[Pseudomonas aeruginosa and cystic fibrosis: non-antibiotic strategy]. / Pseudomonas aeruginosa et mucoviscidose: stratégie non antibiotique.

The Pseudomonas aeruginosa bronchopulmonary infection is, when chronical, a poor prognosis factor for cystic fibrosis children. The higher life expectancy is partly linked to the progresses of the antipseudomonal antibiotherapy: treatment modulated to the stage of infection; possible use of nebulized and oral (ciprofloxacin) antibiotics. But the antipseudomonal strategy does not limit to the antibiotherapy. The preventive approach includes: preservation of a good nutritional status; daily chest physiotherapy with combined use of aerosolized recombinant human DNase for more than 5 years old children with significative bronchorrea; early anti-infammatory treatment with inhaled corticosteroids, despite a poor efficacy proof level; hygiene measures to reduce the environmental and domestic reservoirs and to avoid nosocomial infections. The development of vaccines for the prevention of P. aeruginosa infection is a promising way but stays in the field of clinical research. In case of chronical infection, long-term macrolids reduce the virulence factors expression of the bacteria, leading to antibiofilm properties in spite of subinhibitory concentrations.

[Urolithiasis in cystic fibrosis]. / Lithiase urinaire et mucoviscidose.

The prevalence of urolithiasis is higher in patients with cystic fibrosis than in the normal population. We report on a case of a 12-year-old child with cystic fibrosis who presented flank pain which revealed a calcium oxalate urolithiasis. Evolution was satisfactory with pharmacological and dietary treatment. The principal mechanisms of lithogenesis in patients with cystic fibrosis are described and preventive treatment is discussed.

[Effect of rhDNase on the respiratory function and nutritional status of children and adolescents with mucoviscidosis]. / Effects de la rhDNase sur la fonction respiratoire et le statut nutritionnel de l'enfant et de l'adolescent atteints de mucoviscidose.

BACKGROUND: In 1994 we started recombinant human deoxyribonuclease (rhDNase) in every cystic fibrosis (CF) patient whatever his (her) clinical condition, provided they were aged more than 5 years and forced vital capacity (FVC) was > or = 40%. POPULATION AND METHODS: We reviewed retrospectively the effects of rhDNase in 69 CF children and adolescents during a 2-year follow-up. Patients (35 boys, 34 girls) received 2.5 mg of rhDNase once daily from a mean age of 8.5 years (range 5-16.4). Baseline spirometric values (% predicted) and nutritional status were as followed: FVC = 84.8 +/- 21.7; forced expiratory volume in 1 second (FEV1) = 80.8 +/- 22.2; peak flow = 89.7 +/- 34.2, forced expiratory fraction 25-75% (FEF 25-75) = 71.8 +/- 32.8; Z score weight/height = -0.41 +/- 1.14; Z score weight/age = -0.48 +/- 1.25, body mass index = 15.4 +/- 1.8; caloric intake = 107 +/- 25% of recommended dietary allowances (RDA). Patients had a Shwachman-Kulczycki's score of 87 +/- 9. Spirometric and nutritional data were analysed after 1, 3, 6, 12, 18 and 24 months of treatment and compared to baseline values (changes evaluated as percent change from mean baseline for spirometric data). Shwachman-Kulczycki's score was calculated after 24 months of rhDNase. RESULTS: An improvement of FVC (+10.7%, P < 0.001) and FEV1 (+12%, P < 0.01) was noted after one month of treatment and was maintained throughout the following 2 years around 8.7% (6.4-11.4) for FVC and 8.2% (7.3-9.1) for FEV1, P < or = 0.01. This was particularly observed in children aged 5 to 10 years, in boys and in patients with a baseline FVC under 70% predicted. There was no significant change in FEF 25-75. We observed an improvement of daily caloric intake from the third month (P < 0.05) and of body mass index from the sixth month (P = 0.02). This was particularly noted in girls. Z score weight/age was improved only during the first 3 months of treatment while Z score weight/height increased only after a 2 year follow-up. There was no significant change in Shwachman-Kulczycki's score after 24 months of rhDNase. CONCLUSION: rhDNase in CF children in effective on lung function as well as on nutritional status and the response to this treatment can be evaluated after the first 3 months.

[Liver transplantation in an adolescent with cystic fibrosis]. / Transplantation hépatique chez une adolescente atteinte de mucoviscidose.

BACKGROUND: Orthotopic liver transplantation (OLT) is an effective treatment for patients with cystic fibrosis end stage liver disease, especially those with only mild pulmonary involvement. Long-term follow-up in such transplanted patients is still lacking. CASE REPORT: A 15-year-old girl with cystic fibrosis received an OLT because of severe decompensated cirrhosis. She had been colonized by Pseudomonas aeruginosa for 3 years and had pancreatic insufficiency; she also had mild glucose intolerance. Postoperatively she developed diabetes mellitus requiring insulin therapy for 9 months. Oral cyclosporin was poorly absorbed so that she was given a new emulsion of cyclosporin (Neoral) that was better absorbed. A rapid pubertal catch-up was obtained but the patient remained colonized by Pseudomonas aeruginosa. CONCLUSION: This 3-year postoperative follow-up confirms that OLT can represent a good alternative in those patients with severe liver disease and mild pulmonary involvement.

Tolerance, pharmacokinetics and efficacy of once daily amikacin for treatment of Pseudomonas aeruginosa pulmonary exacerbations in cystic fibrosis patients.

UNLABELLED: Twenty cystic fibrosis patients aged 1.8-22 years (mean +/- SD: 9.6 +/- 4.8 years) with Pseudomonas aeruginosa pulmonary exacerbations were treated with amikacin (AM) (35 mg/kg/day in one daily 30 min infusion) associated with either ceftazidime (200 mg/kg/day in 3 i.v. injections) (n = 19) or imipenem (n = 1) at the same dose. Glomerular and tubular functions (creatinine clearance, 24-h proteinuria, beta 2 microglobulinuria, lysozymuria) and audiometry remained within normal ranges from day 0 to day 14. A peak concentration of AM of 83 +/- 19 mg/l and a trough concentration of 0.8 +/- 0.5 mg/l were observed in blood while AM levels in sputum were above the minimal inhibitory concentration 50 from 30 min to 16 h. No serum accumulation of AM was observed during the treatment. From day 0 to day 14, the following changes were observed: weight/height ratio: 96%-100% (P < 0.001); daily energy intake: 111%-128% of RDA (P < 0.001); prealbumin: 195-290 mg/l (P < 0.001); forced vital capacity (FVC): 66%-81% (P < 0.01); forced expiratory volume in 1 s: 60%-75% (P < 0.01); forced expiratory flow between 25% and 75% of FVC: 42%-56% (P < 0.01); nocturnal SaO2 also improved significantly; cardiac rate decreased from 89 +/- 18/min to 76 +/- 16/min (P < 0.001); respiratory rate decreased from 31 +/- 15/min to 26 +/- 10/min (P < 0.05); inflammatory parameters (white blood cells, polymorphonuclear cells, erythrocyte sedimentation rate) also improved. CONCLUSION: Once daily amikacin administration associated with ceftazidime is well tolerated for the treatment of Pseudomonas aeruginosa pulmonary exacerbations in cystic fibrosis patients. Serum peak levels and diffusion in sputum are higher than with a conventional schedule.

[Frequency of gastroesophageal reflux in infants and in young children with cystic fibrosis]. / Fréquence du reflux gastrooesophagien chez le nourrisson et le jeune enfant atteints de mucoviscidose.

BACKGROUND: Gastro-esophageal reflux (GER) is common in chronic bronchopulmonary diseases, and may aggravate them. The aim of this study was to study frequency and characteristics of GER in infants and toddlers with cystic fibrosis (CF). POPULATION AND METHODS: Twenty-five CF patients, 45 to 954 days-old (mean 219 days) without clinical evidence of bronchopulmonary infection, were studied by prolonged pHmetry (19.45 +/- 1.89 hours). Their pulmonary state was evaluated by the number of previous courses of antibiotic treatment and analysis of the X-rays. RESULTS: Nineteen children had GER (76%); four of them had no clinical sign of GER. All 19 patients had mild or moderate signs of pulmonary impairment. The reflux index (percentage of time with pH < 4) was 12.82%. The GER episodes were more frequent during walking time (p < 0.001), but longer during sleeping time (p < 0.001). CONCLUSIONS: GER is frequent in CF patients, even the youngest. It seems primary, and not related to pulmonary impairment. Its frequency and its potential severity lead to suggesting a pHmetry to every new CF patient.

[Visceral candidiasis in 2 children treated for acute leukemia]. / Candidose viscérale chez deux enfants traités pour leucémie aiguë.

The authors report two cases of splenic and hepatosplenic candidiasis occurring during a protracted neutropenia induced by chemotherapy for acute leukemia (lymphoblastic and myeloblastic respectively). Fungal infection was revealed by persistent or recurrent fever after correction of neutropenia. Diagnosis was suggested by findings at abdominal ultrasonography. It was confirmed by histological analysis which, in the first case, required open liver biopsy. In both cases, lack of improvement of splenic lesions despite treatment with Amphotericin B followed by fluconazole led to splenectomy. Both patients received postoperative anti fungal therapy with Ampho B and at one year's follow up, the patient who had the hepatosplenic candidiasis seems to have recovered.

Longitudinal analysis of infantile growth in children with celiac disease.

The aim of the present study was to describe linear growth of infants with celiac disease, using the "ICP-growth model". Supine length during the first three years of life was studied longitudinally in 63 infants with diagnosed celiac disease. "Undisturbed" linear growth was seen during the first six postnatal months followed by reduced growth during the second half of the first year. After 1.5 years of age a pattern of catch-up growth was observed, leading to an average attained length at 3 years of age similar to that of the controls. According to the "ICP-growth model", normal linear growth can mathematically be represented during the first 3 years of life by an Infancy component with the addition of a Childhood component, the latter acting from the second half of the first postnatal year. The onset of the Childhood component (assumed to represent the age at which growth hormone begins to influence linear growth significantly) was delayed by about an average of 3 months, which is in agreement with the observed reduction in gain during the second half of the first year of life. Children suffering from celiac disease and with "late" onset of the Childhood component were shorter at 1, 2 and 3 years of age than those with "normal" onset. The results of this investigation show that ICP-based growth charts are helpful in detecting and monitoring growth for children with celiac disease, and indicate a possible mechanism whereby malabsorption (and perhaps secondary malnutrition) leads to reduced growth velocity.