Pure red-cell aplasia

This trial tests a combination of two drugs, sirolimus and all-trans retinoic acid (ATRA), for people with autoimmune anemia that has not responded to standard treatments. Autoimmune anemia occurs when the immune system attacks red blood cells, causing fatigue and other symptoms.…

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This phase 2 trial tests whether isatuximab can help people whose blood cell counts stay low after a stem cell transplant for blood cancer. The study enrolls 12 adults whose disease is in remission but who have developed immune-related blood cell deficiencies. Participants receiv…

This study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…

This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease ou…

This study was designed to see if a new monitoring algorithm could detect early signs of ovarian damage in women and girls who had cancer treatment. Researchers planned to compare a group using the new algorithm with a group that received standard care. However, the study was wit…