Specialty Drugs for the Treatment of Short Stature in Children—Pros, Cons, and Perspectives for the Future

2010 ◽  
Vol 06 (01) ◽  
pp. 78
Author(s):  
Michaela B Koontz ◽  
Leona Cuttler ◽  
◽  
Keyword(s):  
Growth Hormone ◽  
Short Stature ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Hormone Deficiency ◽  
Current Status ◽  
Current Evidence ◽  
Recombinant Human Insulin ◽  
Growth Hormone Insensitivity ◽  
Severe Short Stature

Specialty drugs are generally defined as medications that involve special drug handling and/or parenteral administration and are typically used to treat complex medical conditions. They are typically biologicals, often very expensive, and generally prescribed by specialists. The recent surge in use of specialty pharmaceuticals has placed these drugs in the spotlight as policy-makers struggle to contain healthcare costs. Specialty drugs are central to discussions about optimal ways to manage childhood short stature; recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1)—specialty drugs with annual prices of $20,000 to $30,000 per child—are available to treat childhood short stature from specific causes. rhGH and rhIGF-1 revolutionized treatment of severe short stature resulting from growth hormone deficiency and growth hormone insensitivity, respectively. Over the past 20 years, use of rhGH has expanded to other conditions. Expanded use of the newer rhIGF-1 may occur in an analogous manner. This article reviews the background, current status, and potential for these drugs in view of current evidence and policies.

scholarly journals Comparison of Quality of Life Responses From Caregiver and Children Aged ≥7 Years Using the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire, Following 12 Months of Growth Hormone Treatment With Either a Weekly Somatrogon or a Daily Genotropin Injection Schedule

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A674-A674
Author(s):  
Jane Loftus ◽  
Julia Quitmann ◽  
Srinivas Valluri ◽  
Aleksandra Pastrak ◽  
Lawrence Reiter ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Growth Hormone ◽  
Short Stature ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Hormone Treatment ◽  
Hormone Deficiency ◽  
Height Velocity ◽  
Controlled Clinical Trial

Abstract Objective: Paediatric growth hormone deficiency (pGHD) affects 1/4,000 children. Treatment with daily sub-cutaneous injections of recombinant human growth hormone (r-hGH) increase height velocity and quality of life (QoL). A recent randomised controlled clinical trial (NCT02968004) evaluated the efficacy/safety of weekly Somatrogon (hGH-CTP) and daily Genotropin in pGHD. QoL (an exploratory endpoint) was evaluated using the validated Quality of Life in Short Stature Youth (QoLISSY) questionnaire, which includes three subscales (physical, social, emotional) and total score. Methods: The QoLISSY core module was administered to patients (aged 3-11 years [girls], 3-12 years [boys]) and parents in US, UK, Australia, New Zealand, Belarus, Russia, Ukraine and Spain, at Baseline (BL) and 12 months after treatment start. The QoLISSY-CHILD was completed by children aged ≥7 years; QoLISSY-PARENT was completed by the Caregiver for children <7 years, and for some children aged ≥7 years. We report here only the QoLISSY results for children aged ≥7 years (reported from either child or parent). Results: For Total QoLISSY-PARENT, for children aged ≥7 years in the Somatrogon group (N=26), mean scores are 53.65 (BL) and 65.52 (month 12) with mean change of 13.01 (95% Confidence Interval [CI]: 3.99, 22.02). In the Genotropin group (N=28), mean scores are 55.89 (BL) and 63.66 (month 12) with mean change of 6.60 (CI:-0.21, 13.40). For Total QoLISSY-CHILD in the Somatrogon group (N=35), mean scores are 61.48 (BL) and 74.69 (month 12) with mean change of 13.00 (CI: 5.81, 20.19). In the Genotropin group (N=35), these scores are 60.96 (BL) and 69.03 (Month 12) with mean change of 7.84 (CI: 2.71, 12.97). Scores of >70 indicate a good QoL. Conclusions: QoL in children aged ≥7 years improved, following 12 months of either treatment, whether this was reported by caregiver or child. However, these data show that the baseline scores and 12 month scores from the QoLISSY-PARENT in both treatment groups were numerically lower than those reported by the child. This is consistent with the literature¹, in which the caregivers generally report lower QoL scores on behalf of the child. ¹Explaining parent-child (dis)agreement in generic and short stature-specific health-related quality of life reports: do family and social relationships matter? Quitmann et al Health and Quality of Life Outcomes 2016 vol 14, Article 150

The effect of treatment with recombinant human growth hormone (rhGH) on linear growth and adult height in children with idiopathic short stature (ISS): a systematic review and meta-analysis

2020 ◽  
Vol 33 (12) ◽  
pp. 1577-1588
Author(s):  
George Paltoglou ◽  
Ioannis Dimitropoulos ◽  
Georgia Kourlaba ◽  
Evangelia Charmandari
Keyword(s):  
Systematic Review ◽  
Growth Hormone ◽  
Short Stature ◽  
Human Growth Hormone ◽  
Adult Height ◽  
Linear Growth ◽  
Meta Analysis ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Idiopathic Short Stature

AbstractObjectivesIdiopathic short stature (ISS) is a recognized, albeit a controversial indication for treatment with recombinant human growth hormone (rhGH).The objective of the present study was to conduct a systematic review of the literature and meta-analyses of selected studies about the use of rhGH in children with ISS on linear growth and adult height (AH).MethodsA systematic literature search was conducted to identify relevant studies published till February 28, 2017 in the following databases: Medline (PubMed), Scopus and Cochrane Central Registry of Controlled Trials. After exclusion of duplicate studies, 3,609 studies were initially identified. Of those, 3,497 studies were excluded during the process of assessing the title and/or the abstract. The remaining 112 studies were evaluated further by assessing the full text; 21 of them fulfilled all the criteria in order to be included in the current meta-analysis.ResultsChildren who received rhGH had significantly higher height increment at the end of the first year, an effect that persisted in the second year of treatment and achieved significantly higher AH than the control group. The difference between the two groups was equal to 5.3 cm (95% CI: 3.4–7 cm) for male and 4.7 cm (95% CI: 3.1–6.3 cm) for female patients.ConclusionIn children with ISS, treatment with rhGH improves short-term linear growth and increases AH compared with control subjects. However, the final decision should be made on an individual basis, following detailed diagnostic evaluation and careful consideration of both risks and benefits of rhGH administration.

Final Height of Idiopathic Short Stature Children Treated with Recombinant Human Growth Hormone (rhGH)

2011 ◽  
pp. P1-747-P1-747
Author(s):  
Thais C Martins ◽  
Cristiane N Lauretti ◽  
Ivo JP Arnhold ◽  
Berenice B Mendonca ◽  
Alexander AL Jorge
Keyword(s):  
Growth Hormone ◽  
Short Stature ◽  
Human Growth Hormone ◽  
Final Height ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Idiopathic Short Stature

scholarly journals Low FT4 Concentrations around the Start of Recombinant Human Growth Hormone Treatment: Predictor of Congenital Structural Hypothalamic-Pituitary Abnormalities?

2018 ◽  
Vol 89 (2) ◽  
pp. 98-107 ◽  
Author(s):  
Laura van Iersel ◽  
Hanneke M. van Santen ◽  
Gladys R.J. Zandwijken ◽  
Nitash Zwaveling-Soonawala ◽  
Anita C.S. Hokken-Koelega ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Hormone Treatment ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Hormone Treatment ◽  
Hormone Deficiency ◽  
Pituitary Hormone ◽  
Gh Treatment ◽  
Central Hypothyroidism ◽  
Pituitary Disease

Background: Growth hormone (GH) treatment may unmask central hypothyroidism (CeH). This was first observed in children with GH deficiency (GHD), later also in adults with GHD due to acquired “organic” pituitary disease. We hypothesized that newly diagnosed CeH in children after starting GH treatment for nonacquired, apparent isolated GHD points to congenital “organic” pituitary disease. Methods: Nationwide, retrospective cohort study including all children with nonacquired GHD between 2001 and 2011 in The Netherlands. The prevalence of CeH, hypothalamic-pituitary (HP) abnormalities, and neonatal congenital hypothyroidism screening results were evaluated. Results: Twenty-three (6.3%) of 367 children with apparent isolated GHD were prescribed LT4 for presumed CeH within 2 years after starting GH treatment. Similarly to children already diagnosed with multiple pituitary hormone deficiency, 75% of these 23 had structural HP abnormalities. In children not prescribed LT4, low pre- or post-GH treatment FT4 concentrations were also associated with structural HP abnormalities. Neonatal screening results of only 4 of the 23 children could be retrieved. Conclusion: In children with nonacquired, apparent isolated GHD, a diagnosis of CeH after, or a low FT4 concentration around the start of GH treatment, is associated with congenital structural HP abnormalities, i.e., “organic” pituitary disease. Neonatal values could not be judged reliably.

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