At present, a bone marrow transplant is the only cure available for some people living with sickle cell disease. However, it is an intensive treatment that carries a number of risks.

Sickle cell disease (SCD) describes a group of genetic conditions that affect hemoglobin. This molecule is present in red blood cells (RBCs) and enables RBCs to deliver oxygen throughout the body. In SCD, a problem with hemoglobin means that RBCs have a sickle shape and are sticky. This means they cannot function correctly, which can affect the body in multiple ways.

Currently, a bone marrow transplant (BMT) is the only cure for SCD. Bone marrow is a soft, spongey tissue that contains stem cells.

In people without SCD, stem cells can help create a variety of cells, including RBCs. As such, after a transplant, a person may be able to produce healthy RBCs to replace the sickle cells. However, a BMT can be an intensive procedure, and recovery may take a long time.

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Yes, a BMT is currently the only known cure available for some people living with SCD.

The American Society of Hematology (ASH) notes that a BMT, also known as hematopoietic stem cell transplantation, is currently the only established treatment that can restore the healthy production of blood cells, such as RBCs.

However, a BMT is a rigorous procedure and may not be suitable for everyone.

A BMT, which some people may also call a stem cell transplant, is a major medical procedure where a person receives healthy bone marrow tissue from a suitable donor. Bone marrow is the spongey tissue inside bones. This tissue contains stem cells, which have the potential to become a variety of cells, including RBCs.

A doctor performs a BMT in a hospital. Typically, a person will need to stay in the hospital for a couple of weeks before the transplant to prepare. They will also receive special medications and treatments to help prepare their immune system for the procedure.

A person receives the stem cells through an intravenous catheter. The stem cells then travel through the blood to the bone marrow, where they begin to produce healthy blood cells.

A healthcare professional will monitor a person’s recovery after a BMT for up to a year or more. After a BMT, a healthcare team will frequently check a person’s blood counts to ensure new blood cells are growing in their bone marrow. Before a person can leave the hospital, they will receive instructions on how to prevent infections and other possible complications.

A BMT cures SCD by replacing unhealthy blood-forming cells with healthy immature stem cells from a suitable donor. After a person receives the stem cells intravenously, the stem cells travel to the bone marrow where they begin to produce healthy and functioning RBCs.

Typically, for SCD, a person will receive an allogeneic BMT. The term “allogeneic” refers to the stem cells coming from another person. These stem cells can come from a family member, an unrelated donor, or umbilical cord blood.

A person will initially receive treatment, such as chemotherapy, to help kill the cells producing the faulty RBCs. These treatments will also weaken the immune system, which will help prevent the body from rejecting the donor cells.

They will then receive the stem cells through an intravenous tube, similar to a blood transfusion. After a person receives the stem cells, they travel to the bone marrow and replace the unhealthy blood-forming cells. The stem cells then start producing healthy RBCs.

Although BMT currently remains the only cure for SCD, it is not a suitable therapy for everyone. This is typically due to the potential risks of the procedure. As such, a doctor may only recommend it for individuals with severe SCD who experience serious symptoms of the condition.

A person may be suitable for a BMT if they meet the following criteria:

  • They are at risk of severe complications of SCD, which could be fatal.
  • They cannot control SCD with pain management or other treatments.
  • They are often in a hospital to manage the condition.
  • SCD negatively affects their quality of life.

Additionally, before receiving a BMT, it is important for a person to consider all the potential risks of the therapy. For example, the procedure can cause a number of side effects, and having a BMT can be a long and challenging process.

Additionally, before receiving a BMT, a person must have a matched donor of healthy stem cells. Typically, this will be from a full sibling. The sibling must also agree to donate their stem cells.

Potential risks of a BMT can include the following:

  • Graft-versus-host disease (GVHD): GVHD is a serious and potentially life threatening complication of a BMT. It occurs when the donor cells attack the recipient’s healthy cells, which can cause a range of health issues. This is why it is important to match donors before the procedure.
  • Graft failure: If the procedure is unsuccessful, a person will be unable to produce healthy RBCs. This will require a repeat of the procedure.
  • Low blood counts: After chemotherapy and before the stem cells take effect, a person may require blood transfusions to prevent their blood counts from becoming too low.
  • Infections: Preparation for a BMT will involve weakening the immune system. As such, this puts a person at a higher risk of infections.
  • Nutritional problems: Digestive organs, such as the stomach and intestines, are sensitive to chemotherapy. This means a person may experience nausea, vomiting, diarrhea, mouth sores, and a loss of appetite, which could prevent them from receiving sufficient nutrients and lead to malnutrition.
  • Infertility: Many people who receive a BMT may have issues with fertility. This may be due to a complication of chemotherapy. However, a person may be able to collect and store sperm or eggs prior to therapy.
  • Social and emotional concerns: Recovery from a BMT can be lengthy and challenging for both the recipient and their loved ones.

Currently, a BMT is the only cure available for those living with SCD. However, other treatments, such as medications and transfusions, can help manage symptoms.


Medications that a person may receive can include:

  • Voxelotor: Voxelotor is an oral medication that helps prevent the sickle shape and binding together of RBCs. This may help reduce the destruction of RBCs, which improves blood flow and reduces the risk of anemia.
  • Crizanlizumab-tmca: This intravenous medication can help prevent RBCs from sticking to the walls of blood vessels. As such, this can help stop blood flow blockage, inflammation, and possible complications, such as sickle cell pain crises. It is available under the brand name Adakveo.
  • Hydroxyurea: This oral medication can help prevent or reduce several complications of SCD. This can include preventing symptoms of anemia, reducing episodes of pain crises and acute chest syndrome, and reducing the incidence of dactylitis. It is available under the brand name Hydrea.

However, while these medications can help reduce SCD symptoms, they can also cause side effects. If a person experiences any adverse reactions, they should discuss them with a doctor.


A person living with SCD may receive blood transfusions to manage the condition and help prevent potential complications. This procedure involves a person receiving blood that contains healthy and functioning RBCs. Doing so can reduce the symptoms of SCD. Blood transfusions can also help prevent stroke and reduce the risk of potential complications during surgeries.

Possible side effects of transfusions can include infections, iron overload, and transfusion reactions.

Gene therapy

In 2023, the Food and Drug Administration (FDA) approved both Casgevy and Lyfgenia. These two treatment options represent the first cell-based gene therapies for treating SCD.

Both treatments use a person’s own blood stem cells. After modifying the stem cells, a person receives them back through an infusion as part of a stem cell transplant. These help the person to produce functioning RBCs.

Read on to learn more about gene therapy for SCD.

Currently, a bone marrow transplant is the only cure available for sickle cell disease. The treatment involves replacing a person’s blood-forming cells with healthy ones from a donor. The healthy donor cells will then start producing healthy and functional red blood cells.

While a bone marrow transplant is currently the only cure, it is not a suitable therapy for everyone. It is an intensive treatment that can have numerous serious side effects.