| 1. |
- Christiansen, J. S., et al.
(författare)
-
Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations
- 2016
-
Ingår i: European Journal of Endocrinology. - : Oxford University Press (OUP). - 0804-4643 .- 1479-683X. ; 174:6, s. C1-C8
-
Tidskriftsartikel (refereegranskat)abstract
- Objective: The Growth Hormone (GH) Research Society (GRS) convened a workshop to address important issues regarding trial design, efficacy, and safety of long-acting growth hormone preparations (LAGH). Participants: A closed meeting of 55 international scientists with expertise in GH, including pediatric and adult endocrinologists, basic scientists, regulatory scientists, and participants from the pharmaceutical industry. Evidence: Current literature was reviewed for gaps in knowledge. Expert opinion was used to suggest studies required to address potential safety and efficacy issues. Consensus process: Following plenary presentations summarizing the literature, breakout groups discussed questions framed by the planning committee. Attendees reconvened after each breakout session to share group reports. A writing team compiled the breakout session reports into a draft document that was discussed and revised in an open forum on the concluding day. This was edited further and then circulated to attendees from academic institutions for review after the meeting. Participants from pharmaceutical companies did not participate in the planning, writing, or in the discussions and text revision on the final day of the workshop. Scientists from industry and regulatory agencies reviewed the manuscript to identify any factual errors. Conclusions: LAGH compounds may represent an advance over daily GH injections because of increased convenience and differing phamacodynamic properties, providing the potential for improved adherence and outcomes. Better methods to assess adherence must be developed and validated. Long-term surveillance registries that include assessment of efficacy, cost-benefit, disease burden, quality of life, and safety are essential for understanding the impact of sustained exposure to LAGH preparations.
|
|
| 2. |
- Johannsson, Gudmundur, 1960, et al.
(författare)
-
Growth Hormone Research Society perspective on biomarkers of GH action in children and adults
- 2018
-
Ingår i: Endocrine Connections. - : Bioscientifica. - 2049-3614. ; 7:3, s. R126-R134
-
Tidskriftsartikel (refereegranskat)abstract
- Objective: The Growth Hormone Research Society (GRS) convened a Workshop in 2017 to evaluate clinical endpoints, surrogate endpoints and biomarkers during GH treatment of children and adults and in patients with acromegaly. Participants: GRS invited 34 international experts including clinicians, basic scientists, a regulatory scientist and physicians from the pharmaceutical industry. Evidence: Current literature was reviewed and expert opinion was utilized to establish the state of the art and identify current gaps and unmet needs. Consensus process: Following plenary presentations, breakout groups discussed questions framed by the planning committee. The attendees re-convened after each breakout session to share the group reports. A writing team compiled the breakout session reports into a document that was subsequently discussed and revised by participants. This was edited further and circulated for final review after the meeting. Participants from pharmaceutical companies were not part of the writing process. Conclusions: The clinical endpoint in paediatric GH treatment is adult height with height velocity as a surrogate endpoint. Increased life expectancy is the ideal but unfeasible clinical endpoint of GH treatment in adult GH-deficient patients (GHDA) and in patients with acromegaly. The pragmatic clinical endpoints in GHDA include normalization of body composition and quality of life, whereas symptom relief and reversal of comorbidities are used in acromegaly. Serum IGF-I is widely used as a biomarker, even though it correlates weakly with clinical endpoints in GH treatment, whereas in acromegaly, normalization of IGF-I may be related to improvement in mortality. There is an unmet need for novel biomarkers that capture the pleiotropic actions of GH in relation to GH treatment and in patients with acromegaly.
|
|
| 3. |
- Allen, D. B., et al.
(författare)
-
GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults
- 2016
-
Ingår i: European Journal of Endocrinology. - : Oxford University Press (OUP). - 0804-4643 .- 1479-683X. ; 174:2, s. P1-P9
-
Tidskriftsartikel (refereegranskat)abstract
- Recombinant human GH (rhGH) has been in use for 30 years, and over that time its safety and efficacy in children and adults has been subject to considerable scrutiny. In 2001, a statement from the GH Research Society (GRS) concluded that 'for approved indications, GH is safe'; however, the statement highlighted a number of areas for on-going surveillance of long-term safety, including cancer risk, impact on glucose homeostasis, and use of high dose pharmacological rhGH treatment. Over the intervening years, there have been a number of publications addressing the safety of rhGH with regard to mortality, cancer and cardiovascular risk, and the need for long-term surveillance of the increasing number of adults who were treated with rhGH in childhood. Against this backdrop of interest in safety, the European Society of Paediatric Endocrinology (ESPE), the GRS, and the Pediatric Endocrine Society (PES) convened a meeting to reappraise the safety of rhGH. The ouput of the meeting is a concise position statement.
|
|
| 4. |
- Saenger, P, et al.
(författare)
-
Recommendations for the diagnosis and management of Turner syndrome.
- 2001
-
Ingår i: The Journal of clinical endocrinology and metabolism. - : The Endocrine Society. - 0021-972X. ; 86:7, s. 3061-9
-
Tidskriftsartikel (refereegranskat)abstract
- Comprehensive recommendations on the diagnosis of Turner syndrome (TS) and the care of affected individuals were published in 1994. In the light of recent advances in diagnosis and treatment of TS, an international multidisciplinary workshop was convened in March 2000, in Naples, Italy, in conjunction with the Fifth International Symposium on Turner Syndrome to update these recommendations. The present paper details the outcome from this workshop. The genetics and diagnosis of the syndrome are described, and practical treatment guidelines are presented.
|
|
| 5. |
|
|
| 6. |
|
|
| 7. |
- Backeljauw, P, et al.
(författare)
-
Insulin-like growth factor-I in growth and metabolism
- 2010
-
Ingår i: Journal of pediatric endocrinology & metabolism : JPEM. - : Walter de Gruyter GmbH. - 0334-018X .- 2191-0251. ; 23:1-2, s. 3-16
-
Tidskriftsartikel (refereegranskat)
|
|
| 8. |
- Bidarian-Moniri, Armin, et al.
(författare)
-
Autoinflation compared to ventilation tubes for treating chronic otitis media with effusion
- 2022
-
Ingår i: Acta Oto-Laryngologica. - : Informa UK Limited. - 0001-6489 .- 1651-2251. ; 142:6, s. 476-483
-
Tidskriftsartikel (refereegranskat)abstract
- Background Otitis media with effusion (OME) is the most common cause of acquired hearing loss and surgery in children. Autoinflation has been suggested as an alternative treatment for OME. Objectives The aim of the study was to compare treatment outcome with a new autoinflation device versus ventilation tube (VT) surgery or watchful waiting in children with chronic bilateral OME from the waiting list for surgery. Methods Forty-five children performed autoinflation during four weeks, forty-five were submitted to VT surgery, and twenty-three were enrolled as control group. Tympanometry was performed in the autoinflation and the control groups and audiometry in all groups. Results An equivalent hearing improvement was achieved in the autoinflation and the VT group at one (p=.19), six (p=.23) and twelve (p=.31) months with no significant alteration in the control group. In the autoinflation group 80% of the children avoided surgery and no complications were reported compared to 34% complication rate in the VT group. Conclusion Autoinflation achieved an equivalent improvement in hearing thresholds compared to VT surgery for treating OME. Significance Autoinflation may be a reasonable first-line treatment for children with OME to potentially avoid surgery. Article Summary: The Moniri autoinflation device is well tolerated and an effective alternative to ventilation tubes for treatment of chronic otitis media with effusion in young children. What's known on this subject: Previous studies have shown that autoinflation may reduce effusion in children with otitis media with effusion; however limited compliance to treatment, lack of adequate hearing evaluation, short follow-up time and also lack of comparative data to ventilation tube surgery have been reported. What this study adds: A new device was developed to allow for the performance of autoinflation in young children. The effect is compared to ventilation tube surgery and equivalent improvement in hearing is achieved in the short and the long-term follow-up.
|
|
| 9. |
- Wit, J M., et al.
(författare)
-
Personalized Approach to Growth Hormone Treatment: Clinical Use of Growth Prediction Models
- 2013
-
Ingår i: Hormone Research in Paediatrics. - : Karger. - 1663-2818 .- 1663-2826. ; 79:5, s. 257-270
-
Forskningsöversikt (refereegranskat)abstract
- The goal of growth hormone (GH) treatment in a short child is to attain a fast catch-up growth toward the target height (TH) standard deviation score (SDS), followed by a maintenance phase, a proper pubertal height gain, and an adult height close to TH. The short-term response variable of GH treatment, first-year height velocity (HV) (cm/year or change in height SDS), can either be compared with GH response charts for diagnosis, age and gender, or with predicted HV based on prediction models. Three types of prediction models have been described: the Kabi International Growth Hormone Study models, the Gothenburg models and the Cologne model. With these models, 50-80% of the variance could be explained. When used prospectively, individualized dosing reduces the variation in growth response in comparison with a fixed dose per body weight. Insulin-like growth factor-I-based dose titration also led to a decrease in the variation. It is uncertain whether adding biochemical, genetic or proteomic markers may improve the accuracy of the prediction. Prediction models may lead to a more evidence-based approach to determine the GH dose regimen and may reduce the drug costs for GH treatment. There is a need for user-friendly software programs to make prediction models easily available in the clinic.
|
|
