SICKLE CELL DISEASE

This study tested whether L-Arginine, a natural amino acid, can safely help children with sickle cell disease who have increased blood flow speed in their heart (a sign of lung pressure). Fifty kids aged 5-18 took L-Arginine for 3 months. Researchers measured changes in heart blo…

This early-stage study tested a new drug called Pociredir (FTX-6058) in 45 adults with sickle cell disease. The main goal was to check if the drug is safe and how the body processes it. Researchers also looked at whether it could increase fetal hemoglobin, which may help reduce s…

This study tested whether famotidine, a common heartburn medicine, can lower a blood marker linked to pain crises in children with sickle cell disease. Thirty children aged 1 to 17 took the drug for 29 days. The goal was to see if it reduces P-selectin, a protein involved in bloo…

This study tested a drug called etavopivat in 12 children and young adults (ages 12-21) with sickle cell disease. The goal was to see if the drug improves blood flow and oxygen delivery to the brain and muscles. Researchers measured changes in brain blood flow and oxygen use over…

This early-stage study tested a new gene-edited stem cell treatment (BIVV003) in 7 adults with severe sickle cell disease. The goal was to see if the treatment is safe and can help the body make healthy red blood cells. Participants received their own genetically modified stem ce…

This study tested a gene therapy for people with severe sickle cell disease. Doctors took stem cells from the patients, added a working gene to help produce healthy red blood cells, and put the cells back. The goal was to reduce painful crises and other serious complications. Six…

This early-phase study tested whether adding erythropoietin (EPO) to standard hydroxyurea treatment can safely raise hemoglobin levels in adults with sickle cell disease. Seventeen participants with chronic anemia received the combination for 12 weeks. The main goal was to see if…

This study tested whether giving sickle cell patients azathioprine and hydroxyurea for three months before a stem cell transplant from a matched sibling could help prevent graft failure. Twenty adults aged 16 to 60 took part. The goal was to improve disease-free survival and allo…

This study tested a bone marrow transplant from a half-matched (haploidentical) donor in 25 people aged 13 to 40 with severe sickle cell disease. The goal was to replace the faulty blood cells with healthy donor cells using a mild chemotherapy regimen and a drug called cyclophosp…

This study tested a stem cell transplant from closely matched unrelated donors for 8 people with severe sickle cell disease or thalassemia major. The transplant used a special process to remove certain immune cells from the donor cells to reduce the risk of graft-versus-host dise…

This study tested a new oral medicine called NDec in 96 adults with sickle cell disease. The medicine aims to increase hemoglobin levels and reduce painful crises. Participants took capsules twice a week for about a year. The trial compared NDec to placebo and to standard hydroxy…

This study tested a drug called inclacumab in 241 people aged 12 and older with sickle cell disease who often have painful vaso-occlusive crises. The drug works by blocking a protein that helps blood cells stick together, aiming to reduce how often these pain episodes happen. Res…

This early-phase study tested a gene therapy for adults with severe sickle cell disease. Doctors took stem cells from each patient, added a working gene to help produce healthy red blood cells, and gave them back. The main goals were to check safety and see if the treatment could…

This study tested an experimental drug called etavopivat in 53 people with sickle cell disease or thalassemia. The goal was to see if the drug could safely reduce the need for red blood cell transfusions and increase hemoglobin levels. Participants took the drug by mouth, and res…

This study tested an oral medication, epeleuton, in 35 adults with sickle cell disease to understand how the body processes it and whether it can reduce painful blockages in blood vessels (called vaso-occlusive crises). Participants took the drug for 16 weeks while researchers me…

This study tested a bone marrow transplant from a half-matched donor (haploidentical) in 95 children and adults with severe sickle cell disease. The goal was to see if the transplant could help patients live without major complications for two years. The approach used a reduced-i…

This study looked at whether taking vitamin D every day can lower the risk of serious lung problems in children and teens with sickle cell disease. Lung issues like infections and acute chest syndrome are major causes of illness in this group. The study involved 58 participants a…

This study tested a drug called crovalimab in 95 people with sickle cell disease to see if it could prevent painful vaso-occlusive episodes (VOEs), also known as sickle cell crises. Participants received either crovalimab or a placebo alongside their usual treatments. The goal wa…

This study tested whether daily zinc supplements can reduce serious infections in 100 Ugandan children aged 1 to 5 with sickle cell anemia. Half received zinc and half received a placebo, and researchers tracked infections, hospital stays, and side effects. The goal is to find a …

This study tested whether using oxygen at home every night can prevent serious sickle cell complications in pregnant women. The trial included 178 pregnant women with sickle cell disease. The goal was to see if oxygen therapy reduces painful episodes, lung problems, strokes, and …

This study tested a mind-body training program called I-STRONG for teenagers with sickle cell disease who have chronic pain. The program combines gentle movement and mental exercises to help reduce pain and improve daily life. Researchers worked with 45 teens to see if the progra…

This study tested whether using virtual reality hypnosis could lower pain and anxiety in children with sickle cell disease during a painful crisis. Twelve children aged 6 and older participated. Researchers measured pain scores before and after a virtual reality session. The goal…

This study tested a personalized exercise program for 30 older adults with sickle cell disease. The goal was to see if it was safe, doable, and helpful for improving physical health and quality of life. Participants exercised regularly, and researchers tracked how many completed …

This small study looked at whether nitrous oxide (laughing gas) can quickly relieve severe pain in children with sickle cell disease during a vaso-occlusive crisis. Five children aged 8 to 18 who still had high pain after standard treatment received nitrous oxide in the emergency…

This study tested whether guided relaxation and acupuncture can reduce pain and opioid use in 379 adults with sickle cell disease. Participants were randomly assigned to one of these therapies or usual care, and researchers tracked pain levels, sleep, anxiety, and mood over six w…

This study tested whether a medicine called Intravenous Immunoglobulin (IVIG) is safe and works to shorten painful episodes (crises) in people with sickle cell disease. About 300 people aged 6 to 65 took part. The goal was to see if IVIG could reduce how long the pain lasts and t…

This study tested a smartphone-based prayer program called PUSH (Pray Until Something Happens) to help adults with sickle cell disease reduce stress and pain. Forty participants who reported pain and used opioids were included. The goal was to see if adding prayer to standard car…

This study looked at whether a guided relaxation program on a tablet could help adults with sickle cell disease manage pain and stress while in the hospital. Forty-six participants used the program to listen to calming audio-visual recordings. The goal was to see if this simple, …

This study looked at how sickle cell disease affects leg muscle function. Researchers compared 77 people with sickle cell disease to healthy volunteers by measuring muscle strength and fatigue during a short exercise. The goal was to learn if muscle problems contribute to the poo…

This study looked at 70 adults with sickle cell disease to see which lab test—cystatin C or creatinine—better measures kidney function. Participants gave blood and urine samples and had a special dye test to check how well their kidneys work. The goal is to help doctors catch kid…

This study asked over 1,000 emergency doctors in Europe what they would prescribe for a patient with severe belly pain. The patient's race was changed in the story to see if it affected the doctor's choice of pain medicine. The goal was to understand if racial bias plays a role i…

This study looked at how long donated 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 safe vitamin (biotin) and tracked it over time. The goal was to understand why so…

This study surveyed 186 adults with sickle cell disease (SCD) in the U.S. to learn how the COVID-19 pandemic impacted their stress, anxiety, pain, sleep, and healthcare use. Participants completed online questionnaires about their medical history, mental and physical health, and …

This study looked at why people with sickle cell disease get leg ulcers more often than others. Researchers measured blood flow and nerve function in 70 adults with and without ulcers. They also tested blood and wound fluid to find clues about what causes these slow-healing sores…

This study aimed to find the best ways to collect, process, and store umbilical cord blood from newborns with sickle cell disease, sickle cell trait, or no disease. Cord blood is rich in stem cells that might one day be used for treatments like stem cell transplants or gene thera…

This study looked at how often heart iron overload happens in people with thalassemia, sickle cell disease, or myelodysplasia who receive many blood transfusions. Researchers used MRI scans to measure iron levels in the heart and liver of 110 participants. The goal was to better …

This study collected umbilical cord blood from 44 newborns whose mothers carry the sickle cell trait. The goal was to study the cells in the lab and develop better ways to prepare, gene-modify, and preserve them. The research did not involve treating patients directly, but aims t…

This study looked at nearly 1,000 adults with sickle cell anemia to find out how many also develop pulmonary hypertension (high blood pressure in the lungs). Participants had heart ultrasounds and blood tests, then were followed by phone for up to 3 years. The goal was to learn h…

This study looked at whether a special kidney scan (scintigraphy) can find early kidney damage in children and young adults with sickle cell disease. Standard tests often miss these problems until they are advanced. The goal was to improve annual check-ups so that kidney issues c…

This study looked at over 1,700 people with blood cancers or disorders who needed a bone marrow transplant. Researchers compared two strategies: first searching for a matched unrelated donor and then an alternative if needed, versus going straight to an alternative donor. The goa…

This study checked whether etavopivat, a new medicine for sickle cell disease, changes the heart's electrical activity. Thirty-three healthy adults received different doses of the drug, a placebo, or a standard heart medication. The goal was to see if the drug causes any heart rh…

This study looked at what happens in the blood during painful attacks in people with sickle cell disease. Researchers compared blood samples taken during a pain crisis to samples taken when participants were feeling well. The goal was to learn more about the causes of pain and co…

This study followed 94 young people with sickle cell disease as they moved from pediatric to adult medical care. Researchers tracked hospital visits, treatment adherence, and quality of life to learn what makes this transition successful. The goal is to improve programs that help…

This study looked at how adults with sickle cell disease use painkillers at home, including strong opioids and cannabis. Researchers surveyed 257 patients to find out how common addiction is. The goal is to better understand these patterns so doctors can offer better support and …

This study involved 120 children and adults with sickle cell disease to develop a more accurate way to estimate kidney function. Current tests are not designed for sickle cell patients, so researchers are creating a new equation using biological markers. The goal is to improve ho…

This study looked at pregnancy and baby outcomes in 13 women with sickle cell disease who were treated with Adakveo (crizanlizumab) during pregnancy or shortly before. Researchers tracked rates of miscarriage, stillbirth, and birth defects to better understand the drug's safety d…

This early-stage study tested a single dose of a new oral medicine called etavopivat in 24 healthy Chinese volunteers. The main goal was to see how the drug moves through and is processed by the body (pharmacokinetics) and to check its initial safety and tolerability. The results…