Table 1.
Baseline characteristics
|
Median age, years (range) |
73 (33–91) |
|
WHO diagnosis*, n (%) |
|
| t-AML |
16 (10.3) |
| AML-RCA |
16 (10.3) |
| AML-MRF only |
88 (56.8) |
| AML-MRC |
13 (8.4) |
| Preexisting MDS/MPN or MLD |
58 (37.4) |
| Preexisting MDS/MPN or MLD + MRC |
17 (11.0) |
| AML-NOS |
35 (22.6) |
|
Peripheral blood blasts, n (%) |
|
| 0% |
58 (37.4) |
| > 0% |
97 (62.6) |
| Mean,% |
14 |
| Median (range),% |
3 (0–90) |
|
Bone marrow blasts, n (%) |
|
| <20%† |
26 (16.8) |
| 20–30% |
31 (20.0) |
| >30% (off label use) |
98 (63.2) |
| Mean,% |
42 |
| Median (range),% |
35 (0–98) |
|
White blood cell count, n (%) |
|
| Non-MP-AML (< 10 G/l) |
122 (78.7) |
| MP-AML (> 10 G/l) |
33 (21.3) |
|
Transfusion dependence (TD), n (%) |
|
| Any type of TD |
101 (65.2) |
| RBC-TD |
97 (62.6) |
| PLT-TD |
60 (38.7) |
| RBC-TD + PLT-TD |
56 (36.1) |
|
IPSS cytogenetic risk‡, n (%) |
|
| Not evaluable |
11 (7.1) |
| Good |
91 (58.7) |
| Intermediate |
26 (16.1) |
| Poor |
27 (17.4) |
|
MRC cytogenetic risk‡, n (%) |
|
| Not evaluable |
11 (7.1) |
| Good |
3 (1.9) |
| Intermediate |
115 (74.2) |
| High |
26 (16.8) |
|
Comorbidities, n (%) |
|
| Thromboembolic episodes |
21 (13.5) |
| Renal insufficiency |
29 (18.7) |
| Liver disease |
20 (12.9) |
| Diabetes mellitus |
26 (16.8) |
| Coronary artery disease |
34 (21.9) |
| COPD |
15 (9.7) |
| Prior/concomitant malignancies |
35 (22.6) |
|
Number of comorbidities, n (%) |
|
| 0–1 |
66 (42.6) |
| 2–3 |
61 (39.4) |
| > 3 |
28 (18.1) |
|
ECOG Prognostic Score, n (%) |
|
| ECOG <2 |
114 (73.6) |
| ECOG ≥2 |
41 (26.4) |
|
HCT-CI, n (%) |
|
| Low risk |
46 (29.7) |
| Int. risk |
46 (29.7) |
| High risk |
50 (32.2) |
| No data |
13 (8.4) |
|
Treatment prior to azacitidine§, n (%) |
|
| None |
63 (40.6) |
| Erythropoietin stimulating agents |
15 (9.7) |
| G-CSF |
19 (12.3) |
| Thrombopoietin stimulating agents |
1 (0.7) |
| Iron chelation therapy |
5 (3.2) |
| Thalidomide |
3 (1.9) |
| Lenalidomide |
6 (3.9) |
| Low-dose cytarabine |
10 (6.5) |
| Intensive chemotherapy |
60 (38.7) |
| Others |
16 (10.3) |
|
Reason for treatment, n (%) |
|
| 1st line treatment |
63 (40.6) |
| Bridging to allogeneic SCT# |
4 (2.6) |
| Maintenance after CR to conventional chemotherapy |
6 (3.9) |
| No CR to/early relapse after conventional chemotherapy |
45 (29.0) |
| No CR to/early relapse after allogeneic SCT |
5 (3.2) |
| No CR to other prior treatment | 32 (20.6) |
t-AML indicates treatment related AML; AML-RCA, AML with recurrent cytogenetic abnormalities; AML-MRF, AML with MDS related features; MPN, myeloproliferative neoplasia; MLD, multilineage dysplasia; MRC, MDS-related cytogenetics; AML-NOS, AML not otherwise specified; MP, myeloproliferative; COPD, chronic obstructive pulmonary disease; NHL, non-Hodgkin’s lymphoma; ECOG, Eastern Cooperative Oncology Group; CR, complete response; SCT, stem cell transplantation;
*If a patient fulfilled criteria for more than one WHO-category, weighting was performed as follows: t-AML > AML-RCA > AML-MRF.
†BM-blast count was <20%, in those patients with established AML who were refractory to, -or had no CR after-, conventional chemotherapy or allogeneic stem cell transplantation.
‡Pre-treatment cytogenetics were available in 92% of patients and were determined by conventional metaphase karyotyping (46%), interphase-FISH (16%), or both (38%).
§Numbers may add up to >100% as multiple selections were possible.
#None of the patients for whom azacitidine was intended as bridging was in complete remission to prior therapies, and all patients had relapsed after multiple lines of intensive chemotherapy; only one of these patients proceeded to allogeneic SCT (the others died whilst still on, or within 8 weeks after termination of azacitidine treatment due to disease progression and/or infectious complications).