Publications by authors named "Vandana Ayyar-Gupta"
Article Synopsis
- The North Star ambulatory assessment (NSAA) is a key measure for tracking motor function in boys with Duchenne muscular dystrophy (DMD), but the minimal clinically important difference (MCID) for NSAA is not well-established, complicating result interpretation.
- This study aimed to estimate the MCID for NSAA using various methods, including statistical approaches and patient/parent feedback, resulting in estimates of 2.3-3.5 points for boys aged 7 to 10 years.
- Findings highlighted that patients and parents view a complete loss of function in one item or a decline in one to two items as significant changes, enhancing the understanding of clinical relevance in NSAA results.
Aim: To correlate the North Star Ambulatory Assessment (NSAA) and timed rise from floor (TRF) recorded at age of expected peak with age at loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD).
Method: Male children with DMD enrolled in the UK North Start Network database were included according to the following criteria: follow-up longer than 3 years, one NSAA record between 6 years and 7 years 6 months (baseline), at least one visit when older than 8 years. Data about corticosteroid treatment, LOA, genotype, NSAA, and TRF were analysed.
Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy.
J Cachexia Sarcopenia Muscle
April 2022
Background: Duchenne muscular dystrophy (DMD) is caused by DMD mutations leading to dystrophin loss. Full-length Dp427 is the primary dystrophin isoform expressed in muscle and is also expressed in the central nervous system (CNS). Two shorter isoforms, Dp140 and Dp71, are highly expressed in the CNS.
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- Vamorolone is a synthetic steroid that shows strong anti-inflammatory effects, and earlier studies indicated it improved motor function in boys with Duchenne muscular dystrophy (DMD) after 6 months of high-dose treatment.
- This study aimed to evaluate the outcomes of 30 months of ongoing vamorolone treatment in DMD patients, conducted by the Cooperative International Neuromuscular Research Group across multiple locations.
- Results indicated that while the mean time-to-stand velocity of participants on higher doses of vamorolone showed a slight decrease over the treatment period, this change was not statistically significant, suggesting the need for further research.
Growth pattern trajectories in boys with Duchenne muscular dystrophy.
Orphanet J Rare Dis
January 2022
Objectives: The objective of this study is to analyse retrospective, observational, longitudinal growth (weight, height and BMI) data in ambulatory boys aged 5-12 years with Duchenne muscular dystrophy (DMD).
Background: We considered glucocorticoids (GC) use, dystrophin isoforms and amenability to exon 8, 44, 45, 51 and 53 skipping drug subgroups, and the impact of growth on loss of ambulation. We analysed 598 boys, with 2604 observations.
Objective: To compare arthroscopic hip surgery with physiotherapy and activity modification for improving patient reported outcome measures in patients with symptomatic femoroacetabular impingement (FAI).
Design: Two group parallel, assessor blinded, pragmatic randomised controlled trial.
Setting: Secondary and tertiary care centres across seven NHS England sites.