The South African Bone Marrow Registry Successes and

The South African Bone Marrow Registry Successes and challenges: Overcoming Obstacles to Stem Cell Transplantation and delivering on a diverse stem cell registry in South Africa and Africa Dr Charlotte Ingram

SABMR Team Terry Schlaphoff Deputy Director Alicia Venter Search Coordinator Jane Ward Harvest Coordinator Nadia Chalkley Donor Recruitment Lisa Terblanche Search Assistant Gill Bohlmann Accounts

SABMR - Background § § Established in 1991 Non-profit Organisation Board 11 Members 9 Non Exec 2 Exec Hub Centre for SA – Liaison between 137 registries world-wide – Provides MUDs for stem cell transplantation § Facilitated 1 st matched unrelated donor (MUD) bone marrow transplant in South Africa 1997 § Donor recruitment – Past = Sunflower Fund – Present = SABMR

SABMR - Activities § Stem Cell Transplantation offered in 3 regions § Total number of SA patients transplanted – 497 § Total number of SA donors donating stem cells – 162 - For SA patients – 130 - For international patients – 32 – include some siblings

SABMR - Successes § Internationally recognised registry - approved member of the World Marrow Donor Association (WMDA) – Guidelines for the collection and transfer of stem cells § Submits donor data to the WMDA Search & Match Service (WSMS) database – > 34 million donors and cord blood units – Internet search engine § Awarded full WMDA Accreditation Status June 2018 – Achieved in Record Time of 2 years § Started own donor recruitment in Nov 2017 – WMDD Grand Prize 2018

Belgium 4 Netherlands 3 Spain 1 Germany 220 Norway 2 Switzerland 3 Denmark 1 Austria 1 Poland 9 Russia 1 Canada 4 United Kingdom 38 USA 54 France 14 Portugal 2 Taiwan 3 Italy 7 Cyprus 2 Brazil 3 India 1 Israel 7 Singapore 1 South Africa 130 Donors for South African patients Australia 4

3 Major Transplant Challenges Committed Donor Access to Funds Time

3 Major Transplant Challenges Donor recruitment vs. donor attrition 5000 Committed Donor 4500 4000 3500 3000 New 2500 New Deleted 2000 1500 1000 500 0 2011 2012 2013 2014 2015 2016 2017 2018

3 Major Transplant Challenges Committed Donor § Planned donor recruitment vs patient specific donor drives - Aaron Lipschitz - Over 300 applications - Donor identified as match - Local Patient

The Future • Increase donors - Ethnic Diversity • Overcome cultural issues • National footprint

The STATS Ethnicity: 31 December 2018 5. 9% 10% 9. 3% 7. 8% Asian / Indian Black Coloured White Other / Unknown 67%

The STATS • Match 1: 100 000 • More likely within own ethnic group • 2018 – 229 Preliminary Search Requests – 52% searches for Asian, Black and Coloured patients – Only three out of the 40 transplants that year

3 Major Transplant Challenges Committed Donor Global SABMR WMDA STATS 2018

3 Major Transplant Challenges Committed Donor Percentage of Donors Deleted due to Emigration 5. 0% 4. 0% 3. 0% SABMR 2. 0% Global 1. 0% 0. 0% 2010 2011 2012 2013 2014 2015 2016 2017 2018

3 Major Transplant Challenges Committed Donor § Since 2018 the SABMR recruits all of its own donors § Current recruitment initiatives by online platform and donor drives § Frequent contact with all registered donors § Involve them in education and awareness campaigns § § No cost to donor when identified as match, we only ask their time 10 year medical follow-up post donation Possibility to meet their patient after 3 years with mutual consent SABMR strategic goal to increase size of database § ~73 000 tot 90 000 by 2021

Ste m har cell ves t cal edi or m t lan nsp Tra ent em cur Pro Sea Act rch ivat ion Pre Sea limina rch ry req ues t Time Don Ex typ tende ing d Rou HLA nd 1 Ext e n typ ing ded H Rou LA nd 2 Ext e typ nde ing d Rou HLA nd 3 Ma n Ve dat typ rificat ory ing i @ Hon R ited HLA patie typ nt ing Ful l pa tien t. H typ LA ing Lim 3 Major Transplant Challenges *depending on donor profile Prior to 2018: 6 to 8 months

cal est mc ell h arv edi or m Don Ste tien t. H typ LA ing l pa Ma Ver ndato typ ificat ry ing i @ Hon R Ful Tra ent nt nsp la em cur Pro Sea Act rch ivat ion t Pre Sea limina rch ry req ues 3 Major Transplant Challenges Time *depending on donor profile Post 2018: 3 to 4 months

3 Major Transplant Challenges Access to Funds § Donor related costs: § Local donor = R 200 000 – R 300 000 § International donor = R 400 000 – R 800 000 § Medical aid often only covers the cost of a SA donor § SABMR Patient Assistance Program § Financial assistance for South African citizens in need of a MUD SCT and who do not have the financial resources to formally activate their donor search. § Launched in November 2018 – already funded 5 successful MUD SCTs

Registry Challenges § Financial § No state funding § Cost recovery for services not sufficient ∴ Sustainability Plan § Cost of HLA typing § HLA typing from buccal swabs performed in USA § Poor ethnic diversity § Recruited volunteers to help address the issue § Alternative therapies § CAR T-cell and Haploidentical transplantation § Still require MUD BMT support § Decline of donor database noted in 2016 § SABMR started doing it’s own donor recruitment § Implemented a new business model to enable rapid growth

Where do we want to be? As per WHO Haematopoietic Stem Cell Transplantation is: “A routine treatment without equivalent” that is ACCESSIBLE TO ALL IN NEED OF BMT!

Transplant Programmes in SSA �South African �Continuously expanding from 1 to 3 Paediatric �Adult from 5 in 2016 to 12 in 2019 �SABMR (45 MUD transplants/Yr) �Nigeria � 4 Transplants over 5 -6 Yrs �Ghana � 5 Transplants – Sickle Cell only �Kenya �Mainly Sickle Cell - Family & Haploidentical referred to India or SA - cost related

Stem Cell Registries in SSA �South African Bone Marrow Registry (SABMR) � 73 154* �Bone Marrow Registry Nigeria (BMRN) � 924* �The Sunflower Fund � 1 039* �Other - Ghana donor panel �Above excludes EMR Countries- Middle East, North Africa, the Horn of Africa and Central Asia • (21 Member States in Total) *WMDA: 6 August 2019

BMT Options for African Patients �Many African patients in need of a BMT are currently being referred to transplants units in India and South Africa �EMR/AFR High frequency Allogeneic compared to Autologous & almost exclusive use of family donors compared to Global �EMR/AFR tend to use Haploidentical donors rather than cord blood

Current Status of SABMR �SABMR Family Donor Assistance for Africa Kenya Somalia Ghana Nigeria

How could we get there? PUtilisation and expansion of existing services offered by partners PBetter access & more resources for patients in need of transplant PTraining and support programmes in the establishment of new BMT units PEmbrace new developments and treatments PEducation of Health Ministries

Online Recruitment Portal • June 2018 – new website launched

Making it all Worthwhile

“Dear anonymous, “ We know we don’t know who you are, but we know what you are – a hero. You gave our child another chance to lead a normal life. A chance to grow up, learn, play and experience everything this world has to offer. ……. . . Since the transplant almost a year ago, your bone marrow is now their bone marrow. Just know that you are now one with our child, always. A gift that keeps on giving. The world we currently live in is scary, but at the same time it’s beautiful, because there are people like you walking on it. We truly hope that one day we would be able to meet you and thank you in person. We want our child to meet you. We want them to meet the person who saved their life. We want them to know that there are good people on this Earth so that one day, they could be a hero for someone else. Again, thank you for everything you have done. Even though there are over 7 billion people on this Earth, it seems we are all closer than we think. “

Thank you Donors!