Clinical and molecular characteristics of the MPN-BP study cohort
| Characteristic . | Sample DNA obtained . | No sample/DNA not obtained . | Total . |
|---|---|---|---|
| No. of patients | 95 | 82 | 177 |
| No. of centers | 43 | 51 | 73 |
| Patient related | |||
| Age at HCT | |||
| Median (minimum-maximum), y | 59.9 (41.05-81.55) | 58.48 (40.29-74.04) | 58.75 (40.29-81.55) |
| Age group, no. (%) | |||
| 40-49 y | 16 (16.8) | 14 (17.1) | 30 (16.9) |
| 50-59 y | 33 (34.7) | 40 (48.8) | 73 (41.2) |
| 60-69 y | 36 (37.9) | 23 (28) | 59 (33.3) |
| a≥70 y | 10 (10.5) | 5 (6.1) | 15 (8.5) |
| Sex, no. (%) | |||
| Male | 64 (67.4) | 52 (63.4) | 116 (65.5) |
| Female | 31 (32.6) | 30 (36.6) | 61 (34.5) |
| Karnofsky performance score, no. (%) | |||
| 90-100 | 46 (48.4) | 42 (51.2) | 88 (49.7) |
| <90 | 47 (49.5) | 37 (45.1) | 84 (47.5) |
| Missing | 2 (2.1) | 3 (3.7) | 5 (2.8) |
| HCT-CI, no. (%) | |||
| 0 | 16 (16.8) | 17 (20.7) | 33 (18.6) |
| 1-2 | 19 (20) | 12 (14.6) | 31 (17.5) |
| ≥3 | 31 (32.6) | 19 (23.2) | 50 (28.2) |
| NA, before 2007 | 28 (29.5) | 33 (40.2) | 61 (34.5) |
| Missing | 1 (1.1) | 1 (1.2) | 2 (1.1) |
| Disease related | |||
| Type of MPN at original diagnosis, no. (%) | |||
| PV | 26 (27.4) | 20 (24.4) | 46 (26) |
| ET | 25 (26.3) | 26 (31.7) | 51 (28.8) |
| Primary myelofibrosis | 32 (33.7) | 28 (34.1) | 60 (33.9) |
| MPN-unclassifiable | 12 (12.6) | 8 (9.8) | 20 (11.3) |
| Time between diagnosis of MPN to transformation to BP | |||
| Median (minimum-maximum) | 4.02 (0-27.3) | 2.55 (0-30.14) | 3.12 (0-30.14) |
| Age groups, no. (%) | |||
| <2 y | 36 (37.9) | 34 (41.5) | 70 (39.5) |
| 2-5 y | 16 (16.8) | 12 (14.6) | 28 (15.8) |
| >5 y | 36 (37.9) | 27 (32.9) | 63 (35.6) |
| Missing | 7 (7.4) | 9 (11) | 16 (9) |
| WBC at diagnosis of MPN-BP, no. (%) | |||
| ≤30 × 109/L | 64 (67.4) | 57 (69.5) | 121 (68.4) |
| 30-100 × 109/L | 18 (18.9) | 13 (15.9) | 31 (17.5) |
| >100 × 109/L | 5 (5.3) | 4 (4.9) | 9 (5.1) |
| Missing | 8 (8.4) | 8 (9.8) | 16 (9) |
| Cytogenetics, no. (%) | |||
| Favorable | 1 (1.1) | 0 | 1 (0.6) |
| Intermediate | 50 (52.6) | 46 (56.1) | 96 (54.2) |
| Poor-risk | 39 (41.1) | 28 (34.1) | 67 (37.9) |
| Not tested | 2 (2.1) | 3 (3.7) | 5 (2.8) |
| Missing | 3 (3.2) | 5 (6.1) | 8 (4.5) |
| Blast percentage in BM before HCT | |||
| Median (minimum-maximum) | 2 (0-50) | 2 (0-60) | 2 (0-60) |
| Blast % in PB before HCT | |||
| Median (minimum-maximum) | 0 (0-56) | 0 (0-96) | 0 (0-96) |
| Disease status at HCT, no. (%) | |||
| PB/BM blasts <5% | 54 (56.8) | 47 (57.3) | 101 (57.1) |
| Active leukemia* | 41 (43.2) | 35 (42.7) | 76 (42.9) |
| No. of mutations, no. (%) | |||
| 0 | 4 (4.2) | — | |
| 1 | 20 (21.1) | — | |
| 2 | 22 (23.2) | — | |
| 3 | 19 (20) | — | |
| 4 | 15 (15.8) | — | |
| 5 | 7 (7.4) | — | |
| 6 | 5 (5.3) | — | |
| 7 | 1 (1.1) | — | |
| 8 | 2 (2.1) | — | |
| Mutations, no. (%) | |||
| ASXL1 | 21 (22.1) | — | |
| BCOR | 2 (2.1) | — | |
| CALR | 12 (12.6) | — | |
| CBL | 6 (6.3) | — | |
| CEBPA | 1 (1.1) | — | |
| CUX1 | 5 (5.3) | — | |
| DNMT3A | 13 (13.7) | — | |
| EZH2 | 3 (3.2) | — | |
| GATA2 | 2 (2.1) | — | |
| GNAS | 2 (2.1) | — | |
| IDH1 | 2 (2.1) | — | |
| IDH2 | 6 (6.3) | — | |
| JAK2 | 52 (54.7) | — | |
| KRAS | 1 (1.1) | — | |
| MPL | 1 (1.1) | — | |
| NRAS | 4 (4.2) | — | |
| PHF6 | 8 (8.4) | — | |
| PPM1D | 3 (3.2) | — | |
| RAD21 | 1 (1.1) | — | |
| RUNX1 | 13 (13.7) | — | |
| SETBP1 | 4 (4.2) | — | |
| SF3B1 | 10 (10.5) | — | |
| SH2B3 | 4 (4.2) | — | |
| SRSF2 | 15 (15.8) | — | |
| STAG2 | 2 (2.1) | — | |
| TET2 | 18 (18.9) | — | |
| TP53 | 22 (23.2) | — | |
| U2AF1 | 6 (6.3) | — | |
| ZRSR2 | 1 (1.1) | — | |
| Therapy related | |||
| Induction therapy, no. (%) | |||
| 7 + 3 ± other | 49 (51.6) | 51 (62.2) | 100 (56.5) |
| Cytarabine based | 9 (9.5) | 9 (11) | 18 (10.2) |
| Hypomethylating | 6 (6.3) | 1 (1.2) | 7 (4) |
| Other | 2 (2.1) | 1 (1.2) | 3 (1.7) |
| No therapy given | 1 (1.1) | 10 (12.2) | 11 (6.2) |
| Missing | 28 (29.5) | 10 (12.2) | 38 (21.5) |
| Transplant related | |||
| Time from diagnosis of BP to HCT | |||
| Median, mo | 5.23 | 5.28 | 5.26 |
| Time frame, no. (%) | |||
| <6 mo | 55 (57.9) | 50 (61) | 105 (59.3) |
| 6-12 mo | 17 (17.9) | 21 (25.6) | 38 (21.5) |
| ≥12 mo | 23 (24.2) | 11 (13.4) | 34 (19.2) |
| Graft source, no. (%) | |||
| BM | 14 (14.7) | 8 (9.8) | 22 (12.4) |
| PB | 81 (85.3) | 74 (90.2) | 155 (87.6) |
| Donor type, no. (%) | |||
| HLA-identical sibling | 7 (7.4) | 47 (57.3) | 54 (30.5) |
| Other related | 3 (3.2) | 10 (12.2) | 13 (7.3) |
| Well-matched URD | 66 (69.5) | 17 (20.7) | 83 (46.9) |
| Partially matched/mismatched URD | 19 (20) | 8 (9.8) | 27 (15.3) |
| D–R sex match, no. (%) | |||
| M-M | 47 (49.5) | 29 (35.4) | 76 (42.9) |
| M-F | 19 (20) | 17 (20.7) | 36 (20.3) |
| F-M | 16 (16.8) | 23 (28) | 39 (22) |
| F-F | 12 (12.6) | 13 (15.9) | 25 (14.1) |
| Missing | 1 (1.1) | 0 | 1 (0.6) |
| D-R CMV status, no. (%) | |||
| +/+ | 18 (18.9) | 31 (37.8) | 49 (27.7) |
| +/− | 9 (9.5) | 4 (4.9) | 13 (7.3) |
| −/+ | 37 (38.9) | 21 (25.6) | 58 (32.8) |
| −/− | 29 (30.5) | 24 (29.3) | 53 (29.9) |
| Missing | 2 (2.1) | 2 (2.4) | 4 (2.3) |
| Conditioning regimen, no. (%) | |||
| MAC, TBI-based | 8 (8.4) | 13 (15.9) | 21 (11.9) |
| MAC, chemotherapy-based | 46 (48.4) | 26 (31.7) | 72 (40.7) |
| RIC/NST | 41 (43.2) | 43 (52.4) | 84 (47.5) |
| GVHD prophylaxis, no. (%) | |||
| CNI + MMF ± others | 26 (27.4) | 21 (25.6) | 47 (26.6) |
| CNI + MTX ± others | 57 (60) | 42 (51.2) | 99 (55.9) |
| CNI ± others | 8 (8.4) | 11 (13.4) | 19 (10.7) |
| Post-CY | 3 (3.2) | 7 (8.5) | 10 (5.6) |
| Other | 1 (1.1) | 0 | 1 (0.6) |
| Missing | 0 | 1 (1.2) | 1 (0.6) |
| In vivo T-cell depletion, no. (%) | |||
| No | 69 (72.6) | 62 (75.6) | 131 (74) |
| Yes | 26 (27.4) | 20 (24.4) | 46 (26) |
| Planned G-CSF/GM-CSF within 7 d post-HCT, no. (%) | |||
| No | 72 (75.8) | 57 (69.5) | 129 (72.9) |
| Yes | 21 (22.1) | 25 (30.5) | 46 (26) |
| Missing | 2 (2.1) | 0 | 2 (1.1) |
| Year of transplant, no. (%) | |||
| 2001-2005 | 13 (13.7) | 26 (31.7) | 39 (22) |
| 2006-2010 | 42 (44.2) | 30 (36.6) | 72 (40.7) |
| 2011-2015 | 40 (42.1) | 26 (31.7) | 66 (37.3) |
| Follow-up of survivors, median (minimum-maximum), mo | 70.63 (13.55-143.16) | 68.82 (22.2-169.38) | 70.63 (13.55-169.38) |
| Characteristic . | Sample DNA obtained . | No sample/DNA not obtained . | Total . |
|---|---|---|---|
| No. of patients | 95 | 82 | 177 |
| No. of centers | 43 | 51 | 73 |
| Patient related | |||
| Age at HCT | |||
| Median (minimum-maximum), y | 59.9 (41.05-81.55) | 58.48 (40.29-74.04) | 58.75 (40.29-81.55) |
| Age group, no. (%) | |||
| 40-49 y | 16 (16.8) | 14 (17.1) | 30 (16.9) |
| 50-59 y | 33 (34.7) | 40 (48.8) | 73 (41.2) |
| 60-69 y | 36 (37.9) | 23 (28) | 59 (33.3) |
| a≥70 y | 10 (10.5) | 5 (6.1) | 15 (8.5) |
| Sex, no. (%) | |||
| Male | 64 (67.4) | 52 (63.4) | 116 (65.5) |
| Female | 31 (32.6) | 30 (36.6) | 61 (34.5) |
| Karnofsky performance score, no. (%) | |||
| 90-100 | 46 (48.4) | 42 (51.2) | 88 (49.7) |
| <90 | 47 (49.5) | 37 (45.1) | 84 (47.5) |
| Missing | 2 (2.1) | 3 (3.7) | 5 (2.8) |
| HCT-CI, no. (%) | |||
| 0 | 16 (16.8) | 17 (20.7) | 33 (18.6) |
| 1-2 | 19 (20) | 12 (14.6) | 31 (17.5) |
| ≥3 | 31 (32.6) | 19 (23.2) | 50 (28.2) |
| NA, before 2007 | 28 (29.5) | 33 (40.2) | 61 (34.5) |
| Missing | 1 (1.1) | 1 (1.2) | 2 (1.1) |
| Disease related | |||
| Type of MPN at original diagnosis, no. (%) | |||
| PV | 26 (27.4) | 20 (24.4) | 46 (26) |
| ET | 25 (26.3) | 26 (31.7) | 51 (28.8) |
| Primary myelofibrosis | 32 (33.7) | 28 (34.1) | 60 (33.9) |
| MPN-unclassifiable | 12 (12.6) | 8 (9.8) | 20 (11.3) |
| Time between diagnosis of MPN to transformation to BP | |||
| Median (minimum-maximum) | 4.02 (0-27.3) | 2.55 (0-30.14) | 3.12 (0-30.14) |
| Age groups, no. (%) | |||
| <2 y | 36 (37.9) | 34 (41.5) | 70 (39.5) |
| 2-5 y | 16 (16.8) | 12 (14.6) | 28 (15.8) |
| >5 y | 36 (37.9) | 27 (32.9) | 63 (35.6) |
| Missing | 7 (7.4) | 9 (11) | 16 (9) |
| WBC at diagnosis of MPN-BP, no. (%) | |||
| ≤30 × 109/L | 64 (67.4) | 57 (69.5) | 121 (68.4) |
| 30-100 × 109/L | 18 (18.9) | 13 (15.9) | 31 (17.5) |
| >100 × 109/L | 5 (5.3) | 4 (4.9) | 9 (5.1) |
| Missing | 8 (8.4) | 8 (9.8) | 16 (9) |
| Cytogenetics, no. (%) | |||
| Favorable | 1 (1.1) | 0 | 1 (0.6) |
| Intermediate | 50 (52.6) | 46 (56.1) | 96 (54.2) |
| Poor-risk | 39 (41.1) | 28 (34.1) | 67 (37.9) |
| Not tested | 2 (2.1) | 3 (3.7) | 5 (2.8) |
| Missing | 3 (3.2) | 5 (6.1) | 8 (4.5) |
| Blast percentage in BM before HCT | |||
| Median (minimum-maximum) | 2 (0-50) | 2 (0-60) | 2 (0-60) |
| Blast % in PB before HCT | |||
| Median (minimum-maximum) | 0 (0-56) | 0 (0-96) | 0 (0-96) |
| Disease status at HCT, no. (%) | |||
| PB/BM blasts <5% | 54 (56.8) | 47 (57.3) | 101 (57.1) |
| Active leukemia* | 41 (43.2) | 35 (42.7) | 76 (42.9) |
| No. of mutations, no. (%) | |||
| 0 | 4 (4.2) | — | |
| 1 | 20 (21.1) | — | |
| 2 | 22 (23.2) | — | |
| 3 | 19 (20) | — | |
| 4 | 15 (15.8) | — | |
| 5 | 7 (7.4) | — | |
| 6 | 5 (5.3) | — | |
| 7 | 1 (1.1) | — | |
| 8 | 2 (2.1) | — | |
| Mutations, no. (%) | |||
| ASXL1 | 21 (22.1) | — | |
| BCOR | 2 (2.1) | — | |
| CALR | 12 (12.6) | — | |
| CBL | 6 (6.3) | — | |
| CEBPA | 1 (1.1) | — | |
| CUX1 | 5 (5.3) | — | |
| DNMT3A | 13 (13.7) | — | |
| EZH2 | 3 (3.2) | — | |
| GATA2 | 2 (2.1) | — | |
| GNAS | 2 (2.1) | — | |
| IDH1 | 2 (2.1) | — | |
| IDH2 | 6 (6.3) | — | |
| JAK2 | 52 (54.7) | — | |
| KRAS | 1 (1.1) | — | |
| MPL | 1 (1.1) | — | |
| NRAS | 4 (4.2) | — | |
| PHF6 | 8 (8.4) | — | |
| PPM1D | 3 (3.2) | — | |
| RAD21 | 1 (1.1) | — | |
| RUNX1 | 13 (13.7) | — | |
| SETBP1 | 4 (4.2) | — | |
| SF3B1 | 10 (10.5) | — | |
| SH2B3 | 4 (4.2) | — | |
| SRSF2 | 15 (15.8) | — | |
| STAG2 | 2 (2.1) | — | |
| TET2 | 18 (18.9) | — | |
| TP53 | 22 (23.2) | — | |
| U2AF1 | 6 (6.3) | — | |
| ZRSR2 | 1 (1.1) | — | |
| Therapy related | |||
| Induction therapy, no. (%) | |||
| 7 + 3 ± other | 49 (51.6) | 51 (62.2) | 100 (56.5) |
| Cytarabine based | 9 (9.5) | 9 (11) | 18 (10.2) |
| Hypomethylating | 6 (6.3) | 1 (1.2) | 7 (4) |
| Other | 2 (2.1) | 1 (1.2) | 3 (1.7) |
| No therapy given | 1 (1.1) | 10 (12.2) | 11 (6.2) |
| Missing | 28 (29.5) | 10 (12.2) | 38 (21.5) |
| Transplant related | |||
| Time from diagnosis of BP to HCT | |||
| Median, mo | 5.23 | 5.28 | 5.26 |
| Time frame, no. (%) | |||
| <6 mo | 55 (57.9) | 50 (61) | 105 (59.3) |
| 6-12 mo | 17 (17.9) | 21 (25.6) | 38 (21.5) |
| ≥12 mo | 23 (24.2) | 11 (13.4) | 34 (19.2) |
| Graft source, no. (%) | |||
| BM | 14 (14.7) | 8 (9.8) | 22 (12.4) |
| PB | 81 (85.3) | 74 (90.2) | 155 (87.6) |
| Donor type, no. (%) | |||
| HLA-identical sibling | 7 (7.4) | 47 (57.3) | 54 (30.5) |
| Other related | 3 (3.2) | 10 (12.2) | 13 (7.3) |
| Well-matched URD | 66 (69.5) | 17 (20.7) | 83 (46.9) |
| Partially matched/mismatched URD | 19 (20) | 8 (9.8) | 27 (15.3) |
| D–R sex match, no. (%) | |||
| M-M | 47 (49.5) | 29 (35.4) | 76 (42.9) |
| M-F | 19 (20) | 17 (20.7) | 36 (20.3) |
| F-M | 16 (16.8) | 23 (28) | 39 (22) |
| F-F | 12 (12.6) | 13 (15.9) | 25 (14.1) |
| Missing | 1 (1.1) | 0 | 1 (0.6) |
| D-R CMV status, no. (%) | |||
| +/+ | 18 (18.9) | 31 (37.8) | 49 (27.7) |
| +/− | 9 (9.5) | 4 (4.9) | 13 (7.3) |
| −/+ | 37 (38.9) | 21 (25.6) | 58 (32.8) |
| −/− | 29 (30.5) | 24 (29.3) | 53 (29.9) |
| Missing | 2 (2.1) | 2 (2.4) | 4 (2.3) |
| Conditioning regimen, no. (%) | |||
| MAC, TBI-based | 8 (8.4) | 13 (15.9) | 21 (11.9) |
| MAC, chemotherapy-based | 46 (48.4) | 26 (31.7) | 72 (40.7) |
| RIC/NST | 41 (43.2) | 43 (52.4) | 84 (47.5) |
| GVHD prophylaxis, no. (%) | |||
| CNI + MMF ± others | 26 (27.4) | 21 (25.6) | 47 (26.6) |
| CNI + MTX ± others | 57 (60) | 42 (51.2) | 99 (55.9) |
| CNI ± others | 8 (8.4) | 11 (13.4) | 19 (10.7) |
| Post-CY | 3 (3.2) | 7 (8.5) | 10 (5.6) |
| Other | 1 (1.1) | 0 | 1 (0.6) |
| Missing | 0 | 1 (1.2) | 1 (0.6) |
| In vivo T-cell depletion, no. (%) | |||
| No | 69 (72.6) | 62 (75.6) | 131 (74) |
| Yes | 26 (27.4) | 20 (24.4) | 46 (26) |
| Planned G-CSF/GM-CSF within 7 d post-HCT, no. (%) | |||
| No | 72 (75.8) | 57 (69.5) | 129 (72.9) |
| Yes | 21 (22.1) | 25 (30.5) | 46 (26) |
| Missing | 2 (2.1) | 0 | 2 (1.1) |
| Year of transplant, no. (%) | |||
| 2001-2005 | 13 (13.7) | 26 (31.7) | 39 (22) |
| 2006-2010 | 42 (44.2) | 30 (36.6) | 72 (40.7) |
| 2011-2015 | 40 (42.1) | 26 (31.7) | 66 (37.3) |
| Follow-up of survivors, median (minimum-maximum), mo | 70.63 (13.55-143.16) | 68.82 (22.2-169.38) | 70.63 (13.55-169.38) |
CMV, cytomegalovirus; CNI, calcineurin inhibitor; D–R, donor–recipient; G-CSF, granulocyte colony stimulating factor; GM-CSF, granulocyte-macrophage colony stimulating factor; GVHD, graft-versus-host disease; HCT-CI, HCT comorbidity index; HLA, human leukocyte antigen; D-R, donor-recipient; F, female; M, male; MAC, myeloablative conditioning; MMF, mycophenolate mofetil; MTX, methotrexate; post-CY, posttransplant cyclophosphamide; RIC/NST, reduced-intensity/non-myeloablative conditioning; TBI, total body irradiation; URD, unrelated donor; WBC, white blood cell.
Active leukemia, blasts in PB and/or BM ≥5%.