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Table 2 Summary of risk of bias assessment for non-randomised experimental studies

From: A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel

Study ID

Source

Assessment criterion

1

2

3

4

5

6

7

8

9

Bley [18]

Abstract

Yes

NA

NA

No

Unclear

Unclear

NA

Unclear

NA

Bohringer [19]

Abstract

Yes

NA

NA

No

No

Unclear

NA

Unclear

Unclear

Boucher [21]

Full paper

Yes

NA

NA

No

Yes

No

NA

Yes

No

EUROCORD [22]

Full paper

Yes

NA

NA

No

Yes

Unclear

NA

Yes

No

Groeschel [16]

Full paper

Yes

Unclear

Unclear

Yes

Unclear

No

No

Yes

Yes

Kehrer [26]

Full paper

Yes

NA

NA

No

NA

NA

NA

No

Yes

LDM/1 study [25]

Full paper

Yes

NA

NA

No

Unclear

Unclear

NA

Yes

Yes

Martin [23]

Full paper

Yes

NA

NA

No

Yes

Yes

NA

Yes

No

Fumagalli et al. [13]

Full paper

Yes

Yes

Unclear

Yes

Yes

Unclear

Yes

Yes

Yes

Prasad [24]

Full paper

Yes

NA

NA

No

Yes

Unclear

NA

Unclear

No

Singh [20]

Abstract

No

NA

NA

No

No

Unclear

NA

Unclear

Unclear

van Rappard [17]

Full paper

Yes

No

Unclear

Yes

Yes

No

No

Unclear

Unclear

  1. NA not applicable, CUP compassionate use programme, HEP Hospital exemption programme
  2. 1: Is it clear what is the cause (intervention) and what is the effect (outcome) (i.e. no confusion about what comes first)
  3. 2: Were the participants included in any comparisons similar?
  4. 3: Were the participants included in any comparisons receiving similar treatment/care, other than the intervention of interest?
  5. 4: Was there a control group?
  6. 5: Were there multiple measurements of the outcome both pre and post the intervention?
  7. 6: Was follow up complete and if not, were differences between groups in terms of their follow up adequately described and analysed?
  8. 7: Were the outcomes of participants included in any comparisons measured in the same way?
  9. 8: Were outcomes measured in a reliable way?
  10. 9. Was appropriate statistical analysis used?