Summary of findings for the main comparison. First‐line allogeneic hematopoietic stem cell transplantation of HLA‐matched sibling donors compared with first‐line ciclosporin and/or antithymocyte or antilymphocyte globulin for acquired severe aplastic anemia.
First‐line allogeneic hematopoietic stem cell transplantation of HLA‐matched sibling donors compared with first‐line ciclosporin and/or antithymocyte or antilymphocyte globulin for acquired severe aplastic anemia | ||||||
Patient or population: patients with acquired SAA Settings: hospital Intervention: first‐line allogeneic hematopoietic stem cell transplantation of HLA‐matched sibling donors (MSD‐HSCT) Comparison: first‐line ciclosporin and/or antithymocyte or antilymphocyte globulin (IST) | ||||||
Outcomes | Illustrative comparative risks* (95% CI) | Relative effect (95% CI) | No of participants (studies) | Quality of the evidence (GRADE) | Comments | |
Assumed risk | Corresponding risk | |||||
First‐line ciclosporin and/or antithymocyte or antilymphocyte globulin (IST) | First‐line allogeneic hematopoietic stem cell transplantation of HLA‐matched sibling donors (MSD‐HSCT) | |||||
Overall mortality Follow‐up: 2 years | Moderate1 | HR 0.95 (0.45 to 1.91) | 203 (3 studies) | ⊕⊕⊝⊝ low2,3 | ||
31 per 100 | 30 per 100 (15 to 51) | |||||
Treatment‐related mortality Follow‐up: not reported |
See comment | See comment | Not estimable | 54 (2 studies4) | ⊕⊝⊝⊝ very low4 | Case series; 15 of 54 of the MSD‐HSCT group affected |
Health‐related quality of life | See comment | See comment | Not estimable | 0 (0) | See comment | No studies identified |
Graft failure Follow‐up: not reported |
See comment | See comment | Not estimable | 54 (2 studies4) | ⊕⊝⊝⊝ very low4 | Case series; 4 of 54 of the MSD‐HSCT group affected |
No response to IST Follow‐up: not reported |
See comment | See comment | Not estimable | 35 (2 studies4) | ⊕⊝⊝⊝ very low4 | Case series; 16 of 35 of the IST group affected |
Graft‐versus‐host disease Follow‐up: not reported |
See comment | See comment | Not estimable | 52 (2 studies4) | ⊕⊝⊝⊝ very low4 | Case series; 22 of 52 of the MSD‐HSCT group affected |
Secondary clonal disease or malignancies Follow‐up: not reported |
Moderate1 | Peto OR 0.54 (0.07 to 4) | 170 (2 studies) | ⊕⊕⊝⊝ low2,3 | ||
2 per 100 | 1 per 100 (0 to 10) | |||||
*The basis for the assumed risk (e.g. the median control group risk across studies) is provided in footnotes. The corresponding risk (and its 95% confidence interval) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). CI: confidence interval; HR: hazard ratio; IST: immunosuppressive therapy; MSD‐HSCT: first‐line allogeneic hematopoietic stem cell transplantation from HLA‐matched (identical) sibling donor; OR: odds ratio | ||||||
GRADE Working Group grades of evidence High quality: Further research is very unlikely to change our confidence in the estimate of effect. Moderate quality: Further research is likely to have an important impact on our confidence in the estimate of effect and may change the estimate. Low quality: Further research is very likely to have an important impact on our confidence in the estimate of effect and is likely to change the estimate. Very low quality: We are very uncertain about the estimate. |
1Median control group risk across studies. 2'Mendelian randomization'. 3Wide confidence interval. 4Case series.