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Cladribine as First-Line Therapy for Indolent and Advanced Systemic Mastocytosis
ASH 2021 – Systemic Mastocytosis: Wrap-Up
Cladribine has long been established as an efficient therapy in systemic mastocytosis and remains a valuable treatment option even as new therapies emerge.
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First-in-Human Study of Menin Inhibitor SNDX-5613 in MLL-Rearranged and NPM1-Mutant Acute Leukemia
ASH 2021 – AML
In the first-in-human phase 1/2 AUGMENT 101 study, SNDX-5613 demonstrated an acceptable safety profile and promising antileukemic activity in patients with heavily pretreated relapsed/refractory
MLL
-rearranged and
NPM1
-mutated acute leukemias.
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Genetic Testing for Hereditary Alpha-Tryptasemia in Patients Presenting with Mast-Cell Mediator Symptoms in the Absence of Symptoms of Mastocytosis
ASH 2021 – Systemic Mastocytosis: Wrap-Up
Hereditary alpha-tryptasemia may promote development of systemic mastocytosis. Screening for variations in copy number of the alpha tryptase gene may provide early evidence for diagnosis of mastocytosis.
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Allogeneic Stem-Cell Transplantation for Patients with AML in Second Complete Remission Transplanted from Unrelated Donors with Post-Transplant Cyclophosphamide
ASH 2021 – AML
AML patients who underwent allogeneic stem-cell transplantation from unrelated donors while in their second complete remission with post-transplant cyclophosphamide had similar outcomes to those who underwent transplantation while in first complete remission.
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Assessing the Potential Role of Anti-CD117 CAR T-Cells for Targeted Therapy in Advanced Systemic Mastocytosis
ASH 2021 – Systemic Mastocytosis: Wrap-Up
CAR T-cell immunotherapy is highly efficient because of its ability to target specific tumor antigens. Preliminary evidence suggests that CAR T-cells directed against CD117, the KIT receptor, may emerge as a therapeutic option for advanced systemic mastocytosis.
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Ivosidenib plus Azacitidine versus Placebo plus Azacitidine in Patients with Newly Diagnosed AML with an IDH1 Mutation
ASH 2021 – AML
The phase 3 AGILE study demonstrated that ivosidenib + azacitidine therapy provided significant survival benefit compared with placebo plus azacitidine in patients with newly diagnosed
IDH1
mutation–positive AML who were ineligible for intensive chemotherapy.
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Whole Genome Sequencing Detects Chromosomal Aberrations Associated with Systemic Mastocytosis
ASH 2021 – Systemic Mastocytosis: Wrap-Up
Cytogenetic aberrations are rarely found by cytogenetics in systemic mastocytosis but are associated with advanced disease. Whole genome sequencing detects both chromosomal aberrations and non-
KIT
gene mutations and can be used as an alternative to cytogenetics for assessing disease risk.
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Venetoclax plus FLAG-IDA Induction/Consolidation in Newly Diagnosed AML
ASH 2021 – AML
Addition of venetoclax to FLAG-IDA yielded high minimal residual disease–negative composite complete response rates in newly diagnosed patients with AML, accompanied by a favorable safety profile.
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PATHFINDER Study Reveals Potential for Avapritinib to Modify Progression of Advanced Systemic Mastocytosis
ASH 2021 – Systemic Mastocytosis: Wrap-Up
Avapritinib was recently approved for treatment in advanced systemic mastocytosis after demonstrating in clinicals trials the ability to improve hematologic and bone marrow responses in patients.
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The FLT3/SYK Inhibitor HM43239 Shows Activity in Patients with Relapsed or Refractory FLT3-Mutated and Wild-Type AML
ASH 2021 – AML
Results from an ongoing first-in-human phase 1/2 study indicated that the FLT3/SYK inhibitor HM43239 yielded a favorable safety profile and encouraging antileukemic activity in patients with relapsed/refractory AML regardless of
FLT3
mutation status.
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