Benefits and Risks
Compare the different benefits and risks of each treatment.
| Bone Marrow TransplantBone Marrow Transplant | Gene TherapyGene Therapy | Chronic TransfusionsChronic Transfusions | HydroxyureaHydroxyurea | |
|---|---|---|---|---|
| Benefits | ||||
You may be cured, but it might not work for everyone. Some people cannot do these treatments. | Yes, but not for everyone. | Yes, but not for everyone. | ||
You will have less pain, because the treatment helps with pain crises. | Yes, but this treatment can cause pain. | Yes, but this treatment can cause pain. | Yes | Yes, but you can still have a pain crisis. |
It prevents acute chest syndrome (ACS). This is when your sickle cells block blood flow to your lungs. | Yes | Yes | Yes, but you can still get ACS. | Yes, but you can still get ACS. |
After this treatment, you will not need to go to the hospital as often as before for sickle cell problems. | Yes, but you will be in the hospital for transplant. | Yes, but you will be in the hospital for gene therapy. | Yes | Yes |
You will have smaller chance of having a stroke that is caused by sickle cell disease. | Yes | Yes | Yes | |
It can stop or slow organ damage, but it can’t fix damage you already have. | Yes | Yes | Yes, but you can still get new organ damage | Yes |
For this treatment, you do not need a blood donor or a blood-making stem cells donor.It can stop or slow organ damage, but it can’t fix damage you already have. | Yes | Yes | ||
With this treatment, you stop taking sickle cell disease medicine (Example: Hydroxyurea) | Yes | Yes | Yes | |
With this treatment, you need fewer or no blood transfusions. | Yes | Yes, but you need some during gene therapy. | Yes, but you still may need some. | |
| Risks | ||||
Doctors do not know all the risks of this treatment, because it is new. | Yes | |||
This treatment might not work for everyone. Your doctor can talk to you about your options. | Yes | Yes | Yes | |
This is when your body attacks the donor cells. It means treatment did not work. | Yes, but not common. | |||
This is when the donor cells attack your body. GvHD causes health problems that can last a long time. | Yes | |||
Chemotherapy side effects can be hair loss, diarrhea, and other problems. | Yes | Yes | ||
Apheresis has side effects that can be caused by medicine and the collection process. | Yes | Yes | ||
VOD is a problem in your liver that can happen after chemotherapy. | Yes | Yes | ||
Some parts of the treatment cause pain. Your doctors will try to treat and prevent the pain. | Yes | Yes | ||
Some people have a pain crisis when a central line is placed. | Yes | Yes | Yes | |
The treatment can cause cancer, because you will get chemotherapy. | Yes, but not common. | Yes, but not common. | ||
Sickle cell disease medicines have side effects. For example, Hydroxyurea causes myelosuppression, nausea, and more. | Yes | |||
Your immune system cannot fight germs for at least 6 months after treatment. | Yes | Yes | ||
This treatment can damage egg and sperm cells. Some people cannot have children after chemotherapy. | Yes, but fertility preservation is an option. | Yes, but fertility preservation is an option. | ||
Problems from treatment can cause death. This risk is very small and only happens when problems cannot be treated by your doctors. | Yes, but not common. | Yes, but not common. | Yes, but not common. | |
You might feel sad and alone. When you stay in the hospital for many weeks, it can be hard on your mental health. | Yes | Yes | ||
You can have a false positive HIV test. This is when you get an incorrect HIV test result that says you have HIV when, in fact, you do not have HIV in your body. | Yes, if lentivirus used for gene therapy. | |||
You will need medicine to treat problems caused by too much iron in your body. | Yes, but there is medicine to treat it. | |||
Your white blood cells (immune system cells) can attack red blood cells that come from a donor’s blood. It can make it difficult to find blood for transfusions in the future. | Yes | |||
The donor’s red blood cells can breakdown inside your blood. It can cause fever, chills, chest pain, and other problems. | Yes, but not common. | |||
This is when your blood gets thicker and flows more slowly. It can cause a pain crisis. | Yes, but not common. | |||
You can get an infectious disease from blood transfusions. Donor blood is checked for viruses, so this is very rare. | Yes, but very rare. | |||