Economics and Outcome After Hematopoietic Stem Cell Transplantation: A Retrospective Cohort Study

Abstract

Hematopoietic stem cell transplantation (HSCT) is a lifesaving expensive medical procedure. Hence, more transplants are performed in more affluent countries. The impact of economic factors on patient outcome is less defined. We analyzed retrospectively a defined cohort of 102,549 patients treated with an allogeneic (N = 37,542; 37%) or autologous (N = 65,007; 63%) HSCT. They were transplanted by one of 404 HSCT centers in 25 European countries between 1999 and 2006. We searched for associations between center-specific microeconomic or country-specific macroeconomic factors and outcome. Center patient-volume and center program-duration were significantly and systematically associated with improved survival after allogeneic HSCT (HR 0·87; 0·84–0·91 per 10 patients; p < 0·0001; HR 0·90;0·85–0·90 per 10 years; p < 0·001) and autologous HSCT (HR 0·91;0·87–0·96 per 10 patients; p < 0·001; HR 0·93;0·87–0·99 per 10 years; p = 0·02). The product of Health Care Expenditures by Gross National Income/capita was significantly associated in multivariate analysis with all endpoints (R2 = 18%; for relapse free survival) after allogeneic HSCT. Data indicate that country- and center-specific economic factors are associated with distinct, significant, systematic, and clinically relevant effects on survival after HSCT. They impact on center expertise in long-term disease and complication management. It is likely that these findings apply to other forms of complex treatments.

Alloreactivity: the Janus-face of hematopoietic stem cell transplantation

“…There were significant changes in the indications for HSCT over time. 19 'Standard' risk factors and outcome The analysis confirmed previously defined risk factors. 20,21 Outcome differed between the five main disease categories.…”

“…We used both as continuous variables in the model but recoded them into 5 categories to avoid problems with outliers. 19 Calendar year was used as a continuous covariate. Relapse, as provided by the database and non-relapse mortality were acknowledged as competing risks.…”

“…It integrated the key well-described transplant risk factors and adjusted for micro-and macroeconomic risk factors in the analysis. 11,19,20 The large number of patients, the long follow-up time with a minimum of 8 years, and the systematic pattern in all disease categories and disease stages render it likely that the observations of a donor type hierarchy are true findings.…”

“…19 This corresponds to 93% of all patients transplanted during this period by the participating teams with these indications. The patient' survival data were updated as of 1 January 2015.…”

“…In this context, and in view of the need for better data in the present precision medicine initiative, 18 we analyzed a predefined large cohort of patients with a confirmed follow-up of 8 years. 19 We hypothesized a donor type hierarchy based on phenotypic and genotypic differences between donor and recipient in major (defined by HLA) and minor (defined by HY) histocompatibility antigens and analyzed non-relapse mortality, relapse-free and overall survival in specified donor type groups.…”