Email this article The concept of “ambulatory” and “non-ambulatory” in patients with Duchenne muscular dystrophy: definitions and criteria
- Authors: Gremyakova T.A.1, Artemyeva S.V.2, Vashakmadze N.D.3, Vitkovskaya I.P.4, Guzeva V.I.5, Guzeva O.V.5, Kuzenkova L.M.6, Mikhailova S.V.7, Nazarenko L.P.8, Pervunina T.M.9, Pechatnikova N.L.4, Podkletnova T.V.6, Sakbaeva G.E.10, Stepanov A.A.10, Suslov V.M.5, Gremyakova O.I.11, Shakhovskaya N.I.12, Nikitin S.S.13
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Affiliations:
- Charitable Foundation “Gordey”
- Yu. E. Veltishchev Research Clinical Institute of Pediatrics
- Central Clinical Hospital of the Russian Academy of Sciences
- Morozov Children’s City Clinical Hospital of the Department of Health of the City of Moscow
- Saint Petersburg State Pediatric Medical University
- National Medical Research Center for Children’s Health
- Russian Children’s Clinical Hospital
- Research Institute of Medical Genetics
- Institute of Perinatology and Pediatrics
- Central Clinical Hospital with a Polyclinic of the President Administration
- Charitable Foundation “Gordey
- Psycho-neurological Hospital for Children with CNS Lesions with Mental Disorders
- Research Centre for Medical Genetics
- Issue: Vol 12, No 2 (2022)
- Pages: 10-18
- Section: LECTURES AND REVIEWS
- Published: 09.06.2022
- URL: https://nmb.abvpress.ru/jour/article/view/484
- DOI: https://doi.org/10.17650/2222-8721-2022-12-2-10-18
- ID: 484
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Abstract
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disease due to a mutation in the gene encoding dystrophin synthesis. In patients, muscle damage and atrophy progresses, the ability to move independently decreases as well as respiratory and cardiac functions. At various stages of the disease, different methods of care and treatment of patients with DMD are used. The clinical effect of new methods of DMD target therapy may depend on the stage of development of the disease (ambulatory or non‑ambulatory). To date, there are no unified criteria for assessing the status of a patient in terms ambulatory. In clinical trials and real clinical practice, different approaches are used to assess the patient’s status. However, the conclusion about the functional capabilities is critical for patients with DMD as approaches in management of patients in ambulatory and non‑ambulatory stages differ significantly. This necessitates expert consensus to achieve consistency and avoid any of discrepancies on that issue.
The paper reviews the available published data on the concepts of “ambulatory” and “non‑ambulatory” used in clinical trials, real clinical practice, international standards and recommendations. As a conclusion of this analysis, it is proposed in real clinical practice to interpret “ambulation” in DMD patients as ability to walk without the use of assistive devices and without specifying the distance and time, and “non‑ambulation” as condition in which the patient is forced to constantly use a wheelchair both indoors and outdoors.
About the authors
T. A. Gremyakova
Charitable Foundation “Gordey”
Author for correspondence.
Email: tag@dmd-russia.ru
ORCID iD: 0000-0001-7317-3971
Tatyana Andreevna Gremyakova
29 Sokolovo-Meshcherskaya St., Moscow 125466
Russian FederationS. V. Artemyeva
Yu. E. Veltishchev Research Clinical Institute of Pediatrics
Email: fake@neicon.ru
ORCID iD: 0000-0002-8876-7462
2 Taldomskaya St., Moscow 125412
Russian FederationN. D. Vashakmadze
Central Clinical Hospital of the Russian Academy of Sciences
Email: fake@neicon.ru
ORCID iD: 0000-0001-8320-2027
1A Litovskiy Boulevard, Moscow 11759
Russian FederationI. P. Vitkovskaya
Morozov Children’s City Clinical Hospital of the Department of Health of the City of Moscow
Email: fake@neicon.ru
ORCID iD: 0000-0002-0740-1558
1/9 4th Dobryninskiy Lane, Moscow 119049
Russian FederationV. I. Guzeva
Saint Petersburg State Pediatric Medical University
Email: fake@neicon.ru
2 Litovskaya St., Saint Petersburg 194100
Russian FederationO. V. Guzeva
Saint Petersburg State Pediatric Medical University
Email: fake@neicon.ru
2 Litovskaya St., Saint Petersburg 194100
Russian FederationL. M. Kuzenkova
National Medical Research Center for Children’s Health
Email: fake@neicon.ru
ORCID iD: 0000-0002-9562-3774
Build. 1, 2 Lomonosovskiy Prospect, Moscow 119991
Russian FederationS. V. Mikhailova
Russian Children’s Clinical Hospital
Email: fake@neicon.ru
ORCID iD: 0000-0002-2115-985X
117 Leninskiy Prospekt, Moscow 119571
Russian FederationL. P. Nazarenko
Research Institute of Medical Genetics
Email: fake@neicon.ru
ORCID iD: 0000-0002-1861-433X
10 Ushayka River Embankment, Tomsk 634050
Russian FederationT. M. Pervunina
Institute of Perinatology and Pediatrics
Email: fake@neicon.ru
ORCID iD: 0000-0001-9948-7303
2 Akkuratova St., Saint Petersburg 197341
Russian FederationN. L. Pechatnikova
Morozov Children’s City Clinical Hospital of the Department of Health of the City of Moscow
Email: fake@neicon.ru
ORCID iD: 0000-0002-2445-132X
1/9 4th Dobryninskiy Lane, Moscow 119049
Russian FederationT. V. Podkletnova
National Medical Research Center for Children’s Health
Email: fake@neicon.ru
ORCID iD: 0000-0001-6415-156X
Build. 1, 2 Lomonosovskiy Prospect, Moscow 119991
Russian FederationG. E. Sakbaeva
Central Clinical Hospital with a Polyclinic of the President Administration
Email: fake@neicon.ru
ORCID iD: 0000-0002-3651-851X
15 Marshala Timoshenko St., Moscow 121359
Russian FederationA. A. Stepanov
Central Clinical Hospital with a Polyclinic of the President Administration
Email: fake@neicon.ru
ORCID iD: 0000-0001-7634-5783
15 Marshala Timoshenko St., Moscow 121359
Russian FederationV. M. Suslov
Saint Petersburg State Pediatric Medical University
Email: fake@neicon.ru
ORCID iD: 0000-0002-5903-8789
2 Litovskaya St., Saint Petersburg 194100
Russian FederationO. I. Gremyakova
Charitable Foundation “Gordey
Email: fake@neicon.ru
ORCID iD: 0000-0001-8607-1819
29 Sokolovo-Meshcherskaya St., Moscow 125466
Russian FederationN. I. Shakhovskaya
Psycho-neurological Hospital for Children with CNS Lesions with Mental Disorders
Email: fake@neicon.ru
ORCID iD: 0000-0003-3117-636X
1 Ivana Susanina St., Moscow 127486
Russian FederationS. S. Nikitin
Research Centre for Medical Genetics
Email: fake@neicon.ru
ORCID iD: 0000-0003-3292-2758
1 Moskvorechye St., Moscow 115522
Russian FederationReferences
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