Table 1. Individual patient characteristics and responses in 19 patients evaluable for response.
| Universal patient no. | Dose cohort | Disease (FAB/ WHO) | IPSS risk | Age, years | Cytogenetics additional to del(5q) | TP53 mutation (exon) | No. of induction (maintenance) cycles received | Best hematologic response | Best cytogenetic response |
|---|---|---|---|---|---|---|---|---|---|
| 1 | I | RAEB/ RAEB-2 | High | 69 | del(1)(p36p32),+8 | No | 2 (3) | CRi | PR |
| 2 | I | AML/ AML | — | 45 | t(4;17)(q12;q11.2), t(6;8)(p12;q24), −7, +der(7)t(7;13)(p10;q10), −12, +der(12)t(X;12)(q2?;q13), −13, −22 | No | 2 | SD | SD |
| 3 | I | RAEB/ RAEB-2 | High | 69 | −9, +der(9)t(1;9)(q21;q22), del(11)(q14q24) | NA | 2 | SD | PR |
| 4 | I | RAEB/ RAEB-2 | Int-2 | 73 | None | No | 5 | SD | SD |
| 5 | II | RAEB/ RAEB-2 | High | 57 | t(2;9)(p14;q22), −3, +der(3)t(17;3;18;3;18;4;2)(17?-> 17?::3?->3cen->::18?->18?::3?-> 3?::18?->18?::4p13?->4q31?::2?->2?), −4, +der(4)t(4;17)(4p11-> 4q21::17q12->17q23::?), −7, +der(7)t(7;19)(q34;?), iso(11)(q10), −17, +der(17)t(17;19)(p11.2;?), −18, +der(18)t(18;3)(18pter-> 18q11.2::3?), −19 | Yes (6) | 1 | SD | PR |
| 6 | II | AML/ AML | — | 66 | None | Yes (7, 8) | 3 | SD | SD |
| 7 | II | RAEB/ RAEB-2 | Int-2 | 49 | +der(3)t(3;6)(p10;q10), −6, −7, del(12)(p13p11.2), −17, −20, +der(20)t(17;20)(q10;p10) | Yes (6) | 5 | SD | PR |
| 8 | II | AML/ AML | — | 67 | +11, −17 | No | 2 | PD | PD |
| 9 | III | RAEB/ RAEB-2 | High | 75 | der(3)t(3;5)(p11;p13), der(11)t(11;12)(p15;q13), −12, der(17)t(17;20)(p11.2;p11.2), der(18;20)(p10;q13?), −20, +der(20)t(10;18;20)(18pter?-> 18p11.2?::20p11.2::10q24-> 10qter?) | Yes (5) | 3 | SD | SD |
| 10 | III | AML/ sAML | — | 68 | r(11)hrs(11)(q23) | Yes (6) | 4 | SD | SD |
| 11 | III | RAEB/ RAEB-1 | High | 72 | del(7), (q11.2?), −16, +der(16) t(16;21)(p10?;q10?)x2,−21, i(21)(q10), der(22) t(1;22)(p11;p11.1) | NA | 6 | SD | SD |
| 12 | III | AML/ AML | — | 55 | 40–42, X, −X, −3, der(4) t(X;4)(p?;q21), t(4;11)(q11;q21?), der(6)t(6;7;11)(qter->p?::?->?), del(6)(q?), t(7;11)(pter->q22?::?), −11, der(11)t(4;11)(qter->?::q21?-> pter), −14, −16, del(17)(q?), +20, der(20)t(11;20)(?::q13->pter), +22 [cp21] | Yes (5) | 2 | PR | PR |
| 13 | III | RAEB/ RAEB-1 | Int-2 | 60 | −7, −13, +der(13)(q14?q22?), der(17)t(17;22)(p13;p11.2?), −22, +2mar | Yes (5, 6, 7) | 3 (4) | CR | CR |
| 14 | III | RA/ RCMD | Int-2 | 80 | −7, der(17)t(5;17)(q13;p13) | Yes (8) | 1 | CRi | PR |
| 15 | IV | RAEB/ RAEB-2 | Int-2 | 60 | dic(6;18)(p23;p11.1)del(18)(q21), +8 | Yes (5) | 1 | CRi | CR |
| 16 | IV | RAEB/ RAEB-2 | High | 78 | −7, −16, +der(16) dic(16;17)(q13?;p12?), −17 | No | 1 | SD | SD |
| 17 | IV | RAEB/ RAEB-2 | High | 76 | t(1;6)(q42;p22), t(3;4)(p14;p16), −12, der(16), der(18)t(5;18)(q13;q21.3) | Yes (6) | 3 | SD | PD |
| 18 | IV | RAEB/ RAEB-1 | Int-2 | 71 | del(7q), +8, del20(q), +17p, del(18q), del(21q) | No | 3 | SD | SD |
| 19 | IV | AML/ AML | — | 63 | None | Yes (5) | 2 | SD | SD |
Abbreviations: AML, acute myeloid leukemia; CR, complete response; CRi, CR with incomplete recovery of peripheral blood counts; FAB, French-American-British; Int, Intermediate; IPSS, International Prognostic Scoring System; NA, not applicable; PD, progressive disease; PR, partial response; RA, refractory anemia; RAEB, RA with excess blasts; RCMD, refractory cytopenia with multilineage dysplasia; sAML, secondary AML; SD, stable disease; WHO, World Health Organization.