Eight years of growth hormone treatment in children with Prader-Willi syndrome: maintaining the positive effects.

BACKGROUND: The most important reason for treating children with Prader-Willi syndrome (PWS) with GH is to optimize their body composition. OBJECTIVES: The aim of this ongoing study was to determine whether long-term GH treatment can counteract the clinical course of increasing obesity in PWS by maintaining the improved body composition brought during early treatment. SETTING: This was a multicenter prospective cohort study. METHODS: We have been following 60 prepubertal children for 8 years of continuous GH treatment (1 mg/m(2)/d ≈ 0.035 mg/kg/d) and used the same dual-energy x-ray absorptiometry machine for annual measurements of lean body mass and percent fat. RESULTS: After a significant increase during the first year of GH treatment (P < .0001), lean body mass remained stable for 7 years at a level above baseline (P < .0001). After a significant decrease in the first year, percent fat SD score (SDS) and body mass index SDS remained stable at a level not significantly higher than at baseline (P = .06, P = .14, resp.). However, body mass index SDSPWS was significantly lower after 8 years of GH treatment than at baseline (P < .0001). After 8 years of treatment, height SDS and head circumference SDS had completely normalized. IGF-1 SDS increased to +2.36 SDS during the first year of treatment (P < .0001) and remained stable since then. GH treatment did not adversely affect glucose homeostasis, serum lipids, blood pressure, and bone maturation. CONCLUSION: This 8-year study demonstrates that GH treatment is a potent force for counteracting the clinical course of obesity in children with PWS.

[Routine laboratory tests unnecessary for children referred for recurrent wheezing and/or asthma]. / Routinematig laboratoriumonderzoek bij kinderen, verwezen wegens recidiverend piepen en/of astma, niet zinvol.

OBJECTIVE: To investigate the usefulness of laboratory testing and thorax radiography in children, referred to the paediatrician for evaluation of recurrent wheezing. DESIGN: Retrospective. METHODS: In this study, 158 children referred for recurrent wheezing to a specialized child outpatient clinic of the Medisch Centrum Leeuwarden, the Netherlands, in the period 1 January 1994-31 December 1996, were evaluated according to a routine protocol including haemoglobin, ESR, leucocytes, immunoglobulins, sweat chloride levels and allergy testing and chest roentgenograms. It was determined whether these investigations had yielded abnormal results and whether these test results aided in confirming/rejecting the diagnosis of asthma or were helpful in clinical management. RESULTS: In 144 of the 158 (91%) children the diagnosis 'asthma' or 'recurrent wheezing' was made. Although numerous test results were abnormal they were not helpful in establishing the diagnosis. In only one child an abnormal chest radiograph was helpful (the radiograph showed infiltrative abnormalities). Tests for aero-allergy were rarely positive in children younger than 2 years; in children older than 6 years aero-allergy was found frequently, notably to dust mite (41/144). CONCLUSION: The results of this study suggest that--except for allergy testing--routine laboratory testing and chest roentgenograms are not indicated in children referred for evaluation of wheezing disorders. Aero-allergy testing may help to decide on preventive measures.

[Acute renal insufficiency caused by bilateral ureteral obstruction after appendectomy in a 6-year old boy]. / Acute nierinsufficiëntie door bilaterale ureterobstructie na appendectomie bij een 6-jarige jongen.

A 6-year-old boy developed macroscopic haematuria on the 4th day after appendectomy for acute appendicitis, at which the appendix was found to be perforated. During the next few days the urine secretion decreased and malaise, pain in the lower abdomen, nausea and vomiting occurred. On a management of ample fluid administration, the urine secretion recovered and the symptoms subsided in a few days. In the early postoperative stage after appendectomy in children the possibility should be kept in mind of the development of acute renal insufficiency due to bilateral ureteral obstruction as a result of oedema of the posterior bladder wall, even if by means of ultrasonography only mild to moderate abnormalities are noted. Awaiting decompression by means of the introduction of bilateral ureteric stents, in order to prevent irreversible renal damage, supportive therapy with fluid administration depending on the diuresis seems indicated.

[Progressive partial lipodystrophy; an external problem with internal anomalies]. / Progressieve partiële lipodystrofie; een uiterlijk probleem met inwendige afwijkingen.

Progressive partial lipodystrophy (PPL) was diagnosed in two girls aged 6 and 8 years. PPL is characterized by loss of subcutaneous fat, starting in the face and progressing to trunk and arms. Diagnosis is based upon the cachectic appearance and the normal growth parameters. It is a rare disease of unknown aetiology, usually beginning in childhood and more frequent in females. An association with diabetes mellitus, hypertriglyceridaemia and glomerulonephritis has been described. Follow-up should be focused on these and on psychological effects. No causal therapy is available. The facial appearance can be restored by injection of liquid silicones. Life expectancy does not appear to be affected.