Program: Abstracts - Orals, Featured Poster Presentations, and Posters
Session: FP32-Health Outcomes & Quality Improvement
Monday, June 17, 2013: 10:45 AM-11:15 AM
Presentation Start Time: 10:50 AM
Room 301 (Moscone Center)

Poster Board MON-284
Meryl Brod*1, Lise Højbjerre2, Erpur Adalsteinsson2 and Michael Højby2
1The Brod Group, Mill Valley, CA, 2Novo Nordisk A/S, Søborg, Denmark
Introduction:  The ability to assess the full range of impacts of Growth Hormone Deficiency (GHD)   on adult patients with GHD (AGHD) is critical to allow clinicians to better monitor treatment effects and develop tailored treatments with improved outcomes. Unfortunately, the current measures to assess all treatment outcomes in GHD adults have been criticized for not being sensitive to disease severity and expert clinicians have suggested that a new, more sensitive measure is needed.

Methods: The Treatment Related Impact Measure (TRIM-AGHD) was developed according to rigorous scientific principles and following FDA guidelines for the development of patient reported measures , including interviews with  thirty nine  patient  and six clinical experts in three countries (USA, UK, Germany). Based on   qualitative data analysis and a theoretic al model derived from the analysis, a preliminary forty six item TRIM-AGHD was developed and cognitively debriefed to ensure comprehension, readability, acceptability and relevance.

 A validation study was then conducted to assess the measurement and psychometric properties of the preliminary TRIM-AGHD. Eligible, subjects were above 18 years of age, had a diagnosis of GHD of any etiology, received GH replacement therapy for > 1 month, began GH therapy either for the first time or had been off treatment for at least six months. Patients were recruited by physicians from their AGHD patient pool.

Results:  169 patients completed the validation study, mean age 52 years (range 19-79), 73% female, 89% white, and the primary cause of GHD were unknown (38.2%), pituitary tumor or other disorders (34.2%) and head trauma (9.9%).

The final TRIM-AGHD has twenty six items with four distinct domains which were labeled “Energy”, “Psychological”, “Cognitive”, and “Physical”. No problems were found for missing data (≤ 3%) or floor effects (<25%). Internal consistency coefficients (total score and all domains) were acceptable (range 0.82 and 0.95 as was test-retest reliability coefficients (range 0.75 to 0.87) and internal consistency coefficients (range 0.82 and 0.95). All convergent validity hypotheses and all but two of the known group validity hypotheses were met. In patients who had just begun treatment at the start of the validation study a marked improvement in scores were noted for each of the TRIM-AGHD domains (range 13.2 and 18.0 points on a 0-100-point scale) with associated effect sizes ranging from 1.1 to 1.9, indicating that the TRIM-AGHD is sensitive to change. The average time to complete was three minutes.

Conclusions: The TRIM-AGHD can be considered a well-designed, sensitive, responsive, valid and reliable measure of the impact of GH treatment on the functioning and well-being of patients. This measure should prove useful in clinical trials to assess impacts related to AGHD as well as to clinicians in tailoring treatments to patient needs.

Disclosure: MB: Consultant, Novo Nordisk. LH: Employee, Novo Nordisk. EA: Employee, Novo Nordisk, Owner, Novo Nordisk. MH: Employee, Novo Nordisk.

*Please take note of The Endocrine Society's News Embargo Policy at http://www.endo-society.org/endo2013/media.cfm

Sources of Research Support: Novo Nordisk A/S