Study reference

Study characteristics

Patient characteristics ²

Intervention (I)

Comparison / control (C) ³

Follow-up

Outcome measures and effect size ⁴

Comments

PICO 1

Suk,

2014

Type of study: prospective cohort study

Setting and country: single centre, Korea

Funding and conflicts of interest: No external funding sources were utilized in this study. None of the authors received payments or services, either directly or indirectly (i.e., via his or her institution), from a third party in support of

any aspect of this work. None of the authors, or their institution(s), have had any financial relationship, in the thirty-six months prior to submission of this

work, with any entity in the biomedical arena that could be perceived to influence or have the potential to influence what is written in this work. Also, no

author has had any other relationships, or has engaged in any other activities, that could be perceived to influence or have the potential to influence what

is written in this work.

Inclusion criteria: male patients with Duchenne muscular dystrophy and scoliosis, referred by the neurology and neuromuscular rehabilitation group.

Exclusion criteria:

-

N total at baseline:

Intervention: 45

Control: 32

Important prognostic factors²:

Age, mean ± SD:

I: 14.9 year ± 4.2

C: 14.8 year ± 3.6

Groups comparable at baseline? yes

Describe intervention:

instrumented spinal

arthrodesis from the thoracic to the lower lumbar spine (11.1%) or the pelvis (89.9%) with CD Horizon spinal instrumentation (Medtronic, Memphis,

Tennessee)

Describe control:

Not reported

Length of follow-up:

I: 46.4 months ± 18.7

C: 47.1 months ± 23.7

Loss-to-follow-up:

Intervention:

5 (11%)

Reasons

Death; n=2

Lost to follow up; n=3

Control:

6 (19%)

Reasons

Death; n=3

Lost to follow up; n=3

Incomplete outcome data:

Not reported

See tabel 1.

Authors conclusion:

Surgery in patients who had Duchenne muscular dystrophy with scoliosis improved function and decreased

the rate of deterioration of forced vital capacity compared with patients treated conservatively. However, the muscle power

and forced vital capacity decreased in both groups.

Kennedy, 1995

Type of study: prospective cohort study

Setting and country: single centre, United States of America

Funding and conflicts of interest: Not reported

Inclusion criteria: patients with Duchenne muscular dystrophy reviewed between 1960 and 1993 at the Women's and Children's Hospital or the Muscular Dystrophy Clinic at the Regency

Park Centre for Young Disabled, Adelaide,

South Australia.

Exclusion criteria:

-

N total at baseline:

Intervention: 17

Control: 21

Important prognostic factors²:

Age, mean (range):

I: 14.9 years

C: 14.9 years

Cobbs angle, mean ± SD:

I: 57° ± 16.4°

C: 45° ± 29.9°

Groups comparable at baseline? yes

Describe intervention:

Spinal stabilisation

Describe control:

No surgery

Length of follow-up:

I: 2.4 years

C: 2.7 years

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

Spinal stabilisation in Duchenne muscular dystrophy does not alter the decline in pulmonary function, nor

does it improve survival.

Galasko, 1992

Type of study: prospective cohort study

Setting and country: single centre, United Kingdom

Funding and conflicts of interest: This study was supported by a research grant from the Muscular

Dystrophy Group of Great Britain.

No benefits in any form have been received or will be received

from a commercial party related directly or indirectly to the subject of

this article.

Inclusion criteria: Patients with Duchenne muscular dystrophy attending the muscle clinic at the Royal Manchester Children’s Hospital in 1990.

Exclusion criteria:

Not reported

N total at baseline:

I: 32

C: 23

Important prognostic factors²:

Age, mean (range):

I: 14 years

C: 13.6 years

Groups comparable at baseline? Yes

Describe intervention:

Spinal stabilisation using a modified segmental sublaminal wiring technique with facet joint fusion at each level.

Describe control:

No surgery

Length of follow-up:

I: up to 60 months

C: up to 60 months

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

Our results show that, providing the pre-operative

surgical and anaesthetic assessment is carefully done,

extensive spinal stabilisation can be carried out safely in

patients with Duchenne muscular dystrophy.

PICO 2

Galasko, 1995

Type of study: prospective cohort study

Setting and country: single centre, United Kingdom

Funding and conflicts of interest: This work has been supported by grants from Action

Research and the Muscular Dystrophy Group of Great Britain

Inclusion criteria: patients with Duchenne muscular dystrophy attended the Muscle Clinic at the Royal Manchester Children's Hospital

between 1982 and 1993.

Exclusion criteria:

-

N total at baseline:

I: 93

C: 43

Important prognostic factors²:

Not reported

Groups comparable at baseline? Unclear

Describe intervention:

Standing regimen. The boys who accepted the standing

regimen were advised to stand for at least 2 h per day. During the

week this was carried out at school, and at the weekend and during

school holidays it was carried out at home.

Describe control:

None

Length of follow-up. Median (range):

I: 36 months

C: 36 months

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

The results showed that prolongation of standing in patients

with Duchenne muscular dystrophy was associated

with improved lung function, as measured by both the FVC

and PEFR, with reduction in the development and progression

of scoliosis. The most important result in our study was that spinal

stabilisation could improve survival for several years following

operation.

Colbert, 1987

Type of study: prospective cohort study

Setting and country: single centre, United States of America

Funding and conflicts of interest: This study was supported in part by grant G008300042 from the National Institute of Handicapped Research, US Department of Education, Bethesda, MD

Inclusion criteria: All DMD patients whose sitting posterior-anterior spine x-rays revealed a 5° to 15° curve were wasked to wear a modigied Jewett hyperextension brace.

Exclusion criteria:

-

N total at baseline:

I: 7

C: 15

Important prognostic factors²:

Age: 13 years

Average curve: 13.3°

Groups comparable at baseline? YesUnclear

Describe intervention:

Brace which employs an inflatable balloon in the posterior thracolumbar component.

Describe control:

No brace or ceased wearing it.

Length of follow-up:

I: 3.7 years

C: 4 years

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

We conclude that wearing the brace minimally slows scoliosis progression, but does not ultimately prevent a significant curve.

PICO 3

Sengupta, 2002

Type of study: retrospective cohort study

Setting and country: multi centre, United Kingdom

Funding and conflicts of interest: No funds were received in support of this work. No benefits in any

form have been or will be received from a commercial party related

directly or indirectly to the subject of this manuscript.

Inclusion criteria: Patients with Duchenne muscular dystrophy and scoliosis.

Exclusion criteria:

-

N total at baseline:

Intervention: 31

Control: 19

Important prognostic factors²:

Age, mean (range):

I: 14 years (10-17)

C: 11.7 years (9–16)

Groups comparable at baseline? No

Describe intervention:

Fixation to the pelvis using standard Luque instrumentation

technique. The Galveston technique1 was used in 9 cases and the L-rod configuration in 22 cases for pelvic fixation (Figure

1). Autologous bone graft supplemented with femoral head allograft was used for fusion.

Describe control:

Fixation to L5, using Luque rods fixed with sublaminar wires in the thoracic

spine and pedicle screws in the lower three to five lumbar vertebrae. Autologous bone graft and supplemental

femoral head allograft were used for fusion following standard facet excision and decortication. The Luque rods were connected

at the proximal end by H-bars, designed by one author to achieve improved stability, control rotational

forces, and allow spinal growth.

Length of follow-up:

I: 4.6 years (range=0.5 – 11.5)

C: 3.54 years (range=3-5)

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Authors conclusion:

Lumbar fixation to L5 is adequate if the

surgery is performed early, soon after becoming wheelchair bound, and with smaller curves and minimal pelvic obliquity. Use of pedicle screws in lumbar spine provides a solid foundation to maintain the correction over the period of relatively short life expectancy of these children. Pelvic fixation may be necessary in older children, who have larger curves and established pelvic obliquity. In the presence of deteriorating lung function, this is associated

with a greater morbidity and higher complication rate.

Brook, 1996

Type of study: retrospective cohort study

Setting and country: single centre, Australia

Funding and conflicts of interest: Not reported

Inclusion criteria: patients with DMD underwent segmental spinal instrumentation at the Women's and Children's Hospital, Adelaide between 1983 and 1993

Exclusion criteria:

lung function is deemed to be too impaired

N total at baseline:

Intervention: 10

Control: 7

Important prognostic factors²:

Age, mean (range):

14.9 years (10.8 – 18.3)

Groups comparable at baseline? Unclear

Describe intervention:

Luque L-rod technique

Describe control:

Galveston technique of pelvic fixation

Length of follow-up:

I: 3.1 years

C: 2.0 years

Loss-to-follow-up:

12 patients due to death

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

This series demonstrates that fixation to the pelvis is safe and without increased morbidity.

Mubarak, 1993

Type of study: prospective cohort study

Setting and country: single centre, United States of America

Funding and conflicts of interest: Not reported

Inclusion criteria: wheelchair- bound patients with Duchenne muscular dystrophy and spinal deformaites

Exclusion criteria:

-

N total at baseline:

Intervention: 12

Control: 10

Important prognostic factors²:

Age, mean (range):

I: 12 years and 10 months (11y+4mo to 14y+8mo)

C: 13 years and 4 months (10y+6mo to 16y+3mo)

Groups comparable at baseline? yes

Describe intervention:

Instrumentation and fusion tot the sacropelvis

Describe control:

Instrumentation/fusion was extended only to the fifth lumbar level

Length of follow-up:

I: 48 months

C: 34 months

Loss-to-follow-up:

Intervention:

2 (16.7%)

Reasons

Death; n=2

Control:

3 (30%)

Reasons

Death; n=3

Incomplete outcome data:

Not reported

See tabel 1.

Authors conclusion:

The recommendation for operation in such patients should be made when their curve is >20° and if their forced vital capacity is .40%. If treatment is initiated early, Luque instrumentation and fusion from high thoracic (t2 or T3) to the fifth lumbar vertebra should be sufficient.

Debnath, 2011

Type of study: retrospective matched cohort study

Setting and country: single centre, United Kingdom

Funding and conflicts of interest: Not reported

Inclusion criteria: patients with Duchenne muscular dystrophy and spinal deformaites

Exclusion criteria:

-

N total at baseline:

Intervention: 22

Control: 18

Important prognostic factors²:

Age, mean (range):

I: 12.5 years (9 – 16)

C: 11.8 years (9-15)

Cobbs angle, mean (range)°:

I: 45.3 (26-75)

C: 42.8 (28-80)

Groups comparable at baseline? yes

Describe intervention:

SLW fixation was used from T2/3 to pelvis or sacrum

Describe control:

PS fixation was used

from T2/3 to L5

Length of follow-up:

2 years (range= 2 to 13)

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

Spinal instrumentation with pedicle screws for scoliosis

correction in patients with DMD results in a good correction

of the spinal and pelvic deformity. Pedicle instrumentation

gives a solid foundation, on which the rest of the spine

may be held upright, maintain the balance and rotation. Due

to the improvement of lumbar curve and pelvic obliquity by

pedicle instrumentation, a good sitting balance is restored

even without the pelvic or sacral fixation in patients with

pelvic obliquity less than 10°

Alman, 1999

Type of study: retrospective cohort study

Setting and country: multi centre, Canada

Funding and conflicts of interest: No benefits in any form have been received or will be received from a commercial party related directly or indirectly to the subject of this

article.

Inclusion criteria: all patients undergoing spinal surgery for Duchenne muscular dystrophy between 1988 and 1995 at

two institutions where selected cases were fused caudally to

L5.

Exclusion criteria:

-

N total at baseline:

Intervention: 38

Control: 10

Important prognostic factors²:

Age, mean ± SD:

I: 12.8 ± 2.5

C: 13.1 ± 2.8

Cobbs angle, mean ± SD °:

I: 24 ± 11

C: 48 ± 10

Preoperative obliquity, mean ± SD °:

I: 5 ± 4

C: 13 ± 6

Groups comparable at baseline? no

Describe intervention:

Patients with curves measuring less than 40° and with less than 10° of angulation between the sacrum and the caudal

two lumbar vertebrae, were instrumented and fused, stopping caudally at the fifth lumbar vertebra. The procedure was carried out using multiple-level sublaminar wires (Luque) with either a modified unit rod with Galveston

extensions to the pelvis cut off, a modified unit rod with a cross-link placed at the caudal end, or two Luque rods.

Describe control:

The pelvis was included when the curve had a Cobb angle greater than 40° or pelvic obliquity was greater than 10°, using the Galveston technique, with either the unit rod or

two Luque rods. All fusions included facetectomy, decortication and allograft bone.

Length of follow-up:

I:

Preoperative apex above L1: 4.2 ± 2.0

Preoperative apex below L1: 4.8 ± 1.7

C: 4.4 ± 1.6

Loss-to-follow-up:

Not reported

Incomplete outcome data:

Not reported

See tabel 1.

Author’s conclusion:

We recommend fusion to the sacrum for scoliosis in

Duchenne muscular dystrophy, especially for patients

with an apex to their curve below L1.

Study reference

(first author, year of publication)

Bias due to a non-representative or ill-defined sample of patients?¹

(unlikely/likely/unclear)

Bias due to insufficiently long, or incomplete follow-up, or differences in follow-up between treatment groups?²

(unlikely/likely/unclear)

Bias due to ill-defined or inadequately measured outcome ?³

(unlikely/likely/unclear)

Bias due to inadequate adjustment for all important prognostic factors?⁴

(unlikely/likely/unclear)

Suk, 2014

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely

Kennedy, 1995

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely

Galasko, 1992

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely

Galasko, 1995

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely

Colbert, 1987

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely

Sengupta, 2002

Likely (selection bias)

Likely

Likely (soft outcomes)

Likely

Brook, 1996

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely

Mubarak, 1993

Likely (selection bias)

Likely

Likely (soft outcomes)

Likely

Debnath, 2011

Likely (selection bias)

Likely

Likely (soft outcomes)

Likely

Alman, 1999

Likely (selection bias)

Unlikely

Likely (soft outcomes)

Likely