Observations of nonadherence to recombinant human growth hormone therapy in clinical practice.

BACKGROUND: The effectiveness of all prescribed treatments is contingent on patient adherence. The reported levels of adherence to recombinant human growth hormone (r-hGH) therapy are highly variable, but it has been suggested that nonadherence might be as high as 36% to 49%. OBJECTIVES: This commentary discusses the factors that affect long-term adherence to injection treatment, of which r-hGH therapy is a particular challenge. It also explores potential strategies to improve adherence to injection treatments in clinical practice. METHODS: The opinion of the authors was validated and supported by published literature. A PubMed literature search was conducted in November 2006, identifying English-language articles containing key terms growth hormone, adherence, and compliance. RESULTS: This study found that factors associated with poor adherence to injection treatments include patients' lack of understanding of their disease, patient age, chronicity of the disease, complex treatment regimens, and insufficient information on the implications of nonadherence. Strengthening the patient-physician relationship by providing the patient with a clear understanding of his/her disease and the benefits of adherence, making improvements in injection devices, and eliminating subjective illness concepts, might increase adherence to SC injection treatments, thereby reducing increasing health care costs associated with nonadherence. CONCLUSIONS: Poor adherence to r-hGH therapy has a dual effect, in that it leads to reduced efficacy out-comes and increased health care costs. Implementing strategies to improve adherence with injection treatment might be of particular clinical benefit to patients undergoing r-hGH therapy.

Growth hormone treatment in 35 prepubertal children with achondroplasia: a five-year dose-response trial.

BACKGROUND: Achondroplasia is a skeletal dysplasia with extreme, disproportionate, short stature. AIM: In a 5-y growth hormone (GH) treatment study including 1 y without treatment, we investigated growth and body proportion response in 35 children with achondroplasia. METHODS: Patients were randomized to either 0.1 IU/kg (n = 18) or 0.2 IU/kg (n = 17) per day. GH treatment was interrupted for 12 mo after 2 y of treatment in prepubertal patients to study catch-down growth. Mean height SDS (HSDS) at start was -5.6 and -5.2 for the low- and high-dose groups, respectively, and mean age 7.3 and 6.6 y. RESULTS: Mean growth velocity (baseline 4.5/4.6 cm/y for the groups) increased significantly by 1.9/3.6 cm/y during the first year and by 0.5/1.5 cm/y during the second year. During the third year, a decrease of growth velocity was observed at 1.9/1.3 cm/y below baseline values. HSDS increased significantly by 0.6/0.8 during the first year of treatment and in total by 1.3/1.6 during the 5 y of study. Sitting height SDS improved significantly from -2.1/-1.7 to -0.8/0.2 during the study. Body proportion (sitting height/total height) or arm span did not show any significant change. CONCLUSION: GH treatment of children with achondroplasia improves height during 4 y of therapy without adverse effect on trunk-leg disproportion. The short-term effect is comparable to that reported in Turner and Noonan syndrome and in idiopathic short stature.

Chronic mucocutaneous candidiasis and primary hypothyroidism in two families.

UNLABELLED: We describe the clinical and immunological features of two families with chronic mucocutaneous candidiasis (CMC) and primary hypothyroidism. Family A includes three siblings with both candidiasis and hypothyroidism and four individuals with hypothyroidism only. Family B includes four members with candidiasis, of whom one (a male child) also had hypothyroidism. All individuals affected with CMC had suffered from oral candidiasis and onychomycosis since infancy. Facial seborrhoic dermatitis, general folliculitis and scaling blepharitis were main manifestations. Hypothyroidism became evident during childhood. No thyroid antibodies were present in the affected siblings in family A, while the male in family B with hypothyroidism had antibodies against thyroid peroxidase at diagnosis. Immunological evaluation revealed intra-individual variations in serum immunoglobulin levels, lymphocyte subsets and proliferative responses, but there were no consistent abnormalities. Vaccine responses were normal. AIRE gene region microsatellite markers did not segregate with disease nor were autoantibodies typical for autoimmune polyendocrine syndrome type 1 detected in the families. CONCLUSION: The link between hypothyroidism and chronic mucocutaneous candidiasis remains to be identified.