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Allogeneic hematopoietic stem cell transplant (HSCT) plays a pivotal role in the treatment of fit patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), however disease relapse after transplant occurs in around 40% of cases.1 Whilst the hypomethylating agent azacitidine is the standard first-line treatment option for patients who are ineligible for intensive chemotherapy or HSCT, its ability to reduce relapse risk in the post-transplant setting is yet to be proven.
Here, we summarize the results of a phase III trial of azacitidine maintenance following transplant in patients with high-risk AML/MDS (NCT00887068) published by Betül Oran and colleagues in Blood Advances.1
A phase III, open-label, randomized study of azacitidine maintenance in a total of 187 patients, 18–75 years of age, with high-risk AML/MDS achieving a complete remission (CR) following HSCT.
Patients were randomly assigned 1:1 to either:
Selected patient characteristics are shown in Table 1. Of the 93 patients enrolled in the treatment arm, 87 started azacitidine therapy. Although most characteristics were well balanced between arms, the azacitidine arm had more patients with a hematopoietic comorbidity index ≥ 4 (Table 1).
Patients received a median of four treatment cycles. A total of 63 patients were withdrawn from the study due to relapse (n = 29), toxicity (n = 11), patient preference (n = 9), infection (n = 7), logistics (n = 5), or GvHD (n = 2).
Table 1. Selected patient characteristics1
AML, acute myeloid leukemia; HCT-CI, hematopoietic comorbidity index; HSCT, hematopoietic stem cell transplant; CR, complete remission; CR1/CR2, first/second CR; MDS, myelodysplastic syndromes. |
||
Observation (n = 93) |
Azacitidine (n = 87) |
|
---|---|---|
Median age, years (range) |
57.5 (20–75) |
57 (19–72) |
Sex, male, % |
60.6 |
58.6 |
MDS, % |
26.6 |
25.3 |
Cytogenetics, % |
|
|
AML disease status at HSCT, % |
|
|
MDS disease status at HSCT, % |
|
|
HCT-CI, % |
|
|
Patient outcomes are summarized in Table 2.
Table 2. Outcome data1
OS, overall survival; RFS, relapse-free survival. |
|||
Outcome |
Observation |
Azacitidine |
p value |
---|---|---|---|
Median follow-up (n = 88), years |
4.06 |
4.60 |
– |
Median RFS, years |
1.28 |
2.07 |
0.19 |
Median OS, years |
3.56 |
2.52 |
0.43 |
Table 3. Reported Grade ≥ 3 AEs1
*No Grade 5 hematological AEs; only one Grade 5 toxicity, which was aspiration pneumonia. |
||
Grade ≥ 3 AE, no. of events |
Observation n = 215 |
Azacitidine* n = 302 |
---|---|---|
Hematological |
5 |
58 |
Thrombocytopenia |
1 |
29 |
Poor graft function |
2 |
29 |
Non-hematological |
56 |
33 |
Infection |
19 |
13 |
Gastrointestinal |
12 |
0 |
Hepatic |
5 |
9 |
Pulmonary |
6 |
4 |
Skin |
5 |
2 |
Despite promising results from phase II studies reporting improved outcomes after transplant for patients with MDS/AML treated with hypomethylating agents, in this study, azacitidine maintenance failed to improve RFS for patients with AML/MDS in the post-transplant setting, compared to observation alone. When considering data interpretation, the investigators made several noteworthy observations:
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