patient
NMDP and CIBMTR share new, promising stem cell transplantation trial data using mismatched, unrelated donors at the 2024 EHA Congress
Original Publication Date
Article Source
External Web Content
MINNEAPOLIS, June 14, 2024 — NMDPSM, a global nonprofit leader in cell therapy, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) announced interim results from the ACCESS trial as an oral abstract during the European Hematology Association (EHA)…
Alain MIna, MD
Institution
National Cancer Institute - NCI
Physician Status
accepting new patients
Primary Disease Area of Focus
Acute Myeloid Leukemia (AML)
Myelodysplastic Syndromes (MDS)
Non-therapeutic Study Non-therapeutic Study: The purpose of a non-therapeutic study is to increase the understanding of a disease or condition through observation, data collection, or other methods. A non-therapeutic study does not provide treatment or intervention for participants. This type of research contributes to general knowledge that may benefit others in the future by improving treatment or developing new therapies. Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US
Image
Status
Enrolling
Study Date (Range)
-
Bone Marrow Disease(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Age Group
18 years and older
This study aims to capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. As a participant, you will track what you're noticing about your health in the Folia Health app once per week for 6…
FDA approves imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia
Original Publication Date
Article Source
External Web Content
On June 6, 2024, the Food and Drug Administration approved imetelstat (Rytelo, Geron Corporation), an oligonucleotide telomerase inhibitor, for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring four or more red…
Blood Transfusions and Iron Overload
Topic(s)
Living Well with Bone Marrow Failure
Managing Your Treatment
In this webinar, Dr. Thomas Coates will discuss blood transfusions and the potential side effect, iron overload. You may submit questions in advance by emailing help@aamds.org.
Understanding Secondary AML: High-Risk MDS to AML
Topic(s)
Myelodysplastic Syndromes (MDS)
Acute Myeloid Leukemia (AML)
Presenter(s)
Nathan Punwani, MD, MPH
In this webinar, Dr. Nathan Punwani discusses the development of secondary AML from high risk MDS, treatment options and the quality of life impact for patients.
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 Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, ATG) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients with Refractory or Recurrent Severe Aplastic Anemia
Status
Recruiting
Study Date (Range)
-
Bone Marrow Disease(s)
Aplastic Anemia
Age Group
40 years to 75 years
The current standard treatment for aplastic anemia (AA) is immunosuppressive therapy (IST), with only temporary symptom relief and high rates of paroxysmal nocturnal hemoglobinuria, clonal hematopoiesis, and myelodysplastic syndrome. When a matched donor is available, allogeneic…
Kristen Schratz, MD
Institution
Johns Hopkins University
Physician Status
accepting new patients
Primary Disease Area of Focus
Myelodysplastic Syndromes (MDS)
Pediatric
Hiroyuki Takamori, MD
Institution
Osaka Univesity Graduate School of Medicine
Physician Status
accepting new patients
Primary Disease Area of Focus
Paroxysmal Nocturnal Hemoglobinuria (PNH)