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Upon completion of the chapter, the reader will be able to:
Explain the rationale for using hematopoietic stem cell transplant (HSCT) to treat cancer.
Compare the different types of HSCTs, specifically (a) the types of donors (ie, autologous and allogeneic), (b) the source of hematopoietic cells (ie, umbilical cord, peripheral blood progenitor cells, and bone marrow), and (c) the type of preparative regimen (ie, myeloablative and nonmyeloablative).
Develop a plan for monitoring and managing engraftment of hematopoiesis.
List the nonhematologic toxicity to high-dose chemotherapy used in myeloablative preparative regimens, specifically busulfan-induced seizures, hemorrhagic cystitis, gastrointestinal toxicities, and sinusoidal obstruction syndrome.
Explain graft-versus-host disease (GVHD).
Recommend a prophylactic and treatment regimen for GVHD.
Choose an appropriate regimen to minimize the risk of infectious complications in HSCT patients.
Evaluate the long-term health care of HSCT survivors.
Hematopoietic stem cell transplantation (HSCT) is a procedure used primarily in the treatment of hematologic malignancies via high-dose chemotherapy and/or a graft-versus-tumor effect. HSCT may be either autologous, where a patient receives their own bone marrow, or allogeneic, where the patient receives bone marrow from a donor. As an alternative to bone marrow, hematopoietic stem cells may be obtained from the peripheral blood progenitor cells (PBPCs) or umbilical cord blood (UCB). Bone marrow and PBPCs contain pluripotent stem cells and postthymic lymphocytes, which are responsible for long-term hematopoietic reconstitution, called engraftment and immune recovery.1
The rationale of an autologous transplant is to administer a myeloablative preparative regimen and eradicate the patient’s malignancy. Collecting the bone marrow or PBPCs prior to administering high-dose chemotherapy essentially protects the collected PBPCs from the effects of chemotherapy and can restore hematopoiesis. Allogeneic transplants use donor PBPCs to rescue the patient after myeloablative chemotherapy with an added advantage of the donor cells generating an immunologic response toward the recipient’s residual tumor, called the graft-versus-tumor effect. Unfortunately, graft-versus-host disease (GVHD) where the donor cells generate an immunologic response toward the recipient’s normal tissue also occurs.
The type of HSCT performed depends on a number of factors, including the type and status of disease, availability of a compatible donor, patient age, performance status, and organ function. Examples of diseases treated with HSCT are listed in Table 98–1.
Table 98–1Diseases Commonly Treated with HSCT ||Download (.pdf) Table 98–1 Diseases Commonly Treated with HSCT
| ||Autologous Graft ||Allogeneic Graft |
|Cancers || |
Germ cell tumors
Acute myeloid leukemia
Acute lymphoblastic leukemia
Chronic myeloid leukemia
Chronic lymphocytic leukemia
|Other Diseases || |
Paroxysmal nocturnal hemoglobinuria
Sickle cell anemia
Severe combined immunodeficiency
Inborn errors of metabolism...