Consensus and Discordance in the Management of Growth Hormone-Treated Patients: Results of a Knowledge, Attitudes, Beliefs, and Practices Survey

Abstract

Our purpose was to determine pediatric endocrinologists' knowledge, attitudes, beliefs, and practices (KABPs) regarding recombinant human growth hormone (rhGH) treatment, examine care-related attitude consensus or discordance, and identify evidence-based practice gaps. We developed a survey for National Cooperative Growth Study (NCGS) investigators (N = 711) to elicit their KABPs regarding GH stimulation testing as a diagnostic tool, IGF-1 monitoring for safety and dosing guidance, and pubertal dosing. Responses were compared with NCGS data from the last 20 years. Comparison between survey responses and NCGS data revealed potential discrepancies between expressed opinions and actual practice. In conclusion, this KABP survey, combined with NCGS data, suggests changes over time in diagnostic and rhGH-related therapeutic practices. Variability and inconsistency exist between the survey responses and practice trends over time as reflected in the NCGS database. Further study is necessary to provide evidence to guide rhGH treatment decisions.

Identification and management of poor response to growth‐promoting therapy in children with short stature

“…There are no definitive data concerning the long‐term safety of doses higher than 50 μg/kg per day in children with ISS. A GH dose of 70 μg/kg per day was approved in the USA for treatment of GHD in puberty, but this regimen is only used by one‐third of centres . In December 2010, the EMA issued guidance (http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2010/12/news_detail_001167.jsp&murl=menus/news_and_events/news_and_events.jsp&mid=WC0b01ac058004d5c1) not to exceed a GH dose of 50 μg/kg per day based on preliminary data from a French post‐marketing registry study, now published in addition to data from Belgium, the Netherlands and Sweden .…”

“…During the first year of GH therapy, children should be seen at 3–6 monthly intervals for assessment of growth, puberty, mood and body composition and to support compliance with therapy. These visits may be used to judge response to GH, but growth response cannot be reliably assessed at an interval shorter than 1 year GH dose adjustments during the first year of treatment may not have been as common in the past and may be less frequent in some countries than in others …”

How to Optimally Manage Growth Hormone Therapy: Survey of Chilean Pediatric Endocrinologists

“…Alternatively, the variety of cut-offs for GH testing used to define a GHD also remains controversial (particularly with the new generation of immunoassays) [13]. In the survey, it is interesting to note that most clinicians used a cut-off <7 ng/ml, which is stricter than what is still used (10 ng/ml) by the Growth Hormone Research Society [3,4].…”

“…Since 2000, clinician requests for a GH stimulation test had decreased almost 50%. Over the years, requests for an IGF-1 stimulation test increased from 24 to 41% and the use of IGF-1 without a stimulation test increased from 4 to 6% [12,13]. Moreover, within the suggested provocative test for GHD, insulin was mostly used for the induced hypoglycemia test (29%) [11].…”

“…With regard to laboratory assessments, we recommend that blood glucose, thyroid function, HbA1c, and IGF-1 should be monitored every 6–12 months to maintain these parameters within normal levels. Current guidelines consider that IGF-1 monitoring should be performed at least annually as it is the basis for adjusting the GH dose [1,3,4,7,13]. …”