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Treatment of Aplastic Anemia

Previously, we reported that Aplastic Anemia treatments could be somewhat optional - drug treatment or bone marrow transplant, but that isn't exactly correct. According to the Blood Journal, Very Severe Aplastic Anemia should only be treated with a bone marrow transplant unless there is no matching donor and no closely-matching donor.

Victoria was diagnosed with Very Severe Aplastic Anemia. See Diagnosis of Aplastic Anemia.

Both drug treatment alone and the bone marrow transplant treatment will necessarily require immunosuppression.

bone marrow transplant
Blood Stem Cell Transplant

Bone Marrow Transplant and Blood Stem Cell Transplant are often used as interchangeable terminology. A Bone Marrow Transplant refers to the process of harvesting bone marrow and transplanting it usually into anther person's blood stream. A Blood Stem Cell Transplants means the same thing in general conversation, but more specifically in medical conversation, it refers to transplanting only the blood stem cells that were harvested; the rest of the marrow (red cells, white cells, t-cells, etc. are removed from the harvested marrow).

Origin of harvested cells

Bone marrow transplants can be conducted with marrow that is harvested from the patient (autologous bone marrow transplant) or from a donor. Bone Marrow Transplants and Blood Stem Cell Transplants taken from another person are called allogeneic stem cell transplants and are the most common type of transplants. Blood stem cells from an umbilical cord can also used (see Umbilical cord transplants).

Because the marrow of a person with Aplastic Anemia is diseased, an autologous bone marrow transplant is not possible. Autologous bone marrow transplants can be used for patients with Leukemia and no other marrow disease.

Donor matching

Donors are matched through a Human Leukocyte Antigen Test, often referred to as HLA typing, which assesses proteins on the surface of blood cells. It is a DNA-based tissue typing test. Four of six major factors must match: HLA-A, HLA-B, HLA-C and DRB1. For more information about the HLA Locus tissue type matching process, please defer to Marrow.org.

To conduct an HLA tissue typing test, blood is drawn from both the potential recipient of the transplanted cells and the potential donor or donors.

Bone Marrow Harvesting

The process of obtaining the bone marrow containing the blood stem cells from the donor is referred to as a harvesting.

The donor is admitted an an outpatient to the hospital, prepared for the surgical room, and placed under regular anesthesia.

During a bone marrow harvesting, marrow is extracted from the donor's bone marrow through a process similar to that performed during a bone marrow biopsy except that a single bone marrow biopsy may involve some 2-10 small holes into the bone, and a harvest necessary involves many more (approximately 25 small holes into each hip bone at or near the "dimple").

Donor symptoms post-harvest

While it is true that the actual donation process to give marrow is painless, the after effects are not completely painless. Most donors will experience at least some discomfort for up to three months. Some donors will experience painful side effects in the hips. A lessor number of donors will have other side effects including shooting pains down the leg, numbness and pain in the hips when hips are exposed to cold (such as in cold weather areas).

the bone marrow transplant

The donor's bone marrow is filtered to remove bone fragments, and fed into the recipient's blood stream through an IV. Blood stem cells make their way into the recipient's bone marrow. An example is available at: NLM.NIH.Gov.

Bone marrow transplants aren't commonplace, by any means, but many more such procedures are undertaken nowadays than previously.

prior to bone marrow transplant

Prior to the transplant procedure, the recipient patient undergoes a very rigorous treatment procedure including chemotherapy and ATG.

Chemotherapy

Chemotherapy for Aplastic Anemia can include a variety of chemotherapy drugs depending upon the regime selected by the bone marrow transplant team at your medical facility. There are some chemotherapy drugs that have produced harmful effects and death; research should be done on the types of drugs being prescribed by your doctors to determine if they are amongst the listed drugs.

At Froedtert, they used Cytoxan and Methotrexate (also known as MTX or FSK56).

See Cancer.org for information about how these drugs work.

ATG

Anti-thymocyte globulin ATG is a serum developed from horse or rabbit antibodies and used to prevent rejection of transplanted bone marrow or blood stem cells (as well as in organ transplants of other types), and in immunosuppression therapy for Aplastic Anemia when drug therapy alone is sought.

steroids

Full-body Radiation

If the donor and recipient are unrelated, total body radiation will also be administered.

Once the treatment process begins, treatment is irreversible and the patient must receive a bone marrow transplant.

engraftment

Within 3-4 weeks post transplant, new blood stem cells should appear in her bone marrow and begin producing sufficient blood cells to sustain her life. See also: Engraftment.

In some situations, transplants are only partially effective, and while the blood levels may not return to normal levels in those cases, they are high enough to sustain a normal life.

Immunosuppression Therapy

While a patient may opt to undergo immunosuppression therapy instead of a bone marrow transplant, bone marrow transplants are highly recommended for young people; conversely, people who are older (sometimes as young as 30 years old) may be recommended into an immune suppressing treatment program for numerous reasons (age among them).

Drug therapy is also used when a donor cannot be found.

Drug therapy, also called immunosuppression treatment, uses drugs to suppress the immune system. The typical drugs are listed at the American Cancer Society.

Immunosuppression renders the patient susceptible to viral, bacterial and fungal infections. Immunosuppression also weakens the body's entire defense system. The relapse rate for treatment restricted to only immune suppression is 70-80%. In most cases, relapses render the body incapable of receiving a bone marrow transplant at a later time when the immune suppression ceases to treat the disease.

aplastic anemia Information:

For more information about Aplastic Anemia, please consider these sites:

Aplastic Anemia Association

One Life Matters BLOG

Marrow.Org

Bone Marrow Donation

Bone Marrow Testing

Blessings

While it is far from a blessing that Victoria has been diagnosed with Aplastic Anemia, there have been so many blessings along the road to that diagnosis. The elimination of Leukemia, Lymphoma and MDS was a blessing. Her brother and sister both being excellent matches is a blessing. Having a sibling donor and eliminating the need for radiation is a blessing. Her doctors' affirmative confirmation that it is Aplastic Anemia (and not one of the other diseases often producing like symptoms) is a blessing. Even the fact that her levels fell quickly is a blessing; they have been hovering at seriously low levels making her more susceptible to infections, which has been a more serious situation than being able to receive a corrective treatment.