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

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

@SJIA_Foundation
@SJIA_Foundation
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2 months ago 190

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

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

@SJIA_Foundation2 months ago

BMT: How Does It Work and When Can It Be Considered?

Rebecca Marsh, MD

Cincinnati Children's Hospital

                      Disclosures

                      • · Employed part-time by Pharming Healthcare
                      • · Advisory board or consulting - Amgen, AB2Bio, SOBI

                                          I Failed to Find a Good Rheumatology Joke to Open With…

                                                              I Failed to Find a Good Rheumatology Joke to Open With…

                                                              • · They were all too inflammatory!

                                                                                  BMT: How Does It Work?

                                                                                                      BMT: Terminology

                                                                                                      • · For today: Bone Marrow Transplant (BMT) = Hematopoietic Cell Transplant (HCT) = Stem Cell Transplant etc

                                                                                                      Allogeneic

                                                                                                      Autologous

                                                                                                                          Allogeneic BMT Process

                                                                                                                          • · It is like planting a garden

                                                                                                                                              The Garden

                                                                                                                                              First you need to make room for a garden

                                                                                                                                                                  The Garden

                                                                                                                                                                  Then you have to plant the seeds

                                                                                                                                                                                      The Garden

                                                                                                                                                                                      Then you have to wait…

                                                                                                                                                                                                          The Garden

                                                                                                                                                                                                          You have to nurture the new plants... Water, Sun, Food, Love keep harmful things away

                                                                                                                                                                                                                              The Garden

                                                                                                                                                                                                                              … until they are able to survive on their own

                                                                                                                                                                                                                                                  So, What If We Want To Plant Stem Cells Instead of Flowers?

                                                                                                                                                                                                                                                                      Process

                                                                                                                                                                                                                                                                      • 1) Patient
                                                                                                                                                                                                                                                                      • 2) Donor
                                                                                                                                                                                                                                                                      • 3) Conditioning
                                                                                                                                                                                                                                                                      • 4) Graft (Stem Cells)
                                                                                                                                                                                                                                                                      • 5) Recovery

                                                                                                                                                                                                                                                                                          Allogeneic BMT in IEI

                                                                                                                                                                                                                                                                                          · Step 1: Patient

                                                                                                                                                                                                                                                                                          • - Which patients are appropriate to consider for allogeneic BMT?

                                                                                                                                                                                                                                                                                                              In General: 2 Requirements to Consider HCT

                                                                                                                                                                                                                                                                                                              • - #1 : The disease needs to be caused by a genetic disorder (known or unknown) that only or predominantly affects hematopoietic derived cells
                                                                                                                                                                                                                                                                                                              • - #2 : Patients should be at high risk of morbidity or mortality from their disease

                                                                                                                                                                                                                                                                                                              Ogonek et al, 2016

                                                                                                                                                                                                                                                                                                                                  A Lot to Balance!

                                                                                                                                                                                                                                                                                                                                                      Step 2: Find a Donor

                                                                                                                                                                                                                                                                                                                                                                          HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                              HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                                                  HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                                                                      HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                                                                                          HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                                                                                          Endogenous peptide

                                                                                                                                                                                                                                                                                                                                                                                                                                                                              HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                                                                                                              Patient

                                                                                                                                                                                                                                                                                                                                                                                                                                                                              Donor

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                  Source: IT-Ideatlon Department February 2021

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Step 3: Conditioning Regimen

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Transplant Center Conditioning Regimens

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Vs.

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Vs..

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      CCHMC

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Serotherapy A Chemotherapy ABC

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Serotherapy B Chemotherapy BC

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Serotherapy A Chemotherapy BCD

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      CHOP

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      GOSH

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                          Step 4a: Stem Cell Collection

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                              Step 4b: Stem Cell Infusion

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                              • · Infuse stem cell product into patient

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                      Thank you!

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                          Home

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                          Hematopoietic Stem Cell Transplantation for the Pediatric Hematologist[Oncologist Chapter

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                          Immune Reconstitution After Hematopoietic Stem Cell Transplantation

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                              HLA Typing and Donor Search

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                              National Marrow Donor Program?

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                              Donor List Report

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                              R

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                Why do we care about HLA match?

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                GVHD

                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                • - Remains the Biggest Barrier to Successful Outcomes
                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                https://www.aboutkidshealth.ca/article?contentid=1555&language=english#
BMT: How Does It Work and 
When Can It Be Considered?
Rebecca Marsh, MD
Cincinnati Children’s Ho…
1/33
Disclosures
• Employed part-time by Pharming Healthcare
• Advisory board or consulting - Amgen, A…
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)…
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…
15/33
– #1: The disease needs to be 
caused by a genetic disorder 
(known or unknown) that only or 
pr…
16/33
A Lot to Balance!
Transplant 
Risk
Immune 
Disorder 
Risk
Donor Options
Lung Disease
MAS Ne…
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…
23/33
HLA Typing and Donor Search
Patient
Donor
24/33
25/33
Step 3: Conditioning Regimen
Vs. Vs..
CCHMC CHOP GOSH
Transplant Center Conditioning Regimens
S…
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
…
33/33


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