Good psychosocial and physical care has a positive influence on life satisfaction and life expectancy in many diseases, including Duchenne Muscular Dystrophy [1,2,3,4,7]. Duchenne Muscular Dystrophy (DMD) is the most common genetic neuromuscular disorder in children. The pooled global DMD birth prevalence is 19.8 (95% CI:16.6–23.6) per 100,000 live male births [8]. The absence, or near absence, of the protein dystrophin results in a steady loss of vulnerable muscle fibers together with a loss of muscle mass and muscle strength. Patients, mainly boys and men because of its x-linked chromosomal recessive character, become wheelchair-dependent in their early teenage years and subsequently lose their arm functions. Although there are promising treatments that can delay progression, many of these are still in a research stage. This means that patients have to rely on optimal preventative and symptomatic care that may extend their life expectancy to over 30 years of age with good quality of life [7,8,9,10,11]. With increased life expectancy, there is now a significant group of adult patients with DMD. Adult patients with DMD have their own specific challenges, requiring a different and more multisystemic organization of healthcare than children [12].

International care considerations for children with DMD [13,14,15] and additional guidelines for adult patients with DMD have been published [16, 17]. The aim of the care considerations and guidelines is to standardize care for everyone living with DMD and to reach optimal quality of life and life expectancy. Care considerations include 11 topics: considerations for diagnosis, neuromuscular rehabilitation, endocrine, gastrointestinal, respiratory, cardiac, bone health, orthopedic, emergency, psychosocial management, and transition of care across the lifespan. Consensus guidelines for adults describe the focus of care shifting from a preventive towards a treatment approach. Therapy guidelines for adults assist therapists in their clinical practice. Although care considerations and consensus guidelines are freely available online, it is well known that compliance is suboptimal due to multiple reasons [18,19,, 19]. Disregarding the care considerations increases the risk of preventable medical complications and differences in quality of life and life expectancy between care centers and individual patients.

Currently, there are no international agreements on the implementation of the care considerations and guidelines for DMD. Care centers can make their own choice to follow the considerations or not, which increases the risk of considerable differences in the care provided, together with all the associated risks. Patients and families may not be aware of differences in quality of care and are often in a position of being dependent on their care provider. An accreditation or certification program can help overcome this problem. Accredited Duchenne care centers recognize the need for and provide up-to-date, high quality and uniform care. In the United States, Parent Project Muscular Dystrophy (PPMD) has developed the Certified Duchenne Care Center Program [20], but a worldwide applicable accreditation program was lacking. For this reason, the World Duchenne Organization initiated the global Accredited Duchenne Centers (ADC) Program. The aim of the ADC Program is to achieve global, uniform and up-to-date care for all people living with DMD. To stay up to date means also that continuous education is part of the program. Duchenne care centers can obtain accreditation if they provide care according to the international care considerations, consensus guidelines and/or expert opinions. Both healthcare professionals and patients and families are involved in the development of the ADC Program. The aim of this paper is to describe the development and content of the ADC Program.

This paper describes the developmental process and content of the Accredited Duchenne Centers (ADC) Program. The program started in spring 2022 with the preparation phase, in which a program team was formed and the Advisory Board was set up. A definition of accreditation was established (phase 0). Meanwhile, accreditation criteria have been defined based on the international consensus statements, guidelines and expert opinions. Subsequently, pre-visit questionnaires and visitation interviews were developed (phase 1). Also in phase 1, building a database was begun. During the pilot phase (phase 2), there was a call for applications and Duchenne care centers were recruited to participate. The last phase (phase 3) is the implementation of the program worldwide.

Starting the process

The Accredited Duchenne Centers (ADC) Program was initiated and is financed by the World Duchenne Organization (WDO). WDO approached the first author, and together with the WDO a program team was established. The ADC Program team consists of the authors of this article.

Members of the Advisory Board are all professionals with expertise in the field of DMD. Members were recruited via the WDO network. Next to professionals, patient representatives were asked to join the Advisory Board to guarantee the input of patients from the beginning of the program. The international Advisory Board consisted initially of 11 members with a multidisciplinary background from 8 different countries, and is chaired by Prof. Nathalie Goemans. Recently a 12th member has joined the advisory board (see here for members https://accreditedduchenne.center).

The ADC Program team and Advisory Board reached consensus on the following definition for accreditation for children and/or adults: “Accredited Duchenne Centers for children and/or adults provide care according to criteria that are in agreement with the latest care considerations and/or expert opinions for children and/or adults. Care is provided by a multidisciplinary team and may be delivered either by a single center, or shared with other locations; however, the applying center is responsible for the care plan, coordination, communication, documentation and collaboration. Although the definition is comparable for both children and adults, the care considerations or consensus statements on which the criteria are based differ.

A structured process consisting of a literature review by the ADC Program team resulted in a first concept of accreditation criteria for children and adults. Accreditation criteria were primarily based on the latest care considerations and consensus and therapy guidelines for children and adults. Accreditation criteria were reviewed and discussed with the Advisory Board. The first set of accreditation criteria was adjusted based on feedback from the Advisory Board. The current set of accreditation criteria for children and adults for which agreement was reached can be found in Appendix 1 and 2. A minimum set of accreditation criteria was determined and has to be achieved before a visitation can be planned (these are mentioned in Appendix 1 and 2 as SCREENING).

The accreditation procedure was developed by the ADC Program team and the Advisory Board. The process consists of five steps (step 1 Registration, step 2 Screening, step 3 Visitation, step 4 Accreditation, step 5 Maintaining accreditation) which will be further explained in the results section.

Step 1 registration

Centers that apply for accreditation need to start by registering. This can be done via the ADC-website (https://accreditedduchenne.center). Centers complete and submit their registration form on the ADC website. Centers can apply for accreditation for children or adults or for both.

Step 2 screening

After registration, Duchenne centers receive a link to a digital pre-visitation questionnaire to determine whether they provide care according to the care considerations, and/or consensus and therapy guidelines. There are two separate questionnaires, one for pediatric care and one for adult care. Together with the questionnaire(s) centers will be asked to submit a letter of support from the national patient organization. The completed questionnaire(s) and a letter of support are both required prior to a visitation. After assessment of the pre-visitation questionnaire and possible additional questions from the ADC Program team, provided the center fulfilled the pre-visitation criteria, a visitation to the center is planned.

Step 3 visitation

A visitation is performed by a visitation committee consisting of a group of experienced professionals and a patient representative. (Semi) structured interviews, based on all criteria, are conducted with individual professionals (physicians, therapists, psychologists, social workers, nurses) and patients and their families. Interviews per topic of management take approximately 30–90 min. A visitation takes two days if care is given either to pediatric or adult patients. Visitation of a center with a pediatric and an adult team that applies for both pediatric and adult accreditation requires 2–3 days, depending on whether there is one team for both patient groups or two separate teams.

In addition, the national supporting patient organization is asked to send a questionnaire to members who receive care in the applying center. The questionnaires sent to patients receiving care in the applying center are closed questions based on the criteria combined with open questions to allow for specific comments and voicing preferred changes in care management. Their answers and the answers given in the interviews of the patients and their families during the visitation are compared to the answers provided by the professionals on several topics.

Interviews with professionals are recorded and stored according to the FAIR data principles (see results) for which the professionals signed informed consent.

Step 4 accreditation

Interviews are reviewed by the visitation committee, and in case of doubt the Advisory Board is asked for their advice. If criteria for accreditation are met, centers become an Accredited Duchenne Center for pediatric and/or adult care. Centers providing most care according to the care considerations, but not meeting all criteria for accreditation yet, receive advice to make some (minor) changes to the care provided based on written feedback by the ADC Project Team. Follow-up contact by email or an online meeting is planned when these centers have processed the changes. Centers can then obtain their accreditation status.

Step 5 maintaining accreditation

Accreditation will be maintained for five years (unless major changes in health care organization and/or the care provided are made, which the centers are obliged to report). During these five years Accredited Duchenne Centers will need to follow the ongoing education program provided by the WDO in order to remain up-to-date. An online re-accreditation procedure will be scheduled after five years (or earlier if necessary). If needed a new visitation will take place.

To enable transparency of the whole process of accreditation, a FAIR database was set up [21, 22]. FAIR means Findable, Accessible, Interoperable and Reusable. With the help of Mark Wilkinson (BBVA-UPM Industry Chair on Biotechnology, Co-Founder of FAIR Data Systems, Spain) the FAIR database was constructed and the results of the pre-visitation questionnaires and the patients’ questionnaires are directly entered in the database. The FAIR database is queryable as both questions and answers are coded with unique identifiers and transformed into machine-language format, using the Resource Description Framework Standards (W3C Semantic Web Standards, Resource description framework (RDF) https://www.w3.org/RDF/). This makes it distinct from a standard database, which is a long-term document/file repository. It is therefore “ready” to be integrated in the future with third-party data. It is the intention to store the results of the interviews in the database as well. The aim of this database is to gain insight into the barriers and issues to meeting specific criteria and to focus support and education on these topics. An additional database was set up where raw data can be stored, such as the recordings of the interviews. This can be used to listen back in case of confusion or dispute.

Centers that care for persons with DMD were invited to apply for accreditation in February 2023. Interested centers submitted their registration on the ADC website. Several centers applied and 6 centers were selected that fulfilled the visitation criteria based on the pre-visitation questionnaire and were supported by a national patient organization. Three visitations were performed, and after minor changes in care management, all 3 were accredited. After the pilot phase, interviews with the patients and their families were adjusted to a more open question interview. Patient interviews and questionnaires gave good insight into the perspective of patients and their families regarding the care provided by the applying center. These are compared with the answers given by the professionals and can potentially be discussed with the professionals.

Two fully prepared visitations had to be canceled due to reasons beyond our control; one visitation was canceled by the applying center. This experience has changed our procedure regarding visitations: we now ask the applying center to confirm the visitation dates and if they cancel they have to pay a fee to cover any costs already incurred by the WDO.

The aim of this Accredited Duchenne Centers Program is to attain worldwide uniform and up to date optimal care for persons with Duchenne Muscular Dystrophy. For this reason, criteria were formulated based on the standards of care, care considerations and consensus statements. All applying centers will be judged in accordance with these criteria independent of the country and health system they are working in the Certified Duchenne Care Center Program developed in the USA [20] is highly comparable as it is based on the same care considerations. However, this program is focused on the situation in the USA and not every country has the same health organization. We tried to formulate our criteria to be applicable worldwide, as there are several ways to fulfill a criterion. For example, wheelchair prescriptions can be written by occupational therapists or physiotherapists or by a special aids team etc. To reach the criterion attention, must be given to seating position, the ability to still perform activities, sitting without pain and adaptability.

The benefits of the whole process to reach accreditation are a full screening of the team and the care program of the applying center. Suggestions to improve are made and new insights can be gained based on the experience of how other centers provide care. During the application process, the pre-visitation questionnaires and interviews already enable centers to make changes that are supported by the hospital in which they are centered. It gives the patient organization(s) in that country confidence to refer patients and parents to a center working according to the latest care considerations and guidelines. Ongoing education also helps the centers stay up to date. The aim is to build a network of accredited centers that can request advice and learn from each other. The possibility of becoming a member of a visitation team if your center is already accredited will also be offered; this will help to spread knowledge around the world.

A FAIR data system is in place to guarantee a transparent procedure. These are some of the advantages of having a FAIR database:, 1) Records deposited in a ‘container’ are assigned a globally unique persistent identifier (PID) 2) Access to datasets are controlled and include a license of use 3) Metadata remain available over time, even if the dataset is no longer available and 4) Metadata describing the datasets will use controlled vocabularies. Keeping the ADC datasets FAIR over time requires professional data curation and digital preservation [21]. The collected data will be analyzed to identify which criteria are hard to meet for most centers. These results will inform management of the education program. The World Duchenne Organization (WDO) has a yearly Duchenne Care Conference and this year (2024) one of the topics was based on the results of the ADC Program. Specific topics can be addressed through webinars. The attendance of a team at the follow- up education program will be registered.

After discussion with the Advisory Board on the timelines required to update and eventually adjust the criteria, it was agreed to revise the criteria every 5 years. If, however, new guidelines are published or new insights on a specific care field require major changes in care, the criteria will be adjusted sooner and the educational program will focus on these aspects.

Currently the program is being implemented worldwide (phase 3), with a number of visitations performed (N = 10) and planned (N = 1).

The best conclusion is a quote from one of the centers that has been visited and accredited: ´It has been a great privilege and honor to be selected for a visit as part of this ADC Program. We are a passionate team, and the accreditation process has confirmed the standards of care we set and excel in as a team and has also helped identify areas for improvement which we are committed to addressing, with the ultimate goal of trying to achieve excellence for our patients.”

ADC program:

Accredited duchenne centers program

DMD:

Duchenne muscular dystrophy

WDO:

World duchenne organization

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IJMDG is ADC Program leader, KP is ADC Program manager, MJ is Program support manager, sab is Program communication advisor/website manager, NL is Program data manager, EV is Program patient representative, NG is Chair of the ADC Advisory board.

Correspondence to Imelda J. M. de Groot.

Ethical approval was not required: the centers in the pilot phase agreed to participate by applying for accreditation and signing informed consent.

Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Centers that apply for accreditation for children and meet the criteria can become an Accredited Duchenne Care Center for Children. Criteria for children are in agreement with the latest Standards of Care (SoC), consensus guidelines and/or expert opinions.^1,2,3

Criteria for Accreditation for children are (SCREENING means that this criterion is also used in the pre-visitation questionnaire as well as in the visitation interviews):

Genetic testing is used to confirm the diagnosis (SCREENING)

Ambulatory children:

Additionally, in non-ambulatory children:

The primary care provider is part of the care team and works in partnership with the multidisciplinary team

Birnkrant et al. (2018). Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointenstinal and nutritional management. Lancet Neurology; 17(3):251-267.

Birnkrant et al. (2018). Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurology; 17(4):347-361.

Birnkrant et al. (2018). Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across lifespan. Lancet Neurology; 17(5): 445-455.

Accredited Duchenne Centers Program

Criteria for Accreditation – adults (aged ≥ 18 years).

Centers that apply for accreditation for adults and meet the criteria can become an Accredited Duchenne Care Center for Adults. Criteria for adults are in agreement with the latest Standards of Care (SoC), consensus guidelines and/or expert opinions.^1,2,3,4,5

Criteria for Accreditation for adults are (SCREENING means that this criterion is also used in the pre-visitation questionnaire, next to the use in the visitation interviews):

Bone health management

Orthopedic management

• Consider posterior spine fusion during the late non-ambulatory stage for adults with a progressive curve in defined situations (consult with the respiratory physician and cardiologist to ensure that lung and heart function are sufficient to proceed with the surgical intervention)

• Consider surgery on foot and Achilles tendon in selected situations

Participation, psychosocial and palliative care

• A mental health clinician (such as a psychologist or social worker) is part of the multidisciplinary team (SCREENING)

• Mental health and quality of life of adults with DMD and their families are assessed at every clinic (SCREENING)

• Adults with DMD and their families receive ongoing mental support (SCREENING)

• A clinical neuropsychologist is part of the multidisciplinary team, or is available to the team (SCREENING)

• Neuropsychological evaluations and interventions for learning, emotional and behavioral problems are provided (SCREENING)

• Needs for vocational support are assessed

• Age-appropriate independence and social development are encouraged

• A psychiatrist is available to the team (SCREENING)

• Anti-depressant therapy, anxiolytic and/or psychological therapy is considered where appropriate

• Adult DMD patients have the opportunity to discuss end of life issues

• Symptom management is provided to patients with more advanced disease

• Adult DMD patients have access to a palliative care consultant

Transition

• There is close contact between the pediatric and adult health services to facilitate transition and transfer to adult health services (with the pediatric team being responsible for the transition process)

• Families of (young) adults with DMD might need guidance on guardianship or conservatorship

Primary care

• The primary care provider is part of the care team and works in partnership with the multidisciplinary team

Perioperative and anesthetic care

• Anesthetic care is preferably provided by centers with expertise in the care of DMD patients (this may not always be possible, for example in case of an emergency)

• In case of an emergency setting where transfer to the (expert) center is not possible, advice is given to the local hospital (involved) by the expert center (if the expert center knows that a patient is hospitalized in a local hospital; patients are educated by the expert center to inform professionals in local hospitals to get in contact with the expert centers)

• There is always a pre-anesthetic assessment which includes assessments of 1) airway (mouth opening), 2) cardiac status (echo/cMRI) and 3) respiratory status (SCREENING)

• There is close liaison between the surgical, anesthetic, cardiac and respiratory teams

• The anesthetists are aware that DMD patients are at risk of cardiac and respiratory decompensation and steroid induced adrenal insufficiency during and after surgery

• The anesthetists are aware of the risk of acute rhabdomyolysis when using halothanes

Emergency management

• An emergency card is recommended for DMD patients

• In case of an emergency setting where transfer to the (expert) center is not possible, advice is given to the local hospital (involved) by the expert center (if the expert center knows that a patient is hospitalized in a local hospital; patients are educated by the expert center to inform professionals in local hospitals to get in contact with the expert centers)

• Adult DMD patients have a plan to manage steroid-induced adrenal insufficiency during acute illness

Patient satisfaction

Monitoring patient satisfaction with health care services regularly

References

Birnkrant et al. (2018). Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointenstinal and nutritional management. Lancet Neurology; 17(3):251-267.

Birnkrant et al. (2018). Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurology; 17(4):347-361.

Birnkrant et al. (2018). Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across lifespan. Lancet Neurology; 17(5): 445-455.

Narayan S et al. (2022). Adult North Star Network (ANSN): Consensus Document for Therapists Working with Adults with Duchenne Muscular Dystrophy (DMD) – Therapy guidelines. Journal of Neuromuscular Diseases; 9:365-381.

Quinlivan R et al. (2021). Adult North Star Network (ANSN): Consensus Guideline For The Standard Of Care Of Adults With Duchenne Muscular Dystrophy. Journal of Neuromuscular Diseases; 8:899-926.

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