Data quality is emerging as an essential characteristics of all data driven processes. The problem is particularly severe when health or vital statistics are concerned, with important consequences on government intervention policies and distribution of financial resources. In this paper, we deal with the underreporting issue with particular attention on its effects on the estimation of the prevalence of a phenomenon. We propose a non parametric compound Poisson model that allows for the estimation of underreporting probabilities. We will apply the proposed model to original data about the incidence of Chronic Kidney Disease (CKD) in Apulia.
Background: Patients with primary membranous nephropathy (MN) and persistent nephrotic syndrome have a high risk of progression to end-stage renal disease. The Ponticelli protocol (steroids with alkylating agents) is the most effective immunosuppressive therapy for this condition, but it has severe adverse effects. Tacrolimus and rituximab have demonstrated efficacy for remission of nephrotic syndrome in MN with a safer profile. However, the published evidence is largely based on small or short-term observational studies, historical cohorts, comparisons with conservative therapy or clinical trials without appropriate control groups, and there is no head-to-head comparison with the Ponticelli protocol. Methods: The STARMEN randomized clinical trial will compare the efficacy of sequential tacrolimus–rituximab therapy with a modified Ponticelli protocol (steroids plus cyclophosphamide). The trial will also evaluate the role of antibodies against the Mtype phospholipaseA2 receptor (anti-PLA2R) and other antibodies as markers of response to treatment and long-term prognosis Results: The trial has already started with 23 patients having been enrolled as of 1 April 2015, an estimated 21.7% of the estimated sample ; This is an investigator-initiated RCT that has been up to now funded by the European Renal Association (Research Programme, ERA-EDTA), the Instituto de Salud Carlos III (Spanish Government) through specific research projects (FIS 13/02502 and ICI14/00350) and the Spanish Renal Research Network (REDINREN, RD 012/0021).
Altres ajuts: This is an investigator-initiated RCT that has been up to now funded by the European Renal Association (Research Programme, ERA-EDTA), the Instituto de Salud Carlos III (Spanish Government) through specific research projects and the Spanish Renal Research Network (REDINREN, RD 012/0021). ; Patients with primary membranous nephropathy (MN) and persistent nephrotic syndrome have a high risk of progression to end-stage renal disease. The Ponticelli protocol (steroids with alkylating agents) is the most effective immunosuppressive therapy for this condition, but it has severe adverse effects. Tacrolimus and rituximab have demonstrated efficacy for remission of nephrotic syndrome in MN with a safer profile. However, the published evidence is largely based on small or short-term observational studies, historical cohorts, comparisons with conservative therapy or clinical trials without appropriate control groups, and there is no head-to-head comparison with the Ponticelli protocol. The STARMEN randomized clinical trial will compare the efficacy of sequential tacrolimus-rituximab therapy with a modified Ponticelli protocol (steroids plus cyclophosphamide). The trial will also evaluate the role of antibodies against the M-type phospholipase A receptor (anti-PLAR) and other antibodies as markers of response to treatment and long-term prognosis. The trial has already started with 23 patients having been enrolled as of 1 April 2015, an estimated 21.7% of the estimated sample.
