Incidence of SNs by primary NMD with SIRs (N = 5933)
| Primary NMD . | n of survivors (median follow-up, y; range) . | Reported SNs, n (%) . | SIR (95% CI) . | P . |
|---|---|---|---|---|
| Total | 5933 (7.6; 0-20.9) | 71 (1.2) | 11 (9-14) | <.001 |
| Bone marrow failure disorder | ||||
| SAA | 1429 (7.6; 0-20.8) | 16 (1.1) | 8 (4-12) | <.001 |
| FA | 574 (9.1; 0.2-20.4) | 31 (5.4) | 50 (34-71) | <.001 |
| Other marrow failure | 343 (7.7; 0.6-20.3) | 6 (1.7) | 15 (5-32) | <.001 |
| Hemoglobinopathy | ||||
| SCD | 294 (6.1; 0.1-20.1) | 3 (1) | 11 (2-33) | .005 |
| Thalassemia | 572 (5.3; 0.2-20.0) | 0 (0) | — | — |
| Immunodeficiency syndrome | ||||
| SCID | 575 (7.3; 0.2-20.9) | 2 (0.3) | 4 (0.5-14) | .196 |
| Non-SCID PID | 793 (8.0; 0.2-20.5) | 3 (0.4) | 4 (0.7-10) | .107 |
| Histiocytic disorder | 466 (7.1; 0.5-20.2) | 5 (1) | 14 (5-33) | <.001 |
| Metabolic disease, leukodystrophy, other | ||||
| Leukodystrophy | 227 (6.1; 0.2-20.2) | 2 (0.9) | 10 (1-37) | .034 |
| Osteopetrosis | 150 (6.9; 0.2-20.2) | 1 (0.7) | 10 (0.3-56) | .191 |
| Metabolic disorder | 474 (8.3; 0.2-20.4) | 2 (0.4) | 4 (0.5-15) | .172 |
| Autoimmune | 24 (4.7; 0.3-15.8) | 0 (0) | — | — |
| Other | 12 (8.7; 5.5-14.0) | 0 (0) | — | — |
| Primary NMD . | n of survivors (median follow-up, y; range) . | Reported SNs, n (%) . | SIR (95% CI) . | P . |
|---|---|---|---|---|
| Total | 5933 (7.6; 0-20.9) | 71 (1.2) | 11 (9-14) | <.001 |
| Bone marrow failure disorder | ||||
| SAA | 1429 (7.6; 0-20.8) | 16 (1.1) | 8 (4-12) | <.001 |
| FA | 574 (9.1; 0.2-20.4) | 31 (5.4) | 50 (34-71) | <.001 |
| Other marrow failure | 343 (7.7; 0.6-20.3) | 6 (1.7) | 15 (5-32) | <.001 |
| Hemoglobinopathy | ||||
| SCD | 294 (6.1; 0.1-20.1) | 3 (1) | 11 (2-33) | .005 |
| Thalassemia | 572 (5.3; 0.2-20.0) | 0 (0) | — | — |
| Immunodeficiency syndrome | ||||
| SCID | 575 (7.3; 0.2-20.9) | 2 (0.3) | 4 (0.5-14) | .196 |
| Non-SCID PID | 793 (8.0; 0.2-20.5) | 3 (0.4) | 4 (0.7-10) | .107 |
| Histiocytic disorder | 466 (7.1; 0.5-20.2) | 5 (1) | 14 (5-33) | <.001 |
| Metabolic disease, leukodystrophy, other | ||||
| Leukodystrophy | 227 (6.1; 0.2-20.2) | 2 (0.9) | 10 (1-37) | .034 |
| Osteopetrosis | 150 (6.9; 0.2-20.2) | 1 (0.7) | 10 (0.3-56) | .191 |
| Metabolic disorder | 474 (8.3; 0.2-20.4) | 2 (0.4) | 4 (0.5-15) | .172 |
| Autoimmune | 24 (4.7; 0.3-15.8) | 0 (0) | — | — |
| Other | 12 (8.7; 5.5-14.0) | 0 (0) | — | — |