Performance, treatment pathways, and effects of alternative policy options for screening for developmental dysplasia of the hip in the United Kingdom.

AIMS: To compare, using a decision model, performance, treatment pathways and effects of different newborn screening strategies for developmental hip dysplasia with no screening. METHODS: Detection rate, radiological absence of subluxation at skeletal maturity and avascular necrosis of the femoral head, as favourable and unfavourable treatment outcomes respectively, were compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; the addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). RESULTS: Universal or selective ultrasound detects more more affected children (76% and 60% respectively) than clinical screening alone (35%), results in a higher proportion of affected children with favourable treatment outcomes (92% and 88% respectively) than clinical screening alone (78%) or no screening (75%), and the highest proportion of these achieved without recourse to surgery (64% and 79% respectively) compared with clinical screening alone (18%). However, ultrasound based strategies are also associated with the highest number of unfavourable treatment outcomes arising in unaffected children treated following a false positive screening result. The detection rate of clinical screening alone becomes similar to that reported for universal ultrasound when based on studies using experienced examiners (80%) rather than junior medical staff (35%). CONCLUSION: From the largely observational data available, ultrasound based screening strategies appear to be most sensitive and effective but are associated with the greatest risk of potential adverse iatrogenic effects arising in unaffected children.     

Efficiency of alternative policy options for screening for developmental dysplasia of the hip in the United Kingdom

Aims: To assess, using a decision model, the efficiency of ultrasound based and clinical screening strategies for developmental dysplasia of the hip. Methods: The additional cost per additional favourable outcome was compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). Results: Ultrasound based screening strategies are predicted to be more effective but more costly than clinical screening or no screening. Estimated total costs per 100 000 live births are approximately £4 million for universal ultrasound, £3 million for selective ultrasound, £1 million for clinical screening alone, and £0.4 million for no screening. The relative efficiency of selective ultrasound and clinical screening is poorly differentiated, and depends on how infants are selected for ultrasound as well as the expertise of clinical screening examiners. If training costs less than £20 per child screened, clinical screening alone would be more efficient than selective ultrasound. Relative to no screening, each of the 16 additional favourable outcomes achieved as a result of selective ultrasound costs approximately £0.2 million, while each of the five favourable outcomes achieved through universal ultrasound screening, over and above selective ultrasound, costs approximately £0.3 million. Conclusions: Policy choice depends on values attached to the different outcomes, willingness to pay to achieve these and total budget.     

Efficiency of alternative policy options for screening for developmental dysplasia of the hip in the United Kingdom.

AIMS: To assess, using a decision model, the efficiency of ultrasound based and clinical screening strategies for developmental dysplasia of the hip. METHODS: The additional cost per additional favourable outcome was compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). RESULTS: Ultrasound based screening strategies are predicted to be more effective but more costly than clinical screening or no screening. Estimated total costs per 100,000 live births are approximately pound 4 million for universal ultrasound, pound 3 million for selective ultrasound, pound 1 million for clinical screening alone, and pound 0.4 million for no screening. The relative efficiency of selective ultrasound and clinical screening is poorly differentiated, and depends on how infants are selected for ultrasound as well as the expertise of clinical screening examiners. If training costs less than pound 20 per child screened, clinical screening alone would be more efficient than selective ultrasound. Relative to no screening, each of the 16 additional favourable outcomes achieved as a result of selective ultrasound costs approximately pound 0.2 million, while each of the five favourable outcomes achieved through universal ultrasound screening, over and above selective ultrasound, costs approximately pound 0.3 million. CONCLUSIONS: Policy choice depends on values attached to the different outcomes, willingness to pay to achieve these and total budget.     

Management of parapneumonic effusion and empyema

Aims: To gather data on the clinical presentation of parapneumonic effusion and empyema and to examine the effect of different management strategies on short term outcomes. Methods: Retrospective case note review of 48 children admitted to a tertiary unit between January 1998 and March 2001. Effusions were classified into three stages dependent on ultrasound findings. Results: The stage of effusion was not associated with duration of previous symptoms or length of previous admission. An interventional procedure was performed on median day 2 of admission in 46 children: eight (17%) had an intercostal drain alone, 14 (29%) had an intercostal drain followed by intrapleural fibrinolytic therapy, and 24 (50%) had a thoracotomy. Three children who had an initial intercostal drain alone returned to theatre for thoracotomy, and two children who had intrapleural fibrinolysis returned for thoracotomy. Median length of stay (interquartile range) for each initial procedure was 15 days (6–20) for intercostal drain alone, 8 days (6–12) for fibrinolytic therapy, and 6.5 days (5–9) for thoracotomy. Stay for intercostal drain alone was significantly longer than for thoracotomy. Conclusion: Early surgical management of empyema is associated with a favourable outcome.     

Management of parapneumonic effusion and empyema.

AIMS: To gather data on the clinical presentation of parapneumonic effusion and empyema and to examine the effect of different management strategies on short term outcomes. METHODS: Retrospective case note review of 48 children admitted to a tertiary unit between January 1998 and March 2001. Effusions were classified into three stages dependent on ultrasound findings. RESULTS: The stage of effusion was not associated with duration of previous symptoms or length of previous admission. An interventional procedure was performed on median day 2 of admission in 46 children: eight (17%) had an intercostal drain alone, 14 (29%) had an intercostal drain followed by intrapleural fibrinolytic therapy, and 24 (50%) had a thoracotomy. Three children who had an initial intercostal drain alone returned to theatre for thoracotomy, and two children who had intrapleural fibrinolysis returned for thoracotomy. Median length of stay (interquartile range) for each initial procedure was 15 days (6-20) for intercostal drain alone, 8 days (6-12) for fibrinolytic therapy, and 6.5 days (5-9) for thoracotomy. Stay for intercostal drain alone was significantly longer than for thoracotomy. CONCLUSION: Early surgical management of empyema is associated with a favourable outcome.     

Universal versus targeted screening of infants for sickle cell disease: a cost-effectiveness analysis

OBJECTIVE: To compare the health outcomes, costs, and incremental cost-effectiveness of universal neonatal screening for sickle cell disease (SCD) with screening targeted to African Americans. STUDY DESIGN: A cost-effectiveness analysis was done by using a Markov simulation model that considered the costs and outcomes associated with the prevention and treatment of sepsis in those with sickle cell anemia and sickle beta(0)-thalassemia. Three strategies were compared: (1) no screening, (2) targeted screening of African Americans, and (3) universal screening for SCD. RESULTS: In the base case analysis, targeted screening of African Americans compared with no screening cost $6709 per additional year of life saved, and universal screening compared with targeted screening cost $30,760 per additional year of life saved. In a sensitivity analysis, the cost per additional year of life saved with universal screening compared with targeted screening was positively correlated with the delivery rate of targeted screening and was inversely related to the proportion of African Americans in the population. CONCLUSIONS: Targeted screening of African American newborns for SCD compared with no screening is always cost-effective. Universal screening compared with targeted screening always identifies more infants with disease, prevents more deaths, and is cost-effective given certain delivery rates for targeted screening and proportions of African Americans in the population.     

Neurodevelopmental outcome in very-low-birth-weight infants with or without periventricular haemorrhage and/or leucomalacia

The aim of the study was to verify the predictive value of ultrasound performed in the neonatal period for short-term neurodevelopmental prognosis in 122 preterm very-low-birth-weight infants followed-up at 36 months. Neuromotor development was favourable in 53 (87%) subjects with normal ultrasound findings and in 21 (81%) subjects presenting uncomplicated haemorrhage. However, sensory and/or cognitive sequelae developed in 13% and 19% of the two groups, respectively. Outcome was unfavourable in 14 (50%) of 28 patients with ultrasound findings of complicated cerebral haemorrhages and in 5 (71%) of those (7) with ultrasound findings of parenchymal lesions without haemorrhage. Neonatal ultrasound examination seems to be fundamental in predicting neuromotor, but not cognitive, outcome in very-low-birth-weight infants.     

NICU‐only versus universal screening for newborn hearing loss: Population audit

Aim: Targeted newborn hearing screening for infants in neonatal intensive care units (NICUs) may be considered when resources preclude universal newborn hearing screening (UNHS). However, process outcomes have not been compared between stand‐alone NICU hearing screening programs and NICU screening within a full UNHS program. Methods: Comparison of two consecutive hearing screening programs delivered under similar conditions in the four NICUs in Victoria, Australia. All NICU infants were eligible for pre‐discharge automated auditory brainstem response (AABR) hearing screening. Capture, referral and diagnostic data were collected for all NICU infants during the NICU‐only (April 2003–February 2005) and subsequent UNHS (April 2005–June 2006) programs. Results: 4704 eligible infants were admitted during the 23‐month NICU‐only period, and 3160 during the 15‐month UNHS period. Double AABR using ALGO 3i equipment was planned for both programs but, due to clinician concern about this high‐risk clinical population, the NICU‐only protocol was amended to single AABR using AccuScreen equipment. Capture rates were 71.1% (NICU‐only) vs. 95.4% (UNHS) (P < 0.001), successful follow‐up rates were 85.8% vs. 96% (P= 0.004), and mean corrected age at the first audiology appointment was 51.5 vs. 40.2 days (P= 0.05). Conclusions: NICU screening offered within a larger UNHS program outperformed the stand‐alone NICU hearing screening program on all measured parameters. Greater resourcing might address shortcomings of the stand‐alone program but would also reduce its potential savings. The high loss to follow‐up also argues against the often‐advocated approach of referring all NICU infants for diagnostic audiologic testing, bypassing hearing screening altogether.     

Effectiveness of ultrasound screening for developmental dysplasia of the hip

OBJECTIVE: To determine the effectiveness of ultrasound screening for developmental dysplasia of the hip (DDH) after the neonatal period. DESIGN: Prospective cohort study. SETTING: Child health care centres. PARTICIPANTS: Infants attending the child health care centres. INTERVENTIONS: The intervention group (n = 5170) was screened by ultrasound at 1, 2, and 3 months of age. The control group (n = 2066) was screened by routine physical examination as part of the programme for child health surveillance at the child health care centres (CHC screening). For evaluation of the screening, the children in both the intervention and control group received an ultrasound examination after 6 months of age to detect any abnormality that might have been missed by the screening. RESULTS: The sensitivity of the ultrasound screening was 88.5%, and the referral rate 7.6%. As a result of the ultrasound screening, 4.6% of the children were treated. The sensitivity of the CHC screening was 76.4%, with a referral rate of 19.2%. The treatment rate was 2.7%. Of the treated children in the ultrasound screening group, 67% were referred before the age of 13 weeks, whereas in the CHC screening group only 29% were referred before this age. CONCLUSIONS: This study shows that ultrasound screening detects more children with DDH than CHC screening and that more of them are detected at an earlier age. To accomplish this, even fewer children have to be referred. However, even general ultrasound screening seems not to eradicate late cases of DDH. The higher treatment rate in the population screened by ultrasound may be a result of overtreatment.     

Prognosis of neonates with symptomatic respiratory insufficiency surviving with the aid of ventilator therapy.

Twenty-one survivors representing all survivors of neonatal symptomatic respiratory distress treated with intermittent positive pressure ventilation have been follow-up at an age of 2.9 to 7.0 years. The chance of survival proved to be relatively favourable in infants ventilated for perinatal pneumonias and unfavourable in infants ventilated for haemorrhagic diseases and respiratory insufficiency secondary to surgical conditions. At the follow-up half of the children presented with neurological symptoms but only 10% were found severely handicapped. One infant had a tracheostomy due to a laryngeal stenosis, while none developed broncho-pulmonary dysplasia. The late prognosis seemed unfavourable when the children had suffered from severe birth asphyxia and in infants ventilated for prolonged recurrent apnoeic spells. The relation between the clinical indications for ventilator therapy and later outcome is obscured however, by a vase number of complicating perinatal events.     

Paediatric screening for hypercholesterolaemia in Europe

Different screening strategies are currently recommended to identify children with (familial) hypercholesterolaemia in order to initiate early lipid management. However, these strategies are characterised to date by low adherence by the medical community and limited compliance by parents and children. In a literature review, the authors assess which children should undergo screening and which children are in effect identified through the currently recommended strategies. Furthermore, the authors discuss the different screening tools and strategies currently used in Europe and what is known about the negative aspects of screening. The authors conclude that currently recommended selective screening strategies, which are mainly based on family history, lack precision and that a large percentage of affected children who are at increased risk of future coronary artery disease are not being identified. The authors propose universal screening of children between 1 and 9 years of age, a strategy likely to be most effective in terms of sensitivity and specificity for the identification of children with familial hypercholesterolaemia. However, this concept has yet to be proven in clinical practice.     

Selective screening for neonatal galactosemia: an alternative approach

No universal consensus exists for population-based neonatal screening for galactosemia. In our institution, selective screening for classical galactosemia is carried out on infants under 2 wk of age and those with symptoms suggestive of this disorder. Eighteen cases were diagnosed from 25,099 tests done; 17 were symptomatic at the time of diagnosis. CONCLUSION: We suggest that improved clinical vigilance and selective screening would identify most infants with severe galactosemia as early as a population-based program.     

Congenital dislocation of the hip: a prospective study comparing ultrasound and clinical examination

Screening for congenital dislocation of the hip by standard clinical methods and ultrasound was compared prospectively in 1503 newborns (1291 girls and 212 boys). A total of 82.8% of the hips (78.8% of the infants) had well developed acetabulae, 14.5% (17.2% of the infants) were shallow (immature) and 2.7% (4.1% of the infants) were dysplastic. Within 1-3 months 96.7% of the infants with clinically stable, but immature hips normalized, while 3.3% did not improve or worsened, and therapy was therefore initiated. Indications for treatment included dysplastic and/or clinically unstable hips, and a higher number of newborns were treated during the study period than in 1982-85 (31.2 vs 19.7 per 1000, p = 0.0002). Thirty-seven percent of the patients had both clinical and ultrasound findings compatible with congenital dislocation of the hip, while the decision to treat was based on clinical findings alone in 25.0% and on ultrasound findings in 38.0%. Infants born during the study period of nine months had a low incidence of late congenital dislocation of the hip compared with our earlier reported results from 1982-85 (0.9 vs 3.5 per 1000, p = 0.012). The study demonstrated major discrepancies between clinical and ultrasound evaluation of hips in the newborn, and the role of ultrasound in the screening for congenital dislocation of the hip requires further evaluation.     

Outcomes for children with gallbladder abnormalities and sickle cell disease

OBJECTIVE: To determine whether elective cholecystectomy is justifiable in children with sickle cell disease (SCD), gallbladder abnormalities, and minimal clinical symptoms. STUDY DESIGN: A retrospective review comparing clinical presentations and abdominal ultrasound results with outcomes in 146 children with SCD. RESULTS: Ultrasound examination showed sludge or stones in 83 of 146 children (57%). This was found during a diagnostic ultrasound in 59 patients (71%) and during a screening ultrasound in 24 asymptomatic patients (29%). Fifty-four (65%) children with a positive ultrasound underwent cholecystectomy; 13 of these were initially asymptomatic patients who had subsequent development of clinical symptoms. Of the patients with cholecystectomy, 93% had histopathologic evidence of cholecystitis. Perioperative complications were rare, and there were no episodes of postoperative acute chest syndrome. Children who underwent elective surgery had an average 12-days-shorter overall hospital stay than those who underwent emergent surgery (4 vs 16 days, P <.001). CONCLUSIONS: Elective laparoscopic cholecystectomy may be safely performed in children with SCD. Surgery should be strongly considered at the time of gallstone diagnosis before symptoms or complications develop. Histopathologic chronic cholecystitis does not correlate with clinical symptoms.     

PROGNOSIS OF NEONATES WITH SYMPTOMATIC RESPIRATORY INSUFFICIENCY SURVIVING WITH THE AID OF VENTILATOR THERAPY

ABSTRACT. Twenty-one survivors representing all survivors of neonatal symptomatic respiratory distress treated with intermittent positive pressure ventilation have been followed-up at an age of 2.9 to 7.0 years. The chance of survival proved to be relatively favourable in infants ventilated for perinatal pneumonias and unfavourable in infants ventilated for haemorrhagic diseases and respiratory insufficiency secondary to surgical conditions. At the follow-up half of the children presented with neurological symptoms but only 10% were found severely handicapped. One infant had a tracheostomy due to a laryngeal stenosis, while none developed broncho-pulmonary dysplasia. The late prognosis seemed unfavourable when the children had suffered from severe birth asphyxia and in infants ventilated for prolonged recurrent apnoeic spells. The relation between the clinical indications for ventilator therapy and later outcome is obscured however, by a vast number of complicating perinatal events.     

Risk assessment of renal cortical scarring with urinary tract infection by clinical features and ultrasonography.

AIMS: To address some of the issues in the ongoing debate over the optimal diagnostic imaging following childhood urinary tract infection (UTI), by determining the risk of missing renal cortical scarring which would be detected on a technetium-99m dimercaptosuccinic acid (DMSA) gold standard if ultrasound alone were used, factoring for clinical features (upper or lower tract), UTI recurrence, and age group (infants, preschool, or school age). METHODS: Details of UTI clinical features and recurrence were recorded for 990 children with a proven UTI, and their DMSA and ultrasound results were compared for each kidney. RESULTS: The risks of missing DMSA scarring varied between 0.4% (school age children with solitary lower tract UTI) and 11.1% (infants with recurrent upper tract UTI). CONCLUSIONS: UTI clinical features are important in assessing the need for DMSA imaging. Current UK imaging guidelines are endorsed, although preschool children with solitary lower tract UTI remain a controversial group and more attention needs to focused on children with recurrent UTI.     

Outcomes of neonatal screening for hearing loss by otoacoustic emission.

AIM: To assess universal neonatal screening for bilateral hearing impairments averaging 40 dBHL or worse in the better ear, using transient evoked otoacoustic emission screening (TEOAE) testing. METHODS: A three year cohort (14,353 infants born from January 1992 to 1995) was screened and subsequently followed up by hearing surveillance methods, including a distraction test screen from 7 months of age. The entire cohort was used to evaluate the outcome of the screen. A subcohort of 8172 district residents was used to evaluate the continuing worth of the distraction test programme. RESULTS: Nineteen infants (1.3/1000) with a targeted hearing impairment failed the neonatal TEOAE test. Six profoundly deaf infants identified by the TEOAE screen, were fitted with hearing aids at a median age of 16 weeks. One remained without an aid. Of 12 infants with a moderate impairment, only seven accepted hearing aid fitting and the median age of being fitted with an aid increased to 42 weeks. By the time of the analysis 22 children with a targeted hearing impairment (1.5/1000) had been identified from the cohort. Of the three missed neonatally, one was cared for elsewhere, another had a progressive loss, and the third had central deafness. Twenty children were ascertained with a congenital peripheral deafness. Of these, eight (40%) had risk factors identifiable neonatally. Only the child with central deafness was missed by TEOAE screening and subsequently identified by behavioural tests in infancy. The TEOAE screen outperformed the distraction test in terms of processes and yield and was 25% less expensive. CONCLUSIONS: The analysis confirmed the worth within the district of the TEOAE hearing screen. It will thus be continued as a universal neonatal screen with the distraction test being retained as a selective screen in the latter half of infancy.     

A national survey of screening for congenital dislocation of the hip.

OBJECTIVE: To identify current screening and management practices for congenital dislocation of the hip (CDH), and determine the extent to which ultrasound imaging of the hips is practised throughout the United Kingdom and the Irish Republic. METHODS: Postal questionnaire to paediatricians responsible for the routine neonatal care of infants in all maternity units in the UK and the Irish Republic. RESULTS: Questionnaires were returned for 254 maternity units (92% response rate). By 1994, 69% of maternity units had access to ultrasound imaging of the hips, compared to 14% in 1984. Ultrasound imaging of the hip was not used for universal primary screening, but in 93% of units was undertaken for further assessment of infants with clinically detected hip instability or those identified as being at high risk of CDH, or both. Clinical screening of newborn infants was performed by junior paediatricians, but training with a ''Baby Hippy'' hip simulator model was provided in only 37% of units. Treatment of clinically detected hip instability, initiated by an orthopaedic surgeon in 93% of units, varied widely in type and duration. CONCLUSIONS: Ultrasound imaging of the hip is increasingly used in the UK for secondary, rather than primary, screening. Current recommendations are implemented to a variable extent nationally, and the existing wide variation in screening and management for CDH reflects a lack of research evidence to support current screening practices. The effectiveness of screening for CDH needs to be established.     

Developmental dysplasia of the hip. A population-based comparison of ultrasound and clinical findings

Clinical and ultrasound findings were compared in 3613 newborns examined for developmental dysplasia of the hip (DDH) within 48 hours of delivery. Clinical and sonographic hip stability was described as stable, borderline unstable, dislocatable or dislocated, and the morphology on ultrasound as normal, immature or dysplastic. Persistent clinical or sonographic dislocatability or dislocation. major dysplasia or minor dysplasia combined with an unstable femoral head were indications for early treatment. A total of 123 (3.4%) infants were subjected to early treatment. of which 55 (45%) fulfilled the criteria for treatment on both clinical and ultrasound examinations, 52 (42%) were treated on the basis of ultrasound findings alone, and 16 (13%) on the basis of clinical findings alone. Thirty percent of the infants with clinically dislocated or dislocatable hips were judged to have stable or just borderline unstable hips on the first clinical examination. Of 486 (13.5%) infants with sonographically immature or minor dysplastic but stable hips, 472 (97%) normalized spontaneously, while treatment was initiated in 14 (3%) of them at 1-3 months of age because of lack of sonographic improvement. Only one infant presented with late DDH during an observation period of 3 years. Accepting sonographic dysplasia as a criterion for early splinting may result in a treatment rate which is almost twice the rate based on clinical criteria, but late dislocation may be virtually eliminated.     

Is screening ultrasound of the urinary tract indicated in infants with hypertrophic pyloric stenosis?

Aim: Few retrospective studies have evaluated infants with hypertrophic pyloric stenosis (HPS) for associated urological anomalies. They have led to contradictory conclusions. The aim of this study was to evaluate the incidence of urinary tract anomalies in infants with HPS and to establish the clinical significance of this association. Methods: One hundred and twenty-two infants (100 boys) who underwent pyloromyotomy between 1992 and 2002 were prospectively evaluated. Screening ultrasound (Us) of the urinary tract was performed in 107 infants, while 15 did not attend their ultrasound appointment. Results: Renal ultrasound was abnormal in 4 (4%) of 107 screened patients with HPS. Three patients were found to have mild hydronephrosis and, in one patient, a small, normal kidney was detected. Two patients with hydronephrosis had Us follow-up and the third patient underwent Tc-99 mercaptoacetyl triglycine (MAG 3) scan. In all three patients, the hydronephrosis resolved completely on follow-up scan.

Conclusion: The incidence of abnormal renal ultrasound in children with HPS is similar to the reported incidence of 3-6% determined with routine ultrasound screening of healthy newborns. The abnormalities detected were not clinically relevant and did not require surgical intervention. We do not recommend screening of the urinary tract in infants with HPS.