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Pediatric growth hormone deficiency (pGHD) is treated with daily somatropin (recombinant human growth hormone) injections. High rates of discontinuation and poor adherence to treatment, which are associated with worse growth outcomes, have been documented previously, for example in the US and Europe. Discontinuation of somatropin has not yet been evaluated using real-world data in Japan. To describe discontinuation of, and persistence to, daily somatropin treatment among children with pGHD in Japan. This was a retrospective cohort study of children (≥3 and <16 years old) who were prescribed somatropin, using 2 Japan-based databases, Japan Medical Data Center (JMDC) and Medical Data Vision (MDV). Children were required to have ≥1 prescription for somatropin (first prescription = index date) within each study period (1 January 2002-30 September 2021 for JMDC and 1 January 2009-31 October 2021 for MDV) and ≥1 GHD diagnosis code without a somatropin prescription during the 6-months pre-index period. Children were required to be continuously enrolled in the database ≥6 months preceding and ≥3 months following index date. Children were followed for up to 48 months post-index. Early persistence was defined as the proportion of children with ≥1 refill of somatropin subsequent to the initial prescription. Discontinuation was defined as the first observation of a gap in therapy (using >60, > 90, and 120-day gap thresholds) between successive somatropin prescription fill dates. Persistence was defined as continuous refills of somatropin with no gaps in therapy. Time to discontinuation/non-persistence was evaluated using Kaplan-Meier methods, and Cox proportional hazards models identified predictors of time to discontinuation. This analysis utilized de-identified patient data from 2 large, Japanese-based retrospective databases; as such this study does not meet the requirements for institutional review board (IRB) review. Among the children included in this study (JMDC N = 452, MDV N = 573), most were male (JMDC 64.8%, MDV 60.0%). Mean age (standard deviation) was 8.8 (3.6) years in JMDC and 7.5 (3.6) years in MDV. Early persistence was high across both cohorts (JMDC 91.2%, MDV 83.4%). Using the 90-day gap definition for discontinuation, a sizable proportion of children discontinued over the follow-up period: JMDC 19% at 12 months, 35% at 48 months; and MDV 33% at 12 months, 54% at 48 months. Fewer discontinuations were observed with the 120-day gap definition (~16% at 48 months in JMDC, ~ 28% at 48 months in MDV) and more were observed with the 60-day gap definition (~67% at 48 months in JMDC, ~ 83% at 48 months in MDV). No meaningful predictors of discontinuation were identified. Despite high early persistence with somatropin, many children with pGHD in Japan were increasingly non-persistent over time: at 48 months post-index, at least 16% of children discontinued therapy, using the JDMC database and the most conservative measure of gap allowance. These results suggest a need for new strategies to support somatropin medication use over time among children with pGHD in Japan.

Daily injections of recombinant human growth hormone are the standard of care to treat growth failure due to pediatric growth hormone deficiency (GHD). While effective, daily injections are burdensome and can compromise adherence. In recent years, novel injection treatments requiring less frequent administration for growth hormone deficiency (GHD) have been developed. A targeted, pragmatic literature review was conducted to summarize and document the patient experience of moving from daily to less frequent injections, with a specific focus on changing from daily to weekly injection treatments in pediatric GHD (pGHD). Explore and describe the patient experience when switching from a daily to a less frequent injection schedule for GHD. Targeted literature searches were conducted to identify literature describing the patient experience of moving from a daily to weekly injection in GHD. Supplementary searches were conducted to identify literature describing the patient experience of moving from daily to less frequent injection regimens in other medical conditions. Across searches, 1,691 abstracts were reviewed and 13 articles were included in the final analysis. These publications reported that patients moving to less frequent injections across a variety of conditions, including GHD, experienced increased convenience and satisfaction, higher adherence rates, fewer adverse events, and improved quality of life. Less frequent injections were also reported to be at least as efficacious as daily treatments. Less frequent injections in GHD and as other conditions are less burdensome, positively benefit patients, and result in improved adherence that may lead to improved clinical outcomes. Clinicians may consider weekly regimens as an effective alternative for patients, in particular in pGHD, especially when missed injections can negatively impact treatment outcomes. More research is needed to better understand the real-world benefits of injectable therapies that require less frequent administration (e.g., weekly versus daily).

Background Achondroplasia, a disease characterized by disproportionate short stature and increased morbidity, affects daily function and quality of life over the lifetime of the individual. However, data are limited on its economic impact, especially related to healthcare resource utilization (HCRU) and associated costs. This study aimed to characterize the clinical and economic impact of achondroplasia in the US relative to matched non-achondroplasia controls stratified by pediatric and adult populations. Methods This retrospective study used data from the IQVIA PharMetrics Plus national claims database from January 2008 to December 2021. Individuals diagnosed with achondroplasia (index event) between July 2008 and December 2020 were matched on age and sex (1:2 ratio) to non-achondroplasia controls. General comorbidities were evaluated in the pediatric and adult populations. All-cause HCRU and direct medical costs were determined for the 12-month post-index period; out-of-pocket (OOP) costs were also determined. Study variables were analyzed using descriptive statistics. Results A total of 530 individuals with achondroplasia (47.7% pediatric and 52.3% adults) were matched with 1,060 controls. Individuals in the achondroplasia cohort had higher overall comorbidity burdens than controls. HCRU was higher in the achondroplasia cohort relative to controls, with outpatient visits the most frequently used resource. Inpatient visits were the primary driver of mean (SD) total costs, which were 14-fold higher than controls ($28,386 [$259,858] vs $2,031 [$5,418]) in pediatric individuals, and 4-fold higher in adults $21,579 [$58,817] vs $4,951 [$13,020]); prescriptions accounted for 4.7% and 7.4% of total costs in the pediatric and adult achondroplasia cohorts, respectively. The OOP costs were approximately 3-fold higher in both pediatric and adult individuals with achondroplasia relative to controls. Conclusions Individuals with achondroplasia are characterized by a higher comorbidity burden and substantially higher HCRU and related costs relative to matched controls. The results also suggest that despite high HCRU and costs, individuals with achondroplasia likely are not seen by providers early enough nor are they necessarily seen by appropriate specialists, indicating a need for improved care and disease management.

The daily injection burden of recombinant human growth hormone (r-hGH) replacement therapy to treat growth hormone deficiency (GHD) may reduce compliance and limit treatment benefit. Research is needed to evaluate patient preferences for GHD injection regimen and device features. Quantitatively evaluate factors driving preferences for r-hGH injection regimen and device features among pediatric (3-17 years, and caregivers) and adult (≥25 years) patients with GHD using a discrete choice experiment (DCE) approach. The DCE was part of a broader, cross-sectional observational field study to develop clinical outcome assessments (COAs) that assess the experience of patients taking r-hGH injections. Following ethics approval, discrete choice data were collected through an online questionnaire from consented participants recruited from eight sites in the United States. Participants were presented with 20 choice tasks, each comprising different combinations of two profiles. Participants were then shown the same set of three hypothetical device and injection profiles (ie, storage, preparation, injection type device, maintenance, dose setting, injection schedule) and asked whether they would choose each profile over their current device and schedule. Choice-based conjoint analyses were used to estimate the marginal utilities and values for treatment attributes. Subject preferences were estimated at individual and aggregate levels. Two hundred and twenty-four participants completed the DCE (n=75 adults, n=79 adolescent/caregiver dyads, n=70 child/caregiver dyads). Injection schedule was the strongest predictor of choice for the total sample and each patient group. Less frequent injection schedules were more likely to be chosen by participants. A \"ready to use\" injection was preferred, with no preference for auto-injector versus needle-free device. Most participants would choose the hypothetical injection devices and less frequent dosing over their current daily administered device schedule. Patients prefer a less frequent injection regimen for treating GHD. Addressing patient preferences may improve compliance, adherence, and ultimately, clinical outcomes.

IntroductionTransthyretin amyloidosis (ATTR amyloidosis), whether manifesting as familial amyloid polyneuropathy (ATTR-PN) or cardiomyopathy (ATTR-CM), is a progressive, debilitating, and often fatal, rare disease requiring significant caregiver support. This study aims to better characterize the burden of disease for ATTR amyloidosis patients and caregivers.MethodsPatients and caregivers in the USA and Spain were recruited through patient advocacy groups to complete a cross-sectional survey. Assessments included the 12-Item Short Form Health Survey, the Work Productivity and Activity Impairment Questionnaire, the Zarit Burden Interview, pain and symptom measures, health care resource use measures, and caregiving burden measures.ResultsRespondents included 60 ATTR amyloidosis patients and 32 caregivers. Patients registered scores up to two standard deviations below normal for physical health, impairment in quality of life, and reduced work productivity. Patients with liver transplant versus without liver transplant reported better overall outcomes, and those without liver transplant reported a greater impact on physical versus mental health. A high rate of health care utilization in the 3 months prior to the survey was reported by patients. Caregivers reported substantial burden, including poor mental health, work impairment, and much time spent providing care (mean 45.9 h/week). Work productivity was impacted for employed patients and caregivers.ConclusionATTR amyloidosis was associated with high levels of impairment in many domains, including physical health, quality of life, and reduced productivity. Providing care for ATTR amyloidosis patients is associated with a negative impact on the mental health of caregivers. These results highlight the substantial burden of ATTR amyloidosis for patients and caregivers.Trial RegistrationClinicalTrials.gov: NCT01604122.FundingPfizer.

• For children with growth hormone deficiency, once-weekly somatrogon injections were less of a burden than once-daily somatropin injections. • The safety of weekly somatrogon was similar to that of daily somatropin. • Compared with daily somatropin injections, children with growth hormone deficiency may be less likely to miss weekly somatrogon injections.  ○ This is because weekly somatrogon injections were less of a burden and were less likely to interfere with daily activities compared with daily somatropin injections. The purpose of this plain language summary is to help you to understand the findings from recent research. • Somatrogon is used to treat the condition under study that is discussed in this summary. Approval varies by country; please check with your local provider for more details. • The results of this study may differ from those of other studies. Health professionals should make treatment decisions based on all available evidence and not on the results of a single study. This original scientific article on which this summary is based was published in the Journal of the Endocrine Society and can be accessed for free at: https://academic.oup.com/jes/article/6/10/bvac117/6695276. The details of the original article are as follows: Aristides K. Maniatis, Mauri Carakushansky, Sonya Galcheva, Gnanagurudasan Prakasam, Larry A. Fox, Adriana Dankovcikova, Jane Loftus, Andrew A. Palladino, Maria de los Angeles Resa, Carrie Turich Taylor, Mehul T. Dattani, Jan Lebl. Treatment burden of weekly somatrogon versus daily somatropin in children with growth hormone deficiency: a randomized study. J Endocr Soc 2022; 6(10): bvac117. DOI: 10.1210/jendso/bvac117.

Background To evaluate the cost-effectiveness of growth hormone (GH) treatment (Genotropin®) compared with no GH treatment in adults with GH deficiency in a Swedish societal setting. Methods A Markov-type cost-utility simulation model was constructed and used to simulate, for men and women, morbidity and mortality for GH-treated and -untreated individuals over a 20-year period. The calculations were performed using current available prices concerning morbidity-related healthcare costs and costs for Genotropin®. All costs and treatment effects were discounted at 3%. Costs were expressed in Euro (1€ = 9.03 SEK). GH-treated Swedish patients ( n  = 434) were identified from the KIMS database (Pfizer International Metabolic Database) and untreated patients ( n  = 2135) from the Swedish Cancer Registry and the Hospital Discharge Registry. Results The results are reported as incremental cost per quality-adjusted life year (QALY) gained, including both direct and indirect costs for GH-treated versus untreated patients. The weighted sum of all subgroup incremental cost per QALY was €15,975 and €20,241 for men and women, respectively. Including indirect cost resulted in lower cost per QALY gained: €11,173 and €10,753 for men and women, respectively. Key drivers of the results were improvement in quality of life, increased survival, and intervention cost. Conclusions The incremental cost per QALY gained is moderate when compared with informal thresholds applied in Sweden. The simulations suggest that GH-treatment is cost-effective for both men and women at the €55,371 (SEK 500,000 – the informal Swedish cost-effectiveness threshold) per QALY threshold.

PurposeTo report the effects of pegvisomant (PEGV) treatment on patient-reported outcomes in acromegaly patients.MethodsWe conducted an extension study of an open-label, multinational, non-interventional study (ACROSTUDY) evaluating the long-term safety and efficacy of PEGV for acromegaly in routine clinical practice. Enrolled patients were rollover patients from ACROSTUDY, or treatment naïve/semi-naïve (NSN; no PEGV within 6 months of enrollment). Exploratory efficacy endpoints were changes in symptoms with the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ) and quality of life with the Acromegaly Quality of Life questionnaire (AcroQoL) analyzed by controlled or uncontrolled IGF-I levels. Results were analyzed in all patients, in NSN patient subgroup, and by diabetes status.ResultsA total of 544 patients with acromegaly were enrolled, including 434 rollover subjects from ACROSTUDY and 110 NSN patients. Mean PEGV treatment duration was 7.8 years (range, 0–19.6 years). Overall, the majority of PASQ scores improved over time, but there was no significant difference between IGF-I controlled or uncontrolled groups. In the NSN subgroup, most PASQ and AcroQoL scores remained similar to baseline up to 1 year, regardless of IGF-I control. Patients with diabetes reported better PASQ scores over time with PEGV treatment, regardless of IGF-I control. IGF-I normalization increased from 10% of patients at baseline to more than 78% at year 10, with a mean daily PEGV dose of 18.7 mg.ConclusionsOverall, patients treated with PEGV had small improvements in PASQ. While IGF-I normalization increased with PEGV treatment, IGF-I control had no effects on PASQ and AcroQoL scores.

Background/aims:Quantitative data regarding the impact of neovascular age-related macular degeneration (NV-AMD) on individuals and society is a prerequisite for rational decision-making processes when evaluating alternative treatments for the disease.Methods:75 bilateral NV-AMD (patients) and 91 elderly non-AMD (controls) subjects forming the UK cohort of an international cross-sectional, observational study were independently analysed. Subjects completed a telephone survey including the National Eye Institute Visual Function Questionnaire (NEI-VFQ-25), the EuroQol (EQ-5D), the Hospital Anxiety and Depression Scale (HADS), history of falls and health resource utilisation.Results:Patients with NV-AMD reported substantially worse vision-related functioning and overall well-being, including higher depression scores, than controls after adjusting for age, gender and co-morbidities (adjusted mean scores: NEI-VFQ-25 overall 52.7 vs 90.7, p<0.0001; EQ-5D 0.67 vs 0.77, p = 0.0273; HADS depression 6.8 vs 4.0, p = 0.0026). Significantly more patients reported a need for assistance with daily activities compared with controls (25.3% vs 6.6%, p = 0.003). Total annual healthcare utilisation costs were more than sevenfold higher for patients with AMD compared with controls (£3,823.89 vs £517.05, respectively; p<0.0001)Conclusions:Patients with NV-AMD show a significant decline in quality of life and increased need for daily living assistance compared to a control population without AMD. With the availability of effective new therapies there is a need for improved early access to treatment.

Background Somatrogon is a long-acting recombinant human growth hormone (hGH) currently being developed as a once-weekly subcutaneous injectable treatment for pediatric patients with growth hormone deficiency (GHD). A recent global Phase 3 study compared the efficacy and safety of somatrogon administered once weekly with Genotropin administered once daily in pediatric patients with GHD (ClinicalTrials.gov: NCT02968004). Aims To understand and describe physicians’ experiences using once-weekly somatrogon injections compared with once-daily hGH injections during the Phase 3 study. Methods In this quantitative, cross-sectional, observational study, a 14-item multiple choice questionnaire was administered to physicians who were clinical investigators in the Phase 3 study. The questionnaire was designed to assess physicians’ experiences, preferences and satisfaction regarding the use of the once-weekly somatrogon regimen compared with the standard once-daily hGH injection regimen. Survey results were analyzed quantitatively using descriptive statistics. Results A total of 24 physicians who were part of the global Phase 3 study (mean age [SD]: 58.5 [10.1] years; 10 males, 14 females) in 12 different countries completed the on-line survey. Compared with daily injections, most physicians preferred somatrogon (n=18, 75.0%) and reported that somatrogon was more convenient (n=19, 79.2%), less burdensome (n=19, 79.2%) and that they would be more likely to prescribe somatrogon to patients in the future (n=20, 83.3%). In terms of being more likely to support positive long-term growth outcomes, 37.5% (n=9) of physicians selected somatrogon; the remaining physicians (n=15, 62.5%) reported no difference between treatments. The proportion of physicians who were satisfied or very satisfied with somatrogon was 95.8% (n=23), compared with 87.5% (n=21) for daily hGH injections. Most physicians (n=15, 62.5%) reported that daily hGH injections required more effort/much more effort to monitor adherence, compared with somatrogon. Some physicians (n=7, 29.2%) reported that explaining the device instructions to patient/caregivers required more/much more effort for somatrogon compared with daily hGH injections; more than half (n=14, 58.3%) reported no difference in effort required for either regimen. Physicians were divided as to which treatment needed more/much more effort to: (i) explain the injection regimen (n=7 [29.2%] daily injection; n=7 [29.2%] no difference; n=10 [41.7%] somatrogon), (ii) explain what to do if an injection is missed (n=7 [29.2%] daily injection; n=9 [37.5%] no difference; n=8 [33.3%] somatrogon), and (iii) address patient/caregiver questions (n=8 [33.3%] daily injection; n=11 [45.8%] no difference; n=5 [20.8%] somatrogon). Conclusions Overall, physicians from the global Phase 3 study were positive about their experience using somatrogon for the treatment of children with GHD. Physicians generally preferred the once-weekly somatrogon injection regimen, with most physicians reporting somatrogon to be more convenient and less burdensome to patients, indicating that they would be more likely to prescribe somatrogon to their patients in the future. Presentation: Monday, June 13, 2022 12:30 p.m. - 2:30 p.m.