Abstract
Background and Aims
Thrombotic microangiopathy (TMA) is a life-threatening and rare disease associated with a higher risk of dead and chronic renal replacement therapy (CRRT). Eculizumab is highly effective but also expensive. We evaluate the direct economic burden of the TMA, the cost of CRRT, Eculizumab, and the impact of a multidisciplinary team (MDT) after two years of its implementation.
Method
It is a retrospective study, conducted in a 3rd level hospital. We evaluate the risk of i) dead and ii) CRRT need. The number of patients no treated under futility consideration. The cost (euros) for hospitalization at the floor and intensive care unit admission, CRRT and Eculizumab at the pre-MDT implementation (from January 2008 to May 2016) in comparison with the post-MDT period (from May 2018 to Dec 2018). Clinical outcomes: i) risk of death and ii) risk of CRRT need. To determine the cost per patient-year, we calculated the total number of days of hospitalization, the entire months on dialysis or in kidney transplant program (KTx) and the milligrams of Eculizumab used at any period. The total amount divided by the whole years of observation and finally and by the mean number of patients per year diagnosed at any period. The number of patients-year we determined considering the incidence density (ID: cases/1,000,000 person-year). Patients with ADAMTS-13 deficiency were excluded.
Results
Forty-two patients were included. ID increased from 2.3 cases/1,000,000 person-years (n=20) to 11.7 cases/1,000,000 person-year (n=22). Comparing with the pre-MDT period, the number of patients who died increased from 3(15%) to 7(32%), P=0.20; while the risk for CRRT decreased from 9(45%) to 0, P<0.01 [relative risk (95%CI) for no CRRT requirements: 0.55 (0.37 to 0.81)]. One (5%) and three (14%) patients died under futility consideration at the pre- and post-MDT period, respectively (P=0.60). From all the patients who died, only one was in acute dialysis program while 7 showed neurologic damage. The mean cost per patient-year changed from 319,931 to 150,878 euros from the pre- to post-MDT period.
Conclusion
The implementation of an MDT shows a change in the natural history of the disease, where neurological damage emerges as a risk factor associated with mortality instead of CRRT needs.
TMA patients represent a remarkable economic burden, representing an essential challenge for the health system sustainability that could be improved by an MDT.