Baseline patient characteristics
| Variable . | Training cohort . | Validation cohort . | ||
|---|---|---|---|---|
| Total no. (%)* . | Myeloablative no. (%)* . | RIC no. (%)* . | Total no. (%)* . | |
| No. patients | 1539 | 812 | 727 | 672 |
| Median age, y (range) | 49 (18-73) | 43 (18-65) | 56 (18-73) | 42 (18-67) |
| Sex | ||||
| Male | 893 (58) | 442 (46) | 451 (62) | 362 (54) |
| Female | 646 (42) | 370 (54) | 276 (38) | 310 (46) |
| Disease | ||||
| ALL | 150 (10) | 132 (16) | 18 (2) | 93 (14) |
| Intermediate cytogenetics† | 65 (43)‡ | 56 (42)‡ | 9 (50)‡ | N/A |
| Adverse cytogenetics† | 78 (52)‡ | 69 (52)‡ | 9 (50)‡ | N/A |
| Cytogenetics not available | 7 (5)‡ | 7 (5)‡ | 0 (0)‡ | N/A |
| AML | 563 (37) | 349 (43) | 214 (29) | 299 (44) |
| Favorable cytogenetics§ | 27 (5)‡ | 28 (8)‡ | 12 (6)‡ | 34 (11) |
| Intermediate cytogenetics§ | 418 (74)‡ | 236 (68)‡ | 152 (71)‡ | 177 (59) |
| Adverse cytogenetics§ | 90 (16)‡ | 61 (17)‡ | 42 (20)‡ | 88 (29) |
| Cytogenetics not available | 28 (5)‡ | 24 (7)‡ | 8 (4)‡ | 0 (0) |
| CLL | 128 (8)‡ | 23 (3) | 105 (14) | 3 (0) |
| Intermediate cytogenetics‖ | 67 (52)‡ | 8 (35)‡ | 59 (56)‡ | 3 (100) |
| Adverse cytogenetics‖ | 36 (28)‡ | 8 (35)‡ | 28 (27)‡ | 0 (0) |
| Cytogenetics not available | 25 (20)‡ | 7 (30)‡ | 18 (17)‡ | 0 (0) |
| CML | 138 (9) | 109 (13) | 29 (4) | 160 (24) |
| Hodgkin lymphoma | 69 (4) | 10 (1) | 59 (8) | 3 (0) |
| MDS | 193 (13) | 87 (11) | 106 (15) | 91 (14) |
| Intermediate cytogenetics¶ | 102 (53)‡ | 53 (61)‡ | 49 (46)‡ | 44 (48) |
| Adverse cytogenetics¶ | 76 (39)‡ | 27 (31)‡ | 49 (46)‡ | 30 (33) |
| Cytogenetics not available | 15 (8)‡ | 7 (8)‡ | 8 (8)‡ | 17 (19) |
| Multiple myeloma | 46 (3) | 6 (1) | 40 (6) | 2 (0) |
| Myeloproliferative Neoplasms | 29 (2) | 12 (1) | 17 (2) | 0 (0) |
| Non-Hodgkin lymphoma | 223 (14) | 84 (10) | 139 (19) | 21 (3) |
| Indolent B-NHL# | 72 (5) | 22 (3) | 50 (7) | 5 (1) |
| Aggressive B-NHL# | 61 (4) | 25 (3) | 36 (5) | 14 (2) |
| Mantle cell lymphoma | 43 (3) | 13 (2) | 30 (4) | 2 (0) |
| T-cell lymphoma, nodal** | 25 (2) | 10 (1) | 15 (2) | 0 (0) |
| T-cell lymphoma, extranodal** | 22 (1) | 14 (2) | 8 (1) | 0 (0) |
| Stage at SCT | ||||
| CR1/CP | 450 (29) | 291 (36) | 159 (22) | 303 (45) |
| CR > 1 | 223 (14) | 120 (15) | 103 (14) | 83 (12) |
| PR1/AP | 103 (7) | 54 (7) | 49 (7) | 15 (2) |
| PR > 1 | 203 (13) | 48 (6) | 155 (21) | 0 (0) |
| Induction Failure | 178 (12) | 113 (14) | 65 (9) | 0 (0) |
| Relapse/BC | 194 (13) | 102 (13) | 92 (13) | 263 (39) |
| Untreated | 188 (12) | 84 (10) | 104 (14) | 8 (1) |
| Treatment on protocol | N/A | |||
| Yes | 606 (39) | 278 (34) | 328 (45) | |
| No | 933 (61) | 534 (66) | 399 (55) | |
| Donor match | ||||
| MRD | 623 (40) | 367 (45) | 256 (35) | 360 (54) |
| Non-MRD | 916 (60) | 445 (55) | 471 (65) | 312 (46) |
| MUD | 692 (45) | 333 (41) | 359 (49) | 312 (46) |
| MM | 224 (15) | 112 (14) | 112 (15) | 0 (0) |
| Mismatched URD | 201 (13) | 93 (11) | 108 (15) | 0 (0) |
| Mismatched relative | 23 (1) | 19 (2) | 4 (1) | 0 (0) |
| Graft source | ||||
| PB | 1228 (80) | 592 (73) | 636 (87) | 529 (79) |
| BM | 219 (14) | 192 (24) | 27 (4) | 143 (21) |
| PB + BM | 4 (0) | 3 (0) | 1 (0) | 0 (0) |
| UCB | 88 (6) | 25 (3) | 63 (9) | 0 (0) |
| Conditioning | ||||
| Myeloablative | 812 (53) | 621 (92) | ||
| Nonmyeloablative/RIC | 727 (47) | 51 (8) | ||
| GVHD prophylaxis | ||||
| CnI + Mtx | 631 (41) | 407 (50) | 224 (31) | 600 (89) |
| CnI + Siro ± Mtx | 773 (50) | 344 (42) | 429 (59) | 15 (2) |
| CnI + MMF | 68 (4) | 10 (1) | 58 (8) | 52 (8) |
| TCD | 36 (2) | 36 (4) | 0 (0) | 0 (0) |
| Other | 31 (2) | 15 (2) | 16 (2) | 5 (1) |
| CMV serostatus†† | ||||
| Recipient or donor + | 948 (62) | 473 (58) | 475 (65) | 350 (52) |
| Sex matching‡‡ | ||||
| Female to male | 361 (23) | 172 (21) | 189 (26) | 160 (24) |
| Male to female | 333 (22) | 183 (23) | 150 (21) | 165 (25) |
| Female to female | 311 (20) | 185 (23) | 126 (17) | 145 (22) |
| Male to male | 532 (35) | 270 (33) | 262 (36) | 202 (30) |
| Year of HSCT | 2005 | 2005 | 2005 | 2003 |
| (median, range) | (2000-2009) | (2000-2009) | (2000-2009) | (2000-2006) |
| Months of follow-up for survivors | 35 | 40 | 26 | 83 |
| (median, range) | (3-117) | (4-117) | (3-113) | (13-135) |
| Variable . | Training cohort . | Validation cohort . | ||
|---|---|---|---|---|
| Total no. (%)* . | Myeloablative no. (%)* . | RIC no. (%)* . | Total no. (%)* . | |
| No. patients | 1539 | 812 | 727 | 672 |
| Median age, y (range) | 49 (18-73) | 43 (18-65) | 56 (18-73) | 42 (18-67) |
| Sex | ||||
| Male | 893 (58) | 442 (46) | 451 (62) | 362 (54) |
| Female | 646 (42) | 370 (54) | 276 (38) | 310 (46) |
| Disease | ||||
| ALL | 150 (10) | 132 (16) | 18 (2) | 93 (14) |
| Intermediate cytogenetics† | 65 (43)‡ | 56 (42)‡ | 9 (50)‡ | N/A |
| Adverse cytogenetics† | 78 (52)‡ | 69 (52)‡ | 9 (50)‡ | N/A |
| Cytogenetics not available | 7 (5)‡ | 7 (5)‡ | 0 (0)‡ | N/A |
| AML | 563 (37) | 349 (43) | 214 (29) | 299 (44) |
| Favorable cytogenetics§ | 27 (5)‡ | 28 (8)‡ | 12 (6)‡ | 34 (11) |
| Intermediate cytogenetics§ | 418 (74)‡ | 236 (68)‡ | 152 (71)‡ | 177 (59) |
| Adverse cytogenetics§ | 90 (16)‡ | 61 (17)‡ | 42 (20)‡ | 88 (29) |
| Cytogenetics not available | 28 (5)‡ | 24 (7)‡ | 8 (4)‡ | 0 (0) |
| CLL | 128 (8)‡ | 23 (3) | 105 (14) | 3 (0) |
| Intermediate cytogenetics‖ | 67 (52)‡ | 8 (35)‡ | 59 (56)‡ | 3 (100) |
| Adverse cytogenetics‖ | 36 (28)‡ | 8 (35)‡ | 28 (27)‡ | 0 (0) |
| Cytogenetics not available | 25 (20)‡ | 7 (30)‡ | 18 (17)‡ | 0 (0) |
| CML | 138 (9) | 109 (13) | 29 (4) | 160 (24) |
| Hodgkin lymphoma | 69 (4) | 10 (1) | 59 (8) | 3 (0) |
| MDS | 193 (13) | 87 (11) | 106 (15) | 91 (14) |
| Intermediate cytogenetics¶ | 102 (53)‡ | 53 (61)‡ | 49 (46)‡ | 44 (48) |
| Adverse cytogenetics¶ | 76 (39)‡ | 27 (31)‡ | 49 (46)‡ | 30 (33) |
| Cytogenetics not available | 15 (8)‡ | 7 (8)‡ | 8 (8)‡ | 17 (19) |
| Multiple myeloma | 46 (3) | 6 (1) | 40 (6) | 2 (0) |
| Myeloproliferative Neoplasms | 29 (2) | 12 (1) | 17 (2) | 0 (0) |
| Non-Hodgkin lymphoma | 223 (14) | 84 (10) | 139 (19) | 21 (3) |
| Indolent B-NHL# | 72 (5) | 22 (3) | 50 (7) | 5 (1) |
| Aggressive B-NHL# | 61 (4) | 25 (3) | 36 (5) | 14 (2) |
| Mantle cell lymphoma | 43 (3) | 13 (2) | 30 (4) | 2 (0) |
| T-cell lymphoma, nodal** | 25 (2) | 10 (1) | 15 (2) | 0 (0) |
| T-cell lymphoma, extranodal** | 22 (1) | 14 (2) | 8 (1) | 0 (0) |
| Stage at SCT | ||||
| CR1/CP | 450 (29) | 291 (36) | 159 (22) | 303 (45) |
| CR > 1 | 223 (14) | 120 (15) | 103 (14) | 83 (12) |
| PR1/AP | 103 (7) | 54 (7) | 49 (7) | 15 (2) |
| PR > 1 | 203 (13) | 48 (6) | 155 (21) | 0 (0) |
| Induction Failure | 178 (12) | 113 (14) | 65 (9) | 0 (0) |
| Relapse/BC | 194 (13) | 102 (13) | 92 (13) | 263 (39) |
| Untreated | 188 (12) | 84 (10) | 104 (14) | 8 (1) |
| Treatment on protocol | N/A | |||
| Yes | 606 (39) | 278 (34) | 328 (45) | |
| No | 933 (61) | 534 (66) | 399 (55) | |
| Donor match | ||||
| MRD | 623 (40) | 367 (45) | 256 (35) | 360 (54) |
| Non-MRD | 916 (60) | 445 (55) | 471 (65) | 312 (46) |
| MUD | 692 (45) | 333 (41) | 359 (49) | 312 (46) |
| MM | 224 (15) | 112 (14) | 112 (15) | 0 (0) |
| Mismatched URD | 201 (13) | 93 (11) | 108 (15) | 0 (0) |
| Mismatched relative | 23 (1) | 19 (2) | 4 (1) | 0 (0) |
| Graft source | ||||
| PB | 1228 (80) | 592 (73) | 636 (87) | 529 (79) |
| BM | 219 (14) | 192 (24) | 27 (4) | 143 (21) |
| PB + BM | 4 (0) | 3 (0) | 1 (0) | 0 (0) |
| UCB | 88 (6) | 25 (3) | 63 (9) | 0 (0) |
| Conditioning | ||||
| Myeloablative | 812 (53) | 621 (92) | ||
| Nonmyeloablative/RIC | 727 (47) | 51 (8) | ||
| GVHD prophylaxis | ||||
| CnI + Mtx | 631 (41) | 407 (50) | 224 (31) | 600 (89) |
| CnI + Siro ± Mtx | 773 (50) | 344 (42) | 429 (59) | 15 (2) |
| CnI + MMF | 68 (4) | 10 (1) | 58 (8) | 52 (8) |
| TCD | 36 (2) | 36 (4) | 0 (0) | 0 (0) |
| Other | 31 (2) | 15 (2) | 16 (2) | 5 (1) |
| CMV serostatus†† | ||||
| Recipient or donor + | 948 (62) | 473 (58) | 475 (65) | 350 (52) |
| Sex matching‡‡ | ||||
| Female to male | 361 (23) | 172 (21) | 189 (26) | 160 (24) |
| Male to female | 333 (22) | 183 (23) | 150 (21) | 165 (25) |
| Female to female | 311 (20) | 185 (23) | 126 (17) | 145 (22) |
| Male to male | 532 (35) | 270 (33) | 262 (36) | 202 (30) |
| Year of HSCT | 2005 | 2005 | 2005 | 2003 |
| (median, range) | (2000-2009) | (2000-2009) | (2000-2009) | (2000-2006) |
| Months of follow-up for survivors | 35 | 40 | 26 | 83 |
| (median, range) | (3-117) | (4-117) | (3-113) | (13-135) |
ALL indicates acute lymphoblastic leukemia; AML, acute myeloid leukemia; AP, accelerated phase; BC, blast crisis; CLL, chronic lymphocytic leukemia; CML, chronic myelogenous leukemia; CnI, calcineurin inhibitor; CP, chronic phase; CR, complete remission; MDS, myelodysplastic syndrome; MM, mismatched donor; MRD, matched related donor; MTX, methotrexate; MUD, matched unrelated donor; NHL, non-Hodgkin lymphoma; PB, peripheral blood; PR, partial remission; RIC, reduced-intensity conditioning; Siro, sirolimus; TCD, T-cell depletion; UCB, umbilical cord blood; and N/A, not available.
Percentages may not add to 100 because of rounding.
Adverse is complex, t(4;11) or t(9;22).
Percentage of the total number of patients with this disease.
Classified according to Armand et al11 in the training cohort and according to Slovak et al16 in the validation cohort (see text).
Adverse is del17p or del11q.
Classified according to Armand et al.12
Indolent B-NHL includes follicular lymphoma, marginal zone lymphoma, and lymphoplasmacytic lymphoma. Small lymphocytic lymphoma is included in CLL group. Aggressive B-NHL includes diffuse large B-cell lymphoma and transformed indolent B-cell lymphoma.
Nodal T-cell lymphomas include peripheral T-cell lymphoma, not otherwise specified; angioimmunoblastic T-cell lymphoma; and anaplastic T cell lymphoma. Extranodal T-cell lymphomas include hepatosplenic T-cell lymphoma, extranodal NK/T-cell lymphoma, enteropathy-associated T-cell lymphoma, cutaneous T-cell lymphoma/mycosis fungoides, and adult T-cell leukemia/lymphoma.
Data not available for 27 patients.
Data not available for 2 patients.