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Allogeneic stem cell transplantation (allo-SCT) is the current standard of care for patients with poor-risk acute myeloid leukemia (AML).1 Allo-SCT aims at establishing remission by exploiting the graft-versus-leukemia (GvL) effect. Nevertheless, especially in poor-risk patients, disease relapse following allo-SCT is still one of the main reasons of failure in AML.1 Thus, there is a great need for treatments that enhance the GvL effect when combined with allo-SCT, to lead to better outcomes. For this reason, Burak Kalin et al.1 investigated in the phase I/II trial, HOVON 116, the feasibility and preliminary efficacy of epigenetic therapy with panobinostat (PNB) and/or decitabine (DAC) in poor-risk AML patients. The results of this study were published in Blood Advances and are summarized below.
A total of 110 patients who were registered in the study underwent allo-SCT. The key patient baseline characteristics are shown below in Table 1. Briefly, the median age was 59 years, the majority of patients had a World Health Organization (WHO) performance status of 0–1 (83%) and a very poor HOVON/SAKK risk classification (70%)
Table 1. Patient baseline characteristics in HOVON 1161
CR, complete response; CRi, CR with incomplete hematological recovery; EVI1, ecotropic viral integration 1; FC, flow cytometry; HOVON, Dutch-Belgian Cooperative Trial Group for Hematology Oncology; MK, monosomal karyotype; MRD, measurable residual disease; PR, partial response; SAKK, Swiss Group for Clinical Cancer Research; WHO, World Health Organization |
|
Characteristic |
Patient cohort (N = 110) |
---|---|
Median age (range), years |
59 (18–71) |
Male patients, % |
61 |
WHO performance status, % 0 1 2 Unknown |
46 37 10 6 |
Response to induction Cycle 1, % CR/CRi PR Refractory |
61 7 32 |
Response to induction Cycle 2, % CR/CRi PR |
96 4 |
MRD FC (n = 74), % Positive Negative |
2 72 |
HOVON/SAKK risk group, % Poor Very poor MK EVI1 |
30 70 19 25 |
Table 2. Treatment-related toxicities by system in the HOVON 1161
DAC, decitabine; PNB, Panobinostat. |
||||
Treatment-related toxicities |
PNB monotherapy (n = 39), % |
PNB/DAC10 (n = 35), % |
||
---|---|---|---|---|
Grade 2 |
Grade 3 and above |
Grade 2 |
Grade 3 and above |
|
Blood and bone marrow |
2 |
— |
— |
— |
Gastrointestinal |
4 |
4 |
6 |
4 |
Constitutional symptoms |
4 |
2 |
— |
— |
Infections |
7 |
— |
— |
2 |
Metabolic/laboratory |
— |
4 |
— |
4 |
Eye |
2 |
— |
— |
— |
Nervous system |
— |
2 |
— |
2 |
Skin and subcutaneous tissue |
2 |
— |
— |
— |
Table 3. Efficacy outcomes from HOVON 1161
Allo-SCT, allogeneic stem cell transplantation; CIR, cumulative incidence of relapse; NRM, non-relapse mortality; OS, overall survival; PFS, progression-free survival |
|
Outcome |
Patients who underwent allo-SCT, % (N = 110) |
---|---|
2-year CIR |
35 |
2-year NRM |
16 |
2-year PFS |
49 |
2-year OS |
50 |
The results of this phase I/II trial showed that panobinostat monotherapy or in combination with 10 mg/m2 decitabine are feasible and tolerable epigenetic regimens for patients with poor-risk AML, who have undergone allo-SCT. Data did not indicate a synergistic or additive effect of panobinostat and decitabine. Due to the preliminary nature of these results, further large-scale prospective trials are needed to assess the efficacy of these post allo-SCT epigenetic therapies for the treatment of AML patients with poor outcomes.
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