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The clinical impact of immunosuppression termination (IST) after allogeneic haematopoietic stem cell transplantation (allo-HSCT) remains to be fully elucidated. This study was aimed at assessing the impact of IST within 2 years after transplantation on subsequent clinical outcomes. We analysed data for patients from the Transplant Registry Unified Management Program 2 (TRUMP 2) database who…
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Measurable residual disease (MRD) monitoring has become a critical component in the management of
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of…
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Menin inhibition leads to an antileukemic effect through hematopoietic differentiation. Treatment with the menin inhibitor revumenib results in clinical remissions in relapsed or
refractory
refractory:
Not responsive to treatment or cure. For example, refractory anemia is a low red blood cell count that doesn't respond to standard treatments.
(R/R)
acute myeloid leukemia
acute…
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Therapy-related myeloid neoplasm (tMN) is a fatal consequence of exposure to cytotoxic therapy administered in the treatment of cancer. Individuals with pre-existing TP53 clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
(CH) are at high risk of tMN, with avoidance of therapy being the only strategy to reduce tMN risk.…
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Despite concerns about the toxicity of allogeneic hematopoietic cell transplantation (alloHCT) in older patients, prospective data characterizing prevalence or risk stratification for geriatric morbidity such as disability or frailty are limited. We prospectively assessed the prognostic impact of the novel composite health assessment risk model (CHARM), a score established to predict 1-year…
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Post-transplant
cyclophosphamide
cyclophosphamide:
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system.
(PTCy) is associated with infectious and organ…
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Selection of a hematopoietic progenitor cell donor for allogeneic hematopoietic cell transplantation (allo HCT) is essential for treatment planning; however, the parameters that define an "optimal" donor in the modern era are not well defined. Historically, donor-recipient
human leukocyte antigen
human leukocyte antigen:
(LEW-kuh-site ANT-i-jun) One of a group of proteins found on the…
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Recent advances in whole-genome and next-generation sequencing (NGS) have significantly deepened our understanding of the genomic landscape of
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and…
Article last updated
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Patients with
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
,
paroxysmal nocturnal hemoglobinuria…
Conference Event last updated
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Abstract
QuANTUM-First was a randomized trial that demonstrated that the addition of quizartinib, a potent and selective FMS-like tyrosine kinase 3 (FLT3) inhibitor, to induction and consolidation
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be…
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Abstract
Improved ex vivo expansion of human haematopoietic
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem…
(HSCs)…
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Aged individuals with somatic TP53 mutations manifest clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
(CH) and are at high risk of developing myeloid neoplasms. However, the underlying mechanisms are not fully understood. Here we show that inflammatory stress confers a competitive advantage to p53 mutant hematopoietic…
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Abstract
NRAS and KRAS mutations, commonly identified alongside ancestral co-mutations, are generally regarded as pathogenic in adults presenting with monocytosis and/or
cytopenia
cytopenia:
(sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count.
(s). However, their significance in isolation is not well defined. We studied a multi-institutional…
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Chemotherapy
Chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with…
-free approaches could offer comparable efficacy with less…
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Key Takeaways
The combination therapy achieved a 91% overall response rate, with 45% complete remission and 46% marrow complete remission rates.
Median overall survival was 30 months, with 1- and 3-year survival rates of 68.8% and 40.7%, respectively.
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Decitabine
Decitabine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like a…
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Key points
Outpatient low-dose VenAza plus itraconazole reduced early mortality and hospitalizations while maintaining treatment efficacy.
VenAza offers a safer, cost-effective alternative to intensive
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may…
Article last updated
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Highlights
Heme biosynthesis enzymes are variably suppressed in AML
Heme levels are linked with altered leukemic transcriptional programs via BACH1
Heme biosynthesis is a selective dependency in AML, both in vitro and in vivo
Heme starvation disrupts complex IV, inducing copper accumulation and cuproptosis
Summary…
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Key Points
Ivosidenib
Ivosidenib:
A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including acute myeloid leukemia (AML).
,
venetoclax
venetoclax:
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines…
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Recordings:
Aplastic Anemia
Low Risk MDS
High Risk MDS to AML
Transplant and GVHD
MDS/MPN Overlap
A Patient Story: Shauna McMillian
Managing PNH Complications
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Session Recordings Available:
MDS to MPN Overlap from the 2025 Boston Patient and Family Conference
High Risk MDS from the 2025 Boston Patient and Family Conference
Low Risk MDS from the 2025 Boston Patient and Family Conference
What is Aplastic Anemia? From the 2025 Boston Patient and Family Conference
What is PNH? Webinar recorded at the 2025 Boston Patient and Family Conference…
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