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Abstract Aim To investigate the maturation of the peripheral nervous system by analyzing the cross‐sectional area of the sciatic nerve during the first 2 years of life. Methods The sciatic nerve was examined by high‐resolution ultrasound imaging in 52 children aged 0 days to 10 years, 45 of whom were younger than 2 years. The correlation between the cross‐sectional area of the nerve and the age was statistically tested. A logarithmic regression analysis was performed to develop a logarithmic growth model of the cross‐sectional area. Results There is a highly significant correlation between the age and the cross‐sectional area of the sciatic nerve. The growth rate can well be described by a logarithmic model. Interpretation Based on the literature on the maturation of the median nerve and nerve roots and the findings of the present study, we conclude that both the proximal and the distal parts of the nerves of the peripheral nervous system increase simultaneously. What this paper adds Normative values for the size of the sciatic nerve in children.

Abstract Aim To analyze the increase in diameter of the nerve roots C5 and C6 in early childhood. Methods The nerve roots of 56 children aged 0 days to 10 years (47 younger than 2 years) were examined by high‐resolution ultrasound imaging. The correlation of diameter and age was statistically tested and a logarithmic regression analysis was performed to develop a logarithmic growth model. Results The increase in nerve root diameter is greatest during the first 2 years of life and then the growth rate decreases steadily. The relationship between age and diameter follows a logarithmic curve (p

Abstract Background Natural ghrelin, a peptide growth hormone secretagogue, has a therapeutic potential in cachexia. We designed a dose‐finding trial of subcutaneous natural ghrelin to improve nutritional intake (NI) in advanced cancer patients. Methods Advanced cancer patients with cachexia management (symptom management, physiotherapy, nutritional, and psychosocial support) started with ghrelin at 32 μg/kg body weight, followed by 50% dose increases. Patients self‐injected ghrelin twice daily for 4 days followed by a wash‐out period. After reaching the primary endpoint, maximal NI (minimal dose for maximal NI), a maintenance period followed during which patients injected 10 doses of ghrelin per week. Safety parameters, NI, and cachexia outcomes (symptoms, narratives, muscle mass, and strength) were measured over 6 weeks. Results Ten patients with metastatic solid tumours were included, and six (100% male, mean age 61.8 ± 8.5 SD) received ghrelin. Minimal dose for maximal NI was reached in four patients. Three patients reached the end‐of study visit. Ghrelin was well tolerated with variable results on appetite and eating‐related symptoms but a positive effect in the narratives. Mean Functional Assessment of Appetite & Cachexia Therapy score was 6.8 points lower at final measurement compared with baseline, t(5) = 5.98, P

Abstract Background Early diagnosis is of substantial benefit for patients with Pompe disease. Yet underdiagnosing and substantial diagnostic delay are still frequent and the determinants of this are unknown. This study is the first to systematically investigate the diagnostic odyssey in Pompe disease from patients', parents', and physicians' perspectives. Methods Patients with infantile or late onset Pompe disease, their parents as well as their metabolic experts were invited to fill in respective surveys. The survey addressed perceived disease symptoms at onset and during the course of the disease, specialties of involved physicians, activities of patient‐initiated search for diagnosis and the perceived impact of time to diagnosis on outcome. Results of experts' and patients'/parents' surveys were compared and expressed by descriptive statistics. Results and Discussion We collected data on 15 males and 17 females including 9 infantile and 23 late onset Pompe patients. All received the correct diagnosis at a metabolic or musculoskeletal expert center. Patients with direct referral to the expert center had the lowest diagnostic delay, while patients who were seen by several physicians, received the correct diagnosis after 44%‐200% longer delay. The proportion of direct referral varied strongly between pediatricians (57%) and other disciplines (18%‐36%). Conclusion Our study highlights a substantially larger diagnostic delay in Pompe patients that are not directly referred to expert centers for diagnostic work. Our findings may be used to develop more successful strategies for early diagnosis. Synopsis Diagnostic delay in Pompe disease is substantial particularly in patients that are not directly referred to expert centers for diagnostic workup, so facilitating direct referral may be a new strategy for early diagnosis.