Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

Maniatis, Aristides K; Carakushansky, Mauri; Galcheva, Sonya; Prakasam, Gnanagurudasan; Fox, Larry A.; Dankovčíková, Adriana; Loftus, Jane; Palladino, Andrew A.; Resa, Maria de los Angeles; Taylor, Carrie Turich; Dattani, Mehul; Lebl, Jan

doi:10.1210/jendso/bvac117

Cited by 19 publications

(11 citation statements)

References 28 publications

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“…This result is consistent with data from a previous discrete choice experiment study having showed that patients prefer a less frequent injection regimen for treating GHD ( 31 ). Our mean overall life interference total score related to daily injections of somatropin was consistent with the one found in the NCT03831880 trial (27.7 and 24.13), and three times higher than the mean score related to weekly injections of somatrogon (8.63, P < 0.0001 between groups) ( 17 ). As reported in other conditions, a reduced treatment burden may result in improvement treatment adherence ( 12 ).…”

Section: Discussionsupporting

confidence: 88%

“…After first results from a crossover phase III trial which showed that treatment burden of weekly injections of somatrogon was lower than that of somatropin administered once daily in children with GHD (NCT03831880 trial) ( 17 ), the real-world cross-sectional French QOLITHOR study, conducted in 275 pediatric patients including 166 children with GHD, provides complementary information on patient experience as recommended by French, European, and US Health Authorities ( 18 , 19 , 20 , 21 ).…”

Section: Discussionmentioning

confidence: 99%

“…Since treatment burden of the rhGH injection is often a shared experience between patient and caregiver, it was assessed using a Dyad Clinical Outcomes Assessment (DCOA) questionnaire specifically developed and validated prior to this trial to measure rhGH injection treatment burden, with a simultaneous assessment using a parent–child dyad (Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD)) ( 16 ). First results showed that treatment with somatrogon administered once-weekly improved the mean overall life interference total score (primary outcome) after 12 weeks of treatment compared to somatropin administered once-daily (mean respective overall life interference total scores: 8.63 and 24.13; difference between mean scores: –15.49; 95% CI: (–19.71, –11.27); P < 0.0001) ( 17 ).…”

Section: Introductionmentioning

confidence: 99%

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Treatment burden, adherence, and quality of life in children with daily GH treatment in France

Coutant

Tauber²,

Demaret³

et al. 2023

Endocrine Connections

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Objective: To describe in a real-life setting the treatment burden and adherence, and quality of life of children treated with daily injections of growth hormone, and their relationship with treatment duration. Design: This non-interventional, multicenter, cross-sectional French study involved children aged 3 to 17 years treated with daily growth hormone injections. Methods: Based on a recent validated dyad questionnaire, the mean overall Life Interference total score (100=most interference) was described, with treatment adherence and quality of life (QOL) using the Quality of Life of Short Stature Youth questionnaire (100=best). All analyses were performed according to treatment duration prior to inclusion. Results: Among the 275/277 analyzed children, 166 (60.4%) had only growth hormone deficiency (GHD). In the GHD group, mean age was 11.7±3.2 years; median treatment duration was 3.3 years (IQR 1.8-6.4). The mean overall Life Interference total score was 27.7±20.7 (95% CI [24.2;31.2]), with non-significant correlation with treatment duration (p=0.1925). Treatment adherence was good (95.0% of children reported receiving >80% of planned injections over the last month); it slightly decreased with treatment duration (p=0.0364). Children overall QOL was good: 81.5±16.6 and 77.6±18.7 according to children and parents, respectively, but sub scores of the coping and treatment impact domains were <50. Similar results were observed in all patients independently of the condition requiring treatment. Conclusions: This real-life French cohort confirms the treatment burden of daily growth hormone injections, as previously reported in an interventional study.

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Section: Discussionsupporting

confidence: 88%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Treatment burden, adherence, and quality of life in children with daily GH treatment in France

Coutant

Tauber²,

Demaret³

et al. 2023

Endocrine Connections

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“…Long-acting GH preparations (LAGH), injected once weekly as opposed to once daily, have recently been approved for use in the US, EU, Japan, Canada, and Australia and are believed to be a potential solution for improvements in patient adherence ( 18 ). Although no published data have emerged on the impact of LAGH on adherence from real-world use, a recently published clinical trial demonstrated that treatment burden, as evaluated by life interference, was significantly reduced when patients were treated with somatrogon once weekly compared to once daily Genotropin, and the overall treatment experience for somatrogon was preferred by patients and caregivers ( 19 ). Another recent study found a preference for LAGH and reduced treatment burden in patients that switched from daily somatropin to weekly lonapegsomatropin ( 20 ).…”

Section: Discussionmentioning

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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“…Factors contributing to poor adherence include the burden of daily injections, pain caused by injections, storage issues, the need to reconstitute solutions, and managing injections while traveling ( 6 , 12 , 13 ). There is, therefore, a need for GH therapies that require a reduced injection frequency, while still being effective and having a tolerable safety profile compared to daily hGH ( 14 ).…”

Section: Introductionmentioning

confidence: 99%

Long acting growth hormone (LAGH), an update

Grillo,

Frank,

Saenger

2023

Front. Pediatr.

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In 1957, Maurice Raben at Yale was able to isolate and purify growth hormone from cadaveric pituitary glands. Pituitary growth hormone was the only way to treat children with growth hormone (GH) deficiency, until 1985 when recombinant GH became available for daily subcutaneous injection. For many years, the pediatric endocrine community longed for a long-acting recombinant GH formulation that would decrease the inconvenience of daily injections. Several mechanisms were employed to develop a GH that is rapidly absorbed into the blood stream after subcutaneous injection, but provides slow removal from the circulatory system to potentially optimize patient adherence to GH therapy. Four long-acting growth hormones are currently available in the world, or are close to regulatory approval. They are: (1) Pegylated formulations, (2) Prodrug formulations which are converted into active drug, (3) Nonvalent transient albumin binding GH compounds and (4) GH fusion proteins where a protein si fused with GH. All four formulations have undergone detailed phase 3 studies and were found to show non-inferiority in these clinical studies. All four demonstrate a safety and tolerability profile that is comparable to that of daily somatropin with an excellent adherence profile.

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Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

Cited by 19 publications

References 28 publications

Treatment burden, adherence, and quality of life in children with daily GH treatment in France

Treatment burden, adherence, and quality of life in children with daily GH treatment in France

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

Long acting growth hormone (LAGH), an update

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