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Session Recordings Available:
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Recorded Sessions:
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
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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…
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paroxysmal nocturnal hemoglobinuria…
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DEADLINE MAY 8Senators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a bipartisan letter to the Senate in support of increased funding for the Department of Defense’s medical research accounts, including CDMRP and BMFRP.You can help by asking Your Senators to Sign the LetterSenators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a…
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Listen to Barry describe the diagnosis, watch-and-wait, treatment, and survivorship for MDS, or
Myelodysplastic Syndromes
Myelodysplastic Syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or…
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Abstract
Selection of a hematopoietic progenitor cell donor for allogeneic hematopoietic cell transplantation (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
HLA
HLA:
See human leukocyte antigen.
mismatching correlated strongly with risk for
graft-versus-host…
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Abstract
Allogeneic stem cell transplantation (SCT) is a curative treatment in
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People…
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Abstract
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 to treat these conditions.
(Ven) in combination with hypomethylating agents (HMA) (azacitidine or
decitabine
decitabine:
It works by reducing the amount of methylation in the…
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Abstract
Allogeneic stem cell transplantation (SCT) is a curative treatment in
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People…
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Luspatercept generated favorable efficacy and safety outcomes in the treatment of
anemia
anemia:
(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.
in patients with transfusion-dependent, lower-risk MDS.
Data from a systematic review and meta-analysis of 20…
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Key Points
MRD-guided
azacitidine
azacitidine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U…
therapy can effectively prevent or delay…
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Aside from allogeneic transplantation, the current standard-of-care approach for higher-risk
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or…
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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
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potential curative therapy for myelodysplastic syndrome (MDS), recommended in higher risk disease according to the
International Prognostic Scoring System
International Prognostic Scoring System:
A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS)…
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Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potential curative therapy for myelodysplastic syndrome (MDS), recommended in higher risk disease according to the
International Prognostic Scoring System
International Prognostic Scoring System:
A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS)…
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Abstract
The intensity of the conditioning regimen in hematopoietic stem cell transplantation (HSCT) correlates with the risk of relapse, however its potential benefit may be outweighed by the associated risk of toxicity. The addition of total marrow irradiation (TMI) to myeloablative conditioning provides an opportunity to increase intensity with minimal additional toxicity. In this phase II…
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ABSTRACT
The COMMANDS trial established luspatercept as a first-line treatment for
anemia
anemia:
(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.
in transfusion-dependent lower-risk (LR)
myelodysplastic syndromes
myelodysplastic syndromes:
(my…
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2024 Patient and Family Conference in Los AngelesSession Recordings:Transplant for Aplastic Anemia and MDSEmerging Therapies in High-Risk MDSCardiovascular Concerns after HCTSupportive Care ManagementAdvances in Treating Lower-Risk MDSPalliative CareLiving with Chronic GVHDA Patient StoryInherited Bone Marrow Failure SyndromesAdvances in Treating Aplastic Anemia
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