Hyperphagia is a frequent symptom in patients with Prader-Willi syndrome (PWS) and results in marked obesity with the risk of metabolic and cardiovascular complications. Aim: To investigate whether early diagnosis of PWS and strict dietary intervention prevents excessive weight gain in patients with PWS. Patients and Methods: A strict fat reduced and modified carbohydrate diet consisting of 10 kcal/ cm height was provided to nine patients (seven female, two male) diagnosed early with PWS (group A). Patients were prospectively followed at our center with follow-up visits every three months. Eight patients with late diagnosis of PWS served as controls (group B). Body mass index (BMI) SDS and height SDS were compared between these two groups over a ten-year period. Results: At the age of two years height SDS and BMI SDS were significantly lower in group A (-2.9 vs -1.2, ρ <0.05, and BMI SDS -0.1 vs +1.8, ρ <0.05). After ten years BMI SDS increased significantly to +1.2 SDS in group A, but was still significantly lower than in group Β (BMI SDS +2.4), ρ <0.005. Patients without restrictive diet were significantly taller than patients on the diet (height SDS group A -2.8 vs group Β -1.3, ρ <0.05). Conclusion: Early dietary treatment starting at the second year of life and continued until the age of ten years is effective in avoiding excessive weight gain in patients with PWS, but results in shorter stature. Therefore growth hormone may be a useful additional treatment in these patients. KEY WORDSPrader-Willi syndrome, hyperphagia, food craving behavior, low fat and modified carbohydrate diet
Background: Linear growth is the best clinical parameter for monitoring metabolic control in classical congenital adrenal hyperplasia (CAH). Objective: To analyze growth patterns in children with CAH diagnosed by newborn screening and treated with relatively low doses of hydrocortisone during the first year of life. Patients and Methods: 51 patients (27 females) were diagnosed with classical CAH by newborn screening. All patients were treated with relatively low doses of hydrocortisone (9–15 mg/m2 body surface area). 47 patients were additionally treated with fludrocortisone. Results: At birth, height SDS (H-SDS) was 1.1 ± 1 in girls and 0.9 ± 1.5 in boys. After 3 months, H-SDS decreased to 0.4 ± 0.9 in girls and to 0.1 ± 1.3 in boys. Over the 3-year period, H-SDS further decreased to –0.4 ± 1.8 in girls and to –0.8 ± 1 in boys and approached the genetic height potential (target H-SDS of girls –0.5 ± 0.3 and target H-SDS of boys –0.9 ± 0.7). During the first 9 months of age, growth velocity was slightly decreased in girls (18.2 ± 1.9 cm) and boys (17.3 ± 1.6 cm) when compared to a healthy reference population (girls 19.0 ± 3.9 cm and boys 18.7 ± 4.7 cm). At the age of 3 years, bone age was appropriate for chronological age in both girls (2.7 ± 0.5 years) and boys (2.9 ± 0.5 years). Conclusion: Birth length is above average in children with classical CAH, which might be the result of untreated hyperandrogenism in utero. With relatively low doses of hydrocortisone treatment, growth velocity decreases slightly during the first 9 months and H-SDS then approaches the genetic height potential.
Twenty-seven prepubertal boys and 9 prepubertal girls with constitutionally delayed growth were treated with the anabolic steroid oxandrolone for 12 months and followed until they reached final height. Sixteen boys were treated with a mean dose of 0.12 mg/kg.day [low dose (LD)] and 11 boys with a mean dose of 0.22 mg/kg.day [high dose (HD)]. The girls were treated with a mean dose of 0.1 mg/kg.day. Thirteen boys and 9 girls served as controls. On oxandrolone the mean height velocity increased from 4.0 to 8.6 (boys, LD), from 4.3 to 8.9 (boys, HD), and from 4.3 to 8.3 cm/yr (girls). The immediate posttreatment height velocity was significantly higher than the pretreatment height velocity (P less than 0.05), regardless of whether the patients had entered puberty. On oxandrolone the mean ratios of change in bone age/change in chronological age were 2.0 (boys, LD), 2.3 (boys, HD), and 2.0 yr/yr (girls) and continued to be accelerated during the 6 months after treatment. Height predictions at the onset of treatment and after 6 months off treatment were calculated by three different methods: Bayley-Pinneau (BP), Roche-Wainer-Thissen (RWT), and Tanner Mark II (T II). In the boys (LD) mean height predictions increased significantly by the methods of BP (3.3 cm) and RWT (2.9 cm), but not by the method of T II (0.6 cm). In the boys (HD) no significant change in height predictions was noted. In the girls mean height predictions remained unchanged by BP and RWT, but decreased significantly by T II (-2.5 cm). The difference between final height and initial height prediction was taken as a measure of the influence of the treatment on adult height. In all three treatment groups the difference between final height and initial height prediction, calculated with all three methods, did not differ from the control group. We conclude that oxandrolone treatment for 1 yr has no effect on adult height. In spite of this, the use of an anabolic steroid such as oxandrolone may still have value, as an increase in height velocity and an earlier onset of puberty may benefit short children suffering from psychological problems due to delay of growth and development.
Close adherence to the recommended treatment regimen is important for the success of recombinant human growth hormone therapy, although nonadherence can be common. Ease of use and safety during use/storage are among several important factors in the design of a growth hormone injection device intended for long-term use. This study was performed to validate the usability and assess the ease of use of a new pen device (SurePal™) that has been developed to support daily administration of the recombinant human growth hormone product, Omnitrope® (somatropin). The primary objectives of the study were to assess if study participants, representing intended users of the pen in clinical practice, were able to perform an injection procedure into an injection pad effectively and safely and disassemble the pen without receiving a needlestick injury. A total of 106 participants (61 adults and 45 children/adolescents) were enrolled at two study centers (one in the US, one in Germany). Results for both primary usability tasks met the predefined acceptance criteria, with >85% of participants successfully performing each task. All of the other tasks/handling steps assessed were also successfully performed by most participants, with high success rates reflected in the high proportion of participants who classified each task as “very easy” or “easy”. After a second use of the device, 87%–97% of participants rated it as “very easy” or “easy” to use. In summary, the new pen device is safe and easy to use for both adults and children, and will help to support effective, long-term daily administration of the recombinant human growth hormone product, Omnitrope®.
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