Characteristics of all patients with myelofibrosis and of the training and validation cohorts undergoing allogeneic stem cell transplantation.
| Characteristic . | Total cohort (n = 361) . | Training cohort (n = 205) . | Validation cohort (n = 156) . | P . |
|---|---|---|---|---|
| Age, y | ||||
| Median (range) | 56 (18-75) | 57 (29-75) | 55 (18-70) | <.001 |
| Male sex | 211 (58) | 122 (59) | 89 (57) | .667 |
| Diagnosis before transplant | .237 | |||
| PMF | 260 (72) | 153 (75) | 107 (69) | |
| Post-ET/PV | 101 (28) | 52 (25) | 49 (31) | |
| Blood levels, median (range) | ||||
| Hemoglobin, g/dL | 9.5 (5.6-17.9) | 9.5 (5.6-17.9) | 9.5 (5.6-17.7) | .549 |
| Leukocytes, ×109/L | 8.1 (0.4-168.8) | 9.1 (0.8-168.8) | 7.5 (0.4-103.0) | .098 |
| Platelets, ×109/L | 150 (4-2513) | 171 (5-2437) | 124 (4-2513) | .154 |
| Peripheral blasts, % | 1 (0-19) | 1 (0-19) | 1 (0-19) | .271 |
| BM fibrosis grade >1 | 288 (80) | 172 (84) | 116 (74) | .016 |
| KPS, % | .284 | |||
| 90-100 | 208 (58) | 113 (55) | 95 (61) | |
| <90 | 153 (42) | 92 (45) | 61 (39) | |
| Constitutional symptoms | 208 (58) | 148 (73) | 60 (39) | <.001 |
| Transfusion dependence | 150 (42) | 97 (47) | 53 (34) | .002 |
| Cytogenetics | 202 (56) | 150 (73) | 52 (33) | <.001 |
| Driver mutation | .017 | |||
| CALR | 73 (20) | 44 (22) | 29 (19) | |
| MPL | 18 (5) | 10 (5) | 8 (5) | |
| JAK2 | 206 (57) | 126 (62) | 80 (51) | |
| Triple negative | 64 (18) | 25 (12) | 39 (25) | |
| Number of mutations | <.001 | |||
| 0-3 | 303 (84) | 157 (77) | 146 (94) | |
| >3 | 58 (16) | 48 (23) | 10 (6) | |
| DIPSS* | .008 | |||
| Low | 23 (9) | 8 (5) | 15 (14) | |
| Intermediate 1 | 80 (31) | 42 (28) | 38 (36) | |
| Intermediate 2 | 120 (46) | 75 (49) | 45 (42) | |
| High | 37 (14) | 28 (18) | 9 (8) | |
| MIPSS* | <.001 | |||
| Low | 4 (1) | 0 (0) | 4 (4) | |
| Intermediate | 88 (34) | 41 (27) | 47 (44) | |
| High | 168 (65) | 112 (73) | 56 (52) | |
| MYSEC-PM† | .063 | |||
| Low | 24 (24) | 7 (14) | 17 (35) | |
| Intermediate 1 | 39 (38) | 23 (44) | 16 (33) | |
| Intermediate 2 | 25 (25) | 13 (25) | 12 (25) | |
| High | 13 (13) | 9 (17) | 4 (8) | |
| Time to transplant, months | ||||
| Median (range) | 23.3 (0.5-526.5) | 28.4 (0.5-526.5) | 20.0 (1.5-305.3) | .234 |
| Conditioning intensity | <.001 | |||
| Reduced | 230 (64) | 196 (96) | 34 (22) | |
| Myeloablative | 131 (36) | 9 (4) | 122 (78) | |
| CMV status patient/donor | .157 | |||
| −/− | 104 (29) | 63 (31) | 41 (26) | |
| −/+ | 41 (11) | 20 (10) | 21 (13) | |
| +/− | 51 (14) | 23 (11) | 28 (18) | |
| +/+ | 165 (46) | 99 (48) | 66 (42) | |
| HLA-match | .017 | |||
| Matched related | 96 (26) | 51 (25) | 45 (29) | |
| Matched unrelated | 165 (46) | 86 (42) | 79 (51) | |
| Mismatched related | 4 (1) | 1 (1) | 3 (2) | |
| Mismatched unrelated | 96 (26) | 67 (33) | 29 (19) | |
| Graft source | .978 | |||
| PB | 347 (96) | 197 (96) | 150 (96) | |
| BM | 14 (4) | 8 (4) | 6 (4) | |
| Splenectomy before transplant | 48 (13) | 26 (13) | 22 (14) | .755 |
| Ruxolitinib before transplant | 79 (22) | 48 (24) | 31 (20) | .520 |
| Characteristic . | Total cohort (n = 361) . | Training cohort (n = 205) . | Validation cohort (n = 156) . | P . |
|---|---|---|---|---|
| Age, y | ||||
| Median (range) | 56 (18-75) | 57 (29-75) | 55 (18-70) | <.001 |
| Male sex | 211 (58) | 122 (59) | 89 (57) | .667 |
| Diagnosis before transplant | .237 | |||
| PMF | 260 (72) | 153 (75) | 107 (69) | |
| Post-ET/PV | 101 (28) | 52 (25) | 49 (31) | |
| Blood levels, median (range) | ||||
| Hemoglobin, g/dL | 9.5 (5.6-17.9) | 9.5 (5.6-17.9) | 9.5 (5.6-17.7) | .549 |
| Leukocytes, ×109/L | 8.1 (0.4-168.8) | 9.1 (0.8-168.8) | 7.5 (0.4-103.0) | .098 |
| Platelets, ×109/L | 150 (4-2513) | 171 (5-2437) | 124 (4-2513) | .154 |
| Peripheral blasts, % | 1 (0-19) | 1 (0-19) | 1 (0-19) | .271 |
| BM fibrosis grade >1 | 288 (80) | 172 (84) | 116 (74) | .016 |
| KPS, % | .284 | |||
| 90-100 | 208 (58) | 113 (55) | 95 (61) | |
| <90 | 153 (42) | 92 (45) | 61 (39) | |
| Constitutional symptoms | 208 (58) | 148 (73) | 60 (39) | <.001 |
| Transfusion dependence | 150 (42) | 97 (47) | 53 (34) | .002 |
| Cytogenetics | 202 (56) | 150 (73) | 52 (33) | <.001 |
| Driver mutation | .017 | |||
| CALR | 73 (20) | 44 (22) | 29 (19) | |
| MPL | 18 (5) | 10 (5) | 8 (5) | |
| JAK2 | 206 (57) | 126 (62) | 80 (51) | |
| Triple negative | 64 (18) | 25 (12) | 39 (25) | |
| Number of mutations | <.001 | |||
| 0-3 | 303 (84) | 157 (77) | 146 (94) | |
| >3 | 58 (16) | 48 (23) | 10 (6) | |
| DIPSS* | .008 | |||
| Low | 23 (9) | 8 (5) | 15 (14) | |
| Intermediate 1 | 80 (31) | 42 (28) | 38 (36) | |
| Intermediate 2 | 120 (46) | 75 (49) | 45 (42) | |
| High | 37 (14) | 28 (18) | 9 (8) | |
| MIPSS* | <.001 | |||
| Low | 4 (1) | 0 (0) | 4 (4) | |
| Intermediate | 88 (34) | 41 (27) | 47 (44) | |
| High | 168 (65) | 112 (73) | 56 (52) | |
| MYSEC-PM† | .063 | |||
| Low | 24 (24) | 7 (14) | 17 (35) | |
| Intermediate 1 | 39 (38) | 23 (44) | 16 (33) | |
| Intermediate 2 | 25 (25) | 13 (25) | 12 (25) | |
| High | 13 (13) | 9 (17) | 4 (8) | |
| Time to transplant, months | ||||
| Median (range) | 23.3 (0.5-526.5) | 28.4 (0.5-526.5) | 20.0 (1.5-305.3) | .234 |
| Conditioning intensity | <.001 | |||
| Reduced | 230 (64) | 196 (96) | 34 (22) | |
| Myeloablative | 131 (36) | 9 (4) | 122 (78) | |
| CMV status patient/donor | .157 | |||
| −/− | 104 (29) | 63 (31) | 41 (26) | |
| −/+ | 41 (11) | 20 (10) | 21 (13) | |
| +/− | 51 (14) | 23 (11) | 28 (18) | |
| +/+ | 165 (46) | 99 (48) | 66 (42) | |
| HLA-match | .017 | |||
| Matched related | 96 (26) | 51 (25) | 45 (29) | |
| Matched unrelated | 165 (46) | 86 (42) | 79 (51) | |
| Mismatched related | 4 (1) | 1 (1) | 3 (2) | |
| Mismatched unrelated | 96 (26) | 67 (33) | 29 (19) | |
| Graft source | .978 | |||
| PB | 347 (96) | 197 (96) | 150 (96) | |
| BM | 14 (4) | 8 (4) | 6 (4) | |
| Splenectomy before transplant | 48 (13) | 26 (13) | 22 (14) | .755 |
| Ruxolitinib before transplant | 79 (22) | 48 (24) | 31 (20) | .520 |