What the trial was testing
The trial enrolled 382 patients with sickle cell disease. The study was sponsored by St. Jude Children's Research Hospital 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
Patients improved their medication adherence from 40% to 56% of days covered.
Blood advances · 2023 · NCT04080167
These findings — that patients took their hydroxyurea more consistently after using the app — were published in the Blood advances and represent the headline result of the study.
Researchers tracked outcomes across 382 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
Hydroxyurea is an FDA-approved pill that reduces sickle cell complications, but many patients struggle to take it daily. This study showed that a medication-tracking app can help, though the app itself is not yet widely available. Talk to your sickle cell specialist about tools and strategies to help you stay on hydroxyurea consistently.
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
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical or MUD allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.
A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.