Data cleaning Original datan=8479 NCC/ptkgN=7958 NCC10x10E628 8139 NCC/kgPage 1-2) Covariate cleaningcovariate Page Page5-8
Kaplan-Meier method, Log-rank, Cox hazard model Overall survival Cumulative incidence method, Gray test, Competing hazard regression model PltRet /GVHD Logistic model PltRet
Patient characteristics (n=8139) NCC/kg Total Adult 1,134 2,775 2,237 617 6,763 Child 119 305 440 512 1,376 Total 1,253 3, 080 2,677 1,129 8,139 Frequency 0 200 400 600 800 1000 Adult (0.0335-12.1, med2.81) 0 5 10 15 NCC/Pt kg Excel) Frequency 0 100 200 300 Child (0.0639-19.3, med3.51) 0 5 10 15 20 NCC/Pt kg
Neu500 Adult Cumulative incidence (n=5020) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P<0.0000001 97.2% d19 96.7% d18 98.1% d17 98.6% d16 0 20 40 60 80 Days
Neu500 Adult Cumulative incidence (n=4981) Benign (n=206) Malignancy (n=4775) 0.0 0.2 0.4 0.6 0.8 1.0 96.6% d 93.0% d 97.1% d 100 % d 0.0 0.2 0.4 0.6 0.8 1.0 97.9% d 97.5% d 99.1% d 99.2 % d 0 20 40 60 80 0 20 40 60 80
Neu500 Child Cumulative incidence (n=1320) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P=0.000033 93.5% d20 95.6% d19 97.6% d18 97.9% d17 0 20 40 60 80 Days
Neu500 Child Cumulative incidence (n=1320) Benign (n=245) Malignancy (n=1075) 0.0 0.2 0.4 0.6 0.8 1.0 81.8% d19 96.6% d18 95.1% d18 96.7% d16 P=0.012 0.0 0.2 0.4 0.6 0.8 1.0 96.3% d20 95.9% d19 97.4% d18 98.3 % d17 P=0.0008 0 20 40 60 80 Days 0 20 40 60 80 Days
Plt5 Adult Cumulative incidence (n=5039) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P<0.00001 77.1% d36 79.1% d33 83.3% d29 87.1% d27 0 50 100 150 Days
Plt5 Child Cumulative incidence (n=1323) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P<0.000001 76.8% d39 78.7% d40 84.3% d32 88.5% d29 0 50 100 150 Days
Ret1 Adult Cumulative incidence (n=5039) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P<0.00001 87.2% d27 87.3% d25 89.1% d24 91.7% d23 0 20 40 60 80 100 120 Days
Ret1 Child Cumulative incidence (n=1323) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P<0.00001 93.9% d26 93.8% d26 92.7% d24 94.5% d21 0 20 40 60 80 100 120 Days
OS (n=314) 0.00 0.25 0.50 0.75 1.00 Adult/ Benign (n=19) Child/ Benign (n=9) Adult/ Malignancy (n=251) Child/ Malignancy (n=35) 0 1000 2000 3000 4000 Days after SCT
Adult Overall survival (N=5096) 0.00 0.25 0.50 0.75 1.00 Kaplan-Meier survival estimates P=0.0138 0 50 100 150 analysis time cd cd cd cd
Adult Overall survival (N=4868) 0.00 0.25 0.50 0.75 1.00 cd = /H cd = /H cd = /H cd = /H cd = /S cd = /S cd = /S cd = /S 0 50 100 150 Months after SCT
Adult Overall survival Benign disease (n=209) Malignant disease (n=4859) 0.00 0.25 0.50 0.75 1.00 cd = cd = cd = cd = 0.00 0.25 0.50 0.75 1.00 cd = /H cd = /H cd = /H cd = /H cd = /S cd = /S cd = /S cd = /S 0 50 100 150 Months after SCT 0 50 100 150 Months after SCT
Children Overall survival (N=1332) 0.00 0.25 0.50 0.75 1.00 Kaplan-Meier survival estimates P=0.0099 0 50 100 150 analysis time cd cd cd cd
Children Overall survival (N=1245) 0.00 0.25 0.50 0.75 1.00 cd = /H cd = /H cd = /H cd = /H cd = /S cd = /S cd = /S cd = /S 0 50 100 150 Months after SCT
Children Overall survival Benign disease (n=247) Malignant disease (n=1067) cd = /H cd = /S 0.00 0.25 0.50 0.75 1.00 cd = cd = cd = cd = 0 50 100 150 Months after SCT 0.00 0.25 0.50 0.75 1.00 cd = /H cd = /S cd = /H cd = /S cd = /H cd = /S 0 50 100 150 Months after SCT
NRM & Relapse Adult Cumulative incidence (n=4836) Probability 0.0 0.2 0.4 0.6 0.8 1.0 Months after SCT NRM Relapse 43% 23% 39% 21% 42% 19% 33% 18% NRM (P=0.024) Relapse (P=0.10) 0 50 100 150
Probability 0.0 0.2 0.4 0.6 0.8 1.0 NRM Child Cumulative incidence (n=1273) Months after SCT NRM 37% 25% 23% 21% NRM (P=0.013) 0 50 100 150
Relapse Child Cumulative incidence (n=1273) Probability 0.0 0.2 0.4 0.6 0.8 1.0 Relapse 22% 20% 22% 19% Relapse (P=0.83) 0 50 100 150 Months after SCT
agvhdi Adult Cumulative incidence (n=4741) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P=0.0566 (Gray test) 65.4% 67.9% 69.0% 70.8% 0 20 40 60 80 100 Months after SCT
agvhdii Adult Cumulative incidence (n=4741) Probability 0.0 0.2 0.4 0.6 0.8 1.0 42.1% 43.8% 43.1% 43.0% P=0.808 (Gray test) 0 20 40 60 80 100 Months after SCT
agvhdiii Adult Cumulative incidence (n=4741) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P=0.466 (Gray test) 17.0% 18.0% 19.4% 18.4% 0 20 40 60 80 100 Months after SCT
agvhdi Child Cumulative incidence (n=1261) Probability 0.0 0.2 0.4 0.6 0.8 1.0 P=0.0922 (Gray test) 62.4% 70.4% 74.4% 68.4% 0 20 40 60 80 100 Months after SCT
agvhdii Child Cumulative incidence (n=1261) Probability 0.0 0.2 0.4 0.6 0.8 1.0 38.5% 50.0% 45.2% 46.0% P=0.2767 (Gray test) 0 20 40 60 80 100 Months after SCT
agvhdiii Child Cumulative incidence (n=1261) Probability 0.0 0.2 0.4 0.6 0.8 1.0 21.1% 20.1% 22.8% 22.6% P=0.760 (Gray test) 0 20 40 60 80 100 Months after SCT
cgvhd(lm+ex) Adult Cumulative incidence (n=3725) Probability 0.0 0.2 0.4 0.6 0.8 1.0 46.2% 44.0% 43.0% 46.7% P=0.45 (Gray test) 0 50 100 150 Months after day100
cgvhd(lm/ex) Adult Cumulative incidence (n=3725) Probability 0.0 0.2 0.4 0.6 0.8 1.0 LM EX LM EX 16.7% 29.5% 16.1% 27.9% 14.0% 29.0% 15.1% 31.7% P=0.31 P=0.54 (Gray test) 0 50 100 150 Months after day100
cgvhd(lm+ex) Child Cumulative incidence (n=1075) Probability 0.0 0.2 0.4 0.6 0.8 1.0 34.0% 32.2% 33.6% 33.4% P=0.99 (Gray test) 0 50 100 150 Months after day100
cgvhd(lm/ex) Child Cumulative incidence (n=1075) Probability 0.0 0.2 0.4 0.6 0.8 1.0 LM EX LM EX 15.8% 18.2% 15.5% 16.7% 13.0% 20.6% 14.9% 18.5% P=0.81 P=0.72 (Gray test) 0 50 100 150 Months after day100
30Early death Adult P=0.626) Children P=0.194)
Ret NRM (Cox Grade IGVHDHR 1.061)Grade II-IV, III-IV GVHD 30 NRM GVHDaGVHD 4x10 8 /kggvhd GVHD Competing hazardcox
TBIFK MinorMajorTCD) HLAGVHD -2 Logistic model TBI HLA rejection
TBI HLARejection FK Ret HLA
FK HLA Rejection NRM FK HLA Rejection HLAGVHD VPFK Cox
Grade IGVHD HLAGVHDTBI RISTFK Grade II-IVGVHD HLAGVHD Grade III-IVGVHD HLA GVHD FK GVHDLM+EX) DnPtTBIGradeI agvhdgradeiiagvhd
11. HLAGVHD 12. HLAGVHD 13. HLAGVHD DnPt TBI FK 14. T HLAGVHD RIST FK 15.30 THLAGVHD
2x10 8 /Pt.kg68 0.3 2.0 (x10 8 /Pt.kg) 1x10 8 /Pt.kg6 37.5kg
400 2x10 8 /Pt.kg
+400ml