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    NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 2

    NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 2

    S
    @SJIA_Foundation
    8 Followers
    5 months ago 317

    Presentation from Dr. Rebecca Marsh: BMT: How does it work and when it can be considered?

    BMT: How Does It Work and 
When Can It Be Considered?
Rebecca Marsh, MD
Cincinnati Children’s Hospital
    1/33
    Disclosures
• Employed part-time by Pharming Healthcare
• Advisory board or consulting - Amgen, AB2Bio, SOBI
    2/33
    I Failed to Find a Good 
Rheumatology Joke to Open With…
    3/33
    I Failed to Find a Good 
Rheumatology Joke to Open With…
• They were all too inflammatory!
    4/33
    BMT: How Does It Work?
    5/33
    BMT: Terminology
• For today: Bone Marrow Transplant (BMT) = Hematopoietic Cell 
Transplant (HCT) = Stem Cell Transplant etc
Allogeneic Autologous
Stem Cells Stem Cells
    6/33
    Allogeneic BMT Process
• It is like planting a garden
    7/33
    First you need to make room for a garden 
The Garden
    8/33
    Then you have to plant the seeds 
The Garden
    9/33
    Then you have to wait…
The Garden
    10/33
    You have to nurture the new plants...
Water, Sun, Food, Love
keep harmful things away
The Garden
    11/33
    … until they are 
able to survive on their own
The Garden
    12/33
    So, What If We Want To Plant Stem Cells 
Instead of Flowers?
    13/33
    Process
1) Patient
2) Donor
3) Conditioning
4) Graft (Stem Cells)
5) Recovery
    14/33
    • Step 1: Patient
– Which patients are appropriate to consider for allogeneic BMT?
Allogeneic BMT in IEI
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    – #1: The disease needs to be 
caused by a genetic disorder 
(known or unknown) that only or 
predominantly affects 
hematopoietic derived cells 
Ogonek et al, 2016
– #2: Patients should be at high risk of 
morbidity or mortality from their disease
In General: 2 Requirements to Consider HCT
Immune System
    16/33
    A Lot to Balance!
Transplant 
Risk
Immune 
Disorder 
Risk
Donor Options
Lung Disease
MAS New Clinical Trial/
New Therapies
Response to
Therapies
Co-Morbidities
    17/33
    Step 2: Find a Donor
    18/33
    HLA Typing and Donor Search
commons.wikimedia.org/wiki/File:HLA.jpg
    19/33
    HLA Typing and Donor Search
commons.wikimedia.org/wiki/File:HLA.jpg
    20/33
    HLA Typing and Donor Search
commons.wikimedia.org/wiki/File:HLA.jpg
    21/33
    HLA Typing and Donor Search
commons.wikimedia.org/wiki/File:HLA.jpg
    22/33
    HLA Typing and Donor Search
commons.wikimedia.org/wiki/File:HLA.jpg https://www.aboutkidshealth.ca/article?contenti
d=1555&language=english#
GVHD
    23/33
    HLA Typing and Donor Search
Patient
Donor
    24/33
    NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 2 - Page 25
    25/33
    Step 3: Conditioning Regimen
Vs. Vs..
CCHMC CHOP GOSH
Transplant Center Conditioning Regimens
Serotherapy A
Chemotherapy ABC
Serotherapy B
Chemotherapy BC
Serotherapy A
Chemotherapy BCD
    26/33
    Step 4a: Stem Cell Collection
    27/33
    Step 4b: Stem Cell Infusion
• Infuse stem cell product into patient
    28/33
    Step 5
    29/33
    Thank you!
    30/33
    14
7 1 year
    31/33
    HLA Typing and Donor Search
commons.wikimedia.org/wiki/File:HLA.jpg
    32/33
    Why do we care about HLA match?
GVHD
– Remains the 
Biggest Barrier 
to Successful 
Outcomes
https://www.aboutkidshealth.ca/article?contentid=1555&language=english#
    33/33

    NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 2

    • 1. BMT: How Does It Work and When Can It Be Considered? Rebecca Marsh, MD Cincinnati Children’s Hospital
    • 2. Disclosures • Employed part-time by Pharming Healthcare • Advisory board or consulting - Amgen, AB2Bio, SOBI
    • 3. I Failed to Find a Good Rheumatology Joke to Open With…
    • 4. I Failed to Find a Good Rheumatology Joke to Open With… • They were all too inflammatory!
    • 5. BMT: How Does It Work?
    • 6. BMT: Terminology • For today: Bone Marrow Transplant (BMT) = Hematopoietic Cell Transplant (HCT) = Stem Cell Transplant etc Allogeneic Autologous Stem Cells Stem Cells
    • 7. Allogeneic BMT Process • It is like planting a garden
    • 8. First you need to make room for a garden The Garden
    • 9. Then you have to plant the seeds The Garden
    • 10. Then you have to wait… The Garden
    • 11. You have to nurture the new plants... Water, Sun, Food, Love keep harmful things away The Garden
    • 12. … until they are able to survive on their own The Garden
    • 13. So, What If We Want To Plant Stem Cells Instead of Flowers?
    • 14. Process 1) Patient 2) Donor 3) Conditioning 4) Graft (Stem Cells) 5) Recovery
    • 15. • Step 1: Patient – Which patients are appropriate to consider for allogeneic BMT? Allogeneic BMT in IEI
    • 16. – #1: The disease needs to be caused by a genetic disorder (known or unknown) that only or predominantly affects hematopoietic derived cells Ogonek et al, 2016 – #2: Patients should be at high risk of morbidity or mortality from their disease In General: 2 Requirements to Consider HCT Immune System
    • 17. A Lot to Balance! Transplant Risk Immune Disorder Risk Donor Options Lung Disease MAS New Clinical Trial/ New Therapies Response to Therapies Co-Morbidities
    • 18. Step 2: Find a Donor
    • 19. HLA Typing and Donor Search commons.wikimedia.org/wiki/File:HLA.jpg
    • 20. HLA Typing and Donor Search commons.wikimedia.org/wiki/File:HLA.jpg
    • 21. HLA Typing and Donor Search commons.wikimedia.org/wiki/File:HLA.jpg
    • 22. HLA Typing and Donor Search commons.wikimedia.org/wiki/File:HLA.jpg
    • 23. HLA Typing and Donor Search commons.wikimedia.org/wiki/File:HLA.jpg https://www.aboutkidshealth.ca/article?contenti d=1555&language=english# GVHD
    • 24. HLA Typing and Donor Search Patient Donor
    • 26. Step 3: Conditioning Regimen Vs. Vs.. CCHMC CHOP GOSH Transplant Center Conditioning Regimens Serotherapy A Chemotherapy ABC Serotherapy B Chemotherapy BC Serotherapy A Chemotherapy BCD
    • 27. Step 4a: Stem Cell Collection
    • 28. Step 4b: Stem Cell Infusion • Infuse stem cell product into patient
    • 29. Step 5
    • 30. Thank you!
    • 31. 14 7 1 year
    • 32. HLA Typing and Donor Search commons.wikimedia.org/wiki/File:HLA.jpg
    • 33. Why do we care about HLA match? GVHD – Remains the Biggest Barrier to Successful Outcomes https://www.aboutkidshealth.ca/article?contentid=1555&language=english#


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