Therapeutic efficacy of recombinant human growth hormone in children with different etiologies of dwarfism from a pharmacoeconomic point of view.

Treatment outcomes for different causes of childhood dwarfism vary widely, and there are no studies on the economic burden of treatment in relation to outcomes. This paper compared the efficacy and healthcare costs per unit height of recombinant human growth hormone (rhGH) for the treatment of growth hormone deficiency (GHD) and idiopathic short stature (ISS) with a view to providing a more cost-effective treatment option for children. We retrospectively analyzed 117 cases (66 cases of GHD and 51 cases of ISS) of short-stature children who first visited Weifang People's Hospital between 2019.1 and 2022.1 and were treated with rhGH for 1 to 3 years to track the treatment effect and statistically analyzed by using paired t tests, non-parametric tests, and chi-square tests, to evaluate the efficacy of rhGH treatment for GHD and ISS children and the medicinal cost. The annual growth velocity (GV) of children with GHD and ISS increased the fastest during 3 to 6 months after treatment and then gradually slowed down. The GV of the GHD group was higher than that of the ISS group from 0 to 36 months after treatment (P < .05 at 3, 6, 9, and 12 months); the height standard deviation scores (HtSDS) of the children in the GHD and ISS groups increased gradually with the increase of the treatment time, and the changes in the height standard deviation scores (ΔHtSDS) of the GHD group were more significant than those of the ISS group (P < .05 at 3, 6, 9, and 12 months). (2) The medical costs in the pubertal group for a 1-cm increase in height were higher than those of children in the pre-pubertal group at the same stage (3 to 24 months P < .05). The longer the treatment time within the same group, the higher the medical cost of increasing 1cm height. RhGH is effective in treating children with dwarfism to promote height growth, and the effect on children with GHD is better than that of children with ISS; the earlier the treatment time, the lower the medical cost and the higher the comprehensive benefit.

Review of economic analysis of available interventions on idiopathic short stature: A protocol for systematic review and meta-analysis.

BACKGROUND: Idiopathic short stature (ISS) causes a high economic burden worldwide. As part of a research project that synthesizes economic evidence for Korean medicine treatment of ISS, we describe the methods that will be used for the comprehensive review of articles that analyze health-related economic evaluation for available interventions for ISS using a systematic review methodology. METHODS: Eight electronic English, Korean, and Chinese databases will be searched from their inception until December 2020 to identify studies on the economic evaluation of available interventions on ISS, without language, study design, or publication status restrictions. From the included studies, the effectiveness, utility, and cost data will be collected as the outcome measures by two researchers independently. Descriptive analysis of individual studies will be conducted. If it is judged that the interventions and outcomes of the included studies are sufficiently homogeneous, we will attempt a quantitative synthesis through meta-analysis using Review Manager version 5.4 software (Cochrane, London, UK). RESULTS: This study will summarize the evidence regarding the economic evaluation of available interventions for ISS. CONCLUSIONS: The findings of this review will help clinicians and patients in evidence-based decision-making in clinical settings and help policy makers develop effective policies and distribute resources based on the available evidence.

Cost-Conscious Growth-Promoting Treatment: When Discretion Is the Better Part of Value.

Assessing cost-effectiveness of human growth hormone (hGH) treatment to augment height is complicated by uncertainty about how best to measure its therapeutic effect. Cost-conscious growth promotion practice, however, is possible and likely an emerging practical requisite as health care payers increasingly deny the medical necessity of and restrict support for short stature treatment. The increase in denials is not surprising given the expansion and continued high cost of hGH treatment, debate about the value of such treatment, and universal need to restrain burgeoning health care costs. Renunciation of sweeping payer rejection of hGH-for-height treatment is strengthened by cost-conscious practices that (1) recommend no treatment for most short children and restrict treatment to severe, likely disabling short stature; (2) initiate hGH treatment only after evidence-based informed assent; (3) utilize alternative less costly and less invasive options when possible; (4) minimize hGH treatment duration and dosage; and (5) resist enhancement of normal adult stature. A new era of cost-conscious hGH prescribing that prompts thoughtful restraint in hGH use could help preserve hGH approval for children most in need of treatment.

Health-related quality of life and socioeconomic situation among diastrophic dysplasia patients in Finland.

OBJECTIVE: The purpose of the present study was to gain a comprehensive view of the quality of life and socio-economic conditions in a more representative sample of patients with diastrophic dysplasia than previously presented. METHODS: The study sample comprised 115 patients with diastrophic dysplasia, aged over 18 years. The patients were contacted, and 68 patients (59%) agreed to participate in the study. They answered a structured questionnaire, which included the items of RAND-36 and Finn-Health Assessment Questionnaire (Finn-HAQ) questionnaires. The Finn-HAQ items were linked to the categories of the International Classification of Functioning, Disability and Health (ICF). Population controls for matching the participating patients for age and sex were identified in the Finnish population registry. Demographic and social factors (educational status, employment status and household income) were collected in separated questions. RESULTS: RAND-36 showed significantly lower physical functioning in the group of diastrophic dysplasia patients than in the control group. Also, the differences in scores for energy and social functioning were significant. In the mental component scales, no significant difference was found between the groups. When compared with the controls, we found significantly lower levels in all 3 ICF components of functioning in the group of patients when Finn-MDHAQ items linked to ICF were used. Almost 75% of patients with diastrophic dysplasia belonged to the group of people with minor/low income. Some or clear worsening of economic situation due to diastrophic dysplasia was reported by 25 (58%) female and 17 (68%) male patients. CONCLUSION: In their daily living, patients with diastrophic dysplasia have marked physical difficulties, which affect their quality of life, participation in society and their financial situation. It seems that the mental situation is not greatly affected, but a more detailed study is needed to evaluate and illuminate the psychological consequences of this severe skeletal dysplasia. Overall, the pieces of information in the present study are of high importance when designing and reorganizing rehabilitation and in supportive therapy and treatment of patients with diastrophic dysplasia.

[GH treatment in patients with idiopathic short stature]. / Uso de GH em pacientes com baixa estatura idiopática.

Growth hormone has been used in the treatment of patients with idiopathic short stature. Clinical and laboratory criteria are discussed, taking into consideration the indication of GH and the evaluation of its efficacy and individual responsiveness. Anthropometric, psychosocial, ethical, and also cost/benefit aspects must be considered before GH prescription in idiopathic short stature patients.

Uso de GH em pacientes com baixa estatura idiopática: [revisão] / GH treatment in patients with idiopathic short stature: [review]

Pacientes com baixa estatura idiopática podem ser tratados com GH. Os critérios clínicos e laboratoriais utilizados na decisão do uso do GH, bem como no reconhecimento da responsividade dos indivíduos ao tratamento, são discutidos. Não apenas os resultados antropométricos, mas também os aspectos éticos e psicossociais devem ser considerados na avaliação dos custos/benefícios envolvidos no tratamento com GH em pacientes com baixa estatura idiopática.

Growth hormone has been used in the treatment of patients with idiopathic short stature. Clinical and laboratorial criteria are discussed, taking into consideration the indication of GH and the evaluation of its efficacy and individual responsiveness. Anthropometric, psychosocial, ethical, and also cost/benefit aspects must be considered before GH prescription in idiopathic short stature patients.

Does economic inequality affect child malnutrition? The case of Ecuador.

Economic inequality has been hypothesized to be a determinant of population health, independent of poverty and household income. We examined the association between economic inequality and child malnutrition in Ecuador. Economic inequality was measured by the Gini coefficient of household per capita consumption, estimated from the 1990 Census. Childhood stunting, assessed from height-for-age z scores, was obtained from the 1998 Living Standards Measurement Survey (LSMS). We controlled for a range of individual and household covariates, including per capita food consumption, education, housing, ethnicity, fertility, access to health services, diarrhea morbidity, child care, mother's age and diet composition. Stunting still affects 26% of children under five in Ecuador, with higher prevalence in the rural Highlands and among indigenous peoples. Maternal education, basic housing conditions, access to health services, ethnicity, fertility, maternal age and diet composition were independently associated with stunting. However, after controlling for relevant covariates, economic inequality at the provincial scale had a statistically significant deleterious effect on stunting. At municipal or local levels, inequality was not associated with stunting.