Bone Marrow Transplant for Sickle Cell Disease
What is Bone Marrow (Stem Cell) Transplant for Sickle Cell Disease?
This procedure involves replacing bone marrow affected by sickle cell anemia with healthy bone marrow from a donor. The procedure usually uses a matched donor, such as a sibling, who doesn't have sickle cell anemia.
Because of the risks associated with a bone marrow transplant, the procedure is recommended only for people, usually children, who have significant symptoms and complications of sickle cell anemia.
The procedure requires a long hospital stay. After the transplant, you'll receive drugs to help prevent rejection of the donated stem cells. Even so, your body might reject the transplant, leading to life-threatening complications.
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What is Sickle Cell Anemia?
Sickle cell anemia is one of a group of disorders known as sickle cell disease. Sickle cell anemia is an inherited red blood cell disorder in which there aren't enough healthy red blood cells to carry oxygen throughout your body.
Normally, the flexible, round red blood cells move easily through blood vessels. In sickle cell anemia, the red blood are shaped like sickles or crescent moons. These rigid, sticky cells can get stuck in small blood vessels, which can slow or block blood flow and oxygen to parts of the body.
What is a Bone Marrow Transplant?
A bone marrow transplant also called a stem cell transplant is a medical procedure performed to replace bone marrow that has been damaged or destroyed by disease, infection, or chemotherapy. This procedure involves transplanting blood stem cells, which travel to the bone marrow where they produce new blood cells and promote growth of new marrow.
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Bone marrow is the spongy, fatty tissue inside your bones. It creates the following parts of the blood:
. Red blood cells, which carry oxygen and nutrients throughout the body
. White blood cells, which fight infection
. Platelets, which are responsible for the formation of clots
Bone marrow also contains immature blood-forming stem cells known as hematopoietic stem cells, or HSCs. Most cells are already differentiated and can only make copies of themselves. However, these stem cells are unspecialized, meaning they have the potential to multiply through cell division and either remain stem cells or differentiate and mature into many different kinds of blood cells. The HSC found in the bone marrow will make new blood cells throughout your lifespan.
A bone marrow transplant replaces your damaged stem cells with healthy cells. This helps your body make enough white blood cells, platelets, or red blood cells to avoid infections, bleeding disorders, or anemia.
Healthy stem cells can come from a donor, or they can come from your own body. In such cases, stem cells can be harvested, or grown, before you start chemotherapy or radiation treatment. Those healthy cells are then stored and used in transplantation.
What is Allogeneic Stem Cell Transplant?
An allogeneic stem cell transplant uses healthy blood stem cells from a donor to replace your diseased or damaged bone marrow. An allogeneic stem cell transplant is also called an allogeneic bone marrow transplant. The donor must be a close genetic match. Often, a compatible relative is the best choice, but genetic matches can also be found from a donor registry.
Allogeneic transplants are necessary if you have a condition that has damaged your bone marrow cells. However, they have a higher risk of certain complications, such as GVHD. You’ll also probably need to be put on medications to suppress your immune system so that your body doesn’t attack the new cells. This can leave you susceptible to illness.
The success of an allogeneic transplant depends on how closely the donor cells match your own.
About Iranian Surgery
Iranian surgery is an online medical tourism platform where you can find the best Bone Marrow Transplant Surgeons in Iran. The price of a Bone Marrow Transplant surgery in Iran can vary according to each individual’s case and will be determined based on an in-person assessment with the doctor.
For more information about the cost of Bone Marrow Transplant in Iran and to schedule an appointment in advance, you can contact Iranian Surgery consultants via WhatsApp number 0098 901 929 0946. This service is completely free.
Before Bone Marrow Transplant for Sickle Cell Disease
Why it is done
Stem cell transplants offer a potential cure for a child's sickle cell disease. They are usually considered only for children younger than 16 who have:
. Severe sickle cell disease complications, including repeat strokes, episodes of acute chest syndrome, and painful events.
. An available donor (someone who has closely matching bone marrow).
The risks of stem cell transplant become greater as a person gets older and/or develops damage to major organs. For these reasons, a bone marrow transplant is not a treatment option for most adults who have sickle cell disease. But research on bone marrow transplants in adults is ongoing.
Is my child a candidate for a bone marrow transplant?
Your child is a candidate if he or she has:
. Complications due to sickle cell disease such as stroke,
. Frequent pain episodes,
. Frequent chest crisis, or
. Abnormal MRI or ultrasound of the brain
And
. Has a full brother or sister who does not have sickle cell disease and
. Who has a closely matching bone marrow.
Not all brothers and sisters of a patient with sickle cell disease are full matches for bone marrow. However, a sibling who has the sickle cell trait may also donate bone marrow.
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Who can donate stem cells for transplants?
There are 3 main types of stem cell donors:
. Matched related – A brother or sister who has the same bone marrow type and the same mother and father.
. Brothers and sisters are matched through special blood tests called HLA typing.
. Matched Unrelated – volunteers who have the same bone marrow type as the patient.
. Usually these types of donations are matched through national organizations that match donors and patients who have the same type of bone marrow.
. Haploidentical – half-matched family members (usually a mother or father)
. This type of donation is still considered experimental and should be performed only as part of a research study.
Stem cells can be obtained from the donor’s bone marrow or peripheral blood (blood in the veins).
Or in some cases, stem cells are collected from the umbilical cord at the time of birth.
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What are the possible risks of bone marrow transplant?
. Infections – Chemotherapy lowers the white blood cells, which normally fight and prevent infections.
. This puts the patient at high risk for infections, which can be caused by bacteria, fungi, or viruses.
. Medicines are given to fight these germs and prevent these infections. Infections that do not respond to the treatment can lead to death in 5 to 10 percent of patients.
. Graft-versus-host disease (GVHD) – This reaction occurs when the immune cells of the donor (graft cells) sense that cells of the patient (host cells) are different and attack them. This can be a serious side effect of transplant.
. GVHD occurs in up to 10 percent of patients who undergo matched related types of transplants. It can be higher in transplants using other donors.
. This condition can be acute (occurring less than 100 days after the transplant) or chronic (occurring more than 100 days after transplant).
. It may cause damage to the skin, liver, and intestinal tract of the transplanted patient.
. Drugs are given to prevent or limit GVHD. These drugs increase the patient’s risk of infection. However, GVHD that does not respond to treatment can lead to organ damage or even death.
. Graft failure – There is about a 10 percent risk that the new bone marrow from a matched family member will fail to take. The chance of graft failure is higher with other types of donors.
. This means that the patient will not be able to make any white blood cells, red blood cells, or platelets. Typically, the transplant would need to be repeated.
. If there are no more donor cells, stem cells collected from the patient before the transplant can be given back to the patient. This restores the patient’s original bone marrow, which means the sickle cell disease comes back.
. Veno-occlusive disease (VOD) – Blood vessels that lead into and pass through the liver may suffer damage after a bone marrow transplant. This may be caused by the chemotherapy and might lead to swelling and severe liver damage.
. The chances of severe VOD are about 1 out of every 20 cases.
. Nutrition problems – The stomach and intestines are sensitive to chemotherapy. Nausea, vomiting, mouth sores, diarrhea, and loss of appetite may occur.
. Typically, nutrition must be given through the veins until patients are able to eat.
. Low blood counts – While waiting for new stem cells to make normal red blood cells as well as all other types of cells, a patient usually needs transfusions of platelets and red blood cells.
. Social and emotional concerns – A bone marrow transplant is challenging to both the patient and family members. The patient’s routine may change for a while because he will be away from home, isolated from school, friends, and relatives.
. The family is the key to supporting the child through the transplant period.
. The average time spent in the hospital is 4 to 6 weeks.
. Infertility – Most patients who receive a transplant will not be able to have their own children in the future. This is one (1) possible side effect of drugs used while preparing for the transplant; however, there have been patients who were able to conceive children after having a transplant.
During Bone Marrow Transplant for Sickle Cell Disease
How Does It Work?
A bone marrow transplant replaces the cells in your body that make red blood cells, called hematopoietic stem cells, with new ones. That means your body will stop making the sickle-shaped cells that cause the disease.
In the procedure, a doctor takes healthy stem cells from the bone marrow of a donor and injects them into your body, usually through an IV tube into one of your veins. Once inside, the cells go to your bone marrow and start creating healthy blood cells.
While that sounds simple, a bone marrow transplant is a long process. Once you have a donor, you’ll spend several weeks in the hospital and have several more months of follow-up care. The process begins before the actual transplant procedure:
. For 1 to 2 weeks before the transplant, you’ll stay in the hospital, and doctors will give you chemotherapy. These powerful medicines destroy the cells that make abnormal blood cells. They also make your immune system weak, so it won’t reject and attack the new stem cells. You might get radiation therapy as well.
. Then, doctors will inject the donor’s cells into your body. The cells should replace the old bone marrow and start making new, healthy blood cells. Your care team will run tests for about a month to make sure the new cells start to work.
. Once doctors can tell that the transplant has worked, you can leave the hospital. It may take 6-12 months or longer before your blood cells and immune system get back to normal. Your doctor will watch your health closely during this time.
After Bone Marrow Transplant for Sickle Cell Disease
Recovery
When the new stem cells enter your body, they travel through your blood to your bone marrow. In time, they multiply and begin to make new, healthy blood cells. This is called engraftment. It usually takes several weeks before the number of blood cells in your body starts to return to normal. In some people, it may take longer.
In the days and weeks after your bone marrow transplant, you'll have blood tests and other tests to monitor your condition. You may need medicine to manage complications, such as nausea and diarrhea.
After your bone marrow transplant, you'll remain under close medical care. If you're experiencing infections or other complications, you may need to stay in the hospital for several days or sometimes longer. Depending on the risk of complications, you'll need to remain near the hospital for several weeks to months to allow close monitoring.
You may also need periodic transfusions of red blood cells and platelets until your bone marrow begins producing enough of those cells on its own.
You may be at greater risk of infections or other complications for months to years after your transplant. You'll have periodic lifelong follow-up appointments with your doctor to monitor for late complications.
After the last session, the doctor will continue to check the blood each day to determine how well the transplant has worked. They will test whether new cells are beginning to grow in bone marrow.
If a person’s white blood cell count starts to rise, it indicates that the body is starting to create its own blood, indicating that the transplant has been successful.
The amount of time that it takes for the body to recover depends on:
. How well the immune system recovers
. How well the body accepts the new, healthy cells
Many other factors can affect recovery, including:
. Underlying medical conditions
. The use of chemotherapy, radiation, or both
. Where the transplant was performed
. The closeness of the donor match
Some people are able to leave the hospital soon after the transplant, while others need to stay for several weeks or months.
The medical team will continue to monitor the person’s recovery for up to 1 year. Some people find that effects of the transplant remain for life.
Medications
If your bone marrow transplant is using stem cells from a donor (allogeneic transplant), your doctors may prescribe medications to help prevent graft-versus-host disease and reduce your immune system's reaction (immunosuppressive medications).
After your transplant, it takes time for your immune system to recover. During this time, you may be given medications to prevent infections.
Diet and other lifestyle factors
After your bone marrow transplant, you may need to adjust your diet to stay healthy and to prevent excessive weight gain. Your nutrition specialist (dietitian) and other members of your transplant team will work with you to create a healthy-eating plan that meets your needs and complements your lifestyle. Your dietitian can also give you food suggestions to control side effects of chemotherapy and radiation, such as nausea.
Some of your dietitian's recommendations may include:
. Following food safety guidelines to prevent foodborne infections
. Eating a wide variety of healthy foods, including vegetables; fruits; whole grains; lean meats, poultry and fish; legumes; and healthy fats, such as olive oil.
. Limiting salt intake
. Restricting alcohol
. Avoiding grapefruit and grapefruit juice due to their effect on a group of immunosuppressive medications (calcineurin inhibitors)
After your bone marrow transplant, regular physical activity helps you control your weight, strengthen your bones, increase your endurance, strengthen your muscles and keep your heart healthy. As you recover, you can slowly increase your physical activity.
Taking steps to prevent cancer is even more important after your transplant. Don't smoke. Wear sunscreen when you're outside, and be sure to get the cancer screenings your doctor recommends.
Result
Results of many studies show that transplants from matched related donors offer about an 85 percent chance of cure. Other treatment choices are the drug hydroxyurea and chronic red blood cell transfusions. These treatments may lessen the complications of sickle cell disease, but they do not cure the disease