What the trial was testing
The trial enrolled 33 patients with sickle cell disease. The study was sponsored by Children's Hospital Medical Center, Cincinnati and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
Researchers followed patients through treatment and into recovery, tracking the outcomes that mattered most for the disease being studied.
What the results showed
94% of patients with sickle cell disease had reduced exercise capacity on testing.
Pediatric blood & cancer · 2018 · NCT02410811
These findings — that almost all sickle cell patients in the study showed lower-than-normal ability to exercise — were published in the Pediatric blood & cancer and represent the headline result of the study.
Researchers tracked outcomes across 33 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
This was an early-stage study exploring why people with sickle cell disease often struggle with exercise. The findings suggest heart function testing might help identify who needs closer monitoring. Talk to your doctor if you experience unusual fatigue or shortness breath during activity.
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
Physiology and Pathologies Linked to Human Splenic Function : Direct and Ex-vivo Perfusion Explorations
Human splenic physiology remains poorly understood due to lack of functional exploration. However, through its ability to recognize alterations or modifications in circulating cells and to trigger an innate and adaptive response in response to these anomalies, the spleen plays a central role in several diseases affecting blood cells, directly or indirectly. The analysis of the splenic clearance of abnormal cells during ex-vivo perfusions made it possible to clarify the pathogenesis of malaria and the role of the spleen in the adaptive immune response. The study's investigative team wishes to extend these explorations to other human diseases in which the spleen is involved, and to evaluate the preventive or curative potential of substances that can modify the perception of blood cells by the spleen (e.g. monoclonal antibodies directed against circulating cells, among other options).
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.