Table 3:
characteristics of participants with APDS from the treatment and control groups before and after IPTW for the base case IgM analysis
| Before weighting | After weighting | |||||
|---|---|---|---|---|---|---|
| Controls | Treatment | SMDa | Controls | Treatment | SMDa | |
| Age at first IgM test (years) | 3.0 | 3.3 | 0.059 | 3.3 | 3.3 | 0 |
| Sex (% male) |
54.1 | 49.0 | 0.102 | 56.8 | 56.8 | 0 |
| APDS subtype (% APDS2) |
18.9 | 28.6 | 0.228 | 23.4 | 23.4 | 0 |
| Baseline IgM (g/L) | 22.9 | 10.8 | 1.274 | 17.8 | 17.8 | 0 |
Results are reported for the base case analysis, in which missing data were handled via complete case analysis. Age at first IgM test, baseline serum IgM levels, APDS mutation status and sex were adjusted for in the propensity score model, and data were censored at first occurrence of HSCT in the outcome model. After weighting, the effective sample size was n = 28 in both the control and treatment group.
aStandardised mean difference ≥0.1 indicates imbalance.
Abbreviations: APDS: activated PI3K delta syndrome; ATO: average treatment effect in the overlap; HSCT: haematopoietic stem cell transplant; IgM: immunoglobulin M; IPTW: inverse probability of treatment weighting; PI3K: phosphoinositide 3-kinase; SMD: standardised mean difference.