SICKLE CELL DISEASE

This study tested whether giving two drugs (azathioprine and hydroxyurea) for three months before a stem cell transplant could help the transplant work better in adults with sickle cell disease. The goal was to help patients' bodies accept the donor cells more completely, potenti…

This early-stage study tested a one-time treatment for adults with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used gene editing to potentially fix the disease-causing problem, and then transplanted the modified cells back. The main goals were …

This early-stage study tested a one-time gene therapy for people with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used a modified virus to insert a healthy gene into them in the lab, and then gave the corrected cells back to the patient. The go…

This study tested a bone marrow transplant procedure for people with severe sickle cell disease who don't have a perfectly matched donor. The approach uses a half-matched family donor and a milder chemotherapy regimen to reduce side effects. Researchers aimed to cure the disease …

This early-stage study tested a potential one-time treatment for adults with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used a modified virus to insert a healthy gene into them, and then gave them back to the patient. The goal was to see if th…

This study tested whether adding a hormone called erythropoietin (EPO) to the standard drug hydroxyurea could safely improve chronic anemia in adults with sickle cell disease. The small, early-phase trial involved 17 participants who were already on stable hydroxyurea treatment. …

This study tested whether a potential new medicine for sickle cell disease, called etavopivat, affects the heart's electrical activity in healthy people. It involved 33 healthy volunteers who received different doses of the drug, a placebo, or an approved comparison drug to check…

This study tested whether a nutritional supplement called L-Arginine is safe and can help control a serious heart complication in children with sickle cell disease. It involved 50 children, aged 5 to 18, who already had signs of this heart issue. Researchers gave them L-Arginine …

This study tested an oral medication called etavopivat in people with sickle cell disease or thalassemia. The main goals were to see if the drug was safe and if it could help patients need fewer blood transfusions or have higher hemoglobin levels. The trial included 53 participan…

This completed Phase 3 trial tested whether inclacumab, given as an IV infusion every 12 weeks, could reduce the frequency of painful vaso-occlusive crises in people with sickle cell disease. The study involved 241 participants aged 12 and older who had experienced 2-10 painful c…

This early-stage study tested the safety and effects of an experimental oral medication called FTX-6058 in adults with sickle cell disease. Forty-five participants took the daily pill for 12 weeks while researchers monitored for side effects and measured how the drug affected the…

This study tested whether giving pregnant women with sickle cell disease extra oxygen at home could prevent serious pain episodes and other complications for both mother and baby. It was a large, completed Phase 3 trial involving 178 women across France. Researchers compared stan…

This study aimed to find the best strategy for finding bone marrow transplant donors for patients with blood cancers and disorders. It compared whether patients should first search for a perfectly matched unrelated donor or go directly to alternative donors like family members or…

This study tested a new oral medicine called NDec to see if it could help adults with sickle cell disease. The goal was to see if taking NDec capsules twice a week for about a year could improve blood health and reduce painful crises. Participants were randomly assigned to receiv…

This study tested whether taking a daily vitamin D pill could reduce serious lung problems in children and teens with sickle cell disease. Researchers compared daily vitamin D to a larger monthly dose over two years to see which was better at preventing infections, asthma attacks…

This study tested whether famotidine, a safe and widely used heartburn medication, could help children with sickle cell disease. Researchers gave the drug to 30 children for 29 days to see if it reduced a specific marker of blood vessel inflammation linked to painful blockages. T…

This study aimed to find the most accurate blood test to measure kidney function in adults with sickle cell disease. Kidney problems are common and serious in this condition, so early detection is key. Researchers compared a standard test to a newer one, hoping to improve how doc…

This study tested whether giving a daily zinc supplement could help prevent serious infections in young children with sickle cell anemia. One hundred children in Uganda, aged 1 to 5 years, were randomly assigned to receive either zinc or a placebo pill for six months. Researchers…

This study developed and tested a new telehealth program called I-STRONG to help teenagers with sickle cell disease manage chronic pain. The program combines breathing exercises, relaxation techniques, and gentle movement training, and was specifically adapted with input from pat…

This study tested whether a single dose of an immune protein therapy (IVIG) could safely shorten severe pain crises in people with sickle cell disease. About 300 children and adults were given either IVIG or a placebo saline solution during a hospital stay for a pain crisis. Rese…

This study explored whether a virtual reality (VR) session with a hypnotic script could help reduce pain and anxiety for children experiencing a severe sickle cell pain crisis. Researchers observed 12 children over age 6 who were hospitalized for these crises. The goal was to see…

This study tested whether a simple, non-drug approach could help adults with sickle cell disease manage pain and stress while in the hospital. Researchers had 46 patients try a tablet program with guided relaxation videos to see if it was practical and helpful. The goal was to se…

This study tested whether acupuncture and guided relaxation techniques could help people with sickle cell disease manage their chronic pain and reduce their need for opioid painkillers. It involved 379 participants across three major university health systems. The research aimed …

This study tested nitrous oxide (laughing gas) as a potential alternative to repeated opioid painkillers for children and teens experiencing severe sickle cell pain crises. Researchers wanted to see how well and for how long the gas could relieve pain in the emergency room after …

This study tested a personalized exercise program adapted for adults aged 40 and older with sickle cell disease. Researchers wanted to see if the program was practical, safe, and acceptable to participants, and if it could help improve their physical health and quality of life. T…

This study aimed to understand what causes the sudden, severe pain attacks experienced by people with sickle cell disease. Researchers collected blood samples from 99 participants—both people with sickle cell and healthy volunteers—during pain attacks and between attacks. By anal…

This study aimed to find out how common and serious high blood pressure in the lungs (pulmonary hypertension) is for adults with sickle cell disease. Researchers enrolled 986 adults to check their heart health with an ultrasound and then followed up with phone calls for over thre…

This study aimed to understand how the COVID-19 pandemic impacted the daily lives, stress levels, and healthcare use of adults living with sickle cell disease (SCD). Researchers surveyed 186 adults with SCD in the US, asking about their mental health, pain, and access to medical …

This study looked at how emergency room doctors make decisions about prescribing strong pain medication for sickle cell disease patients. Researchers presented 1,063 emergency physicians across Europe with a fictional patient case and asked what treatment they would choose. The g…

This study aimed to create a more accurate kidney function test specifically for people with sickle cell disease. Researchers tested 120 children and adults with sickle cell to compare a new calculation method against the standard one. The goal is to improve how doctors monitor k…

This study aimed to understand how young adults with sickle cell disease move from pediatric to adult healthcare. Researchers followed 94 patients aged 16-21 to track their hospital visits, medication use, and quality of life during this critical transition period. The goal was t…

This study aimed to learn the best methods for collecting, processing, and storing umbilical cord blood from babies. The goal was to preserve the stem cells in this blood for potential future use in advanced treatments for sickle cell disease, such as stem cell transplants or gen…

This early study tested a new medicine called etavopivat, which is being developed for sickle cell disease and thalassemia. It involved 24 healthy Chinese volunteers to see how their bodies process a single dose of the drug and to check for any side effects. The main goal was to …

This study aimed to understand how often dangerous iron builds up in the heart of patients who need frequent blood transfusions for conditions like thalassemia, sickle cell disease, or myelodysplasia. Researchers observed 110 patients across 14 centers, using heart and liver MRI …

This study aimed to understand how adults with sickle cell disease manage their chronic and severe pain. Researchers surveyed 257 patients to learn how often they use strong painkillers (opioids) and cannabis or CBD at home, and to check for signs of addiction. The goal was to ga…

This study aimed to understand why painful, slow-healing leg ulcers are so common in adults with sickle cell disease. Researchers compared 70 adults with and without ulcers, measuring blood flow, nerve function, and substances in the blood and wound fluid. The goal was to learn m…

This study aimed to understand why people with sickle cell disease often have reduced physical fitness. Researchers compared leg muscle strength and fatigue during exercise between 77 sickle cell patients and healthy individuals. The goal was to determine if muscle dysfunction co…

This study collected umbilical cord blood from newborns at risk for sickle cell disease to use in laboratory experiments. Researchers aimed to study these cells to develop future gene therapy methods. The study involved 44 participants and did not provide direct treatment to part…