Three-year Experience With Access to Nationally Funded Growth Hormone (GH) Replacement for GH-deficient Adults

I.M. Holdaway; P. Hunt; P. Manning; W. Cutfield; G. Gamble; N. Ninow; D. Staples-Moon; P. Moodie; S. Metcalfe

Disclosures

Clin Endocrinol. 2015;83(1):85-90. 

In This Article

Abstract and Introduction

Abstract

Objective Treatment of growth hormone (GH)-deficient adults with GH has been shown to improve a range of metabolic abnormalities and enhance quality of life. However, the results of access to nationally funded treatment have not been reported.

Design Retrospective case series auditing nationally funded treatment of defined GH-deficient adults in New Zealand, with carefully designed entry and exit criteria overseen by a panel of endocrinologists.

Patients Applications for 201 patients were assessed and 191 approved for funded treatment over the initial 3 years since inception. The majority had GH deficiency following treatment of pituitary adenomas or tumours adjacent to the pituitary.

Results After an initial 9-month treatment period using serum IGF-I measurements to adjust GH dosing, all patients reported a significant improvement in quality of life (QoL) score on the QoL-AGHDA® instrument (baseline (95%CI) 19 (18–21), 9 months 6 (5–7·5)), and mean serum IGF-I SD scores rose from -3 to zero. Mean waist circumference decreased significantly by 2·8 ± 0·6 cm. The mean maintenance GH dose after 9 months of treatment was 0·39 mg/day. After 3 years, 17% of patients had stopped treatment, and all of the remaining patients maintained the improvements seen at 9 months of treatment.

Conclusion Carefully designed access to nationally funded GH replacement in GH-deficient adults was associated with a significant improvement in quality of life over a 3-year period with mean daily GH doses lower than in the majority of previously reported studies.

Introduction

Growth hormone deficiency in children has been identified as a treatable cause of short stature for many years. GH deficiency is also associated with a range of clinical problems in adults including adverse effects on body composition, impaired muscle and cardiac function, reduced quality of life secondary to changes in mood, motivation and processing of information, abnormal lipid levels, increased insulin resistance and abnormal endothelial function.[1–3] Growth hormone treatment has been shown to improve these abnormalities and enhance quality of life.[4–8] Several medium-term follow-up programmes have been developed to assess patients' response to treatment, such as the KIMS[9] and HypoCSS[10] databases, and a number of reviews have assessed the clinical response to growth hormone therapy based on randomized or open label trials of replacement therapy in deficient adults.[11,12] Cost–utility analyses of treatment have been performed, particularly in the United Kingdom,[13] leading to funding of growth hormone treatment in the UK according to strict guidelines (see NICE Technical Appraisal 64 – Growth hormone deficiency (adults), August 2003). Growth hormone treatment for symptomatic GH-deficient adults is carried out in a number of countries often funded through medical insurance programs.

The Pharmaceutical Funding Agency (PHARMAC) is the New Zealand Government crown entity that manages funding of pharmaceuticals for treatment of individuals within New Zealand (population 4·5 million). After submissions from the NZ Society of Endocrinology (NZSE), PHARMAC approved funding for the treatment of GH-deficient adults that commenced in April 2010. This report details early experience over the first 3 years of funding.

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