MEK inhibitors for neurofibromatosis type 1 manifestations: Clinical evidence and consensus

PMK De Blank, AM Gross, S Akshintala… - Neuro …, 2022 - academic.oup.com
The wide variety of clinical manifestations of the genetic syndrome neurofibromatosis type 1
(NF1) are driven by overactivation of the RAS pathway. Mitogen-activated protein kinase …

Bedside to bench in juvenile myelomonocytic leukemia: insights into leukemogenesis from a rare pediatric leukemia

TY Chang, CC Dvorak, ML Loh - Blood, The Journal of the …, 2014 - ashpublications.org
Juvenile myelomonocytic leukemia (JMML) is a typically aggressive myeloid neoplasm of
childhood that is clinically characterized by overproduction of monocytic cells that can …

RAS/CBL mutations predict resistance to JAK inhibitors in myelofibrosis and are associated with poor prognostic features

G Coltro, G Rotunno, L Mannelli, C Mannarelli… - Blood …, 2020 - ashpublications.org
The dysregulation of the JAK/STAT pathway drives the pathogenesis of myelofibrosis (MF).
Recently, several JAK inhibitors (JAKis) have been developed for treating MF. Select …

A gain-of-function p53 mutant synergizes with oncogenic NRAS to promote acute myeloid leukemia in mice

A Rajagopalan, Y Feng, MB Gayatri… - The Journal of …, 2023 - Am Soc Clin Investig
We previously demonstrated that a subset of acute myeloid leukemia (AML) patients with
concurrent RAS pathway and TP53 mutations have an extremely poor prognosis and that …

[HTML][HTML] An International MDS/MPN Working Group's perspective and recommendations on molecular pathogenesis, diagnosis and clinical characterization of …

TI Mughal, NCP Cross, E Padron, RV Tiu… - …, 2015 - ncbi.nlm.nih.gov
In the 2008 WHO classification, chronic myeloid malignancies that share both
myelodysplastic and myeloproliferative features define the myelodysplastic …

Turning the tide in myelodysplastic/myeloproliferative neoplasms

MWN Deininger, JW Tyner, E Solary - Nature Reviews Cancer, 2017 - nature.com
Myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) are aggressive
myeloid malignancies recognized as a distinct category owing to their unique combination of …

Dual targeting of JAK2 and ERK interferes with the myeloproliferative neoplasm clone and enhances therapeutic efficacy

S Brkic, S Stivala, A Santopolo, J Szybinski, S Jungius… - Leukemia, 2021 - nature.com
Myeloproliferative neoplasms (MPN) show dysregulated JAK2 signaling. JAK2 inhibitors
provide clinical benefits, but compensatory activation of MAPK pathway signaling impedes …

[HTML][HTML] DNA-hypomethylating agents as epigenetic therapy before and after allogeneic hematopoietic stem cell transplantation in myelodysplastic syndromes and …

C Flotho, S Sommer, M Lübbert - Seminars in Cancer Biology, 2018 - Elsevier
Myelodysplastic syndrome (MDS) is a clonal bone marrow disorder, typically of older adults,
which is characterized by ineffective hematopoiesis, peripheral blood cytopenias and risk of …

Expression of NrasQ61R and MYC transgene in germinal center B cells induces a highly malignant multiple myeloma in mice

Z Wen, A Rajagopalan, ED Flietner… - Blood, The Journal …, 2021 - ashpublications.org
NRAS Q61 mutations are prevalent in advanced/relapsed multiple myeloma (MM) and
correlate with poor patient outcomes. Thus, we generated a novel MM model by …

Phase II trial of MEK inhibitor binimetinib (MEK162) in RAS-mutant acute myeloid leukemia

A Maiti, K Naqvi, TM Kadia, G Borthakur… - … Myeloma and Leukemia, 2019 - Elsevier
Background Relapsed and refractory (R/R) acute myeloid leukemia (AML) continues to be a
therapeutic challenge with poor outcomes. Dysregulation of the mitogen-activated protein …