Exploring Awareness of and Self‐Reported Adherence to Neuromuscular Clinical Practice Guidelines Among Australian and New Zealand Health Professionals: A Cross‐Sectional Survey Study

ABSTRACT

Background and Purpose

There is limited evidence describing awareness and adherence to clinical practice guidelines for neuromuscular disorders. This study aimed to assess awareness of and self‐reported adherence to clinical practice guidelines for neuromuscular disorders among Australian and New Zealand health professionals.

Methods

A cross‐sectional survey of health professionals working with individuals with neuromuscular disorders was conducted to describe awareness of three neuromuscular guidelines. Self‐reported adherence to recommendations for allied health assessments in Duchenne muscular dystrophy and exercise and physical activity in paediatric Charcot‐Marie‐Tooth was also assessed.

Results

Sixty‐four surveys were received; 50/64 (84%) worked in metropolitan areas, 44/64 (69%) were allied health professionals and 35/64 (55%) had less than 5 years’ experience in neuromuscular disorders. Sixty participants provided responses about guideline awareness. Awareness was 67% (n = 40/60)—DMD diagnosis and management care considerations, 42% (n = 25/60)—Clinical practice guidelines for allied health and nursing assessment and management of Duchenne muscular dystrophy and 33% (n = 20/60)—Clinical practice guideline for the management of paediatric Charcot‐Marie‐Tooth disease. Those who were aware of the guidelines felt that they influenced their practice. Self‐reported adherence to evidence‐based assessments in Duchenne muscular dystrophy ranged from 24% (use of dynamometry) to 90% (not using skinfolds). Twenty‐four (24/64; 38%) respondents managed paediatric Charcot‐Marie‐Tooth and most recommended exercise and physical activity for management of Charcot‐Marie‐Tooth‐related impairments.

Discussion

This study provides insights into guideline self‐reported adherence among Australian and New Zealand health professionals treating individuals with neuromuscular disorders. Targeted strategies to increase guideline awareness and adherence are vital to ensure standardised and equitable care in readiness for prospective disease‐modifying therapies.

1. Introduction

Neuromuscular diseases arising in infancy and childhood include a diverse group of conditions including Duchenne muscular dystrophy (DMD) and Charcot‐Marie‐Tooth disease (CMT). Considerable progress in clinical management of these diseases has occurred over the past 2 decades, including the development of clinical practice guidelines. The first international and multidisciplinary guidelines for DMD published in 2010 [1, 2], transformed the international standard for best practice management of people with DMD. Subsequently, guidelines were released for other neuromuscular diseases [3, 4, 5, 6] and the DMD guidelines were updated in 2018 [7, 8, 9]. More recently, two Australian‐led guidelines for neuromuscular diseases have been developed [10, 11].

Despite the proliferation of guidelines for neuromuscular diseases very little is known about health professional adherence to guidelines among those who care for individuals with neuromuscular diseases. Understanding if evidence to practice gaps exist, including barriers and enablers to uptake is important in the development of implementation strategies to support the provision of equitable and standardised best practice care. In this era of disease modifying therapies [12, 13], focused efforts to enhance the implementation of best practice care is needed to both assess the effect of and optimise the outcome of these therapies.

The aim of this study was to describe awareness to three neuromuscular guidelines in DMD and CMT among health professionals in Australia and New Zealand. In addition, we aimed to assess self‐reported adherence and identify barriers to adherence to key recommendations from the DMD and CMT guidelines.

2. Methods

A cross‐sectional study of Australian and New Zealand health professionals was conducted via an electronic survey between April and June 2023. Any medical, nursing or allied health professional (‘health professionals’) involved in clinical care of people with DMD (adult or paediatric) and/or children and adolescents (‘children’) with CMT were eligible to complete the survey. Health professionals who did not have a clinical caseload involving people with DMD or CMT were ineligible. A survey link was distributed via email to Australian and New Zealand adult and paediatric neuromuscular clinics, key providers of disability care, professional networks, via social media, and the Australasian Neuromuscular Network (www.ann.org.au). Snowballing maximised recruitment. Participation was voluntary and informed consent was implied by completion of the online survey. The survey was developed by the lead authors and piloted by the remaining authors. To support content validity of the survey, all authors contributing to the survey had expertise in neuromuscular disorders including neuromuscular guideline development and/or implementation science. Also, survey questions assessing self‐reported adherence directly aligned with guideline statements. The full survey is included in Material S1.

Survey participants were asked about their professional background, location of their work (state or territory of Australia, North or South Island of New Zealand), health service setting, the population subgroup they care for (adults or children or both) and years of practice.

Health professionals were asked about awareness and influence on their practice of guidelines relating to clinical care in DMD and CMT; namely awareness of international guidelines including DMD diagnosis and management care considerations (‘Guideline 1 (DMD—Care considerations)’) developed for an international context [7, 8, 9], the Clinical practice guidelines for allied health and nursing assessment and management of Duchenne muscular dystrophy (‘Guideline 2 (DMD—Allied health)’), developed for Australian and New Zealand context [10] and the Clinical practice guideline for the management of paediatric Charcot‐Marie‐Tooth disease (‘Guideline 3 (CMT—Paediatric)’) developed for an international context [11].

Health professionals caring for individuals with DMD were asked to self‐report adherence and barriers to adherence to seven evidence‐based and two consensus‐based recommendations for assessments from Guideline 2 (DMD—Allied health). These related to goniometry, handheld dynamometry (HHD), 6‐min walk test (6MWT) [14], North Star Ambulatory Assessment (NSAA) [15], Performance of Upper Limb 2·0 (PUL 2·0) [16], Egen Klassifikation Scale Version 2 (EK2) [17], skinfolds, height and weight (Box 1). These recommendations were chosen as they had either strong evidence or strong consensus supporting the recommendations. Self‐reported adherence reports whether a health professional was using the assessment recommended in Guideline 2 (DMD—Allied health) (either completing the assessment themselves or using information from the assessments completed by another health professional) or not using the recommended assessment. Health professionals could also indicate if the assessment was not relevant to their scope practice (e.g. goniometry would be not relevant for scope of practice of dietitians). Whilst self‐reporting is subject to several limitations including subjectivity and overestimation of true compliance, it was utilised in this survey as it was feasible to collect responses from health professionals across diverse locations [21].

Box 1. Recommendations assessed in the survey.

Guideline 2 (DMD—allied health) recommendations for assessment [10]

Evidence‐based recommendations

• We suggest the use of goniometry to measure range of motion.

• We suggest the use of handheld dynamometry to assess strength.

• We suggest the 6‐min walk test (6MWT) [14] be used to assess mobility.

• We suggest the North Star Ambulatory Assessment (NSAA) [15] be used to assess function.

• We suggest the Performance of the Upper Limb (PUL) [16] assessment be used.

• We suggest the Egen Klassifikation Scale (EK scale) [17] be used to assess activities of daily living in non‐ambulant patients.

• We suggest skinfold measures not be used to estimate body composition.

Consensus based recommendations

• From age 2 to 18 years, height and weight should be measured and assessed at least 6 monthly and tracked using the Centers for Disease Control and Prevention (CDC) 2000 growth charts [18] to identify at risk patterns of growth.

• Over 18 years of age, weight changes should be monitored at least 6 monthly to identify at risk nutritional status.

Guideline 3 (CMT—paediatric) recommendations for exercise and physical activity [11]

Evidence‐based recommendations

• We recommend the use of progressive resistance exercise of the ankle dorsiflexors to improve muscle strength and slow progression of muscle weakness.

Consensus based recommendations

• A tailored strength training programme should be encouraged, under the supervision of the child's treating physician and/or allied health clinician. The clinician should provide guidance in order to ensure that the exercise program is carried out safely, monitor progress and modify the program as appropriate.

• Strength training should start at low resistance and gradually build up (as per generic paediatric recommendations from the American Academy of Sports Medicine [19].

• Strength training of proximal/core muscles (trunk, hip and shoulder girdle) should be encouraged.

• Exercise programmes should include rest days to encourage recovery. i.e. strength training should occur on non‐consecutive days for the same muscle group (as per generic recommendations from the National Strength and Conditioning Association [20].

• Exercise should cease temporarily, and the exercise regimen should be modified, if there are any signs of exercise‐induced muscle damage (e.g. excessive muscle cramping, dark brown urine).

• Stretching of joints prone to contracture should form part of rehabilitation management.

• Activities such as balance retraining or core and postural strengthening tailored to the individual child should be used to improve balance.

• Child‐appropriate or adolescent‐appropriate recreational/community‐based activities (e.g. dance, Pilates, non‐contact martial arts and play‐based therapy) may be used to improve balance.

• Stretching of involved muscle groups may be used for the treatment of cramps.

• Physical therapy such as stretching and strengthening should be used for musculoskeletal pain.

For health professionals caring for children with CMT, we were interested in their awareness and self‐reported adherence (including barriers) to evidence‐ and consensus‐based recommendations from Guideline 3 (CMT—Paediatric) relating to exercise and physical activity (Box 1). Exercise is a planned and structured form of physical activity with the purpose to improve or maintain an element of fitness (strength, endurance, flexibility and/or aerobic fitness) [22]. Physical activity can be planned and organised or informal and incidental [22]. Exercise and physical activity were of particular interest as 88% of international experts in the field had identified this area as a research priority in CMT [11]. These recommendations relate to a single evidence‐based exercise for weakness [23], and other expert‐consensus physiotherapeutic and non‐pharmacological recommendations for the management of weakness, contracture, balance, muscle cramp and musculoskeletal pain [11]. Self‐reported adherence reports whether a health professional was recommending exercise or physical activity for weakness, contractures, balance, cramp and pain, respectively, as outlined in Guideline 3 (CMT—Paediatric). The management of these symptoms are important priorities among healthcare consumers living with CMT [20, 24, 25, 26]. Since Guideline 3 refers to the American Academy of Pediatrics Strength Training by Children and Adolescents Council of Sports Medicine and Fitness (2008) [19] and strength guidelines from the American College of Sports Medicine (ACSM) Exercise testing and prescription [27], we also assessed awareness of these guidelines.

The electronic survey was completed anonymously via a secure, web‐based survey platform (REDCap). All survey data, including incomplete data were included in the analysis. When reporting data, responses relating to profession and geographic location were grouped so as not to identify specific individuals or services.

Data were analysed using descriptive statistics (Stata v. 18) and IBM SPSS Statistics version 25 (2017, Amronk, NY). Associations between guideline awareness and health professional characteristics were explored (Chi² or Fishers exact). A post hoc power analysis (G∗Power 3.1) indicated that considering a sample of 64 participants and α = 0.05, the power for detecting a small (Cohen's w = 0.1), medium (Cohen's w = 0.3) and large (Cohen's w = 0.5) effect in a Chi² analysis was 13%, 67% and 98%, respectively [28, 29].

Qualitative methodology using the Theoretical Domains Framework (TDF) [30] mapped open text answers relating to self‐reported adherence and barriers to uptake of the DMD guidelines. Firstly, operational definitions of the TDF domains in relation to the survey were drafted and agreed upon by three researchers. Then, two researchers independently mapped the responses to one or more of the TDF domains. For each response, one primary domain was selected which the researchers thought mostly strongly represented the open text response. The researchers then met to agree upon the final domain selection(s). Where consensus could not be agreed upon, a third researcher (MK) was consulted. Agreement between the two primary raters was 84% (Cohen's Kappa = 0.4636 indicating moderate agreement).

Ethics approval was provided by The Royal Children's Hospital Melbourne Human Research Ethics Committee (HREC 94359).

3. Results

Responses from 64 health professionals (medical, 20/64 (31%), nursing and allied health, 44/64 (69%)) were received. Most respondents worked in metropolitan locations (54/64, 84%), in a neuromuscular clinic (34/64, 53%) and with children with DMD (44/64, 69%) (Table 1).

Table 1.

Characteristics of survey respondents.

Characteristics	Total n (%)
Location (n = 64)
Metropolitan	54 (84.4%)
Rural	10 (15.6%)
Profession (n = 64)
Medical	20 (31.3%)
Allied health	44 (68.8%)

		Metro		Rural
Physiotherapist	28 (43.8%)	24 (85.7%)		4 (14.3%)
Neurologist	12 (18.8%)	11 (91.7%)		1 (8.3%)
Occupational therapist	8 (12.5%)	5 (62.5%)		3 (37.5%)
Nurse	3 (4.7%)	3 (100%)		0
Dietitian	2 (3.1%)	1 (50%)		1 (50%)
Orthopaedic surgeon	2 (3.1%)	2 (100%)		0
Speech pathologist	2 (3.1%)	2 (100%)		0
Exercise physiologist	1 (1.6%)	1 (100%)		0
Respiratory physician	1 (1.6%)	1 (100%)		0
Other	5 (7.8%)	4 (80%)		1 (20%)
State or territory (n = 64)
Victoria	18 (28.1%)
Western Australia	16 (25%)
New South Wales	15 (23.4%)
Queensland	8 (12.5%)
Northern Territory	2 (3.1%)
Tasmania	2 (3.1%)
Australian Capital Territory	1 (1.6%)
New Zealand	2 (3.1%)
Work Setting (n = 64)
Hospital Neuromuscular Clinic (NMC)	34 (53.1%)
Community	33 (51.6%)
Both NMC and community	3 (4.7%)
Both NMC and Hospital other	1 (1.6%)

Pt age group (n = 64)	DMD		CMT
Child	40 (62.5%)		22 (34.8%)
Adults	14 (21.9%)		10 (15.6%)
Both	4 (6.3%)		2 (3.1%)
Do not see/missing	6 (9.4%)		30 (46.9%)
Years of practice (n = 64)
0 to < 5 years	35 (54.7%)
5 to 15 years	19 (29.7%)
> 15 years	10 (15.6%)

Sixty respondents provided information to explore associations between guideline awareness and professional characteristics (Table 2). Forty (40/60, 67%), 25/60 (42%) and 20/60 (33%) were aware of Guidelines 1 (DMD—Care considerations), 2 (DMD—Allied health) and 3 (CMT—Paediatric), respectively, with no association between guideline awareness and profession. Years of practice was associated with awareness of guidelines. Practitioners with 5 or more years’ experience in neuromuscular diseases were more likely to know about Guideline 1 (p = 0.03) and 3 (p = 0.005). Across all three guidelines, community‐based health professionals were less aware of the guidelines compared to those in specialist neuromuscular clinics. More health professionals working in neuromuscular clinics (n = 33, one did not provide a response to awareness questions) were aware of Guideline 1 (26/33, 79%); fewer were aware of Guideline 2 (17/33, 52%) and Guideline 3 (15/33, 46%) (Table 2). There was a low number of rural‐based respondents, and few were aware of the guidelines.

Table 2.

Associations between guideline awareness (yes) and health professional characteristics.

	Total Cohort n (%)	Guideline 1 DMD—International		Guideline 2 DMD—Allied health		Guideline 3 CMT—Paediatric
		Yes, n (%)	p = *	Yes, n (%)	p = *	Yes, n (%)	p = *
Total	60# (100)	40 (67)	—	25 (42)	—	20 (33)	—
Profession
Medical	20 (33)	14 (70)	0.70	7 (35)	0.46	9 (45)	0.18
Nursing/Allied health	40 (67)	26 (66)		18 (45)		11 (28)
Location
Metropolitan	50 (83)	36 (72)	0.07	22 (44)	0.49	18 (36)	0.47
Rural	10 (17)	4 (40)		3 (30)		2 (20)
Work settinga
Hospital NMC	33 (57)	26 (79)	0.03	17 (52)	0.09	15 (46)	0.03
Community	29 (48)	15 (52)	0.02	8 (28)	0.03	5 (17)	0.01
Patient age groupa
Child	45 (75)	30 (66)	0.10	22 (49)	0.07	17 (38)	0.34
Adults	20 (33)	14 (70)	0.70	6 (30)	0.20	5 (25)	0.33
Years of practice
0 to < 5 years	32 (53)	18 (57)	0.03	10 (31)	0.19	5 (16)	0.005
5 to 15 years	19 (31)	17 (90)		11 (58)		11 (58)
> 15 years	9 (16)	5 (56)		4 (44)		4 (44)

^* χ ² (or Fishers exact (two‐tailed) where cell number were < 5) was used to assess associations between health professional awareness of the guidelines and characteristics. Guideline 1 [7, 8, 9] Guideline 2 [10] Guideline 3 [11].

^a Separate analysis for Work Setting and Patient age group were completed because respondents could tick all that apply meaning some health professionals worked in both hospital and community settings and across children and adults. NMC Neuromuscular clinic.

^# Sample size is 60 as 4 respondents did not provide an answer.

Most health professionals aware of the guidelines reported the guidelines had influenced or somewhat influenced their practice (Guideline 1—37/40 (93%), Guideline 2—21/25 (84%) and Guideline 3—15/20 (75%)). Reasons provided for the guidelines not influencing practice included: unsure how to change their practice (n = 2); lack of resources to change practice (n = 1); lack of support from their organisation to implement clinical guidelines (n = 1); recommendations did not consider the local healthcare context and models of care (n = 1); lack of time to review the guidelines (n = 1); and inability to access guidelines online (n = 1). For Guideline 3 (CMT—Paediatric) three adult‐focused health professionals reported the guideline was not relevant to their area of practice.

Fifty‐eight of 64 (91%) health professionals provided care for children and/or adults with DMD and 24/58 (41%) were aware of Guideline 2 (DMD—Allied health). Survey responses to self‐reported adherence and barriers to use of selected assessment recommendations from Guideline 2 were provided by 52 health professionals (Table 3). Over half of respondents reported using goniometry, NSAA and anthropometric measures either performed by themselves or utilising measures assessed by another health professional. The lowest self‐reported adherence was associated with the recommendation for handheld dynamometry (12/52, 23%) whilst the highest self‐reported adherence was not conducting skinfold measures (47/52, 90%). Approximately half of health professionals who assessed weight and height were completing these measures every 6 months as recommended (Figure 1). Barriers to assessment adherence varied; however, lack of access to equipment/treatment space, lack of knowledge of the assessment and insufficient time were barriers identified across all assessments. Free text comments on ‘other’ barriers to adherence were summarised and mapped to the TDF (Material S2).

Table 3.

Health professionals’ self‐reported adherence and barriers to adherence to assessment measures recommended by Guideline 2 (DMD—allied health) (n = 52).

	n (%)
	Goniometry	Handheld dynamometry	6‐min walk test	North Star Ambulatory Assessment	Performance of upper limb 2·0	Egen Klassifikation Scale Version 2	Skinfoldsa	Weight	Height
Yes, I perform this assessment myself	16 (31)	5 (10)	12 (23)	13 (25)	1 (2)	7 (14)	1 (2)	9 (17)	7 (13)
I use measures from this assessment performed by another health professional	12 (23)	7 (14)	13 (25)	17 (33)	12 (23)	9 (17)	4 (8)	24 (46)	25 (48)
No, I do not use this assessment	12 (23)	28 (54)	12 (23)	8 (15)	26 (50)	21 (40)	31 (60)	9 (17)	12 (23)
This assessment is not relevant to my scope of practice	12 (23)	12 (23)	15 (29)	14 (27)	13 (25)	15 (29)	16 (31)	10 (19)	8 (15)
Why ‘No’?a	(n = 12)	(n = 28)	(n = 12)	(n = 8)	(n = 26)	(n = 21)	N/A	(n = 9)	(n = 12)
Lack of access to appropriate equipment or treatment space	3 (25)	17 (61)	1 (8)	1 (13)	6 (23)	2 (10)		4 (44)	3 (25)
Insufficient training	2 (17)	4 (14)	2 (17)	4 (50)	10 (38)	9 (43)		0 (0)	1 (8)
Insufficient time with patients/workload issues	8 (67)	10 (36)	7 (58)	3 (38)	6 (23)	6 (29)		1 (11)	4 (33)
I don't find it clinically useful	4 (33)	10 (36)	0 (0)	0 (0)	0 (0)	0 (0)		5 (56)	5 (42)
Patients don't like it	1 (8)	0 (0)	1 (8)	0 (0)	0 (0)	0 (0)		0 (0)	0 (0)
I am not familiar with this measure	2 (17)	2 (7)	1 (8)	2 (25)	12 (46)	9 (43)		1 (11)	2 (17)
Other	0 (0)	2 (7)	3 (25)	3 (38)	3 (12)	5 (24)		3 (33)	1 (8)

^a Skinfolds are not recommended so we did not ask why participants did not complete this measure. Self‐reported adherence to this recommendation is reversed scored; 10% are using the measure so adherence to the recommendation is 90%.

Figure 1.

Frequency of weight and height assessments in patients with DMD completed by health professionals.

Content analysis identified five prominent ‘other’ domains influencing health professionals’ use of (or choosing not to use) recommended assessments. The most frequently identified domains were: (1) ‘Intentions’ (both a clear intent to use, as well as intent to not use the assessment in practice); (2) ‘Knowledge’ (unfamiliarity with the assessment or knowledge of the DMD disease process); (3) ‘Beliefs about consequences’ (the assessment would be discouraging for a patient whose physical function was declining); (4) ‘Memory, attention and decision’ (clear decisions to not use the assessment when it did not apply to the specific clinic population, e.g. mostly non‐ambulatory) and (5) ‘Environmental context and resources’ (working in contexts in which the assessment was not appropriate to the age group).

Twenty‐four health professionals provided care in paediatric CMT; only 13/24 (54%) were aware of Guideline 3 (CMT—Paediatric), and all responded to questions on awareness and self‐reported adherence to evidence‐ and consensus‐based recommendations (Table 4). Five health professionals (all physiotherapists) were aware of the American Academy of Pediatrics Strength training by Children and Adolescents statement [19] and six (four physiotherapists and two other) were aware of the ACSM exercise prescription guidelines [27]. Twenty of 24 respondents recommended exercise or physical activity for weakness and to manage pain (83%), 19/24 for contracture (79%), 18/24 for balance (75%) and 15/24 for cramp (63%) (Table 4). Those who did not recommend exercise or physical activity stated it was outside of their scope of practice. Most respondents recommended supervision of strength and balance exercise by physiotherapists (strength n = 20; balance n = 17) and exercise physiologists (strength n = 10; balance n = 6). One respondent recommended parents be trained to supervise exercise and physical activity.

Table 4.

Health professional prescription and barriers to prescription of exercise and physical activity as outlined in Guideline 3 (CMT—paediatric) (n = 24).

	n (%)
	Weakness	Contracturea , b	Balance	Crampc , b	Paind , b
Yes, I do recommend exercise or physical activity	20 (83)	19 (79)	18 (75)	15 (63)	20 (83)
No, I do not recommend exercise or physical activity	4 (17)	5 (21)	6 (25)	8 (33)	5 (21)
Other	—	1 (4)e	—	3 (13)f	5 (21)g
Why ‘No’?b	n = 4	n = 5	n = 6	n = 8	n = 5
Lack of access to appropriate equipment or treatment space	0 (0)	0 (0)	1 (17)	1 (13)	0 (0)
Insufficient training	1 (25)	2 (40)	1 (17)	1 (13)	0 (0)
Insufficient time with patients/workload issues	0 (0)	0 (0)	0 (0)	0 (0)	0 (0)
I don't find it clinically useful	0 (0)	0 (0)	0 (0)	0 (0)	1 (20)
Patients don't like it	0 (0)	0 (0)	0 (0)	0 (0)	0 (0)
I am not familiar with exercise treatment(s) for…	—	1 (20)	1 (17)	3 (38)	0 (0)
It is not within my scope of practice	3 (75)	4 (80)	4 (67)	3 (38)	4 (80)
Other	0 (0)	0 (0)	0 (0)	1 (13)	0 (0)

^a Do you recommend the following exercise or physical activity for contracture management? Options: stretching, other or ‘I do not recommend stretching’.

^b ‘Select all that apply’ Respondents could tick more than one response.

^c Do you recommend the following exercise or physical activity for cramp? Options: Stretching of muscle group(s) for cramp, I do not recommend stretching for cramp, Other.

^d Do you recommend the following physical therapy for musculoskeletal pain? Options: Stretching of muscle group(s) and/or joints or Strengthening exercise, I do not recommend physical therapy for musculoskeletal pain, Other.

^e Contracture, other = serial casting.

^f Cramp, other = heat, massage, warm bath or gentle movement.

^g Pain, other = hydrotherapy, heat or cold, supportive, orthotics or defer to physiotherapist.

Figure 2 illustrates types of exercise prescribed or recommended by health professionals for weakness (n = 20) and balance (n = 18) in paediatric CMT. Half the respondents (10/20, 50%) recommended strengthening be undertaken 2 to 3 times per week, allowing for a rest day between sessions. Stretching was recommended to manage contractures (19/24, 79%) and cramps (15/24, 63%) (Table 4). Twenty of 24 (83%) health professionals recommended stretching and strengthening to manage pain (Table 4). Reported barriers to exercise prescription included insufficient training and a lack of knowledge (familiarity) with prescribing exercise or outside their scope of practice (Table 4).

Figure 2.

Types of exercise and physical activity recommended by health professionals for strengthening (20 respondents; dark grey bars) and balance (18 respondents; light grey bars) in paediatric CMT including those that are evidence‐based and those recommended by the CMT clinical practice guideline. LL, lower limb; PRT progressive resistance training; UL, upper limb.

4. Discussion

Our survey identified approximately two‐thirds of health professionals working in neuromuscular disease were aware of international care considerations for DMD, however only one‐third were aware of Australian‐led international allied health DMD guidelines [10] and paediatric CMT guidelines [11]. In those aware, the majority identified the guidelines had influenced or somewhat influenced their practice. However, less than half of respondents reported using evidence‐based allied health assessments in DMD as recommended in Guideline 2. Despite low guideline awareness, most health professionals working in paediatric CMT prescribed exercise and physical activity reflective of Guideline 3 [11] likely reflecting general clinical practice. In addition, most felt programmes should be supervised by a physiotherapist or exercise physiologist, supporting expert consensus [11].

Awareness is an important factor that promotes guideline implementation [31]. Guideline awareness in our survey was also associated with years of practice with health professionals with 5–15 years of experience reporting greatest awareness. Up to half of the health professionals with more than 15 years of experience were not aware of any of the guidelines, similar to another study reporting a negative correlation between years of practice and evidence‐based practice skills [32]. The length of practice (> 15 years) also reflects the period before DMD guidelines were first published in 2010 [1, 2]. It is also important to consider the influence of recruitment strategies on awareness. Most participants in this small sample were recruited via specialist neuromuscular clinics and networks, so the awareness of neuromuscular guidelines may be increased compared to non‐specialist health professionals.

Considering guideline self‐reported adherence in the context of reported barriers helps to understand evidence‐to‐practice gaps. Common barriers included lack of resources (time and equipment) and paucity of training and knowledge of assessments and appropriate exercise prescription. For example, the lowest self‐reported adherence to DMD assessments was observed for handheld dynamometry (HHD) with access to appropriate equipment or treatment space a commonly reported barrier. Despite good reliability and minimal measurement error [10], quantitative strength testing requires costly equipment (HHD), more typically available in tertiary specialist centres. Equipment restrictions were also highlighted for the measurement of weight, possibly due to lack of wheelchair scales outside of hospital environments. However, under two‐thirds of practitioners reported monitoring weight and height, indicating significant barriers to this practice beyond equipment restrictions. Unsurprisingly, other consistently reported barriers included insufficient time, training and familiarity with the measure. Perceived clinical utility impeded adoption of some assessments, and this sentiment extended to include assessments health professionals perceived were discouraging for their patient, for example measurement of physical function decline. There were less variations in the reported barriers to exercise and physical activity prescription with health professionals only identifying training and unfamiliarity with prescription of exercise. The lower response rate of health professionals working in CMT likely limited the identification of barriers to implementation.

Potential implementation strategies to address the barriers to guideline self‐reported adherence can be identified using theoretical frameworks such as the capability, opportunity and motivation system (COM‐B) and the related behaviour change wheel [33]. Guidelines are in fact an implementation strategy; however, increasing awareness of the guidelines assessed here via communication and/or marketing strategies to both specialist and non‐specialist neuromuscular clinicians could be considered combined with education and subsequent training opportunities, if required. There would also need to be environmental restructuring, requiring financial input, to ensure that resources such as equipment and health professional time are available to adhere to guideline recommendations.

Most available literature assesses guideline compliance to the first international recommendations for DMD [1, 2] and are based on key medical management and monitoring [34, 35, 36, 37]. To our knowledge, there have been no studies assessing compliance to CMT guidelines. A more recent Dutch study assessed barriers to implementing the revised care considerations for DMD [38]. Dutch health professionals identified similar barriers to those found in our analysis including lack of familiarity/awareness, lack of outcome expectancy and external barriers including resources. One point of difference was Dutch health professionals identified lack of consensus with the guideline as a barrier; however, this was not evident in our survey among Australian and New Zealand health professionals. While the respondents to our survey identified assessment recommendations may be discouraging for adult patients with DMD, they did not disagree with the recommendation per se. Guideline 2 (DMD—Allied health) was developed for the Australian and New Zealand healthcare context which may explain the general agreement and highlights the importance of developing or translating existing guidelines within the local healthcare context [38].

Exercise and physical activity modalities are widely used to manage weakness, contracture and balance in many neurological conditions, however application in children with CMT may not be as widely understood. The uptake of progressive resistance training of the foot and ankle muscles by 60% of respondents is likely artificially high reflecting the local experience of neuromuscular health professionals who may be aware of this Australian study [23]. It was interesting to note, hydrotherapy was most frequently prescribed for weakness. Hydrotherapy is a common physiotherapeutic modality, an appealing (and fun) activity and accessible in the community. However, there is little evidence base for hydrotherapy in children with CMT. Only a single study of aquatic therapy versus land‐based therapy in adults with peripheral neuropathies found comparable effects on function [39], with no studies known conducted among children with CMT. This is despite the strong clinical use of hydrotherapy as a therapeutic modality for children. The common reporting of age‐appropriate community‐based recreational and play‐based activities reflects the age group of this patient population and the remit of general paediatric therapists to make exercise and physical activity meaningful (and fun) [40]. The high proportion of health professionals recommending exercise and physical activity likely reflects general clinical practice and management rather than solely awareness of Guideline 3.

The value of standardised assessment in neuromuscular diseases may be unclear to health professionals. Whilst guidelines seek to reduce unwarranted variation and improve equity and access to care, the cost‐effectiveness of providing best practice care in DMD and CMT is difficult to establish. However, this value may become more apparent with emerging disease modifying therapies [12, 13]. To facilitate post approval surveillance, the use of outcome measures and assessments by health professionals outside of clinical trial setting needs to be considered. The NSAA, 6MWT and the PUL are identified by the European Medicines Agency as outcomes to capture treatment efficacy in DMD [18]. In this survey, the use of these assessment and outcomes, either completed by the health professional or performed by another was relatively low. Targeted dissemination and implementation strategies are required to increase the uptake of standardised assessments in readiness to monitor disease modifying therapies across all neuromuscular diseases in the clinical setting [41].

Study limitations included a small sample size, albeit in rare disease in relatively small (country) populations, and a biased group of informed responders likely due to the method of survey distribution (i.e. via neuromuscular professional networks). Therefore, associations should be interpreted with caution. However, the sample size is similar to recent comparisons from the Netherlands [38] and United States [42]. Snowballing prevented calculation of a definitive response rate. Snowball sampling may reduce sample diversity [43]. In this study, snowball sampling within neuromuscular networks may have inflated estimates of guideline awareness as community‐based and rural health professionals who are more likely naïve to guidelines due (potentially) to reduced access to resources (e.g. medical library), supervision or professional development opportunities may not have been reached by the sampling strategy. Equally, rural health professionals are likely to have a negligible neuromuscular case load (e.g. 1–2 patients) and may have felt the survey irrelevant to their practice. Further, there were a relative lower number of health professionals managing paediatric CMT compared to those who care for people with DMD. Greater representation of those managing DMD possibly reflects disease severity and less engagement with health and medical resources by people with CMT despite greater prevalence. Because of these limitations, awareness and self‐reported adherence is likely overestimated and more representative of neuromuscular specialists rather than general health professionals.

This study identified awareness (or lack thereof) of guidelines, evidence‐to‐practice gaps and provides valuable insights into local practice relating to assessment of DMD and exercise and physical activity management in paediatric CMT. The insights gained will inform targeted dissemination and implementation strategies to improve awareness and adherence to guidelines among Australian and New Zealand health professionals and can be used to support similar investigations in other countries. Strategies need to be tailored to the broad range of healthcare professionals providing care for individuals with neuromuscular diseases, as the survey demonstrates characteristics of health professionals influence guideline awareness. Implementation research within neuromuscular diseases is vital to ensure the benefits of clinical practice guidelines, including standardised and equitable care, are realised in anticipation of future therapy development.

Funding

The authors have nothing to report.

Ethics Statement

Ethics approval was provided by The Royal Children's Hospital Melbourne Human Research Ethics Committee (HREC 94359).

Conflicts of Interest

The authors declare no conflicts of interest.

Supporting information

Supporting File 1

Supporting File 2

Kennedy R. A., Klaic M., Donlevy G., et al., “Exploring awareness of and self‐reported adherence to neuromuscular clinical practice guidelines among Australian and New Zealand health professionals: a cross‐sectional survey study,” Journal of Evaluation in Clinical Practice 32 (2026): e70624. 10.1111/jep.70624.

Data Availability Statement

The data that support the findings of this study are available from the corresponding author upon reasonable request.