retest reliability was acceptable for the Physical, Emotional, and Overall domains of the GHD-CTB versions, and the Emotional and Overall domains of the GHD-PTB (intraclass correlation coefficient above 0.70). All but one of the convergent validity hypotheses for the GHD-CTB versions and all hypotheses for the GHD-PTB were proven (r > 0.40). Known-groups validity hypotheses were significant for length of time to administer the injections in the GHD-CTB versions (p < 0.001 for Physical, Emotional, and Overall, and p < 0.01 for Interference) and whether parents/guardians versus child gave the injections more often for the Emotional domain of the GHD-PTB (p < 0.05). Associated effect sizes ranged from −0.27 to −0.57 for GHD-CTB versions and from −0.74 to −0.69 for the GHD-PTB, indicating that the measures are sensitive to change. Anchor-based patient and parent/guardian ratings of severity suggest preliminary meaningful change thresholds (GHD-CTB: 6 points for Physical score, 9 for Emotional, and 6 for Interference; GHD-PTB: 10 points for Emotional and 6 for Interference scores). Conclusions The psychometric properties of the GHD-CTB-Child, GHD-CTB-Observer, and GHD-PTB support the validity of their use as PRO and ObsRO measures to capture the experiences associated with treatment burden for children with GHD and their parents/guardians in both clinical and research settings. The Clinicaltrials.gov registration number NCT02580032 was first posted October 20, 2015. Extended author information available on the last page of the article M. Brod et al.
Purpose The purpose of this study was to conduct cognitive debriefing (CD) interviews with adults... more Purpose The purpose of this study was to conduct cognitive debriefing (CD) interviews with adults diagnosed with chronic hypoparathyroidism (HP) to assess the content validity of the SF-36v2® Health Survey Acute (SF-36v2) measure in this population. Methods CD interviews were conducted with adults with HP in the United States (US). Interviews were conducted by a trained moderator using a semi-structured interview guide, employing a think-aloud method in conjunction with verbal probing. Participants were asked whether each item was understandable, relevant, important, and sensitive to change in relation to HP. Additionally, comprehension of instructions, response options, and the appropriateness of a 1-week recall period was assessed. Results Sixteen adults with HP participated in individual CD telephone interviews. All items in the SF-36v2 were reported to be understood, relevant, important, and sensitive to change by at least half, and in most cases, by a strong majority of study p...
cohort with out-of-pocket costs. Retention was measured in both cohorts between January 2016 and ... more cohort with out-of-pocket costs. Retention was measured in both cohorts between January 2016 and January 2017 using a Kaplan-Meier approach. A sensitivity analysis was performed to investigate the cost of retention for increase incremental increase in out-of-pocket cost. ReSultS: Brand 1 Co-pay cohort are retained on average 27 days longer than the Cash cohort (p< 0.05), whereas the Brand 2 Co-pay cohort are retained on average 51 days longer than the Cash cohort. The sensitivity analysis of retention for Brand 1 Cash cohort showed that patients with higher out-of-pocket cost have significantly lower retention than patients with lower out-of-pocket cost (p< 0.001). ConCluSionS: Co-pay cards have the potential to improve patient retention for patients experiencing access barriers due to high out-of-pocket costs. We recommended channeling the Co-pay card patient assistant program to patients with high out-of-pocket costs to achieve the biggest impact on retention.
Clinical Assessment and Comorbidities of Sleep Disorders, 2019
Background: Obstructive sleep apnea (OSA) with excessive daytime sleepiness (EDS) can impair qual... more Background: Obstructive sleep apnea (OSA) with excessive daytime sleepiness (EDS) can impair quality of life (QOL) and may go undiagnosed. Objectives: To examine the diagnosis experience and impacts of EDS with OSA on QOL in the US. Methods: Focus groups in 3 US cities with 42 patients experiencing EDS with OSA; coded transcripts qualitatively analysed using adapted grounded theory approach. Results: Prior to diagnosis, 40% (n=17) of patients were aware of their EDS and 74% (n=31) were informed of their other OSA symptoms by a spouse/partner, family or friends. About half (n=22, 52%) waited an average of 11.4 (range 1-37) years to seek medical care for OSA; 32% (n=7/22) had thought their symptoms were normal rather than a sign of OSA. Reasons for seeking care were: input from a loved one (n=21, 50%); self-concern about symptoms (n=7, 17%); and impaired driving (n=5, 12%). Twenty-seven patients discussed referral pathways: 15/27 (56%) first saw a primary care physician and 12/27 (44%...
total, intensification required 7.0 HCP visits [95% CI, 6.6-7.3] and 30.1 weeks [95% CI, 28.4-31.... more total, intensification required 7.0 HCP visits [95% CI, 6.6-7.3] and 30.1 weeks [95% CI, 28.4-31.7], on average, excluding referral time. Among intensified patients, physicians reported that, on average, 57.0% reached their HbA1c target after 2.6 HCP visits [95% CI, 2.5-2.8] and 37.8% discontinued basal-bolus. ConCluSionS: Intensifying T2D patients to basal-bolus requires use of expensive healthcare resources and is time-consuming. Study limitations include possible overlap in the intensification training and titration process. Similar findings have been reported for the costs of intensifying T2D patients on basal insulin to basal with GLP-1.
Conclusions The burden of GHD in children and adolescents is considerable and not limited to shor... more Conclusions The burden of GHD in children and adolescents is considerable and not limited to short stature. The severity of GHD impact on children and adolescents appears to be variable and individualized, but these data indicate that early identification and growth hormone treatment may lead to fewer impacts.
Introduction: Post-prandial hyperglycemia (PPH) among people with diabetes is a well-known clinic... more 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 Electronic supplementary material The online version of this article (
The objective of this study was to develop and psychometrically evaluate a general measure of pat... more The objective of this study was to develop and psychometrically evaluate a general measure of patients' satisfaction with medication, the Treatment Satisfaction Questionnaire for Medication (TSQM). The content and format of 55 initial questions were based on a formal conceptual framework, an extensive literature review, and the input from three patient focus groups. Patient interviews were used to select the most relevant questions for further evaluation (n = 31). The psychometric performance of items and resulting TSQM scales were examined using eight diverse patient groups (arthritis, asthma, major depression, type I diabetes, high cholesterol, hypertension, migraine, and psoriasis) recruited from a national longitudinal panel study of chronic illness (n = 567). Participants were then randomized to complete the test items using one of two alternate scaling methods (Visual Analogue vs. Likert-type). A factor analysis (principal component extraction with varimax rotation) of spe...
Content validity of patient-reported outcome measures (PROs) has been a focus of debate since the... more Content validity of patient-reported outcome measures (PROs) has been a focus of debate since the 2006 publication of the U.S.
Background Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappr... more Background Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone with associated significant physical and cognitive symptoms. This study evaluated the psychometric properties of the Hypoparathyroidism Patient Experience Scales (HPES), which were developed as disease-specific, patient-reported outcome (PRO) measures to assess the symptoms and impacts associated with HP in adults. Methods Data from a non-interventional, observational study ( N = 300) and a Phase 2 clinical trial ( N = 59) were used in the psychometric evaluation. Observational and trial assessments included: an online validation battery (baseline or screening) and retest (approximately 2 weeks after baseline or screening). In the trial, the primary efficacy endpoint was assessed at week 4 through re-administration of the HPES and validation battery subset. The observational study’s larger sample size allowed for evaluation of the...
The aim of this study was to perform psychometric testing of the Growth Hormone Deficiency-Child ... more The aim of this study was to perform psychometric testing of the Growth Hormone Deficiency-Child Impact Measure (GHD-CIM): a patient-reported outcome (PRO) for children with GHD aged 9 to < 13 years and an observer-reported outcome (ObsRO) for parents/guardians of children who are unable to answer for themselves. A non-interventional, multicenter, clinic-based study was conducted in 30 private-practice and large institutional sites in the US and the UK. Psychometric analyses were conducted following an a priori validation statistical analysis plan. A preliminary examination of the data determined a PRO version for children aged 9 to < 13 years was not psychometrically sound and therefore the decision was made to have only an ObsRO measure of the GHD-CIM, which would be suitable for children aged 4 to < 13 years. The GHD-CIM ObsRO validity analyses included 98 parents/guardians. Factor analyses identified three domains: Physical Functioning (PHYS), Social Well-Being (SWB), a...
Background Currently, there is limited research on how having a child diagnosed with achondroplas... more 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 includ...
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