title: Discontinuation of eculizumab maintenance treatment for atypical hemolytic uremic syndrome: a report of 10 cases.
abstract: Atypical hemolytic uremic syndrome (aHUS) is a life-threatening thrombotic microangiopathy, and as many as 70% of patients with aHUS have mutations in the genes encoding complement regulatory proteins. Eculizumab, a humanized recombinant monoclonal antibody targeting C5, has been used successfully in patients with aHUS since 2009. The standard maintenance treatment requires life-long eculizumab therapy, but the possibility of discontinuation has not yet been tested systematically. We report the safety of discontinuing eculizumab treatment in 10 patients who stopped treatment with the aim of minimizing the risk of adverse reactions, reducing the risk of meningitis, and improving quality of life while also reducing the considerable treatment costs. Disease activity was monitored closely at home by means of urine dipstick testing for hemoglobin. During the cumulative observation period of 95 months, 3 of the 10 patients experienced relapse within 6 weeks of discontinuation, but then immediately resumed treatment and completely recovered. Our experience supports the possibility of discontinuing eculizumab therapy with strict home monitoring for early signs of relapse in patients with aHUS who achieve stable remission.
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy. Electronic address: ardissino@centroseu.org.
authors.forename: Gianluigi
authors.initials: G
authors.lastname: Ardissino
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Sara
authors.initials: S
authors.lastname: Testa
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Ilaria
authors.initials: I
authors.lastname: Possenti
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Tel
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Fabio
authors.initials: F
authors.lastname: Paglialonga
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Stefania
authors.initials: S
authors.lastname: Salardi
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Silvana
authors.initials: S
authors.lastname: Tedeschi
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Mirco
authors.initials: M
authors.lastname: Belingheri
authors.affiliation: Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Cugno
mesh_terms: D000293:Adolescent; D000328:Adult; D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage / Q000009:adverse effects / Q000191:economics; D065766:Atypical Hemolytic Uremic Syndrome / Q000175:diagnosis / Q000188:drug therapy / Q000191:economics / Q000503:physiopathology / Q000523:psychology; D002648:Child; D002675:Child, Preschool; D051056:Complement Inactivating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000191:economics; D017281:Cost of Illness; D064420:Drug-Related Side Effects and Adverse Reactions / Q000145:classification / Q000209:etiology / Q000517:prevention & control*; D005260:Female; D006456:Hemoglobinuria / Q000175:diagnosis / Q000209:etiology; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D008991:Monitoring, Physiologic / Q000379:methods; D011788:Quality of Life; D012008:Recurrence; D012074:Remission Induction; D016896:Treatment Outcome; D016482:Urinalysis / Q000379:methods; D028761:Withholding Treatment
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D051056:Complement Inactivating Agents; C481642:eculizumab
keywords: Atypical hemolytic uremic syndrome; discontinuation; eculizumab