Genetics of MDS

S Ogawa - Blood, The Journal of the American Society of …, 2019 - ashpublications.org
Our knowledge about the genetics of myelodysplastic syndromes (MDS) and related
myeloid disorders has been dramatically improved during the past decade, in which …

Biology and relevance of human acute myeloid leukemia stem cells

D Thomas, R Majeti - Blood, The Journal of the American …, 2017 - ashpublications.org
Evidence of human acute myeloid leukemia stem cells (AML LSCs) was first reported nearly
2 decades ago through the identification of rare subpopulations of engrafting cells in …

Mutations in AML: prognostic and therapeutic implications

CD DiNardo, JE Cortes - Hematology 2014, the American …, 2016 - ashpublications.org
Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized
by the proliferation and aberrant differentiation of immature clonal myeloid cells. The …

Molecular MRD status and outcome after transplantation in NPM1-mutated AML

R Dillon, R Hills, S Freeman, N Potter… - Blood, The Journal …, 2020 - ashpublications.org
Relapse remains the most common cause of treatment failure for patients with acute myeloid
leukemia (AML) who undergo allogeneic stem cell transplantation (alloSCT), and carries a …

Outcomes of older patients with NPM1-mutated AML: current treatments and the promise of venetoclax-based regimens

CA Lachowiez, S Loghavi, TM Kadia, N Daver… - Blood …, 2020 - ashpublications.org
Abstract Nucleophosmin-1 mutations (NPM1+) occur in∼ 30% of acute myeloid leukemia
(AML) patients. Although typically associated with favorable prognosis, the beneficial impact …

Genetically distinct leukemic stem cells in human CD34 acute myeloid leukemia are arrested at a hemopoietic precursor-like stage

L Quek, GW Otto, C Garnett, L Lhermitte… - Journal of Experimental …, 2016 - rupress.org
Our understanding of the perturbation of normal cellular differentiation hierarchies to create
tumor-propagating stem cell populations is incomplete. In human acute myeloid leukemia …

A phase 1 clinical trial of single-agent selinexor in acute myeloid leukemia

R Garzon, M Savona, R Baz, M Andreeff… - Blood, The Journal …, 2017 - ashpublications.org
Selinexor is a novel, first-in-class, selective inhibitor of nuclear export compound, which
blocks exportin 1 (XPO1) function, leads to nuclear accumulation of tumor suppressor …

AML engraftment in the NOD/SCID assay reflects the outcome of AML: implications for our understanding of the heterogeneity of AML

DJ Pearce, D Taussig, K Zibara, LL Smith, CM Ridler… - Blood, 2006 - ashpublications.org
The nonobese diabetic/severe combined immunodeficient (NOD/SCID) assay is the current
model for assessment of human normal and leukemic stem cells. We explored why 51% of …

Prevalence, clinical profile, and prognosis of NPM mutations in AML with normal karyotype

N Boissel, A Renneville, V Biggio, N Philippe… - Blood, 2005 - ashpublications.org
Mutation of the nucleophosmin (NPM) gene has been reported as the most frequent
mutation in acute myeloid leukemia (AML), especially in the presence of a normal karyotype …

Midostaurin in patients with acute myeloid leukemia and FLT3-TKD mutations: a subanalysis from the RATIFY trial

MT Voso, RA Larson, D Jones, G Marcucci… - Blood …, 2020 - ashpublications.org
The results from the RATIFY trial (ClinicalTrials. gov: NCT00651261; CALGB 10603)
showed that midostaurin combined with standard chemotherapy significantly improved …