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The page below is a sample from the LabCE course Hematopoietic Stem Cell Transplantation. Access the complete course and earn ASCLS P.A.C.E.-approved continuing education credits by subscribing online.

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Auer Rods in myeloblast

Treatment of Hematologic Diseases by HSC Transplantation

The first successful treatment of hematologic disease by bone marrow transplant of an HLA-identical sibling occurred in 1972 for a patient with aplastic anemia. In 1977 researchers at the Fred Hutchinson Cancer Research Center in Seattle, Washington, published a study of transplantation of 100 patients with acute leukemia that were treated with bone marrow from HLA-identical siblings. Today, autologous and allogeneic transplants from HLA-matched donors have become standard therapies for patients with malignant and nonmalignant hematologic diseases.
In a 2015 report by the Center for International Blood & Marrow Transplant Research (CIBMTR), the most common indications for HSC transplants overall were multiple myeloma and non-Hodgkin lymphoma (NHL). Most of these patients received autologous transplants. Patients with acute myeloid leukemia and myelodysplasia were the most common recipients of allogeneic transplants, accounting for more than 50% of all allogeneic HSC transplants. Other allogeneic transplant patients were diagnosed with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), myeloproliferative diseases (MPD), chronic lymphocytic leukemia (CLL), and aplastic anemia. Patients with Hodgkin’s lymphoma (HL) were more likely to receive an autologous transplant. A flowchart for transplantation for a patient with AML appears in the image on the lower right.
Hematologic disorders treated by HSC transplants
Acute nonlymphoblastic leukemia
Aplastic anemia
Acute lymphoblastic leukemia
Fanconi anemia
Hairy cell leukemia
Diamond-Blackfan syndrome
Sickle cell disease
Chronic myeloid leukemia
Chronic lymphocytic leukemia
Paroxysmal nocturnal hemoglobinuria
Hodgkin’s disease
Non-Hodgkin lymphoma
Congenital neutropenia
Multiple myeloma
Chediak-Higashi syndrome
Chronic granulomatous disease
Glanzmann’s thrombasthenia
Gaucher’s disease
HSC transplants are most commonly used when the patient has a disease with a low probability of cure from other treatments (eg, chemotherapy or radiation therapy), or a disease that is incurable (eg, myelodysplasia).
The potential for curing patients with hematologic disorders has been well documented. With improvements in HLA typing, management of GVHD, and prevention of infectious diseases, more patients, especially older patients, have been successfully cured of their disease.
Acute myeloid leukemia (AML)
AML is the most common hematologic disorder treated by allogeneic HSC transplants. The average age for these patients is 45 years and approximately 25% of patients with this diagnosis will require a transplant. Patients with risk factors are more likely to receive a transplant at an early stage in their disease. These risk factors include age over 60, extremely high WBC count, previous myelodysplasia, and two or more induction cycles to obtain complete remission. The outcomes for AML patients with transplants from unrelated donors is about the same as for matched related donors. Improvements in HLA typing and supportive care have improved survival rates for AML patients. The major cause of treatment failure is disease recurrence. Relapse can occur at rates as high as 40% and methods to reduce this complication are under investigation.