Bone Marrow Transplant for Sickle Cell Disease

This trial examines how a bone marrow transplant from a mismatched donor might benefit individuals with severe sickle cell disease and other blood disorders requiring regular transfusions. Known as a T-Cell Depleted Alternative Donor Bone Marrow Transplant, this approach aims to increase transplant accessibility by using donors who are not a perfect match. The study also seeks to reduce complications such as graft-versus-host disease, where donor cells attack the recipient's body. Suitable candidates include those with sickle cell disease experiencing frequent pain episodes or chest problems, or those dependent on regular blood transfusions. As a Phase 1, Phase 2 trial, this research focuses on understanding the treatment's function in people and measuring its effectiveness in an initial, smaller group.

The trial information does not specify if you need to stop taking your current medications. However, it does mention that hydroxyurea must have been tried and failed for patients with sickle cell disease, which might imply some changes in medication. It's best to discuss your specific medications with the trial team.

Previous studies have shown promise for bone marrow transplants from alternative donors, with certain immune cells (T-cells) removed, in patients with severe sickle cell disease. Research indicates that this method, which uses a specific type of donor stem cell, is generally well-tolerated. Removing T-cells reduces the risk of complications like graft-versus-host disease, where donor cells attack the recipient’s body.

One study found that this T-cell depleted approach led to successful engraftment, meaning the donor cells started to grow and produce new blood cells without severe complications in patients with sickle cell disease. Other studies have reported that patients did not experience serious issues like sinusoidal obstruction syndrome (a liver condition) or veno-occlusive disease (a type of blood vessel blockage in the liver).

Unlike the standard treatments for sickle cell disease, such as hydroxyurea or regular blood transfusions, this approach uses a T-cell depleted alternative donor bone marrow transplant. Researchers are excited because this method specifically targets and depletes CD3+/CD19+ cells, reducing the risk of graft-versus-host disease, which is a common complication of transplants. Additionally, by using HLA mismatched or haploidentical donors, it increases the availability of potential donors, making transplants more accessible for patients. This innovative technique aims to provide a more effective and safer option for those with sickle cell disease, offering hope for improved outcomes.

Research has shown that using specially prepared stem cells from partially matched donors can help treat severe sickle cell disease. In this trial, participants will receive a T-Cell Depleted Alternative Donor Bone Marrow Transplant. Earlier studies demonstrated that patients who received these transplants successfully had the new stem cells start working in their bodies. This method reduces the risk of a common problem where the new stem cells attack the body. Early research indicates that this approach can work even for patients who typically have fewer donor options.¹²³⁴⁵