Study design

Two groups of participants were included in a controlled before and after design, using a mixed-methods approach to assess the effect of rehabilitation on their well-being. The first group (of 30 participants) received rehabilitation care as usual. Next, the healthcare professionals were trained in providing the developed capability care. Then, the second group (of 30 participants) received capability care. Participants were blinded for treatment group. Data were collected at baseline (T0) and 6 months after the initial measurements (follow-up, T1). After the T1 measurements participants were asked whether they had a suspicion of treatment group and if they could elaborate on this suspicion. A more extensive description of the study design [12] and the capability care intervention [13] are published separately.

Participants

Participants were recruited at the Radboud university medical center of expertise for NMD, Nijmegen, The Netherlands, via the departments of Rehabilitation and Neurology between 11 November 2020 and 21 June 2023. All persons with planned appointments were screened by their rehabilitation physician or nurse practitioner for eligibility. Participants were recruited and included before their yearly appointments for ‘analysis and advice’ at the expertise outpatient rehabilitation clinic of the department of Rehabilitation. The study protocol was approved by the medical research ethics committee CMO Regio Arnhem-Nijmegen (NL72794.091.20) and registered at trialregister.nl (NL8946) on 12 October 2020. All participants provided written informed consent. Participants were adults with a diagnosis of FSHD or DM1 with a current rehabilitation aim. See Table 1 for an overview of all inclusion and exclusion criteria.

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Table 1. Inclusion and exclusion criteria of the ReCap-NMD study.

https://doi.org/10.1371/journal.pone.0332388.t001

Intervention

Participants in both groups had one day of appointments for ‘analysis and advice’ at the multidisciplinary outpatient rehabilitation clinic. As the Radboud university medical center is a Center of Expertise for both FSHD and DM1, persons from throughout the Netherlands have appointments with the multidisciplinary rehabilitation team. Prior to their visit, it was determined which healthcare professionals they have consultations with during this day. This was determined together with the person with NMD and depended on their type of NMD and personal circumstances. They always had an appointment with the rehabilitation physician and with one, several or all of the following healthcare professionals: nurse practitioner, physical therapist, occupational therapist, speech and language therapist and/or dietician. After the individual consultations, the multidisciplinary team discussed their findings to provide a rehabilitation advice, which was communicated and discussed with the person with NMD at an additional (phone) appointment with the rehabilitation physician, to make a shared decision on the next steps.

The usual care group received rehabilitation care as usual at the outpatient rehabilitation clinic. The capability care group received capability care: the multidisciplinary team was trained to provide care from a capability perspective. The structure of the day of appointments is similar for both groups. What is changed in capability care is the focus of the conversations between the person with NMD and the healthcare professionals, as well as the analysis and advice by the multidisciplinary rehabilitation team, which were conducted, discussed and formulated from a capability perspective. Capability care can focus both on facilitating and achieving functionings (beings and doings) as well as looking for alternative functionings that fulfil the same underlying value, thereby contributing to a persons’ well-being. To facilitate a conversation on broader aspects that impact on well-being, persons with NMD receive a preparation letter and healthcare professionals are provided with guiding questions and practical tools to use. A more extensive description of capability care and its development is published elsewhere [13].

Outcome measures

We selected the Canadian Occupational Performance Measure performance (COPM-P) and satisfaction (COPM-S) scales as primary outcome measure to assess the effect of capability care and usual rehabilitation on participants well-being. According to the capability approach, well-being is determined by the capabilities a person has. As there is no measure to determine all possible capabilities, we chose to measure realised capabilities; these are the beings and doings (functionings in the capability approach). The COPM is an individualized outcome measure that identifies priorities in a person’s meaningful occupations. We therefore used this as a proxy for measuring a change in functionings, implying that a change in capabilities has also taken place.

The primary outcome was the mean score of the participants self-rated performance (COPM-P) on, and satisfaction (COPM-S) with 3–5 self-selected daily occupations [14]. Through a semi-structured interview, participants identified and prioritized 3–5 problems in their occupational performance. These occupations were subsequently rated on a 10-point scale for perceived performance (1 = impossible; 10 = very well able) and similar for perceived satisfaction (1 = not satisfied at all; 10 = fully satisfied). The COPM is a reliable and valid instrument [15–17], and has been shown to be a sensitive and clinically relevant outcome measure in a number of rehabilitation studies [15,18–21].

To uncover the diverse effects of care, we selected a broad range of secondary outcome measures on participation, capability well-being, and health-related quality of life. These included the Dutch versions of the Utrecht Scale for Evaluation of Rehabilitation Participation (USER-P) [22], EuroQol-5D-5L (EQ-5D-5L) [23,24], ICEpop CAPability measure for Adults (ICECAP-A)[25,26] and Medical Outcome Study Short Form-36 v2 (SF-36) [27]. For the USER-P, we used the scores on the three subscales frequency, restrictions, and satisfaction. These scores have a range from 0–100 with higher scores indicating higher participation (higher frequency, less restrictions, more satisfaction) [22,28]. For the EQ-5D-5L we used the visual analogue scale (VAS) and the level sum score (LSS). The VAS ranges from 0–100 with a higher score indicating a better health; the LSS ranges from 5–25 with a higher score indicating a worse health state [29,30]. The ICECAP-A comprises 5 items, which are capabilities important to one’s well-being: (1) stability, i.e., being able to feel settled and secure in all areas of life; (2) attachment, i.e., being able to have love, friendship, and support; (3) autonomy, i.e., being able to be independent; (4) achievement, i.e., being able to achieve and progress in all aspects of life; (5) enjoyment, i.e., being able to have a lot of enjoyment and pleasure [25]. We used the sum score ranging from 4–20 and the tariff value (calculated based on the general UK population [31]) ranging from 0–1. Higher scores indicate a higher capability well-being [25]. The SF-36 consists of eight subscales (physical functioning, role limitations due to physical health problems, role limitations due to emotional health problems, vitality, mental health, social functioning, bodily pain, general health) with scores ranging from 0–100; a higher score indicating a higher health-related quality of life [27,32].

Sample size calculation

The sample size was calculated based on the primary outcome measure. The expected difference between both groups is 1.4 for COPM-P and 1.9 for COPM-S. These values represent a clinically relevant improvement as perceived by the person [19]. The standard deviations in both groups and for both COPM-P and COPM-S are expected to be 1.9 at 6 months follow-up (T1) [20]. Based on these assumptions, a sample size of at least 24 participants per group will yield a power of 80% to show a statistical difference between usual rehabilitation and capability care group at a significance level of 0.05. These calculations are based on ANCOVA-models with the outcomes at 6 months as dependent variables, corrected for the value at baseline (T0) and an assumed correlation of 0.5 between the outcome values at T0 and T1 [33]. Considering dropout, we aimed to include 30 participants in each group. We aimed for 20 participants with FSHD and 10 with DM1 per group, as more persons with FSHD visit the outpatient clinic than persons with DM1.

Data collection

Participant data were collected 1–2 weeks before their appointments at the department of Rehabilitation (baseline, T0) and 6 months after the initial measurements (follow-up, T1). The primary outcome measure, the COPM, is a semi-structured interview and was administered by an independent research assistant (occupational therapist) during an online or phone appointment. The secondary outcome measures were questionnaires sent to the participants using the electronic data platform Castor EDC [34]. The results of the COPM were manually entered into Castor EDC by the research assistant. The COPM results are also reported in the electronic health record visible to the healthcare team (so that the occupational therapist does not need to repeat the COPM, as it is often used in clinical practice). Adverse events or irregularities affecting protocol adherence were registered in Castor EDC by the primary researchers (BB and EP).

Statistical analysis

As described in the study protocol [12], the primary outcome measure was analysed using ANCOVA models with the follow-up scores at T1 of both groups as dependent variable. Covariates were the scores at baseline (T0) of both groups, age, sex and type of NMD. Group was a fixed factor. For an intervention effect to be present, both the COPM-P and COPM-S needed to show a significant difference between groups (p < 0.05).

To determine whether both capability care and usual rehabilitation had an effect, paired T-tests were performed for the capability care and usual care group separately. The dependent variables were the T0 and T1 scores on the COPM-P and COPM-S. For an intervention effect to be present, both the COPM-P and COPM-S needed to show a significant effect (one-sided p < 0.025); we hypothesized an improvement on T1 compared to T0.

For the secondary outcome measures, ANCOVA models were fitted with the follow-up scores at T1 as dependent variables. Covariates were the T0 score of both groups, age, sex and type of NMD. Group was a fixed factor. Additionally, paired T-tests were performed for the secondary outcome measures, similar to those described for the primary outcome measure.

Incomplete measures (i.e., a participant that did not complete all COPM interviews and/or questionnaires) were excluded from analysis. If only part of the data from a subject was missing, this subject was not excluded from all analyses. For example, if the primary outcome measure was complete but the secondary outcome measures were missing, this subject was only excluded for analysis of the secondary outcome measures. All statistical analyses were performed using Statistical Package for Social Sciences (SPSS) version 29.

Participants

A total of 101 persons were pre-screened by their physician or nurse practitioner and contacted by a researcher (EP or BB). Of these, 64 persons agreed to participate (see Fig 1). The foremost reasons for declining participation (similar for both types of NMD) were: rehabilitation appointments planned too short to be able to complete baseline measurements in time (n = 10), too time or energy consuming (n = 8), inclusion already complete for their type of NMD (FSHD or DM1) (n = 6) (pre-screening had continued, while sufficient participants with the type of NMD were already included) and no current rehabilitation aims (n = 5) (see Fig 1). Of the 64 participants, six were excluded or dropped out during the study. Three (two DM1, one FSHD) could not formulate a current rehabilitation aim during the COPM interview with the research assistant, one postponed appointments to a time where capability care training of the professionals had already started (and therefore could no longer be included in the usual care group), one cancelled appointments, and we lost contact with one before the first measurement. In total, 58 participants completed baseline measurements for the primary outcome measure; however, one participant did not complete any of the T1 measurements and was excluded from analysis. Therefore, data from 57 participants were used for analysis. Of these, three participants did not complete all measurements (see Fig 1 and Table 2). Additionally, one participant only completed the ICECAP-A and no other secondary outcome measures.

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Table 2. Baseline characteristics of the participants included for analysis.

https://doi.org/10.1371/journal.pone.0332388.t002

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Fig 1. Flowchart of the inclusion process.

DM1: Myotonic dystrophy type 1; FSHD: Facioscapulohumeral muscular dystrophy; NMD: neuromuscular disease.

https://doi.org/10.1371/journal.pone.0332388.g001

For the usual care group, baseline measurements took place between November 2020 and August 2021, with follow-up between May 2021 and February 2022. For the capability care group, baseline measurements took place between March 2022 and December 2022 with follow-up between October 2022 and June 2023.

Table 2 shows the characteristics of the participants included for analysis for both usual and capability care. The groups seemed comparable at first glance with respect to type of NMD, sex, age (Table 2) and baseline values of the primary and secondary outcome measures (Table 5 and S1-S5 Tables).

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Table 3. Suspicion of treatment group by participants.

https://doi.org/10.1371/journal.pone.0332388.t003

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Table 4. Confidence intervals and p-values for the effect of group on the Canadian Occupational Performance Measure performance (COPM-P) and satisfaction (COPM-S) scales.

https://doi.org/10.1371/journal.pone.0332388.t004

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Table 5. Means and standard deviations of the scores on the Canadian Occupational Performance Measure performance (COPM-P) and satisfaction (COPM-S) scales for both groups at baseline (T0) and 6-month follow-up (T1).

https://doi.org/10.1371/journal.pone.0332388.t005

Table 3 shows whether participants had a suspicion of which treatment group they were assigned to. In the usual care group, 28% had a correct suspicion. In the capability care group, 43% had a correct suspicion. When we look at the reasons that participants provided, in each group 4 participants that suspected usual care mentioned that the care is not different from what they had experienced previously. In the usual care group all 7 participants (24%) that suspected capability care provided reasons such as personalised treatment, professionals took the time for my story and they really helped me with my questions; in the capability care group 3 participants (11%) provided similar reasons. In the capability care group 6 participants (21%) provided reasons that could be attributed to capability care, such as a focus on my possibilities (1 participant), attention for mental health as well as physical symptoms (1 participant), a different way of asking questions (1 participant) and attention for values (3 participants).

Canadian Occupational Performance Measure

We fitted ANCOVA models to determine whether capability care had a larger effect on the COPM-P and COPM-S scales compared to usual rehabilitation. The covariates age, sex and type of NMD had no significant effect on the outcome. The baseline score had a significant effect on the outcome for both COPM-P (F(1,50) = 25.852, p < 0.001) and COPM-S (F(1,50) = 13,840, p < 0.001). Group showed no significant effect for neither COPM-P (F(1,50) = 0.004, p = 0.947) or COPM-S (F(1,50) = 0.401, p = 0.530) (see also Table 4).

Paired T-tests were performed to determine whether there was a difference between baseline and follow-up for both groups. Means and standard deviations for both groups are shown in Table 5. At follow-up, both the usual care and capability care group showed an improvement on COPM-P (t(28) = 4.104, p < 0.001 and t(26) = 2.995, p = 0.003, respectively) and COPM-S (t(28) = 4.564, p < 0.001 and t(26) = 3.433, p = 0.001, respectively).

Secondary outcome measures

ANCOVA models were fitted to determine whether capability care had a larger effect than usual rehabilitation care on all secondary measures. An overview of the baseline and follow-up scores of the secondary measures can be found in the supporting information (S1-S5 Tables). For all measures, the baseline score of the respective subscales showed a significant effect (all p < 0.001) on the outcome. For 6 of the 15 subscales, some of the covariates age, sex and type of NMD showed a significant effect (see S5 Tables). Group showed no significant effect for all subscales of all secondary outcome measures.

Paired T-tests were performed to determine whether there was a difference between baseline and follow-up for both groups. These showed no significant difference between baseline and follow-up for all secondary outcome measures.

Comparing capability care to usual care

Overall, we found no significant differences between the capability care group and usual care group. This might indicate insufficient contrast between the two groups, which may be due to: 1) the choice of outcome measures; 2) the content of capability care; and/or 3) the implementation of capability care. Below, we will discuss each of these options.

Effect of rehabilitation: changes over 6 months

The results showed a significant improvement at follow-up on both subscales of the COPM. However, these did not reach the level of clinically relevant improvement. Initially, we decided to use values of 1.4 on the COPM-P and 1.9 on the COPM-S as indication of clinically relevant improvement as perceived by the person [12,19]. However, recent discussions on the cut-off value for clinically relevant improvement concluded that there is insufficient evidence to determine a cut-off value [39]. It is also suggested that this value may vary depending on person characteristics, their occupational performance issues, and contextual factors [39]. Therefore, we cannot determine whether our results show a clinically relevant improvement over time. However, the observed improvements on the COPM, ranging from 0.8 to 1.1 six months after a visit to the multidisciplinary outpatient rehabilitation clinic, suggest that the day of ‘analysis and advice’ along with the subsequent interventions, contributed to positive changes in participants’ occupational performance and satisfaction.

Study design

We used a controlled before-after study design with two groups of participants, whereby participants were blinded for group allocation. Due to the outpatient clinic setup, it was not possible to blind the professionals, e.g., by training half of the healthcare professionals and randomise participants. This might be considered a limitation; however, it is also a strength that the same healthcare professionals provided care in both groups before and after training in capability care.

In addition to the quantitative outcome measures reported in this article, we used several qualitative methods, such as audio-recording of consultations and multidisciplinary team meetings, access to the notes in the electronic health record, interviews with participants and focus groups with healthcare professionals. The use of this mixed-methods design may provide us with more in-depth understanding of the current results and the added value of the capability approach in rehabilitation for persons with NMD and will be published separately.

Future directions

Future studies are needed on how to measure well-being change in a care setting such as rehabilitation, that focuses on achieving individualized goals to enhance a person’s well-being. Additionally, further research in a rehabilitation treatment setting is needed, as we hypothesize that the contrast between groups will be larger in a treatment setting. Furthermore, the use of the capability approach is not limited to rehabilitation. Future studies in healthcare can contribute to the body of knowledge on using the capability approach in clinical intervention development.