Association of Daily Growth Hormone Injection Adherence and Height Among Children With Growth Hormone Deficiency

Loftus, Jane; Miller, Bradley S.; Parzynski, Craig S.; Alvir, José; Chen, Yong; Jhingran, Priti; Gupta, Anu; DeKoven, Mitch; Divino, Victoria; Tse, Jenny; He, Jing; Wajnrajch, Michael

doi:10.1016/j.eprac.2022.02.013

Cited by 15 publications

(12 citation statements)

References 24 publications

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“…In 1 study, one-quarter of children treated with daily GH therapy reported missing 2 or more injections per week ( 5 ). Low adherence negatively affects growth outcomes ( 6 ) and is partly attributed to injection pain and frequency of injections ( 5 , 7 ).…”

mentioning

confidence: 99%

Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial

Miller

Blair

Rasmussen

et al. 2022

The Journal of Clinical Endocrinology &Amp; Metabolism

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Context Somapacitan, a once-weekly reversible albumin-binding GH derivative, is evaluated in children with GH deficiency (GHD). Objective To demonstrate efficacy and safety of somapacitan versus daily GH. Design REAL4 is a randomised, multi-national, open-labelled, active-controlled parallel group phase 3 trial, comprising a 52-week main trial and three-year extension (NCT03811535). Setting Eighty-six sites across 20 countries. Patients 200 treatment-naïve patients were randomized and exposed. Interventions Patients were randomized 2:1 to somapacitan (0.16 mg/kg/week) or daily GH (Norditropin®; 0.034 mg/kg/day), administered subcutaneously. Main outcome measures The primary endpoint was annualized height velocity (HV; cm/year) at week 52. Additional assessments included HV standard deviation score (SDS), height SDS, bone age, IGF-I SDS, patient-reported outcomes, and safety measures. Results Estimated mean HV at week 52 was 11.2 and 11.7 cm/year for somapacitan and daily GH, respectively. Non-inferiority was confirmed. Changes in HVSDS, height SDS, bone age, and IGF-I SDS from baseline to week 52 were similar between treatment groups. At week 52, mean IGF-I SDS values were similar between treatment groups and within normal range (-2 to +2). Safety of somapacitan was consistent with the well-known daily GH profile. Low proportions of injection-site reactions were reported for somapacitan (5.3%) and daily GH (5.9%). Both treatments similarly reduced disease burden from baseline to week 52, while a greater treatment burden reduction was observed for somapacitan. Conclusions Similar efficacy for somapacitan compared to daily GH was demonstrated over 52 weeks of treatment with comparable safety and mean IGF-I SDS levels in treatment-naïve children with GHD.

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mentioning

confidence: 99%

Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial

Miller

Blair

Rasmussen

et al. 2022

The Journal of Clinical Endocrinology &Amp; Metabolism

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“…2 The relationship between poor adherence and poor response to r-hGH therapy is well established. 53 While enthusiasm and support for digital health technologies was slow at first, these efforts have accelerated with broader awareness and acceptance amongst both patients and HCPs. New digital technologies will evolve and the introduction of innovations and new technologies, while providing challenges for patients and HCPs, have the potential to further improve the personalized management of the GH-deficient patient receiving r-hGH therapy.…”

Section: Discussionmentioning

confidence: 99%

Adherence to r-hGH Therapy in Pediatric Growth Hormone Deficiency: Current Perspectives on How Patient-Generated Data Will Transform r-hGH Treatment Towards Integrated Care

Savage¹,

Fernández-Luque²,

Graham³

et al. 2022

PPA

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Pediatric growth hormone (GH) deficiency is a licensed indication for replacement therapy with recombinant human growth hormone (r-hGH). Treatment, consisting of daily subcutaneous injections, extends from the time of diagnosis until cessation of linear growth at completion of puberty. Suboptimal adherence to r-hGH therapy is common and has been well documented to substantially impair the growth response and achievement of the optimal goal which is attainment of adult height within the genetic target range. The causes of poor adherence are complex and include disease-, patient-, doctor-, and treatment-related factors. Interventions for suboptimal adherence are important for a long-term successful outcome and can include both face-to-face and digital strategies. Face-to-face interventions include behavioral change approaches such as motivational interviewing and nonjudgmental assessment. Medical and nursing staff require training in these techniques. Digital solutions are rapidly advancing as evidenced by the electronic digital auto-injector device, easypod® (Merck Healthcare KGaA, Darmstadt, Germany), which uses the web-based easypod® connect platform allowing adherence data to be transmitted electronically to healthcare professionals (HCPs), who can then access GH treatment history, enhancing clinical decisions. Over the past 10 years, the multi-national Easypod® Connect Observational Study has reported high levels of adherence (>85%) from up to 40 countries. The easypod® connect system can be supported by a smartphone app, growlink™, which facilitates the interactions between the patients, their care team, and patient support services. HCPs are empowered by new digital techniques, however, the human-digital partnership remains essential for optimal growth management. The pediatric patient on r-hGH therapy will benefit from these innovations to enhance adherence and optimize long-term response.

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“…A New Zealand-based study, using the rate of returned vials, found that children with good compliance (defined as no more than one missed dose per week) had significantly greater linear growth ( 5 ). A retrospective cohort study of US claims data showed that adherent children grew an additional 1.8 cm over 1 year of treatment than non-adherent children ( 6 ). A UK-based study of 75 pGHD children who attended regional pediatric endocrinology clinics found that almost 1 in 4 (23%) missed >2 injections per week and this was associated with lower predicted height velocities ( 7 ).…”

Section: Discussionmentioning

confidence: 99%

“…Suboptimal adherence to somatropin has been well documented in many populations and associated with a negative impact on growth response ( 4 – 6 ). UK-based studies have also demonstrated the association between adherence to treatment and improved growth outcomes.…”

Section: Introductionmentioning

confidence: 99%

Persistence with daily growth hormone among children and adolescents with growth hormone deficiency in the UK

Loftus

Wogen²,

Oliveri³

et al. 2022

Front. Endocrinol.

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BackgroundChildren with growth hormone deficiency (GHD) are treated with daily somatropin injections; however, poor treatment persistence and adherence have been recognized previously and have been shown to negatively impact growth outcomes. A recent real-world study of a US pediatric GHD population found that a substantial proportion of children discontinued somatropin therapy, but similar data for a real-world UK population are lacking.ObjectivesTo describe the discontinuation of, and persistence with, daily somatropin treatment among children with GHD in the UK.MethodsThis was a retrospective cohort study of children (≥3 and <16 years old) with ≥1 medication prescription for daily injectable somatropin from 1 July 2000 to 31 December 2020 in the IQVIA Medical Research DATA (IMRD) database. Early persistence was defined as the proportion of children prescribed ≥1 somatropin refill (≥2 prescriptions). Discontinuation was defined as the first date at which a medication gap for somatropin (of >60 or >90 days between prescriptions) occurred. Kaplan–Meier methods were used to evaluate persistence (non-discontinuation) over time to assess time to first discontinuation event. Cox proportional hazards models were used to evaluate the relationship between patient characteristics and time to medication discontinuation.ResultsAmong the cohort identified in this study (n = 117), the majority (n = 84, 71.8%) had 48 months of available follow-up; 56.4% were boys and the mean (median) age was 8.6 (8.0) years. About 98% exhibited early persistence, but persistence over the follow-up period decreased with follow-up duration. Using the conservative 90-day gap definition of persistence, an estimated 72.4%, 52.8%, and 43.3% were persistent at 12, 36, and 48 months. Lower persistence rates were observed using the 60-day definition. No significant patient predictors of time to discontinuation were identified.ConclusionsDespite high early persistence with somatropin, a high percentage of children with GHD were increasingly non-persistent over time. More than 1 in 4 were non-persistent at 12 months and more than 1 in 2 were non-persistent at 48 months of follow-up. These results suggest that strategies to support improved medication-taking behavior among children with GHD in the UK are warranted.

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Association of Daily Growth Hormone Injection Adherence and Height Among Children With Growth Hormone Deficiency

Cited by 15 publications

References 24 publications

Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial

Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial

Adherence to r-hGH Therapy in Pediatric Growth Hormone Deficiency: Current Perspectives on How Patient-Generated Data Will Transform r-hGH Treatment Towards Integrated Care

Persistence with daily growth hormone among children and adolescents with growth hormone deficiency in the UK

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