2026 Tampa Patient and Family Conference
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 c
patient
Clinical Trial Clinical Trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease. Trials are in four phases. Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment. A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
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Status
Recruiting
Study Date (Range)
-
Bone Marrow Disease(s)
Myelodysplastic Syndromes (MDS)
Age Group
18 years and older
The primary goal of this RENEW study is to find out how well elritercept works in lowering the need for red blood cell (RBC) transfusions. Other aims are to learn how well elitercept works in reducing the need for RBC transfusions over longer periods of time or in adults with…
First Responder Stunned by Severe Aplastic Anemia Diagnosis: Greg's Story
In November 2016, at just 36 years old, Greg was diagnosed with severe aplastic anemia aplastic anemia:
Basiliximab in the Prophylaxis of aGVHD for Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Thalassemia Major: A Prospective, Multicenter, Open-Label, Randomized Controlled Study
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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) in thalassemia major (TM) patients undergoing Matched unrelated donor hematopoietic stem-cell…
Inhibiting ferroptosis enhances ex vivo expansion of human haematopoietic stem cells
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Abstract Improved ex vivo expansion of human haematopoietic stem cells (HSCs) would considerably advance transplantation and genome-engineered therapies, yet existing culture methods still allow substantial HSC loss. Here we show that this attrition is driven largely by…
Rusfertide Continues to Show Strong Results for Polycythemia Vera
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Rusfertide sustained hematocrit control and sharply reduced phlebotomy need through week 52 in PV, showing durable efficacy and a consistent safety profile. Treatment with the hepcidin mimetic rusfertide continued to demonstrate sustained hematocrit control below 45% and high…
Mutant p53 promotes clonal hematopoiesis through generating a chronic inflammatory microenvironment
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Aged individuals with somatic TP53 mutations manifest clonal hematopoiesis (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…
Donor Age and Pre-Transplant Transfusion Burden Independently Predict GVHD and Relapse-Free Survival (GRFS) in Aplastic Anemia
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Abstract Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for aplastic anemia (AA), especially in younger patients with matched sibling donors (MSD). However, in low-resource settings, delayed access to HSCT and pre-transplant complications can…
Isolated KRAS and NRAS mutations in adults with monocytosis and/or cytopenia(s)
Original Publication Date
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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(s). However, their significance in isolation is not well defined. We studied a multi-institutional…
Basiliximab in the Prophylaxis of aGVHD for Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Thalassemia Major: A Prospective, Multicenter, Open-Label, Randomized Controlled Study
Original Publication Date
Article Source
External Web Content
To the Editor, We present the first prospective, multicenter, randomized controlled trial (RCT) evaluating Basiliximab prophylaxis for acute graft-versus-host disease (GVHD) in thalassemia major (TM) patients undergoing Matched unrelated donor hematopoietic stem-cell…