Bone Marrow and Stem Cell Transplantation

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DESCRIPTION

  • Hematopoietic stem cell transplantation (HSCT) is the infusion of progenitor stem cells with the intention of restoring hematopoiesis and immunity. Stem cells are infused into the peripheral blood of the recipient using a central venous catheter, similar to a blood transfusion. They then hone to the bone marrow niche and over the next 2 to 4 weeks differentiate into mature blood components.
  • HSCT is currently used in the treatment of malignancies, to replace an absent or poorly functioning hematopoietic or immune system, or for the treatment of nonmalignant, genetic disease.
  • Product types:
    • Bone marrow transplantation (BMT): Stem cells are obtained from bone marrow under general anesthesia.
    • Peripheral blood stem cell transplantation (PBSCT): Stem cells are mobilized to the periphery with cytokines (e.g., granulocyte colony-stimulating factor [G-CSF]) and collected by apheresis.
    • Umbilical cord blood transplantation (UCBT): Stem cells are collected from the umbilical cord and placenta immediately after birth.
  • Donor types:
    • Allogeneic (allo-HSCT): Donor hematopoietic stem cells are used. The donor may be related or unrelated to the recipient.
    • Syngeneic: The donor is an identical twin.
    • Autologous (auto-HSCT): The patient’s own hematopoietic stem cells are used. Stem cells are collected from the patient prior to administration of stem cell-toxic therapy.

EPIDEMIOLOGY

  • In 2018, >5,000 pediatric HSCTs (in patients aged <18 years) were reported. Of those transplants, the majority (~75%) were allogeneic with the remainder (~25%) being autologous.
  • Between 2014 and 2019, among pediatric recipients of allogeneic HSCT, unrelated donors were the most common donor type followed, in order, by matched related donor, umbilical cord blood, and other related donor transplants.
  • In the pediatric population, BMT is more common than PBSCT or UCBT for allogeneic transplants.

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