Low dose growth hormone treatment in infants and toddlers with Prader-Willi syndrome is comparable to higher dosage regimens

Scheermeyer, Elly; Harris, Mark; Hughes, Ian; Crock, Patricia; Ambler, Geoffrey; Verge, Charles F.; Bergman, Philip S.; Werther, George A.; Craig, Maria E.; Choong, Catherine S.; Davies, Peter S. W.

doi:10.1016/j.ghir.2017.03.001

Cited by 16 publications

(14 citation statements)

References 35 publications

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“…In the past, a start of GHT during early childhood, especially before the onset of obesity at around 2 years of age, was widely recognized as beneficial [14]. More recent findings demonstrate that additional benefits can be obtained when treatment is started from as early as 2 to 6 months of life age [19,24,25]. Thus, starting GHT earlier during the first year of life and/or immediately after the diagnosis of PWS has become increasingly popular [14].…”

Section: Introductionmentioning

confidence: 99%

Early start of growth hormone is associated with positive effects on auxology and metabolism in Prader-Willi-syndrome

Magill

Laemmer

Woelfle

et al. 2020

Orphanet J Rare Dis

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Background Prader-Willi-Syndrome (PWS) is characterized by hypothalamic-pituitary dysfunction. Recent research suggests starting growth hormone-treatment (GHT) as soon as possible. The aim of this study is to analyze possible differences in auxological parameters, carbohydrate and lipid metabolism between two groups of children with PWS that started GHT either during or after their first year of life. Study design Retrospective longitudinal study of 62 children (31 males) with genetically confirmed PWS. Upon diagnosis all children were offered GHT, some started immediately, others commenced later. Cohort A (n = 21; 11 males) started GHT at 0.3–0.99 yrs. (mean 0.72 yrs) and Cohort B (n = 41; 20 males) commenced GHT at 1.02–2.54 yrs. (mean 1.42 yrs) of age. Fasting morning blood samples and auxological parameters were obtained before the start of therapy and semi-annually thereafter. Differences between the two cohorts were estimated with a linear mixed-effect model. Results Mean length/height-SDSPWS differed significantly between the groups [1 yr: A: 0.37 (±0.83) vs B: 0.05 (±0.56); 5 yrs.: A: 0.81 (±0.67) vs B: 0.54 (±0.64); p = 0.012]. No significant differences were found in BMI, lean body mass or body fat. Low-density cholesterol was significantly lower in A than in B [LDL: 1 yr: A: 79 (±20) mg/dl vs B: 90 (±19) mg/dl; 5 yrs.: A: 91(±18) mg/dl vs 104 (±26) mg/dl; p = 0.024]. We found significant differences in the glucose homeostasis between the groups [fasting insulin: p = 0.012; HOMA-IR: p = 0.006; HbA1c: p < 0.001; blood glucose: p = 0.022]. Conclusions An early start of GHT during the first year of life seems to have a favorable effect on height-SDS and metabolic parameters.

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Section: Introductionmentioning

confidence: 99%

Early start of growth hormone is associated with positive effects on auxology and metabolism in Prader-Willi-syndrome

Magill

Laemmer

Woelfle

et al. 2020

Orphanet J Rare Dis

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“…Although the approved rhGH dosage for patients with PWS is usually higher (1.0 mg/m 2 /d, which corresponds to 0.033–0.035 mg/kg/d), it is recommended to start therapy with a lower dose of 0.5 mg/m 2 /d [ 4 , 48 ]. The latest studies confirm the sustained effectiveness in terms of auxological response and safety of lower rhGH doses in infants with PWS (0.64 mg/m 2 /d) [ 49 ]. However, earlier rhGH dose–response studies have shown that a lower dose may not be beneficial for optimising and maintaining the improvement in body composition [ 3 , 7 , 38 , 50 ].…”

Section: Discussionmentioning

confidence: 84%

Effects of Recombinant Human Growth Hormone Treatment, Depending on the Therapy Start in Different Nutritional Phases in Paediatric Patients with Prader–Willi Syndrome: A Polish Multicentre Study

Lecka-Ambroziak

Wysocka‐Mincewicz

Doleżal-Ołtarzewska

et al. 2021

JCM

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Recombinant human growth hormone (rhGH) treatment is an established management in patients with Prader–Willi syndrome (PWS), with growth promotion and improvement in body composition and possibly the metabolic state. We compared anthropometric characteristics, insulin-like growth factor 1 (IGF1) levels, metabolic parameters and the bone age/chronological age index (BA/CA) in 147 children with PWS, divided according to age of rhGH start into four groups, corresponding to nutritional phases in PWS. We analysed four time points: baseline, rhGH1 (1.21 ± 0.81 years), rhGH2 (3.77 ± 2.17 years) and rhGH3 (6.50 ± 2.92 years). There were no major differences regarding height SDS between the groups, with a higher growth velocity (GV) (p = 0.00) and lower body mass index (BMI) SDS (p < 0.05) between the first and older groups during almost the whole follow-up. IGF1 SDS values were lower in group 1 vs. other groups at rhGH1 and vs. groups 2 and 3 at rhGH2 (p < 0.05). Glucose metabolism parameters were favourable in groups 1 and 2, and the lipid profile was comparable in all groups. BA/CA was similar between the older groups. rhGH therapy was most effective in the youngest patients, before the nutritional phase of increased appetite. We did not observe worsening of metabolic parameters or BA/CA advancement in older patients during a comparable time of rhGH therapy.

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“…GH therapy improves growth velocity and adult height for individuals affected by PWS and maintains lean body mass with age regardless of GH secretory status at baseline . The benefits of initiating GH therapy have been demonstrated for infants as young as 2–4 months of age and through early childhood . Currently, many children with PWS receive GH therapy, which lowers the body mass index (BMI) and, on average, provides the capacity to maintain BMI below two standard deviations above the age‐ and gender‐matched reference values of the British 1990 normative growth charts for the duration of therapy …”

Section: Resultsmentioning

confidence: 99%

“…There have been limited Australian studies of PWS . This gap is particularly relevant for Australian families with the rollout of the National Disability Insurance Scheme where caregivers must provide evidence of therapeutic benefit from supports and services to receive funding for their child.…”

Section: Discussionmentioning

confidence: 99%

Requirements for improving health and well‐being of children with Prader‐Willi syndrome and their families

Mackay

McCallum

Ambler

et al. 2019

J Paediatrics Child Health

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Prader‐Willi syndrome (PWS) is a rare genetic condition with multi‐system involvement. The literature was reviewed to describe neurodevelopment and the behavioural phenotype, endocrine and metabolic disorders and respiratory and sleep functioning. Implications for child and family quality of life were explored. Challenging behaviours contribute to poorer well‐being and quality of life for both the child and caregiver. Recent evidence indicates healthy outcomes of weight and height can be achieved with growth hormone therapy and dietary restriction and should be the current target for all individuals with PWS. Gaps in the literature included therapies to manage challenging behaviours, as well as understanding the effects of growth hormone on respiratory and sleep function. New knowledge regarding the transition of children and families from schooling and paediatric health services to employment, accommodation and adult health services is also needed. Developing a national population‐based registry could address these knowledge gaps and inform advocacy for support services that improve the well‐being of individuals with PWS and their families.

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Low dose growth hormone treatment in infants and toddlers with Prader-Willi syndrome is comparable to higher dosage regimens

Cited by 16 publications

References 35 publications

Early start of growth hormone is associated with positive effects on auxology and metabolism in Prader-Willi-syndrome

Early start of growth hormone is associated with positive effects on auxology and metabolism in Prader-Willi-syndrome

Effects of Recombinant Human Growth Hormone Treatment, Depending on the Therapy Start in Different Nutritional Phases in Paediatric Patients with Prader–Willi Syndrome: A Polish Multicentre Study

Requirements for improving health and well‐being of children with Prader‐Willi syndrome and their families

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