Characteristics and outcome of study patients (N=18).
| Pt. No. | Sex | Age (yr) | FAB | Cytogenetics at diagnosis | Combined molecularabnormalitya) | Induction | CRd) | HSCT donor type (HLA) | Dx to HSCT |
CR to HSCT(M) | Conditio-ning regimen | Engraft-ment (D) | HSCT Compli-cation | OS (entry to death, M) | Relapse (RFS, M)e) | HSCT to last follow up (M) | Outco-me |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | F | 19 | M4 | 46, XX | BH-ACb) | Yes | MUD(10/10) | 7 | 5 | TBIAcFluda | 16 | - | 16 | Yes (4) | 9 | DOD | |
| 2 | M | 11 | M2 | 46,XY,t(5;21;8) (q13;q22;q22)/46,XY | AML1/ETO | BH-AC | Yes | MUD(9/10) | 4 | 3 | TBIAcFluda | 14 | skin aGVHD |
21 | No | 16 | TRM |
| 3 | F | 9 | M2 | 49,XX,+8,+11,+18/46,XX | BH-AC | Yes | MUD(10/10) | 7 | 6 | TBIAcFluda | 14 | VOD, liver cGVHD | 19 | No | 11 | TRM | |
| 4 | F | 16 | M4 | 46, XX | BH-AC | Yes | MUD(10/10) | 7 | 6 | TBIAcFluda | 19 | lung cGVHD | 157 | No | 150 | Alive | |
| 5 | F | 14 | M4 | 46,XX,t(6;9)(p23;q34)46,XX,inv(1)(p13q21),t(6;9)(p23;q34) | DEK/NUP214 | BH-AC | Yes | MUD(9/10) | 5 | 4 | TBIAcFluda | 19 | - | 130 | No | 124 | Alive |
| 6 | F | 7 | M5 | 46, XX | BH-AC | Yes | MUD(9/10) | 3 | 2 | BuFluVPATG | 17 | lung cGVHD | 108 | No | 104 | Alive | |
| 7 | M | 9 | M1 | 46, XY | BH-AC | Yes | MRD | 6 | 4 | TBIAcFluda | 12 | - | 13 | Yes (4) | 6 | DOD | |
| 8 | M | 12 | M1 | 46, XY | BH-AC | Yes | MRD | 4 | 3 | BuFluVPATG | 11 | VOD | 106 | No | 102 | Alive | |
| 9 | F | 10 | M1 | 46,XX,t(7;11) (p15;p15) | BH-AC | Yes | MRD | 4 | 3 | BuFluVPATG | 11 | - | 36 | Yes (28) | 32 | TRM | |
| 10 | F | 15 | M2 | 46,XX,t(8;21) (q22;q22) | AML1/ETO | BH-AC | Yes | MUD(10/10) | 5 | 4 | BuFluVPATG | 11 | - | 106 | No | 100 | Alive |
| 11 | M | 18 | M1 | 46,XY,t(6;9)(p23;q34)/46,XY | DEK/NUP214 | BH-AC | Yes | MRD | 5 | 4 | BuFluVPATG | 10 | VOD, liver cGVHD | 7 | No | 2 | TRM |
| 12 | F | 11 | M1 | 46 XY | BH-AC | Yes | MRD | 4 | 2 | BuFluVPATG | 10 | VOD | 32 | Yes (12) | 27 | DOD | |
| 13 | M | 6 | M4 | 46, XY | FLT3/TKD | BH-AC | Yes | MUD(10/10) | 4 | 3 | BuFluVPATG | 11 | VOD, |
101 | No | 96 | Alive |
| 14 | F | 13 | M1 | 46,XX,16qh+ | BH-AC | Yes | MUD(10/10) | 4 | 1 | BuFluVPATG | 13 | skin aGVHD | 50 | No | 46 | Alive | |
| 15 | M | 16 | M2 | 45,X,-Y,t(8;21)(q22;q22) | AML1/ETO | Cytarabinec) | Yes | Haplo-identical | 7 | 5 | BuFluCy | 20 | - | 73 | No | 66 | Alive |
| 16 | F | 13 | M4 | 46,XX,t(11;19) (q23;p13.3) | MLL | Cytarabine | Yes | Haplo-identical | 5 | 3 | BuFluCy | 18 | - | 70 | No | 65 | Alive |
| 17 | M | 16 | M4 | 46, XY | Cytarabine | No | (-) | - | - | (-) | - | - | 6 | No | - | DOD | |
| 18 | F | 14 | M1 | 46, XX | NPM1 | Cytarabine | Yes | Haplo-identical | 4 | 3 | BuFluCy | 16 | - | 13 | No | 9 | Alive |
a)All patients were tested for
Abbreviations: aGVHD, acute graft versus host disease; BuFluCy, busulfan, fludarabine, cyclophosphamide; BuFluVPATG, Busulfan, etoposide, thymoglobulin, fludarabine; cGVHD, chronic graft versus host disease; CR, complete remission; DOD, died of disease; Dx, diagnosis; FAB, French–American–British classification; HSCT, hematopoietic stem cell transplant; MRD, matched related donor; MUD, matched unrelated donor; TBIAcFluda, TBI, AraC, thymoglobulin, fludarabine; TRM, treatment related mortality; VOD, veno-occlusive disease.