TABLE 1.
Patient Characteristics
| Characteristic | No. of Patients (%)
|
|
|---|---|---|
| HAPLO Arm, n = 60 | 9/10 MUD Arm, n = 46 | |
| Median age [range], y | 45 [20–63] | 51 [20–64] |
| Recipient sex | ||
| Men | 29 (48) | 23 (50) |
| Women | 31 (52) | 23 (50) |
| Karnofsky performance status | ||
| ≥90 | 53 (88) | 40 (87) |
| <90 | 7 (12) | 6 (13) |
| Donor sex | ||
| Men | 35 (58) | 22 (48) |
| Women | 25 (42) | 24 (52) |
| Donor relationship to recipient | ||
| Unrelated | NA | 46 (100) |
| Parent | 8 (13) | NA |
| Child | 24 (40) | NA |
| Sibling | 27 (45) | NA |
| Cousin | 1 (2) | NA |
| HLA locus mismatch location | ||
| A | 0 (0) | 20 (44) |
| B | 0 (0) | 6 (13) |
| C | 0 (0) | 16 (35) |
| DRB1 | 0 (0) | 2 (4) |
| DQB1 | 0 (0) | 2 (4) |
| A/B/C/DRB1/DQB1 | 42 (70) | 0 (0) |
| B/C/DRB1/DQB1 | 7 (12) | 0 (0) |
| A/B/DRB1/DQB1 | 4 (7) | 0 (0) |
| A/B/C/DQB1 | 2 (3) | 0 (0) |
| A/B/C/DRB1 | 1 (2) | 0 (0) |
| A/DRB1/DQB1 | 2 (3) | 0 (0) |
| A/B/DRB1 | 1 (2) | 0 (0) |
| DRB1/DQB1 | 1 (2) | 0 (0) |
| Hematopoietic cell transplant-comorbidity index | ||
| 0–1 | 31 (52) | 22 (48) |
| 2–3 | 19 (32) | 16 (35) |
| >3 | 10 (17) | 8 (17) |
| Disease risk indexa | ||
| Very high | 5 (8) | 3 (7) |
| High | 18 (30) | 15 (33) |
| Intermediate | 29 (48) | 12 (26) |
| Low | 8 (13) | 12 (26) |
| NA | 0 (0) | 4 (9)b |
| No. of prior autologous hematopoietic cell transplantations | ||
| 0 | 53 (88) | 38 (83) |
| 1 | 6 (10) | 6 (13) |
| 2 | 1 (2) | 2 (4) |
| No. of prior chemotherapy cycles | ||
| 0–3 | 48 (80) | 36 (78) |
| >3 | 12 (20) | 10 (22) |
| Conditioning intensity | ||
| FM100 | 20 (33) | 18 (39) |
| FM140 | 40 (67) | 28 (61) |
| Graft source | ||
| Bone marrow | 58 (97) | 38 (83) |
| Peripheral blood | 2 (3) | 8 (17) |
| CMV status | ||
| Patient and donor negative | 6 (10) | 5 (11) |
| Patient or donor positive | 54 (90) | 41 (89) |
| Diagnosis | ||
| AML/MDS | 33 (55) | 18 (39) |
| Secondary AML/MDSc | 7 (12) | 7 (15) |
| ALL | 7 (12) | 5 (11) |
| Non-Hodgkin lymphoma | 8 (13) | 11 (24) |
| Hodgkin lymphoma | 2 (3) | 2 (4) |
| Myeloproliferative disease | 9 (15) | 4 (9) |
| Multiple myeloma | 1 (2) | 2 (4) |
| Aplastic anemia | 0 (0) | 4 (9) |
| Disease stage For acute leukemia | ||
| CR1/CR2 | 24 (67) | 9 (56) |
| ≥CR3/CRp | 6 (17) | 5 (31) |
| Active disease | 6 (17) | 2 (13) |
| For lymphoma | ||
| CR | 3 (30) | 8 (62) |
| PR | 5 (50) | 3 (23) |
| Chemoresistant | 2 (20) | 2 (15) |
| For multiple myeloma | ||
| PR | 1 (100) | 1 (50) |
| Very good PR | 0 (0) | 1 (50) |
Abbreviations: 9/10 MUD, HLA-mismatched unrelated donor; ALL, acute lymphoblastic leukemia; AMS/MDS, acute myeloid leukemia/myelodysplastic syndrome; CMV, cytomegalovirus; CR1, first complete remission; CR2, second complete remission; CR3, third complete remission; CRp, complete remission with incomplete count recovery; DQB1, DQ β1; DRB1, D-related β1; FM100, the same regimen as FM140 with a lower melphalan dose of 100 mg/m2; FM140, 1 intravenous dose of 140 mg/m2 melphalan on day −7, 1 intravenous dose of 5 mg/kg thiotepa on day −6, and 4 intravenous doses of 40 mg/m2 fludarabine (1 daily on days −5 through −2); HAPLO, haploidentical donor; HLA, human leukocyte antigen; NA, not applicable; PR, partial remission.
Disease risk was assessed using the disease risk index described by Armand et al, 2012.30
These patients had aplastic anemia.
Secondary AML/MDS was defined as disease secondary to a previous hematologic disorder or therapy related.