Patients’ characteristics
| Parameters . | Value (%) or range (min, max) . |
|---|---|
| Age, median (min-max) | 56 (19-84) |
| Sex male, n (%) | 20 (49) |
| Disease type, n (%) | |
| R/R Ph+ BCP-ALL | 33 (80.5) |
| LBC-CML | 8 (19.5) |
| No. of previous TKIs, median (min-max) | 3 (2-5) |
| Previous use of ponatinib, n (%) | 38 (92.7) |
| Prior CAR T-cell therapy, n (%) | 2 (4.8) |
| Prior allo-HSCT, n (%) | 18 (43.9) |
| Previous line of therapy, n (%) | 41 |
| First line (LBC-CML) | 2 (4.9) |
| Second line of treatment | 7 (17.1) |
| Third line of treatment or more | 32 (78) |
| Disease status, n (%) | |
| Hematological relapse | 24 (58.5) |
| Refractory | 5 (12.2) |
| CNS-only relapse | 1 (2.4) |
| Molecular relapse | 7 (17.1) |
| Complete remission (intolerance) | 4 (9.8) |
| CNS involvement at time of ASC initiation, n (%) | 8 (19) |
| ABL mutations analyzed before ASC treatment, n (%) | 35 (85.4) |
| Absence of mutations, n (%) | 8 (22.9) |
| Presence of mutations, n (%) | 27 (77.1) |
| T315I | 14 (51.9) |
| E255V/K | 2 (7.4) |
| T315I + E255V | 3 (11.1) |
| T315I + E255K + M244T | 1 (3.7) |
| T315I + V299L | 1 (3.7) |
| E255K + G250E + Y253H | 1 (3.7) |
| F311L | 1 (3.7) |
| F317L | 1 (3.7) |
| Y253H | 1 (3.7) |
| E255K + E255V | 1 (3.7) |
| T315A + V299L | 1 (3.7) |
| ASC dose, n (%) | |
| High dose (200 mg twice daily) | 34 (82.9) |
| Low dose (40 mg twice daily) | 7 (17.1) |
| Associated treatment, n (%) | 41 |
| ASC monotherapy, including 2 patients with ITT, n (%) | 20 (48.8) |
| ASC in combination, n (%) | 21 (51.2) |
| High dose chemotherapy | 2 (4.9) |
| Low dose chemotherapy | 8 (19.5) |
| Immunotherapy (blinatumomab or InO) | 6 (14.6) |
| Other TKI | 3 (7.3) |
| DLI | 1 (2.4) |
| CAR T cell | 1 (2.4) |
| Hematological response rate (efficacy population), n (%) | 36 |
| CR | 28 (77.8) |
| CRi | 2 (5.6) |
| Failure | 6 (16.7) |
| MRD response in CR + CRi patients with evaluable bone marrow samples, n (%) | 23 |
| No CMR | 10 (43.5) |
| CMR (BCR ABL <0.01% in bone marrow) | 13 (56.5) |
| Post ASC treatment, n (%) | 37 |
| No HSCT or CAR T cells | 27 (73) |
| Allo-HSCT | 3 (8) |
| CAR T cell | 5 (13.5) |
| Allo-HSCT + CAR T cell | 2 (5.4) |
| Parameters . | Value (%) or range (min, max) . |
|---|---|
| Age, median (min-max) | 56 (19-84) |
| Sex male, n (%) | 20 (49) |
| Disease type, n (%) | |
| R/R Ph+ BCP-ALL | 33 (80.5) |
| LBC-CML | 8 (19.5) |
| No. of previous TKIs, median (min-max) | 3 (2-5) |
| Previous use of ponatinib, n (%) | 38 (92.7) |
| Prior CAR T-cell therapy, n (%) | 2 (4.8) |
| Prior allo-HSCT, n (%) | 18 (43.9) |
| Previous line of therapy, n (%) | 41 |
| First line (LBC-CML) | 2 (4.9) |
| Second line of treatment | 7 (17.1) |
| Third line of treatment or more | 32 (78) |
| Disease status, n (%) | |
| Hematological relapse | 24 (58.5) |
| Refractory | 5 (12.2) |
| CNS-only relapse | 1 (2.4) |
| Molecular relapse | 7 (17.1) |
| Complete remission (intolerance) | 4 (9.8) |
| CNS involvement at time of ASC initiation, n (%) | 8 (19) |
| ABL mutations analyzed before ASC treatment, n (%) | 35 (85.4) |
| Absence of mutations, n (%) | 8 (22.9) |
| Presence of mutations, n (%) | 27 (77.1) |
| T315I | 14 (51.9) |
| E255V/K | 2 (7.4) |
| T315I + E255V | 3 (11.1) |
| T315I + E255K + M244T | 1 (3.7) |
| T315I + V299L | 1 (3.7) |
| E255K + G250E + Y253H | 1 (3.7) |
| F311L | 1 (3.7) |
| F317L | 1 (3.7) |
| Y253H | 1 (3.7) |
| E255K + E255V | 1 (3.7) |
| T315A + V299L | 1 (3.7) |
| ASC dose, n (%) | |
| High dose (200 mg twice daily) | 34 (82.9) |
| Low dose (40 mg twice daily) | 7 (17.1) |
| Associated treatment, n (%) | 41 |
| ASC monotherapy, including 2 patients with ITT, n (%) | 20 (48.8) |
| ASC in combination, n (%) | 21 (51.2) |
| High dose chemotherapy | 2 (4.9) |
| Low dose chemotherapy | 8 (19.5) |
| Immunotherapy (blinatumomab or InO) | 6 (14.6) |
| Other TKI | 3 (7.3) |
| DLI | 1 (2.4) |
| CAR T cell | 1 (2.4) |
| Hematological response rate (efficacy population), n (%) | 36 |
| CR | 28 (77.8) |
| CRi | 2 (5.6) |
| Failure | 6 (16.7) |
| MRD response in CR + CRi patients with evaluable bone marrow samples, n (%) | 23 |
| No CMR | 10 (43.5) |
| CMR (BCR ABL <0.01% in bone marrow) | 13 (56.5) |
| Post ASC treatment, n (%) | 37 |
| No HSCT or CAR T cells | 27 (73) |
| Allo-HSCT | 3 (8) |
| CAR T cell | 5 (13.5) |
| Allo-HSCT + CAR T cell | 2 (5.4) |
CMR, complete molecular response; DLI, donor lymphocyte infusion; InO, inotuzumab ozogamicin; ITT, intrathecal therapy; min, minimum; max, maximum.