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

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

• 25.

• 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#