Sickle cell disease – treatment

Blood and bone marrow transplants are a potential curative treatment for some people with sickle cell disease. A successful transplant requires a suitable donor, usually a close relative.

Another name for blood and bone marrow transplant is hematopoietic stem cell transplant. The person receiving a transplant is often called a “recipient.” The person who donates cells for transplantation is called a “donor.”

Human leukocyte antigen (HLA) testing helps assess donor and recipient compatibility. HLA plays an important role in the body's immune response to foreign substances. They constitute a person's tissue type and vary from person to person.

Currently, most blood and bone marrow transplants are performed on children who have suffered complications such as stroke, acute chest attacks, or recurrent pain attacks. For adults, transplants are riskier. Some people may undergo a transplant if medicines do not work well to prevent the development of severe pain.

Scientists are researching new ways to help more people with sickle cell disease receive transplants. These methods include blood and bone marrow transplant techniques when there is no compatible donor in the family or in children and adults who are older than most recipients.

Blood and bone marrow transplants are successful in about 90% of children when the donor is related and HLA-matched.. Even with such a high success rate, there are still risks to undergoing a transplant. Although healthcare providers take many steps to prevent transplant complications, they can still occur.

Possible complications include:

• Graft-versus-host disease. It is caused when transplanted cells attack the recipient's organs.
• Increased risk of some cancers later in life
• infertility
• seizure
• serious infection
• Transplant failure causing sickle cell disease recurrence

Approximately 5% of recipients under 16 years of age die from transplant surgery. Approximately 9% of recipients over the age of 16 die after transplantation. For some recipients, the transplant does not cure the long-term pain associated with sickle cell disease. Transplants cannot always repair all the damage caused by sickle cell disease.

To prepare donors for blood and bone marrow transplants, donors administer “mobilization” drugs to increase the number of donors.

stem cells

In their bloodstream. Next, the donor collects stem cells from the donor's blood.

To make room for the donor's cells in the recipient's bone marrow, health care providers give the recipient “conditioning” chemotherapy drugs or use radiation therapy. The donor then injects the donor's cells into the recipient's blood. The cells travel in the bloodstream to the marrow cavity inside the bone. Once in the bone marrow, the cells produce healthy red blood cells with normal, non-sickle hemoglobin.

Because transplantation can affect future fertility, prospective transplant recipients should discuss fertility preservation options with their health care provider before transplantation.