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General FAQ about Bone Marrow & Peripheral Blood Stem Cell Donation

  1. What is bone marrow and what does it do?

  2. What diseases or disorders can bone marrow transplants or peripheral blood stem cells help with?

  3. Why do those with leukemia, and other patients, need bone marrow transplants or peripheral blood stem cells?

  4. What types of stem cell transplants are available for those with the above diseases?

  5. Is the actual bone marrow what is necessary for the patient to recover?

  6. What are hematopoeitic stem cells?

  7. Why does chemotherapy or radiation therapy kill healthy cells as well as the intended cancerous cells?

  8. Which is better for the recipient to receive, bone marrow or peripheral blood stem cells?

  9. Are umbilical cord blood stem cells better that bone marrow or peripheral blood stem cells?

  10. How many patients are in need of bone marrow transplants (BMT) or peripheral blood stem cells?

  11. How many registered bone marrow or peripheral blood stem cell donors are there?

  12. What is Graft Versus Host Disease (GVHD)?

  13. Is it dangerous to donate your bone marrow or peripheral blood stem cells?


1. What is bone marrow and what does it do?
Bone marrow is the spongy organ found in the core of our bones.  Bone marrow is responsible for manufacturing red and white blood cells and platelets needed for proper clotting.

  • Plasma constitutes about 55% of whole blood.  It is the watery, yellowish-clear portion of the blood which is comprised about 90% water and 10% protein, amino acids, hormones, and waste to be excreted by the renal system.  

  • Red blood cells (also known as erythrocytes, and RBCs) contain hemoglobin (which gives blood it's red color when rich in oxygen) and are responsible for transporting oxygen to the tissues.  RBCs also help remove CO2, a byproduct of your body's normal chemical reactions.  RBCs have a life span of about 120 days so obviously it is necessary for the body to regularly produce more.  

  • White blood cells (also known as leukocytes, WBCs) are responsible for fighting off disease, infection and sickness.  Your WBC's are your body's defense system.  There are 5 main types of WBCs.  A class called 'granulocytes' consist of basophils, eosinophils and neutrophils. The remaining two are lymphocytes and monocytes and help fight infection via the lymph nodes and spleen.  That may seem like a lot of WBCs but in actuality, there are still more RBCs than white.  But a healthy body can produce more WBC's if needed to fight off an infection.  This is usually experienced when your lymph nodes swell and become tender.  It is a normal reaction when your body is fighting off an infection.  The life span of white blood cells range from a few days to a few months  depending upon the type.

  • Platelets (also known as thrombocytes) are responsible for hemostasis, otherwise known as clotting, or coagulation.  The lifespan of a platelet is about 10 days.  Although more complicated than outlined below, in short your platelets arrive at an injury first and begin plugging the hole in a blood vessel to prohibit blood loss.  Then the clotting factors in your plasma make the clot more stable by producing strands of fibrin.  This reaction is triggered by the platelets reaction upon being exposed to the surrounding collagen fibers.  When your platelets react this way, it triggers a chain reaction in your other platelets and WBCs in the vicinity of the wound.  If you didn't have platelets and clotting factors, every time you cut yourself you would bleed to death because it would not stop until there was no pressure to pump the blood out anymore.  

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2. What diseases or disorders can bone marrow transplants (BMT) or peripheral blood stem cell transplants (PBSCT)  help with?
Diseases which may benefit from stem cell transplantation obtained by bone marrow or peripheral blood stem cell donors include:

  • Leukemia

    • Acute Lymphocytic Leukemia (ALL)

    • Acute Myeloid Leukemia (AML)

    • Chronic Lymphocytic Leukemia (CLL)

    • Chronic Myeloid Leukemia (CML)

  • Hodgkin's lymphoma

  • Non-Hodgkin's lymphoma

  • Aplastic Anemia

  • Severe Combined Immunodeficiency (SCID, or "bubble boy" disease)

  • Fanconi Anemia

  • Sickle Cell Anemia

  • Beta-thalassemia

  • Osteosarcoma

  • Myeloma

  • Myelodysplastic Syndromes (MDS)

    • Refractory Anemia
    • Refractory Anemia with Sideroblasts
    • Refractory Anemia with Excess Blasts
    • Chronic Myelomonocytic Leukemia
    • Refractory Anemia with Excess Blasts in Transformation
    • Preleukemia including Aplastic Anemia caused by environmental exposure
    • PH negative Chronic Myelocytic Leukemia

The most comon types of cancer treated from bone marrow or peripheral blood stem cell transplants are leukemia, lymphoma, multiple myeloma, and neuroblastoma.

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3. Why do those with leukemia, and other patients, need bone marrow transplants or peripheral blood stem cells?
Patients who have any of the above and other disorders may have diseased or damaged bone marrow and may not be able to produce sufficient amounts of healthy blood cells, including WBCs.  As the patient undergoes treatment, the WBC count declines further resulting in a disorder called neutropenia.  Medications such as filgrastim (Neupogen®), which is a growth factor, can stimulate the body to produce more WBCs.  Some patients will undergo filgrastim therapy before a transplant to increase the WBC output which helps the patient fight off infection which may prove deadly with a suppressed immune system.  Not all patients with leukemia will be administered filgrastim.  However, those with acute myelcytic leukemia often receive this drug.  It is, however, often used after a bone marrow or peripheral blood stem cell transplant to help reduce the chance of infection and fever.  It also triggers the production of stem cells, or non-progenitor cells, which are removed from the patient before chemotherapy, and re-introduced via IV after the diseased cells are thought to be eradicated.  These cells later turn into healthy cells which seed the bone marrow and produce vital blood cells.

Radiation and chemotherapy further damage the bone marrow, making it even more difficult for a patient to fight off even the common cold.  Bone marrow and stem cells can only be transplanted after the patient's own tissues have been eradicated with chemotherapy or radiation treatments.   If all goes well, the healthy new cells will then increase in number and become that patients new immune system.  In essence, transplants allow the patient to eventually produce healthy cells and platelets.

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4. What types of stem cell transplants are available for those with the above diseases?
There are three types of transplant options available to those who have received chemotherapy or radiation therapy:

  • Allogenic - cells from a patient's parent, sibling or an unrelated donor.  The cells can be removed via surgical bone marrow harvesting or peripheral blood stem cells harvesting via apheresis.

  • Autologous transplants - stem cells from the patient which have been banked prior to the onslaught of disease or stimulated to grow via filgrastim therapy and removed before chemotherapy, or during a remission, can also be used.  Cord blood is also a source of autologous stem cells.  Cord blood is kept (banked) and stored cryogenically. Using autologous banked cord blood stem cells is rare as most patients do not have their stem cells banked at birth.  Harvested adult, autologous stem cells are is an option if the patient is able to produce enough stem cells for re-introduction.  For treatment to be effective, the diseased cells are removed from the marrow via chemo or radiation and the banked cord blood stem cells or adult-harvested stem cells can be injected into the patient's blood.  *Autologous transplants are not possible in those who produce abnormal stem cells, such as aplastic anaemia.

  • Syngeneic  transplants - cells from the patient's identical twin. Obviously only available to those who have twins.  The cells can be removed via surgical bone marrow harvesting or peripheral blood stem cells harvesting via apheresis.

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5. Is the actual bone marrow what is necessary for the patient to recover?
Well, yes and no.  The patient actually needs the stem cells derived from the donor's, or his or her own, bone marrow to eventually seed the bones and grow into healthy marrow so it can produce healthy blood cells.  In allogenic and syngeneic transplants, this marrow is essentially the donor's but becomes the patient's.  In order to survive, we need our own bone marrow to produce blood cells and platelets.  Since the patient's bone marrow's ability to produce cells has been destroyed by disease and treatment, the donor's bone marrow will eventually make it possible for patient to produce their own blood cells.  You cannot live without healthy bone marrow for very long at all.  Transfusions are often necessary during treatment since the patient cannot produce enough of these blood cells.

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6. What are hematopoetic cells?
Hematopoetic cells are the cells needed to produce blood cells.  They are stem cells which will grow into blood cells such as WBC, RBC and platelets.

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7. Why does chemotherapy or radiation therapy kill healthy cells as well as the intended cancerous cells?
Cancerous cells divide very rapidly, far more rapidly than healthy, disease-free cells.  Chemotherapy and radiation therapy is used to kill these rapidly dividing cells.  But, unfortunately bone marrow cells also divide very rapidly and are inadvertently destroyed along with the cancer cells.  This is why a transplant is necessary after radiation or chemotherapy therapy.  The new stem cells are needed to replace the ones which are now gone.  Without bone marrow, your body cannot produce blood cells. You would eventually die of infection, not be able to give your body the oxygen it needs as well as remove the waste created by your body's cells and also would bleed out because you would not be able to clot.

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8. Which is better for the recipient to have, a bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT)?
While either can be very beneficial to the patient and highly increase their chances of a full recovery, they are one and the same.  When the bone marrow is collected from an allogeneic source, it is filtered to remove participles of bone and fat.  RBC's may also be removed if the blood type of the donor is different from the patient.
  It is the stem cells contained within the serum extracted that is injected into the bloodstream of the recipient patient, and is what makes it possible for patients to grow new, disease-free marrow.  In either BMT or PBSCT, the donor is put on filgrastim therapy to increase the amount of stem cells.  Hematopoietic stem cells are the cells needed for life, that will seed themselves into the marrow to begin producing their own blood cells.  Unfortunately, there are more stem cells found in the bone marrow than in the blood, even with filgrastim therapy.  So, obtaining enough stem cells this way can be difficult.

Whether or not one is better than the other depends upon the study.  However, according to one finding ["Hematopoietic stem cell transplantation for treatment of acute leukemia" Harada, M. - Department of Internal Medicine II, Okayama University Medical School, Japan] autologous PBSCT is increasingly being preferred over autologous BMT, as well as allogeneic PBSCT over allogeneic BMT.  The conclusion is that over all, PBSCT is becoming the treatment option of choice with those who qualify.  This is good news since PBSCT is easier on the donor than BMT as it is non surgical and has no true downtime other than slight bone discomfort due to filgrastim therapy.

 According to others, however, BMT still remains in favor.

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9. Are umbilical cord blood stem cells better that bone marrow or peripheral blood stem cells?
Cord blood stem cells contain hematopoietic cells and cells which can pretty much develop into any tissue type.  According to the Genetic Science Leaning Center - University of Utah, cord blood stem cells are less likely to be rejected by the body.    They also advise that since the cord blood does not have mature immune cells, they are less likely to attack the patient, triggering what is known as Graft-versus-Host Disease, or GVHD.

Nonetheless, cord blood is often in short supply as most people do not think to, or are not willing to, donate their baby's cord blood after birth.  Many times, the umbilical cord is simply wasted and thrown away.

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10. How many patients are in need of bone marrow transplants (BMT) or peripheral blood stem cells?
It is estimated that 35,000 children and adults in the United States are diagnosed every year with a condition which could be treated with a bone marrow transplant.  The more donors that sign up, the better the chances of a recipient finding his or her lifesaving match.  Possibly you!

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11. How many registered bone marrow or peripheral blood stem cell donors are there?
At the time of this writing, the National Marrow Donor Program (NMDP) boasts a registry of over 5.5 million donors.   While you may feel that certainly sounds like a lot, the chances of finding a match have been reported as much as 1:20,000 and as little as 1:100,000; the ratio is even higher for minority groups.  This ratio significantly lowers the chances of funding a life saving match.  All the more reason to become a donor.

As of November 2005, the World Marrow Donors Association boasted 10 million registered bone marrow or blood stem cell donors in the whole world. Incredible!!!

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12. What is Graft Versus Host Disease (GVHD)?
Graft-versus-Host Disease, or GVHD, is a disorder triggered by an immune response of the donor's stem cells in the recipient's body.  The Graft is the stem cell and the host is the recipient.  The donor's stem cells recognize the recipient's tissues as a foreign body and begin to attack it.  It is essentially a secondary disease as it is a disorder on top of the already present disease, such as leukemia.  The incidence is increased when there is a non-perfect match, or a less than 6 out of 6 HLA (human leukocyte antigen) match, although it can happen at any time.

GVHD occurs less in cord blood stem cell transplants.  It has even been shown to occur less in allogeneic BMTs if the stem cells are cryopreserved first.  A patient can be conditioned with immunosuppressants such as tacrolimus (FK-506) and cyclosporine, antimetabolites (methotrexate) and/or with steroids to help lower the chances of GVHD.

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13. Is it dangerous to donate your bone marrow or peripheral blood stem cells?
While there are inherent risks to any surgical procedure, the risks are nominal compared to the benefits they will bring.  However, you must know the risks before you are informed.  

  • Filgrastim and Pegfilgrastim Use: The risks, complications or side effects related to filgrastim therapy proceeding the donation of either BM or PBSC are as follows: bone pain (common), headache, a feeling of pressure in the bones, inability to get comfortable, swelling or redness at the injection site, nausea. **Contact your doctor if you should experience chills, congestion, diarrhea, or rash.  If you should experience shortness of breath, wheezing, sweating, increase in blood pressure or heart rate, swelling of the ice, mucous membranes and a tightness in the throat and chest, excessive pain, fever (over 100.5º F) seek medical attention immediately and also contact your doctor to let them know.  Also let your doctor know if you experience pain or swelling around a wound or wound healing problems. If you notice any swelling or painful areas in the upper left area of your abdomen you must call your doctor immediately.  Spleen enlargement and rupture have occurred in some patients, although it is rare, in those who take filgrastim or pegfilgrastim.  Do not take filgrastim or pegfilgrastim if you are pregnant or nursing as its effects have not been studied in this regard.  Read more about a common pegfilgrastim product used, Neulasta.

  • Bone Marrow Aspiration: Risks associated with anesthesia such as allergic reaction, asthma attacks in those susceptible and who under go General anesthesia, and other medication allergies.  Infection resulting in skin necrosis, substantial scarring, keloids in those who are prone are also risks of bone marrow donation.

    


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