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Aplastic Anemias
&
Blood Marrow Transplant II
The basics…what you need to know
Dr. Sookun Rajeev K
(MD)
Dept of General Medicine
Anna Medical College
Types of Transplant
 Autologous (your own cells)
 Allogeneic
 cells from another person

Sibling

Unrelated Donor

Parent or relative
 or source: Umbilical cord
Hematopoietic Progenitor
Cell Sources
Bone Marrow
PBSC (peripheral blood
stem cells)
Umbilical Cord
Best Allogeneic Blood/Bone
Marrow Donor is a brother or
sister
 Only 25% of patients are that
lucky!
 There is a 1 in 4 chance that any child
will match another child of the same
parents
 Major obstacle in the treatment of
patients who would benefit from
an allogeneic transplant.
Bone Marrow
 Standard source of hematopoietic
cells for more than 30 years.
 Transplant physicians may select
marrow because:
 Extensive clinical data are available
about marrow transplant outcomes
 Extensive information is available about
the marrow donation experience
Peripheral Blood Stem
Cell
 Autologous transplants rely almost
exclusively on PBSC rather than marrow
due to:
 Easier collection of cells
 More rapid hematopoietic recovery
 Decreased costs
 We also use this method in certain
instances for allogeneic transplants in
pediatrics.
Umbilical Cord Blood
 Physicians may consider umbilical cord
blood a good choice particularly for patients
who need an unrelated donor and have an
uncommon HLA type or are in urgent need
of a transplant.
 HLA mismatch is better tolerated – even
with haploidentical donors
 Available more quickly than marrow or
PBSC unrelated donors
 Reduced incidence and severity of GVHD
Transplant Process (5
steps)
(1) Conditioning,
(2) Stem cell infusion,
(3) Neutropenic phase,
(4) Engraftment phase
(5) Post-engraftment period.
Conditioning Phase
 The conditioning period typically lasts 7-10
days.
 The purposes are (by delivery of
chemotherapy and/or radiation)
 to eliminate malignancy
 to provide immune suppression to prevent
rejection of new stem cells
 create space for the new cells
 Radiation and chemotherapy agents differ
in their abilities to achieve these goals.
Stem cell processing and
infusion
 Infusion - 20 minutes to an hour,
varies depending on the volume
infused. The stem cells may be
processed before infusion, if
indicated. Depletion of T cells can be
performed to decrease GVHD.
 Premedication with acetaminophen
and diphenhydramine to prevent
reaction.
Stem cell processing and
infusion
 Infused through a CVL, much like a blood
transfusion.
 Anaphylaxis, volume overload, and a (rare)
transient GVHD are the major potential
complications involved.
 Stem cell products that have been
cryopreserved contain dimethyl sulfoxide
(DMSO) as a preservative and potentially
can cause renal failure, in addition to the
unpleasant smell and taste.
Neutropenic Phase
 During this period (2-4 wk), the patient
essentially has no effective immune system.
 Healing is poor, and the patient is very
susceptible to infection.
 Supportive care and empiric antibiotic
therapy are the mainstays of successful
passage through this phase.
Engraftment Phase
 During this period (several weeks),
the healing process begins with
resolution of mucositis and other
lesions acquired. In addition, fever
begins to subside, and infections
often begin to clear. The greatest
challenges at this time are
management of GVHD and prevention
of viral infections (especially CMV).
Post-engraftment Phase
 This period lasts for months to
years. Hallmarks of this phase
include the gradual development
of tolerance, weaning off of
immunosuppression, management
of chronic GVHD, and
documentation of immune
reconstitution.
Graft versus Host Disease
(GVHD)
• If donor cells see the host cells as
foreign, the donor cells will attack
the host.
• Skin, gut, and liver most likely to be
affected.
• Acute < 100 days after the
transplant
• Chronic > 100 days
Acute Graft versus Host Disease of Skin
Graft Versus Host Disease of the Skin: Grade IV
Chronic Extensive Graft versus Host Disease
INFECTIONS POST TRANSPLANT
Other Problems Encountered
 Hemorrhagic Cystitis
 VOD (venoocclusive disease of the
liver) or SOS (solid organ syndrome)
 Organ Toxicity (lung, heart, kidney)
 Idiopathic Pneumonia Syndrome

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Aplastic Anemias & Bone Marrow Transplant II by Dr. Sookun Rajeev Kumar

  • 1. Aplastic Anemias & Blood Marrow Transplant II The basics…what you need to know Dr. Sookun Rajeev K (MD) Dept of General Medicine Anna Medical College
  • 2. Types of Transplant  Autologous (your own cells)  Allogeneic  cells from another person  Sibling  Unrelated Donor  Parent or relative  or source: Umbilical cord
  • 3. Hematopoietic Progenitor Cell Sources Bone Marrow PBSC (peripheral blood stem cells) Umbilical Cord
  • 4. Best Allogeneic Blood/Bone Marrow Donor is a brother or sister  Only 25% of patients are that lucky!  There is a 1 in 4 chance that any child will match another child of the same parents  Major obstacle in the treatment of patients who would benefit from an allogeneic transplant.
  • 5. Bone Marrow  Standard source of hematopoietic cells for more than 30 years.  Transplant physicians may select marrow because:  Extensive clinical data are available about marrow transplant outcomes  Extensive information is available about the marrow donation experience
  • 6. Peripheral Blood Stem Cell  Autologous transplants rely almost exclusively on PBSC rather than marrow due to:  Easier collection of cells  More rapid hematopoietic recovery  Decreased costs  We also use this method in certain instances for allogeneic transplants in pediatrics.
  • 7. Umbilical Cord Blood  Physicians may consider umbilical cord blood a good choice particularly for patients who need an unrelated donor and have an uncommon HLA type or are in urgent need of a transplant.  HLA mismatch is better tolerated – even with haploidentical donors  Available more quickly than marrow or PBSC unrelated donors  Reduced incidence and severity of GVHD
  • 8. Transplant Process (5 steps) (1) Conditioning, (2) Stem cell infusion, (3) Neutropenic phase, (4) Engraftment phase (5) Post-engraftment period.
  • 9. Conditioning Phase  The conditioning period typically lasts 7-10 days.  The purposes are (by delivery of chemotherapy and/or radiation)  to eliminate malignancy  to provide immune suppression to prevent rejection of new stem cells  create space for the new cells  Radiation and chemotherapy agents differ in their abilities to achieve these goals.
  • 10. Stem cell processing and infusion  Infusion - 20 minutes to an hour, varies depending on the volume infused. The stem cells may be processed before infusion, if indicated. Depletion of T cells can be performed to decrease GVHD.  Premedication with acetaminophen and diphenhydramine to prevent reaction.
  • 11. Stem cell processing and infusion  Infused through a CVL, much like a blood transfusion.  Anaphylaxis, volume overload, and a (rare) transient GVHD are the major potential complications involved.  Stem cell products that have been cryopreserved contain dimethyl sulfoxide (DMSO) as a preservative and potentially can cause renal failure, in addition to the unpleasant smell and taste.
  • 12. Neutropenic Phase  During this period (2-4 wk), the patient essentially has no effective immune system.  Healing is poor, and the patient is very susceptible to infection.  Supportive care and empiric antibiotic therapy are the mainstays of successful passage through this phase.
  • 13. Engraftment Phase  During this period (several weeks), the healing process begins with resolution of mucositis and other lesions acquired. In addition, fever begins to subside, and infections often begin to clear. The greatest challenges at this time are management of GVHD and prevention of viral infections (especially CMV).
  • 14. Post-engraftment Phase  This period lasts for months to years. Hallmarks of this phase include the gradual development of tolerance, weaning off of immunosuppression, management of chronic GVHD, and documentation of immune reconstitution.
  • 15. Graft versus Host Disease (GVHD) • If donor cells see the host cells as foreign, the donor cells will attack the host. • Skin, gut, and liver most likely to be affected. • Acute < 100 days after the transplant • Chronic > 100 days
  • 16. Acute Graft versus Host Disease of Skin
  • 17. Graft Versus Host Disease of the Skin: Grade IV
  • 18. Chronic Extensive Graft versus Host Disease
  • 20. Other Problems Encountered  Hemorrhagic Cystitis  VOD (venoocclusive disease of the liver) or SOS (solid organ syndrome)  Organ Toxicity (lung, heart, kidney)  Idiopathic Pneumonia Syndrome

Editor's Notes

  1. Human Leukocyte Antigen
  2. Central Venous Line