The Anthony Nolan Trust

1. The Anthony Nolan Trust Pauline Makoni Operations Manager HTA 18.09.08

2. 1971 Anthony Nolan born with Wiskott-Aldrich syndrome 1973 first unrelated donor transplant performed (Seattle) 1974 first volunteer donor register, The Anthony Nolan Trust 1979 Anthony died The start of unrelated donor registries

3. Bone Marrow Registers NMDP 4,956,003 (82%) Germany 3,291,880 (51%) Anthony Nolan 392,558 (79%) Israel E.M. 374,340 (52%) Italy 352,666 (63%) Japan 312,061 (100%) BBMR 305,345 (91%) Taiwan 277,294 (86%) Others 2,179,243 Total 12,441,390 From Bone Marrow Donors Worldwide 26.08.08

4. Activities • Prototype for registers world wide • Maintain register of tissue types of prospective PBSC and BM donors • Aim to increase register to assist patients of all ethnicities, nationally and internationally, in finding a matched unrelated donor • 70% of all patients unable to find related donor • Target young adults and minorities • Donor advocacy • Unregulated locally other than JMAC till inception of HTA: ensures we aim to meet EU standards and boosts our local and international credibility

5. Donor Recruitment and Patient Searches Recruitment • Recruit adult volunteer donors (18-40) from the general population by running recruitment clinics around the country. Donation permitted up to 60 • Currently over 391,000 donors on our register • HLA type donors in our CPA and EFI accredited laboratories • 400 new donors HLA-A, B, C, DRB1 and CMV tested each week Patient Searches • Perform searches of registers and cord blood banks worldwide to find matching donors for patients requiring transplants • 1,260 new UK patients referred in 2007 (50% increase 2003-8) • 14,865 search requests received worldwide in 2007

6. Consent HTA is responsible for ensuring compliance to consent code and by an independent assessment process approves BM donations from children and adults. Premise for unrelated donations: consent gained from adults who are capable (as defined by HTA code on consent) of making informed decisions and that information is disseminated to donors on an ongoing basis by different methods (written, spoken) Consent is required at/for: • Registration: for initial HLA typing • CT: HLA typing and virology prior to final selection • Donation: at medical and day of donation – BM, PBSC and DL – covers collection, storage, discard etc • Cryopreservation: prior to collection if necessary • Research: ethics approved research studies

7. Information dissemination process ensures donors • are knowledgeable about procedures, risks and likely outcome for recipients • agree to be tested for infectious disease markers and understand results communication procedure • are willing to donate to an anonymous recipient anywhere in the world (anonymity maintained for a minimum of two years) • know they are free to withdraw at any time (the effect this will have on a patient at the work-up to donation stage is made clear) • voluntarily provide consent for altruistic donation • understand follow up procedures

8. Graft Selection Unrelated Donors (HPC-M and HPC-A) • New blood samples: Confirmatory typing and virology on potentially matched donors • 4538 donors tested • 2007: 484 work ups received • 368 MUDs to patients (221 UK/Ireland and 147 overseas) • 241 donations imported Cord blood units • Initially to treat aplastic anaemia - 1988 • 212 cords imported to date for single and double tx (77 in 2007)

9. International Activity • regulates and coordinates registry activities and standards • over 120 000 potential recipients, approx. 16 000 new searches each year • 42% of world wide unrelated donor transplants are performed using donors from another country • 3,029 patients received HPC-M (BM) • 5,974 patients received HPC-A (PBSC) • 1,350 patients received HPC-C (cord blood) (World Marrow Donor Association 2007 statistics)

10. HPC provided by ANT to overseas patients in 2007 (147) Patients in 23 countries received donations from an Anthony Nolan Trust donor -62% BM/PBSC exported stayed within EU, -28% to North America -10% elsewhere

11. Source of imported haematopoietic stem cells for UK patients in 2007 - Total 318 UK patients received transplants from donors from 20 different countries -59% from EU countries -35% from North America -6% from elsewhere

12. Cord Blood Bank and Research Institute Cord Blood Bank • Collection at King’s College, London • Processing and storage at CBB in Nottingham • Placed on Anthony Nolan register • Research alternative uses of cord blood Research Institute • HLA: to improve tx outcomes • G-CSF: proof of safety

13. Accreditation and Regulation • Registry WMDA accredited. International donor registries must ensure that the organisations they work with comply with standards which are in keeping with those of the World Marrow Donor Association, HTA, JACIE • Importation/exportation from registers compliant with WMDA standards to transplant centres with acceptable transplantation credentials • EU Tissues and Cell Directive (EUTCD) regulates international activity: implemented into UK law via Quality and Safety Regulations and regulated in UK by HTA (facilitates tracing of tissue and cell donations, cross border activity and safeguards quality). • Laboratories: CPA and EFI accredited • Collection/Transplant centres: JACIE accredited • HTA: 2 licences - registry (incl. activity of labs, CCs, TCs) and cord blood bank - regulates testing, storing, processing, use and distribution of human tissue or cells used in treatment - Serious Event/Accident Report (SEAR) Ensures any trends identified early and investigated for benefit of the donor

14. Registered Charity No. 803716 / SC038827 www.anthonynolan.org.uk