1. STEM CELL DONOR REGISTRIES A. Vanhonsebrouck, MD E. Baudoux, MD 25/04/20121ARMB-KAGB

2. Hematopoietic stem cell transplantation (HSCT) has become an accepted therapy for many congenital or acquired disorders of the hematopoietic system and has seen major changes in indications and use of transplant techniques over the years. 25/04/2012ARMB-KAGB2 EBMT Annual report, 2010

3. Principle 25/04/2012ARMB-KAGB3 Blood RBC Platelets WBC Bone Marrow

4. Stem cell sources 25/04/2012ARMB-KAGB4 PBSCBMCB

5. Brief History Decade Event 1950 Survival of irradiated animals after letal irradiation+infusion BM First attempts humans : treatment of hematological diseases (Mathé-France, Donnall Thomas-USA) 1960Failure of BM transplantation due to severe adverse reactions 1970 First studies of BM transplantations in leukemic patients with HLA matched Donors Hematopoietic progenitors in Cord Blood (CB) First BM Donor Registries 1980 Nobel Prize for the HLA system: Jean Dausset (France) First CB transplantation: E. Gluckman (France) Development of BM Donor Registries 1990 Nobel Prize for transplantation of organs/ cells: D. Thomas (USA) Considerable growth of BM Donor Registries and CB Banks 2000Steep increase in use of unrelated donor stem cells (CB-donors) 201019 Million donors available -0.5 Million CBU 25/04/2012ARMB-KAGB5

6. Human Leucocyte Antigens 25/04/2012ARMB-KAGB6 Father Mother Children

7. Donors –CBU worldwide 25/04/2012ARMB-KAGB7 BMDW:19 Million BMDW:0,5 Million BMDW : Bone Marrow Donors Worldwide WMDA : World Marrow Donor Association

8. MDPB-REGISTRY (Marrow Donor Program Belgium) HISTORY : 1989 Belgian Stem Cell Donor Registry ADDRESS : MOTSTRAAT 40, 2800 MECHELEN COMPOSITION MDPB-REGISTRY TASK: FINANCIAL – ADMINISTRATIVE MANAGEMENT STAFF: DIRECTOR: A. VANHONSEBROUCK Adjunct Director + 2 Search coordinators MDPB-VZW/ASBL TASK: MEDICAL AND SCIENTIFIC ADVISE PRESIDENT : E. BAUDOUX Representatives of Transplant Center (TC), Donor Center (DC), Cord Blood Bank (CBB) and Collection Center (CC). 25/04/2012ARMB-KAGB8

9. MDPB-REGISTRY Goals To provide stem cells from an HLA identical unrelated donor to national/international patients Stem cell transplantation for patients with hematological diseases (malignant and non-malignant) o Related : familial transplantation: 1/4 perfect match o Unrelated : 1/50.000 perfect match National Collaboration o 9 donor centers/ 10 transplant centers/ 5 cord blood banks/ 5 collection centers Part of international collaboration o BMDW : 19 million donors – 500.000 CBU o Cooperation with 65 stem cell donor Registries – 44 Cord Blood Banks 25/04/2012ARMB-KAGB9

10. MDPB-REGISTRY 25/04/2012ARMB-KAGB10 4 56972 8275 65 18 500000 2011

11. MDPB-REGISTRY 25/04/2012ARMB-KAGB11 2011 MDP-B Registry (Syrenad) Unrelated donors Belgian Patients 5 Cord blood banks >16 000 CBUs 7 donor centers >60 000 Donors 9 Transplant centers (national patients) Internet EMDIS Searching for national donors Searching for International donors 65 INTERNATIONAL REGISTRIES 44 CB BANKS >18 M DONORS>500 000 CBUS

12. MDPB-R Donor inventory 25/04/2012ARMB-KAGB12 N = 60346 Donors

13. MDPB-R Cord blood inventory 25/04/2012ARMB-KAGB13 N=16088 CBU

14. MDPB-R Search-Transplant activity 25/04/2012ARMB-KAGB14 BELGIAN PATIENTS

15. MDPB-R Transplant activity 25/04/2012ARMB-KAGB15

16. MDPB-R Transplant activity 25/04/2012ARMB-KAGB16

17. MDPB-R SWOT analysis 25/04/2012ARMB-KAGB17 S TRENGHTS WELL-INTEGRATED NETWORK between partners ( DC, CBB,TC, Col.C, HLA lab) Connected to REGISTRIES WORLDWIDE ACTIVE participation with INTERNATIONAL GROUPS (WMDA, Netcord) W EAKNESSES SIZE (international context) LOW INSTITUTIONAL support MOST DONORS ARE FROM ABROAD (for transplantations of Belgian Patients) Potential solutions reachable Cancer plan Official mandate to operate O PPORTUNITIES ACCREDITATION Investment in NEW DATA EXCHANGE TOOLS INCREASE INTERNATIONAL COOPERATION NEW RECRUITMENT STRATEGIES (fe minorities) CONTINUOUS RECRUITMENT ACTION T HREATS AGE/ HLA TYPING level of donor population BIGGER transnational structures with ample funding MORE BELGIAN STEM CELL DONORS (for Belgian-international Patients)

18. MDPB-R PROJECTS 1.Donor Recruitment Action Plan MDPB-R PROJECTS 1.Donor Recruitment Action Plan SITUATION before 2008 - number of donors : 45.000 - donor data base is aging (drop-off >60 y) - data HLA typing is poor/ median age: 45-50 y CONTRACT with RIZIV-INAMI - 5 years (2008-2013) : 10.000 new donors - HLA typing better quality : molecular methods (A,B,C,DR) - Recruitment campaigns donor centers and transfusion centers ACTUAL RESULTS -SUCCESS ! Total amount : 60346 new donors 12.627 CHALLENGE : CONTINUOUS RECRUITMENT AFTER 2013 25/04/2012ARMB-KAGB18

19. Donor Recruitment action Plan 25/04/2012ARMB-KAGB19

20. MDPP-R PROJECTS 1.Donor Recruitment Action Plan 25/04/2012ARMB-KAGB20 RKVL : ANT+BTOSJB : St Jan BrugesVUB : UZ Brussel GOD : Mont GodinneSFS : Service du Sang KUL : UZ Leuven

21. MDPB-R PROJECTS 2. CANCER PLAN - CANCERPLAN launched in 2008 – actions were defined -STRUCTURAL SUPPORT OF TISSUE BANKS FOR CELL THERAPY and THERAPY with HSC and CB by financing (personnell – storage – general costs) 13 hospitals receive funding -MEETING MDPB-R and delegates of CANCER CENTRE 2011 25/04/2012ARMB-KAGB21 NEW PROPOSALS – EVALUTION FOLLOWS Optimizing test capacity of TISSUE TYPING LABORATORIES RECRUITMENT CAMPAIGNS to sensitize stem cell donors Improve the POSITION of the stem cell donor COOPERATION between MDPB-R, MDPB-vzw and BHS Financing the TRANSPLANT REGISTRY:central database with followup of patients

22. MDPB-R PROJECTS 3. IT APPLICATION -January 2008 : Software SYRENAD (FGM - France) webbased application for exchange of information between centers (Donor Centre,TC, CBB, Collection Centers) and Registries -EMDIS (European Marrow Donor Information System) (15 countries ) electronic communication between MDPB/intern.Registries encrypted secured email based communication system -PROMETHEUS : FUTURE SOFTWARE APPLICATION already used in more than 10 registries EMDIS : connection with 27 countries 25/04/2012ARMB-KAGB22

23. IT application – Syrenad -Prometheus Global search: 28 Emdis connections US NMDP US Caitlin Canada South Africa Taiwan Cyprus Czech (Plzen) Poland Slovakia SYRENAD: 15 Denmark BBMR Finland Israel Australia PROMETHEUS : + 12 EMDIS connections EUROPE :Germany,Switzerland,Spain, France,Italy, Netherlands,UK-Wales, Austria, Sweden, Norway, Czech R. 25/04/201223ARMB-KAGB

24. MDPB-R PROJECTS 4. MEDIUM SIZED REGISTRIES -Medium Sized Registries : 20.000-100.000 donors -countries : Switzerland, Belgium, Sweden, Spain, Cyprus, Norway, Finland, Czech Republic, Ireland, Wales, Austria, Netherlands, Denmark -First meeting sept 28, 2011 -Purpose : maintain and strengthen viability of « medium sized » registries by increasing efficiency and diversity in a situation with enhanced needs and limited resources 25/04/2012ARMB-KAGB24 ACTION PLAN : PHASE IPHASE II Improve recruitment of suitable donorsIT Indicators for benchmarkingEU application HLA typing, strategy, efficiencyQuality Management Refining CT work upHLA expertise Regulatory Issues

25. THANK YOU ! MORE INFO 25/04/2012ARMB-KAGB25 Annual report 2011 will be available on the website www.infodonor.be end of June 2012