Beta thalassemia

This observational study tests whether hetrombopag, a platelet-boosting drug, can safely speed platelet recovery in children with thalassemia who receive a half-matched (haploidentical) stem cell transplant. Thirty children aged 2 to 17 will be followed for 28 days after transpla…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This study tests whether giving blood transfusions more liberally (when hemoglobin drops to 9 g/dL) versus more restrictively (when it drops to 7.5 g/dL) reduces death and serious complications like heart attack, stroke, or kidney injury in patients aged 70 and older undergoing n…

This study tested whether giving children with transfusion-dependent thalassemia a combination of hydroxyurea and thalidomide works better than either drug alone. Ninety children aged 3-18 were split into three groups: one getting both drugs, one getting only hydroxyurea, and one…

This early-stage study tested a new gene-edited stem cell transplant in 2 children with severe β-thalassemia, a serious blood disorder requiring lifelong transfusions. The treatment aims to help the body produce healthy red blood cells and reduce or stop the need for transfusions…

This phase 2 trial tested whether combining thalidomide and hydroxyurea can reduce the need for blood transfusions in people with transfusion-dependent beta thalassemia. The study enrolled 100 patients over age 2 in Pakistan, where thalassemia is common and life expectancy is low…

This pilot study tested a stem cell transplant from closely matched unrelated donors for 8 patients with severe sickle cell disease or thalassemia major. The donated stem cells were specially processed to remove certain immune cells to reduce the risk of rejection and graft-versu…

This completed Phase 4 trial tested two drugs—hydroxyurea and metformin—in 96 people with transfusion-dependent beta-thalassemia. Participants took one of the two drugs for a year. Researchers measured changes in fetal hemoglobin levels and how often blood transfusions were neede…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This early-phase study tested a new gene-edited stem cell treatment (CS-101) in 5 people with beta-thalassemia, a severe blood disorder requiring regular blood transfusions. The goal was to see if the treatment is safe and can help patients produce healthy red blood cells, potent…

This study tested a new treatment called ET-01 in 3 people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular blood transfusions. The treatment uses the patient's own stem cells, which are modified and then transplanted back. The goal was to see i…

This early-stage study tested a single dose of a gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The main goals were to check safety and see if the treatment helps the body make more healthy hemoglobin. …

This study tested whether red blood cells from umbilical cord blood are safe and work as well as standard adult donor blood for transfusions in extremely preterm infants (born before 28 weeks). 41 babies received small transfusions, and researchers monitored their vital signs, ox…

This study tested a once-weekly injection called dulaglutide (a GLP-1 drug) against daily insulin in 80 children aged 10-18 with transfusion-dependent thalassemia and diabetes. The goal was to see if the weekly shot could control blood sugar just as well or better than insulin ov…

This Phase 2 study tested an experimental drug called etavopivat in 53 people with thalassemia or sickle cell disease. The goal was to see if the pill could safely reduce the number of red blood cell transfusions needed and raise hemoglobin levels. Participants took 400 mg of eta…

This study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…

This small study tested whether L-glutamine, a common supplement, can reduce high blood pressure in the lungs of adults with non-transfusion-dependent thalassemia. Eight participants took either glutamine plus standard care or standard care alone for 60 days. The main goal was to…

This study tested a new MRI technique called QSM to measure iron levels in the liver more accurately than current methods. Researchers compared MRI results from 55 patients before liver transplant with chemical analysis of their removed livers. The goal is to improve diagnosis an…

This study looked at 100 children with beta-thalassemia major and iron overload to see if simple blood tests (ferritin and hepcidin) can detect liver scarring (fibrosis) as accurately as other non-invasive methods. Researchers measured these blood markers and compared them to ult…

This study looked at how long donor red blood cells survive in children with sickle cell disease who get regular blood transfusions. Researchers labeled a small part of the transfused blood with a harmless marker and tracked it over time. The goal was to better understand why som…

This study explored whether low hemoglobin (anemia) is linked to an enlarged heart (cardiomegaly) in children up to 18 years old. Researchers used chest X-rays and echocardiograms to check for heart enlargement in 56 anemic children. The goal was to better understand how anemia a…

This study tested a new, simpler method to measure iron buildup inside red blood cells of people with beta thalassemia major. The goal was to find a cheaper and easier way to monitor iron overload compared to current tests. Researchers analyzed blood samples from 100 patients to …

This study screened 8,400 people living in Chattogram, Bangladesh to find out how common abnormal hemoglobin types are. Researchers used a blood test called HbA1c capillary electrophoresis to detect conditions like HbE trait. The goal was to understand the prevalence of these inh…

This completed study looked at how often hemoglobin disorders (like thalassemia and sickle cell disease) occur in people visiting a hospital in Chattogram, Bangladesh. Researchers tested blood samples from 760 volunteers using advanced methods to find different hemoglobin types. …

This completed study checked how often inherited blood disorders (hemoglobinopathies) occur in people from Mardan, Pakistan, and nearby areas. Researchers analyzed blood samples from 839 people using special lab tests. The goal was to gather local data to help improve public heal…