Growth hormone therapy and treatment outcomes: current clinical practice of the Gulf Cooperation Council

Abstract

Over the last 20 years, recombinant human growth hormone (somatropin) has been the cornerstone of managing children with growth hormone deficiency (GHD). Although both international and national guidelines for growth hormone (GH) therapy exist, there is currently no consensus on the optimal use of GH therapy in Gulf Cooperation Council (GCC) countries. The goals of GH therapy are to normalize height during childhood, attain normal adult height and correct metabolic abnormalities related to GHD. However, extended use of GH >50 µg/kg/day may increase frequency of adverse events. Here, we report the proceedings from a meeting of nine GCC pediatric endocrinology experts, which took place in Beirut in November 2011. The meeting was also attended by three European counterparts and aimed to provide consensus on best practice in the management of children with GHD in the GCC based on current local medical and regulatory environments.

Keywords::

• adherence
• diagnosis
• growth hormone deficiency
• response
• therapy

Acknowledgements

We wish to thank Professors Peter Bang, Fritz Haverkamp and Sandro Loche, the three international experts who delivered short presentations at the meeting and participated in discussions. The meeting was sponsored by Merck Serono S.A. (Merck Serono S.A., Coinsins, Switzerland, is an affiliate of Merck KGaA, Darmstadt, Germany). The authors also wish to express their gratitude to Merck Serono S.A. which developed the premeeting survey based on collaboration with two experts (F. Haverkamp and S. Norgren).

Financial & competing interests disclosure

Khaled Esmat is an employee of Merck Serono Middle East, a branch of Merck Serono S.A., Coinsins, Switzerland, an affiliate of Merck KGaA, Darmstadt, Germany. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. Writing assistance was provided by PHOCUS Medical Communications. We acknowledge Dominique Spirig of PHOCUS Services Ltd, funded by Merck Serono S.A., for providing medical writing support.

Key issues

• The majority (7/8) of Gulf Cooperation Council (GCC) experts adhere to international guidelines for growth hormone (GH) therapy, rather than to national ones.

• Most experts at this meeting (9/12) were satisfied with the range of GH therapies currently available; major concerns were inadequate efficacy (5/12) and cost (4/12). However, no single optimal approach to GH therapy currently exists given the individual genetic nature of both the diagnosis and the response.

• Early and improved diagnosis was most frequently (7/12) considered a single critical step in optimizing GH therapy. Therapy initiated at the earliest opportunity in prepubertal individuals with GH deficiency is likely to lead to a final height within the genetic range (i.e., around the mid-parental height).

• Most GCC experts (7/8) assessed their patients’ responses to GH therapy every 3 months during the first year of treatment; of all the experts at the meeting, 11 out of 12 recommended monitoring IGF-1 levels to achieve optimal outcomes.

• The majority (10/12) of experts expected the optimal effects of GH therapy during the first year of treatment, with height velocity (7/8), IGF-1 levels (6/8) and height standard deviation score (ΔHSDS) (5/8) regarded by most GCC experts as important quantitative growth response parameters.

• The preferred definition for a ‘good’ response during the first year of therapy was ΔHSDS >1.0 (6/12). Most of the experts surveyed agreed that the best definition for a ‘poor’ response was ΔHSDS <0.4 (7/12).

• The majority of the GCC experts (6/8) recognized the variable adherence to GH therapy as the central issue and the most likely cause for a suboptimal growth response, and motivational conversations with the patient (8/8) were universally recommended. Automatic recording of doses administered were recommended by most experts at the meeting (11/12), as well as social/economic and therapy-related interventions and healthcare team education.

Notes