title: Real-World Experience and Impact of Canakinumab in Cryopyrin-Associated Periodic Syndrome: Results From a French Observational Study.
abstract: The ENVOL study was designed to assess the psychosocial impact of disease and therapy in a French cohort of cryopyrin-associated periodic syndromes (CAPS) patients (and caregivers) treated with canakinumab.
  
  
  
  The ENVOL study was a multicenter, observational study of CAPS patients given ≥1 canakinumab dose. Data were collected before treatment, at 6 and 12 months afterward, and at the last visit. Patients and caregivers completed questionnaires assessing changes from the 12 months of pretreatment to 12 months prior to interview. Data were analyzed retrospectively.
  
  
  
  The study included 10 physicians and 68 patients (53 adults, 15 children). Sixty-five patients (95.6%) were still receiving canakinumab at the last visit (median 5 years after starting therapy). The mean ± SD score for patient-reported general health increased from 7 ± 2.9 before canakinumab to 2.7 ± 2.7 after treatment (P < 0.001). Physical and emotional symptoms resolved or improved in a substantial proportion of patients, including bodily pain (38 of 46 patients), fever (32 of 39), skin disease (35 of 41), fatigue (31 of 47), self-confidence (29 of 46), and energy (34 of 47). Social activity, relationships, sexuality, and energy measures improved in >40% of respondents. Caregivers spent a median of 3 versus 0.5 hours/week on care in the 12 months of pretreatment versus 12 months prior to interview (P < 0.001). Following treatment, patients required fewer consultations with general practitioners (mean ± SD per patient per year: 5.2 ± 7.4 versus 8.5 ± 7.2 pretreatment), internists/rheumatologists/dermatologists (2.0 ± 2.1 versus 3.7 ± 3.9), and pediatricians (1.8 ± 1.5 versus 4.4 ± 4.2).
  
  
  
  Long-term treatment with canakinumab achieves a highly relevant improvement in the physical, emotional, and social lives of patients with CAPS, accompanied by a marked reduction in support required from caregivers and in health care consultations.
authors.affiliation: CEREMAI, Hôpital Bicêtre, APHP, University of Paris Sud, Saclay, Le Kremlin Bicêtre, France.
authors.forename: I
authors.initials: I
authors.lastname: Kone-Paut
authors.affiliation: Université Paris-Descartes-Sorbonne Paris, Institut IMAGINE, Hôpital Necker-Enfants Malades, Assistance Publique Hôpitaux de Paris, Paris, France.
authors.forename: P
authors.initials: P
authors.lastname: Quartier
authors.affiliation: Hôpital Saint Antoine, Paris, France.
authors.forename: O
authors.initials: O
authors.lastname: Fain
authors.affiliation: Hôpital Tenon, DHU I2B, Paris, France.
authors.forename: G
authors.initials: G
authors.lastname: Grateau
authors.affiliation: Hôpital Pellegrin, Bordeaux, France.
authors.forename: P
authors.initials: P
authors.lastname: Pillet
authors.affiliation: Clinique Hôpital Lapeyronie, Montpellier, France.
authors.forename: P
authors.initials: P
authors.lastname: Le Blay
authors.affiliation: Hôpital Saint André, Bordeaux, France.
authors.forename: F
authors.initials: F
authors.lastname: Bonnet
authors.affiliation: CHU de Rennes, Rennes, France.
authors.forename: V
authors.initials: V
authors.lastname: Despert
authors.affiliation: Hôpital Tenon, DHU I2B, Paris, France.
authors.forename: K
authors.initials: K
authors.lastname: Stankovic-Stojanovic
authors.affiliation: Novartis Pharma SAS, Rueil-Malmaison, France.
authors.forename: L
authors.initials: L
authors.lastname: Willemin
authors.affiliation: Novartis Pharma SAS, Rueil-Malmaison, France.
authors.forename: S
authors.initials: S
authors.lastname: Quéré
authors.affiliation: Novartis Pharma SAS, Rueil-Malmaison, France.
authors.forename: O
authors.initials: O
authors.lastname: Reigneau
authors.affiliation: Hôpital Huriez, CHRU de Lille, FHU IMMINeNT, Université de Lille, France.
authors.forename: E
authors.initials: E
authors.lastname: Hachulla
mesh_terms: D000293:Adolescent; D000328:Adult; D000911:Antibodies, Monoclonal / Q000008:administration & dosage*; D061067:Antibodies, Monoclonal, Humanized; D002648:Child; D002675:Child, Preschool; D056587:Cryopyrin-Associated Periodic Syndromes / Q000175:diagnosis* / Q000188:drug therapy* / Q000453:epidemiology; D004334:Drug Administration Schedule; D005260:Female; D005602:France / Q000453:epidemiology; D006801:Humans; D008297:Male; D012189:Retrospective Studies
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; C541220:canakinumab