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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…
Article last updated
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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…
Article last updated
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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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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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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
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)…
Article last updated
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Abstract
Health-related quality of life (HRQoL) of patients with myeloproliferative neoplasms (MPNs) may be impaired across several domains. In this multicenter observational study, we evaluated HRQoL and symptoms in a cohort of MPN patients with validated measures, including the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), the…
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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
Conference Event last updated
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Rusfertide sustained
hematocrit
hematocrit:
(hi-MA-tuh-crit) A blood test that measures the percentage of the blood made up of red blood cells. This measurement depends on the number of red blood cells and their size. Hematocrit is part of a complete blood count. Also called HCT, packed cell volume, PCV.
control and sharply reduced phlebotomy need through week 52 in PV, showing durable…
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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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To the Editor,
We present the first prospective, multicenter, randomized controlled trial (RCT) evaluating Basiliximab prophylaxis for acute
graft-versus-host disease (GVHD)
graft-versus-host disease (GVHD):
Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient'…
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Jen had a great life. She was married with two children, and she’s just transitioned from teaching to school counseling. At 41, life was busy yet wonderful.Her symptoms were unusual, but not alarming. The leg pain was new, as was the bruising. Then fatigue set in, far beyond usual tiredness from her schedule. A visit to the doctor did not offer a definitive diagnosis. They suggested it was…
Patient Chronicle last updated
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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
Conference Event last updated
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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…
Conference Event last updated
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
MDS/MPN Overlap
PNH and Current Therapies
Living Well with Bone Marrow Failure
Judy's Story of PNH
Transplant and GVHD
Aplastic Anemia
PNH Complications
High Risk MDS
Conference Event last updated
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Dr. Rory Shallis explains the treatment,
Imetelstat
Imetelstat:
Who should not take RYTELO?
Before taking RYTELO, tell your healthcare provider about all your medical conditions, including if you:
are pregnant or plan to become pregnant. RYTELO may harm your unborn baby and may cause loss of pregnancy. Tell your healthcare provider right away if you…
, for patients with Low Risk MDS…
Podcast Episode last updated
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Meet Evan. In a recent conversation with the
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…
and MDS…
Article last updated
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To the editor,
Mutations in nucleophosmin 1 (NPM1-mut) are detected in approximately 30% of newly diagnosed (ND)
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…
Article last updated
.