How to treat VEXAS syndrome: a systematic review on effectiveness and safety of current treatment strategies
Z Boyadzhieva, N Ruffer, I Kötter, M Krusche - Rheumatology, 2023 - academic.oup.com
Objectives To evaluate the effectiveness and safety of current treatment strategies for the
vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome. Methods A …
vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome. Methods A …
Comparison of the International Consensus and 5th WHO edition classifications of adult myelodysplastic syndromes and acute myeloid leukemia
B Falini, MP Martelli - American Journal of Hematology, 2023 - Wiley Online Library
Several editions of the World Health Organization (WHO) classifications of lympho‐
hemopoietic neoplasms in 2001, 2008, and 2016 served as the international standard for …
hemopoietic neoplasms in 2001, 2008, and 2016 served as the international standard for …
Outcome prediction in myelodysplastic neoplasm undergoing hematopoietic cell transplant in the molecular era of IPSS-M
Outcome of patients with myelodysplastic neoplasms/syndromes (MDS) is closely related to
the biology and molecular genetics of the disease as well as its status prior to allogeneic …
the biology and molecular genetics of the disease as well as its status prior to allogeneic …
Real-world use and outcomes of hypomethylating agent therapy in higher-risk myelodysplastic syndromes: why are we not achieving the promise of clinical trials?
AM Zeidan, T Salimi, RS Epstein - Future Oncology, 2021 - Taylor & Francis
Myelodysplastic syndromes are hematological malignancies characterized by ineffective
hematopoiesis and a high risk of progression to acute myeloid leukemia. Hypomethylating …
hematopoiesis and a high risk of progression to acute myeloid leukemia. Hypomethylating …
STIMULUS-MDS2 design and rationale: a phase III trial with the anti-TIM-3 sabatolimab (MBG453)+ azacitidine in higher risk MDS and CMML-2
AM Zeidan, A Giagounidis, MA Sekeres, Z Xiao… - Future …, 2023 - Taylor & Francis
Patients with higher-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic
leukemia (CMML) unfit for hematopoietic stem cell transplantation have poor outcomes …
leukemia (CMML) unfit for hematopoietic stem cell transplantation have poor outcomes …
Eltrombopag for low-risk myelodysplastic syndromes with thrombocytopenia: interim results of a phase ii, randomized, placebo-controlled clinical trial (EQOL-MDS)
PURPOSE In myelodysplastic syndromes (MDS), severe thrombocytopenia is associated
with poor prognosis. This multicenter trial presents the second-part long-term efficacy and …
with poor prognosis. This multicenter trial presents the second-part long-term efficacy and …
Long-term efficacy and safety of luspatercept for anemia treatment in patients with lower-risk myelodysplastic syndromes: the phase II PACE-MDS study
U Platzbecker, KS Götze, P Kiewe… - Journal of Clinical …, 2022 - ascopubs.org
Clinical trials frequently include multiple end points that mature at different times. The initial
report, typically based on the primary end point, may be published when key planned co …
report, typically based on the primary end point, may be published when key planned co …
Have we reached a molecular era in myelodysplastic syndromes?
MT Voso, C Gurnari - Hematology, 2021 - ashpublications.org
Myelodysplastic syndromes (MDS) are characterized by heterogeneous biological and
clinical characteristics, leading to variable outcomes. The availability of sophisticated …
clinical characteristics, leading to variable outcomes. The availability of sophisticated …
Diagnostic capabilities, clinical features, and longitudinal UBA1 clonal dynamics of a nationwide VEXAS cohort
C Gurnari, MR Pascale, A Vitale, E Diral… - American Journal of …, 2024 - Wiley Online Library
VEXAS is a prototypic hemato‐inflammatory disease combining rheumatologic and
hematologic disorders in a molecularly defined nosological entity. In this nationwide study …
hematologic disorders in a molecularly defined nosological entity. In this nationwide study …
[HTML][HTML] Autoimmune manifestations in VEXAS: opportunities for integration and pitfalls to interpretation
A Bruno, C Gurnari, T Alexander, JA Snowden… - Journal of Allergy and …, 2023 - Elsevier
VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) is a novel entity
manifesting with a multiplicity of clinical features. Somatic mutations of the UBA1 gene in …
manifesting with a multiplicity of clinical features. Somatic mutations of the UBA1 gene in …