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Title: | Low dose growth hormone treatment in infants and toddlers with Prader-Willi syndrome is comparable to higher dosage regimens. | Authors: | Hughes I.;Davies P.S.W.;Bergman P. ;Harris M. ;Choong C.S.;Scheermeyer E.;Crock P.A.;Craig M.E.;Werther G.;Verge C.F.;Ambler G. | Institution: | (Scheermeyer) Faculty of Medicine, Primary Care Clinical Unit, The University of Queensland, Brisbane, Australia (Scheermeyer, Davies) Child Health Research Centre, The University of Queensland, Brisbane, Australia (Harris) Lady Cilento Children's Hospital, Brisbane, Australia (Harris, Hughes) Mater Research Institute - UQ, The University of Queensland, Brisbane, Australia (Crock) John Hunter Children's Hospital, School of Medicine and Public Health, University of Newcastle, Newcastle, Australia (Ambler, Craig) The Children's Hospital at Westmead and Discipline of Child and Adolescent Health, The University of Sydney, Sydney, Australia (Verge) Sydney Children's Hospital, School of Women's and Children's Health, University of New South Wales, Sydney, Australia (Bergman) Monash Medical Centre, Melbourne, Australia (Werther) The Royal Children's Hospital, Melbourne, Australia (Choong) Princess Margaret Hospital, School of Paediatrics and Child Health, University of Western Australia, Perth, Australia | Issue Date: | 27-Apr-2017 | Copyright year: | 2017 | Publisher: | Churchill Livingstone | Place of publication: | United Kingdom | Publication information: | Growth Hormone and IGF Research. 34 (pp 1-7), 2017. Date of Publication: 01 Jun 2017. | Journal: | Growth Hormone and IGF Research | Abstract: | Objective Evaluate benefit and risk of low dose growth hormone treatment (GHT, 4.5 mg/m2/week) in very young children with Prader-Willi Syndrome (PWS). Design Prospective longitudinal clinical intervention. Methods We evaluated 31 infants (aged 2-12 months) and 42 toddlers (13-24 months) from the PWS-OZGROW database for height, weight and BMI using the World Health Organization standard deviation scores (SDSWHO) and PWS specific BMI (SDSPWS), bone age, insulin-like growth factor 1 (IGF-I) levels and adverse events over 3 years of GHT. Results At commencement of GHT infants had a lower BMI SDSWHO (- 0.88 vs 0.40) than toddlers, while toddlers had a lower height SDSWHO (- 1.44 vs - 2.09) (both P < 0.05). All increased height SDSWHO (2 year delta height infants + 1.26 SDS, toddlers + 1.21 SDS), but infants normalised height sooner, achieving a height SDS of - 0.56 within 1 year, while toddlers achieved a height SDS of - 0.88 in two years. BMI SDSWHO increased, while BMI SDSPWS decreased (both P < 0.0001) and remained negative. The GHT response did not differ with gestation (preterm 23%) or genetic subtype (deletion vs maternal uniparental disomy). Bone age advancement paralleled chronological age. All children had low serum IGF-I at baseline which increased, but remained within the age-based reference range during GHT (for 81% in first year). Four children had spinal curvature at baseline; two improved, two progressed to a brace and two developed an abnormal curve over the observation period. Mild to severe central and/or obstructive sleep apnoea were observed in 40% of children prior to GHT initiation; 11% commenced GHT on positive airway pressure (PAP), oxygen or both. Eight children ceased GHT due to onset or worsening of sleep apnoea: 2 infants in the first few months and 6 children after 6-24 months. Seven resumed GHT usually after adjusting PAP but five had adenotonsillectomy. One child ceased GHT temporarily due to respiratory illness. No other adverse events were reported. Two children substantially improved their breathing shortly after GHT initiation. Conclusion Initiation of GHT in infants with 4.5 mg/m2/week was beneficial and comparable in terms of auxological response to a dose of 7 mg/m2/week. Regular monitoring pre and post GH initiation assisted in early detection of adverse events. IGF-I levels increased with the lower dose but not excessively, which may lower potential long-term risks.Copyright © 2017 Elsevier Ltd | DOI: | http://monash.idm.oclc.org/login?url=http://dx.doi.org/10.1016/j.ghir.2017.03.001 | Link to associated publication: | Click here for full text options | PubMed URL: | 28427039 [http://www.ncbi.nlm.nih.gov/pubmed/?term=28427039] | ISSN: | 1096-6374 | URI: | https://repository.monashhealth.org/monashhealthjspui/handle/1/38381 | Type: | Article | Subjects: | sleep disordered breathing/si [Side Effect] spine disease treatment response treatment withdrawal *human growth hormone/ae [Adverse Drug Reaction] *human growth hormone/do [Drug Dose] *human growth hormone/dt [Drug Therapy] *human growth hormone/ec [Endogenous Compound] somatomedin C/ec [Endogenous Compound] longitudinal study adenotonsillectomy airway pressure article body height body mass body weight bone age child disease course disease severity drug megadose drug withdrawal female gestational age hormone blood level human infant low drug dose major clinical study male *Prader Willi syndrome/dt [Drug Therapy] prospective study respiratory tract disease/si [Side Effect] gestational age hormone blood level human infant longitudinal study low drug dose major clinical study male *Prader Willi syndrome / *drug therapy prospective study respiratory tract disease / side effect treatment response treatment withdrawal body height Article airway pressure adenotonsillectomy sleep disordered breathing / side effect body weight bone age child disease course disease severity drug megadose drug withdrawal female spine disease body mass |
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