What the trial was testing
The BEYOND enrolled 145 patients with thalassemia. The study was sponsored by Celgene 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
77% of patients on luspatercept saw their hemoglobin rise by at least 1 g/dL over 12 weeks.
The Lancet. Haematology · 2022 · NCT03342404
These findings — that had meaningful increases in hemoglobin levels compared to none on placebo — were published in the The Lancet. Haematology and represent the headline result of the study.
Researchers tracked outcomes across 145 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 thalassemia, 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
Luspatercept is now FDA-approved for certain types of beta-thalassemia. If you have non-transfusion-dependent beta-thalassemia with low hemoglobin, talk to your hematologist about whether luspatercept might help reduce your symptoms and improve your blood counts.
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 thalassemia trials
Autologous Testicular Tissue Transplantation
Freezing testicular tissue of prepubertal boys is a method for preserving spermatogonial stem cells in case of imminent gonadotoxic treatment during childhood. In case of total azoospermia or absence of spermatozoa that can be used for intra-cytoplasmic injection (ICSI) in adulthood, the investigators intend to perform the first in men autologous testicular tissue transplantation to restore fertility.
Study of the Role of Genetic Modifiers in Hemoglobinopathies
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).