Patient-, transplant-, treatment-, and organ-related RF and CoM variables influencing 2-year TRM
Variable . | Total cases (%) . | 2-y TRM (95% CI) . | HR (95% CI) . | P value . |
---|---|---|---|---|
All patients | 582 | 14.8% (12-17.9) | ||
Age, y | ||||
<2 | 242 (42) | 14.8% (10.6-19.7) | ||
2-5 | 160 (27) | 12.9% (8.2-18.7) | 1.09 (0.86-1.38) | .471 |
6+ | 180 (31) | 16.0% (10.9-21.9) | ||
Continuous | 1.01 (0.96-1.06) | .811 | ||
Patient sex | ||||
Male | 360 (62) | 15.9% (11.4-21.2) | 1.01 (0.96-1.06) | .349 |
Female | 222 (38) | 13.8% (10.4-17.7) | ||
Weight | ||||
Continuous | 1.00 (0.99-1.02) | .733 | ||
Disease category | ||||
Malignant diseases | 165 (28) | 15.0% (10.0-21.0) | 1.14 (0.75-1.74) | .535 |
Nonmalignant diseases | 417 (72) | 14.5% (11.2-18.1) | ||
Presence of sex mismatch (yes) | 264 (46) | 14.4% (10.4-19.1) | 1.08 (0.73-1.61) | .696 |
CMV mismatch (yes) | 188 (33) | 15.3% (10.5-20.9) | 1.08 (0.71-1.65) | .89 |
Stem cell source | ||||
BM | 339 (58) | 11.4% (8.2-15.2) | 1.53 (1.01-2.34) | .047 |
PBSC | 171 (29) | 20.7% (14.9-27.2) | 1.14 (0.60-2.18) | .692 |
Cord | 72 (12) | 15.5% (8.2-24.8) | ||
Period of transplantation (y) | ||||
2000-2005 | 167 | 17.4% (12-23) | 0.98 (0.75-1.27) | .853 |
2006-2011 | 205 | 13.1% (14.6-27.5) | ||
2012-2016 | 210 | 14% (9.6-19.2) | ||
Donor type | ||||
Mismatched donor | 190 (33) | 24.1% (18.2-30.5) | 2.15 (1.45-3.20) | .0005 |
Matched donor | 392 (67) | 10.0% (7.2-13.3) | ||
Transplant | ||||
>1 transplant | 47 (8) | 22% (11-33) | 1.40 (0.71-2.76) | .328 |
No previous transplant | 535 (92) | 14% (11-17) | ||
Conditioning | ||||
MAC | 263 (45) | 13% (9-17) | 0.82 (0.60-1.12) | .21 |
RIC/MIC | 278 (48) | 16% (12-21) | ||
None | 41 (7) | 18% (8-31) | ||
TBI | ||||
TBI-based conditioning | 85 (15%) | 18% (11-27) | 1.41 (0.86-2.32) | .168 |
Non-TBI based | 496 (85) | 14% (11-17) | ||
Use of serotherapy | ||||
Yes | 382 (66) | 16% (13-20) | 1.16 (0.76-1.78) | .487 |
No | 200 (34) | 11% (7-16) | ||
Use of any GVHD prophylaxis | ||||
Yes | 549 (94) | 14.4% (11.5-17.5) | 1.47 (0.72-2.97) | .288 |
No | 33 (6) | 18.8% (7.6-33.7) | ||
Pre-HSCT infections | ||||
Presence of probable/confirmed fungal infection | ||||
Yes | 50 (9) | 12.3% (5-23.1) | 0.81 (0.38-1.74) | .591 |
No | 531 (91) | 14.8% (11.9-18.1) | ||
Presence of disseminated BCGiosis | ||||
Yes | 12 (2) | 16.7% (2.7-41.3) | 0.95 (0.23-3.90) | .944 |
No | 569 (98) | 14.6% (11.8-17.7) | ||
Presence of EBV Day -10 to day 0 | ||||
Yes | 9 (2) | 12.5% (0.7-42.3) | 1.34 (0.37-4.82) | .658 |
No | 572 (98) | 14.7% (11.9-17.8) | ||
Presence of AdV Day -10 to day 0 | ||||
Yes | 6 (1) | 0% | Dropped | |
No | 575 (99) | 14.8% (12.0-17.9) | ||
Presence of CMV Day -10 to day 0 | ||||
Yes | 18 (3) | 28% (10-49) | 1.74 (0.68-4.41) | .246 |
No | 564 (97) | 14% (11-17) | ||
Presence of respiratory viral infection Day -10 to day 0 | ||||
Yes | 26 (4) | 31% (15-49) | 2.26 (1.04-4.91) | .039 |
No | 556 (96) | 14% (11-17) | ||
Need to continue antimicrobials at D0 | ||||
Yes | 104 (18) | 19% (12-28) | 1.32 (0.81-2.16) | .263 |
No | 478 (82) | 13.6% (10.6-16.9) | ||
Cardiac variables | ||||
Presence of heart valve disease∗ | ||||
Yes | 15 (3) | 13.3% (2.2-34.6) | 0.84 (0.42-1.68) | .616 |
No | 566 (97) | 14.7% (11.8-17.8) | ||
Presence of a cardiac anomaly | ||||
Yes | 31 (5) | 19.5% (7.9-34.9) | 1.12 (0.49-2.55) | .79 |
No | 551 (95) | 14.3% (11.5-17.5) | ||
Pulmonary variable | ||||
Presence of dyspnea at slight activity | ||||
Yes | 3 (1) | 0% | Dropped | |
No | 578 (99) | 14.7% (11.9-17.8) | ||
Presence of dyspnea at rest or requiring oxygen | ||||
Yes | 21 (4) | 9.5% (1.6-26.1) | 0.50 (0.12-2.00) | .325 |
No | 560 (96) | 14.8% (12.0-18.0) | ||
Presence of pulmonary hypertension | ||||
Yes | 2 (<1) | 0% | Dropped | |
No | 579 (99) | 14.7% (11.9-17.8) | ||
Presence of obstructive sleep apnea | ||||
Yes | 9 (2) | 33.3% (7.8-6.2) | 2.12 (0.66-6.79) | .208 |
No | 572 (98) | 14.3% (11.5-17.4) | ||
Being on assisted ventilation at D0 | ||||
Yes | 4 (1) | 25.0% (0.9-66.5) | 1.63 (0.17-5.4) | .670 |
No | 578 (99) | 14.6% (11.8-17.6) | ||
Requiring assisted ventilation at any time (including D0) | ||||
Yes | 74 (13) | 22% (13-32) | 1.48 (0.87-2.50) | .146 |
No | 508 (87) | 14% (11-17) | ||
Presence of any respiratory structural abnormality† | ||||
Yes | 13 (2) | 23% (6-47) | 1.91 (0.72-5.06) | .19 |
No | 13 (2) | 14% (12-18) | ||
Cerebrovascular CoMs | ||||
Encephalopathy | ||||
Yes | 4 (1) | 14.6% (11.8-17.6) | 1.52 (0.22-10.3) | .669 |
No | 577 (99) | 25% (0.9-66.5) | ||
Hydrocephalus | ||||
Yes | 7 (1) | 14.3% (0.7-46.5) | 1.63 (0.43-6.13) | .472 |
No | 574 (99) | 14.6% (11.8-17.7) | ||
Sinus thrombosis | ||||
Yes | 2 (<1) | 0% | Dropped | |
No | 579 (99) | 14.7% (11.9-17.8) | ||
PRES | ||||
Yes | 3 (1) | 67% (5-95) | 5.21 (1.56-17.3) | .007 |
No | 569 (99) | 14% (12-17) | ||
Epilepsy requiring medical intervention | ||||
Yes | 6 (1) | 50% (11-80) | 3.90 (1.03-14.8) | .045 |
No | 576 (99) | 14% (11-17) | ||
Presence of any CNS structural abnormality‡ | ||||
Yes | 11 (2) | 45% (17-71) | 2.95 (1.26-6.92) | .013 |
No | 571 (98) | 14% (11-17) | ||
Presence of any of the following: CNS infection, PRES, epilepsy, or CNS structural abnormality | ||||
Yes | 32 (6) | 47% (29-63) | 3.85 (2.17-6.83) | .0005 |
No | 550 (94) | 13% (10-16) | ||
Gastrointestinal disease variables | ||||
Presence of chronic diarrhea | ||||
Yes | 44 (8) | 20.9% (10.3-33.9) | 1.35 (0.71-2.54) | .361 |
No | 538 (92) | 14.1% (11.3-17.2) | ||
Active infectious diarrhea at D0 | ||||
Yes | 12 (2) | 42% (15-67) | 3.59 (1.27-10.1) | .016 |
No | 540 (98) | 14% (11-17) | ||
Continuation on TPN at D0 | ||||
Yes | 46 (8) | 26.5% (14.7-39.8) | 1.74 (0.93-3.25) | .08 |
No | 46 (8) | 13.6% (10.8-6.7) | ||
Perianal inflammation/fistula | ||||
Yes | 5 (1) | 0% | Dropped | |
No | 576 (99) | 14.7% (11.9-17.8) | ||
Endocrine disease variables | ||||
Diabetes requiring treatment | ||||
Yes | 3 (1) | 0% | Dropped | |
No | 578 (99) | 14.7% (11.9-17.8) | ||
Presence of any other endocrine disease on treatment§ | ||||
Yes | 21 (4) | 22.0% (6.7-42.7) | 1.49 (0.63-3.48) | .362 |
No | 561 (96) | 14.4% (11.6-17.5) | ||
Hepatobiliary disease, renal and biochemical variables | ||||
Moderate/severe hepatobiliary | 153 | 16.6% (11.1-23.2) | 1.33 (1.06-1.67) | .014 |
Mild hepatobiliary | 198 | 18% (12.9-23.7) | ||
No abnormality | 231 | 10.5% (6.9-15) | ||
Serum creatinine | ||||
>1.5 ULN | 29 | 14.3% (8.3-22.0) | 1.1 (0.9-1.27) | .693 |
Above ULN to 1.5 ULN | 72 | 13.2% (10.2-16.8) | ||
Normal | 481 | 13.5% (10.3-17.1) | ||
Presence of renal malformation | ||||
Yes | 5 (1) | 20% (1-58) | 1.29 (0.18-9.48) | .801 |
No | 577 (99) | 15% (12-18) | ||
Serum albumin level | ||||
<25 g/L | 10 (2) | 50.0% (18.4-75.3) | 1.80 (1.23-2.65) | .003 |
≥25-35 g/L | 155 (27) | 20.7% (14.6-27.5) | ||
>35 g/L | 416 (52) | 11.5% (8.6-14.9) | ||
Other comorbidities | ||||
Having a prior surgery | ||||
Yes | 56 (10) | 13.0% (5.7-23.4) | 0.93 (0.46-1.86) | .838 |
No | 525 (90) | 14.8% (11.9-18.1) | ||
Having a prior malignancy | ||||
Yes | 15 (3) | 18.3% (2.9-44.4) | 1.83 (0.76-4.40) | .180 |
No | 566 (97) | 14.6% (11.8-17.7) | ||
Prior autoimmunity or immune dysregulation | ||||
Yes | 63 | 14.4% (11.5-17.6) | 1.02 | .949 |
No | 519 | 16.45% (8.4-26.8) | ||
Being on prednisolone ≥0.3 mg/kg per day at D0 | ||||
Yes | 44 (8) | 27.5% (15.3-21.1) | 1.98 (1.08-3.61) | .026 |
No | 537 (92) | 13.6% (10.8- 16.4) |
Variable . | Total cases (%) . | 2-y TRM (95% CI) . | HR (95% CI) . | P value . |
---|---|---|---|---|
All patients | 582 | 14.8% (12-17.9) | ||
Age, y | ||||
<2 | 242 (42) | 14.8% (10.6-19.7) | ||
2-5 | 160 (27) | 12.9% (8.2-18.7) | 1.09 (0.86-1.38) | .471 |
6+ | 180 (31) | 16.0% (10.9-21.9) | ||
Continuous | 1.01 (0.96-1.06) | .811 | ||
Patient sex | ||||
Male | 360 (62) | 15.9% (11.4-21.2) | 1.01 (0.96-1.06) | .349 |
Female | 222 (38) | 13.8% (10.4-17.7) | ||
Weight | ||||
Continuous | 1.00 (0.99-1.02) | .733 | ||
Disease category | ||||
Malignant diseases | 165 (28) | 15.0% (10.0-21.0) | 1.14 (0.75-1.74) | .535 |
Nonmalignant diseases | 417 (72) | 14.5% (11.2-18.1) | ||
Presence of sex mismatch (yes) | 264 (46) | 14.4% (10.4-19.1) | 1.08 (0.73-1.61) | .696 |
CMV mismatch (yes) | 188 (33) | 15.3% (10.5-20.9) | 1.08 (0.71-1.65) | .89 |
Stem cell source | ||||
BM | 339 (58) | 11.4% (8.2-15.2) | 1.53 (1.01-2.34) | .047 |
PBSC | 171 (29) | 20.7% (14.9-27.2) | 1.14 (0.60-2.18) | .692 |
Cord | 72 (12) | 15.5% (8.2-24.8) | ||
Period of transplantation (y) | ||||
2000-2005 | 167 | 17.4% (12-23) | 0.98 (0.75-1.27) | .853 |
2006-2011 | 205 | 13.1% (14.6-27.5) | ||
2012-2016 | 210 | 14% (9.6-19.2) | ||
Donor type | ||||
Mismatched donor | 190 (33) | 24.1% (18.2-30.5) | 2.15 (1.45-3.20) | .0005 |
Matched donor | 392 (67) | 10.0% (7.2-13.3) | ||
Transplant | ||||
>1 transplant | 47 (8) | 22% (11-33) | 1.40 (0.71-2.76) | .328 |
No previous transplant | 535 (92) | 14% (11-17) | ||
Conditioning | ||||
MAC | 263 (45) | 13% (9-17) | 0.82 (0.60-1.12) | .21 |
RIC/MIC | 278 (48) | 16% (12-21) | ||
None | 41 (7) | 18% (8-31) | ||
TBI | ||||
TBI-based conditioning | 85 (15%) | 18% (11-27) | 1.41 (0.86-2.32) | .168 |
Non-TBI based | 496 (85) | 14% (11-17) | ||
Use of serotherapy | ||||
Yes | 382 (66) | 16% (13-20) | 1.16 (0.76-1.78) | .487 |
No | 200 (34) | 11% (7-16) | ||
Use of any GVHD prophylaxis | ||||
Yes | 549 (94) | 14.4% (11.5-17.5) | 1.47 (0.72-2.97) | .288 |
No | 33 (6) | 18.8% (7.6-33.7) | ||
Pre-HSCT infections | ||||
Presence of probable/confirmed fungal infection | ||||
Yes | 50 (9) | 12.3% (5-23.1) | 0.81 (0.38-1.74) | .591 |
No | 531 (91) | 14.8% (11.9-18.1) | ||
Presence of disseminated BCGiosis | ||||
Yes | 12 (2) | 16.7% (2.7-41.3) | 0.95 (0.23-3.90) | .944 |
No | 569 (98) | 14.6% (11.8-17.7) | ||
Presence of EBV Day -10 to day 0 | ||||
Yes | 9 (2) | 12.5% (0.7-42.3) | 1.34 (0.37-4.82) | .658 |
No | 572 (98) | 14.7% (11.9-17.8) | ||
Presence of AdV Day -10 to day 0 | ||||
Yes | 6 (1) | 0% | Dropped | |
No | 575 (99) | 14.8% (12.0-17.9) | ||
Presence of CMV Day -10 to day 0 | ||||
Yes | 18 (3) | 28% (10-49) | 1.74 (0.68-4.41) | .246 |
No | 564 (97) | 14% (11-17) | ||
Presence of respiratory viral infection Day -10 to day 0 | ||||
Yes | 26 (4) | 31% (15-49) | 2.26 (1.04-4.91) | .039 |
No | 556 (96) | 14% (11-17) | ||
Need to continue antimicrobials at D0 | ||||
Yes | 104 (18) | 19% (12-28) | 1.32 (0.81-2.16) | .263 |
No | 478 (82) | 13.6% (10.6-16.9) | ||
Cardiac variables | ||||
Presence of heart valve disease∗ | ||||
Yes | 15 (3) | 13.3% (2.2-34.6) | 0.84 (0.42-1.68) | .616 |
No | 566 (97) | 14.7% (11.8-17.8) | ||
Presence of a cardiac anomaly | ||||
Yes | 31 (5) | 19.5% (7.9-34.9) | 1.12 (0.49-2.55) | .79 |
No | 551 (95) | 14.3% (11.5-17.5) | ||
Pulmonary variable | ||||
Presence of dyspnea at slight activity | ||||
Yes | 3 (1) | 0% | Dropped | |
No | 578 (99) | 14.7% (11.9-17.8) | ||
Presence of dyspnea at rest or requiring oxygen | ||||
Yes | 21 (4) | 9.5% (1.6-26.1) | 0.50 (0.12-2.00) | .325 |
No | 560 (96) | 14.8% (12.0-18.0) | ||
Presence of pulmonary hypertension | ||||
Yes | 2 (<1) | 0% | Dropped | |
No | 579 (99) | 14.7% (11.9-17.8) | ||
Presence of obstructive sleep apnea | ||||
Yes | 9 (2) | 33.3% (7.8-6.2) | 2.12 (0.66-6.79) | .208 |
No | 572 (98) | 14.3% (11.5-17.4) | ||
Being on assisted ventilation at D0 | ||||
Yes | 4 (1) | 25.0% (0.9-66.5) | 1.63 (0.17-5.4) | .670 |
No | 578 (99) | 14.6% (11.8-17.6) | ||
Requiring assisted ventilation at any time (including D0) | ||||
Yes | 74 (13) | 22% (13-32) | 1.48 (0.87-2.50) | .146 |
No | 508 (87) | 14% (11-17) | ||
Presence of any respiratory structural abnormality† | ||||
Yes | 13 (2) | 23% (6-47) | 1.91 (0.72-5.06) | .19 |
No | 13 (2) | 14% (12-18) | ||
Cerebrovascular CoMs | ||||
Encephalopathy | ||||
Yes | 4 (1) | 14.6% (11.8-17.6) | 1.52 (0.22-10.3) | .669 |
No | 577 (99) | 25% (0.9-66.5) | ||
Hydrocephalus | ||||
Yes | 7 (1) | 14.3% (0.7-46.5) | 1.63 (0.43-6.13) | .472 |
No | 574 (99) | 14.6% (11.8-17.7) | ||
Sinus thrombosis | ||||
Yes | 2 (<1) | 0% | Dropped | |
No | 579 (99) | 14.7% (11.9-17.8) | ||
PRES | ||||
Yes | 3 (1) | 67% (5-95) | 5.21 (1.56-17.3) | .007 |
No | 569 (99) | 14% (12-17) | ||
Epilepsy requiring medical intervention | ||||
Yes | 6 (1) | 50% (11-80) | 3.90 (1.03-14.8) | .045 |
No | 576 (99) | 14% (11-17) | ||
Presence of any CNS structural abnormality‡ | ||||
Yes | 11 (2) | 45% (17-71) | 2.95 (1.26-6.92) | .013 |
No | 571 (98) | 14% (11-17) | ||
Presence of any of the following: CNS infection, PRES, epilepsy, or CNS structural abnormality | ||||
Yes | 32 (6) | 47% (29-63) | 3.85 (2.17-6.83) | .0005 |
No | 550 (94) | 13% (10-16) | ||
Gastrointestinal disease variables | ||||
Presence of chronic diarrhea | ||||
Yes | 44 (8) | 20.9% (10.3-33.9) | 1.35 (0.71-2.54) | .361 |
No | 538 (92) | 14.1% (11.3-17.2) | ||
Active infectious diarrhea at D0 | ||||
Yes | 12 (2) | 42% (15-67) | 3.59 (1.27-10.1) | .016 |
No | 540 (98) | 14% (11-17) | ||
Continuation on TPN at D0 | ||||
Yes | 46 (8) | 26.5% (14.7-39.8) | 1.74 (0.93-3.25) | .08 |
No | 46 (8) | 13.6% (10.8-6.7) | ||
Perianal inflammation/fistula | ||||
Yes | 5 (1) | 0% | Dropped | |
No | 576 (99) | 14.7% (11.9-17.8) | ||
Endocrine disease variables | ||||
Diabetes requiring treatment | ||||
Yes | 3 (1) | 0% | Dropped | |
No | 578 (99) | 14.7% (11.9-17.8) | ||
Presence of any other endocrine disease on treatment§ | ||||
Yes | 21 (4) | 22.0% (6.7-42.7) | 1.49 (0.63-3.48) | .362 |
No | 561 (96) | 14.4% (11.6-17.5) | ||
Hepatobiliary disease, renal and biochemical variables | ||||
Moderate/severe hepatobiliary | 153 | 16.6% (11.1-23.2) | 1.33 (1.06-1.67) | .014 |
Mild hepatobiliary | 198 | 18% (12.9-23.7) | ||
No abnormality | 231 | 10.5% (6.9-15) | ||
Serum creatinine | ||||
>1.5 ULN | 29 | 14.3% (8.3-22.0) | 1.1 (0.9-1.27) | .693 |
Above ULN to 1.5 ULN | 72 | 13.2% (10.2-16.8) | ||
Normal | 481 | 13.5% (10.3-17.1) | ||
Presence of renal malformation | ||||
Yes | 5 (1) | 20% (1-58) | 1.29 (0.18-9.48) | .801 |
No | 577 (99) | 15% (12-18) | ||
Serum albumin level | ||||
<25 g/L | 10 (2) | 50.0% (18.4-75.3) | 1.80 (1.23-2.65) | .003 |
≥25-35 g/L | 155 (27) | 20.7% (14.6-27.5) | ||
>35 g/L | 416 (52) | 11.5% (8.6-14.9) | ||
Other comorbidities | ||||
Having a prior surgery | ||||
Yes | 56 (10) | 13.0% (5.7-23.4) | 0.93 (0.46-1.86) | .838 |
No | 525 (90) | 14.8% (11.9-18.1) | ||
Having a prior malignancy | ||||
Yes | 15 (3) | 18.3% (2.9-44.4) | 1.83 (0.76-4.40) | .180 |
No | 566 (97) | 14.6% (11.8-17.7) | ||
Prior autoimmunity or immune dysregulation | ||||
Yes | 63 | 14.4% (11.5-17.6) | 1.02 | .949 |
No | 519 | 16.45% (8.4-26.8) | ||
Being on prednisolone ≥0.3 mg/kg per day at D0 | ||||
Yes | 44 (8) | 27.5% (15.3-21.1) | 1.98 (1.08-3.61) | .026 |
No | 537 (92) | 13.6% (10.8- 16.4) |
95% CI, 95% confidence interval; AdV, adenovirus; CMV, cytomegalovirus infection; CSA, cyclosporine; EBV, Epstein-Barr virus; TPN, total parenteral nutrition.
Apart from trivial regurgitant lesions.
This includes bronchiectasis, chronic lung disease, cystic fibrosis, bronchopulmonary fistula, and laryngomalacia.
Macrocephaly/microcephaly/computed tomography changes showing cerebral abnormalities.
Includes insulin-dependent diabetes mellitus, Addison’s disease, syndrome of inappropriate antidiuretic hormone secretion, hypoparathyroidism, recurrent hypoglycemia, hypercalcemia, and hypothyroidism.