Pedsql duchenne muscular dystrophy download assessment pdf

Significant relationships between quality of life and fatigue overlap with studies in the literature related to the effects of fatigue on the quality of life of children with DMD. Thus, the result was considered to be acceptable when the structural characteristic of the questionnaire was regarded. Thus, collection of data regarding fatigue from both parents and children is important in terms of accurate assessment of fatigue from different perspectives in clinical practice.

The limited number of patients with DMD and the inclusion of only physically active, ambulant children in current study can be considered as a limitation.

Also, the missing information on the daily doses of steroids for each child is another limitation of the study. The scale can be considered as a promising tool to be used as an outcome measure that evaluates fatigue and quality of life from the perspectives of parents and children with DMD in clinical trials in the future. Further studies are needed to be performed on a large number of patients with wide range of functionality to investigate broader use of the Turkish version of PedsQL-MFS.

Authors have no conflict of interests, and the work was not supported or funded by any drug company. Readers may copy, distribute, and display the work for non-commercial purposes with the proper citation of the original work. NOTE: We only request your email address so that the person you are recommending the page to knows that you wanted them to see it, and that it is not junk mail.

We do not capture any email address. Skip to main content. Research Article Original Article. Open Access. Subjects A total of 71 patients with DMD, whose functional levels were between 1—3 Level 1: The child is able to walk and climb the stairs, independently; Level 2: The child is able to walk and climb the stairs by using handrails, taking less than 12 seconds; Level 3: The child climbs the stairs slowly, taking more than 12 seconds according to the Brooke Lower Extremity Functional Classification BLEFC , 10 aged between 5—12 years, who were on corticosteroids for more than 6 months, and who were still ambulant were included in the study with their parents.

The translation steps were as follows; I. Internal Consistency Reliability The standardized Cronbach alpha coefficient was used to detect the internal consistency of the scale. Test-Retest Reliability The same physiotherapist applied the questionnaire twice to 71 children and their parents with a 2-week interval to test the test-retest reliability of the PedsQL-MFS. Results Seventy-one boys with DMD aged 55 to months, and 69 mothers View this table: View inline View popup.

Figure 1 Bland-Altman Plot for test-retest reliability of child self-report. Figure 2 Bland-Altman Plot for test-retest reliability of parent proxy report.

Construct Validity Table 3 presented the confirmatory factor analysis results of the 3-factor model of the child self-report and parent proxy report.

Table 3 3-factor model results of confirmatory factor analysis. Footnotes Disclosure. Neurol India 56 , — Rev Bras Fisioter 14 , — Lancet Neurol 9 , 77 — Cell 51 , — Ann Readapt Med Phys 49 , — Neuromuscul Disord 23 , — Dubowitz V , ed Muscle disorders in childhood W. Saunders , Edinburgh UK , Romani A The treatment of fatigue.

After thinking aloud their thought processes, however, participants were able to respond readily to these items, and did not suggest any modifications. From the outset, the need to distinguish between the impact of having bladder accidents and performing a bladder program on HRQoL was established by the research team and expert panel, and was confirmed from the Modified Delphi surveys. Participants were able to differentiate between the items pertaining to bladder accidents and bladder program, and responded appropriately.

During the first round of interviews, two participants with SCI and one parent discussed how muscle spasms impeded the ability to perform transfers. The definition provided for this domain was considered to be helpful, particularly for children of ages 8— Participants in the 5—7-year age group did not know what a pressure injury was, nor did they understand after it was explained to them, so this domain was removed from the Young Child version.

Once the participants heard the correct pronunciation and read it correctly themselves, most of them readily recognized the terminology. Following this modification, all participants were able to read these words without difficulty. Parents of toddlers commented that it is not possible to determine if their child experiences symptoms of OH as toddlers cannot clearly communicate their discomfort other than by crying or being irritable.

On the other hand, parents did say they are able to detect signs of AD in their child, such as facial flushing and sweating.

Children of ages 5—7 did not understand OH or AD. This domain was well accepted, particularly by the parents who voiced appreciation for items assessing community participation for their children. There were, however, several responses pertaining to self-care activities, such as brushing teeth and washing face. Initially, this domain contained items on all of the SHCs e. Accordingly, these items were removed. The other items assessing treatment, peer relationship, and body image were all considered relevant issues that youth with SCI worry about.

At the conclusion of each cognitive interview session, participants were asked to talk freely about their thoughts on the module. Two children in the 8—year age group commented on the length, but said that they were able to complete the module easily. All participants responded positively regarding content and readability. Table 4 lists a few notable comments. Incorporation of expert opinion by professionals in pediatric SCI and detailed feedback obtained from cognitive interviews of individuals living with an SCI allowed for item development and content validity for this multidimensional HRQoL measurement instrument specific to youth and emerging adults with SCI.

The FDA Guidance strongly recommends in-depth focus group or individual interviews in the early stages of item development to determine concepts most relevant to the consumer [ 22 ]. Given the highly heterogeneous presentation of SCI depending on the severity and level of injury, however, there was a possibility that focus group or individual interviews would not be able to adequately capture the full spectrum of relevant issues encountered in SCI.

Thus, the input obtained from the multidisciplinary expert panel and modified Delphi surveys allowed for a comprehensive collection of relevant concepts affecting HRQoL in youth with SCI, which were then used to develop the preliminary module.

The subsequent cognitive interviews conducted using the preliminary modules provided very insightful information on SCI-specific and age-appropriate content and wording. Not surprisingly, the majority of the modifications were made to the Parent of Toddler and Young Child versions due to the developmental stage of the child precluding them from performing activities or understanding the items.

The addition of brief definitions and examples for domains pertaining to SHCs was considered to be appropriate and informative, particularly for participants who had not previously experienced the specific condition. This study is limited by the lower proportion of participants with tetraplegia who completed the cognitive interviews, potentially resulting in less information on HRQoL issues related to having higher levels of SCI.

In addition, there was a significantly lower number of nonwhite individuals providing their perspectives. As the next step of instrument development, an internet-based field test of the newly developed module is currently underway, and this will be followed by quantitative methods to determine the reliability and validity of the instrument.

The Parent of Toddler version contains 38 items in 9 domains, the Young Child version contains 56 items in 9 domains, and all other versions contain 67 items in 12 domains. Data sharing is not applicable to this paper as no datasets were generated or analyzed during this study. Post M, Noreau L.

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We do not believe that the construct validity of a disability domain would differ across patient populations, but rather that some items may be far more or far less likely to be endorsed in some patient populations. Such differences might result in dependency between items which challenges unidimensionality. Based on our analysis for example, we opted to retain more items in the mobility domain than the mobility short form, in order to clearly capture the range of mobility disability and to retain items deemed relevant by the caregivers.

This decision was motivated by a desire for better content validity, while at the same time it undermined unidimensionality i. Thus, construct validity for a disability domain measure is not different for those with DMD, but some items used to measure the construct may function in a different way. The resulting measure includes eight domains that reflect the conceptualization based on caregiver input.

Six of these domains had strong psychometric characteristics, and two of which are better conceptualized as clinimetric and are scored using raw sums. The resulting measure can be scored quickly and manually or using the IRT-scoring tables to provide more precise metrics. The scores resulting from the two methods are highly correlated.

The caregivers included in this study were generally parents of the person with DMD. As is the case with most childhood-onset health conditions, parents bear the greatest responsibility for their child.

While siblings, other relative, and paid aides may also provide caregiving support, our study predominantly reflects parental caregivers. Future research might explicitly focus on siblings, other relatives, and paid aides in furthering the validation of this condition-specific proxy report measure of DMD disability.

Input from clinicians would be helpful in ascertaining how helpful this proxy-reported information is in the clinical setting. Of note, the mobility scale posed many challenges for psychometric scale development. Most pointedly, exclusion of key items might have resolved issues of local dependence i. An approach that was blinded to item content might have produced a short form that had good psychometric characteristics.

However, this approach ignored the reality that most people with DMD use a wheelchair at a relatively young age and, as their disability progresses, lose the ability to walk a block, walk across the room, or get up from the floor. This initial version of the DMD-specific proxy-report measure built on the Lowes Algorithm and reduced the number of items presented by about eight. The tailored administration is now only one or two items shorter than the full administration because the psychometric analyses supported removing a number of items from the final item set.

Even if one were to rely only on one to two questions to determine wheelchair use to tailor the mobility and sleep-device symptoms items, the total number of items administered would be about the same in a tailored versus not-tailored administration.

Thus, tailoring does not reduce the length of the measures and would not be recommended. Future research will investigate whether full administration of all items within the mobility and sleep-device symptoms domains renders any differences in terms of simple structure and model fit. The data enabled careful psychometric modeling that considered relevant subgroups. The limitations of the study must be acknowledged, however. First and foremost, the study is only able to address the cross-sectional characteristics of the measure.

Longitudinal construct validity [ 52 ] was not addressed, in particular responsiveness to clinically important change [ 53 ] and stability in the face of no change [ 54 ]. Second, as with any scale development, its validation is iterative. Future work should continue the validation of the new measure in an independent data set.

Such future work might explicitly include a Patient and Public Involvement phase that expands the role of the DMD caregivers to be partners at all stages of the research [ 55 , 56 , 57 , 58 ]. Third, it would be worthwhile to investigate differential item function related to gender and education.

While this investigation is beyond the scope of the present study, future work might address this important aspect of item function. Fourth, the new measure is explicitly for proxy assessment, not patient assessment. This decision was made because the study was focused on DMD caregivers, not patients. Accordingly, we did not collect patient data in conjunction with the proxy-reported measure. Future work might utilize the patient-reported versions of the proxy domains and items used, and validate this set of PROMIS scales for use in patient-reported research.

Future research should also address the relationship between caregiver proxy report and DMD patient self-report. Past research done by members of our group with caregivers of children with genetic disorders revealed that caregivers preferred the former to the latter because it allowed for positive aspects of caregiving which were broadly acknowledged by study participants [ 21 ]. Capturing the patient voice: implementing patient-reported outcomes across the health system.

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J Clin Neuromuscul Dis. Propp R. Development and psychometric evaluation of the muscular dystrophy child health index of life with disabilities MDCHILD questionnaire in children with Duchenne muscular dystrophy. University of Toronto Canada ; Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments. Health Qual Life Outcomes. Upon termination of the User Agreement between the User and MRT, the User shall immediately cease the use of the e-PRO and shall destroy any material related to the e-PRO in the system by erasing them from the magnetic mediate on which they are stored and certify in writing that they have been destroyed.

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