Is a bone marrow transplant effective in treating sickle cell?

A bone marrow transplant (BMT) is currently the sole known cure for sickle cell disease (SCD), a group of genetic conditions that affect hemoglobin in red blood cells. Hemoglobin enables red blood cells to carry oxygen throughout the body, but in SCD, a problem with hemoglobin causes the cells to take on a sickle shape and become sticky. A BMT involves receiving healthy bone marrow tissue from a suitable donor, which contains stem cells that can produce healthy red blood cells. However, this rigorous procedure may not be suitable for everyone and carries potential risks, including graft-versus-host disease, graft failure, low blood counts, infections, nutritional problems, infertility, and social and emotional concerns.

Although a BMT is the only cure for SCD, not everyone is a candidate for the procedure. Individuals with severe SCD who experience serious symptoms, are at risk of complications, and cannot control their symptoms with other treatments may be considered. Before undergoing a BMT, it is important to consider the potential risks and side effects associated with the therapy. Additionally, a matched donor of healthy stem cells, typically from a full sibling, is required for the transplant.

Other treatments for managing SCD include medications like voxelotor, crizanlizumab-tmca, and hydroxyurea, which can help reduce symptoms but may have side effects. Blood transfusions can also be used to manage the condition and prevent complications, but they can carry risks such as infections, iron overload, and transfusion reactions. In 2023, the Food and Drug Administration (FDA) approved cell-based gene therapies, Casgevy and Lyfgenia, which use a person’s own modified stem cells to produce functioning red blood cells. Although a BMT remains the only cure for SCD, these alternative treatments provide potential options for managing the condition.

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