Steroid versus non-steroid regimen in treating duchenne muscular dystrophy: an observational study from south India
By: Sattenapalli, N. C
.
Contributor(s): Areti, A. R.
Publisher: Mumbai Indian Journal of Pharmaceutical Science 2022Edition: Vol.84(1), Jan-Feb.Description: 80-86p.Subject(s): PHARMACEUTICSOnline resources: Click here
In:
Indian journal of pharmaceutical sciencesSummary: There are no large studies from Southern India describing the treatment regimen of the Duchenne
muscular dystrophy which is the most common muscular dystrophy. The current observational study
aimed to compare steroid versus non-steroid treatment outcomes in Duchenne muscular dystrophy
diagnosed population of South India. Immunohistochemically/genetically confirmed Duchenne muscular
dystrophy patients diagnosed between February 2019 and March 2021 were ambispectively included. The
main outcomes assessed in the study were loss of ambulation with respect to age, disease progression of
motor milestones, medical research council grade, change in body weight and reported adverse events.
We correlated these outcomes comparatively for both intervention groups (steroid and non-steroid). A
total of 274 Duchenne muscular dystrophy patients were included in the study. The mean minimal age of
onset of disease was 2.0±1.2 y. 32.85 % patients were treated with steroids and 67.15 % patients were on
non-steroidal treatment. Comparatively, both regimen using patients demonstrated loss of ambulation at
13 y of age. Gait, stairs, Gower, chair scores reported loss of motor functions at 13 y and 25 y for steroid
and non-steroid using patients respectively. Medical research council grade improved in steroid using
patients to a greater extent compared to non-steroid using patients. Change in body weight and adverse
events were comparatively low in non-steroid using patients to that of steroid using patients. The pattern
of primary outcome measures in this large cohort is comparable despite variability in medical care. The
contemporary observational study adds to the real-world evidence in guiding clinicians towards better
approach in treating Duchenne muscular dystrophy patient populationa.
| Item type | Current location | Call number | Status | Date due | Barcode | Item holds |
|---|---|---|---|---|---|---|
Articles Abstract Database
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School of Pharmacy Archieval Section | Not for loan | 2022-2222 |
There are no large studies from Southern India describing the treatment regimen of the Duchenne
muscular dystrophy which is the most common muscular dystrophy. The current observational study
aimed to compare steroid versus non-steroid treatment outcomes in Duchenne muscular dystrophy
diagnosed population of South India. Immunohistochemically/genetically confirmed Duchenne muscular
dystrophy patients diagnosed between February 2019 and March 2021 were ambispectively included. The
main outcomes assessed in the study were loss of ambulation with respect to age, disease progression of
motor milestones, medical research council grade, change in body weight and reported adverse events.
We correlated these outcomes comparatively for both intervention groups (steroid and non-steroid). A
total of 274 Duchenne muscular dystrophy patients were included in the study. The mean minimal age of
onset of disease was 2.0±1.2 y. 32.85 % patients were treated with steroids and 67.15 % patients were on
non-steroidal treatment. Comparatively, both regimen using patients demonstrated loss of ambulation at
13 y of age. Gait, stairs, Gower, chair scores reported loss of motor functions at 13 y and 25 y for steroid
and non-steroid using patients respectively. Medical research council grade improved in steroid using
patients to a greater extent compared to non-steroid using patients. Change in body weight and adverse
events were comparatively low in non-steroid using patients to that of steroid using patients. The pattern
of primary outcome measures in this large cohort is comparable despite variability in medical care. The
contemporary observational study adds to the real-world evidence in guiding clinicians towards better
approach in treating Duchenne muscular dystrophy patient populationa.
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