What the trial was testing
The BMTCTN1507 enrolled 95 patients with sickle cell disease. The study was sponsored by Medical College of Wisconsin and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Pain crises dropped from an average of 5.6 events in the two years before transplant to 0.9 events in the two years after.
Blood advances · 2023 · NCT03263559
These findings — that patients had far fewer painful sickle cell crises after receiving donor stem cell transplants — were published in the Blood advances and represent the headline result of the study.
Researchers tracked outcomes across 95 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with sickle cell disease, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Stem cell transplants from matched donors are FDA-approved for sickle cell disease and can dramatically reduce pain crises. This is a major procedure with risks, so it's typically reserved for severe cases. Talk to your doctor about whether you're a candidate and what newer gene therapy options might also be available.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open sickle cell disease trials
Study of Vitamin B12 Metabolism in Children With Sickle Cell Disease Exposed to MEOPA
Short description of the protocol intended for the lay public. Include a brief statement of the study hypothesis (Limit : 5000 characters) The sickle cells anemia is a monogenic disease linked to the presence of Hemoglobin S due to a mutation in the Hemoglobin Beta chain. The lack of circulating oxygen induces a polymerization of the Hemoglobin S which change the red cell conformation into sickle. Those cells interact and causes vaso-occlusive crisis (CVO). The MEOPA is a medical gas used as an antalgic and a sedative especially in sickle cells disease patients. The nitrous oxide, oxide the cobalt ion in the vitamin B12 which inactivate it irreversibly creating a functional deficiency. During the metabolism of vitamin B12, homocysteine is transformed in methionine which is used in to form the myelin sheath and helped in producing DNA. Numerous studies already shown that the longer the exposition to MEOPA is the greater the functional deficiency of vitamin B12 occur. A few studies shown a symptomatic deficiency of vitamin B12 due to the exposition of MEOPA in sickle cells patient but there is no explanation on the necessary amount of exposure or if some patients are more at risk. When there is a deficiency of vitamin B12 the symptoms can go from a simple orthostatic hypotension to a combined spinal sclerosis. The participation to the study will be proposed to every patient hospitalized for a CVO in the follow up of the emergency room visit or directly in pediatric reanimation. During a usual blood test, a small amount of blood (4mL) will be collected in addition to dose the Vitamin B12, the vitamin B9, the homocysteine, and the methionine. A small amount of urine will also be collected to dose the methylmalonic acid, all those elements are a part of the metabolism of B12 vitamin. The same sample will be taken on the day of departure of the hospital. During the hospitalization the pain management, a daily neurological exam, and the exposition to the MEOPA will be assessed meticulously. An appointment will take place at 7 days and at one month after the hospital departure to evaluate the possible neurological defect. Each patient can only be included once.
Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT)
The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT.