Cure For Thalassemia

A bone marrow transplant delivers healthy bone marrow stem cells into the patient. It replaces bone marrow that is either not working properly or has been destroyed (ablated) by chemotherapy or radiation.

Transplant - bone marrow; Stem cell transplant; Hematopoietic stem cell transplant; Reduced intensity, nonmyeloablative transplant; Mini transplant; Allogenic bone marrow transplant; Autologous bone marrow transplant; Umbilical cord blood transplant

Bone marrow is the soft, fatty tissue inside your bones. Stem cells are immature cells in the bone marrow. Some stem cells grow into different parts of your blood. These parts are:

• Red blood cells (which carry oxygen to your tissues)
• White blood cells (which fight infection)
• Platelets (which help your blood clot)

In a bone marrow transplant, you will receive healthy stem cells after your own bone marrow has been destroyed.

There are three kinds of bone marrow transplants:

• Autologous bone marrow transplant. "Auto" means "self." Stem cells are taken from the patient before the patient gets chemotherapy or radiation treatment. When chemotherapy or radiation is done, the patient gets their stem cells back. This is called a "rescue" transplant.
• Allogeneic bone marrow transplant. "Allo" means "other." Stem cells come from another person, who is called a donor. Donor stem cells come from the donor’s bone marrow or their blood. Most times, a donor must have the same genetic typing as the patient, so that their blood "matches" the patient’s. Special blood tests will tell whether a possible donor is a good match for the patient. A patient’s brothers and sisters have the highest chance of being a good match, but sometimes parents and children of the patient and other relatives may be matches. Donors who are not related to the patient may be found through national bone marrow registries. These are lists of people who have offered to be donors.
• Umbilical cord blood transplant. Stem cells are taken from an umbilical cord right after delivery of an infant. The stem cells are tested, typed, counted, and frozen until they are needed for a transplant.

Most patients get high doses of chemotherapy, radiation, or both, before the bone marrow transplant. This is called ablative (or myeloablative) treatment. It kills any cancer cells that might remain, and it makes room in the bone marrow for the new stem cells to grow.

Today, some patients are getting less chemotherapy and radiation before their transplant. This is called a reduced intensity (nonmyeloablative) or "mini" transplant.

After the patient gets chemotherapy and radiation, a doctor will do the stem cell transplant. The patient gets the stem cells through a tube called a central venous catheter. The cells go right into the bloodstream.

This delivery of cells is called an infusion. It may take up to several hours. It is not surgery. It is similar to a blood transfusion. The stem cells find their way into the bone marrow, where they may begin reproducing and making healthy new blood cells.

Donors must have minor surgery to collect their bone marrow and stem cells. They will be unconscious and pain-free (under general anesthesia) while their bone marrow is removed from their hip bone.

When receiving stem cells, a patient may have these symptoms:

• Pain
• Chills
• Fever
• Hives
• Chest pain
• Drop in blood pressure
• Shortness of breath
• Nausea
• Flushing
• Headache
• Funny taste in the mouth

Bone marrow or stem cell transplant may be recommended for:

• Certain cancers, such as leukemia, lymphoma, and multiple myeloma
• Illnesses where the bone marrow does not produce the right kind of or enough cells. Some of these are:
  • Sickle cell anemia
  • Aplastic anemia
  • Thalassemia
  • Congenital neutropenia
  • Severe immunodeficiency syndromes
• Rescue transplant to replace bone marrow, when treatment for cancer has destroyed a patient’s bone marrow

All bone marrow transplants have risks. The risk is higher or lower depending on many factors. Some of these factors are:

• What disease you have
• What type of treatment (chemotherapy, radiation) you have before the bone marrow transplant
• How old you are
• How healthy you are when you have your transplant
• How good a match your donor is
• What type of bone marrow transplant patient you are having (autologous, allogeneic, or umbilical cord blood)

Complications you may have are:

• Infections: These may be very serious.
• Bleeding: This can happen in the lungs, the intestines, brain, and any other part of the body.
• Anemia
• Diarrhea, nausea, and vomiting
• Pain
• Severe mucositis (inflammation and soreness) in the mouth, throat, esophagus, and stomach
• Damage to the kidneys, liver, lungs, and heart
• Cataracts
• Early menopause
• Graft failure, which means that the new cells do not settle into the body and start producing stem cells
• Graft-versus-host disease: This is when your donor’s cells attack your own body. In the first few months after the transplant, symptoms may be a skin rash, diarrhea, or abnormal liver tests. Later, symptoms may be dry eyes or mouth, tightness of the skin, scarring in the lungs, chronic diarrhea, and other problems.
• Children who get transplants may have delayed growth.

Your health care provider will ask you about your health record and do a physical exam. You will also have many tests before your treatment begins.

Your doctor or nurse should discuss the emotional stress of having a bone marrow transplant. You may want to meet with a mental health counselor talk. Talking with your family and children to help them understand and prepare is important.

You will need to make plans for when you have the transplant. Items to consider are:

• Household chores
• Care of pets
• Payment of bills
• Bank or financial statements
• Schedules and care for your children
• Insurance coverage
• Arranging medical leave from work
• Advanced care directives

You may need to find housing for yourself or your family near the hospital.

Before the transplant, one or two catheters are inserted into larger blood vessels, usually in the neck, for you to receive treatments, fluids, and sometimes nutrition.

Bone marrow transplant patients usually go to medical centers, or hospitals, that specialize in this treatment. Most times the patient will stay in a bone marrow transplant unit in the center to limit their chance of getting an infection.

Some patients who get autologous transplants do not have to stay in the hospital or medical center. They can get the transplant as an outpatient.

Some patients may have part of their autologous or allogeneic transplant done as an outpatient.

Most patients will be in the hospital for 4 to 6 weeks. During this time, you will be isolated and watched closely because of the increased risk of infection.

While you are in the hospital, these things may happen:

• Close monitoring of your blood and vital signs
• You may have to take antibiotics, antifungal medications, and antivrial medications to prevent or treat infection.
• You may need many blood transfusions.
• You may have to stay in a room where special things are done to prevent infection.
• You may get calories and proteins through an intravenous (IV) line until you can eat on your own, and until your diarrhea, nausea, or mouth sores have cleared up.
• You may have to take medicine to prevent graft-versus-host disease.

How well you do after transplant greatly depends on these things:

• What type of bone marrow transplant you had
• How well your donor’s cells match yours
• What type of cancer or illness you have
• Your age and overall health
• What type of chemotherapy or radiation therapy you had before your transplant
• What kind of complications happened after the transplant
• Your genetic make-up

Possible results of a bone marrow transplant are complete cure of the illness being treated, a partial cure, or death. Death may be caused by complications of the bone marrow transplant or because the transplant did not work to treat the illness.

If the transplant works, you can go back to most of your normal activities as soon as you feel well enough. Most times it takes up to 12 months to recover fully.

Bishop MR, Pavletic SZ. Hematopoietic stem cell transplantation. In: Abeloff MD, Armitage JO, Niederhuber JE, Kastan MB, McKena WG, eds. Clinical Oncology. 4th ed. Philadelphia, Pa: Elsevier Churchill Livingstone; 2008:chap 32.

Vose JM, Pavletic SZ. Hematopoietic stem cell transplantation. In: Goldman L, Ausiello D. Cecil Medicine. 23rd ed. Philadelphia, Pa: Saunders Elsevier; 2007:chap 184.