A bone marrow transplant is a treatment that can potentially cure sickle cell disease. It replaces the blood-forming stem cells that produce sickled red blood cells with healthy stem cells from a donor.

However, a transplant is a major procedure with serious risks, and not everyone with sickle cell disease is eligible. This article explains how transplantation works, its potential benefits and risks, who may be considered, and the barriers that can affect access.

What Is a Bone Marrow Transplant?

A bone marrow transplant is also called a hematopoietic stem cell transplant (HSCT). The healthy blood-forming stem cells used for a transplant may come from a donor’s bone marrow, bloodstream, or stored umbilical cord blood. Your transplant team considers the donor and stem cell source when determining the best approach for you. The donor can also be a related or unrelated donor, with the best recommendation for using a matched sibling donor when available.

🗳️ What matters most to you when discussing a bone marrow transplant with your care team?

Understanding the potential benefits

Understanding the possible risks

Knowing what recovery may involve

Knowing what other options are available

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The transplant process starts by finding a donor whose tissue type closely matches yours, which can lower the risk of complications. Before receiving the donor cells, you receive treatment to prepare your bone marrow and immune system for the transplant. This may include chemotherapy, medicines that suppress the immune system, and sometimes radiation, depending on the transplant plan.

The healthy donor stem cells are then given through a vein, similar to a blood transfusion.

After the infusion, the donor stem cells travel to the bone marrow and begin making new blood cells including normal hemoglobin, a process called engraftment. You are hospitalized for this process, since you will need close monitoring because your immune system is weakened and complications such as infections can occur. During recovery, your healthcare team will regularly check your blood counts, including your red blood cells, white blood cells, and platelets.

Risks and Side Effects

Bone marrow transplant can cause short-term side effects from the treatment (such as chemotherapy or radiation) used before the transplant. These may include nausea, fatigue, mouth sores, hair loss, and low blood cell counts. A weakened immune system also increases the risk of serious infections during the early recovery period and is the reason you are hospitalized for the transplant procedure.

Serious complications can occur if the donor cells do not successfully settle in the bone marrow, known as graft failure. Another risk is graft-versus-host disease (GVHD), which occurs when the donor’s immune cells attack the recipient’s tissues. GVHD can affect areas such as the skin, digestive system, and liver and may become a long-term condition.

Some transplant complications can appear months or years after treatment. These may include infertility, organ damage, and hormonal problems. The likelihood of these complications varies based on factors such as age, overall health, donor type, and type of transplant treatment.

Who May Be Considered for Treatment

People with more severe sickle cell disease may be considered for a bone marrow transplant, especially when they have serious or frequent complications. Doctors may also consider how well standard treatments are controlling the disease and whether complications continue despite treatment.

Bone marrow transplants have historically been performed more often in children with severe sickle cell disease. However, newer transplant approaches have made the treatment a viable option for adults as well.

Donor availability is also important because finding a suitable donor can influence whether transplantation is an option and the risks involved. When a matched sibling donor is available, the compatibility generally lowers the risk of complications and is associated with excellent transplant outcomes.

Insurance and Cost

The cost of a bone marrow transplant can be substantial and may include testing, hospitalization, medications, and follow-up care. Before treatment, contact your insurance provider and transplant center to confirm coverage and expected out-of-pocket costs.

A case manager or patient navigator can help you explore payment assistance and other resources that may reduce financial strain.

What Happens After a Bone Marrow Transplant?

Recovery from a bone marrow transplant does not end when you leave the hospital. Your transplant team will continue checking your blood counts as your bone marrow and immune system recover. You may also need medications to prevent infections or other transplant complications.

Follow-up visits allow your care team to make sure the donor cells continue working properly and to watch for complications such as infection, graft failure, or graft-versus-host disease (GVHD). Even though you may feel very well after transplant, it is important to keep following with the medical team to make sure screening and close follow up is occuring.

Some problems can develop later in recovery, so staying connected with your transplant and sickle cell care teams remains important even when you are feeling well. Your healthcare team may monitor your heart, lungs, liver, and other areas that can be affected by sickle cell disease or transplant treatment.