The Memorial Sloan Kettering Cancer Center is sponsoring a study investigating the merits of immunosuppressive therapy in individuals with sickle cell disease (SCD) or β-thalassemia receiving an allogeneic hematopoietic cell transplantation (HCT).
HCT can lead to severe adverse effects, such as graft failure or graft vs host disease. Scientists have proposed administering immunosuppressive drugs, such as fludarabine and dexamethasone, as prophylactic therapy to lower the risk of an overactive immune system compromising the health status of patients receiving HCT.
However, scientists first need to know how the body of participants will respond to these medications, and whether another drug, such as cyclophosphamide or mycophenolate mofetil, may be more effective.
The research team is hence conducting a study to investigate the efficacy of immunosuppressive drugs in increasing the safety profile of patients with SCD/β-thalassemia who receive HCT. This clinical trial is currently still at the recruitment phase. Researchers hope to enroll 24 participants aged 2 to 50 years for the interventional phase 2 study.
Participants must have a diagnosis of SCD/β-thalassemia and demonstrate signs of active disease. They must also have adequate function of all major organs. The exclusion criteria includes having a prior myeloablative allogeneic HCT, a malignancy diagnosis (unless in remission), or an inability to commit to medical therapy/follow-up.
Read more about SCD etiology
Different immunosuppressive drugs will be administered at various predetermined dates. For example, dexamethasone will be administered prior to surgery, while cyclophosphamide will be administered after surgery.
The primary and only outcome measure of this study is the percentage of patients with treatment-related mortality or primary graft failure within the 1st year of undergoing HCT. The study has an estimated completion date of February 9, 2026.
A study of immune suppression treatment for people with sickle cell disease or β-thalassemia who are going to receive an allogeneic hematopoietic cell transplantation (HCT). ClinicalTrials.gov. February 21, 2023. Accessed February 24, 2023.