The association between demographics and treatment outcome
| Characteristic . | No. of pts . | No. of events . | 4-y EFS rate, % (95% CI) . | P value∗ . | No. of TRM events . | 4-y CI TRM, % (95% CI) . | P value∗ . | No. of progressive cancer events . | 4-y CI progressive cancer, % (95% CI) . | P value∗ . | No. of second malignancy events . | 4-y CI second malignancy, % (95% CI) . | P value∗ . | No. of deaths unrelated to cancer . | 4-y CI deaths unrelated to cancer, % (95% CI) . | P value∗ . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Total | 185 | 90 | 47.9 (40.8-56.2) | — | 47 | 25.9 (19.5-32.4) | — | 26 | 14.5 (9.2-19.8) | 7 | 4.9 (1.3-8.4) | 10 | 6.5 (2.5-10.5) | |||
| Sex | .225 | .866 | .384 | .772 | .214 | |||||||||||
| Female | 72 | 40 | 41.9 (31.6-55.6) | 19 | 25.5 (17.3-33.7) | 12 | 14.9 (7.2-22.6) | 3 | 4.5 (0-9.5) | 6 | 9.7 (2.2-17.2) | |||||
| Male | 113 | 50 | 51.8 (42.7-62.8) | 28 | 26.6 (16.2-36.9) | 14 | 25.0 (11.9-38.1) | 4 | 5.4 (0.2-10.5) | 4 | 4.4 (0.1-8.8) | |||||
| Age at cancer diagnosis, y | .019 | .029 | .084 | .754 | <.001 | |||||||||||
| 0-4 | 40 | 12 | 69.1 (55.9-85.4) | 7 | 17.5 (5.5-29.4) | 2 | 6.3 (0-14.7) | 3 | 8.4 (0-17.7) | 0 | 0 | |||||
| 5-9 | 77 | 38 | 45.3 (34.6-59.4) | 15 | 19.8 (10.7-28.9) | 17 | 28.7 (15.8-41.7) | 2 | 3.6 (0-8.7) | 4 | 6.7 (0.2-13.3) | |||||
| 10-14 | 34 | 18 | 39.8 (25-63.3) | 10 | 31.1 (14.6-47.6) | 4 | 17.3 (1.1-33.4) | 1 | 4.1 (0-12.2) | 3 | 11.8 (0-25.2) | |||||
| ≤15 | 34 | 22 | 33.7 (20.8-54.6) | 15 | 44.6 (27.3-61.8) | 3 | 14.4 (0-30.5) | 1 | 3.1 (0-9.3) | 3 | 10.2 (0-21.5) | |||||
| Treatment era | .758 | .192 | .126 | <.001 | .426 | |||||||||||
| 1985-1999 | 28 | 16 | 41.6 (26.6-65) | 7 | 25.0 (8.5-41.4) | 4 | 15.9 (0.9-31.0) | 4 | 15.1 (1-29.3) | 1 | 3.8 (0-11.3) | |||||
| 2000-2009 | 66 | 33 | 49.6 (38.8-63.4) | 22 | 33.3 (21.8-44.8) | 5 | 9.4 (0.3-18.4) | 3 | 4.7 (0-10.1) | 3 | 4.5 (0-9.6) | |||||
| 2010-2023 | 91 | 41 | 48 (37.4-61.7) | 18 | 20.5 (11.9--29.1) | 17 | 26.3 (14.9-37.7) | 0 | 0 | 6 | 11 (1.8-20.2) | |||||
| Tumor type | .004 | .211 | .360 | .459 | <.001 | |||||||||||
| Mature B | 85 | 47 | 38.9 (28.9-52.3) | 22 | 26.5 (16.9-36.1) | 15 | 23.5 (11.7-35.3) | 1 | 1.9 (0-5.6) | 9 | 13.8 (5.1-22.4) | |||||
| Lymphoblastic | 79 | 27 | 63.9 (53.8-75.9) | 15 | 19.4 (10.5-28.2) | 8 | 13.0 (4.5-21.6) | 4 | 6.1 (0.2-11.9) | 0 | 0 | |||||
| Hodgkin lymphoma | 15 | 10 | 31.1 (14.2-68) | 6 | 40 (13.9-67.4) | 2 | 18.3 (0-43.9) | 1 | 7.8 (0-23.9) | 1 | 8.9 (0-26.9) | |||||
| Other | 6 | 6 | 4 | 1 | 1 | 0 | ||||||||||
| Baseline neurological status | .009 | .208 | .288 | .045 | .687 | |||||||||||
| Asymptomatic | 28 | 8 | 71.4 (56.5-90.3) | 5 | 17.8 (3.3-32.3) | 1 | 4.7 (0-14.1) | 1 | 3.6 (0-10.6) | 1 | 3.6 (0-10.6) | |||||
| Atactic gait | 64 | 28 | 50.8 (39-66.2) | 13 | 21.3 (10.8-31.7) | 8 | 17.1 (5.7-28.4) | 3 | 5.9 (0-12.5) | 4 | 8.4 (0.3-16.4) | |||||
| Wheelchair bound | 59 | 35 | 37.3 (26.4-52.8) | 19 | 33.0 (20.7-45.4) | 11 | 26.1 (10.9-41.2) | 0 | 0 | 5 | 10.2 (1.6-18.8) | |||||
| Unknown | 34 | 19 | 10 | 6 | 3 | 0 | ||||||||||
| Baseline respiratory status | .021 | .001 | .645 | <.001 | .374 | |||||||||||
| Asymptomatic | 66 | 22 | 63 (51.7-76.8) | 9 | 14.2 (5.5-22.9) | 9 | 18.2 (7.1-29.2) | 2 | 3.8 (0-9.0) | 2 | 3.9 (0-9.4) | |||||
| Recurrent respiratory infections (≥3 per y) | 60 | 30 | 44.9 (33-61.2) | 13 | 22.0 (11.3-32.8) | 9 | 18.4 (7.0-29.7) | 3 | 6.4 (0-13.8) | 5 | 11.1 (1.5-20.6) | |||||
| Chronic bronchitis/bronchiectasis | 25 | 18 | 28 (14.9-52.5) | 13 | 52 (31.8-72.2) | 2 | 7.7 (0-22.7) | 0 | 0 | 3 | 12 (6.8-25.4) | |||||
| Unknown | 34 | 20 | 12 | 6 | 2 | 0 | ||||||||||
| ATM mutation class | <.001 | .017 | .922 | .464 | <.001 | |||||||||||
| Absent ATM kinase activity | 77 | 43 | 39.4 (29-53.3) | 28 | 37.6 (26.4-48.7) | 10 | 17.2 (5.9-28.5) | 1 | 1.6 (0-4.7) | 4 | 7.6 (0.1-15) | |||||
| Residual ATM kinase activity | 25 | 5 | 78.7 (63.7-97.2) | 1 | 4.0 (0-11.8) | 3 | 13.8 (0-28.8) | 1 | 4.4 (0-13) | 0 | 0 | |||||
| Unknown | 83 | 42 | 18 | 13 | 5 | 6 |
| Characteristic . | No. of pts . | No. of events . | 4-y EFS rate, % (95% CI) . | P value∗ . | No. of TRM events . | 4-y CI TRM, % (95% CI) . | P value∗ . | No. of progressive cancer events . | 4-y CI progressive cancer, % (95% CI) . | P value∗ . | No. of second malignancy events . | 4-y CI second malignancy, % (95% CI) . | P value∗ . | No. of deaths unrelated to cancer . | 4-y CI deaths unrelated to cancer, % (95% CI) . | P value∗ . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Total | 185 | 90 | 47.9 (40.8-56.2) | — | 47 | 25.9 (19.5-32.4) | — | 26 | 14.5 (9.2-19.8) | 7 | 4.9 (1.3-8.4) | 10 | 6.5 (2.5-10.5) | |||
| Sex | .225 | .866 | .384 | .772 | .214 | |||||||||||
| Female | 72 | 40 | 41.9 (31.6-55.6) | 19 | 25.5 (17.3-33.7) | 12 | 14.9 (7.2-22.6) | 3 | 4.5 (0-9.5) | 6 | 9.7 (2.2-17.2) | |||||
| Male | 113 | 50 | 51.8 (42.7-62.8) | 28 | 26.6 (16.2-36.9) | 14 | 25.0 (11.9-38.1) | 4 | 5.4 (0.2-10.5) | 4 | 4.4 (0.1-8.8) | |||||
| Age at cancer diagnosis, y | .019 | .029 | .084 | .754 | <.001 | |||||||||||
| 0-4 | 40 | 12 | 69.1 (55.9-85.4) | 7 | 17.5 (5.5-29.4) | 2 | 6.3 (0-14.7) | 3 | 8.4 (0-17.7) | 0 | 0 | |||||
| 5-9 | 77 | 38 | 45.3 (34.6-59.4) | 15 | 19.8 (10.7-28.9) | 17 | 28.7 (15.8-41.7) | 2 | 3.6 (0-8.7) | 4 | 6.7 (0.2-13.3) | |||||
| 10-14 | 34 | 18 | 39.8 (25-63.3) | 10 | 31.1 (14.6-47.6) | 4 | 17.3 (1.1-33.4) | 1 | 4.1 (0-12.2) | 3 | 11.8 (0-25.2) | |||||
| ≤15 | 34 | 22 | 33.7 (20.8-54.6) | 15 | 44.6 (27.3-61.8) | 3 | 14.4 (0-30.5) | 1 | 3.1 (0-9.3) | 3 | 10.2 (0-21.5) | |||||
| Treatment era | .758 | .192 | .126 | <.001 | .426 | |||||||||||
| 1985-1999 | 28 | 16 | 41.6 (26.6-65) | 7 | 25.0 (8.5-41.4) | 4 | 15.9 (0.9-31.0) | 4 | 15.1 (1-29.3) | 1 | 3.8 (0-11.3) | |||||
| 2000-2009 | 66 | 33 | 49.6 (38.8-63.4) | 22 | 33.3 (21.8-44.8) | 5 | 9.4 (0.3-18.4) | 3 | 4.7 (0-10.1) | 3 | 4.5 (0-9.6) | |||||
| 2010-2023 | 91 | 41 | 48 (37.4-61.7) | 18 | 20.5 (11.9--29.1) | 17 | 26.3 (14.9-37.7) | 0 | 0 | 6 | 11 (1.8-20.2) | |||||
| Tumor type | .004 | .211 | .360 | .459 | <.001 | |||||||||||
| Mature B | 85 | 47 | 38.9 (28.9-52.3) | 22 | 26.5 (16.9-36.1) | 15 | 23.5 (11.7-35.3) | 1 | 1.9 (0-5.6) | 9 | 13.8 (5.1-22.4) | |||||
| Lymphoblastic | 79 | 27 | 63.9 (53.8-75.9) | 15 | 19.4 (10.5-28.2) | 8 | 13.0 (4.5-21.6) | 4 | 6.1 (0.2-11.9) | 0 | 0 | |||||
| Hodgkin lymphoma | 15 | 10 | 31.1 (14.2-68) | 6 | 40 (13.9-67.4) | 2 | 18.3 (0-43.9) | 1 | 7.8 (0-23.9) | 1 | 8.9 (0-26.9) | |||||
| Other | 6 | 6 | 4 | 1 | 1 | 0 | ||||||||||
| Baseline neurological status | .009 | .208 | .288 | .045 | .687 | |||||||||||
| Asymptomatic | 28 | 8 | 71.4 (56.5-90.3) | 5 | 17.8 (3.3-32.3) | 1 | 4.7 (0-14.1) | 1 | 3.6 (0-10.6) | 1 | 3.6 (0-10.6) | |||||
| Atactic gait | 64 | 28 | 50.8 (39-66.2) | 13 | 21.3 (10.8-31.7) | 8 | 17.1 (5.7-28.4) | 3 | 5.9 (0-12.5) | 4 | 8.4 (0.3-16.4) | |||||
| Wheelchair bound | 59 | 35 | 37.3 (26.4-52.8) | 19 | 33.0 (20.7-45.4) | 11 | 26.1 (10.9-41.2) | 0 | 0 | 5 | 10.2 (1.6-18.8) | |||||
| Unknown | 34 | 19 | 10 | 6 | 3 | 0 | ||||||||||
| Baseline respiratory status | .021 | .001 | .645 | <.001 | .374 | |||||||||||
| Asymptomatic | 66 | 22 | 63 (51.7-76.8) | 9 | 14.2 (5.5-22.9) | 9 | 18.2 (7.1-29.2) | 2 | 3.8 (0-9.0) | 2 | 3.9 (0-9.4) | |||||
| Recurrent respiratory infections (≥3 per y) | 60 | 30 | 44.9 (33-61.2) | 13 | 22.0 (11.3-32.8) | 9 | 18.4 (7.0-29.7) | 3 | 6.4 (0-13.8) | 5 | 11.1 (1.5-20.6) | |||||
| Chronic bronchitis/bronchiectasis | 25 | 18 | 28 (14.9-52.5) | 13 | 52 (31.8-72.2) | 2 | 7.7 (0-22.7) | 0 | 0 | 3 | 12 (6.8-25.4) | |||||
| Unknown | 34 | 20 | 12 | 6 | 2 | 0 | ||||||||||
| ATM mutation class | <.001 | .017 | .922 | .464 | <.001 | |||||||||||
| Absent ATM kinase activity | 77 | 43 | 39.4 (29-53.3) | 28 | 37.6 (26.4-48.7) | 10 | 17.2 (5.9-28.5) | 1 | 1.6 (0-4.7) | 4 | 7.6 (0.1-15) | |||||
| Residual ATM kinase activity | 25 | 5 | 78.7 (63.7-97.2) | 1 | 4.0 (0-11.8) | 3 | 13.8 (0-28.8) | 1 | 4.4 (0-13) | 0 | 0 | |||||
| Unknown | 83 | 42 | 18 | 13 | 5 | 6 |
Data shown for 185 treated patients.
P values are based on a comparison performed at a predefined time point of 4 years from cancer diagnosis on the basis of log-log transformation.