Affiliation:
1. The Brod Group (M.B.), Mill Valley, California 94941;
2. Department of Global Development (L.H., J.E.A., M.H.R.), Novo Nordisk A/S, DK-2860 Søborg, Denmark
Abstract
Context:
Approximately 50 000 adults in the United States are diagnosed with GH deficiency, which has negative impacts on cognitive functioning, psychological well-being, and quality of life.
Objective:
This paper presents development and validation of a patient-reported outcome measure (PRO), the Treatment-Related Impact Measure-Adult Growth Hormone Deficiency (TRIM-AGHD). The TRIM-AGHD was developed to measure the impact of GH deficiency and its treatment.
Design and Settings:
The development and validation of the TRIM-AGHD was conducted according to the Food and Drug Administration guidance on the development of PROs. Concept elicitation, conducted in three countries included interviews with patients, clinical experts, and literature review. Qualitative data were analyzed based on grounded theory principles, and draft items were cognitively debriefed. The measure underwent psychometric validation in a US clinic-based population. An a priori statistical analysis plan included assessment of the measurement model, reliability, and validity. Item functioning was reviewed using item response theory analyses.
Patients or Other Participants:
Forty-eight patients and six clinical experts participated in concept elicitation and 169 patients completed the validation study.
Main Outcome Measure:
TRIM-AGHD was measured.
Results:
Factor analysis resulted in four domains: energy level, physical health, emotional health, and cognitive ability. The item response theory confirmed adequate item fit and placement within their domain. Internal consistency ranged from 0.82 to 0.95 and test-retest ranged from 0.80 to 0.92. All prespecified hypotheses for convergent validity and all but two for discriminant validity were met.
Conclusions:
The final 26-item TRIM-AGHD can be considered a reliable and valid PRO of the impact of disease and treatment for adult GH deficiency.
Subject
Biochemistry (medical),Clinical Biochemistry,Endocrinology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference33 articles.
1. The growth hormone deficiency syndrome in adults;Cuneo;Clin Endocrinol (Oxf),1992
2. The Human Growth Foundation. Adult growth hormone deficiency explained. 2013. http://www.hgfound.org/res_aghd.explained.asp. Accessed April 11, 2013.
3. Growth hormone deficiency in adulthood and the effects of growth hormone replacement: a review. Growth Hormone Research Society Scientific Committee;Carroll;J Clin Endocrinol Metab,1998
4. Body composition, bone metabolism, and heart structure and function in growth hormone (GH)-deficient adults before and after GH replacement therapy at low doses;Amato;J Clin Endocrinol Metab,1993
5. Changes in muscle volume, strength, and bioenergetics during recombinant human growth hormone (GH) therapy in adults with GH deficiency;Janssen;J Clin Endocrinol Metab,1999
Cited by
8 articles.
订阅此论文施引文献
订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献