Table 3.
Patient characteristics and outcomes.
| Patients' N° | Level/dose of Tocilizumab | Gender | ECOG PS | Caryotype | Molecular assessment | ELN2017 classification | Response to treatment according to ELN 2017 classification | MRD | Cytogenetic remission | Treatment received after induction | Allo-HCST | Relapse | Cause of death | Status at last follow-up |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 01 | L1 : 4 mg/kg | M | 1 | Complex with del5q | Flt3 neg/idh1 and 2 neg/npm1 neg | Unfavourable | Primary refractory disease | NA | NA | Salvage with azacitidine = failed; 2nd attempt with low dose arac + venetoclax = CR | yes | Yes (before transplant) | Leukaemia | Dead |
| 02 | L1 : 4 mg/kg | F | 0 | trisomy 11 | Flt3 neg/idh1 neg/IDH2 POS/npm1 neg | Intermediate | CR | neg | not done | Consolidation with azacitidine (1 course) | yes | no | NA | Alive, in CR |
| 03 | L2 : 6 mg/kg | F | 0 | complex | npm1 neg/Flt3 neg | Unfavourable | CRi | neg | yes | Consolidation with Idarubicin (1 day) + cytarabine; 1 course | yes | no | Infection and MOF | Dead, in CR |
| 04 | L2 : 6 mg/kg | F | 0 | Normal | Flt3neg, NPM1neg, IDHneg, CEBP alpha neg; EZH2 pos | intermediate | CR | 0.16% | NA | Consolidation with Idarubicin (1 day) + cytarabine; 2 courses | Yes | No | NA | Alive, in CR |
| 05 | L3 : 8 mg/kg | M | 0 | Normal | RUNX1 pos and ASXL1 pos | unfavourable | CR | Negative | NA | Consolidation with Idarubicin (1 day) + cytarabine; 1 course | Yes | No | GVHD | Dead, in CR |
| 06 | L3 : 8 mg/kg | M | 0 | 2 anomalies including monosomy 7 | Mutations in JAK2, TET2, DNMT3A, MPL and NFE2 | Unfavourable | CRi | Not done | Yes | Consolidation with Idarubicin (1 day) + cytarabine; 1 course | Yes | No | NA | Alive, in CR |
| 07 | L3 : 8 mg/kg | M | 2 | del 5q, monosomy 7 and MECOM rearrangement | 2 mutations of TP53 + 1 mutation of CXCR4 | Unfavourable | CR | 0.6% | Yes | Consolidation with Idarubicin (1 day) + cytarabin; 1 course | No | Yes | Leukaemia | Dead |
| 08 | L3 : 8 mg/kg | F | 0 | Complex with del5q | del TP53 | Unfavourable | CRi | Not done | No | Salvage with azacitidine + venetoclax = failed; 2nd attempt with high dose of cytatabine = failed; sequential Allo-HSCT = CR | Yes | Yes (before transplant) | Engraftment syndrom | Dead, in CR |
| 09 | L3 : 8 mg/kg | M | 1 | normal | Mutation of: IDH2, ASXL1, SRSF2 et RUNX1 | Unfavourable | CRi | Negative | NA | Consolidation with azacitidine (1 course) | Yes | No | NA | Alive, in CR |
| 10 | L3 : 8 mg/kg | F | 1 | Normal | NPM1neg, Flt3neg, IDH1 pos, IDH 2 neg, DNMT3 pos | Intermediate | NA | NA | NA | NA | No | NA | Infection during induction | Dead |
| 11 | L3 : 8 mg/kg | F | 0 | Monosomy 7 | no mutations on a 22 gene NGS panel (including npm1 and Flt3) | Unfavourable | MLFS | Not done | Not done | Salvage with azacitidine + venetoclax = failed; Allo-HSCT with active disease | yes | Yes (before transplant) | NA | Alive, in CR |
| 12 | L3 : 8 mg/kg | F | 0 | Normal | Mutation of ASXL1 | Unfavourable | NA | NA | NA | NA | no | NA | Probable cerebral haemorrhage during induction | Dead |