[Development in conservatively treated scoliosis in patients with myelomeningocele (patients of the years 1964-1977)]. / Evolution des scolioses traitées conservativement chez les patients atteints de myéloméningocèle (patients des années 1964-1977).

The advances in urological and neurosurgical management in myelomeningocoele patients have led to an increased survival rate. The extremely complex spinal deformity presents a major treatment challenge. Clinical and radiological evolution of scoliosis treated conservatively for at least 10 years was studied. Eighty-nine myelomeningocoele patients born between 1964 and 1977 were reviewed. Thirty-one (35%) were noted to have scoliosis (10 congenital type, 21 developmental type). All congenital forms showed rapid progression despite brace treatment, often to curvatures in excess of 100 degrees. Among the developmental type, curves detected prior to 10 years (10 cases before 5 years, 6 cases between 6 and 10 years) were rapidly progressive beyond 70 degrees. Curves detected after 10 years (5 patients) never exceeded 30 degrees. Because of this natural history, conservative treatment should be limited to developmental forms less than 50 degrees. Spinal stabilization is indicated in all curvatures over 50 degrees without awaiting adulthood.

[Psychosocial approach to cystic fibrosis in adults]. / Abord psychosocial de la mucoviscidose de l'adulte.

Progress in therapeutics has transformed care for cystic fibrosis. Physicians now see adults with a chronic disease requiring more global care. To improve the patient's quality of life, it is essential to take into account the social and occupational aspirations as well as their psychological resistance and self-determination. We emphasize the need for perfect synergy between medical and social care givers and on the importance of careful transition from pediatric to adult care, including during the time of major surgical procedures.

Comments on the management of newborn with spina bifida cystica--active treatment or no treatment.

From 1977-1984 46 children with myelomeningocele (MMC) have been admitted immediately following birth. According to Lorber's adverse criteria, 8 received no active treatment (17%) (rejected group). The other 38 were operated on within the first 24 hours (83%) (selected group). The first group had a mean of 3 criteria per patient. The decision not to treat was taken in consultation with the parents, social factors playing an important rôle, too. 5 children died within a few days to 2 months subsequent to an infection. 3 survived and were shunted at 2-4 months at the request of the parents. With the exception of 2, all mothers agreed with the former decision 1-8 years later. Among the 38 patients with active treatment 12 should not have been operated on (26% of all MMC cases), because they had adverse criteria too though less than the rejected group. Among these 12 patients, there are more shunt patients, and their walking capacity is inferior to that of the remaining 26, but there is no difference in respect of their intellectual capacity. Without additional measures Lorber's criteria refer rather to come than to spontaneous mortality. They predict subsequent walking capacity and bladder function correctly, but not the intelligence, and patients with good intellectual capacity may thus be excluded from treatment. To avoid this, at least 3 adverse criteria and/or the three last ones (hydrocephalus, birth injury, major congenital defects) as well as social factors, should be taken into account. Following selection the paediatric surgeon should remain available for the parents' support and reevaluation of the child.

Psychosocial aspects in the treatment of children with myelomeningocele: an assessment after a decade.

The aim of this study was to recognize the possible psychological advantages when children with a severe CNS disorder like myelomeningocele (MMC) are given very early rehabilitation treatment. One hundred and seven newborns with MMC seen between 1971-1992 were prospectively analysed with respect to two different therapeutic approaches. The children born during the period 1971-1980 did not receive very early therapeutic rehabilitation treatment, whereas those born during the period 1981-1992, received this treatment. In the latter group, special attention was paid to support an improvement in the difficult relationship between the parents and the child with MMC as well as between parents and caregivers. The following statistically significant differences between the two treatment programmes were found: (1) all children achieved independent locomotion at 5 years, in the very early intervention group, compared to only 35% (P < 0.001) in the group without this programme. Orthopaedic operations in the first-mentioned group were markedly reduced; (2) urological surgery decreased drastically in the group with very early urodynamic rehabilitation. Thus, there were 0.6 operations per patient in the older group, but only 0.06 operations per patient in the younger one (P < 0.001); (3) normal schooling was reached by 76% (22/29) and social continence by 80% (23/29) of the children with very early interventional therapy. In the older group only 54% reached normal schooling (P < 0.05) and 29% social continence (P < 0.001). The very early co-ordinated medical and physiotherapeutic rehabilitation treatment of children with MMC usually reduces the psychosocial stress and improves the quality of life of these children and of their families.

[Experiences with the early treatment of osteogenesis imperfecta]. / Erfahrungen mit der Frühbehandlung der Osteogenesis imperfecta (OI).

Between 1973 and 1988 twenty children with osteogenesis imperfecta were treated in the Department of Paediatric Surgery at the University of Berne, Switzerland. Our initial experience with the first 15 children, who had virtually no treatment during infancy and early childhood showed that they later developed severe soft tissue and skeletal deformities. Since resulting contractures and curvatures of the long bones are difficult to correct, we changed our therapeutic approach. Traditional therapy in OI was limited to the correction of bony malformations. Considering the fact, that the different elements of the locomotor system are part of a functional entity, we began early treatment combining physiotherapy and surgery.

Relationship between clinical conditions, radiographic findings and pulmonary functions in patients with cystic fibrosis.

Clinical and radiological findings, quantified by special scores, in 36 patients with cystic fibrosis are compared with lung function measurements, including the ratio of RV/TLC and FEV/VC and the arterial blood gases at rest and during exercise. Although respiratory function tests were found to correlate well with both the pulmonary finding score and the chest radiographic score, distinction of three severity groups was possible only by a vector calculation of the blood gases in PaO2-PaCO2-diagram, at rest and during exercise. Thus, measurements of pO2 and pCO2 at rest and on exercise appear to be a helpful adjuvant to routine spirometry for the individual appraisal of the degree of lung involvement, performance and care level.

Relative underweight in cystic fibrosis and its prognostic value.

On the basis of observations in 117 children with cystic fibrosis, seen from January 1956 to June 1976, it is demonstrated that the relative underweight (weight loss corrected for height) is most pronounced in children with predominantly pulmonary sypmtoms. The degree of underweight closely correlates inversely with survival. Because of its prognostic value, it is recommended that this clinical parameter be included in the checkups which are periodically carried out on children suffering from cystic fibrosis.

[Double-blind study on the anti-convulsive effect of phenobarbital and valproate in the Lennox syndrome]. / Doppeltblind-Untersuchung über die antikonvulsive Wirkung von Phenobarbital und Valproat beim Lennox-Syndrom.

In a double-blind crossover trial valproate was compared with phenobarbital with regard to anticonvulsive activity and tolerance in 17 epileptic children (mean age 55 +/- 26 months) with Lennox syndrome. Valproate in association with a phenobarbital dose reduced by about 40% proved to be to a statistically significant degree more active against epileptic seizures than phenobarbital alone. No difference in the effect on the EEG tracings was observed. Valproate appeared to be somewhat more active than phenobarbital with regard to behaviour, but the difference was not significant. Tolerance to both products was equally good.

Conventional and spectral EEG analysis in children treated with cytotoxic agents.

One hundred and six EEG investigations were carried out in 17 children with various types of neoplastic disease without cerebral involvement during one or more courses of treatment with cytotoxic agents EEGs were recorded before and 24 hr after administration of the drugs. The EEGs were evaluated visually and by spectral analysis. A transient slowing of the dominant frequency in the alpha band by about 1 Hz and a decrease in the relative power of alpha activity by 20-30% was observed in only 4 patients. These children did not show any clinical or biochemical signs of neurotoxicity. The children did not receive the same antineoplastic treatment. One patient received very high dose methotrexate, 2 patients received vincristine combined with other cytotoxic agents and the other patient received L-asparaginase. It is suggested that EEG changes in patients receiving intravenous cytotoxic treatment usually occur only where there is pre-existing impairment of the blood--cerebrospinal fluid barrier or blood-brain barrier. No clinical signs of epilepsy, new epileptiform waves in the EEG or long-term changes in the background activity of the EEG were observed in this pilot study.

Electroencephalographic findings in children treated with cytotoxic agents.

106 EEG investigations were carried out in 17 children with various types of neoplastic disease without cerebral involvement during one or more courses of treatment with cytotoxic agents. EEGs were recorded before and 24 h after administration of the drugs. A transient slowing of the dominant frequency in the alpha-band by about 1 c/s and a decrease in the relative power of alpha-activity by 20-30% was observed in only 4 patients. These children did not receive the same antineoplastic treatment. 1 patient received very high dose methotrexate, 2 patients received vincristine combined with other cytotoxic agents, and the other patient received L-asparaginase. It is suggested that EEG changes in patients receiving intravenous cytotoxic treatment usually occur only where there is a preexisting impairment of the blood-cerebrospinal fluid barrier or the blood-brain barrier.

Phenytoin therapy for epileptic children: evaluation of salivary and plasma concentrations and of methods of assessing compliance.

Monitoring phenytoin therapy in children is difficult because they fear blood sampling, yet frequent control of phenytoin levels may be necessary because of the age-dependence of phenytoin metabolism. In 22 patients aged between six and 15 years, phenytoin concentrations were studied in mixed saliva and parotid saliva and were compared with plasma levels from blood obtained at the same time. For mixed saliva, the saliva: plasma concentration ratio was 0.11 +/- SD 0.04 and in parotid saliva it was 0.10 +/- 0.06. In addition, compliance was studied in 13 children, each receiving between three and seven different quantities of phenytoin. Clinical judgement about compliance agreed well with the two laboratory measures. A graphic analysis of the relationship between plasma concentration and dosage is proposed as practical method of assessing compliance, because it appears to be both simple and reliable, and therefore suitable for routine use.

The age at onset of chronic Pseudomonas aeruginosa colonization in cystic fibrosis--prognostic significance.

To evaluate the prognostic significance of the age at onset of chronic Pseudomonas aeruginosa colonization (OPCP) with respect to pulmonary disease progression in patients with cystic fibrosis (CF), a retrospective long-term analysis using annual chest radiographs was performed on 54 CF patients. Thirty-seven patients (68%) were chronically colonized before the age of 12 years (group 1), 17 patients (32%) thereafter (group 2). These two groups did not significantly differ in terms of mean duration of follow up (16.2 +/- 5.9 years), sex, CF genotypes, colonization with other respiratory pathogens, supportive medical treatment and death rate during the study period. Chest radiographs were evaluated according to the Chrispin-Norman score, increasing scores representing increasing severity of respiratory disease. In both groups, progression of score means was not accelerated of score means was not accelerated up to 6 years after OCPC (Scores at OCPC set 0; mean score +/- SEM 6 years prior to OCPC -5.6 +/- 2.0; 10 years after OCPC +3.6 +/- 0.7 points). Patients chronically colonized prior to age 12 years (group 1) scored significantly higher between age 2 and 11 years (maximum difference at age 8 years [mean +/- SEM]: 9.4 +/- 0.7 vs. 4.3 +/- 1.3 points; P = 0.002) as compared to group 2. After age 11 years, mean scores were similar in both groups, since in group 2 scores increased rapidly after age 8 years. We conclude that OCPC did not cause an immediate acceleration of CF lung disease judged by serial chest radiographs. Rapid progression in group 2 (OCPC after age 12 years) was independent of OCPC since it occurred earlier. These data indicate that OCPC may be a marker rather than the cause of respiratory disease progression.

[Course and quantitative evaluation of the lung involvement in cystic fibrosis]. / Verlauf und quantitative Erfassung des pulmonalen Befalls bei zystischer Fibrose.

On the basis of clinical observations the course of pulmonary involvement in cystic fibrosis is studied, and an attempt at classification into three severity groups is made. Clinical and radiological findings, quantified by special scores, are presented and correlations between scores and lung function data are demonstrated. Although respiratory function tests - in particular the ratio of residual volume to total lung capacity (RV/TLC) - correlate with the clinical and chest radiographic scores, division into three severity degrees is only reliable for groups of patients. In the individual patient the clinical, radiological and lung function findings must be taken into account together. Assessment according to a scoring system may be helpful.

Acute metabolic alkalosis in cystic fibrosis: prospective study and review of the literature.

Between January 1993 and April 1994, 5 patients with cystic fibrosis, aged 4-9 months, were admitted to the Department of Pediatrics, University of Berne, Switzerland, with acute, severe metabolic alkalosis (sodium < 133 mmol/l, plasma potassium < 3.5, chloride < 85, bicarbonate > 35.0 mmol/l, blood pH > 7.43). 87 cases of acute metabolic alkalosis complicating cystic fibrosis reported in the literature between 1951 and 1995 were also reviewed. Our cases and those described in the literature demonstrate that acute metabolic alkalosis occurs in patients aged 2 years or less. Anorexia, vomiting, respiratory exacerbation, fever, and body weight loss often precede metabolic alkalosis. Furthermore, metabolic alkalosis is a common initial presentation of cystic fibrosis, suggesting that this diagnosis should be considered in the context of unexplained metabolic alkalosis.

Effect of high-affinity anti-Pseudomonas aeruginosa lipopolysaccharide antibodies induced by immunization on the rate of Pseudomonas aeruginosa infection in patients with cystic fibrosis.

Patients with cystic fibrosis (CF; N = 26) and with no prior history of infection with Pseudomonas aeruginosa were immunized with an octavalent O-polysaccharide-toxin A conjugate vaccine. During the next 4 years, 16 patients (61.5%) remained free of infection and 10 (38.5%) became infected. Total serum antilipopolysaccharide (LPS) antibody levels induced by immunization were comparable in infected and noninfected patients. In contrast, 12 of 16 noninfected versus 3 of 10 infected patients (p = 0.024) mounted and maintained a high-affinity anti-LPS antibody response. When compared retrospectively with the rate in a group of age- and gender-matched, nonimmunized, noncolonized patients with CF, the rate at which P. aeruginosa infections were acquired was significantly lower (p < or = 0.02) among all immunized versus nonimmunized patients during the first 2 years of observation. Subsequently, only those immunized patients who maintained a high-affinity anti-LPS antibody response had a significant reduction (p < or = 0.014) in the rate of infection during years 3 and 4. Smooth, typeable strains of P. aeruginosa predominated among immunized patients; rough, nontypeable strains were most frequently isolated from nonimmunized patients. Mucoid variants were isolated from one immunized patient versus six nonimmunized patients. These results indicate that the induction of a high-affinity P. aeruginosa anti-LPS antibody response can influence the rate of infection in patients with CF.