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Articles by Norbert Vey
Ivosidenib monotherapy in IDH1 mutated myelodysplastic neoplasm/syndrome
Abstract Ivosidenib (IVO) is an oral inhibitor of mutant IDH1 (IDH1m) approved for treatment of IDH1m acute myeloid leukemia (AML) in association with Azacitidine (AZA). We investigated safety and efficacy of IVO monotherapy in patients with IDH1m myelodysplastic neoplasm/syndrome (MDS).
Phase 1 clinical trial of S64315, an intravenous MCL1 inhibitor, in patients with myeloid malignancies
Abstract Preclinical data support myeloid cell leukemia 1 (MCL1) inhibition in the treatment of acute myeloid leukemia (AML). Patients with relapsed/refractory myelodysplastic syndrome (n = 1) or AML (n = 32) or older patients with untreated AML ineligible to receive intensive chemotherapy (n = 5) were treated in this phase 1 study of S64315, an MCL1 inhibitor (NCT02979366). Seven dose levels (50, 100, 200, 250, 300, 400, and 500 mg) were tested.
Venetoclax–Azacitidine Versus Azacitidine for the Treatment of Primary Refractory or First Relapsed Acute Myeloid Leukemia. An IPC‐DATAML‐MSKCC Retrospective Study
To the Editors, Relapsed or refractory (R/R) AML have a poor outcome when treated with intensive chemotherapy (≈30% response rate) [1], or azacitidine (AZA, ≈20% response rate) [2-5]. Both strategies are associated with a poor overall survival (OS) ranging between 6 and 9 months. Venetoclax (VEN)–AZA has been approved for newly-diagnosed AML patients ineligible for IC [6].
Eltrombopag in chronic myelomonocytic leukemia with severe thrombocytopenia. A Groupe Francophone des Myélodysplasies (GFM) study - Leukemia
Severe thrombocytopenia (<50 ×109/L) occurs in 10–30% of Chronic Myelomonocytic Leukemias (CMML) and leads to significant mortality through increased risk of bleeding and disease progression [1]. Its management is ill codified [2]. The thrombopoietin receptor agonist Eltrombopag improves platelet counts in lower-risk myelodysplastic syndromes [3]. Its activity in CMML has been investigated in retrospective case series of up to eleven patients [4,5,6], or as part of a phase I MDS trial [7].
Venetoclax Resistance in Acute Myeloid Leukemia
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GVHD prophylaxis with post-transplant cyclophosphamide results in lower incidence of GVHD and allows faster immunosuppressive treatment reduction compared to antithymocyte globulin in 10/10 HLA-matched unrelated allogeneic hematopoietic cell transplantati
Antithymocyte globulin (ATG) as part of graft-versus-host disease (GVHD) prophylaxis for peripheral blood stem cells (PBSC) transplant significantly reduced the risk of GVHD and thus progressively became a standard, notably for unrelated donor (UD) allogeneic cell transplantation (allo-HCT) [1, 2].
A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune/inflammatory disorders and VEXAS syndrome associated with MDS and CMML | Leukemia
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Lomustine is beneficial to older AML with ELN2017 adverse risk profile and intermediate karyotype: a FILO study
Abstract We previously reported the benefit of lomustine addition to conventional chemotherapy in older acute myeloid leukemias with nonadverse chromosomal aberrations in the LAM-SA 2007 randomized clinical trial (NCT00590837). A molecular analysis of 52 genes performed in 330 patients included in this trial, 163 patients being treated with lomustine in combination with idarubicin and cytarabine and 167 without lomustine, identified 1088 mutations with an average of 3.3 mutations per patient.
Blurring lines between treatment intensity and patient fitness in elderly people with AML
The simplistic view in the onco-haematology field dichotomises the elderly patient population (ie, those aged ≥60 years) into two groups: the fit and the unfit for intensive treatment. Fitness—or rather, so-called unfitness—is defined on the basis of a combination of factors that have been associated with poor outcomes after intensive chemotherapy.
Targeting apoptosis in acute myeloid leukaemia
Acute myeloid leukaemia (AML) is a molecularly and clinically heterogeneous disease, and its incidence is increasing as the populations in Western countries age. Despite major advances in understanding the genetic landscape of AML and its impact on the biology of the disease, standard therapy has not changed significantly in the last three decades. Allogeneic haematopoietic stem cell transplantation remains the best chance for cure, but can only be offered to a minority of younger fit patients.
Elacytarabine - A New Agent in the Treatment of Relapsed/Refractory Acute Myeloid Leukaemia
Acute myeloid leukaemia (AML) is a genetically heterogeneous group of leukaemias that result from clonal transformation of haematopoietic precursors through the acquisition of chromosomal rearrangements and multiple gene mutations. In the absence of treatment, AML progresses rapidly, resulting in bone marrow failure, anaemia, fatal infection, bleeding and organ infiltration.
Phase 1 dose-escalation study of oral abexinostat for the treatment of patients with relapsed/refractory higher-risk myelodysplastic syndromes, acute myeloid leukemia, or acute lymphoblastic leukemia: Leukemia & Lymphoma: Vol 0, No 0
Abexinostat (S 78454, PCI-24781) is an oral hydroxamate-based Class I and II HDAC inhibitor that has demonstrated potent preclinical activity in hematologic and solid tumors [ 29 Rivera-Del Valle N, Gao S, Miller CP, et al. PCI-24781, a novel hydroxamic acid HDAC inhibitor, exerts cytotoxicity and histone alterations via caspase-8 and FADD in leukemia cells. Int J Cell Biol. 2010;2010:207420. [CrossRef] , [PubMed] , 30 Buggy JJ, Cao ZA, Bass KE, et al.
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