Bone Marrow/Stem Cell Transplantation for the Treatment of Patients with Malignant Diseases (Leukemia, Lymphoma or Myeloma)

Review Article

J Stem Cell Res Transplant. 2020; 7(1): 1031.

Bone Marrow/Stem Cell Transplantation for the Treatment of Patients with Malignant Diseases (Leukemia, Lymphoma or Myeloma)

Berhanu AD*, Abrha GT and Kasegn MM

Department of Biotechnology, Mekelle University, Ethiopia

*Corresponding author: Berhanu AD, Department of Biotechnology, Mekelle University, Ethiopia

Received: December 14, 2019; Accepted: January 13, 2020; Published: January 20, 2020


A stem cell transplant is a procedure that replaces unhealthy blood-forming cells with healthy ones. The aim of this review is to provide detail information for the readers about the curative treatment option of bone marrow/stem cell transplantations for the treatment of patients with malignant diseases, mainly leukemia, lymphoma or myeloma and to familiarize with a relatively recent technique of bone marrow/stem cell transplants. Bone marrow/Stem cell transplantation procedures continue to be improved, making transplantation a treatment option for more patients. Certain other marrow disorders are also treated with bone marrow/stem cell transplantation. For some patients, this complex procedure offers a curative treatment option. Since blood and marrow are both good sources of stem cells for transplantation, the term “stem cell transplantation” has replaced “bone marrow transplantation” as the general term for this procedure. The abbreviation “BMT” is now used to represent blood and marrow transplantation. There are many terms for transplantation, including Bone Marrow Transplantation (BMT), marrow or umbilical cord blood transplantation, Hematopoietic Stem Cell Transplantation (HSCT). These are all different names for the same procedure. Today, the stem cells used for transplantation can come from marrow, peripheral blood or umbilical cord blood. Peripheral blood is now the most common source of stem cells for transplantation.

Keywords: Blood cells; Cancer; Marrow Disorders; Stem Cells; Sources of Stem Cells


ALL: Acute Lymphoblastic Leukemia; BMT: Bone Marrow Transplantation; DLI: Donor Lymphocyte Infusion; GVHD: Graft- Versus-Host Disease; HLA: Human Leukocyte Antigens; HSCT: Hematopoietic Stem Cell Transplantation; HZV: Herpes Zoster Virus; NMDP: National Marrow Donor Program; Pbscs: Peripheral Blood Stem Cells; RIC: Reduced Intensity Conditioning Stem Cell Transplant; SCT: Stem Cell Transplantation; SLE: Systemic Lupus Erythematosus


Bone marrow/Stem cell transplantation is a procedure that can restore marrow function for patients who have had severe marrow injury or abnormalities of the immune system. Marrow injury can occur because of primary marrow failure, destruction or replacement of marrow by disease, or intensive chemical or radiation exposure. The basis for stem cell transplantation is that all blood cells and immune cells arise from stem cells in marrow. At the turn of the 20th century, scientists began to formulate the idea that a small number of cells in the marrow might be responsible for the development of all blood cells. They began to refer to them as “stem cells.” The scientific exploration of marrow transplantation as a form of cancer treatment began at the end of World War II. Bone marrow transplantation and hematopoietic stem cell transplantation have been used with increasing frequency to treat numerous malignant and nonmalignant diseases forty years ago. Post-World War II “Cold War” fears of nuclear warfare stimulated interest in the effects of radiation on the human body. Early studies with animals quickly revealed that bone marrow was the organ most sensitive to the damaging effects of radiation. The reinfusion of marrow cells was subsequently used to rescue lethally irradiated animals. In the 1950s, patients were given lethal doses of radiation to treat leukemia. Although many had hematologic recovery following this treatment, all patients eventually succumbed to relapse of their malignancies or to infections. In the 1950s and 1960s, almost 200 allogeneic marrow transplants were performed in humans, with no long-term successes. However, during this time, transplantation using identical twin donors brought a fair amount of success and provided a crucial foundation to continued clinical research in the field [1].

In 1968, the first major landmark in bone marrow transplantation occurred with successful allogeneic transplantations performed for an infant with X-linked lymphopenic immune deficiency and for another with Wiskott-Aldrich syndrome [2,3]. These successes were followed by reports of effective transplantation for aplastic anemia and, later, for leukemia. Advances in histocompatibility testing and development of marrow donor registries, such as the National Marrow Donor Program (NMDP), have facilitated the use of unrelated donors, thus expanding the number of patients who can receive transplants [4].

In 1988, successful transplantation occurred in a young boy with Fanconi anemia using umbilical cord blood collected at the birth of his sibling [5]. The patient remains alive and well to this date. In 1992, a patient was successfully transplanted with cord blood instead of bone marrow for the treatment of leukemia. Over the past decade, the use of cord blood has rapidly expanded [6].

The 5-year leukemia-free survival rate in 503 children with Acute Lymphoblastic Leukemia (ALL) who received a transplant of umbilical cord blood that was mismatched for either one or two Human Leukocyte Antigens (HLA) was similar when compared with the survival rate of 282 children who received bone marrow transplants [7].

In addition to bone marrow and cord blood, Peripheral Blood Stem Cells (PBSCs) have gained popularity as a source of stem cells since their initial introduction in the 1980s. The most important cell needed for successful transplantation is the hematopoietic stem cell. Currently, the major sources of stem cells for transplantation include bone marrow, peripheral blood, and cord blood [8].

Necessities of Bone Marrow/Stem Cell Transplantation

Bone marrow basics

Bone marrow is soft, spongy tissue found inside the bone, such as the hip and thighbones. All blood starts out as stem cells, “parent cells” produced in the bone marrow. Stem cells develop into one of the three types of mature blood cells. These are red blood cells, white blood cells and platelets and then enter into the bloodstream. Red blood cells carry oxygen throughout the body. White blood cells fight infection and platelets cause blood to clot. Bone marrow was the first source used for stem cell transplants because it has a rich supply of stem cells. The bones of the hip, chest (sternum/breastbone) and pelvis contain the largest amount of marrow and stem cells. For this reason, cells from these bones are used most often for a bone marrow/stem cell transplant. Enough marrow must be removed to collect a large number of healthy stem cells. For a bone marrow/stem cell transplant, the donor gets general anesthesia (drugs are used to put the patient into a deep sleep). Several large needle sticks are made through the skin into the back of the pelvic bone to remove marrow. The thick, liquid marrow is pulled out through the needle [9].

The harvested marrow is filtered, stored in a special solution in bags, and then frozen. When the marrow is to be used, it is thawed and then given just like a blood transfusion. The stem cells travel to the recipient’s bone marrow. There, they engraft or “take” over time and begin to make blood cells. Signs of the new blood cells usually can be measured in the patient’s blood tests in about 2 to 4 weeks [10].

Why Transplant Bone Marrow/Stem Cell?

The basic idea behind BMT/SCT is to allow high doses of chemotherapy and/or radiation therapy in order to kill rapidly dividing cells in the body and to make room for new healthy cells. Cancer cells, like other cells in the body divide rapidly. Though these treatments are among the most effective weapons against many forms of cancer, they don’t have precise aim and they cannot target only diseased cells. As a result, many normal rapidly dividing cells, including stem cells are also destroyed during the treatment. Therefore, “rescue” with transplanted bone marrow or stem cells enables the patient to produce new blood cells to replace those destroyed during treatment.

Types of Bone Marrow/Stem Cell Transplantations

There are two main types of bone marrow or stem cell transplants: these are autologous (the patient’s own bone marrow or stem cells are used) and allogeneic (a donor supplies the marrow or stem cells). A syngeneic transplant is a type of allogeneic transplant of marrow or stem cells from an identical twin.

Autologous bone marrow/stem cell transplantation

“Auto” means “self.” This type of transplantation involves the use of a patient’s own stem cells. Immature marrow cells (stem cells) are collected from the patient himself/herself before he/she receives high-dose chemotherapy or radiation treatment, and then frozen. The thawed cells are returned to the patient after he or she has received intensive chemotherapy and/or radiation therapy for his or her disease. This is called a “rescue” transplant [11]. The primary purpose of an autologous transplant is to allow the patient to be given high doses of chemotherapy with or without radiation that would otherwise be too toxic to tolerate because the marrow would be severely damaged. Such high doses of treatment can sometimes overcome resistance of the disease to standard doses of chemotherapy. Autologous bone marrow/stem cell transplantation is used mainly to treat people who have a blood cancer diagnosis, but may be used to treat patients who have some other types of cancer. Autologous transplantation requires that an individual have sufficient numbers of healthy stem cells in the marrow or blood. For example, in patients with acute leukemia, remission must be achieved before the patient’s marrow or blood is harvested and frozen for later use (Figure 1).

Citation: Berhanu AD, Abrha GT and Kasegn MM. Bone Marrow/Stem Cell Transplantation for the Treatment of Patients with Malignant Diseases (Leukemia, Lymphoma or Myeloma). J Stem Cell Res Transplant. 2020; 7(1): 1031.