Use of supported standing in males with Duchenne muscular dystrophy: Individual and family perspectives

2.1 Design

The current study utilized a mixed methods research (MMR) design to achieve the study aim. MMR utilizes both quantitative and qualitative data analysis and integrates the data to draw interpretations based on the combined strengths of both data sets to better understand the research problem [16, 17]. This study utilized a concurrent triangulation design, a validating quantitative data model of MMR. This model involves collecting quantitative and qualitative data simultaneously from a survey and using the qualitative information to validate the quantitative results [16, 18].

Upon approval from the Institutional Review Board, data collection occurred via administration of a cross-sectional survey (see Appendix 1) that included both categorical and open-ended questions and was administered online via Research Electronic Data Capture (REDCap) tools hosted at Cincinnati Children’s Hospital Medical Center [19, 20]. REDCap is a secure, web-based software platform designed to support data capture for research studies, providing 1) an intuitive interface for validated data capture; 2) audit trails for tracking data manipulation and export procedures; 3) automated export procedures for seamless data downloads to common statistical packages; and 4) procedures for data integration and interoperability with external sources. Respondents’ consent was implied through completion of the survey. All responses were anonymous. Two versions of the survey were administered: one version for parents/caregivers of individuals diagnosed with DMD that will be referred to as the caregiver survey questionnaire (CSQ), and another version for adult individuals diagnosed with DMD that will be referred to as the DMD survey questionnaire (DMDSQ). Survey types differed in two ways. First, the CSQ included an item asking about the number of children in the household diagnosed with DMD. Respondents could take the survey multiple times to capture data relevant to each of their children diagnosed with DMD. Second, questions were framed differently between the two survey types to tailor them to the identity of the respondent (i.e., parent/caregiver vs. individual with DMD). Both survey types collected identical data.

Inclusion criteria for completing the survey was based on responses to screening questions 1–3 (refer to Appendix 1). Question 1 confirmed that the respondent was either a parent or caregiver of or an individual with DMD. The second question confirmed that the individual with DMD was male. The third question confirmed that the respondent was at least 18 years old and that the individual with DMD was at least 10 years old. The study excluded respondents who lacked the English literacy necessary to complete the survey.

All respondents who met the inclusion criteria were asked if they were familiar with the use of standers for individuals with DMD (Appendix 1: CSQ 1; DMDSQ 1) and answered demographic survey questions (Appendix 1: Demographic Questions). Those who were familiar with the use of standers, but who were not currently using a stander (Appendix 1: CSQ 3; DMDSQ 1), answered two additional questions: (1) reasons that may keep their child/themselves from using a stander and (2) any other thoughts they would like to share. Those respondents who were familiar with the use of standers and currently using a stander answered all survey questions.

2.2 Recruitment of participants

Recruitment occurred simultaneously through two sources. The first recruitment source was Parent Project Muscular Dystrophy (PPMD), a national public organization for DMD. The survey was sent to 1,660 unique email addresses of male PPMD registrants with DMD living in the United States. The second recruitment source was patients seen in the Comprehensive Neuromuscular Center at Cincinnati Children’s Hospital Medical Center, which serves patients both nationally and internationally. Four hundred and eighty-eight emails were sent to families of males with DMD via this recruitment source. Respondents were asked to follow an emailed link to the online survey. Follow-up emails were distributed at two and four weeks. Investigators allowed two months from the initial email distribution date to complete the survey.

2.3 Quantitative analysis

Quantitative analysis explored responses to categorical survey questions pertaining to stander acquisition, initiation of stander use, stander dose, and location of use (Appendix 1: CSQ 3–14; DMD 2–13). Where applicable (Appendix 1: CSQ 6, 8, 9, 13, 14; DMD 5, 7, 8, 12, 13), survey responses were categorized according to dose of stander use, utilizing the frequency, intensity, timing, and type (FITT) principle [21, 22]. Frequency was defined as the number of days per week of stander use. Intensity was determined by the amount of upright position achieved in the stander using pictures in the survey (Appendix 1: CSQ 13; DMDSQ 12). Timing was defined as the duration of stander use in number of minutes per day. Lastly, type was described by the type of stander, i.e., a power standing feature in a wheelchair (PSW) or sit-to-stand stander (STS).

Descriptive statistics were computed using Microsoft Excel (Microsoft Office 365 ProPlus 2013, Version 15.0.4893.1002) for analysis of ambulatory status, initiation of stander use, stander dose, stander acquisition, and location of stander use. Frequencies and percentages were derived for categorical variables. Comparative statistical analyses were conducted using SAS^® version 9.4 (SAS Institute, Inc., Cary, NC) to compare LOA and start of stander use, reported dose versus recommended dose, and dose by stander type. Pearson’s correlation analysis was performed to compare LOA to start of stander use. Student’s t-tests were performed to determine if respondents’ stander use matched recommended dosing (a duration of 60–90 minutes at a frequency of at least five days per week), as well as the total dose equal to the product of the minutes per day and the days per week. Two-sample Student’s t-tests were used to measure differences in use between the two stander types (p < 0.05). Satterthwaite approximation was used for the two-sample t-tests. The relationship between dose and stander type was evaluated using Fisher’s Exact Test.

Two neuromuscular physical therapists reviewed open-ended survey responses related to liked or disliked features of the stander and what would make using a stander easier (Appendix 1: CSQ 16–18; DMDSQ 15–17) and utilized thematic analysis to analyze the data. Categories were analyzed using descriptive statistics in Microsoft Excel. Frequencies and percentages were derived for categorical variables.

2.4 Qualitative analysis

Open-ended survey responses related to problems encountered when trying to get a stander, reasons the individual with DMD does not use a stander, and perceptions around importance/usefulness of standers (Appendix 1: CSQ 15, 19–21; DMDSQ 14, 18–20) were analyzed using a deductive qualitative content analysis approach [23]. Responses were coded according to the International Classification of Function (ICF) for analysis. The ICF [24], a commonly used [25] and validated framework [26] that extends beyond medical diagnoses to classify functioning and disability on both the individual and population level, was used to create a categorization matrix following coding. The ICF framework is grouped into five domains: body functions, activities and participation, environmental factors, body structures and personal factors. Four of these domains have been classified into a hierarchically nested structure with up to four different levels (Fig. 1) that become more detailed as they unfold [24, 27].

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Fig. 1

Taxonomy of the ICF.

Two neuromuscular physical therapists met multiple times to critically reflect on survey responses, review the responses for content, and code them to the first level (i.e., chapters) by using key words in the responses and aligning them with the chapter definitions [24, 26]. If responses contained information that was relevant across multiple chapters, then all relevant chapters were included. Descriptive statistics were computed using Microsoft Excel. Frequencies and percentages were derived for coded categorical variables.

Integration of the qualitative and quantitative results occurred as the categorical and open-ended survey responses were merged in a joint display [16, 28]. Meta-inferences (i.e., interpretations based on the qualitative and quantitative findings) were drawn to determine whether confirmation or disconfirmation occurred between the datasets [16, 28]. Confirmation occurred if the findings from both types of data reinforced the results from the other [28]. Disconfirmation occurred if the two datasets were inconsistent or disagreed with each other [28].

Questions specific to funding sources and parties who facilitated stander acquisition permitted multiple response options and are outlined in Table 2. Respondents reported similar methods of obtaining PSWs and STSs.

Participants were also asked to describe barriers to obtaining a stander (Table 3). Qualitative results coded according to the ICF revealed that environmental factors represented the most common hindrance, with nearly half (n = 16) of those who responded to this question reporting economic services challenges. These challenges were associated with cost, insurance approval/denial process, and time from ordering to delivery of the standing device. With seven respondents, activity and participation barriers related to furnishing a place to live (e.g., finding adequate space to store the stander) represented the second most common hindrance.

The average age of LOA (defined here as the inability to walk 10 meters without assistance) for individuals with DMD was 13.6±3.0 years (range: 8–22). The average age at which individuals with DMD began using a stander was 13.9±4.0 years (range: 8–31). Seventeen of 42 respondents reported that the individual with DMD was ambulatory when stander use was initiated. Four of 42 respondents reported the individual with DMD using a stander was currently ambulatory. There was a positive correlation between age of LOA and age of start of stander use (r = 0.61, p < 0.0001, n = 36), with a later onset of LOA associated with a later initiation of stander use (see Fig. 3).

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Fig. 3

Comparison of Age of Loss of Ambulation (LOA) to age of start of stander use.

Of the 42 respondents who reported stander use, 13 reported using a PSW (including Permobil F5 and Permobil C400), and 27 reported using a STS as the stander type. Two respondents did not report the type they used. Regarding frequency and timing, respondents reported using the stander 4.5±1.7 days per week (range: 1–7) and 44.8±30.2 minutes per day (range: 10–180) on days when they used the stander. Most respondents (n = 31) reported spending their time in the stander all at once; fewer (n = 11) reported using the stander multiple times throughout the day. Half of the respondents (n = 21) reported the individual with DMD became fully upright when using the stander, seven became nearly upright, seven became moderately upright, and seven became mildly upright. Figure 4 illustrates comparisons of respondents’ frequency, intensity, and timing of use by stander type.

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Fig. 4

Comparison of Frequency, Intensity, and Timing by Type (FITT) of stander.

Of those who reported using a PSW or STS type, 10 participants reported standing at a dose of 60–90 minutes at least five days a week. Respondents’ number of days per week of stander use were not significantly less than five days per week (mean = 4.5, 95% confidence interval [CI] = 4.0–5.0, p = 0.06). Minutes per day of stander use were significantly less than 60–90 minutes (mean = 44.8, 95% CI = 35.4–54.2, p = 0.002). Similarly, respondents’ total dose was significantly lower than the recommended 300 minutes (mean value = 227, 95% CI = 165–289, p = 0.02). Additional analyses revealed no significant differences in use between stander types (STS or PSW) for either days per week (mean difference = –0.8, 95% CI = –1.9–0.4, p = 0.15) or minutes per day (mean difference = 1.5, 95% CI = –19.5–22.5, p = 0.87). The relationship between total dose and stander type was not significant (mean difference = –20.4, 95% CI = –154.7–113.9, p = 0.76).

Respondents were asked to report all locations in which they used their standers. Thirty-eight of the 42 respondents reported use at home, including 24 who used a STS, 12 who used a PSW, and two who reported using “other” stander types. Nine used their stander at school, one during therapy appointments, and four at “other” locations (e.g., “Voice rehearsal” [P3]). Seven respondents with a PSW and two with a STS reported stander use at multiple locations.

3.5 Perceptions regarding features of standers

Respondents also described preferred features of their stander. For the 42 respondents who were currently using a stander, the most preferred features in order of preference were the tray (n = 12), the ability to use the stander anywhere/anytime among PSW users (n = 9), the ease of adjustment and movement into the standing position among STS users (n = 8), the ability to move a STS within the environment (n = 2), and the supportive nature of the PSW (n = 1). While not directly “features” of a stander, some respondents reported benefits associated with stander use, including the ability to participate with peers and urinate while standing, as well as reporting that the stander helped with flexibility, bone density, and scoliosis prevention. One respondent also noted the stander was “fun to use” (P55).

Respondents also reported stander features they disliked. Nine respondents mentioned various components of the stander (e.g., arm rest height, tray height, head rest, knee pads, etc.). Equally disliked was the overall process for set up and optimal adjustment of the devices (n = 9). Other negative features included neither device being supportive enough (n = 3), the PSW not providing enough of a stretch (n = 2), the size/bulkiness of the STS (n = 5), and difficulty transferring into the STS (n = 5).

3.6 What would make using the stander easier

In response to the question, “What would make using the stander easier?” for those 42 respondents who were currently using a stander, 15 respondents reported overall improved design/product modification and reduced size of the devices would improve ease of use. Nine respondents reported that an altogether different product or incorporation of the stander into their power wheelchair would make it easier to use. Other responses included ease of transferring into the device (n = 4), nothing (n = 4), motivation for consistent use (n = 2), financial barriers (n = 2), and support for proper fitting of the device (n = 1).

3.7 Perceptions regarding importance/usefulness of standers

The 42 respondents who were currently using a stander also reported on the importance/usefulness of standers. Parents/caregivers completing the survey were asked why using a stander is important/useful from both their perspective and their child’s (Appendix 1: CSQ 19-20). Individuals with DMD completing the survey were also asked this question from both perspectives (Appendix 1: DMDSQ 18-19). When analyzing the results, the parent/caregiver perspectives were combined from both survey questions (Appendix 1: CSQ 20; DMDSQ 19), as were the individual with DMD perspectives (Appendix 1: CSQ 19; DMDSQ 18). Responses spanned all four domains of the ICF (Table 3). The majority of responses were best characterized in the body functions domain (n = 54), specifically within the mobility of joint functions chapter, with 24 from the individual with DMD’s perspective and 30 from the caregiver’s perspective. The second most common response (n = 32) was best characterized in the body structures domain, specifically the bones (i.e., bone health) chapter, with 12 from the individual with DMD’s perspective and 20 from the caregiver’s perspective. Additionally, there were multiple responses that spanned many chapters of the activities and participation domain (n = 39), 23 from the individual with DMD’s perspective and 16 from the caregiver’s perspective.

3.8 Reasons stander not used

All 120 survey respondents who were familiar with stander use for individuals with DMD were asked to share reasons that may prevent stander use, regardless of stander use history, and 111 responded (Table 3). Responses spanned the four domains of the ICF. The greatest number of responses aligned with the mobility of joint functions chapter (i.e., range of motion) of the body functions domain (n = 19) and the economic services chapter of the environmental services domain (n = 19). Other commonly reported barriers included the time management chapter of the body functions domain (n = 14) and the ensuring one’s physical comfort (n = 15) and walking (n = 13; e.g., “currently he is still ambulatory. . . ” [P117]) chapters of the activity and participation domain.

3.9 Integration of quantitative and qualitative results

The qualitative findings confirmed the quantitative results regarding initiation of stander use and the frequency and timing (duration) of stander use. The quantitative results informed when stander use was initiated and the frequency and timing of stander use. The qualitative analysis provided insights into why stander use was primarily initiated after LOA and at less than the recommended frequency and duration.

As illustrated in Table 4, individuals with DMD delayed stander use until after LOA due to a lack of education and awareness about the benefits of early initiation, perceptions of stander use prior to LOA, and difficulties with insurance approval. Lack of time and energy to complete a supported standing program and the presence of contractures that made it difficult for the individual with DMD to tolerate standing contributed to decreased duration of stander use.

The qualitative findings also confirmed the quantitative results indicating primary use of the stander within the home regardless of stander type and the use of the PSW in multiple locations. The preference for use of the stander in the home may be due to the time it takes to get set up comfortably in the stander and the perception of others when in the stander (Table 4). The versatility of a PSW to assist with specific activities outside of the home was consistent with the use of the PSW in multiple locations compared with those using a STS.

Other qualitative findings disconfirmed the quantitative results regarding stander type. Whereas the quantitative results revealed that more participants reported having a STS, the qualitative analysis revealed preferences for the PSW. Reported space limitations and difficulties with transferring in and out of a separate device (STS) illustrated these preferences. Economic and health services barriers, including limited insurance coverage, prolonged process for obtaining standers, and cost of a PSW, may explain this discrepancy (Table 4).

4.1 Strengths and limitations

Combining the quantitative data with the qualitative data in the mixed methods analysis provided a better understanding of stander use than either form alone and allowed for triangulation of findings [17]. By expanding on the quantitative data, the qualitative findings provided greater insight into potentially modifiable barriers to stander use. This study was limited by factors inherent in any cross-sectional survey [33]. Specific to this study, individuals may have received the survey through two different recruitment methods, thus reducing the response rate. As this study utilized a sample of convenience and the response rate was low, it may not be representative of the DMD population at large. When responding to demographic questions, the majority of respondents reported having private rather than public insurance and reported an annual household income of over $80,000, which may have influenced the variability of responses.

Consistent with prior research [15], few respondents reported stander use, which further limited the generalizability of the findings. While there were limited responses from individuals with DMD, it is possible that a caregiver and their adult child with DMD may have both completed the survey and thus potentially overrepresented their experiences. While two authors participated in the qualitative analysis, independent coding was not feasible. However, due to the clarity of the codes based on the definitions available for the ICF chapters, this does not likely constitute a substantial limitation.