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

- · Employed part-time by Pharming Healthcare
- · Advisory board or consulting - Amgen, AB2Bio, SOBI
- · They were all too inflammatory!
- · For today: Bone Marrow Transplant (BMT) = Hematopoietic Cell Transplant (HCT) = Stem Cell Transplant etc
- · It is like planting a garden
- 1) Patient
- 2) Donor
- 3) Conditioning
- 4) Graft (Stem Cells)
- 5) Recovery
- - Which patients are appropriate to consider for allogeneic BMT?
- - #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
Disclosures

I Failed to Find a Good Rheumatology Joke to Open Withâ¦

I Failed to Find a Good Rheumatology Joke to Open Withâ¦

BMT: How Does It Work?

BMT: Terminology

Allogeneic
Autologous


Allogeneic BMT Process







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 waitâ¦

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






Allogeneic BMT in IEI

· Step 1: Patient
In General: 2 Requirements to Consider HCT


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