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Objective: To determine current expert opinion and recommendations regarding the controversial issue of the use of growth hormone (GH) to treat short children who do not have classical GH deficiency (non-GHD children).Study Design: Analysis of a national survey mailed to 534 US physician experts on the management of short stature (pediatric endocrinologists) with a response rate of 81.3%.Main Outcome Measure: The experts' GH treatment recommendations.Results: The physicians reported that approximately 58% of their current patients undergoing GH therapy have classical GH deficiency, while 42% have other conditions. The proportion of physicians who recommended GH treatment of short non-GHD children ranged from 1% to 74% over all case scenarios presented. The likelihood of GH being recommended depended on the physiological growth characteristics of the child (ie, the child's height, growth rate, and predicted adult height), contingency factors (ie, strong family wishes or a reduction in GH cost), and physician beliefs (ie, the impact of short stature on well-being, the effectiveness of GH therapy). Each of these factors exerted highly significant, independent, and additive effects on decisions to recommend GH.Conclusion: Our results indicate that many pediatric endocrinologists consider GH treatment appropriate for selected short non-GHD children, going beyond current Food and Drug Administration-approved indications for GH. Decisions to recommend GH for a non-GHD child rest on a combination of medical, social, and perceptual factors; variations in treatment patterns stem from variations in these influences. Future GH use will likely be determined not only by the results of controlled trials, but also by family preferences, producer pricing, and physician perceptions of the value of height and GH therapy. [ABSTRACT FROM AUTHOR]

Context: There is concern in both the medical community and the general public about mechanisms of medical decision making and the interplay of physician and insurer decisions in determining access to care.Objective: To examine the medical process influencing access to growth hormone (GH) therapy for childhood short stature by comparing coverage policies of US insurers with the treatment recommendations of US physicians.Design and Participants: Independent national representative surveys were mailed to insurers (private, Blue Cross/Blue Shield, health maintenance organizations, programs for Children with Special Health Care Needs, and Medicaid programs, n=113), primary care physicians (n=1504), and pediatric endocrinologists (n=534) with response rates of 75%, 60%, and 81%, respectively. Each survey included identical case scenarios. Primary care physicians were asked decisions about referrals to pediatric endocrinologists. Endocrinologists were asked GH treatment recommendations. Insurers were asked coverage decisions for GH therapy.Main Outcome Measures: Insurer coverage decisions for GH in specific case scenarios were compared with the recommendations of primary care physicians and pediatric endocrinologists.Results: Physician recommendations and insurance coverage decisions differed strikingly. For example, while 96% of pediatric endocrinologists recommended GH therapy for children with Turner syndrome, insurer policies covered GH therapy for only 52% of these children. Overall, referral and treatment decisions by physicians resulted in recommendations for GH therapy in 78% of children with GH deficiency, Turner syndrome, or renal failure; of those recommended for treatment, 28% were denied coverage by insurers. Similarly, GH therapy would be recommended by physicians for only 9% of children with idiopathic short stature, but insurers would not cover GH for the vast majority of these children. Furthermore, the data indicated considerable variation among insurers regarding coverage policies for GH (P<.01).Conclusions: Access to GH therapy differs depending on the type of insurance coverage. The deep discord between physician recommendations and insurance coverage decisions, exemplified by these findings, represents a major challenge to mechanisms of health care decision making, access, and costs. [ABSTRACT FROM AUTHOR]

Context: Extremely low-birth-weight (ELBW) children have high rates of chronic conditions during childhood. Information on their trajectory of health during adolescence is needed for health care planning., Objective: To examine changes in the rates of chronic conditions between the ages of 8 and 14 years among ELBW children compared with normal-birth-weight (NBW) controls., Design, Setting, and Participants: Cohort study conducted from 2004 through 2009 of 181 ELBW children (weight < 1 kg) and 115 NBW controls of similar sociodemographic status born from 1992 through 1995 in Cleveland, Ohio., Main Outcome Measures: Rates of chronic conditions overall (measured with the revised Questionnaire for Identifying Children With Chronic Conditions) and rates of asthma and obesity., Results: The overall rates of chronic conditions did not change significantly between the ages of 8 and 14 years among ELBW children (75% at age 8 years vs 74% at age 14 years) or NBW controls (37% at age 8 years vs 47% at age 14 years). In generalized estimating equations logistic regression adjusting for sociodemographic status, sex, and race, ELBW children continued to have a higher rate of chronic conditions than NBW controls at age 14 years (74% vs 47%, respectively, adjusted odds ratio [AOR], 2.8 [95% confidence interval {CI}, 1.7 to 4.6]). Rates of asthma requiring medication did not change between the ages of 8 and 14 years among ELBW children (23% at both ages) but increased among NBW controls from 8% at age 8 years to 17% at age 14 years (P = .002). Differences in rates of asthma between ELBW and NBW children were no longer significant at the age of 14 years (23% vs 17%, respectively; AOR, 1.5 [95% CI, 0.8 to 2.8]). Mean z scores for body mass index increased in ELBW children from 0.06 at age 8 years to 0.38 at age 14 years (P <.001) and rates of obesity increased from 12% at age 8 years to 19% at age 14 years (P = .02). However, the scores and rates did not change among NBW controls such that at the age of 14 years the differences between ELBW and NBW children in mean z scores for body mass index (0.38 vs 0.56, respectively; adjusted mean difference -0.2 [95% CI, -0.5 to 0.1]) or rates of obesity (19% vs 20%, respectively; AOR, 1.1 [95% CI, 0.6 to 2.0]) were not significant., Conclusions: Among ELBW children, rates of overall chronic conditions and asthma did not change between the ages of 8 and 14 years but the rate of obesity increased. Compared with NBW controls, the rates of chronic conditions were higher but there were no significant differences in the rates of asthma or obesity.

Context: Use of growth hormone (GH) therapy to promote growth in children with idiopathic short stature is controversial. A fundamental issue underlying the controversy is uncertainty about the magnitude of effectiveness of GH for this condition., Objective: To determine the effect of GH on short- and long-term growth in idiopathic short stature., Study Design: Systematic review of controlled and uncontrolled studies., Data Sources: MEDLINE (1985-2000), key journals, cross-referencing of bibliographies, abstract booklets, and experts., Study Selection and Data Extraction: We performed a meta-analysis of all studies satisfying the inclusion criteria for idiopathic short stature: initial height below the 10th percentile, normal stimulated GH levels (>10 microg/L), absence of comorbid conditions, no previous GH therapy, treatment with biosynthetic GH, and inclusion of major outcome measures., Primary Outcome Measures: Growth velocity and height SD score (number of SDs from mean height for age and sex) at baseline and after 1 year to evaluate the short-term effect of GH. Adult height was analyzed to evaluate the long-term effect of GH., Data Synthesis: Ten controlled trials (434 patients) and 28 uncontrolled trials (655 patients) met the inclusion criteria. While baseline growth velocities were equivalent at baseline, 1-year growth velocity of the GH-treated group significantly exceeded that of controls by 2.86 cm/y. Similarly, in uncontrolled trials, growth velocity increased after 1 year, and height SD score increased from -2.72 at baseline to -2.19. In controlled studies, the adult height of the GH-treated group significantly exceeded controls by 0.84 SD, and in uncontrolled trials the adult height attained after GH treatment (-1.62 SDs) exceeded that predicted at baseline (-2.18 SDs). These results suggest an average gain in adult height of approximately 4 to 6 cm (range, 2.3-8.7 cm) with GH therapy. Given current treatment costs, this corresponds to more than $35 000 per inch (2.54 cm) gained in adult height in idiopathic short stature., Conclusions: Treatment with GH results in short-term increases in growth for children with idiopathic short stature, and long-term GH can increase adult height. These results are fundamental to decisions about GH use and raise questions about the goals of treatment. Use of GH for idiopathic short stature in clinical practice will depend on its efficacy in promoting growth and the value of this effect to families, physicians, and third-party payers.

Objective: To assess the psychosocial functioning of adults who were evaluated as children for short stature and were not treated with human growth hormone., Design: Inception cohort study., Setting: Hospital-based pediatric endocrinology clinic., Participants: From 1975 to 1980, medical record review indicated that 181 of the children referred to our clinic for concerns about short stature were non-growth hormone deficient. In 1992 and 1993, we were able to recruit 35 of these patients for a follow-up study. Eligible subjects were at least 18 years of age at the time of follow-up., Main Outcome Measures: Standardized self-report questionnaires assessed various domains of psychosocial adjustment. Also, a brief test of intellectual functioning was administered and subjects underwent a semistructured in-person interview to evaluate pragmatic functioning and experiences associated with short stature., Results: Few significant differences between the study sample and standardization samples were found on measures of psychosocial and intellectual functioning. Within-group childhood height during the first evaluation appointment was not significantly associated with most adult measures of psychosocial adjustment. Shorter adult stature was significantly associated with lower educational achievement, lower self-esteem, and greater emotional distress., Conclusions: The absence of significant psychosocial distress or impairment in these subjects brings into question one basis for hormonal treatment for non-growth hormone deficient short stature; that short stature in childhood is likely to lead to psychological dysfunction in adulthood. The results, however, also suggest that shorter stature in adulthood may constitute a psychosocial stressor, increasing vulnerability across several domains.

Pediatricians face clinical and ethical dilemmas about therapy to augment growth in short children who do not meet classic criteria for growth hormone (GH) deficiency. Biologic norms of health are unhelpful because of the uncertain relationship between stature, GH secretion, health, and disease. Instead, we suggest that GH therapy be evaluated from the perspective of cultural norms. We compare GH therapy for short normal children with currently accepted therapies for non--life-threatening pediatric conditions such as well-child care, cosmetic therapy, treatment of psychological problems, and invasive outpatient therapy for chronic conditions. Based on this analysis, we argue that the burdens of therapy, the uncertainty about long-term risks and benefits, the unclear therapeutic end point, and the implications for child health policy place routine GH therapy for children without documented deficiency of GH secretion outside current pediatric ethical norms. Such therapy is properly administered within a comprehensive clinical research protocol.