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Marrow Matters

Aplastic Anemia Treatment

Twenty years ago, Aplastic Anemia was a death sentence. Today, treatments are available that can treat the disease and even cure it.

Aplastic Anemia is a life-threatening disease in which the bone marrow fails to produce blood cells. Aplastic Anemia has three classifications: Moderate Aplastic Anemia, Severe Aplastic Anemia, or Very Severe Aplastic Anemia.

The diagnostic process often takes some time because there is no single test to determine if a person has Aplastic Anemia; rather, it is a diagnosis derived from excluding all other diseases and confirming that the cellularity of the bone marrow is sufficient failing and blood stem cells are dying and turning into fat deposits within the bone marrow to be called 'Aplastic Anemia'.

The mainstream treatments are divided into two categories: bone marrow transplant or drugs only.

DO NOT TAKE HOLISTIC CHINESE HERBS FOR THE TREATMENT OF APLASTIC ANEMIA. See DO NOT TAKE HERBAL MEDS.

bone marrow transplant
Blood Stem Cell Transplant

Medical science has used bone marrow transplants for decades to treat a variety of diseases. While bone marrow transplants aren't yet commonplace, they are done frequently in the US and held by the medical community as the only cure to Aplastic Anemia.

Typically, a person with Aplastic Anemia will undergo some of its cruel effects before being able to receive their transplants. Those effects can include the follow problems and many more.

Becoming nuetropenic, which means, in short, that the body does not have enough fighter white cells to ward off infections that a healthy person wards off daily let alone major contagions.
Fatigue - not the kind of tiredness after a bad night's sleep, but rather the feeling of total exhaustion you may have felt after being up for many consecutive days.
Crashed levels - a person with Aplastic Anemia already has low blood counts (meaning that their blood count levels are below the bottom of the recommended level), but as the disease progresses, the marrow produces less and less cells until it is producing none or nearly none. While our blood cells die naturally, our healthy bone marrow keeps up with the demands of our body, but a person with Aplastic Anemia has a failed marrow - their blood cells die, but the marrow cannot keep up with the body's demand. They must have transfusions to prevent them from going into a coma for lack of oxygen supply due to too few red cells carrying oxygen to their organs and brain, to prevent them from bleeding to death.

Aside from the emotional aspects of a bone marrow transplant, the actual transplant is quite rudementary compared to what the patient has undergone previously. It is in fact much like a blood transfusion.

What is a bone marrow transplant?

In simple terms, a donor is found, a harvest of the donor's blood stem cells is performed, and the blood stem cells are fed into the patient's bloodstream via an IV or into a port or line just as if they were getting an infusion of packed red cells.

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

Bone Marrow Transplant and Blood Stem Cell Transplant are terminologies that are often used interchangeably. A Bone Marrow Transplant refers to the process of harvesting bone marrow and transplanting it into another person's blood stream. A Blood Stem Cell Transplant 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). Studies have indicated that there are advantages to transplanting only the blood stem cells; other studies have been done involving transplantation of the blood stem cells and certain white blood cells.

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 (they cannot transplant themselves with their own diseased marrow). Autologous bone marrow transplants can be used for patients with Leukemia and other bone marrow diseases. Whether an autologous transplant is possible is only able to be determined by your medical professionals.

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. See also Long Term Effects From Bone Marrow Transplants.

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

Prednisone, one of the most commonly known steroids and one used in the treatment of Aplastic Anemia, as well as many other diseases such as Graft-versus-Host Disease.

Full-body Radiation

If the donor and recipient are unrelated, total body radiation will also be administered. Related donor blood stem cells do not require radiation.

engraftment

Within 3-4 weeks post transplant, new blood stem cells should appear in the recipient's 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.

Drug Therapy
Immunosuppression Therapy

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

Immunosuppression therapy occurs when a patient takes a drug that causes their immune system to be suppressed. More specifically, there are several types of immunosuppressant drugs, such as Prograf (Tachrolimus) and Cellcept (Mycophenolate), which act upon various white cells; Prograf, for example, reduces interleukin-2 production by T-cells.

Immunosuppression therapy is used most often when a matched bone marrow donor cannot be found.

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.

Immunosuppression renders the patient susceptible to viral, bacterial and fungal infections, as well as numerous other issues including cancers. 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. See Long Term Effects of Immunosuppression, and Avoid & Prevent Infections.

herbal treatments

DO NOT - REPEAT - DO NOT TAKE HERBAL SUPPLEMENTS TO TREAT APLASTIC ANEMIA.

The single most frequently asked question by medical staff of a person who is suspected of having Aplastic Anemia or a related disease is, "Have you been taking a herbal supplement?" The reason that doctors ask if the patient has been on ANY herbal drug is because they can cause Aplastic Anemia, as well as other diseases.

Herbal Drugs Do Not Treat Aplastic Anemia.