clinical characteristics at or prior to baseline and during follow-up by group for the base case IgM analysis, after weighting
| At or prior to baseline . | During follow-up . | |||||
|---|---|---|---|---|---|---|
| Control . | Treatment . | SMDa . | Controlb . | Treatment . | SMDa . | |
| Age at index date, years (median [IQR]) | 15.00 [8.00, 24.01] | 16.78 [14.00, 20.00] | <0.001 | |||
| Presence of lymphoproliferationb (% yes) | 85.8 | NA | 70.1 | NA | ||
| HSCTc (% yes) | 0 | 0 | <0.001 | 5.2 | 0 | 0.333 |
| Infections and infestations (excluding EBV and CMV) (% yes) | 98.9 | 47.6 | 1.424 | 100.0 | 65.9 | 1.016 |
| Any autoimmune cytopenia (% yes) | 46.9 | 34.5 | 0.255 | 11.8 | 5.8d | 0.215 |
| Malignancy (% yes) | 17.9 | 3.7 | 0.468 | 14.0 | 3.9 | 0.361 |
| Concomitant medications | ||||||
| IRT (% yes) | 80.1 | 70.5 | 0.224 | 86.6 | 26.9 | 1.510 |
| Antibiotics (% yes) | 78.6 | 82.1 | 0.088 | 71.4 | 53.5 | 0.376 |
| mTOR inhibitore (% yes) | 33.4 | 23.6 | 0.220 | 33.4 | 0.0 | 1.002 |
| Rituximab (% yes) | 5.1 | 0.0 | 0.328 | 5.1 | 0.0 | 0.328 |
| At or prior to baseline . | During follow-up . | |||||
|---|---|---|---|---|---|---|
| Control . | Treatment . | SMDa . | Controlb . | Treatment . | SMDa . | |
| Age at index date, years (median [IQR]) | 15.00 [8.00, 24.01] | 16.78 [14.00, 20.00] | <0.001 | |||
| Presence of lymphoproliferationb (% yes) | 85.8 | NA | 70.1 | NA | ||
| HSCTc (% yes) | 0 | 0 | <0.001 | 5.2 | 0 | 0.333 |
| Infections and infestations (excluding EBV and CMV) (% yes) | 98.9 | 47.6 | 1.424 | 100.0 | 65.9 | 1.016 |
| Any autoimmune cytopenia (% yes) | 46.9 | 34.5 | 0.255 | 11.8 | 5.8d | 0.215 |
| Malignancy (% yes) | 17.9 | 3.7 | 0.468 | 14.0 | 3.9 | 0.361 |
| Concomitant medications | ||||||
| IRT (% yes) | 80.1 | 70.5 | 0.224 | 86.6 | 26.9 | 1.510 |
| Antibiotics (% yes) | 78.6 | 82.1 | 0.088 | 71.4 | 53.5 | 0.376 |
| mTOR inhibitore (% yes) | 33.4 | 23.6 | 0.220 | 33.4 | 0.0 | 1.002 |
| Rituximab (% yes) | 5.1 | 0.0 | 0.328 | 5.1 | 0.0 | 0.328 |
aStandardised differences of ≥0.1 are considered meaningful.
bEver-present lymphoproliferation, infections and concomitant medications are used in the absence of data variables in the ESID registry.
cHSCT during follow-up represents patients in the cohort who were censored during follow-up though HSCT occurred only after the observation period.
dCytopenias during the leniolisib clinical studies were based on adverse event reports, and were due to any cause (so may not have been due to autoimmunity).
eUsing definition based on any mention of mTOR throughout ESID variables.
Abbreviations: CMV: cytomegalovirus; EBV: Epstein-Barr virus; ESID: European Society of Immunodeficiencies; HSCT: haematopoietic stem cell transplant; IQR: interquartile range; IRT: immunoglobulin replacement therapy; mTOR: mammalian target of rapamycin; NA: not applicable; SMD: standardised mean difference.
clinical characteristics at or prior to baseline and during follow-up by group for the base case IgM analysis, after weighting
| At or prior to baseline . | During follow-up . | |||||
|---|---|---|---|---|---|---|
| Control . | Treatment . | SMDa . | Controlb . | Treatment . | SMDa . | |
| Age at index date, years (median [IQR]) | 15.00 [8.00, 24.01] | 16.78 [14.00, 20.00] | <0.001 | |||
| Presence of lymphoproliferationb (% yes) | 85.8 | NA | 70.1 | NA | ||
| HSCTc (% yes) | 0 | 0 | <0.001 | 5.2 | 0 | 0.333 |
| Infections and infestations (excluding EBV and CMV) (% yes) | 98.9 | 47.6 | 1.424 | 100.0 | 65.9 | 1.016 |
| Any autoimmune cytopenia (% yes) | 46.9 | 34.5 | 0.255 | 11.8 | 5.8d | 0.215 |
| Malignancy (% yes) | 17.9 | 3.7 | 0.468 | 14.0 | 3.9 | 0.361 |
| Concomitant medications | ||||||
| IRT (% yes) | 80.1 | 70.5 | 0.224 | 86.6 | 26.9 | 1.510 |
| Antibiotics (% yes) | 78.6 | 82.1 | 0.088 | 71.4 | 53.5 | 0.376 |
| mTOR inhibitore (% yes) | 33.4 | 23.6 | 0.220 | 33.4 | 0.0 | 1.002 |
| Rituximab (% yes) | 5.1 | 0.0 | 0.328 | 5.1 | 0.0 | 0.328 |
| At or prior to baseline . | During follow-up . | |||||
|---|---|---|---|---|---|---|
| Control . | Treatment . | SMDa . | Controlb . | Treatment . | SMDa . | |
| Age at index date, years (median [IQR]) | 15.00 [8.00, 24.01] | 16.78 [14.00, 20.00] | <0.001 | |||
| Presence of lymphoproliferationb (% yes) | 85.8 | NA | 70.1 | NA | ||
| HSCTc (% yes) | 0 | 0 | <0.001 | 5.2 | 0 | 0.333 |
| Infections and infestations (excluding EBV and CMV) (% yes) | 98.9 | 47.6 | 1.424 | 100.0 | 65.9 | 1.016 |
| Any autoimmune cytopenia (% yes) | 46.9 | 34.5 | 0.255 | 11.8 | 5.8d | 0.215 |
| Malignancy (% yes) | 17.9 | 3.7 | 0.468 | 14.0 | 3.9 | 0.361 |
| Concomitant medications | ||||||
| IRT (% yes) | 80.1 | 70.5 | 0.224 | 86.6 | 26.9 | 1.510 |
| Antibiotics (% yes) | 78.6 | 82.1 | 0.088 | 71.4 | 53.5 | 0.376 |
| mTOR inhibitore (% yes) | 33.4 | 23.6 | 0.220 | 33.4 | 0.0 | 1.002 |
| Rituximab (% yes) | 5.1 | 0.0 | 0.328 | 5.1 | 0.0 | 0.328 |
aStandardised differences of ≥0.1 are considered meaningful.
bEver-present lymphoproliferation, infections and concomitant medications are used in the absence of data variables in the ESID registry.
cHSCT during follow-up represents patients in the cohort who were censored during follow-up though HSCT occurred only after the observation period.
dCytopenias during the leniolisib clinical studies were based on adverse event reports, and were due to any cause (so may not have been due to autoimmunity).
eUsing definition based on any mention of mTOR throughout ESID variables.
Abbreviations: CMV: cytomegalovirus; EBV: Epstein-Barr virus; ESID: European Society of Immunodeficiencies; HSCT: haematopoietic stem cell transplant; IQR: interquartile range; IRT: immunoglobulin replacement therapy; mTOR: mammalian target of rapamycin; NA: not applicable; SMD: standardised mean difference.
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