Helen Baldomero

Hematopoietic Stem Cell Transplantation: A Global Perspective

CONTEXT Hematopoietic stem cell transplantation (HSCT) requires significant infrastructure. Little is known about HSCT use and the factors associated with it on a global level. OBJECTIVES To determine current use of HSCT to assess differences in its application and to explore associations of macroeconomic factors with transplant rates on a global level. DESIGN, SETTING, AND PATIENTS Retrospective survey study of patients receiving allogeneic and autologous HSCTs for 2006 collected by 1327 centers in 71 participating countries of the Worldwide Network for Blood and Marrow Transplantation. The regional areas used herein are (1) the Americas (the corresponding World Health Organization regions are North and South America); (2) Asia (Southeast Asia and the Western Pacific Region, which includes Australia and New Zealand); (3) Europe (includes Turkey and Israel); and (4) the Eastern Mediterranean and Africa. MAIN OUTCOME MEASURES Transplant rates (number of HSCTs per 10 million inhabitants) by indication, donor type, and country; description of main differences in HSCT use; and macroeconomic factors of reporting countries associated with HSCT rates. RESULTS There were 50 417 first HSCTs; 21 516 allogeneic (43%) and 28 901 autologous (57%). The median HSCT rates varied between regions and countries from 48.5 (range, 2.5-505.4) in the Americas, 184 (range, 0.6-488.5) in Asia, 268.9 (range, 5.7-792.1) in Europe, and 47.7 (range, 2.8-95.3) in the Eastern Mediterranean and Africa. No HSCTs were performed in countries with less than 300,000 inhabitants, smaller than 960 km(2), or having less than US

Risk Score for Outcome After Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Analysis

680 gross national income per capita. Use of allogeneic or autologous HSCT, unrelated or family donors for allogeneic HSCT, and proportions of disease indications varied significantly between countries and regions. In linear regression analyses, government health care expenditures (r(2) = 77.33), HSCT team density (indicates the number of transplant teams per 1 million inhabitants; r(2) = 76.28), human development index (r(2) = 74.36), and gross national income per capita (r(2) = 74.04) showed the highest associations with HSCT rates. CONCLUSION Hematopoietic stem cell transplantation is used for a broad spectrum of indications worldwide, but most frequently in countries with higher gross national incomes, higher governmental health care expenditures, and higher team densities.

Blood and marrow transplantation activity in Europe 1996

It was investigated whether the European Group for Blood and Marrow Transplantation risk score, previously established for chronic myeloid leukemia, could be used to predict outcome after allogeneic hematopoietic stem cell transplantation (HSCT) for hematological disease in general.

The EBMT activity survey: 1990-2010

Transplant activity by members of the European Group for Blood and Marrow Transplantation (EBMT) and related European teams is reported for 1996 by indication, donor type and stem cell source. Bearing in mind reports from previous years, the annual numbers of transplants for each indication, transplant rates for each participating country, changes in transplant rates by indication and changes in donor types and stem cell sources are described. A total 14 593 blood or marrow transplants, performed in Europe by 382 teams from 31 countries, were reported in 1996. Of these, 4393 (30%) were allogeneic and 10 200 (70%) were autologous transplants. Of the autologous transplants, 978 (10%) were bone marrow derived, 9222 (90%) from peripheral blood stem cells or combined bone marrow and peripheral blood stem cell transplants. Of the allogeneic transplants, 3252 (74%) were bone marrow and 1141 (26%) were peripheral blood stem cell transplants. Main indications in 1996 were leukemias with 4961 transplants (34%), 70% allogeneic and 30% autologous; lymphomas with 5505 transplants (38%), 6% allogeneic and 94% autologous; solid tumours with 3484 transplants (24%), 1% allogeneic and 99% autologous; non-malignant disorders with 643 transplants (4%), 92% allogeneic and 8% autologous. There are major differences between countries. Transplant rates per 10m inhabitants per country ranged from 0 to >500 (median 202 per 10 m inhabitants). The most pronounced increase since 1990 for new indications in autologous transplants was observed in multiple myeloma and carcinoma of the breast. These data reflect recent changes and present status of blood and marrow transplantation in Europe. They provide a basis for patient counselling and health care planning.

Severe events in donors after allogeneic hematopoietic stem cell donation

A total of 654 centers from 48 countries were contacted for the 2010 survey. In all, 634 centers reported a total of 33 362 hematopoietic SCT (HSCT) with 30 012 patients receiving their first transplant (12 276 allogeneic (41%) and 17 736 autologous (59%)). Main indications were leukemias: 9355 (31%; 93% allogeneic), lymphoid neoplasias specifically Non Hodgkin’s lymphoma, Hodgkin’s lymphoma and plasma cell disorders: 17 362 (58%; 12% allogeneic), solid tumors: 1585 (5%; 6% allogeneic) and non-malignant disorders: 1609 (6%; 88% allogeneic). There were more unrelated donors than HLA-identical sibling donors (53% versus 41%); the proportion of peripheral blood as stem cell source was 99% for autologous and 71% for allogeneic HSCT. Cord blood was primarily used in allogeneic transplants (6% of total) with three autologous cord blood HSCT being reported. The number of transplants has increased by 19% since 2005 (allogeneic 37% and autologous 9%) and continued to increase by about 1100 HSCT per year since 2000. Patterns of increase were distinct and different. The data show the development of transplantation in Europe since 1990, with the number of patients receiving a HSCT increasing from 4200 to over 30 000 annually. The most impressive trend seen is the steady increase of unrelated donor transplantation, in parallel to the availability of unrelated donors through donor registries.

Hematopoietic SCT in Europe 2013: Recent trends in the use of alternative donors showing more haploidentical donors but fewer cord blood transplants

The risk for donors of allogeneic hematopoietic stem cells transplants is generally considered negligible. This study shows that hematopoietic stem cell donation is associated with a small but definite risk of fatalities and serious adverse events Background The risk for donors of allogeneic hematopoietic stem cells transplants is generally considered negligible. Scattered reports of severe complications and a recent controversy on hematopoietic malignancies after granulocyte colony-stimulating factor administration have challenged this opinion. Design and Methods Three hundred and thirty-eight allogeneic transplant teams from 35 primarily European countries were asked to report numbers of fatalities, severe adverse events and hematologic malignancies occurring among their hematopoietic stem cell donors. Results Two hundred and sixty-two of the 338 teams (77.5%) responded to a first survey (1993–2002) and 169 of the 262 responder teams (65%) to a second survey (2003–2005). They had performed a total of 51,024 first allogeneic hematopoietic stem cell transplantations, of which 27,770 were bone marrow and 23,254 peripheral blood. They observed five donor fatalities, one after a bone marrow donation and four after peripheral blood donation (incidence 0.98 per 10,000 donations; 95% CI 0.32–2.29), 37 severe adverse events (7.25/10,000; 95% CI 5.11–9.99), of which 12 in bone marrow donors (4.32/10,000; 95% CI 2.24–7.75) and 25 in peripheral blood donors (10.76/10,000; 95% CI 6.97–15.85; p<0.05) and 20 hematologic malignancies (3.92/10,000; 95% CI 2.39–6.05), of which 8 after donating bone marrow and 12 after donating peripheral blood stem cells. The observed incidence rate of hematologic malignancies did not exceed the expected incidence in an age- and sex-adjusted general population. Conclusions Hematopoietic stem cell donation is associated with a small but definite risk of fatalities and serious adverse events. True incidences might be higher, due to potential underreporting by study design. A continuous, standardized donor follow-up is needed to define donor risk groups and to monitor intermediate and long-term sequelae.

The EBMT activity survey 2009: trends over the past 5 years

A record number of 39 209 HSCT in 34 809 patients (14 950 allogeneic (43%) and 19 859 autologous (57%)) were reported by 658 centers in 48 countries to the 2013 survey. Trends include: more growth in allogeneic than in autologous HSCT, increasing use of sibling and unrelated donors and a pronounced increase in haploidentical family donors when compared with cord blood donors for those patients without a matched related or unrelated donor. Main indications were leukemias, 11 190 (32%; 96% allogeneic); lymphoid neoplasias, 19 958 (57%; 11% allogeneic); solid tumors, 1543 (4%; 4% allogeneic); and nonmalignant disorders, 1975 (6%; 91% allogeneic). In patients without a matched sibling or unrelated donor, alternative donors are used. Since 2010 there has been a marked increase of 96% in the number of transplants performed from haploidentical relatives (802 in 2010 to 1571 in 2013), whereas the number of unrelated cord blood transplants has slightly decreased (789 in 2010 to 666 in 2013). The use of donor type varies greatly throughout Europe.

Results of the EBMT activity survey 2005 on haematopoietic stem cell transplantation: focus on increasing use of unrelated donors

Six hundred and twenty-four centers from 43 countries reported a total of 31 322 hematopoietic SCT (HSCT) to this 2009 European Group for Blood and Marrow Transplantation (EBMT) survey with 28 033 first transplants (41% allogeneic, 59% autologous). The main indications were leukemias (31%; 92% allogeneic), lymphomas (58%; 12% allogeneic), solid tumors (5%; 6% allogeneic) and non-malignant disorders (6%; 88% allogeneic). There were more unrelated than HLA-identical sibling donors (51 vs 43%) for allogeneic HSCT; the proportion of peripheral blood as stem cell source was 99% for autologous and 71% for allogeneic HSCT. Allogeneic and autologous HSCT continued to increase by about 1000 HSCT per year since 2004. Patterns of increase were distinct and different. In a trend analysis, allogeneic HSCT increased in all World Bank Categories (P=0.01, two sided; all categories), autologous HSCT increased in middle- (P=0.01, two sided) and low-income (P=0.01, two sided) countries. EBMT practice guidelines appeared to have an impact on trend, with a clear increase in absolute numbers within the categories ‘standard’ and ‘clinical option’ for both allogeneic and autologous HSCT (P=0.01, two sided; for both allogeneic and autologous HSCT) and a clear decrease in autologous HSCT for the ‘developmental’ and ‘generally not recommended’ indications (P=0.01, two sided). These data illustrate the status and trends of HST in Europe.

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

This EBMT activity report documents the haematopoietic stem cell transplantation (HSCT) activity in Europe in 2005. It provides numbers of HSCT by indication, donor type and stem cell source, lists the new practice of planned double transplants with allogeneic after autologous HSCT and concentrates on the increasing role of unrelated transplants over the last years. In 2005, there were 24 168 first HSCT, 8890 allogeneic (37%), 15 278 autologous (63%) and 3773 additional re- or multiple transplants reported from 597 centres in 43 participating countries. Main indications were leukaemias (7404 (31%; 82% allogeneic)); lymphomas (13 825 (57%; 89% autologous)); solid tumours (1655 (7%; 92% autologous)) and non-malignant disorders (1131 (5%; 93% allogeneic)). A total of 671 planned allogeneic after autologous HSCT were reported; the majority for myeloma (52%), lymphoma (28%) and acute myeloid leukaemia (11%). Compared to 2004, there was a 20% increase in allogeneic HSCT; numbers of autologous HSCT remained constant. The most noticeable increase was in unrelated HSCT, which comprise 41% of all allogeneic HSCT. Unrelated HSCT were preferentially performed for leukaemias and in countries with high income according to World Bank criteria. These data illustrate the current experience in Europe and form the basis for patient counselling and decisions making at health care institutions.

EBMT activity survey 2004 and changes in disease indication over the past 15 years

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 km(2), and a gross national income per person of US