Journal of Pediatric Psychology Advance Access published online on July 22, 2008
Journal of Pediatric Psychology, doi:10.1093/jpepsy/jsn067
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Psychosocial Adjustment in Males with Duchenne Muscular Dystrophy: Psychometric Properties and Clinical Utility of a Parent-report Questionnaire
1Kempenhaeghe Epilepsy Centre, Heeze, 2Franciscusoord, Childhood Rehabilitation Centre, SRL, Valkenburg, 3Department of Neurology, University Hospital Maastricht, 4Baylor College of Medicine, Texas Children's Hospital and 5Department of Psychology, Maastricht University
All correspondence concerning this article should be addressed to Jos G. M. Hendriksen, Kempenhaeghe Epilepsy Centre, Department of Behavioral Sciences, P.O. Box 61, 5590 AB Heeze, The Netherlands. E-mail: hendriksenj{at}kempenhaeghe.nl
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Abstract |
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Objective The primary aim of this study was to establish the psychometric properties and clinical utility of the Personal Adjustment and Role Skills Scale (PARS-III) for assessing psychosocial adjustment in males with Duchenne muscular dystrophy (DMD). Methods The parents of 287 male patients with DMD aged 5–18 years completed the PARS-III and Revised Rutter Scale. Results The
coefficients and factor analysis indicated good reliability and validity. Overall psychosocial adjustment was not significantly different in DMD compared to males with other chronic medical conditions and was positively associated with increases in age. A clinical cutoff score for screening in the DMD population is also reported. Conclusions The PARS-III is a reliable and valid index of youth psychosocial adjustment in DMD and can be used for both clinical screening and research purposes. Key words: Duchenne muscular dystrophy; psychosocial adjustment; questionnaire study.
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder occurring in approximately 1 in 3,500 live male births (Anderson, Head, Rae, & Morley, 2002
). Mutations in the dystrophin gene result in the absence of dystrophin or a dysfunctional dystrophin protein, thereby compromising the structural integrity of skeletal and cardiac muscle cells. DMD is characterized by progressive muscle weakness and subsequent fatigue, respiratory difficulties and cardiac complications. The typical age of diagnosis for DMD is 2–7 years (Emery & Muntoni, 2003), and most males become wheelchair dependent by the age of 12 years. During adolescence ventilation is usually necessary and they eventually become totally dependent upon caregivers for basic self-care. Death typically occurs by the early twenties, most commonly caused by respiratory or cardiac complications (Emery & Muntoni, 2003).
Research on DMD has understandably been focused primarily on muscle degeneration and its treatment. However, DMD is complex and examination of additional factors is necessary for a holistic understanding of the disorder and the implications for patient outcome. For example, emerging evidence indicates that the absence of dystrophin results in disordered architecture of the central nervous system (Anderson et al., 2002), suggesting that DMD should not only be viewed merely as a "muscle disorder," but also as a disorder affecting the brain. Studies documenting the higher prevalence of cognitive and learning problems (Cotton, Voudouris, & Greenwood, 2001; Hendriksen & Vles, 2006; Hinton, Fee, Goldstein, & De Vivo, 2007; Hinton & Goldstein, 2007) and neuropsychiatric disorders in boys with DMD (Hendriksen & Vles, 2008; Stein, Pachter, Schwartz, & Taras, 2004; Wu, Kuban, Allred, Shapiro, & Darras, 2005) support this view, and can be factors that impose additional stress on patients and their families. Furthermore, advances in medical care have prolonged life expectancy, resulting in a new population of adults with DMD who have unique psychosocial issues related to independence and vocational planning that are not routinely being addressed (Rahbek et al., 2005). Other factors associated with the disease, including its life limiting nature, physical restrictions, medical complications, and issues related to the provision care can contribute to the emotional burden of patients and their families (Nereo, Fee & Hinton, 2003). Taken together, these findings suggest that there are multiple factors in DMD that can have an impact on a child's ability to cope with his disorder. Despite the obvious implications of this, there are relatively few studies examining the cumulative impact these stressors can have on behavioral, emotional, and social functioning in the DMD population (Poysky, 2007).
While some studies have lumped boys with DMD together with other neuromuscular disorders (Darke, Bushby, Le Couteur, & McConachie, 2006), we found five studies that focused specifically on populations of boys with DMD. They indicated that between 30% and 50% have psychosocial problems including general emotional or behavioral disturbance (Firth, Gardner-Medwin, Hosking, & Wilkinson, 1983; Leibowitz & Dubowitz, 1981; Polakoff, Morton, Koch, & Rios, 1998), symptoms of depression (Fitzpatrick, Barry, & Garvey, 1986), and social problems (Hinton, Nereo, Fee, & Cyrulnik, 2006). However, these studies are limited in that they have neglected the control for impact of corticosteroid treatment, employed small sample sizes, did not examine the relationship between age and functioning, or utilized measures that may not have been designed for use in chronic illness populations.
There are few standardized instruments specifically developed to assess psychosocial functioning in children with a chronic illness (Harris, Canning, & Kelleher, 1996; Walker, Stein, Perrin, & Jessop, 1990). The Child Behavior Checklist—CBCL (Achenbach & Ruffle, 2000) is one of the most widely used measures for the assessment of behavior problems and domains of competence, but some have suggested that its use may not always be appropriate for children with chronic illness (Perrin, Stein, & Drotar, 1991; Walker et al., 1990). More specifically, the primary aim of the CBCL is to assist in the identification and diagnosis of psychopathology. It does not take into account the stress and emotional reactions that are a normal part of adjustment to a chronic, fatal illness (Eiser, 1990). In addition, there are items on the CBCL that may be overly sensitive to illness-related variables (in particular items related to somatic complaints). As such, sole reliance on the CBCL when assessing psychosocial functioning in DMD may result in over identification or mislabeling of normal behavior as pathological. The Strengths and Difficulties Questionnaire (Goodman, 1997), which is commonly used across Europe, has the same drawback in that it measures general behavior problems without special consideration of illness-related factors.
The Pediatric Quality of Life Inventory—PedsQL (Varni, Seid, & Kurtin, 2001) was specifically developed for children with medical conditions and is commonly used in pediatric chronic illness studies to measure aspects of health-related quality of life (HRQOL), including physical, emotional, social and school functioning. However, the PedsQL has a limited number of items assessing adjustment to chronic illness, and items are not anchored to specific behaviors, suggesting that more comprehensive assessment of psychosocial factors may be necessary in some clinical and research settings.
To address these limitations the Personal Adjustment and Role Skills Scale, 3rd edition (PARS-III) was specifically developed to measure psychosocial adjustment in children with chronic physical illnesses (Stein & Jessop, 1990). Psychosocial adjustment can be defined as the adaptive task of managing upsetting feelings and frustrations aroused by the illness, and preserving an emotional balance (Moos & Tsu, 1977). It is the outcome of a process in which children attempt to meet the demands of their illness with their emotional/social resources and coping patterns. Amongst other things, the PARS-III assesses peer relationships, independence, and participation in age-appropriate tasks; all of which appear to be particularly important domains related to the adjustment of children who have a chronic illness (Immelt, 2006; Witt, Riley, & Coiro, 2003), and which may vary in significance according to the age of the child. In addition, strength of the PARS-III is its exclusion of items based on physical symptoms, such as pain, fatigue, and headaches (Walker et al., 1990). The PARS-III has been used to assess psychosocial functioning in a variety of nonDMD chronic-illness populations with mixed results (Chernoff, Ireys, DeVet, & Kim, 2002; Harris et al., 1996; LeBovidge, Lavigne, & Miller, 2005; Witt et al., 2003). One of the shortcomings of the PARS-III is the lack of an established cutoff score. In the original study by Walker et al. (1990), a cutoff of 1 SD below the mean was proposed to identify subjects who were at an increased risk for adjustment problems as compared to their affected peers. The male participants’ mean score was 85.5; SD = 12.1 resulting in a cutoff score of 73. Based on these results, Witt and colleagues (2003) utilized a cutoff score of 73 for a different pediatric illness sample resulting in 11.1% of the children in their study being identified as having significant psychosocial adjustment problems. In another study, Chernoff et al. (2002) utilized a –1 SD procedure that was derived from their own pediatric illness sample. They reported a cutoff score of 78 with 19% falling below the cutoff point (lower scores indicate poorer adjustment). The most important issue with respect to cutoff scores therefore is the inconsistency across studies.
Taken together, these findings emphasize the need for additional research in the psychosocial adjustment of males with DMD, and the PARS-III appears to be an appropriate tool for this purpose. However, mixed findings in other populations suggest that reliability, validity, and clinical cutoff scores should be established especially for DMD prior to its adoption for clinical and research purposes.
The primary aim of the present study was to establish the psychometrics of the PARS-III for use in the DMD population. We hypothesized (Hypothesis 1) that the factor structure of the PARS-III in males with DMD would be the same as was documented in the original study by Walker et al. (1990). We also hypothesized that the PARS-III would reach acceptable levels of reliability and validity (Hypothesis 2).
Our secondary aim was to describe psychosocial adjustment in males with DMD, as this is a group that is chronically under-represented in pediatric psychology research. Because of the complex nature of DMD and the many associated stressors, we hypothesized that problems in overall psychosocial adjustment will be more common in males with DMD than in a clinical reference group (Hypothesis 3). Although it may be somewhat contrary to logic, adjustment ability has been reported to improve with age in other chronic illnesses (Eiser & Jenney, 2007; LeBlanc, Goldsmith, & Patel, 2003). As such, we hypothesized that improved overall adjustment scores would be associated with increased age in males with DMD (Hypothesis 4). However, some have recently suggested that there may be specific problem areas that appear across different age groups in DMD; increased social problems in younger boys and increased depression and anxiety in older boys (Hinton & Goldstein, 2007). We therefore hypothesized that adjustment scores for peer relations would improve with age (Hypothesis 5) and that depression/anxiety scores would become poorer with increased age (Hypothesis 6).
The third aim of our study was to establish the clinical utility of the PARS-III by identifying a cutoff score that can be used to identify patients within the DMD population who are at risk for having greater problems with adjustment.
Finally, corticosteroids are commonly used in males with DMD to prolong ambulation and maintain pulmonary functioning, but there continues to be some debate regarding the impact of this treatment on behavioral functioning (Biggar, Harris, Eliasoph & Alman, 2006; Dubowitz, 2005; Hendriksen & Vles, 2008). Given the opportunity, we decided to examine this by conducting exploratory analyses comparing psychosocial adjustment in participants who are on steroid treatment and those who are not.
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Participants
Parents of patients with DMD were recruited from members registered with two-parent advocacy groups, the Dutch and American Parent Project Muscular Dystrophy organizations. Representatives of the respective organizations contacted parents and requested their participation in the study via letter (Dutch), or e-mail (American). Parent Project Muscular Dystrophy (PPMD), founded in 1994, is a nonprofit grassroots parent organization focused entirely on Duchenne and Becker muscular dystrophies. Approval was obtained from the local ethics committee of Maastricht University, and information was provided in the letter or email that addressed requirements of adequate informed consent. Only boys with a diagnosis of DMD were included in the current study. Of the 112 Dutch parents who were contacted by a letter, 63 of them participated (56%) by completing the questionnaire. Of the 1,587 American parents contacted by e-mail, 351 of them participated (22%). The participating parents and their sons lived in areas spread throughout the Netherlands and the US. The mean age of the males was 11.9 (SD = 5.2), with a minimum age of 3 years and a maximum of 38 years. As 47 males were 18 years or older and thus in the adult age range they were excluded in further analyses. Also, the 17 boys aged 3 and 4 years were excluded from further analyses, as the PARS-III was not designed for this age group. The final sample consisted of 287 boys (mean age = 10.9; SD = 3.5). The mean age of initial DMD diagnosis was 3.8 years (SD = 2.0). Parents were asked to give their educational status (highest grade of school which they completed) on a 5-point scale ranging from "some high school or less" to "professional or graduate degree" as is done in the Child Health Questionnaire (Landgraf, Abetz, & Ware, 1999
), as an estimate of their social–economic status. Kolmogorov–Smirnov test was performed to test whether the distribution of education was normal in both the US and Dutch sample. Educational status in the US sample (D[225] = 0.21, p <.001), and the Dutch sample (D[55] = 0.23, p <.001), were both negatively skewed toward higher levels of educational attainment, suggesting that there was a response bias toward more education and higher socio-economic status in responders than would typically be expected in the general populations. The majority of questionnaires were completed by mothers: 73% in the US sample and 58% in the Dutch sample. Questionnaires were completed by both parents in 10% of the US sample and 29% of the Dutch sample; remaining questionnaires were completed by fathers: 15% in US sample and 11% in the Dutch sample. No information on race/ethnicity of the responder was collected as these variables are not routinely collected in the Netherlands. The survey was completed anonymously, and demographic information was not collected for nonresponders.
Measures
Parents were asked to complete a survey questionnaire constructed for the purpose of this study. It included the PARS-III, the Revised Rutter Scale (RRS) (Parker, Yiming, Tan, & Rutter, 2001), and other various items assessing demographics (age of patient with DMD, parental educational status) and disease parameters (e.g., use of steroids).
Psychosocial Adjustment and Role Skills Scale III (Stein & Jessop, 1990)
This instrument is a brief parent-completed index of youth psychosocial adjustment. All 28 items use a 4-point interval rating scale: "never or rarely," "sometimes," "often," or "always or almost always." Of them, 20 items are "reverse scored." Item responses are assigned 1–4 points on a likert-type scale, and the summation of all item responses results in the total score. Summation of specific items also yields six factor-derived psychosocial subscales: peer relations, dependency, hostility, productivity, anxiety/depression, and withdrawal. Higher scores indicate better adjustment (Stein & Jessop, 1990; Walker et al., 1990). In the original study, by Walker et al. conducted with 450 children with a variety of chronic medical conditions (including asthma, seizure disorders, cystic fibrosis, cerebral palsy, and endocrine disorders) the reliability (coefficient ) of the total summary score was.88 overall, with subscales ranging from.70 to.80. Construct validity of the six subscales was supported by principal component factor analysis and concurrent validity was adequate, as supported by significant correlations in the expected directions with the Child Behavior Checklist (Achenbach & Ruffle, 2000) and the Health Resources Inventory (Gesten, 1976). Because girls were reported to have slightly higher (better) scores on the total scale and four of the subscales (peer relations, dependency, hostility, and productivity), only the results of the males from their study will be used in comparison to our results, which also consists only of male subjects.
Similar to the original Walker et al. (1990) study, we elected to set the cutoff score at –1 SD below the mean of our sample. This was judged to be an adequate and understandable method of identifying males with DMD who are at risk for having adjustment problems as compared to other males with DMD.
Revised Rutter Scale (Parker et al., 2001)
This is an 8-item parent-completed measure of emotional distress in children (Parker et al., 2001). The scale has four items derived from the emotional difficulties RRS subscale and four additional items assessing lack of enjoyment, irritability, withdrawal, and poor concentration (Parker et al., 2001). Parents are required to state whether the item "does not apply," "applies somewhat," or "certainly applies" (with respective scores of 0, 1, and 2). Higher scores indicate higher levels of global emotional stress in children. Research (Parker et al., 2001) has demonstrated that the RRS has sound psychometric properties, e.g., inter-rater reliability.84.
Data Analysis
The various analyses were performed using SPSS PC (version 11.0). Descriptive statistics i.e., mean and SD were initially calculated. We used a structural equation-based confirmatory factor analysis (CFA) using LISREL version 8.30 (Jöreskog and Sörbom, 1999) to determine if the items of the PARS-III loaded on the six domains as predicted. The goodness-of-fit for the factor solution was evaluated by calculating the Comparative Fit Index (CFI) and the standardized root mean square residual (SRMR). To evaluate the goodness-of-fit, we used the combination rules recommended by Hu and Bentler (1999) suggesting a good fit if both the CFI-value is 
.95 and the SRMR is 
.09. Internal consistency reliability coefficients (Cronbach's ) were calculated for the PARS-III total score and six subscales. A coefficient of .80 was set as the minimum limit for acceptance for the total score, and a coefficient of .70 was set as the minimum level of acceptance for subscale scores (Sattler, 2001). Convergent validity of the PARS-III questionnaire was assessed by calculating correlation levels (Spearman's correlation) between the total scores on the PARS-III and the total scores of the RRS. A coefficient of –.60, with a statistical significance at p <.05, was set as the minimum level of acceptance for the total scale scores. T-tests were used to compare the overall score and subscale scores of patients with DMD to total and subscale scores for a male clinical reference group that has been previously reported (Walker et al., 1990), and to compare the overall and subscale scores of participants taking steroid medicine with those who were not. Bivariate correlations were computed to analyze the relationship between age as a continuous variable and psychosocial adjustment.
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Primary Analysis
Each item of the PARS-III questionnaire had <1.4% missing data. This means that the questionnaire was completed for a valid population number of 282 males with DMD. The results of the confirmatory factor analysis are presented in Table I. Our data proved to have a good fit as CFI was.96 and SRMR was.06. Factor loadings >.40 are presented in Table I. The item-factor correlations, which are also presented in Table I, were estimated with the use of the maximum likelihood factor analyses within the SPSS software program. The factors in our study corresponded closely with the factor structure, that was originally published for the PARS-III, thereby justifying the use of the six previously identified subscales of psychosocial adjustment. The Pearson correlations, corrected for attenuation, for the six factors are reported in Table II. Patterns of subscale intercorrelations provide data for construct validity. The correlations ranged from.18 (p =.002) to.55 (p
.001), indicating significant relationships among the subscales. In Table III, the mean scores, SD and SE for the total PARS-III score and the six subscales are described. Our descriptive statistics suggest that an appropriate cutoff score (mean – 1 SD as suggested by Pless et al., 1994 and Witt et al., 2003) would be 72.3.
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The internal consistency of the total scale was good with
at.91. The internal consistency of the subscales was acceptable with subscale 's ranging from.74 for dependency to.89 for hostility.
Convergent validity of the PARS-III was supported by the negative correlations of the PARS-III with the RRS. The Spearman correlation between RRS scores and PARS-III scores within our group of patients with DMD was significant (r's = –.65, p .001). This means that better adjustment (high PARS-III scores) was associated with lower emotional distress (low RRS scores). The scores for the six PARS-III subscales were also negatively correlated with the emotional distress scores of the RRS: ranging from –.35 (p .001) for peer relations to –.61 (p .001) for anxiety/depression.
Results of one sample t-tests comparing our results with a clinical reference group (Walker et al., 1990) are presented in Table IV. Comparison of total scores indicated no significant difference between groups. However, analyses of subscales revealed some interesting differences for the six domains of psychosocial adjustment: males with DMD were rated as having significantly worse peer relations and productivity in comparison to males with other chronic conditions, but were rated as less dependent on caregivers and as having less anxiety and depression.
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Bivariate correlations were calculated to determine the relationship between age and PARS-III scores. Results are presented in Table V. As can be seen, there was a significant positive relationship between age and total score on the PARS-III questionnaire, indicating that increases in age corresponded with increases in overall psychosocial functioning. Subscale analyses resulted in a negative correlation between the peer relations subscale score and age, indicating that poorer peer relations are associated with increased age. Dependency, hostility, and productivity scores were positively related to age, suggesting improved functioning in these areas as patient age increases. In addition, the anxiety/depression and withdrawal subscale scores were not significantly correlated with patient age.
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Exploratory Analyses
The total PARS-III scores of 161 males in our study using steroids (56%) were compared to 118 nonusers (41%). T-test indicated no significant difference between the two groups (t = 1.37; p =.17). Analyses of subscales indicated a small but significant difference (t = 2.33; p =.02) for the withdrawal subscale when boys using steroids (M = 14.6) were compared to nonusers (M = 14.0).
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Discussion |
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Our results confirm Hypotheses 1 and 2, indicating that the PARS-III is a reliable and valid measure for screening psychosocial adjustment in the DMD population. Internal consistency reliability was good overall and was somewhat stronger than what was published in the original study, possibly reflecting the increased homogeneity of our sample. Convergent validity was adequate; however, this is to be expected in a broad-based screening measure. There was strong support for good construct validity, as the six factors previously identified by Stein and Jessop (1990
) were replicated in our sample. We believe that these findings, its ease of administration and scoring, and its focus on factors that are important in chronic illnesses make the PARS-III an useful and promising clinical and research measure for screening psychosocial adjustment in boys and adolescents with DMD. However, additional cross-validation studies are needed, such as cross-cultural differences in psychosocial functioning in males with DMD. Also, further research is needed on possible contributory factors (e.g., physical impairments like wheelchair dependence, physical complaints, and intelligence) as well as outcomes factors such as school performance, behavioral functioning and family burden. This is the first study to compare the psychosocial adjustment of a large sample of patients with DMD to findings reported in males with other chronic illnesses. We were encouraged to see that patients with DMD do not appear to be at significantly greater risk for adjustment problems than boys with other types of chronic conditions, thereby confirming Hypothesis 3. It seems counter intuitive that boys with DMD are rated as less dependent than boys with other medical conditions. However, this finding may reflect the fact that boys with DMD are being rated by their parents as better able to decide things for themselves, asking less unnecessary questions, or asking for less help in things when compared to boys with other types of chronic conditions.
Our study also demonstrated that patient age is an important factor when conceptualizing psychosocial adjustment in males with DMD. In general, there was a trend for overall psychosocial adjustment to be rated higher as age increased, confirming Hypothesis 4. It is difficult to interpret this finding, as the logical assumption would be that more problems should arise as the disease progresses. However, this is consistent with what has been reported in other conditions (Eiser & Jenny, 2007; LeBlanc, Goldsmith & Patel, 2003). These results may reflect the improvement in cognitive and problem-solving skills that some have proposed to occur with increased age in boys with DMD (Cotton, Voudouris & Greenwoud, 2005) or possibly the development of effective coping strategies that occurs over time.
Despite the trend in overall improvement with age, the domain of peer relations was an exception. Contrary to what we assumed in Hypothesis 5, a decrease in functioning in peer relations was related to increased age. Although age is not a direct proxy for disease severity, the most obvious interpretation of this is that there is a decrease in access to social, recreational, and educational/vocational opportunities as physical impairment progresses and health deteriorates. Emotional factors such as anxiety and depression could also potentially contribute to peer problems; our data show a modest but significant relationship between these domains (r =.29 between the anxiety/depression and peer relations scores).
Because the PARS-III is not designed to establish psychiatric diagnosis and has not been normed on the normal population, there is the risk that the PARS-III may under-identify psychopathology (Harris et al., 1996). The lack of norms for the general population also does not allow for greater understanding of the clinical relevance and implications of individual PARS-III scores. However, the use of the clinical cutoff that was established in this study (total score <72) will hopefully allow professionals to quickly identify patients who are at risk for having adjustment problems, and who may benefit from more comprehensive evaluations (such as clinical evaluation, pathology-focused rating scales, or neuropsychological testing).
While it is well known that common side effects of corticosteroid treatment include behavioral and emotional problems (Biggar et al., 2006; Dubowitz, 2005), our results are consistent with those of Hinton et al. (2007), who found that steroids were not associated with the behavioral outcome of children in their study. Our results indicated that there was no significant difference in overall psychosocial adjustment between males with DMD in our study who were taking steroids as compared to those who were not. Although our model limits the conclusions that can be drawn from this finding, there was no indication that steroids played a major role in negatively modifying psychosocial adjustment in our sample. In contrast, subscale analyses indicated that participants who were using steroids showed less withdrawal than peers. Our cross-sectional model limits our ability to determine if this finding was possibly indicative of a beneficial causative effect (e.g., participants were less withdrawn because steroids increased mobility and reduced medical complications) or a self-selection bias effect (e.g., males with pre-existing emotional problems or withdrawal are less likely to be treated with steroids).
In general, our study has several strengths including (a) the focus is on males with DMD, which is an under-researched population, (b) the use of an international sample of participants, which enhances the generalizability of our findings, (c) the use of few exclusionary criteria, which can also enhance external validity, and (d) the recruitment of a relatively large sample given the rarity of this illness. However, there are a number of limitations to our study that should be addressed in future research. As previously mentioned, one of these limitations is the use of a cross-sectional methodology. Because our data are cross-sectional, we were limited in our ability to examine causative factors and change over time. In particular, longitudinal studies are necessary to further elucidate the relationship between age, steroid use and various areas of adjustment. Longitudinal models will also allow greater examination of other important topics such as the overall lifetime risk of problems with psychosocial adjustment dysfunction, or the identification of factors that predict positive outcome. Given the fact that poor to moderate correlations between parent and child ratings on different measures have been reported elsewhere (Eiser & Jenney, 2007), another limitation of our study is our sole reliance on parent report. This method of data collection has the potential to be influenced by parental stress and/or emotional maladjustment (Abi Daoud, Dooley & Gordon, 2004), and can also be problematic because some of the findings may reflect shared method variance. A multi-informant perspective is preferred and future cross-validation studies should include both parent and patient self-report whenever possible, as is done in epilepsy for instance (Buck, Smith, Appleton, Baker, & Jacoby, 2007).
The trend toward higher educational achievement in our study and the low-response-rate suggests the possibility of a selection bias in the participants of our study, which may have had an impact on the external validity of our results. More specifically, families with higher education levels may have greater access to financial or other resources that may serve to reduce stressors, thereby resulting in our study under-representing psychosocial adjustment in patients from families with less educational opportunities.
There was also a difference in response rates for the Dutch and American samples in our study. The reason for this is not fully understood, and may have been the result of the method by which the parents were contacted or societal/cultural differences. Also, it is unkown to what extent differences between the rates of Dutch and American mothers completing the questionnaires might have had on the results.
In regards to the general trend for improved overall psychosocial functioning as age increases, the possibility of an attrition/mortality bias has been suggested by others (Miller, Tunnecliffe, & Douglas, 1985). More specifically, there is the possibility that young men with DMD who have better psychosocial functioning have a less severe form of the disease, and therefore a longer lifespan. However, given that we only included boys who were 18 years of age or younger, the impact of an attritional bias is less likely.
While this study identified a cutoff for the PARS-III that can be helpful in clinical practice, the development of clinical norms based on a demographically representative sample would be highly useful. Also, further research is needed on repeated administration of the PARS-III, as this might be of value in clinical practice so as to help elucidate the individual developmental psychosocial profile of males with DMD. Repeated administration of the PARS-III might also contribute to monitoring psychosocial outcome in future scientific research on potential treatments for DMD which are currently in progress, like corticosteroid treatment and other clinical trials.
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The authors wish to thank the Duchenne Parent Project Netherlands and the Parent Project Muscular Dystrophy (USA) for their support and participation.
Conflict of Interest: None declared.
Received May 1, 2007; revision received May 31, 2008; accepted June 2, 2008
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