Sickle Cell FAQs
Question: Can bone marrow transplants cure sickle cell disease?
Is this an effective procedure?
Answer: Bone marrow transplants are not routinely offered to patients with sickle cell disease because this type of treatment is still in the experimental stages. For that reason, many insurers may not cover the cost. Further, it is an expensive procedure that can run into the hundreds of thousands of dollars. Not only is it an expensive procedure, but the success rate varies and is dependent on multiple factors. There are several factors to consider when evaluating a person as a candidate for a sickle cell bone marrow transplant:
- 1.) Is there a donor available who has a full immunologic match with the recipient?
- Finding a donor with a full immunologic match offers the highest probability of success. Usually, brothers and sisters without sickle cell disease are the ideal candidates to donate bone marrow. If a full immunologic match is unavailable, the chances of success are dramatically decreased; such a compromised bone marrow transplant may result in graft rejection or graft versus host disease (an immune system response to the transplant in which the body rejects donated bone marrow cells and may result in other organ damage). This complication can result in the bone marrow recipient still having sickle cell disease, having severe damage done to his/her body, or in extreme cases, death.
- 2.) What is the health status of the recipient?
- In order to consider a bone marrow transplant, the person must have severe sickle cell disease but be in relatively good physical condition. The treatment used in preparing the person to receive a bone marrow transplant is extremely hard on the body. A person may experience overwhelming infection; failure of the vital organs such as the kidney, liver or lungs; and/or uncontrolled bleeding. Any of these complications may lead to death.
- 3.) Do the potential benefits of the bone marrow transplant outweigh the risks?
- Young children have the best chance of a successful outcome with this treatment, as adults may already have organ damage from their sickle cell disease. Overall, the success rate for bone marrow transplant for sickle cell patients is between 80-85%, with a 5-8% chance of death and a 10-12% risk of still having sickle cell disease post transplant. Also, the long-term consequences for sickle cell patients who receive this treatment are not well known.
There are a number of bone marrow transplant centers in the United States that perform bone marrow transplants for sickle cell disease. If you have further questions about this treatment, please contact the IHTC at info@ihtc.org or 317.871.0011, ext. 225, and we can provide you additional information as well as arrange an evaluation to determine whether bone marrow transplantation is suitable for you.
Question: Does having sickle cell disease prevent pregnancy? If not, how will my baby be affected?
Answer: Having sickle cell disease does not prevent pregnancy. However, if you are taking hydroxyurea (a drug that helps decrease the frequency of painful episodes) for your disease, you should plan your pregnancy with your hematologist. Hydroxyurea treatment should be suspended well before pregnancy, allowing adequate time for the substance to exit your system. Even if you are not taking hydroxyurea, you should consult your hematologist and a high—risk obstetrician. These specialists can advise you on how to best increase your chances of having a complication-free pregnancy, a healthy baby and how to adjust your medications to prevent possible birth defects.
How your baby is affected by your sickle cell disease is dependent on many factors including: the type of sickle cell disease you have, whether your partner has sickle cell disease or is a carrier of the sickle cell trait, and whether your partner has any other defect in his hemoglobin. If you have sickle cell disease and your partner does not, all your children will inherit the sickle cell trait. If both you and your partner have sickle cell disease, all your children will be born with sickle cell disease. If you have sickle cell disease and your partner has the sickle cell trait, then your children have a 50% chance of having either sickle cell disease or carrying the sickle cell trait.
If you are unsure, a simple blood test can determine if you or your partner carry the sickle cell trait or another hemoglobin defect that can affect your offspring. Genetic counseling can also be arranged for you and your partner through the Indiana Hemophilia & Thrombosis Center. Please contact us at info@ihtc.org or 317.871.0000 and ask for the genetics counselor.
Question: What are the symptoms of carrying the sickle cell trait?
Answer: Carrying the sickle cell trait is not the same as having sickle cell disease; in fact, sickle cell trait is not considered a disease. Persons with sickle cell trait may have some blood in their urine and a slight increase in the tendency to experience kidney infections. They may experience some pain if they are at very high altitudes (higher than 10,000 feet) without oxygen assistance. They are not affected while riding in commercial aircraft, as the cabins are pressurized and the oxygen levels are supplemented. These problems are rare in persons with sickle cell trait; those with the trait rarely have trait-related medical problems.
Question: Is treatment with hydroxyurea safe and effective?
Answer: Hydroxyurea has been found to be effective in the majority of sickle cell patients who receive this treatment. Hydroxyurea decreases the frequency of painful episodes, decreases the incidence of acute chest syndrome (a complication of sickle cell disease characterized by changes on chest x–ray, pain, decreased blood counts and sometimes respiratory failure), and reduces or prevents priapism (persistent erection of the penis, accompanied by pain and tenderness). Hydroxyurea is the only drug approved specifically for the treatment of sickle cell disease. Side effects are usually minimal and seem to decrease over time as the patient continues the treatment. The most serious side effects are reversible by decreasing the dose or halting treatment. Your hematologist can provide more specific information about side affects associated with hydroxyurea.
