Results of a phase II study of thalidomide and azacitidine in patients with clinically advanced myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and low blast count acute myeloid leukemia (AML)

2016 ◽  
Vol 58 (2) ◽  
pp. 298-307 ◽  
Author(s):  
Melita Kenealy ◽  
Nigel Patton ◽  
Robin Filshie ◽  
Andrew Nicol ◽  
Shir-Jing Ho ◽  
...  
Keyword(s):  
Acute Myeloid Leukemia ◽  
Phase Ii ◽  
Myelodysplastic Syndromes ◽  
Myeloid Leukemia ◽  
Phase Ii Study ◽  
Chronic Myelomonocytic Leukemia ◽  
Blast Count ◽  
Myelomonocytic Leukemia ◽  
Acute Myeloid

scholarly journals Eprenetapopt Plus Azacitidine in TP53-Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia: A Phase II Study by the Groupe Francophone des Myélodysplasies (GFM)

2021 ◽  
pp. JCO.20.02342
Author(s):  
Thomas Cluzeau ◽  
Marie Sebert ◽  
Ramy Rahmé ◽  
Stefania Cuzzubbo ◽  
Jacqueline Lehmann-Che ◽  
...  
Keyword(s):  
Acute Myeloid Leukemia ◽  
Adverse Events ◽  
Phase Ii ◽  
Myelodysplastic Syndromes ◽  
Myeloid Leukemia ◽  
Phase Ii Study ◽  
International Prognostic Scoring System ◽  
High Risk Population ◽  
Very High ◽  
Acute Myeloid

PURPOSE TP53-mutated ( TP53m) myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) have very poor outcome irrespective of the treatment received, including 40% responses (20% complete remission [CR]) with azacitidine (AZA) alone, short response duration, and a median overall survival (OS) of approximately 6 months. Eprenetapopt (APR-246), a novel first-in-class drug, leads to p53 protein reconformation and reactivates its proapoptotic and cell-cycle arrest functions. PATIENTS AND METHODS This phase II study assessed the safety and efficacy of eprenetapopt in combination with AZA in untreated high or very high International Prognostic Scoring System-R TP53m MDS and AML patients. RESULTS Fifty-two TP53m patients (34 MDS, 18 AML [including seven with more than 30% blasts]) were enrolled. In MDS, we observed an overall response rate (ORR) of 62%, including 47% CR, with a median duration of response at 10.4 months. In AML, the ORR was 33% including 17% CR (27% and 0% CR in AML with less than and more than 30% marrow blasts, respectively). Seventy-three percent of responders achieved TP53 next-generation sequencing negativity (ie, variant allele frequency < 5%). The main treatment-related adverse events were febrile neutropenia (36%) and neurologic adverse events (40%), the latter correlating with a lower glomerular filtration rate at treatment onset ( P < .01) and higher age ( P = .05), and resolving with temporary drug interruption without recurrence after adequate eprenetapopt dose reduction. With a median follow-up of 9.7 months, median OS was 12.1 months in MDS, and 13.9 and 3.0 months in AML with less than and more than 30% marrow blasts, respectively. CONCLUSION In this very high-risk population of TP53m MDS and AML patients, eprenetapopt combined with AZA was safe and showed potentially higher ORR and CR rate, and longer OS than reported with AZA alone.

Azacitidine access program for Belgian patients with myelodysplastic syndromes, acute myeloid leukemia or chronic myelomonocytic leukemia

2014 ◽  
Vol 31 (1) ◽  
pp. 35-42
Author(s):  
S. Meers ◽  
D. Selleslag ◽  
H. Potier ◽  
A. Glasmacher ◽  
P. Mineur ◽  
...  
Keyword(s):  
Acute Myeloid Leukemia ◽  
Myelodysplastic Syndromes ◽  
Myeloid Leukemia ◽  
Chronic Myelomonocytic Leukemia ◽  
Myelomonocytic Leukemia ◽  
Access Program ◽  
Acute Myeloid
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