Table 3.

Patient-, transplant-, treatment-, and organ-related RF and CoM variables influencing 2-year TRM

VariableTotal 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)   
VariableTotal 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.

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