Hematopoietic Cell Transplantation Overview

The collection and transplantation of hematopoietic stem cells, using various sources like bone marrow, peripheral blood, and umbilical cord blood.

HCT is used to treat diseases affecting the blood and immune system, including cancers and genetic disorders.

A graft-versus-tumor effect occurs when donor immune cells attack and destroy remaining cancer cells in the recipient.

HCT involves replacing the recipient's abnormal blood-forming system with a healthy one from a donor.

The hematopoietic stem cell possesses remarkable regenerative capacity, allowing it to rebuild the entire blood-forming system.

Stem cells can travel to the bone marrow after intravenous injection, making transplantation possible.

Stem cells survive freezing and thawing, allowing for long-term storage and a readily available source for transplantation.

A single stem cell is capable of regenerating the entire lymphohematopoietic system of an adult mouse.

A treatment approach for cancer where high doses of chemotherapy or radiation are used to destroy cancerous cells, followed by the transplantation of healthy bone marrow from a compatible donor.

A less intense conditioning approach that involves lower doses of chemotherapy and radiation, aiming to reduce the toxicity of the procedure while still achieving engraftment.

Graft-versus-tumor (GVT) effect refers to the immune response of donor T cells attacking and destroying residual cancer cells in the recipient.

Graft-versus-host disease (GVHD) occurs when donor T cells attack the recipient's tissues, causing organ damage. It is a potential complication of allogeneic transplantation.

A type of conditioning regime that is minimal in intensity, aiming to achieve engraftment with the least possible damage to the patient's body.

A type of conditioning regime that causes significant but not necessarily fatal myelosuppression. It is less intense than conventional high-dose regimens.

The process of collecting bone marrow from a donor for transplantation.

The procedure involves aspirating bone marrow from the posterior and sometimes anterior iliac crests of the donor.

Proteins presented by HLA molecules that can be derived from sources outside the body (exogenous, like during infections) or from within the body (endogenous).

The rejection of a transplanted organ by the recipient's immune system due to differences in HLA (Human Leukocyte Antigen) proteins.

A potentially life-threatening complication after a transplant where the donor's immune cells attack the recipient's body.

Genes responsible for encoding HLA proteins. These genes are very close together on a chromosome so they are inherited together.

The probability that a full sibling will match a patient's HLA type is 1 in 4.

A registry that helps find HLA-matched unrelated donors for patients needing transplants. It allows patients to connect with potential donors who are genetically compatible.

The chance of finding an HLA-matched unrelated donor for a patient is low but not impossible. The NMDP has a large database of volunteers which increases the chances of finding a match.

A newer approach to preventing GVHD that uses high-dose cyclophosphamide after transplantation. This allows for transplants between donors and recipients who share only one HLA haplotype.

A procedure in which hematopoietic stem cells are transferred from a donor to a recipient, replacing the recipient's diseased or damaged bone marrow. This can be used to treat a variety of conditions, including certain cancers, blood disorders, and immune deficiencies.

A type of anemia characterized by a failure of the bone marrow to produce red blood cells. It can be caused by a variety of genetic and environmental factors.

A rare genetic disorder that affects bone development. It is characterized by abnormally dense bones due to an inability of osteoclasts (bone-resorbing cells) to break down bone.

A group of genetic disorders caused by the buildup of specific substances in cells. These disorders can affect various parts of the body, including the brain, liver, spleen, and nervous system.

A type of leukemia that affects the myeloid cells, which are responsible for producing components of the blood, including white blood cells, red blood cells, and platelets. It is typically characterized by rapid growth of abnormal myeloid cells in the bone marrow.

The process of killing cancerous cells using chemotherapy drugs.

A state where the cancer is completely eliminated from the body.

A situation where leukemia returns after a period of remission.

Patients with myeloma who experience disease progression after initial treatment may benefit from allogeneic or autologous transplantation.

Studies show that including autologous transplantation in the initial treatment for myeloma improves disease-free and overall survival.

Adding lenalidomide maintenance therapy after transplantation in myeloma patients further enhances survival benefits.

The use of autologous transplantation followed by non-myeloablative allogeneic transplantation has yielded mixed results in myeloma treatment.

In testicular cancer patients with platinum-based chemotherapy failure, high-dose chemotherapy with autologous stem cell support offers a cure in ~50% of cases.

High-dose chemotherapy with autologous stem cell support is being investigated for various solid tumors like neuroblastoma and pediatric sarcomas.

High-dose chemotherapy with autologous stem cell support is most effective in patients with limited disease and tumors sensitive to conventional chemotherapy.

Patients who experience relapse after autologous transplantation may respond to further chemotherapy and could be candidates for allogeneic transplantation, especially if the initial remission was long.

Allogeneic hematopoietic cell transplantation can cure patients with primary myelofibrosis or myelofibrosis secondary to polycythemia vera or essential thrombocythemia, with 5-year progression-free survival rates exceeding 65%.

Patients with disseminated intermediate- or high-grade non-Hodgkin's lymphoma who have failed first-line chemo might still be cured with a transplant in relapse or second remission.

Allogeneic transplantation is generally reserved for patients with myelodysplasia who have an International Prognostic Scoring System (IPSS) score of Int-2 or higher, or for selected patients with an IPSS score of Int-1 who have other poor prognostic features.

In many cases, complete remissions have been achieved with allogeneic transplantation for advanced-stage diseases. Disease-free survival rates at 3 years are around 50%.

Reduced-intensity conditioning regimens followed by allogeneic transplantation have shown high rates of complete and lasting responses in patients with recurrent indolent lymphomas.

Between 20 and 65% of patients with myelodysplasia appear to be cured with allogeneic transplantation.

Most experts prefer autologous transplantation for patients with intermediate- or high-grade non-Hodgkin's lymphoma because it has fewer complications and similar survival rates compared to allogeneic transplantation.

The role of transplantation in Hodgkin's disease is similar to that in intermediate- and high-grade non-Hodgkin's lymphoma.