DMD 2
DMD 2
DMD 2
Al-Madinah Al-Munawara
Ministry of Health Maternity and Children’s Hospital
Medical Rehabilitation Dep. Physical Therapy
Done by:
Ahdab Aljohani
Arwa Al-johani
Study 1:
Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an
external pilot randomized controlled trial
P (Population):
40 ambulatory boys aged from 7-16 years old with genetically confirmed
DMD, North Star ambulatory assessment < 8
I (Intervention):
Standardised AT was prescribed and delivered by an AT- trained
physiotherapist (with specialist knowledge of DMD), based on the study
protocols detailed in a manual, in 30-min sessions twice weekly in an NHS
pool with a temperature of 34–36 °C. Standardised LBT stretches and
exercises were prescribed by a specialist physiotherapist at the baseline
appointment, based on the study protocols detailed in a manual.
C (Comparison):
they were randomly allocated in a 1:1 ratio by a centralised web-based
randomisation system provided by the Sheffield Clinical Trials Research Unit
(CTRU) to AT plus land-based therapy (LBT) or LBT alone , the control group
receive AT plus (LBT )
Those randomised to receive AT were asked to perform LBT on four of the
other 5 days of the week, while those in the control group were asked to
perform LBT 6 days/week
O (Outcome)
Over 6 months, 348 boys were screened: most lived too far from centres or were
enrolled in other trials; 12 (30% of the targets) were randomised to AT (n = 8) or
control (n = 4). The mean change in NSAA at 6 months was −5.5 (SD 7.8) in the
control arm and −2.8 (SD 4.1) in the AT arm. Harms included fatigue in two boys,
pain in one. Physiotherapists and parents valued AT but believed it should be
delivered in community settings. Randomisation was unattractive to families, who
had already decided that AT was useful and who often preferred to enrol in drug
studies. The AT prescription was considered to be optimised for three boys, with
other boys given programmes that were too extensive and insufficiently focused.
Recruitment was insufficient for VoI analysis.
Study 2:
Efficacy of two intervention approaches on functional walking capacity and
balance in children with Duchene muscular dystrophy
P (Population):
Thirty ambulatory boys aged from 6-10 years were diagnosed with DMD, having
grade 3+ muscle strength in lower limbs and trunk muscles and having a
functional ROM for UL and LL joints.
I (Intervention):
Group A: 15 Children were trained on the bicycle ergometer for 20 min, at three times a
week for three successive months. Group B: 15 children underwent treadmill training for
20 minutes. Both groups received a PT program 3x/ week.
The program included gentle stretching for (biceps brachii, hamstrings, and calf muscles).
Isometric for (quadriceps, hamstrings, anterior tibial group, calf muscles, biceps and
triceps). Every contraction was held and relaxed for 5, 5 reps.
Reference: Sherief, A. E. A. A., Abd ElAziz, H. G., & Ali, M. S. (2021). Efficacy of two intervention approaches on functional walking
capacity and balance in children with Duchene muscular dystrophy. Journal of musculoskeletal & neuronal interactions, 21(3), 343–350.
Study 3:
Assisted Bicycle Training Delays Functional Deterioration in Boys With Duchenne
Muscular Dystrophy: The Randomised Controlled Trial “No Use Is Disuse”
P (Population): the study population age < 6 and Boys were eligible if
they were in their late ambulatory phase and showed a labored gait and/or had
difficulties with rising from the floor. Boys were included if they needed more
than 5 seconds to rise from the floor, were not able to rise from the floor, were not
able to cycle without electric assistance, or needed a wheelchair to move over a
long (>500 m) distance. Wheelchair-dependent boys were eligible if they were
able to touch the top of their head with both hands or were able to use a hand-
operated wheelchair
I (Intervention): the intervention was assisted bicycle training of the legs and
arms during 24 weeks.
C (Comparison): a 2:1 ratio the the control group received usual care during
24 weeks. Usual care means that boys were allowed to continue their activities,
including physiotherapy, but they did not receive any specific intervention. The
control group received the same training after the 24-week waiting period. The
intervention and control groups were followed up until 56 and 60 weeks after
study entry
O (Outcome):
The results suggest that assisted bicycle training of the legs and arms is feasible
and safe for both ambulant and wheelchair-dependent children and may decline
the deterioration due to disuse. Progressive deterioration, however, may
compromise the design of trials for DMD.