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 the transplantation of 100 patients with acute leukemia 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 were multiple myeloma and non-Hodgkin lymphoma (NHL). Most of these patients received autologous transplants. Patients with Hodgkin’s lymphoma (HL) were also more likely to receive an autologous transplant. 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.
Table 4. Hematologic Disorders Treated by HSC Transplants.| Malignant | Nonmalignant |
| Acute non-lymphoblastic leukemia | Aplastic anemia |
| Acute lymphoblastic leukemia | Fanconi anemia |
| Hairy cell leukemia | Diamond-Blackfan syndrome |
| Myelodysplasia | Sickle cell disease |
| Chronic myeloid leukemia | Thalassemia |
| Chronic lymphocytic leukemia | Paroxysmal nocturnal hemoglobinuria |
| Hodgkin's disease | Myelofibrosis |
| Non-Hodgkin lymphoma | Congenital neutropenia |
| Multiple myeloma | Chediak-Higashi syndrome |
| Chronic granulomatous disease |
| Glanzmann's thrombasthenia |
| Gaucher's disease |
| Mucopolysaccharidosis |
HSC transplants are most commonly used when the patient has an incurable disease (e.g., myelodysplasia) or with a low probability of cure from other treatments (e.g., chemotherapy or radiation therapy).
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 are about the same as for matched related donors. Improvements in HLA typing and supportive care have improved survival rates for AML patients. The primary 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.
