PEDIATRICS Vol. 120 No. 5 November 2007, pp. 1044-1052 (doi:10.1542/10.1542/peds.2007-0159)
ARTICLE |
Health Status and Health-Related Quality of Life Preference-Based Outcomes of Children Who Are Aged 7 to 9 Years and Have Bilateral Permanent Childhood Hearing Impairment
a National Perinatal Epidemiology Unit and Health Economics Research Centre, University of Oxford, Oxford, United Kingdom
b Department of Child Health, University of Southampton, Southampton, United Kingdom
c Institute of Child Health, University College London, London, United Kingdom
d Department of Audiology, Whipps Cross University Hospital, London, United Kingdom
| ABSTRACT |
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OBJECTIVE. The objective of this study was to describe the health status and health-related quality of life preference-based outcomes of children with diagnosed bilateral permanent childhood hearing impairment and a comparison group of English-speaking children with normal hearing.
METHODS. We studied 120 children who were aged 7 to 9 years and had bilateral permanent childhood hearing impairment of moderate or greater severity, identified from a cohort of 156733 children who were born in 8 districts of southern England, and 63 English-speaking children with normal hearing and the same place of birth and age at assessment. Principal caregivers were interviewed by using the Health Utilities Index Mark III questionnaire for proxy-assessed usual health status assessment. Levels of function within each of the 8 attributes of the Health Utilities Index Mark III (cognition, vision, hearing, speech, ambulation, dexterity, emotion, and pain) were recorded.
RESULTS. Bilateral permanent childhood hearing impairment is associated with significantly increased proportions of suboptimal levels of function and significantly lower single-attribute utility scores in 6 of the 8 attributes of the Health Utilities Index Mark III: vision, hearing, speech, ambulation, dexterity, and cognition. Compared with the children with normal hearing, the mean multiattribute utility score for the children with hearing impairment was significantly lower for both the whole group and the moderate, severe, and profound severity subgroups. The differences in the distributions of the multiattribute utility scores between the children with hearing impairment as a group and the children with normal hearing and between each of the severity subgroups and the children with normal hearing all were statistically significant.
CONCLUSIONS. This study provides rigorous evidence of an association between bilateral permanent childhood hearing impairment and diminished health status and health-related quality of life preference-based outcomes during midchildhood.
Key Words: hearing impairment health status health-related quality of life preferences utility scores
Abbreviations: EQ-5D—EuroQol 5-dimension instrument 16D—16-dimension measure of health-related quality of life 17D—17-dimension measure of health-related quality of life SF-6D—short-form 6-dimension measure of health-related quality of life HL—hearing level UNS—universal newborn screening
Bilateral permanent childhood hearing impairment of moderate or greater severity in the early years affects approximately 1 in 750 children, and >80% is present at birth.1–3 The consequences of the condition may include lifelong impairment in language skills and possible delays in social development and academic achievement.2–5 The severity of the disability that is experienced by the child is influenced by the degree and duration of hearing loss, the age at which the hearing loss first appears, and the hearing frequencies affected.6 The severity of disability is also influenced by coexisting disabilities that the child has, such as visual impairment, learning difficulties, and cerebral palsy.6 Developmental delays are apparent for children with severe and profound hearing, as well as for those who have moderate and less severe impairment.7–9
Most studies that describe the long-term outcomes of children with hearing impairment focus on speech and language impairment and fail to capture all functional, neurodevelopmental, and behavioral outcomes that might be of interest.2 In recent years, a number of investigators have recognized the importance of measuring the impact of childhood conditions across multiple domains. Instruments that can be used to measure the multiple health impacts of bilateral permanent childhood hearing impairment include multidimensional health profiles, which measure aspects of physical, mental, and social well-being across several domains. Wake et al10 used a multidimensional health profile, the Child Health Questionnaire, to measure parent-reported health-related quality of life in 7- to 8-year-old children with congenital hearing loss. Another group of instruments that can be used to measure the multiple health impacts of bilateral permanent childhood hearing impairment are multiattribute utility measures, which are health status classification systems with preexisting preference weights that can be attached to each permutation of responses. A particular advantage of multiattribute utility measures is that they generate composite utility scores that reflect population preferences for the overall health state that is being measured and can be incorporated into cost–utility analyses of screening, diagnostic, preventive, or therapeutic interventions.
The multiattribute utility measures that have been developed include the Quality of Well-being Scale,11 Rosser-Kind Classification of Illness States,12 Health Utilities Index,13 EQ-5D,14 16D,15 17D,16 Assessment of Quality of Life instrument,17 and SF-6D.18 The Health Utilities Index is the most widely used of the multiattribute utility measures within the childhood context. Three studies have used the Health Utilities Index as the basis for estimating preference-based health outcomes in economic evaluations of pediatric cochlear implantation.19–21 The objective of this study was to use the Health Utilities Index to describe the health status and health-related quality of life preference-based outcomes of children who were aged 7 to 9 years and had diagnosed bilateral permanent childhood hearing impairment of moderate or greater severity and a comparison group of English-speaking children with normal hearing.
| METHODS |
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Study Background and Sample
This study was undertaken as part of a larger program of research, the Hearing Outcomes Project, which focused on the speech and oral language abilities of children with bilateral permanent childhood hearing impairment.22 The study sample included all children with bilateral permanent childhood hearing impairment of at least 40 dB hearing level (HL) identified from a cohort of 156733 children who were born in 8 districts of southern England: Southampton, Portsmouth, Swindon, Bath, Waltham Forest, Hillingdon, Redbridge, and Brent and Harrow. The districts broadly reflected the nation's socioeconomic characteristics with unemployment rates in 2001 in Southampton, Portsmouth, Swindon, Bath, Waltham Forest, Hillingdon, Redbridge, and Brent and Harrow of 2.88%, 3.09%, 2.46%, 1.98%, 4.92%, 2.73%, 3.64%, and 4.14%, respectively, compared with a national average of 3.35%.23 Similarly, the districts broadly reflected the nation's underlying health characteristics with the proportion of the population with a limiting long-term illness in Southampton, Portsmouth, Swindon, Bath, Waltham Forest, Hillingdon, Redbridge, and Brent and Harrow estimated at 17.43%, 17.44%, 15.26%, 15.85%, 16.54%, 14.88%, 16.30%, and 15.28%,respectively, compared with a national average of 17.93%.23 The severity of hearing impairment, age at confirmation, and age at institution of management of cases of bilateral permanent childhood hearing impairment were obtained by review of the case records of pediatric audiologists, audiology scientists, family practitioners, speech and language therapists, and other professionals involved in each district. Children with a known postnatal cause of bilateral permanent childhood hearing impairment (eg, bacterial meningitis) were not included in the study sample. Four of the districts in which children were identified (Southampton, Portsmouth, Swindon, and Bath) are in the Wessex region and provided the sample for the years 1993 to 1996 for the Wessex controlled trial of universal newborn screening (UNS) for permanent childhood hearing impairment.24, 25 The other 4 districts in which children were identified are in the Greater London region. During the period 1992 to 1997, 2 of these districts (Waltham Forest and Hillingdon) had operated a UNS program and their 2 neighboring districts (Redbridge, and Brent and Harrow) had not operated a UNS program. Both of the London districts that were operating UNS programs had reported on the impact of UNS on early diagnosis of permanent childhood hearing impairment and collected data prospectively in this birth cohort.26, 27 In the case of Waltham Forest, this had been a prospective comparison with the Redbridge district. Thus, approximately one half of the entire birth cohort of this study had been in a target population for UNS for permanent childhood hearing impairment with >90% of the sample included from birth in prospective studies of permanent childhood hearing impairment.
At the age of 7 to 9 years, the study children were traced; the principal caregiver was then contacted by mail, regardless of the current place of residence, and permission was requested to participate in this study. When consent was provided, 2 researchers who were unaware of the child's early hearing or audiologic history arranged a time convenient for a visit to the child's home. One researcher interviewed the principal caregiver, who was usually the mother, which included completion of a battery of research instruments. The child was simultaneously assessed by the other researcher in a separate room with the following: Test of Reception of Grammar28; British Picture Vocabulary Scale (receptive language)29; Renfrew Bus Story Test (expressive language)30; and Raven's Progressive Matrices Test (nonverbal abilities).31 For the purpose of comparison with a group of children without permanent bilateral permanent childhood hearing impairment, a group of English-speaking children with normal hearing were also identified and assessed using the same measures. The children with normal hearing were identified using an algorithm developed by the investigators that calculated in an incremental manner the age distribution of the children who had hearing impairment and were recruited in each district during 4-month periods. The algorithm calculated the dates of birth of the required number of children with normal hearing within each district for each 4-month period. Each district child health officer was then asked to select randomly from routine data collection systems children who were born in the target hospitals on specified dates and approach their principal caregiver for participation in the study (with a target of 4 principal caregivers to be approached for each child with hearing impairment). This process was designed to recruit a group of children who had normal hearing and similar place of birth and age at assessment to the children with hearing impairment. Full details on the tracing and recruitment procedures of the children with hearing impairment and children with normal hearing and the measures used to assess outcomes are reported elsewhere.22
Measurement of Health Status and Health-Related Quality of Life Preference-Based Outcomes
As part of the home visit, the principal caregiver was interviewed about the health status and health-related quality of life of the child using the Health Utilities Index. The principal caregiver was considered the appropriate subject for the task because pilot research had indicated that the comprehension level for the Health Utilities Index is somewhat high for a pediatric sample in which a number of children may have developmental disabilities.
The Health Utilities Index is a family of preference-based, multiattribute utility measures.13 The principal caregiver was interviewed using the unedited 15-item questionnaire for proxy-assessed usual health status assessment, which was obtained from the Health Utilities Index developers and covers both Mark II and Mark III health status classification systems. The "usual" health focus of the questions has previously been applied in population health surveys, in which short-term illnesses such as the flu are not the major concern.32 The Mark III classification system is now recommended by the developers because of its broad applicability in both clinical and general population health studies, improvements in a number of definitions, and an increased orthogonality of its attributes for structural independence.32 The Health Utilities Index Mark III health status classification system covers 8 attributes: cognition, vision, hearing, speech, ambulation, dexterity, emotion, and pain. Function within each attribute is graded on a 5- or 6-point scale corresponding to level of severity, ranging from normal function (level 1) to severe impairment (level 5 or 6). Responses to the Health Utilities Index Mark III health status classification system were converted into single-attribute and multiplicative multiattribute utility scores using published utility functions.33, 34 Single-attribute utility scores for the Health Utilities Index Mark III are defined on a scale from 0.00 (reflecting the preference score for the lowest level of function for the attribute) to 1.00 (reflecting the preference score for the highest level of function for the attribute). Multiattribute utility scores for the Health Utilities Index Mark III, in contrast, are based on the permutation of responses across the 8 attributes and are expressed on an interval scale ranging from –0.36 (representing the health state with the lowest level of function for all attributes) to 1.00 (representing the health state with the highest level of function for all attributes). The multiattribute utility scoring algorithm for the Health Utilities Index Mark III can be summarized as u* = 1.371(b1x b2x b3x b4x b5x b6x b7x b8) – 0.371, where u* is the utility score for the overall health state being measured and the bj's are substituted from a table of coefficients provided by the Health Utilities Index developers for the appropriate attribute and level.34 For development of the single-attribute and multiplicative multiattribute utility scoring algorithms, a random sample of 504 general population adults living in the city of Hamilton, Ontario, Canada, had previously been asked to value selected health states using both a visual analog scaling technique and a standard gamble instrument. Additional details on the utility algorithms for the Health Utilities Index Mark III are reported elsewhere.33, 34
Ethics Approval
The South and West Multi-center Research Ethics Committee, England, approved this study, and participating principal caregivers provided written informed consent.
Statistical Analysis
A detailed statistical analysis plan was used to compare the health status and health-related quality of life preference-based outcomes of the children with bilateral permanent childhood hearing impairment and the children with normal hearing. The children with bilateral permanent childhood hearing impairment were stratified according to severity of hearing loss on the basis of their most recent audiologic records. The levels of severity comprised moderate (40–69 dB HL), severe (70–94 dB HL), and profound (
95 dB HL) hearing impairment according to 4-frequency averaging of the pure-tone thresholds from 500 to 2000 Hz (or, if pure-tone thresholds were unavailable, sound fields and electrophysiologic test results).
Differences in baseline characteristics between the children with hearing impairment and the children with normal hearing were tested using the Pearson
2 test. For each of the 8 attributes of the Health Utilities Index Mark III, we compared the proportion of children with suboptimal levels of function (defined as below level 1 function) using Fisher's exact test for equality of proportions. Differences in the single-attribute and multiattribute utility scores were tested using 2-sample t tests for unequal variance. Differences in the distributions of the multiattribute utility scores were tested using the nonparametric 2-sample Kolmogorov-Smirnov test. Finally, we performed a Tobit regression to explore the effects of severity of hearing impairment (none, moderate, severe, and profound) on the Health Utilities Index Mark III utility score. Tobit regression was required to account for the censoring of the dependent variable, the utility score, which has an upper value of 1.0. Other covariates included in the model were experience of other significant medical condition and disorders, such as cerebral palsy (no and yes), and social class of the head of household (higher occupations, intermediate occupations, lower occupations, never worked, and long-term unemployed). Because many comparisons were made, we used a conservative P value threshold of <.01 as a qualitative indicator of statistical significance. Statistical analyses were conducted by using Stata 9.0 (Stata Corp, College Station, TX).
| RESULTS |
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A total of 168 children with bilateral permanent childhood hearing impairment and without a known postnatal cause of the impairment were identified among the 156733 children born between 1993 and 1996 in the 4 districts in the Wessex region or between 1992 and 1997 in the 4 districts in the Greater London region. Of these 168 children, 6 were not approached because this was deemed inappropriate by their audiologist as a result of acute problems with their health at the time of recruitment, and 2 could not be traced. Of the remaining 160 children, 120 (75%; 67 boys and 53 girls) of mean age 7.9 years (range: 5.4–11.7 years) and their principal caregivers agreed to participate. Fifteen (9%) children and their principal caregivers refused study participation, and 25 (16%) children and their principal caregivers did not respond to repeated study requests. No statistically significant differences were found between participants and nonparticipants with respect to age at follow-up, gender, and severity of hearing loss. A group of 63 (19.4%) of the 325 approached children who had normal hearing (37 boys and 26 girls) and a mean age of 8.1 years (range: 6.3–9.8 years) and their principal caregivers also agreed to participate.
No statistically significant differences were identified in the distributions of age at follow-up, gender, mother's highest educational qualification, social class of the head of household, or use of English as the first language at home between the children with bilateral permanent childhood hearing impairment and the children with normal hearing (Table 1). Of the 120 study children with bilateral permanent childhood hearing impairment, 65 (54.2%) had moderate hearing impairment, whereas 29 (24.2%) and 26 (21.7%) had severe and profound hearing impairment, respectively. Bilateral permanent childhood hearing impairment had been confirmed by 9 completed months of age in 57 (47.5%) children, between 10 and 18 completed months of age in 27 (22.5%) children, and at a later point in 36 (30.0%) children. Eighty-six (71.6%) of the 120 study children with bilateral permanent childhood hearing impairment used oral communication, 16 (13.3%) used signing, 11 (9.2%) used a combination of oral and signing, and 7 (5.8%) used nonverbal communication and gestures. Thirteen (10.8%) of the 120 study children with bilateral permanent childhood hearing impairment had cochlear implants, 4 (3.3%), 1 (0.8%), and 8 (6.7%) of whom had moderate, severe, and profound hearing impairment, respectively. Cerebral palsy, learning disabilities, and asthma were identified in 5 (4.2%), 8 (6.7%), and 12 (10.0%) of the children with bilateral permanent childhood hearing impairment, respectively. The frequencies (proportions) of these diagnosed conditions in the children with normal hearing were 0 (0%), 0 (0%), and 5 (7.9%), respectively.
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Comparisons of the frequency and proportion of suboptimal levels of function between the children with bilateral permanent childhood hearing impairment and the children with normal hearing are shown in Table 2 for each of the 8 attributes of the Health Utilities Index Mark III. In 6 of the 8 attributes (vision, hearing, speech, ambulation, dexterity, and cognition), there were significantly higher proportions of suboptimal levels of function among children with bilateral permanent childhood hearing impairment (P
.01); however, there were no statistically significant differences in the proportions of suboptimal levels of function in the emotion (P = .539) and pain (P = .832) attributes between the 2 groups of children.
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When compared with the children with normal hearing, the single-attribute utility scores were significantly lower for the children with hearing impairment as a group in 6 of the 8 attributes of the Health Utilities Index Mark III (vision, hearing, speech, ambulation, dexterity, and cognition; P < .01; Table 3). This was also true for the moderate, severe, and profound severity subgroups for the hearing and speech attributes (P < .01). In addition, among the children with severe hearing impairment, the single-attribute utility score was significantly lower for the cognition attribute when compared with the children with normal hearing (P < .01).
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Table 4 presents descriptive statistics for the multiattribute utility scores for the comparison groups. These multiattribute utility scores summarize population preferences for the overall health state of the child across the 8 attributes of the Health Utilities Index Mark III. The mean multiattribute utility score for the children with hearing impairment as a cohort was 0.626, compared with 0.920 for the children with normal hearing, a mean difference in utility score of 0.294 that was statistically significant (P < .001). The mean multiattribute utility scores were also significantly lower in each of the 3 severity subgroups than in the group with normal hearing (P < .001).
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Table 5 presents the distributions of multiattribute utility scores for the children with hearing impairment and children with normal hearing across the utility scale of the Health Utilities Index Mark III. The differences in the distributions of the multiattribute utility scores between the children with hearing impairment as a group and the children with normal hearing and between each of the severity subgroups and the children with normal hearing all were statistically significant (P < .001).
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Finally, the Tobit regression revealed that after controlling for other significant medical conditions and disorders and social class of the head of household, the mean multiattribute utility score for children with moderate, severe, and profound hearing impairment was 0.283, 0.477, and 0.411 less, respectively, than that for children with normal hearing (P < .0001; Table 6).
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| DISCUSSION |
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This study revealed that bilateral permanent childhood hearing impairment is associated with significantly increased proportions of suboptimal levels of function and significantly lower single-attribute utility scores in 6 of the 8 attributes of the Health Utilities Index Mark III: vision, hearing, speech, ambulation, dexterity, and cognition. In addition, bilateral permanent childhood hearing impairment is associated with a 0.294 reduction in the mean multiattribute utility score, a difference that is not only statistically significant but also far exceeds the 0.030 minimally important difference in utility score postulated in the literature as clinically important for evaluative purposes.35, 36
A multiple Tobit regression that was conducted as part of the analytical strategy revealed that even after controlling for the presence of other significant medical conditions and disorders, children with bilateral permanent childhood hearing impairment had lower multiattribute utility scores, on average, than children with normal hearing. Although some evidence exists to suggest that the impact of significantly reduced auditory input on adverse health outcomes might be mediated through increased risk for isolation, social phobias, and impulsivity,37 additional research is required to elucidate the complex biopsychosocial pathways that are likely to be involved. A surprising aspect of our results is that the decrement (reduction) in the mean multiattribute utility score associated with severe hearing impairment is higher than that associated with profound hearing impairment (0.477 vs 0.411). This finding reflects differences in resource use and costs and multidimensional health profile measures between the severity subgroups reported elsewhere10, 38 and also merits additional research.
We are aware of only 1 other study in the published literature that estimated levels of health utility for children with bilateral permanent childhood hearing impairment of moderate or greater severity.21 As with our study, the principal caregivers were interviewed about the health status and health-related quality of life of the child using the Health Utilities Index Mark III. The distributions of the multiattribute utility scores for the moderate, severe, and profound severity groups were broadly consistent with the descriptive statistics presented in this article. Barton et al,21 however, failed to control for confounding factors in their analyses and, consequently, were unable to make any firm assertions about the probable bounds of the effects of bilateral permanent childhood hearing impairment on the utility scores. A number of studies have applied multiple regression methods to general population data from the United States to estimate the marginal disutility of chronic conditions in adults.39, 40 The marginal disutility of the vast majority of chronic conditions was found to be smaller than that for the hearing impairment states revealed by our multiple Tobit regression, suggesting that our results are likely to be of relevance to clinical and policy decision makers.
There are a number of strengths to this study. First, it is based on a cohort of children drawn from defined geographic areas rather than clinic-based populations; consequently, selection biases are unlikely to represent a major problem. Second, children were recruited from 8 districts of southern England that broadly reflect the nation's socioeconomic and underlying health characteristics; therefore, the study is likely to have high external validity.23 Third, the study used validated and reliable approaches to measure the health status, health-related quality of life preference-based outcomes, and severity of hearing impairment of the study children. Fourth, the analysis used a geographic- and age-based comparison group that was specifically recruited for this study rather than data from siblings, which are prone to biases as a result of continuously changing developmental profiles throughout childhood, or comparisons with British population norms for which limited data are available.
There are 3 broad caveats to the study findings, which should be borne in mind by readers. First, despite a rigorous process designed to recruit an appropriately sized comparison group, only 19.4% of the children with normal hearing and their principal caregivers who were approached agreed to participate in the study. However, in no case did a
2 analysis of the background sociodemographic variables in Table 1 show a significant difference between the children with hearing impairment and children with normal hearing. Consequently, we are confident that the low response rate did not systematically bias our results. Second, each child's health status and health-related quality of life were assessed by the principal caregiver rather than the child himself or herself. At the outset of the study, it was considered on the basis of preliminary research that the comprehension level required for the Health Utilities Index Mark III is somewhat high for a pediatric sample in which a number of children may have developmental disabilities. Empirical evidence of the concordance between child and parent ratings of health status suggests that parents are able to rate accurately observable behaviors, such as physical functioning and physical symptoms, but are less successful at identifying social or emotional impairments41; however, there is no consistent evidence to suggest that parents or other principal caregivers consistently either underreport or overreport these impairments,42 which suggests that there are unlikely to be systematic biases in the reporting of functional impairments. Third, although the Health Utilities Index is the most widely used of the multiattribute utility measures within the childhood context, the underlying preference weights for the Mark III health status classification system were derived from a survey of Canadian adults. Recent research suggests that our approach of indirectly estimating preference-based outcomes by attaching population-derived utility scores to Health Utilities Index Mark III health states may be a poor substitute for directly measured utility scores43; however, the cognitive requirements entailed in directly estimating utility scores for health states using techniques such as the visual analog, standard gamble, and time trade-off approaches precluded a direct measurement approach among our pediatric sample.41 Moreover, evidence from empirical studies suggests that our approach of indirectly estimating preference-based outcomes does not result in systematic biases in group differences in utility scores.43
| CONCLUSIONS |
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This study has revealed an association between bilateral permanent childhood hearing impairment and diminished health status and health-related quality of life preference-based outcomes in midchildhood. The data generated by the study provide a basis for informing service provision and for evaluating the outcomes of preventive and treatment interventions in this area. In addition, the preference-based outcomes reported in this study can be synthesized with economic data to generate cost-effectiveness data using a composite quality-adjusted life-year outcome metric. Their use in follow-up studies in midchildhood, however, is not yet routine, and more methodologic work is required, particularly with regard to eliciting descriptions and underlying preferences for the health states experienced from the children themselves.
| ACKNOWLEDGMENTS |
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The Hearing Outcomes Project was supported by a grant from the Wellcome Trust (reference No. 061839). Dr Petrou is supported by a United Kingdom Medical Research Council senior nonclinical research fellowship. The National Perinatal Epidemiology Unit is funded by the Department of Health, England.
We gratefully acknowledge the help and assistance received from children, families, school staff, specialist teachers of the hearing impaired, speech and language pathologists, and audiology professionals in the local area teams.
| FOOTNOTES |
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Accepted May 25, 2007.
Address correspondence to Stavros Petrou, PhD, National Perinatal Epidemiology Unit, University of Oxford (Old Road Campus), Headington, Oxford OX3 7LF, United Kingdom. E-mail: stavros.petrou{at}npeu.ox.ac.uk
The views contained in this article are those of the authors and not necessarily the funding bodies.
The authors have indicated they have no financial relationships relevant to this article to disclose.
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