Prednisolone versus dexamethasone in croup: a randomised equivalence trial

Background

Croup remains a common respiratory problem presenting to emergency departments. A single oral treatment of oral dexamethasone results in improved outcome. Prednisolone has similar pharmacokinetic properties and has a significant advantage in that it is commercially available in liquid preparations.

Objective

To ascertain whether a single oral dose of prednisolone was equivalent to a single oral dose of dexamethasone (matched for potency) in children with mild to moderate croup.

Design

A double blind, randomised, controlled equivalence trial

Setting

Tertiary paediatric emergency department.

Patients

133 children aged 3 to 142 months presenting with mild to moderate croup.

Interventions

Children received either a single oral dose of dexamethasone 0.15 mg/kg or single oral dose of prednisolone 1 mg/kg.

Outcome

The main outcome measure was unscheduled re‐presentation to medical care as determined by telephone follow up at 7 to 10 days. Croup score, adrenaline (epinephrine) use, time spent in the emergency department, and duration of croup and viral symptoms were secondary outcome measures.

Results

Children treated with prednisolone were more likely to re‐present: 19 of 65 children (29%) reattended medical care compared with 5 of 68 (7%) from the dexamethasone group. The confidence intervals around this 22% difference in outcome were 8% to 35%, outside the 0% to 7.5% range of equivalence. There were no significant differences in other outcome measures.

Conclusion

A single oral dose of prednisolone is less effective than a single oral dose of dexamethasone in reducing unscheduled re‐presentation to medical care in children with mild to moderate croup.     

Is omitting post urinary‐tract‐infection renal ultrasound safe after normal antenatal ultrasound? An observational study

Background

Guidelines recommend obtaining a renal ultrasonogram (RUS) for young children after a first urinary tract infection (UTI).

Objective

The aim of the current study was to assess the concordance of prenatal and post‐UTI RUS findings in children with a first simple UTI.

Methods

This was a prospective study and included all children aged 5 years or younger who were hospitalised with a first simple UTI (determined as clinical response and normalisation of temperature within 48 h on initiation of antibacterial therapy with no complications). Data were collected from each child regarding the results of prenatal and post‐UTI RUS.

Results

Overall, 250 children were included in the study and the results of late‐pregnancy and post‐UTI RUS were available for 84% (n = 209). Complete concordance between the two RUS was demonstrated in 96% (n = 201). The predictive value of normal antenatal to normal post‐UTI RUS was 96% (95% CI: 93% to 99%). These results include four children with mild transient pelvic dilatation. In eight children in whom renal anomalies were demonstrated only in post‐UTI RUS, the influence of these anomalies on the children''s management was negligible.

Conclusions

Prenatal‐RUS have been performed in most children <5 years old hospitalised with a first simple UTI. Concordance with post‐infection tests is very high. Findings which appear only in post‐infectious RUS usually have negligible effects on children''s management. Thus, in such children with normal antenatal RUS omitting post‐UTI RUS could be considered.     

Reference chart for relative weight change to detect hypernatraemic dehydration

Objective

The validity of the rule of thumb that infants may have a weight loss of 10% in the first days after birth is unknown. We assessed the validity of this and other rules to detect breast‐fed infants with hypernatraemic dehydration.

Design

A reference chart for relative weight change was constructed by the LMS method. The reference group was obtained by a retrospective cohort study.

Participants

1544 healthy, exclusively breast‐fed infants with 3075 weight measurements born in the Netherlands and 83 cases of breast‐fed infants with hypernatraemic dehydration obtained from literature.

Results

The rule of thumb had a sensitivity of 90.4%, a specificity of 98.3% and a positive predictive value of 3.7%. Referring infants if their weight change is below −2.5 SDS (0.6th centile) in the reference chart in the first week of life and using the rule of thumb in the second week had a sensitivity of 85.5%, a specificity of 99.4% and a positive predictive value of 9.2%.

Conclusions

The rule of thumb is likely to produce too many false positive results, assuming that for screening purposes the specificity needs to be high. A chart for relative weight change can be helpful to detect infants with hypernatraemic dehydration.     

Infant feeding in the second 6 months of life related to iron status: an observational study

Objective

To investigate the relationship between iron status in infancy and type of milk and weaning solids consumed.

Design

An observational cohort study.

Setting

928 term infants from the Avon Longitudinal Study of Parents and Children in 1993–94.

Methods

Haemoglobin and ferritin concentrations at 8 and 12 months were assessed in relation to type and quantity of milk intake at 8 months.

Results

By WHO criteria, 22.7% of the infants were anaemic at 8 months and 18.1% at 12 months. More breast‐ than formula‐fed infants were anaemic at 8 and 12 months. Cows'' milk as the main drink was associated with increased anaemia at 12 months and low ferritin at 8 and 12 months. No association was found between any nutrients and haemoglobin concentrations. Protein and non‐haem iron intakes were positively associated with ferritin concentrations and calcium intake negatively. This effect was more marked in infants being fed cows'' milk. More than 25% of infants in the breast milk and cows'' milk groups and 41% of infants having >6 breast feeds per day had iron intakes below the lower reference nutrient intake. Feeding cows'' milk or formula above 600 ml or >6 breast feeds per day was associated with lower intakes of solids.

Conclusions

Both breast and cows'' milk feeding were associated with higher levels of anaemia. Satisfactory iron intake from solids in later infancy is more likely if formula intake is <600 ml per day and breast feeds are limited to <6 feeds per day. Cows'' milk should be strongly discouraged as a main drink before 12 months.     

Increasing prevalence of hypospadias in Western Australia, 1980-2000

Objectives

Hypospadias, a common birth defect, has shown widespread variation in reported rates and temporal trends across countries over the last 30 years. The aim of this study was to determine the prevalence and trends of hypospadias in an Australian population.

Design

Population‐based study of all male infants born in Western Australia (WA) between 1980 and 2000 diagnosed with hypospadias and notified to the WA Birth Defects Registry.

Main outcome measures

Prevalence of hypospadias, birth outcome and association with other congenital anomalies, stratified by degree‐of‐severity.

Results

1788 cases of hypospadias were registered in WA in 1980–2000 with an overall prevalence of 34.8 (95% confidence interval (CI): 33.2 to 36.4) cases per 10 000 births. The prevalence increased by 2.0% per annum (95% CI: 1.2% to 2.8%) from 27.9 in 1980 to 43.2 per 10 000 births in 2000 (p<0.001). Hypospadias was mild in 84% of cases, moderate‐severe in 11% and unspecified in 5%, with the number of moderate‐severe hypospadias almost doubling over time (p<0.01). There were 1465 (82%) cases of isolated hypospadias and 323 (18%) had co‐existing anomalies. Infants with co‐existing genital (relative risk (RR) 4.5; 95% CI: 3.3 to 6.1) or non‐genital (RR 1.5; 95% CI: 1.0 to 2.2) anomalies were more likely to have moderate‐severe hypospadias compared with isolated cases.

Conclusion

Hypospadias affects one in 231 births and has been reported to have increased significantly over the last 20 years. Future investigation of the aetiology of hypospadias is important to identify potentially modifiable risk factors and ensure optimal male reproductive health in the future.     

A low cost, colour coded, hand held spring scale accurately categorises birth weight in low resource settings

Aims

To determine the accuracy of a low cost, spring calibrated, hand held scale in classifying newborns into three weight categories (⩾2500 g, 2000–2499 g, <2000 g).

Methods

The test device was compared to a gold standard digital baby scale with precision to 2 g. In Sarlahi district, Nepal, 1890 newborns were eligible for the study. Measurements were collected for both the test device and the digital scale from 1820 (96.3%) newborns.

Results

The overall low birth weight (LBW) prevalence rate for the gold standard digital scale was 28.1% (511/1820). Sensitivity (93.7%) and specificity (97.6%) of the test device was high compared to LBW classifications based on digital weight measurements. Classification of infants into the <2000 g category was 5.0% and 4.7% for the gold standard and test device, respectively. Sensitivity and specificity of the test device in identifying infants <2000 g was 87.8% and 99.6%, respectively. Positive predictive values were high (>91%) for both weight categories

Conclusions

This low cost, simple‐to‐use device classified infants into weight categories with a high degree of consistency and accuracy that exceeds that of surrogate measures. This new device is useful for identifying and targeting life saving interventions for LBW, high risk infants in settings where infants are born in the home and conventional weighing scales are unavailable.     

Nasopharyngeal aspiration for diagnosis of pulmonary tuberculosis.

BACKGROUND: Confirmation of pulmonary tuberculosis (PTB) in young children is difficult as they seldom expectorate sputum. AIM: To compare sputa obtained by nasopharyngeal aspiration and by sputum induction for staining and culture of Mycobacterium tuberculosis. PATIENTS AND METHODS: Patients from Mulago Hospital, Kampala with symptoms suggestive of PTB were considered for inclusion in the study. Those with a positive tuberculin test and/or a chest radiograph compatible with tuberculosis were recruited. Infection with human immunodeficiency virus (HIV) was confirmed by duplicate enzyme-labelled immunosorbent assay or in children <15 months by polymerase chain reaction (PCR). Direct PCR was undertaken on 82 nasopharyngeal aspirates. RESULTS: Of 438 patients referred, 94 were recruited over a period of 5 months. Median (range) age was 48 (4-144) months. Of 63 patients tested, 69.8% were infected with HIV. Paired and uncontaminated culture results were available for 88 patients and smear results for 94 patients. Nasopharyngeal aspirates were smear-positive in 8.5% and culture-positive in 23.9%. Induced sputa were smear-positive in 9.6% and culture positive in 21.6%. Overall, 10.6% were smear-positive, 25.5% were culture-positive and 26.6% had smear and/or culture confirmed tuberculosis. Direct PCR on nasopharyngeal aspirates had a sensitivity of 62% and specificity of 98% for confirmation of culture-positive tuberculosis. CONCLUSIONS: Nasopharyngeal aspiration is a useful, safe and low-technology method for confirmation of PTB and, like sputum induction, can be undertaken in outpatient clinics.     

Prevalence of overweight and obesity in the Netherlands in 2003 compared to 1980 and 1997

Objective

To assess the prevalence of overweight and obesity in children living in the Netherlands and compare the findings with the Third and Fourth National Growth Studies carried out in 1980 and 1997, respectively.

Design and methods

Data were obtained from the child health care system. International cut‐off points for body mass index (BMI) were used to determine overweight and obesity. Cases were weighted for ethnicity and municipality size in such a way that the sample matched the distribution in the general population. The LMS method was used to calculate the age‐related distribution of BMI, and the prevalence was calculated from the fitted distribution.

Patients

Data on 90 071 children aged 4–16 years were routinely collected by 11 community health services during 2002–2004.

Results

On average, 14.5% of the boys and 17.5% of the girls were overweight (including obesity), which is a substantial increase since 1980 (boys 3.9%, girls 6.9%) and 1997 (boys 9.7%, girls 13.0%). Similarly, 2.6% of the boys and 3.3% of the girls aged 4–16 years were obese, which is much higher than in 1980 (boys 0.2%, girls 0.5%) and 1997 (boys 1.2%, girls 2.0%). At the age of 4, 12.3% of the boys and 16.2% of the girls were already overweight.

Conclusions

The prevalence of overweight and obesity in the Netherlands is still rising, and at an even faster rate than before. Evidence‐based interventions are needed to counter the obesity epidemic, and there is an urgent need for pre‐school intervention programmes.     

Ethnic and gender differences in body fat in British schoolchildren as measured by DXA

Background

There are known to be ethnic differences in body composition in adults which are related to ethnic differences in adult disease.

Objectives

To evaluate gender and ethnic differences in percentage body fat in British schoolchildren and to compare these differences with classification of obesity using body mass index (BMI) criteria.

Design

A cross‐sectional study of 1251 healthy children and adolescents aged 5–18 years from white, South Asian and African‐Caribbean ethnic groups. Percentage body fat was determined by dual x ray absorptiometry and the subjects classified using BMI criteria for overweight and obesity.

Results

Significant gender differences in percentage body fat were seen, with girls having higher values from the age of 5 years. Girls had 3.8% higher percentage body fat at 5 years of age increasing to 12.9% at 18 years of age. Significant ethnic differences were found, with South Asian girls and boys having the highest percentage body fat from 5 and 7 years of age, respectively. These differences increased with age, being most significant in the teenage years. Although South Asian girls and boys were over‐represented in the group containing children with more than 25% body fat (p<0.0001, χ² test), African‐Caribbean subjects were more likely to be classified as obese using BMI criteria.

Conclusions

There are clear gender and ethnic differences in percentage body fat in British schoolchildren which may relate to known differences in the risk of type 2 diabetes in adolescence and adulthood. BMI criteria for defining overweight and obesity do not accurately identify ethnic differences in body fat.     

An 8 year study of risk factors for SIDS: bed-sharing versus non-bed-sharing

Background

It is unclear if it is safe for babies to bed share with adults. In Ireland 49% of sudden infant death syndrome (SIDS) cases occur when the infant is bed‐sharing with an adult.

Objective

To evaluate the effect of bed‐sharing during the last sleep period on risk factors for SIDS in Irish infants.

Design

An 8 year (1994–2001) population based case control study of 287 SIDS cases and 831 controls matched for date, place of birth, and sleep period. Odds ratios and 95% confidence intervals were calculated by conditional logistic regression.

Results

The risk associated with bed‐sharing was three times greater for infants with low birth weight for gestation (UOR 16.28 v 4.90) and increased fourfold if the combined tog value of clothing and bedding was ⩾10 (UOR 9.68 v 2.34). The unadjusted odds ratio for bed‐sharing was 13.87 (95% CI 9.58 to 20.09) for infants whose mothers smoked and 2.09 (95% CI 0.98 to 4.39) for non‐smokers. Age of death for bed‐sharing and sofa‐sharing infants (12.8 and 8.3 weeks, respectively) was less than for infants not sharing a sleep surface (21.0 weeks, p<0.001) and fewer bed‐sharing cases were found prone (5% v 32%; p = 0.001).

Conclusion

Risk factors for SIDS vary according to the infant''s sleeping environment. The increased risk associated with maternal smoking, high tog value of clothing and bedding, and low z scores of weight for gestation at birth is augmented further by bed‐sharing. These factors should be taken into account when considering sleeping arrangements for young infants.     

Randomised trial of a parenting intervention during neonatal intensive care

Objective

To evaluate the influence of parenting intervention on maternal responsiveness and infant neurobehavioural development following a very premature birth.

Design

Cluster‐randomised controlled trial, with a crossover design and three‐month washout period.

Setting

Six neonatal intensive care units.

Patients

Infants born <32 weeks'' gestation.

Intervention

The Parent Baby Interaction Programme (PBIP) is a supportive, educational intervention delivered by research nurses in the neonatal intensive care unit, with optional home follow‐up for up to six weeks after discharge.

Main outcome measures

Parenting stress at 3 months adjusted age, as measured by the Parenting Stress Index (PSI). Other outcomes included the Neurobehavioural Assessment of the Preterm Infant (NAPI) and maternal interaction as assessed by the Nursing Child Assessment Teaching Scale (NCATS) and the responsivity subscale for Home Observation for Measurement of the Environment (HOME).

Results

112 infants were recruited in the intervention phases and 121 in the control phases. Mean standardised NAPI scores at 35 weeks did not differ between the PBIP and control groups. Both groups had low but similar NCATS caregiver scores before discharge (36.6 in the PBIP group and 37.4 in control, adjusted mean difference −0.7, 95% CI −2.7 to 1.4). At three months, adjusted age mean PSI scores for the PBIP group were 71.9 compared with 67.1 for controls (adjusted mean difference 3.8, 95% CI −4.7 to 12.4). NCATS scores and HOME responsivity scores were similarly distributed between the groups.

Conclusion

This early, nurse‐delivered, parent‐focused interaction programme intervention had no measurable effects on short‐term infant neurobehavioural function, mother–child interaction or parenting stresses.     

Comparison of alteplase and heparin in maintaining the patency of paediatric central venous haemodialysis lines: a randomised controlled trial

Objectives

To determine whether the tissue plasminogen activator, alteplase, is more effective than heparin in preventing blood clots developing in children''s haemodialysis central lines between dialysis sessions.

Design

A prospective double‐blind, within‐patient multiperiod cross‐over controlled trial of instilling a “lock” of either heparin 5000 U/ml or alteplase 1 mg/ml into the central lines of two children haemodialysed twice weekly, and seven dialysed thrice weekly, over 10 weeks.

Setting

A UK paediatric nephrology unit.

Main outcome measures

Weight of blood clot aspirated from the line at the start of the next dialysis session.

Results

The odds of a clot forming was 2.4 times greater with heparin than alteplase (95% CI 1.4 to 4.0; p = 0.001), and when present they were 1.9 times heavier (31 vs 15 mg; 95% CI 1.5 to 2.4; p<0.0005). There was no effect of inter‐dialytic interval. One child required an alteplase infusion to clear a blocked line following a heparin lock. We subsequently changed our routine locks from heparin to alteplase. Comparing the year before and after that change, the incidence of blocked lines requiring an alteplase or urokinase infusion fell from 2.7 to 1.2 per child (p<0.03), and the need for surgical replacements from 0.7 to nil (p<0.02).

Conclusion

Alteplase is significantly more effective than heparin in preventing clot formation in central haemodialysis lines. This reduces morbidity and improves preservation of central venous access. It is more expensive, though relatively economic if packaged into syringes and stored frozen until needed, but reduces the costs of unblocking or replacing clotted lines.     

Vaccine-related pain: randomised controlled trial of two injection techniques

Objective

To compare acute pain response during immunisation in infants using a slow standard of care injection technique versus a rapid pragmatic technique.

Design

Randomised controlled trial.

Setting

Single‐centre, urban paediatric primary care practice.

Subjects

Healthy infants 4–6 months of age receiving their routine DPTaP‐Hib immunisation.

Interventions

Standard of care group: slow aspiration prior to injection, slow injection and slow withdrawal. Pragmatic group: no aspiration, rapid injection and rapid withdrawal.

Main outcome measures

Immediate infant pain measured by the Modified Behavior Pain Scale (MBPS), crying and parent/paediatrician visual analogue scale (VAS).

Results

113 infants participated; there were no observed differences in age, birth order or prior analgesic use. Mean MBPS scores (95% confidence interval (CI)) were higher (p<0.001) for the standard group compared to the pragmatic group, 5.6 (5 to 6.3) vs 3.3 (2.6 to 3.9). The standard group was more likely to cry, 47/57 (82%) vs 24/56 (43%), to cry longer, median (interquartile range (IQR)) 14.7 s (8.7–35.6) vs 0 s (0–11.30), and to take longer to have the vaccine injected, median (IQR) 8.8 s (7.9–10.3) vs 0.9 s (0.8–1.1), p<0.001 for all comparisons. The median (IQR) VAS scores by parents and paediatricians were higher for the standard group: VAS parent, 3.5 (1.6–5.5) vs 1.9 (0.1–3.1) and VAS paediatrician, 2.8 (2.0–5.1) vs 1.4 (0.2–2.4). There were no adverse events.

Conclusion

Immunisation using a pragmatic rapid injection technique is less painful than a slow standard of care technique and should be recommended for routine intramuscular immunisations.     

Prediction of vesicoureteral reflux after a first febrile urinary tract infection in children: validation of a clinical decision rule

Aims

To test the reproducibility of a highly sensitive clinical decision rule proposed to predict vesicoureteral reflux (VUR) after a first febrile urinary tract infection in children. This rule combines clinical (family history of uropathology, male gender, young age), biological (raised C reactive protein), and radiological (urinary tract dilation on renal ultrasound) predictors in a score, and provides 100% sensitivity.

Methods

A retrospective hospital based cohort study included all children, 1 month to 4 years old, with a first febrile urinary tract infection. The sensitivities and specificities of the rule at the two previously proposed score thresholds (⩽0 and ⩽5) to predict respectively, all‐grade or grade ⩾3 VUR, were calculated.

Results

A total of 149 children were included. VUR prevalence was 25%. The rule yielded 100% sensitivity and 3% specificity for all‐grade VUR, and 93% sensitivity and 13% specificity for grade ⩾3 VUR. Some methodological weaknesses explain this lack of reproducibility.

Conclusions

The reproducibility of the previously proposed decision rule was poor and its potential contribution to clinical management of children with febrile urinary tract infection seems to be modest.     

Stress in mothers of young children with eczema

Objective

To assess parental stress levels of mothers of children less than 6 years old with eczema and compare these levels with those reported for other chronic childhood illnesses.

Methods

Mothers were recruited from hospital‐based out‐patient clinics (55%) or while their child was an in‐patient (45%) for management of eczema. Maternal stress was measured utilising the Parenting Stress Index‐Long Form (PSI) in 33 mothers. The severity of the eczema at the time of interview was documented by the Eczema Area and Severity Index (EASI) score and the Investigators'' Global Assessment (IGA) score.

Results

The children with eczema had a mean age of 2.8 years. Mothers of children aged 5 years or less with eczema exhibited significantly higher total stress scores (mean PSI 259.6, 95% CI 244.9 to 274.3) as compared to mothers of normal children (PSI 222.8, 95% CI 221.4 to 224.2) and children with other chronic disorders such as insulin‐dependent diabetes (PSI 218.1, 95% CI 204.7 to 231.6) and profound deafness (PSI 221.7, 95% CI 206.4 to 237.0). Stress scores in the parental domain (138.2, 95% CI 128.9 to 147.6) did not differ significantly from the scores of parents of children with severe disabilities such as those requiring home enteral feeding (135.2, 95% CI 129.3 to 141.1) and those with Rett syndrome (132.8, 95% CI 125.0 to 140.6).

Conclusions

Moderate to severe childhood eczema should be regarded as a significant illness in which maternal stress is equivalent to that associated with the care of children with severe developmental and physical problems.     

Prevalence rates for constipation and faecal and urinary incontinence

Objective

To evaluate the prevalence rates for constipation and faecal and urinary incontinence in children attending primary care clinics in the United States.

Methods

Retrospective review of case records of all children, 4–17 years of age, seen for at least one health maintenance visit during a 6 month period and followed from birth or within the first 6 months of age in our clinics. We reviewed all charts for constipation, faecal incontinence and urinary incontinence.

Results

We included 482 children in the study, after excluding 39 children with chronic diseases. The prevalence rate for constipation was 22.6% and was similar in boys and girls. The constipation was functional in 18% and acute in 4.6%. The prevalence rate for faecal incontinence (⩾1/week) was 4.4%. The faecal incontinence was associated with constipation in 95% of our children. The prevalence rate for urinary incontinence was 10.5%; 3.3% for daytime only, 1.8% for daytime with night‐time and 5.4% for night‐time urinary incontinence. Faecal and urinary incontinence were significantly more commonly observed in children with constipation than in children without constipation.

Conclusion

The prevalence rates were 22.6% for constipation, 4.4% for faecal incontinence and 10.5% for urinary incontinence in a US primary care clinic. Children with constipation had higher prevalence rates for faecal and urinary incontinence than children without constipation. Boys with constipation had higher rates of faecal incontinence than girls with constipation.     

Neonatal vitamin K prophylaxis in Great Britain and Ireland: the impact of perceived risk and product licensing on effectiveness

Objective

To determine current use of vitamin K (VK) prophylaxis in newborns and review the efficacy and effectiveness of regimens used.

Design

Efficacy and effectiveness calculated using current practice details, data from Southern Ireland and two previous surveys, together with contemporaneous studies of vitamin K deficiency bleeding (VKDB).

Setting

Current survey: United Kingdom (Great Britain and Northern Ireland). Efficacy and effectiveness tables: United Kingdom and Southern Ireland.

Main outcome measures

Current VK prophylaxis following uncomplicated term deliveries. Relative risk of VKDB calculated for the VK actually received and for “intention to treat”.

Results

Questionnaire response rate 95% (n = 243), all recommending VK prophylaxis. No association between unit size and route of administration. For uncomplicated term deliveries, 60% recommended intramuscular (IM) prophylaxis, 24% oral and 16% offered both routes without bias. All units offering IM gave a single dose, mostly 1 mg Konakion Neonatal. Oral regimens showed more variation: two thirds gave 2 mg (range 0.5–2 mg), the number of doses ranged from 1 to 11 and many used preparations off‐licence or the unlicensed Orakay. IM prophylaxis, if given, provided the best protection (most efficacious) against VKDB. However, on an intention‐to‐treat basis (effectiveness), there is no statistically significant difference between the risks of VKDB after intended IM VK and after oral prophylaxis intended to continue beyond a week.

Conclusions

Although the principles of VK prophylaxis is now accepted by all, there is no uniformity in practice. Omission of prophylaxis appears to be a greater problem for IM than for multi‐dose oral prophylaxis, affecting overall effectiveness.     

The usefulness of bone marrow aspiration in the diagnosis of Niemann-Pick disease type C in infantile liver disease

Background

Niemann–Pick disease type C (NPC) is a fatal, autosomal recessive lysosomal storage disease which may present in infancy with cholestatic jaundice and/or hepatosplenomegaly. In cholestatic patients with splenomegaly, a bone marrow aspirate has been advocated as a relatively accessible tissue to demonstrate storage phenomena. Typically in patients with NPC, macrophages with abnormal cholesterol storage, so called foam cells, can be detected in the bone marrow.

Aim

To review our experience of bone marrow aspiration in children with NPC presenting with infantile liver disease.

Methods

A retrospective analysis of 11 consecutive children (8 males) from Birmingham Children''s Hospital with NPC presenting with infantile liver disease was undertaken. The diagnosis of NPC was confirmed in all cases by demonstrating undetectable or low rates of cholesterol esterification and positive filipin staining for free cholesterol in cultured fibroblasts.

Results

The median age at presentation was 1.5 months (range 0.5–10). Bone marrow aspirates showed storage cells in only 7/11 cases. Bone marrow aspirates which had storage cells were undertaken at a median age of 11 months while those with no storage cells were undertaken at median age 2.3 months. The overall sensitivity of bone marrow aspirates for detecting storage cells in children presenting with infantile liver disease was 64%; however, for children who had bone marrow aspirates in the first year of life it was only 57%.

Conclusions

The sensitivity of bone marrow aspirate for the diagnosis of NPC disease in patients presenting with infantile liver disease was lower than previously reported. Where NPC is suspected clinically, definitive investigations should be undertaken promptly. There is a need to develop sensitive screening methods for NPC in children presenting with infantile liver disease.     

Does splenectomy in cystic fibrosis related liver disease improve lung function and nutritional status? A case series

Aims

To review the effect of total splenectomy on lung function and nutrition in children with cystic fibrosis related liver disease (CFLD) and associated portal hypertension. The stated indications for surgery and the short and long term risks of the procedure were also documented.

Method

Over a 25 year period from January 1980 to June 2005, approximately 650 patients with cystic fibrosis (CF) were treated at the Royal Children''s Hospital, Melbourne, Australia. Nine patients with CFLD who underwent a splenectomy during that time were identified and their medical records were reviewed.

Results

FEV₁% predicted dropped by −16±11% in the two years pre‐splenectomy. This contrasts with the increase in FEV₁% predicted of 2±16% in the two years post‐splenectomy (p = 0.05). The cumulative gain in WAZ score (ΔWAZ pre) over the two years prior to splenectomy of 0.045±0.69 was not significantly different from the cumulative gain in WAZ score (ΔWAZ post) for the two years after splenectomy of 0.15±0.36 (p = 0.65). The average age at splenectomy was 14.8 years (SD = 3 years). The average weight of an excised spleen was 983 g (SD = 414 g). There were no deaths associated with splenectomy. The median length of follow up post‐splenectomy was 6.0 years (range 0.7–15.8). There were no episodes of bacterial peritonitis or overwhelming sepsis.

Conclusions

Splenectomy may have a beneficial effect on lung function although this may not manifest itself until the second year post‐splenectomy. Splenectomy in patients with CFLD appears to be a safe procedure.