Quality of life and psychological well‐being in GH‐treated, adult PWS patients: a longitudinal study

Bertella, Laura; Mori, Ileana; Grugni, Graziano; Pignatti, Riccardo; Ceriani, Florencia; Molinari, Enrico; Ceccarelli, Anna; Sartório, Alessandro; Vettor, Roberto; Semenza, Carlo

doi:10.1111/j.1365-2788.2006.00878.x

Cited by 52 publications

(46 citation statements)

References 37 publications

(26 reference statements)

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Two studies showed that GH treatment can improve the physical and psychological aspects of health-related quality of life (HRQOL) in adolescents and adults with PWS [7,8], but information about HRQOL or the effect of GH treatment on HRQOL in children with PWS is scarce. One study investigated HRQOL in 19 adolescents after 10 years of GH treatment and observed a lower HRQOL than in the general population on the items of mobility, breathing, speech, mental function, going to school, and getting and having friends [9].…”

Section: Introductionmentioning

confidence: 99%

Beneficial Effect of Growth Hormone Treatment on Health-Related Quality of Life in Children with Prader-Willi Syndrome: A Randomized Controlled Trial and Longitudinal Study

Bakker

Siemensma²,

Rijn³

et al. 2015

Horm Res Paediatr

View full text Add to dashboard Cite

Background/Aims: Growth hormone (GH) treatment is beneficial for children with Prader-Willi syndrome (PWS), but data about health-related quality of life (HRQOL) and effects of GH treatment are scarce. We, therefore, investigated the effects of GH treatment on HRQOL in PWS children. Methods: In a randomized controlled GH trial including 26 PWS children and during an 11-year longitudinal GH study in 76 children, we annually assessed HRQOL recorded by patients and parents, using a generic questionnaire (DUX25), containing 4 subdomains (Physical, Home, Social, and Emotional) and a PWS-specific questionnaire (DUXPW). Results: At baseline, PWS children rated HRQOL similar to or higher than healthy and obese children. GH-treated children reported an increase in HRQOL in the Physical and Social subdomains and the DUXPW compared to untreated children. Parents reported an increase in the Physical and Emotional subdomains and borderline in the total DUX25 compared to parents of untreated children. During the 11 years of GH treatment, the Physical subdomain continued to improve, according to parents, whereas the Home, Social and Emotional subdomains, the total DUX25, and the DUXPW remained similar, according to children and parents. Conclusions: PWS children rated HRQOL equally to or better than healthy and obese children. HRQOL increased during GH treatment, in contrast to HRQOL of untreated children. This effect was sustained during long-term GH treatment. PWS children consider themselves quite happy, despite some difficulties related to the syndrome.

show abstract

Section: Introductionmentioning

confidence: 99%

Beneficial Effect of Growth Hormone Treatment on Health-Related Quality of Life in Children with Prader-Willi Syndrome: A Randomized Controlled Trial and Longitudinal Study

Bakker

Siemensma²,

Rijn³

et al. 2015

Horm Res Paediatr

View full text Add to dashboard Cite

show abstract

“…We identified nine papers that looked at QoL in other growth-related conditions treated with growth hormone. [36][37][38][39][40][41][42][43][44] Again these presented a variety of results, but only limited evidence that there is a strong link between QoL and final height gain. Several of the studies called for further research into the links between QoL and short stature and the tool with which this is evaluated.…”

Section: Resultsmentioning

confidence: 99%

Growth monitoring for short stature: update of a systematic review and economic model

Craig

Fayter²,

Stirk³

et al. 2011

Health Technol Assess

View full text Add to dashboard Cite

An electronic version of this title, in Adobe Acrobat format, is available for downloading free of charge for personal use from the HTA website (www.hta.ac.uk). A fully searchable DVD is also available (see below).Printed copies of HTA journal series issues cost £20 each (post and packing free in the UK) to both public and private sector purchasers from our despatch agents.Non-UK purchasers will have to pay a small fee for post and packing. For European countries the cost is £2 per issue and for the rest of the world £3 per issue. How to order:-fax (with credit card details) -post (with credit card details or cheque) -phone during office hours (credit card only).Additionally the HTA website allows you to either print out your order or download a blank order form. Contact details are as follows:Synergie UK (HTA Department) Digital House, The Loddon Centre Wade Road Basingstoke Hants RG24 8QW Email: orders@hta.ac.uk Tel: 0845 812 4000 -ask for 'HTA Payment Services' (out-of-hours answer-phone service) Fax: 0845 812 4001 -put 'HTA Order' on the fax header Payment methods Paying by chequeIf you pay by cheque, the cheque must be in pounds sterling, made payable to University of Southampton and drawn on a bank with a UK address.Paying by credit card You can order using your credit card by phone, fax or post. SubscriptionsNHS libraries can subscribe free of charge. Public libraries can subscribe at a reduced cost of £100 for each volume (normally comprising 40-50 titles). The commercial subscription rate is £400 per volume (addresses within the UK) and £600 per volume (addresses outside the UK). Please see our website for details. Subscriptions can be purchased only for the current or forthcoming volume.How do I get a copy of HTA on DVD?Please use the form on the HTA website (www.hta.ac.uk/htacd/index.shtml). HTA on DVD is currently free of charge worldwide.The website also provides information about the HTA programme and lists the membership of the various committees. ii ii HTA NIHR Health Technology Assessment programmeThe Health Technology Assessment (HTA) programme, part of the National Institute for Health Research (NIHR), was set up in 1993. It produces high-quality research information on the effectiveness, costs and broader impact of health technologies for those who use, manage and provide care in the NHS. 'Health technologies' are broadly defined as all interventions used to promote health, prevent and treat disease, and improve rehabilitation and long-term care. The research findings from the HTA programme directly influence decision-making bodies such as the National Institute for Health and Clinical Excellence (NICE) and the National Screening Committee (NSC). HTA findings also help to improve the quality of clinical practice in the NHS indirectly in that they form a key component of the 'National Knowledge Service' . The HTA programme is needs led in that it fills gaps in the evidence needed by the NHS. There are three routes to the start of projects. First is the commissioned route. Suggestions for resea...

show abstract

“…Bertella et al 23) evaluated quality of life by physical and psychological well-being questionnaires (SF 36) self-administered by the patients or their caregivers. They found significant improvements in both scales, with greater improvements noted in patient questionnaires than in questionnaires completed by their caregivers.…”

Section: ) Quality Of Lifementioning

confidence: 99%

Growth Hormone Therapy in Adults with Prader-Willi Syndrome

Cho¹

2015

Journal of mucopolysaccharidosis and rare disease

View full text Add to dashboard Cite

Prader-Willi syndrome (PWS) is a complex multisystem genetic disorder characterized by hypothalamic-pituitary dysfunction. Many features of PWS indicate a deficiency in growth hormone (GH) production, and these findings provide a rationale for GH therapy in PWS. It is possible that rhGH therapy could have beneficial effects in adults with PWS, similar to those in adults with GH deficiency (GHD) of non-syndromic cause. However, there is a paucity of data on the use of GH in adults with PWS. Here, the previous studies about efficacy and safety of rhGH therapy in PWS adults are summarized. Briefly, rhGH therapy in PWS adults may improve body composition, leading to increased lean body mass and decreased fat mass, as well as decreased subcutaneous and visceral adiposity without overall changes in body mass index. There may be at least transient deterioration in glucose homoeostasis in some PWS patients on rhGH therapy, which requires further study. In addition, clinical care guidelines for rhGH therapy in adults with PWS were suggested.

show abstract

Quality of life and psychological well‐being in GH‐treated, adult PWS patients: a longitudinal study

Abstract: Our findings suggest that the amelioration of QoL and psychological status is sustained in patients who continue GH treatment.

Cited by 52 publications

References 37 publications

Beneficial Effect of Growth Hormone Treatment on Health-Related Quality of Life in Children with Prader-Willi Syndrome: A Randomized Controlled Trial and Longitudinal Study

Beneficial Effect of Growth Hormone Treatment on Health-Related Quality of Life in Children with Prader-Willi Syndrome: A Randomized Controlled Trial and Longitudinal Study

Growth monitoring for short stature: update of a systematic review and economic model

Growth Hormone Therapy in Adults with Prader-Willi Syndrome

Contact Info

Product

Resources

About