Main characteristics of a series of 5183 patients with MF undergoing allo-HCT and of the training and test cohorts
Characteristic . | Group . | Missing (%) . | Total cohort . | Training set . | Test set . | P value . |
---|---|---|---|---|---|---|
N (%) . | n (%) . | n (%) . | ||||
No. of patients | 5183 (100) | 3887 (100) | 1296 (100) | |||
Age at allo-HCT, y∗ | 58.3 (52-63.5) | 58.2 (51.8-63.4) | 58.6 (52.7-63.8) | .072 | ||
Age at allo-HCT | <60 y | 3003 (57.9) | 2274 (58.5) | 729 (56.2) | .164 | |
Patient sex | Male | 3242 (62.6) | 2412 (62.1) | 830 (64.0) | .21 | |
Year of MF diagnosis | <2000 | 615 (11.9) | 455 (11.7) | 160 (12.3) | .21 | |
2000-2010 | 1765 (34.1) | 1310 (33.7) | 455 (35.1) | |||
2010-2015 | 1422 (27.4) | 1057 (27.2) | 365 (28.2) | |||
≥2015 | 1381 (26.6) | 1065 (27.4) | 316 (24.4) | |||
MF type | PMF | 3743 (72.2) | 2807 (72.2) | 936 (72.2) | 1.00 | |
SMF | 1440 (27.8) | 1080 (27.8) | 360 (27.8) | |||
JAK2 inhibitor treatment before allo-HCT | Yes | 1503 (29) | 1039 (28.2) | 764 (27.9) | 275 (29.2) | .459 |
Genotype | JAK2+ | 2422 (46.7) | 2156 (78.1) | 1625 (77.7) | 531 (79.3) | .43 |
MPL+ | 94 (3.4) | 68 (3.3) | 26 (3.9) | |||
CALR+ | 377 (13.7) | 290 (13.9) | 87 (13.0) | |||
Triple negative | 134 (4.9) | 108 (5.2) | 26 (3.9) | |||
Constitutional symptoms at allo-HCT | Yes | 3089 (59.6) | 911 (43.5) | 693 (44.3) | 218 (41.3) | .255 |
Hemoglobin at allo-HCT, g/dL∗ | 2575 (49.7) | 9.2 (8.1-10.5) | 9.2 (8.1-10.5) | 9.2 (8.2-10.6) | .43 | |
Leukocyte count at allo-HCT, ×109/L∗ | 2600 (50.2) | 6.9 (3.5-14.4) | 6.8 (3.5-14.3) | 7 (3.6-14.4) | .336 | |
Blood blasts at allo-HCT, %∗ | 3095 (59.7) | 1 (0-3) | 1 (0-3) | 1 (0-3) | .637 | |
Platelets at allo-HCT, ×109/L∗ | 2640 (50.9) | 117 (53-242.5) | 114.5 (53-237) | 125 (51-274) | .039 | |
Splenectomy before allo-HCT | Yes | 2536 (48.9) | 324 (12.2) | 231 (11.7) | 93 (14.0) | .129 |
Spleen size below costal margin, cm, by physical examination∗ | 3857 (74.4) | 5 (0-10) | 5 (1-11) | 5 (0-10) | .184 | |
Spleen span by ultrasound or CT scan, max diameter, cm∗ | 4283 (82.6) | 20 (16-23) | 20 (16-23) | 20 (16.2-23) | .72 | |
HCT-CI risk group | Low risk (0 points) | 1360 (26.2) | 2041 (53.4) | 1538 (53.7) | 503 (52.4) | .745 |
Intermediate risk (1-2 points) | 910 (23.8) | 674 (23.5) | 236 (24.6) | |||
High risk (≥3 points) | 872 (22.8) | 651 (22.7) | 221 (23.0) | |||
KPS score at allo-HCT | 90-100 | 489 (9.4) | 3111 (66.3) | 2316 (65.8) | 795 (67.7) | .508 |
80 | 1232 (26.2) | 937 (26.6) | 295 (25.1) | |||
<80 | 351 (7.5) | 266 (7.6) | 85 (7.2) | |||
DIPSS risk group at allo-HCT | Low risk | 2715 (52.4) | 60 (2.4) | 40 (2.2) | 20 (3.2) | .286 |
Intermediate-1 | 919 (37.2) | 695 (37.7) | 224 (36.0) | |||
Intermediate-2 | 954 (38.7) | 702 (38.0) | 252 (40.4) | |||
High risk | 535 (21.7) | 408 (22.1) | 127 (20.4) | |||
CIBMTR risk score at allo-HCT | Low | 2640 (50.9) | 1020 (40.1) | 763 (39.6) | 257 (41.6) | .641 |
Intermediate | 1313 (51.6) | 1004 (52.2) | 309 (50.0) | |||
High | 210 (8.3) | 158 (8.2) | 52 (8.4) | |||
Donor type | Identical sibling | 1534 (29.6) | 1147 (29.5) | 387 (29.9) | .919 | |
MRD (other than sibling) | 45 (0.9) | 34 (0.9) | 11 (0.8) | |||
MMRD | 339 (6.5) | 249 (6.4) | 90 (6.9) | |||
MUD | 2175 (42.0) | 1646 (42.3) | 529 (40.8) | |||
MMUD | 673 (13.0) | 504 (13.0) | 169 (13.0) | |||
Unrelated, number of mismatches unknown | 417 (8.0) | 307 (7.9) | 110 (8.5) | |||
Recipient-donor match | Recipient male with donor male | 76 (1.5) | 2297 (45.0) | 1714 (44.7) | 583 (45.9) | .614 |
Recipient male with donor female | 895 (17.5) | 666 (17.4) | 229 (18.0) | |||
Recipient female with donor male | 1147 (22.5) | 878 (22.9) | 269 (21.2) | |||
Recipient female with donor female | 768 (15.0) | 578 (15.1) | 190 (14.9) | |||
Donor age, y∗ | 1132 (21.8) | 36.6 (27.2-49.8) | 36.7 (27.2-49.6) | 36.1 (27.1-50.1) | .902 | |
CMV serology in patient/donor | −/− | 233 (4.5) | 1437 (29.0) | 1056 (28.4) | 381 (30.8) | .174 |
−/+ | 464 (9.4) | 348 (9.4) | 116 (9.4) | |||
+/− | 1002 (20.2) | 776 (20.9) | 226 (18.3) | |||
+/+ | 2047 (41.4) | 1533 (41.3) | 514 (41.6) | |||
Stem cell source | Bone marrow | 409 (7.9) | 305 (7.8) | 104 (8.0) | .884 | |
Peripheral blood | 4774 (92.1) | 3582 (92.2) | 1192 (92.0) | |||
Busulfan- or melphalan-based conditioning regimen | Busulfan based | 89 (1.7) | 3522 (69.1) | 2642 (69.2) | 880 (69.0) | .861 |
Melphalan based | 718 (14.1) | 533 (14.0) | 185 (14.5) | |||
Other regimen | 854 (16.8) | 644 (16.9) | 210 (16.5) | |||
Conditioning drugs | BuCy w/wo others (MAC) | 146 (2.8) | 166 (3.3) | 137 (3.6) | 29 (2.3) | .324 |
FluBu w/wo others (MAC) | 1153 (22.9) | 858 (22.7) | 295 (23.4) | |||
FluTreo w/wo others (MAC) | 160 (3.2) | 121 (3.2) | 39 (3.1) | |||
TBI w/wo Cy w/wo others (MAC) | 135 (2.7) | 111 (2.9) | 24 (1.9) | |||
Others (MAC) | 155 (3.1) | 116 (3.1) | 39 (3.1) | |||
FluBu w/wo others (RIC) | 2135 (42.4) | 1588 (42.1) | 547 (43.3) | |||
FluMel w/wo others (RIC) | 579 (11.5) | 428 (11.3) | 151 (12.0) | |||
FluTreo w/wo others (RIC) | 143 (2.8) | 105 (2.8) | 38 (3.0) | |||
TBI w/wo Cy w/wo Flu w/wo others (RIC) | 275 (5.5) | 206 (5.5) | 69 (5.5) | |||
Others (RIC) | 136 (2.7) | 105 (2.8) | 31 (2.5) | |||
Conditioning regimen intensity | MAC | 75 (1.4) | 1802 (35.3) | 1366 (35.7) | 436 (34.1) | .32 |
RIC | 3306 (64.7) | 2463 (64.3) | 843 (65.9) | |||
TBI | Yes | 37 (0.7) | 420 (8.2) | 326 (8.4) | 94 (7.3) | .219 |
T-cell depletion | No | 148 (2.9) | 1356 (26.9) | 1010 (26.8) | 346 (27.4) | .451 |
Yes in vivo, no ex vivo | 3585 (71.2) | 2696 (71.4) | 889 (70.5) | |||
Yes ex vivo, no in vivo | 17 (0.3) | 10 (0.3) | 7 (0.6) | |||
Yes in vivo + ex vivo | 77 (1.5) | 58 (1.5) | 19 (1.5) | |||
ATG | Yes | 120 (2.3) | 3387 (66.9) | 2568 (67.7) | 819 (64.5) | .038 |
Alemtuzumab | Yes | 167 (3.2) | 306 (6.1) | 218 (5.8) | 88 (7.0) | .146 |
GVHD prophylaxis group | Post-Cy | 112 (2.2) | 415 (8.2) | 311 (8.2) | 104 (8.2) | .064 |
ATG + CNI + MMF | 1398 (27.6) | 1064 (28.0) | 334 (26.3) | |||
ATG + CNI + MTX | 1352 (26.7) | 1021 (26.9) | 331 (26.0) | |||
ATG + CNI | 354 (7.0) | 276 (7.3) | 78 (6.1) | |||
ATG w/wo other(s) | 195 (3.8) | 150 (3.9) | 45 (3.5) | |||
Post-Cy + ATG | 88 (1.7) | 57 (1.5) | 31 (2.4) | |||
CNI only | 184 (3.6) | 129 (3.4) | 55 (4.3) | |||
CNI + MMF w/wo other | 434 (8.6) | 311 (8.2) | 123 (9.7) | |||
CNI + MTX w/wo other | 522 (10.3) | 393 (10.3) | 129 (10.1) | |||
Other | 129 (2.5) | 88 (2.3) | 41 (3.2) |
Characteristic . | Group . | Missing (%) . | Total cohort . | Training set . | Test set . | P value . |
---|---|---|---|---|---|---|
N (%) . | n (%) . | n (%) . | ||||
No. of patients | 5183 (100) | 3887 (100) | 1296 (100) | |||
Age at allo-HCT, y∗ | 58.3 (52-63.5) | 58.2 (51.8-63.4) | 58.6 (52.7-63.8) | .072 | ||
Age at allo-HCT | <60 y | 3003 (57.9) | 2274 (58.5) | 729 (56.2) | .164 | |
Patient sex | Male | 3242 (62.6) | 2412 (62.1) | 830 (64.0) | .21 | |
Year of MF diagnosis | <2000 | 615 (11.9) | 455 (11.7) | 160 (12.3) | .21 | |
2000-2010 | 1765 (34.1) | 1310 (33.7) | 455 (35.1) | |||
2010-2015 | 1422 (27.4) | 1057 (27.2) | 365 (28.2) | |||
≥2015 | 1381 (26.6) | 1065 (27.4) | 316 (24.4) | |||
MF type | PMF | 3743 (72.2) | 2807 (72.2) | 936 (72.2) | 1.00 | |
SMF | 1440 (27.8) | 1080 (27.8) | 360 (27.8) | |||
JAK2 inhibitor treatment before allo-HCT | Yes | 1503 (29) | 1039 (28.2) | 764 (27.9) | 275 (29.2) | .459 |
Genotype | JAK2+ | 2422 (46.7) | 2156 (78.1) | 1625 (77.7) | 531 (79.3) | .43 |
MPL+ | 94 (3.4) | 68 (3.3) | 26 (3.9) | |||
CALR+ | 377 (13.7) | 290 (13.9) | 87 (13.0) | |||
Triple negative | 134 (4.9) | 108 (5.2) | 26 (3.9) | |||
Constitutional symptoms at allo-HCT | Yes | 3089 (59.6) | 911 (43.5) | 693 (44.3) | 218 (41.3) | .255 |
Hemoglobin at allo-HCT, g/dL∗ | 2575 (49.7) | 9.2 (8.1-10.5) | 9.2 (8.1-10.5) | 9.2 (8.2-10.6) | .43 | |
Leukocyte count at allo-HCT, ×109/L∗ | 2600 (50.2) | 6.9 (3.5-14.4) | 6.8 (3.5-14.3) | 7 (3.6-14.4) | .336 | |
Blood blasts at allo-HCT, %∗ | 3095 (59.7) | 1 (0-3) | 1 (0-3) | 1 (0-3) | .637 | |
Platelets at allo-HCT, ×109/L∗ | 2640 (50.9) | 117 (53-242.5) | 114.5 (53-237) | 125 (51-274) | .039 | |
Splenectomy before allo-HCT | Yes | 2536 (48.9) | 324 (12.2) | 231 (11.7) | 93 (14.0) | .129 |
Spleen size below costal margin, cm, by physical examination∗ | 3857 (74.4) | 5 (0-10) | 5 (1-11) | 5 (0-10) | .184 | |
Spleen span by ultrasound or CT scan, max diameter, cm∗ | 4283 (82.6) | 20 (16-23) | 20 (16-23) | 20 (16.2-23) | .72 | |
HCT-CI risk group | Low risk (0 points) | 1360 (26.2) | 2041 (53.4) | 1538 (53.7) | 503 (52.4) | .745 |
Intermediate risk (1-2 points) | 910 (23.8) | 674 (23.5) | 236 (24.6) | |||
High risk (≥3 points) | 872 (22.8) | 651 (22.7) | 221 (23.0) | |||
KPS score at allo-HCT | 90-100 | 489 (9.4) | 3111 (66.3) | 2316 (65.8) | 795 (67.7) | .508 |
80 | 1232 (26.2) | 937 (26.6) | 295 (25.1) | |||
<80 | 351 (7.5) | 266 (7.6) | 85 (7.2) | |||
DIPSS risk group at allo-HCT | Low risk | 2715 (52.4) | 60 (2.4) | 40 (2.2) | 20 (3.2) | .286 |
Intermediate-1 | 919 (37.2) | 695 (37.7) | 224 (36.0) | |||
Intermediate-2 | 954 (38.7) | 702 (38.0) | 252 (40.4) | |||
High risk | 535 (21.7) | 408 (22.1) | 127 (20.4) | |||
CIBMTR risk score at allo-HCT | Low | 2640 (50.9) | 1020 (40.1) | 763 (39.6) | 257 (41.6) | .641 |
Intermediate | 1313 (51.6) | 1004 (52.2) | 309 (50.0) | |||
High | 210 (8.3) | 158 (8.2) | 52 (8.4) | |||
Donor type | Identical sibling | 1534 (29.6) | 1147 (29.5) | 387 (29.9) | .919 | |
MRD (other than sibling) | 45 (0.9) | 34 (0.9) | 11 (0.8) | |||
MMRD | 339 (6.5) | 249 (6.4) | 90 (6.9) | |||
MUD | 2175 (42.0) | 1646 (42.3) | 529 (40.8) | |||
MMUD | 673 (13.0) | 504 (13.0) | 169 (13.0) | |||
Unrelated, number of mismatches unknown | 417 (8.0) | 307 (7.9) | 110 (8.5) | |||
Recipient-donor match | Recipient male with donor male | 76 (1.5) | 2297 (45.0) | 1714 (44.7) | 583 (45.9) | .614 |
Recipient male with donor female | 895 (17.5) | 666 (17.4) | 229 (18.0) | |||
Recipient female with donor male | 1147 (22.5) | 878 (22.9) | 269 (21.2) | |||
Recipient female with donor female | 768 (15.0) | 578 (15.1) | 190 (14.9) | |||
Donor age, y∗ | 1132 (21.8) | 36.6 (27.2-49.8) | 36.7 (27.2-49.6) | 36.1 (27.1-50.1) | .902 | |
CMV serology in patient/donor | −/− | 233 (4.5) | 1437 (29.0) | 1056 (28.4) | 381 (30.8) | .174 |
−/+ | 464 (9.4) | 348 (9.4) | 116 (9.4) | |||
+/− | 1002 (20.2) | 776 (20.9) | 226 (18.3) | |||
+/+ | 2047 (41.4) | 1533 (41.3) | 514 (41.6) | |||
Stem cell source | Bone marrow | 409 (7.9) | 305 (7.8) | 104 (8.0) | .884 | |
Peripheral blood | 4774 (92.1) | 3582 (92.2) | 1192 (92.0) | |||
Busulfan- or melphalan-based conditioning regimen | Busulfan based | 89 (1.7) | 3522 (69.1) | 2642 (69.2) | 880 (69.0) | .861 |
Melphalan based | 718 (14.1) | 533 (14.0) | 185 (14.5) | |||
Other regimen | 854 (16.8) | 644 (16.9) | 210 (16.5) | |||
Conditioning drugs | BuCy w/wo others (MAC) | 146 (2.8) | 166 (3.3) | 137 (3.6) | 29 (2.3) | .324 |
FluBu w/wo others (MAC) | 1153 (22.9) | 858 (22.7) | 295 (23.4) | |||
FluTreo w/wo others (MAC) | 160 (3.2) | 121 (3.2) | 39 (3.1) | |||
TBI w/wo Cy w/wo others (MAC) | 135 (2.7) | 111 (2.9) | 24 (1.9) | |||
Others (MAC) | 155 (3.1) | 116 (3.1) | 39 (3.1) | |||
FluBu w/wo others (RIC) | 2135 (42.4) | 1588 (42.1) | 547 (43.3) | |||
FluMel w/wo others (RIC) | 579 (11.5) | 428 (11.3) | 151 (12.0) | |||
FluTreo w/wo others (RIC) | 143 (2.8) | 105 (2.8) | 38 (3.0) | |||
TBI w/wo Cy w/wo Flu w/wo others (RIC) | 275 (5.5) | 206 (5.5) | 69 (5.5) | |||
Others (RIC) | 136 (2.7) | 105 (2.8) | 31 (2.5) | |||
Conditioning regimen intensity | MAC | 75 (1.4) | 1802 (35.3) | 1366 (35.7) | 436 (34.1) | .32 |
RIC | 3306 (64.7) | 2463 (64.3) | 843 (65.9) | |||
TBI | Yes | 37 (0.7) | 420 (8.2) | 326 (8.4) | 94 (7.3) | .219 |
T-cell depletion | No | 148 (2.9) | 1356 (26.9) | 1010 (26.8) | 346 (27.4) | .451 |
Yes in vivo, no ex vivo | 3585 (71.2) | 2696 (71.4) | 889 (70.5) | |||
Yes ex vivo, no in vivo | 17 (0.3) | 10 (0.3) | 7 (0.6) | |||
Yes in vivo + ex vivo | 77 (1.5) | 58 (1.5) | 19 (1.5) | |||
ATG | Yes | 120 (2.3) | 3387 (66.9) | 2568 (67.7) | 819 (64.5) | .038 |
Alemtuzumab | Yes | 167 (3.2) | 306 (6.1) | 218 (5.8) | 88 (7.0) | .146 |
GVHD prophylaxis group | Post-Cy | 112 (2.2) | 415 (8.2) | 311 (8.2) | 104 (8.2) | .064 |
ATG + CNI + MMF | 1398 (27.6) | 1064 (28.0) | 334 (26.3) | |||
ATG + CNI + MTX | 1352 (26.7) | 1021 (26.9) | 331 (26.0) | |||
ATG + CNI | 354 (7.0) | 276 (7.3) | 78 (6.1) | |||
ATG w/wo other(s) | 195 (3.8) | 150 (3.9) | 45 (3.5) | |||
Post-Cy + ATG | 88 (1.7) | 57 (1.5) | 31 (2.4) | |||
CNI only | 184 (3.6) | 129 (3.4) | 55 (4.3) | |||
CNI + MMF w/wo other | 434 (8.6) | 311 (8.2) | 123 (9.7) | |||
CNI + MTX w/wo other | 522 (10.3) | 393 (10.3) | 129 (10.1) | |||
Other | 129 (2.5) | 88 (2.3) | 41 (3.2) |
ATG, antithymocyte globulin; Bu, busulfan; CNI, calcineurin inhibitor; CT, computed tomography; Cy, cyclophosphamide; DIPSS, Dynamic International Prognostic Scoring System; Flu, fludarabine; MAC, myeloablative conditioning; Mel, Melphalan; MMF, mycophenolate mofetil; MMRD, mismatched related donor; max, maximum; MMUD, mismatched unrelated donor; MRD, matched related donor; MTX, methotrexate; MUD, matched unrelated donor; RIC, reduced intensity conditioning; TBI, total body irradiation; Treo, treosulfan; w/wo, with/without.
Values are median (interquartile range).