WHO- Global perspective of hematopoieitic stem cell transplantation

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1 WHO- Global perspective of hematopoieitic stem cell transplantation José R. Núñez MD, PhD WHO Dietger Niederwieser MD University Hospital, Leipzig, Germany

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6 Over the past 50 years, the transplantation of human organs, tissues and cells has become a worldwide practice which has extended and greatly enhanced the quality of hundreds of thousands of lives. 6

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9 9 Patient Safety Programme

10 Advances in the Field of HCT Liver-Tx and Auto BMT Graft-versusbreast cancer Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization mod. from Appelbaum NEJM 2007

11 Societies active in early Stem Cell Transplantation Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization

12 Global Donation Central database Standards Accreditation Policies Education Worldwide Network for Blood and Marrow Transplantation

13 Percentage stem cell donations provided for national and international patients 75.0 Percentage Year Stem Cell Donations International Provided Stem Cell Donations National Provided Worldwide Network for Blood and Marrow Transplantation

14 Article 1 of WHO Constitution: "The attainment by all people of the highest possible level of health."

15 15 Patient Safety Programme

16 Foundation of WBMT Lyon 2007 Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization

17 First Meeting of the WBMT Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization

18 18 Patient Safety Programme

19 Role of Global and Regional Scientific and Professional Societies in the Global Governance of HSCT Developing and monitoring global codes of practice about ethical issues along the Guiding Principles Promoting global good practice in relation to quality and safety Advocating for globally harmonized practices such as accreditation and use of consistent information standards In collaboration with all stakeholders Monitoring regional and global access Monitoring outcome data Contributing to vigilance and surveillance And ensure transparency 19 Patient Safety Programme

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22 Foundation of WBMT Lyon 2007 by Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization

23 Latin American Blood Marrow Transplantation Group (LABMT) 23 Patient Safety Programme

24 Foundation of the Latin America Blood and Marrow Transplantation Group (LABMT) Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization

25 Latin American Blood Marrow Transplantation Group (LABMT) African Blood and marrow Transplantation Group (AFBMT) 25 Patient Safety Programme

26 Foundation of the African Blood and Marrow Transplantation Group (AfBMT) Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization

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28 Latin American Blood Marrow Transplantation Group (LABMT) African Blood and marrow Transplantation Group (AFBMT) Meetings (Hanoi; Salvador; Cape Town) 28 Patient Safety Programme

29 Increasing access to the appropriate therapy Increasing access to the appropriate therapy 29 Patient Safety Programme

30 Latin American Blood Marrow Transplantation Group (LABMT) African Blood and marrow Transplantation Group (AFBMT) Meetings (Hanoi; Salvador; Cape Town) Twining (Mongolia; Philippines; Qatar; Myanmar) Dissemination 1 million SCT; global press release Annual Survey with biannual publication (2006, , 1 million) 30 Patient Safety Programme

31 WBMT Global Survey Allogeneic HSCT Autologous HSCT Total HSCT 31 Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization Patient Safety Programme HB:Sept 2016

32 WBMT: number of reporting teams per country The Americas Eastern Mediterranean/Africa Europe South East Asia/Western Pacific Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Feb 2016

33 WHO Regions 4 Countries Global Survey 2013 data Teams Year of Transplant reporting Patients allogeneic (47%) autologous (53%) Donor type family (49%) sibling/twin haplo-identical relative (23%) unrelated (51%) cord blood Worldwide Worldwide Network for for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Jan 2017

34 WBMT Global Survey Increase in the numbers of transplants and reporting teams Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Jan 2017

35 Global Activity Survey: Allogeneic Autologous Acute Leuk/other leuk/mds/mps % Chronic Leuk Lymphoproliferative disorders % Solid Tumors Non Malignant disorders % BMF % Others Total % Leukemias % PCD % Lymphomas % Solid Tumors % Non Malignant disorders % Others Total % Total % Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Jan 2017

36 Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Jan 2017 Transplant Type by Region: 2013 Main indication Allogeneic HSCT Autologous HSCT Total Europe (47%) The Americas (30%) South East Asia / Western Pacific Eastern Mediterranean / Africa (20%) (3%) Total (47%) (of 79) reporting countries contributing teams

37 Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Jan 2017 WBMT Global Survey Changes in donor choice Unrelated incl. CB Family Unrelated CB Haploidentical 3000 H S C T H S C T

38 Transplant Rates in World Regions TR HSCT /10 million inhabitans Family Unrelated Allo Auto Total LABMT EUROPE USA/Canada APBMT EMRO Total worldwide (median) Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Feb 2016

39 GNI/capita, HCE/capita and transplant rate ranking of Latin American countries performing HSCT. Venezuela Argentina Brazil Uruguay 6 4 Chile 2 GNI/cap Peru 0 Colombia HCE/cap TR tot Paraguay Costa Rica Panama Ecuador Mexico Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization HB: Feb 2016

40 WBMT Global Survey: Unrelated grafts 40 Worldwide Network for Blood and Marrow Transplantation NGO in official relations with World Health Organization Patient Safety Programme

41 Latin American Blood Marrow Transplantation Group (LABMT) African Blood and marrow Transplantation Group (AFBMT) Meetings (Hanoi; Salvador; Cape Town) Twining (Mongolia; Philippines; Qatar; Myanmar) Dissemination 1 million SCT; global press release Annual Survey with biannual publication (2006, , 1 million) Legislation (Pharmacopoea) Pharmacovigilance (Notify library 41 Patient Safety Programme

42 Bologna Initiative for Global Vigilance and Surveillance BIG V&S 42 Dr Luc Noël EHT/CPR "Clinical Procedures" Patient Safety Programme

43 The NOTIFY Library # More than 900 documented case types can be searched in a structured way by organ, tissue or cell type and/or incident type # associated bibliographic references and expert analyses. # Reactions # Infectious transmissions of all types, malignancy transmissions, and genetic transmissions to transplant recipients and to the offspring of children born from donated gametes. # Events # caused by process failures and reactions in living donors # Site and search tools publicly available (without username and password) # Language specific interface in Regions. 43 Patient Safety Programme

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48 The Melphalan story / Shortage of freezing bags / Fukoshima accident

49 Latin American Blood Marrow Transplantation Group (LABMT) African Blood and marrow Transplantation Group (AFBMT) Meetings (Hanoi; Salvador; Cape Town) Twining (Mongolia; Philippines; Qatar; Myanmar) Dissemination 1 million SCT; global press release Annual Survey with biannual publication (2006, , 1 million) Legislation (Pharmacopoea) Pharmacovigilance (Notify library Model for other societies the champion WHO 49 Patient Safety Programme

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53 CARs: different approaches A. Fesnak et al, Nature 2016

54 Gene Therapy comes of Age Gene editing L. Yi et al, Biochimica et Biophysica Acta 2016

55 GP 5 Free donation and no purchase of human transplant as such, but cost &expenditures recoveryt GP 3 Maximizing DD Protecting LD GP 6 Promoting No advertising GP 7 Reasonability For transplant origin GP 8 Justifiable fees GP 2 Death No conflict GP 1 Consent DD GP 4 Protecting the incompetent GP 9 Equitable allocation GP 10 Monitoring long term outcomes. Quality and safety of procedures and products GP 11 Transparency, openness to scrutiny, anonymity 63 th World Health Assembly, 2010

56 56 Patient Safety Programme

57 57 Service Delivery Safety

58 Good governance mechanisms e.g. registries e.g. preventive measures to limit the demand e.g. Oversight of the process e.g. increasing donations e.g. including all stakeholders

59 Human Cell, Tissue and Organ Donation and Transplantation Necessity of a legal framework 59

60 60 Patient Safety Programme

61 Global Governance 61 Patient Safety Programme

62 Thank you 62 Worldwide Network for Blood and Marrow Transplantation Patient Safety Programme

63 63 Patient Safety Programme

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