Elsevier

The Lancet Haematology

Volume 2, Issue 3, March 2015, Pages e91-e100
The Lancet Haematology

Articles
One million haemopoietic stem-cell transplants: a retrospective observational study

https://doi.org/10.1016/S2352-3026(15)00028-9Get rights and content

Summary

Background

The transplantation of cells, tissues, and organs has been recognised by WHO as an important medical task for its member states; however, information about how to best organise transplantation is scarce. We aimed to document the activity worldwide from the beginning of transplantation and search for region adapted indications and associations between transplant rates and macroeconomics.

Methods

Between Jan 1, 2006, and Dec 31, 2014, the Worldwide Network for Blood and Marrow Transplantation collected data for the evolution of haemopoietic stem-cell transplantation (HSCT) activity and volunteer donors in the 194 WHO member states.

Findings

953 651 HSCTs (553 350 [58%] autologous and 400 301 [42%] allogeneic) were reported by 1516 transplant centres from 75 countries. No transplants were done in countries with fewer than 300 000 inhabitants, a surface area less than 700 km2, and a gross national income per person of US$1260 or lower. Use of HSCT increased from the first transplant in 1957 to almost 10 000 by 1985. We recorded a cumulative total of about 100 000 transplants by 1995, and an estimated 1 million by December, 2012. Unrelated donor registries contributed 22·3 million typed volunteer donors and 645 646 cord blood products by 2012. Numbers of allogeneic HSCTs increased in the past 35 years with no signs of saturation (R2=0·989). Transplant rates were higher in countries with more resources, more transplant teams, and an unrelated donor infrastructure.

Interpretation

Our findings show achievements and high unmet needs and give guidance for decisions; to grant access for patients, to provide a donor infrastructure, and to limit overuse by defining risk and region adapted indications for HSCT as an efficient and cost-effective approach for life-threatening, potentially curable diseases.

Funding

Funding for this study was indirectly provided by support of the WBMT.

Introduction

WHO has recently recognised the broad idea of provision of medical products of human origin as an important global medical task.1 The transplantation of cells, tissues, and organs has extended the lifespan of hundreds of thousands of patients worldwide and enhanced their quality of life. As part of this success, the availability of products and procedures has decreased; expectations of patients in need have triggered organ trade and stem cell tourism. As a consequence, guidelines by WHO declare that regulation of transplantation on a national level is a governmental responsibility.1, 2, 3, 4, 5 Data collection and data analysis have been recognised as integral parts of the treatment to achieve efficient and cost-effective use of resources. Harmonised data for use and trends worldwide are a key prerequisite.6, 7 Information about how to best support introduction of HSCT and how this technique has spread remains scarce. Traditional methods of health technology assessment are likely to fail in view of the vast heterogeneity of approaches and small number of transplantation studies being done.8, 9

Haemopoietic stem-cell transplantation (HSCT) is an established but complex treatment for selected patients with severe congenital or acquired disorders of the haemopoietic system10, 11, 12 and some other life-threatening disorders. Achievements with HSCT have stimulated research in the biology, property, and functions of stem cells, and treatment of cancer generally. Novel technologies to generate, expand, and maintain stem and precursor cells create hope for custom tailored stem cells. Ideas for use range from single organ disease to trauma repair or partial organ replacement.13, 14, 15, 16 HSCT has evolved from experimental bone marrow transplantation more than half a century ago to an accepted and successful treatment; therefore, the long experience and past errors and successes might serve to improve access for all patients in need and as guidance for the application of medical products of human origin generally.

The Worldwide Network for Blood and Marrow Transplantation (WBMT), an HSCT umbrella organisation recognised by WHO as a non-governmental organisation, has taken up the task of helping with HSCT worldwide. On the occasion of the one millionth transplant at the end of 2012, we present the worldwide diffusion of HSCT from its beginning up to now.17

Section snippets

Patients and study design

In this retrospective observational survey we analysed the number of HSCTs worldwide from the first published series (to the best of our knowledge) of bone marrow transplants in 195718 to Dec 31, 2012 (table 1). Data for HSCT were collected by WBMT through its network of international or regional member organisations; data for registered unrelated donors, on registered cord blood products and on their use were collected through the World Marrow Donor Association. The organisations providing the

Results

Numbers of HSCT increased after an initial period of limited activity continually to 10 000 HSCTs worldwide by 1985, to roughly 50 000 by 1991, to about 100 000 by 1995, to roughly 500 000 by 2005, and doubled to about 1 million by December, 2012 (table 1 and figure 1A). When HSCT first started, this technique was restricted to a few countries (appendix pp 4–11). By the end of 2012, HSCT had spread slowly but steadily to 75 countries; 35 started with allogeneic HSCT, 23 with allogeneic and

Discussion

These global findings show striking differences in absolute transplant numbers, frequency of transplant, and spread of HSCT. The different patterns are affected mainly by macroeconomic factors related to availability of resources and infrastructure. We noted major differences in use of autologous and allogeneic HSCT (panel). Global numbers of allogeneic HSCT increased with no signs of saturation but substantial differences between regions were present. Of note, increase in absolute numbers was

References (46)

  • M Sleeboom-Faulkner

    Experimental treatments: regulating stem-cell therapies worldwide

    Nature

    (2013)
  • WHO guiding principles on human cell, tissue and organ transplantation

    Transplantation

    (2010)
  • M Aljurf et al.

    Challenges and opportunities for HSCT outcome registries: perspective from international HSCT registries experts

    Bone Marrow Transplant

    (2014)
  • T Stafinski et al.

    Funding the unfundable: mechanisms for managing uncertainty in decisions on the introduction of new and innovative technologies into healthcare systems

    Pharmacoeconomics

    (2010)
  • SR Tunis et al.

    Coverage options for promising technologies: Medicare's ‘coverage with evidence development’

    Health Aff (Millwood)

    (2006)
  • EA Copelan

    Hematopoietic stem-cell transplantation

    N Engl J Med

    (2006)
  • P Ljungman et al.

    Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe 2009

    Bone Marrow Transplant

    (2010)
  • MC Pasquini et al.

    2010 report from the Center for International Blood and Marrow Transplant Research (CIBMTR): current uses and outcomes of hematopoietic cell transplants for blood and bone marrow disorders

    Clin Transpl

    (2010)
  • DR Higgs

    A new dawn for stem-cell therapy

    N Engl J Med

    (2008)
  • I Martin et al.

    The survey on cellular and engineered tissue therapies in Europe in 2011

    Tissue Eng Part A

    (2014)
  • MN Hirt et al.

    Cardiac tissue engineering: state of the art

    Circ Res

    (2014)
  • Media fact sheet: 1 million blood stem cell transplants Worldwide

  • EE Osgood et al.

    Aplastic anemia treated with daily transfusions and intravenous marrow; a case report

    Ann Intern Med

    (1939)
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    Dr Blume died on Jan 9, 2013

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