Next‐generation sequencing of baseline genetic mutations and outcomes of eltrombopag and azacitidine therapy in patients with myelodysplastic syndromes and thrombocytopenia: Data from the SUPPORT clinical trial
Pedro Marques Ramos,
Jeea Choi,
Catarina D. Campbell,
Ying A. Wang,
Celine Pallaud,
Michael Dickinson,
Amit Verma,
Moshe Mittelman,
Uwe Platzbecker,
Honar Cherif,
Pierre Fenaux
Affiliations
Pedro Marques Ramos
Novartis Pharma AG Basel Switzerland
Jeea Choi
Novartis Pharmaceuticals Corporation East Hanover New Jersey USA
Catarina D. Campbell
Novartis Institutes of BioMedical Research Cambridge Massachusetts USA
Ying A. Wang
Novartis Global Drug Development Cambridge Massachusetts USA
Celine Pallaud
Novartis Pharma AG Basel Switzerland
Michael Dickinson
The Sir Peter MacCallum Department of Oncology The University of Melbourne Melbourne Australia
Amit Verma
Division of Medical Oncology Department of Medicine Albert Einstein College of Medicine New York New York USA
Moshe Mittelman
Tel Aviv Sourasky Medical Center Tel Aviv University Tel Aviv Israel
Uwe Platzbecker
Medical Clinical and Policlinic Hematology and Cellular Therapy University Hospital Leipzig Leipzig Germany
Honar Cherif
Department of Medical Sciences Uppsala University Uppsala Sweden
Pierre Fenaux
Hôpital Avicenne Assistance Publique‐Hôpitaux de Paris/University Paris XIII Bobigny France
Abstract Eltrombopag has been previously shown to be effective in reversing azacitidine‐mediated thrombocytopenia. This was further investigated in the SUPPORT trial, a phase III study assessing the efficacy/safety of eltrombopag plus azacitidine in patients with intermediate‐ to high‐risk myelodysplastic syndromes and thrombocytopenia. The results did not support a clinical benefit for the addition of eltrombopag to azacitidine. We investigated if the somatic mutational profiles in the patient cohort were associated with treatment outcomes. Based on the available data, we observed no imbalance in the mutational profiles between treatment arms or a clear association between identified somatic mutations and clinical outcomes.
