BONE MARROW AND

UMBILICAL CORD BLOOD

TRANSPLANTATION
Hematopoietic stem cells
l Stem cell sources
l Autologous
l Allogeneic
l Syngeneic (identical twin)
l Related
l Unrelated
l Stem cell type
l Bone marrow
l Cord blood
l PBSC
Indication of UCB or BM or PBSC transplantation

l Nonmalignant diseases
l Inherited metabolic disorders
l Inherited immune disorders
l Inherited red cell disorders
l Marrow failure states
l Autoimmune diseases (experimental)

l Malignant/premalignant diseases
l Acute lymphoblastic leukemia (ALL)
l Acute myelogenous leukemia (AML)
l Chronic myelogenous leukemia (CML)
l Juvenile myelomonocytic leukemia
l Myelodysplastic syndromes
l Plasma cell disorders
l Hodgkin and non-
non-Hodgkin lymphoma
 The transplant process
l Donor and recipient:
l HLA typing
l Hepatitis B and C, HIV, CMV etc
l Donor
l Umbilical cord blood collection or bone
marrow aspiration
l Recipient
l Pre conditioning
l Stem cells (UCB or BM) infusion
 The transplant process

l Conditioning
l 7-10 day
l chemotherapy and/or radiation to
l eliminate malignancy,
l prevent rejection of new stem cells, and
l create space for the new cells.

l Stem cell processing and infusion

l The stem cells may be processed before
infusion.
 The transplant process
l Neutropenic phase
l 2-4 wk,
l no effective immune system.
l susceptible to infection.
l Supportive care and empiric antibiotic therapy
l Total parenteral nutrition
l Engraftment phase
l several weeks
l the healing process begins with resolution of
mucositis and other lesions acquired.
l fever begins to subside, and infections often begin to
clear.
l management of GVHD and prevention of viral
infections (especially CMV).
 The transplant process
l Postengraftment phase
l months to years.
l Hallmarks of this phase include the gradual
development of tolerance, weaning off of
immunosuppression, management of chronic
GVHD, and documentation of immune
reconstitution.
l Most patients need reimmunization, usually
beginning 1 year posttransplant.
 Cord blood advantages and
disadvantages

Advantages
l An abundantly available source of stem cells
l can be harvested at no risk to the mother or infant
l Ethically is acceptable
l Low viral contamination
l T lymphocyte less than of adult –less GVHD
l a smaller number of cells needed to obtain engraftment.
l A larger percentage of primitive cells exists in cord blood
than in bone marrow.
l more cells are in a highly proliferative state in cord blood,
and these typically would be in a quiescent state in bone
marrow.
 Cord blood advantages and
disadvantages

Disadvantage
l small number of cells collected - limits the size of the
recipient
l only one chance to engraft with a cord blood unit,
l a higher rate of nonengraftment. This is especially
evident in the bone marrow failure diseases, especially
aplastic anemia.
l delayed engraftment of neutrophils (23 d, compared to
10-14 d for marrow and 7-12 d with PBSCs).
UCB transplantation in thalassemia
l Donor is related or sibling
l Unaffected (non thalassemic) – Prenatal
Diagnosis (Pre implantation genetic
diagnosis)
l HLA matched

l Unrelated donor
Centers which have performed UCBT and
BM transplantation in thalassemia

l Italy
l China
l Australia
l Thailand
l Taiwan
l Singapore
l USA
l Canada
l Others