title: The efficacy and safety of rituximab in treating childhood nephrotic syndrome: an Italian perspective.
abstract: BACKGROUND
  Nephrotic syndrome is a disorder characterized by proteinuria, hypoalbuminemia and dyslipidemia. Low-dose alternate-day steroid regimen is the standard of care. In case of relapse or significant adverse events, steroid-sparing agents may be used. This analysis was aimed at assessing the efficacy and safety of rituximab for the treatment of children with nephrotic syndrome.
  
  
  RESULTS
  Four studies were included in the final meta-analysis. The end-point of our analysis was the percentage of patients in remission at 6 months. Pooled data from the four studies favours the use of rituximab (RR 5.25, 95 % CI: 3.05-9.06; p < 0.0001). As regards the safety data, rituximab has a limited number of adverse effects, the most common of which occur during the infusions.
  
  
  CONCLUSIONS
  In Italy, the off-label use of drugs is regulated by Law 648/96. In our opinion, there are three scientific requirements to merit a conditional national reimbursement for rituximab in nephrotic syndrome: 1. favourable clinical efficacy and safety data; 2. no available alternatives; 3. outcome data collecting by AIFA through prescribers. In conclusion, our results report a significant incremental benefit of adding rituximab to corticosteroid and/or calcineurin inhibitors for the treatment of nephrotic syndrome.
authors.affiliation: Hospital Pharmacy, Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Milano, Italy. dario.maratea@gmail.com.
authors.forename: Dario
authors.initials: D
authors.lastname: Maratea
authors.affiliation: School of Specialization in Hospital Pharmacy, School of Pharmacy, University of Milano, Milano, Italy.
authors.forename: Monica
authors.initials: M
authors.lastname: Bettio
authors.affiliation: Hospital Pharmacy, Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Milano, Italy.
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Corti
authors.affiliation: Pediatric Nephrology and Dialysis Unit, Department of Clinical Sciences and Community Health, University of Milan, Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Milano, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Montini
authors.affiliation: Hospital Pharmacy, Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Milano, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Venturini
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007558:Italy; D008297:Male; D009404:Nephrotic Syndrome / Q000175:diagnosis / Q000188:drug therapy*; D061214:Patient Safety; D011379:Prognosis; D018570:Risk Assessment; D000069283:Rituximab / Q000008:administration & dosage*; D016896:Treatment Outcome
chemical_list: D000305:Adrenal Cortex Hormones; D000069283:Rituximab
keywords: 648/96 law; Nephrotic syndrome; Off label; Reimbursement; Rituximab