Individual Quality Assessment of Autografting by Probability Estimation for Clinical Endpoints: A Prospective Validation Study from the European Group for Blood and Marrow Transplantation
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文摘
The aim of supportive autografting is to reduce the side effects from stem cell transplantation and avoid procedure-related health disadvantages for patients at the lowest possible cost and resource expenditure. Economic evaluation of health care is becoming increasingly important. We report clinical and laboratory data collected from 397聽consecutive adult patients (173 non-Hodgkin lymphoma, 30 Hodgkin lymphoma, 160 multiple myeloma, 7聽autoimmune diseases, and 28 acute leukemia) who underwent their first autologous peripheral blood stem cell transplantation (PBSCT). We considered primary endpoints evaluating health economic efficacy (eg, antibiotic administration, transfusion of blood components, and time in hospital), secondary endpoints evaluating toxicity (in accordance with Common Toxicity Criteria), and tertiary endpoints evaluating safety (ie, the risk of regimen-related death or disease progression within the first year after PBSCT). A time-dependent grading of efficacy is proposed with day 21 for multiple myeloma and day 25 for the other disease categories (depending on the length of the conditioning regimen) as the acceptable maximum time in hospital, which together with antibiotics, antifungal, or transfusion therapy delineates four groups: favorable (鈮?聽days on antibiotics and no transfusions; 鈮?1 [25] days in hospital), intermediate (from 7 to 10聽days on antibiotics and <3 transfusions, 鈮?1 to 25聽days in hospital or 鈮?聽days on antibiotics and no transfusions; from 21 to 30聽days [25 to 34] in hospital), unfavorable (>7聽days on antibiotics, >3 but <6 transfusions; >30/34聽days in hospital after transplantation), and very unfavorable (>10聽days on antibiotics, >6 transfusions; >30 to 34聽days in hospital). The multivariate analysis showed that (1) PBSC harvests of 鈮?聽脳聽106/kg CD34聽+聽cells in 1 apheresis procedure were associated with a favorable outcome in all patient categories except acute myelogenous leukemia and acute lymphoblastic leukemia (P聽=聽.001), (2) 鈮?聽脳聽106/kg CD34聽+聽cells infused predicted better transplantation outcome in all patient categories (P聽<聽.0001) except acute myelogenous leukemia and acute lymphoblastic leukemia, (3) 1 or 2 aphereses (P聽=聽.001) predicted good outcome, (4) toxicity increased with higher graft volume reinfused (>500聽mL) (P聽=聽.002), and (5)聽patients with a central venous catheter during both collection and infusion of PBSC had a more favorable outcome post-PBSCT than peripheral access (P聽=聽.007). The type of mobilization regimen did not affect the outcome of auto-PBSCT. The present study identified predictive variables, which may be useful in future individual pretransplantation probability evaluations with the goal to improve supportive care.
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