1. New Approaches for Transplant Patients Linda J Burns, MD Medical Director, Health Services Research Program National Marrow Donor Program (NMDP)/Be the Match May 21, 2016 Leukemia Research Foundation 1

2. Learn About Be The Match Types of transplants When to consider an unrelated donor transplant Finding an unrelated donor How Be The Match can assist you A look to the future 2

3. Be The Match: Dedicated to Patients

4. Be The Match: People Helping People 4

5. Types of Transplants and Cell Sources There are two types of transplant – autologous and allogeneic There are three sources of stem cells for transplantation: bone marrow, peripheral blood stem cells (PBSC), and cord blood 7 Operated by the National Marrow Donor Program ®.

6. Total Number of Transplants Pasquini MC, Zhu X. Current uses and outcomes of hematopoietic stem cell transplantation: CIBMTR Summary Slides, 2015.

7. Reasons for Transplant

10. The Transplant Process 15 Operated by the National Marrow Donor Program ®.

11. Reduced-intensity Regimen (non-myeloablative or “mini”) Lower doses of chemotherapy with or without radiation –Main goal: suppressing the recipient immune system to allow donor cells to engraft Works for patients with: –Slower growing diseases –Who are less likely to tolerate the side effects of a high dose transplant Primary benefit of this type of transplant is the immune effect: Graft vs. Leukemia/Lymphoma 18 Operated by the National Marrow Donor Program ®.

12. Standard-intensity Regimen (myeloablative) Higher doses of radiation/chemotherapy administered with goal of killing ALL of the patient’s stem cells and diseased cells Works for patients with: –More aggressive diseases –Who are younger and overall healthy Usually causes more side effects 17 Operated by the National Marrow Donor Program ®.

13. Allogeneic Transplant Family donor Unrelated donor 13

14. When to Consider an Unrelated Donor Transplant When you need an allogeneic transplant and a family member is not able to donate for you Outcomes comparable to family donor for many diseases –Advances in HLA typing to identify the “best” match –New preparative regimens –Improved supportive care 14

15. How an Unrelated Donor or Cord Blood Unit Is Found Your transplant center is responsible for searching for a donor or cord blood unit(s) Be The Match Registry –Includes 12.5 million donors and 209,000 cord blood units –Has access to 25 million potential donors and more than 622,000 cord blood units on U.S. and global registries 12 Operated by the National Marrow Donor Program ®.

16. HLA: Genetic Identity Human Leukocyte Antigens (HLA) are proteins found on the surface of most cells in the body The immune system uses HLA to verify that a given cell is part of the body and not foreign Finding a suitably-matched donor is important to reduce the risk of post-transplant complications There are many different variations of the HLA molecules (HLA-A, -B, -C, -DRB1, -DQ, -DP) 8 Operated by the National Marrow Donor Program ®.

17. HLA Haplotypes Possible sets of HLA combinations, termed “haplotypes,” are in the billions –Similar to a set of lottery numbers –Everyone gets one haplotype from each parent Haplotypes often follow ethnicity (but not always) 9 Operated by the National Marrow Donor Program ®.

18. Diversity of Be The Match Registry 13 Operated by the National Marrow Donor Program ®.

19. NMDP: How We Can Assist You Confidential one-on-one support and navigation Tailored educational materials Financial resources Fundraising information Insurance appeals Peer-to-peer connection Caregiver support 24 Operated by the National Marrow Donor Program ®.

20. Pre-Transplant Patient Materials Booklets: –Transplant Basics –Allogeneic Transplant: How to plan and what to expect Fact sheets: –Basics of Blood or Marrow Transplant –Allogeneic Transplant Process –Autologous Transplant Process –Preparing for Transplant –Being a Transplant Caregiver Videos: –Basics of Blood and Marrow Transplant

21. Post-Transplant Patient Materials Fact sheets on chronic GVHD –Eyes, lungs, mouth, skin & connective tissues –Available summer 2016: GI tract, genitals –Developed with experts from the Chronic GVHD Consortium Living Now newsletter series –Magazine series with 6 issues plus special caregiver issue –Mailed automatically to patients who receive an NMDP facilitated transplant –Available to order for others

22. Post-Transplant Guidelines 22 Operated by the National Marrow Donor Program ®. Mobile app –Customizable 6-month, 12-month and 2+ year checkup guidelines –Chronic GVHD symptom checker –Set reminders (appointments, medications, etc.) –Search “transplant guidelines” in app stores to download Also available online and print at BeTheMatch.org/patient-after

23. Free Webcasts for Patients and Families Living Now: –Taking Care of Yourself –Your Role in Managing Your Chronic GvHD –Staying Healthy After Transplant –Your Journey Ahead –Making Meaning and Moving Forward Visit: BeTheMatch.org/careguide 23

24. Look to the Future Research will enable: Every patient who needs a transplant to receive one when they need it More patients to be cured of their disease Us to understand and improve outcomes that matter most to patients 24

25. Take Home Points Be The Match helps transplant centers identify unrelated stem cells (donor or umbilical blood) for their patients in need of a transplant Survival following an unrelated transplant is similar to a transplant using a family member as a donor Be The Match has many programs to help you and your family through the transplant journey The future is bright for patients needing a transplant 25