Now showing 1 - 10 of 37
  • Publication
    One million haemopoietic stem-cell transplants: a retrospective observational study
    (Elsevier, 2015-03)
    Gratwohl, Alois
    ;
    Pasquini, Marcelo
    ;
    Aljurf, Mahmoud
    ;
    Atsuta, Yoshiko
    ;
    Baldomero, Helen
    ;
    Foeken, Lydia
    ;
    Gratwοhl, Μichael
    ;
    Bouzas, Luis Fernando
    ;
    Confer, Dennis
    ;
    ;
    Gluckman, Eliane
    ;
    Greinix, Hildegard
    ;
    Horowitz, Mary
    ;
    Iida, Minako
    ;
    Lipton, Jeff
    ;
    Madrigal, Alejandro
    ;
    Mohty, Mohamad
    ;
    Noel, Luc
    ;
    Novitzky, Nicolas
    ;
    Nunez, José
    ;
    Oudshoorn, Machteld
    ;
    Passweg, Jakob
    ;
    van Rood, Jon
    ;
    Szer, Jeff
    ;
    Blume, Karl
    ;
    Appelbaum, Frederic
    ;
    Kodera, Yoshihisa
    ;
    Niederwieser, Dietger
    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.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 300
  • Publication
    Quantitative and Qualitative Differences in Use and Trends of Hematopoietic Stem Cell Transplantation : A Global Observational Study
    (Ferrata Storti Foundation, 2013-08)
    Gratwohl, Alois
    ;
    Baldomero, Helen
    ;
    Gratwοhl, Μichael
    ;
    Aljurf, Mahmoud
    ;
    Bouzas, Luis
    ;
    Horowitz, Mary
    ;
    Kodera, Yoshihisa
    ;
    Lipton, Jeff
    ;
    Iida, Minako
    ;
    Pasquini, Marcelo
    ;
    Passweg, Jakob
    ;
    Szer, Jeff
    ;
    Madrigal, Alejandro
    ;
    ;
    Niederwieser, Dietger
    After 55 years since its first publication stem cell transplantation is considered the optimal treatment option for specific hematological and non-hematological diseases. There is considerable interest in understanding differences of its use and trends at a global level. This analysis aims at analyzing differences in indications, world regions and macroeconomic factors over a three year period. Data from 2006-2008 were obtained from WBMT member registries and from transplant centers in countries without registries, pooled and duplicate reporting removed. Population and macroeconomic data were collected from the World Bank and from the International Monetary Fund. Transplant rates were analyzed by indication, donor type, country, and World Health Organization regional offices areas and related to selected health care indicators using single and multiple linear regression analyses. A total of 146,808 patients after stem cell transplantation were reported by 1,411 teams from 72 countries over 5 continents. Annual number of transplants (+11%) steadily increased, but preferentially in high (p=0.02) and not in low or medium income countries. Highest increase was observed in the Asia Pacific region and, among alllogeneic transplants, in myelodysplasia, chronic lymphocytic and acute leukemias, nonmalignant diseases (>+21%). Among autologous transplantation autoimmune and lymphoproliferative diseases increased. A clear negative trend was seen in allogeneic for chronic myelogenous leukemia and in autologous transplants for leukemias and solid tumors. Transplant rates (p< 0.01), donor type (p< 0.01) and disease indications (p < 0.01) differed significantly between countries and regions and were associated with Gross National Income/capita (p < 0.01) but showed a wide variation of explanatory content by donor type and disease indication. An increase of stem cell transplant activity is observed worldwide but with significant regional differences. The preferential increase in high income country indicates a widening gap between low and high income countries.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 93
  • Publication
    The EBMT Activity Survey 2009: Trends over the Past 5 Years
    (Nature Publishing Group, 2011-02-28)
    Baldomero, Helen
    ;
    Gratwohl, Alois
    ;
    Gratwοhl, Μichael
    ;
    Tichelli, André
    ;
    Niederwieser, Dietger
    ;
    Madrigal, Alejandro
    ;
    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.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 131
  • Publication
    Hematopoietic Stem Cell Transplantation: A Global Perspective
    (American Medical Association, 2010-04-28)
    Gratwohl, Alois
    ;
    Baldomero, Helen
    ;
    Aljurf, Mahmoud
    ;
    Pasquini, Marcelo
    ;
    Bouzas, Luis
    ;
    Yoshimi, Ayami
    ;
    Szer, Jeff
    ;
    Lipton, Jeff
    ;
    Schwendener, Alvin
    ;
    Gratwοhl, Μichael
    ;
    ;
    Niederwieser, Dietger
    ;
    Horowitz, Mary
    ;
    Kodera, Yoshihisa
    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 km2, or having less than US $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 (r2 = 77.33), HSCT team density (indicates the number of transplant teams per 1 million inhabitants; r2 = 76.28), human development index (r2 = 74.36), and gross national income per capita (r2 = 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.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 522
  • Publication
    Changes in the use of hematopoietic stem cell transplantation: a model for diffusion of medical technology
    (Ferrata Storti Foundation, 2010-04-01)
    Gratwohl, Alois
    ;
    Schwendener, Alvin
    ;
    Baldomero, Helen
    ;
    Gratwοhl, Μichael
    ;
    Apperley, Jane
    ;
    Niederwieser, Dietger
    ;
    Background Innovations in hematology spread rapidly. Factors affecting the speed of introduction, international diffusion, and durability of use of innovations are, however, poorly understood. Design and Methods We used data on 251,106 hematopoietic stem cell transplants from 591 teams in 36 European countries to analyze the increase and decrease in such transplants for breast cancer and chronic myeloid leukemia and the replacement of bone marrow by peripheral blood as the source of stem cells as processes of diffusion. Regression analyses were used to measure the quantitative impact of defined macro- and microeconomic factors, to look for significant associations (t-test), and to describe the coefficient of determination or explanatory content (R2). Results Gross national income per capita, World Bank category, team density, team distribution, team size, team experience and, team innovator status were all significantly associated with some or all of the changes. The analyses revealed different patterns of associations and a wide range of explanatory content. Macro- and micro-economic factors were sufficient to explain the increase of allogeneic hematopoietic stem cell transplants in general (R2 = 78.41%) and for chronic myeloid leukemia in particular (R2 = 79.39%). They were insufficient to explain the changes in stem cell source (R2 =26.79% autologous hematopoietic stem cell transplants; R2 = 9.67% allogeneic hematopoietic stem cell transplants) or the decreases in hematopoietic stem cell transplants (R2 =10.22% breast cancer; R2=33.17% chronic myeloid leukemia). Conclusions The diffusion of hematopoietic stem cell transplants is more complex than previously thought. Availability of resources, evidence, external regulations and, expectations were identified as key determinants. These data might serve as a model for diffusion of medical technology in general.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 35
  • Publication
    Predictability of hematopoietic stem cell transplantation rates
    (Ferrata Storti Foundation, 2007-12-01)
    Gratwohl, Alois
    ;
    Baldomero, Helen
    ;
    Schwendener, Alvin
    ;
    Gratwohl, Michael
    ;
    Apperley, Jane
    ;
    Niederwieser, Dietger
    ;
    Background and Objectives Hematopoietic stem cell transplantation (HSCT) is a complex and expensive procedure. Trends in the use of this procedure have appeared erratic in the past. Information on future needs is essential for health care administrators. Design and Methods We analyzed the evolution of transplant rates, e.g. numbers of transplants per 10 million inhabitants, in Europe from 1990 to 2004 for all major disease categories and used Gross National Income (GNI) per capita, team density (numbers of teams per 10 million inhabitants) and team distribution (numbers of teams per 10,000 km2) to measure the impact of economic factors in participating countries. Trends were compared by regression analyses, and countries were grouped by World Bank definitions into high, middle and low income categories. Results Transplant rates increased over time with nearly linear trends, in clear association with GNI per capita (R2=0.72), and distinct by World Bank category within a narrow window of variation for both autologous HSCT (R2=0.95, 0.98 and 0.94 for high, middle and low income categories, respectively) and allogeneic HSCT (R2=0.99, 0.96 and 0.95 for high, middle and low income categories, respectively) when breast cancer (autologous) and chronic myeloid leukemia (allogeneic) were excluded. Team density (R2=0.72) and team distribution (R2=0.51) were also associated with transplant rates. Interpretation and Conclusions Transplant rates for HSCT in Europe are highly predictable. They are primarily influenced by GNI per capita. The absence of saturation and a nearly linear trend indicate that infrastructure lags behind medical needs. Isolated changes in single disease entities can easily be recognized. Hematopoietic stem cell transplantation (HSCT) is considered the treatment of choice for many patients with severe malignant or non-malignant, acquired or congenital disorders of the hematopoietic system or with chemosensitive, radiosensitive or immunosensitive tumors. HSCT has evolved over the last decades from an experimental procedure to the standard of care and is integrated into the treatment algorithm for many disease categories from diagnosis.1-4 Better supportive care, increased donor pools and novel conditioning regimens have extended its use to new categories of patients and disease indications. However, HSCT is a high-cost procedure and can present a financial challenge for patients and health care systems in any country.5-7 A correlation between the economic strength of individual countries and transplant rates, i.e. the number of transplants per number of inhabitants, was reported earlier by the European Group for Blood and Marrow Transplantation (EBMT).8, 9 This correlation explained some of the differences in numbers of transplants between Eastern and Western European countries. Transplant rates were higher in countries with higher Gross National Income (GNI) or higher health care expenditures (HCE) per capita. It is easy to understand that health care providers would like to have information on future needs. HSCT is a complex procedure, and is dependent on the availability of a specific infrastructure, trained medical personnel and support staff. Providing the infrastructure and its mandatory quality management requirements takes time and, therefore, at least short-term predictions are warranted.10 This issue became first apparent with the sudden increase of autologous HSCT for breast cancer in the 1990s.11, 12 There was a massive increase of such transplants within a few years from nearly none to more than 5000 in Europe alone in 1996, followed by a similarly rapid decline. A similar phenomenon was observed a few years later with an increase and then rapid decrease in allogeneic HSCT for chronic myeloid leukemia in the late 1990s and the first few years of the new century. In 1999, chronic myeloid leukemia was the most frequent indication for an allogeneic HSCT worldwide. When imatinib mesylate, a specific inhibitor of the BCR/ABL tyrosine kinase, was introduced, transplant rates for chronic myeloid leukemia dropped and are still continuing to do so.13-15 These two observations created the feeling amongst hospitals and health care institutions that transplant rates were erratic, rapidly changing and unpredictable. A fear of having too much infrastructure became prevalent. Making use of its annual activity surveys, the EBMT made an attempt to gain insight into the mechanisms of the evolution in HSCT. Based on the data from 1990 to 2000, short-term predictions were attempted and extrapolations made in 2000 for the transplant rates in 2003.16 These predictions were tested for their validity with the 2003 final data and extended throughout the observation period of the activity survey. The results of this analysis confirm the accuracy of the predictions made in 2000. They show that the changing usage of HSCT for breast cancer and chronic myeloid leukemia are the exception. As a rule, and adjusted for economic factors, transplant rates are highly predictable, increasing over time with nearly linear trends and in association with national income. There is no indication of saturation and the need for more infrastructure remains as urgent as before.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 31
  • Publication
    Predictability of hematopoietic stem cell transplantation rates
    (Il Pensiero Scientifico, 2007-12-01)
    Gratwohl, Alois
    ;
    Baldomero, Helen
    ;
    Schwendener, Alvin
    ;
    Gratwοhl, Μichael
    ;
    Apperley, Jane
    ;
    Niederwieser, Dietger
    ;
    Background and Objectives Hematopoietic stem cell transplantation (HSCT) is a complex and expensive procedure. Trends in the use of this procedure have appeared erratic in the past. Information on future needs is essential for health care administrators. Design and Methods We analyzed the evolution of transplant rates, e.g. numbers of transplants per 10 million inhabitants, in Europe from 1990 to 2004 for all major disease categories and used Gross National Income (GNI) per capita, team density (numbers of teams per 10 million inhabitants) and team distribution (numbers of teams per 10,000 km2) to measure the impact of economic factors in participating countries. Trends were compared by regression analyses, and countries were grouped by World Bank definitions into high, middle and low income categories. Results Transplant rates increased over time with nearly linear trends, in clear association with GNI per capita (R2=0.72), and distinct by World Bank category within a narrow window of variation for both autologous HSCT (R2=0.95, 0.98 and 0.94 for high, middle and low income categories, respectively) and allogeneic HSCT (R2=0.99, 0.96 and 0.95 for high, middle and low income categories, respectively) when breast cancer (autologous) and chronic myeloid leukemia (allogeneic) were excluded. Team density (R2=0.72) and team distribution (R2=0.51) were also associated with transplant rates. Interpretation and Conclusions Transplant rates for HSCT in Europe are highly predictable. They are primarily influenced by GNI per capita. The absence of saturation and a nearly linear trend indicate that infrastructure lags behind medical needs. Isolated changes in single disease entities can easily be recognized.
    Type:
    Journal:
    Volume:
    Issue:
    Scopus© Citations 31
  • Publication
    Hematopoietic stem cell transplants for chronic myeloid leukemia in Europe - Impact of cost considerations
    (Nature Publ. Group, 2007-03-01)
    Gratwohl, Alois
    ;
    Baldomero, Helen
    ;
    Schwendener, Alvin
    ;
    Gratwοhl, Μichael
    ;
    Urbano-Ispizua, Alvaro
    ;
    The tradeoff between ongoing expensive drug therapies in general or once in a lifetime procedure becomes relevant. Transplant rates for chronic myeloid leukemia across Europe provide an example. Economics matter as well as evidence.
    Scopus© Citations 30