Recombinant growth hormone therapy in children with short stature in Kuwait: a cross-sectional study of use and treatment outcomes

BMC Endocrine Disorders

Iman Al-Basari¹  Kholoud Hassan¹  Abdullah Al-Taiar³  Dalia Al-Abdulrazzaq² 

[1] Department of Pediatrics, Mubarak Al-Kabeer Hospital, Ministry of Health, Safat, Kuwait;Department of Pediatrics, Faculty of Medicine, Kuwait University, Safat 13110, Kuwait;Department of Community Medicine, Faculty of Medicine, Kuwait University, Safat 13110, Kuwait

关键词: Middle East;    Kuwait;    Therapy;    Growth Hormone;    Short Stature;   

Others  :  1234624
DOI  :  10.1186/s12902-015-0073-7

 received in 2015-07-21, accepted in 2015-11-25,  发布年份 2015

【 摘 要 】

Background

Recombinant Growth hormone (rGH) therapy is approved in many countries for treatment of short stature in a number of childhood diagnoses. Despite the increasing body of international literature on rGH use, there is paucity of data on rGH use in Kuwait and the broader Middle-East which share unique ethnic and socio-cultural backgrounds. This study aimed to describe the pattern of use and treatment outcomes of rGH therapy in Kuwait.

Methods

This is a cross-sectional retrospective review of children treated with rGH in the Department of Pediatrics, in a major hospital in Kuwait between December 2013 and December 2014. Data were extracted using standard data extraction form and the response to rGH therapy was defined as a gain of ≥ 0.3 standard deviation score (SDS) of height per year.

Results

A total of 60 children were treated with rGH in the center. Their Median (Interquartile) age at rGH initiation was 9.0 (6.2, 10.7) years. The most common indications for rGH therapy were Growth Hormone Deficiency (GHD) 23 (38.3 %), Idiopathic Short Stature (ISS) 12 (20.0 %) and Small for Gestational Age (SGA) 9 (15.0 %). After excluding patients with TS, no significant differences were found in gender of those who received rGH therapy in all indications combined or in each group (p ≥ 0.40). At 1-year follow-up, children in all groups had median height SDS change of ≥ 0.3 SDS except for children with ISS. Age at rGH initiation was negatively associated with 1-year treatment response, Adjusted odds ratio (AOR) 0.56 (95 % CI: 0.04–1.49); p = 0.011).

Conclusions

GHD is the most common indication of rGH therapy. All indications except for ISS showed significant 1-year treatment response to therapy. Treatment outcomes in patients with ISS should be further investigated in Kuwait. Younger age at initiation of rGH therapy was independently associated with significant response to therapy suggesting the importance of identifying children with short stature and prompt initiation of rGH therapy.

【 授权许可】

   
2015 Al-Abdulrazzaq et al.

【 图 表 】

Fig. 1.

【 参考文献 】

• [1]Gharib H, Cook DM, Saenger PH, Bengtsson BA, Feld S, Nippoldt TB et al.. American Association of Clinical Endocrinologists medical guidelines for clinical practice for growth hormone use in adults and children--2003 update. Endocr Pract. 2003; 9(1):64-76.
• [2]Wilson TA, Rose SR, Cohen P, Rogol AD, Backeljauw P, Brown R et al.. Update of guidelines for the use of growth hormone in children: the Lawson Wilkins Pediatric Endocrinology Society Drug and Therapeutics Committee. J Pediatr. 2003; 143(4):415-21.
• [3]Richmond E, Rogol AD. Current indications for growth hormone therapy for children and adolescents. Endocr Dev. 2010; 18:92-108.
• [4]Lee PA, Sävendahl L, Oliver I, Tauber M, Blankenstein O, Ross J et al.. Comparison of response to 2-years' growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies. Int J Pediatr Endocrinol. 2012; 2012(1):22. BioMed Central Full Text
• [5]Carel JC, Ecosse E, Nicolino M, Tauber M, Leger J, Cabrol S et al.. Adult height after long term treatment with recombinant growth hormone for idiopathic isolated growth hormone deficiency: observational follow up study of the French population based registry. BMJ. 2002; 325(7355):70.
• [6]Hintz RL, Attie KM, Baptista J, Roche A. Effect of growth hormone treatment on adult height of children with idiopathic short stature. Genentech Collaborative Group. N Engl J Med. 1999; 340(7):502-7.
• [7]Park HK, Lee HS, Ko JH, Hwang IT, Hwang JS. Response to three years of growth hormone therapy in girls with Turner syndrome. Ann Pediatr Endocrinol Metab. 2013; 18(1):13-8.
• [8]Bannink E, Djurhuus CB, Christensen T, Jøns K, Hokken-Koelega A. Adult height and health-related quality of life after growth hormone therapy in small for gestational age subjects. J Med Econ. 2010; 13(2):221-7.
• [9]Bryant J, Djurhuus CB, Christensen T, Jøns K, Hokken-Koelega A. Recombinant growth hormone for idiopathic short stature in children and adolescents. Cochrane Database Syst Rev. 2007; 3: Article ID CD004440
• [10]Public Authority for Civil Information (PACI), Population Statistical Reports. 2014 [cited 2015 July 12th]; Available from:. https://www. paci.gov.kw/Home.aspx
• [11]Cohen P, Rogol AD, Deal CL, Saenger P, Reiter EO, Ross JL et al.. Consensus statement on the diagnosis and treatment of children with idiopathic short stature: a summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop. J Clin Endocrinol Metab. 2008; 93(11):4210-7.
• [12]Lee PA, Chernausek SD, Hokken-Koelega AC. Czernichow P; International Small for Gestational Age Advisory Board. International Small for Gestational Age Advisory Board consensus development conference statement: management of short children born small for gestational age, April 24-October 1, 2001. Pediatrics. 2003; 111(6 Pt 1):1253-61.
• [13]Clayton PE, Cianfarani S, Czernichow P, Johannsson G, Rapaport R, Rogol A. Management of the child born small for gestational age through to adulthood: a consensus statement of the International Societies of Pediatric Endocrinology and the Growth Hormone Research Society. J Clin Endocrinol Metab. 2007; 92(3):804-10.
• [14]WHO Child Growth Standards based on length/height, weight and age. Acta Paediatr Suppl. 2006; 450:76-85.
• [15]Ranke MB, Lindberg A, Board KI. Observed and predicted growth responses in prepubertal children with growth disorders: guidance of growth hormone treatment by empirical variables. J Clin Endocrinol Metab. 2010; 95(3):1229-37.
• [16]Bell J, Parker KL, Swinford RD, Hoffman AR, Maneatis T, Lippe B. Long-term safety of recombinant human growth hormone in children. J Clin Endocrinol Metab. 2010; 95(1):167-77.
• [17]Carel JC, Ecosse E, Landier F, Meguellati-Hakkas D, Kaguelidou F, Rey G et al.. Long-term mortality after recombinant growth hormone treatment for isolated growth hormone deficiency or childhood short stature: preliminary report of the French SAGhE study. J Clin Endocrinol Metab. 2012; 97(2):416-25.
• [18]Ross J, Lee PA, Gut R, Germak J. Factors influencing the one- and two-year growth response in children treated with growth hormone: analysis from an observational study. Int J Pediatr Endocrinol. 2010; 2010:494656. BioMed Central Full Text
• [19]Darendeliler F, Lindberg A, Wilton P. Response to growth hormone treatment in isolated growth hormone deficiency versus multiple pituitary hormone deficiency. Horm Res Paediatr. 2011; 76 Suppl 1:42-6.
• [20]Huang YH, Wai YY, Van YH, Lo FS. Effect of growth hormone therapy on Taiwanese children with growth hormone deficiency. J Formos Med Assoc. 2012; 111(7):355-63.
• [21]Deodati A, Cianfarani S. Impact of growth hormone therapy on adult height of children with idiopathic short stature: systematic review. BMJ. 2011; 342:c7157.
• [22]Grimberg A, Huerta-Saenz L, Grundmeier R, Ramos MJ, Pati S, Cucchiara AJ, et al. Gender Bias in U.S. Pediatric Growth Hormone Treatment. Sci Rep. 2015;5:11099.
• [23]Grimberg A, Stewart E, Wajnrajch MP. Gender of pediatric recombinant human growth hormone recipients in the United States and globally. J Clin Endocrinol Metab. 2008; 93(6):2050-6.
• [24]Hughes IP, Choong CS, Cotterill A, Harris M, Davies PS. Gender bias in children receiving growth hormone treatment. J Clin Endocrinol Metab. 2010; 95(3):1191-8.
• [25]Renes JS, Willemsen RH, Mulder JC, Bakker-van Waarde WM, Rotteveel J, Oostdijk W et al.. New insights into factors influencing adult height in short SGA children: Results of a large multicentre growth hormone trial. Clin Endocrinol (Oxf). 2015; 82(6):854-61.
• [26]Reiter EO, Price DA, Wilton P, Albertsson-Wikland K, Ranke MB. Effect of growth hormone (GH) treatment on the near-final height of 1258 patients with idiopathic GH deficiency: analysis of a large international database. J Clin Endocrinol Metab. 2006; 91(6):2047-54.
• [27]Ranke MB, Lindberg A, Price DA, Darendeliler F, Albertsson-Wikland K, Wilton P et al.. Age at growth hormone therapy start and first-year responsiveness to growth hormone are major determinants of height outcome in idiopathic short stature. Horm Res. 2007; 68(2):53-62.
• [28]Finkelstein BS, Imperiale TF, Speroff T, Marrero U, Radcliffe DJ, Cuttler L. Effect of growth hormone therapy on height in children with idiopathic short stature: a meta-analysis. Arch Pediatr Adolesc Med. 2002; 156(3):230-40.
• [29]Ranke MB. Towards a consensus on the definition of idiopathic short stature. Horm Res. 1996; 45 Suppl 2:64-6.
• [30]Growth Hormone Research S. Consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency in childhood and adolescence: summary statement of the GH Research Society. GH Research Society. J Clin Endocrinol Metab. 2000; 85(11):3990-3.
• [31]Rose SR, Shulman DI, Larsson P, Wakley LR, Wills S, Bakker B. Gender does not influence prepubertal growth velocity during standard growth hormone therapy--analysis of United States KIGS data. J Pediatr Endocrinol Metab. 2005; 18(11):1045-51.
• [32]Lee PA, Germak J, Gut R, Khutoryansky N, Ross J. Identification of factors associated with good response to growth hormone therapy in children with short stature: results from the ANSWER Program(R). Int J Pediatr Endocrinol. 2011; 2011:6. BioMed Central Full Text