SICKLE CELL DISEASE

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 inclacumab in 241 people with sickle cell disease aged 12 and older. The goal was to see if it could reduce the number of painful vaso-occlusive crises (VOCs) over 48 weeks compared to a placebo. Participants received either the drug or a placebo, …

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 were enrolled, and the main goal was to see if at least a 1 g/dL increase in hemoglobi…

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

This early-phase study tested a gene therapy for adults with severe sickle cell disease. Doctors took the patients' own blood stem cells, added a working gene to help produce normal red blood cells, and infused them back. The goal was to see if the treatment is safe and could red…

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 gentle chemotherapy regimen and a drug called cyclopho…

This study tested whether famotidine, a common heartburn medicine, can lower a blood marker linked to painful blockages 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 i…

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 a new oral medicine called NDec in 96 adults with sickle cell disease. The goal was to see if it can safely raise hemoglobin levels and reduce painful crises. Participants took capsules twice a week for about a year, and some also continued their usual hydroxyur…

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 it could improve blood flow and oxygen delivery to the brain and muscles. Participants took the drug for a period, and researchers measured cha…

This early-stage study tested a new treatment called BIVV003 for adults with severe sickle cell disease. The approach uses a person's own blood stem cells, which are gene-edited in a lab and then given back after chemotherapy. The main goals were to see if the treatment is safe a…

This study tested an oral medication called epeleuton in 35 adults with sickle cell disease. The goal was to see how the drug moves through the body, how it affects blood cells, and if it can reduce painful vaso-occlusive crises. Participants took the drug for 16 weeks, and resea…

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 possible lung pressure issues). Fifty children aged 5-18 took part. Researchers measured changes in heart bl…

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

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

This study tested whether daily zinc supplements can lower the number of serious infections in young children (ages 1 to 5) with sickle cell anemia in Uganda. Half of the 100 children received zinc and half received a placebo, and researchers tracked infections over time. The goa…

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 medicine called crovalimab to see if it can prevent painful vaso-occlusive episodes (VOEs) in people with sickle cell disease. 95 participants received either crovalimab or a placebo alongside their usual treatments. The goal was to reduce the number of VOEs r…

This study looked at whether using virtual reality to guide children into a hypnotic state could help reduce pain and anxiety during a sickle cell crisis. Twelve children aged 6 and older participated while hospitalized. Researchers measured pain scores before and after the virtu…

This study tested whether a medicine called Intravenous Immunoglobulin (IVIG) is safe and works for treating sudden, severe pain episodes in people with sickle cell disease. About 300 people took part. The goal was to see if IVIG could shorten the pain crisis and reduce the need …

This study tested a smartphone-based prayer program called PUSH (Pray Until Something Happens) to see if it could help adults with sickle cell disease feel less stress and pain. 40 people who had pain and used opioids took part. They listened to guided prayer recordings on their …

This study tested whether guided relaxation or acupuncture can help reduce chronic pain and opioid use in people with sickle cell disease. About 379 adults with sickle cell disease and chronic pain took part across three hospitals. Participants were randomly assigned to receive g…

This study tested whether a personalized exercise program, adapted from a program called Gerofit, is safe and doable for older adults with sickle cell disease. Thirty participants tried the program to see if it could improve their physical health and quality of life. The focus wa…

This study tested a program called I-STRONG, which combines mental and physical training to help teenagers with sickle cell disease cope with pain. 45 teens took part to see if the program was practical and well-liked. The goal was to reduce pain severity and its impact on daily …

This study tested nitrous oxide (laughing gas) as a pain reliever for children aged 8 to 18 with sickle cell disease who were having a severe pain crisis. The goal was to see if it could quickly lower pain when standard treatments like morphine were not enough. Only 5 participant…

This study tested whether a guided relaxation program on a tablet could help adults with sickle cell disease better manage pain and stress while in the hospital. 46 participants used the program to listen to and view calming scenes. The goal was to see if this simple, drug-free a…

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 normal hemoglobin. Researchers enrolled 307 pregnant women to donate cord blood after delivery. The stored blood may be used in…

This study followed 94 young people with sickle cell disease as they moved from pediatric to adult medical care. Researchers tracked hospital visits, medication adherence, and quality of life to understand what makes this transition successful. The goal is to use these insights t…

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 looked at how sickle cell disease affects leg muscles. Researchers compared muscle strength and fatigue in 77 people with and without sickle cell disease during a short exercise. The goal was to understand if muscle problems contribute to poor physical fitness in sickl…

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 1,753 patients needing a bone marrow transplant for blood cancers or other serious blood disorders. It compared two approaches: first searching for a matched unrelated donor, or going straight to an alternative donor (like a half-matched family member). The g…

This study asked over 1,000 emergency doctors in Europe what they would prescribe for a patient with severe belly pain. The goal was to see if a patient's race influences whether doctors give morphine. The study did not involve actual patients, but used a made-up case to uncover …

This study looked at how common iron buildup in the heart is among 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 be…

This study looked at nearly 1,000 adults with sickle cell anemia to find out how many also develop pulmonary hypertension, a condition where blood pressure in the lungs is too high. Participants had heart ultrasounds and blood tests, and were followed by phone for up to 3 years. …

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 study collected umbilical cord blood from 44 newborns whose mothers had sickle cell trait or disease. The goal was to study the cells in the lab, learn more about the disease, and develop ways to prepare and gene-modify these cells for future treatments. No treatment was giv…

This study looked at whether a special kidney scan (scintigraphy) can find kidney problems earlier than standard tests in children and young adults with sickle cell disease. 18 participants were checked during their annual visit. The goal is to prevent serious kidney damage later…

This study tested a new medicine called etavopivat, being developed for sickle cell disease, to see if it changes the heart's electrical activity. Thirty-three healthy adults received different doses of the drug, a placebo, or an approved heart medicine. The goal was to check for…

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

This study looked at why people with sickle cell disease get leg ulcers. Researchers measured blood flow and nerve function in 70 adults with and without ulcers. They also checked blood and wound fluid for clues. The goal was to better understand the problem, not to test a new tr…

This study looked at what happens in the blood of people with sickle cell disease during painful attacks. 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 these painf…

This study looked at 70 adults with sickle cell disease to find the most accurate way to measure kidney function. Participants gave blood and urine samples and had special imaging tests. The goal was to see if a newer blood test (cystatin C) is better than the standard one (creat…

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…