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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'…
Article last updated
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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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Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for
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…
Article last updated
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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'…
Article last updated
.
NIH
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…
shows promise for people with treatment-limited, life-…
Article last updated
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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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Curtis et al. (July 17 issue)1 report evidence of longer
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's body as foreign and mount an immune response. GVHD most commonly effects the…
Article 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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Abstract
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…
(AA) transformation into
myelodysplastic…
Article last updated
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Steroid-refractory GvHD (SR-GvHD) following allogeneic transplant is a major clinical challenge and is associated with substantial mortality [1]. While the European Society for Bone and Marrow Transplantation (EBMT) recommends ruxolitinib for second-line treatment of GvHD [2], some patients may experience side effects or refractoriness, and therefore require alternative treatment options [2].…
Article last updated
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Abstract
Purpose
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for advanced hematologic malignancies. HSCT using
human leukocyte antigen
human leukocyte antigen:
(LEW-kuh-site ANT-i-jun) One of a group of proteins found on the surface of white blood cells and other cells. These antigens differ from person to person. A human leukocyte antigen test is…
Article last updated
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Evan biked daily. He had no symptoms. The diagnosis was fast, but surprising. Evan shares his remarkable story in this episode.
Podcast Episode last updated
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Recorded Sessions:
Nutrition for Aplastic Anemia, MDS, or PNH
Managing PNH Treatments
Advances in Aplastic Anemia
What is PNH?
Transplant and GVHD: Aplastic Anemia and PNH
Inherited Bone Marrow Failure (focus on pediatrics)
Expert Panel: Pediatric Bone Marrow Failure
Life After Diagnosis - Pediatrics
Pediatric MDS
Transitioning to Adult Care
Pediatric PNH
Pediatric Aplastic…
Conference Event last updated
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Data from APPULSE-PNH may support oral
iptacopan
iptacopan:
FABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It was approved by the U.S. Food and Drug Administration in December 2023. Fabhalta is taken twice a day in a capsule form.
What is FABHALTA?
…
as a potentially practice-changing…
Article last updated
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Low rates of chronic graft-versus-host disease with ruxolitinib maintenance following allogeneic HCT
Key Points
Prolonged ruxolitinib administration after allogeneic HCT is associated with low rates of clinically significant chronic GVHD.
Abstract
Despite recent advances in
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…
Article last updated
.
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.
plus cyclosporin, a calcineurin inhibitor, significantly improved…
Article last updated
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Shane Pope talks about living with PNH, or Paroxysmal Nocturnal Hemglobinuria, in this recorded conversation with Leigh Clark. Listen or read the transcript below.
Podcast Episode last updated
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