PMID- 37662020
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230928
IS  - 2327-2236 (Electronic)
IS  - 2326-697X (Print)
IS  - 2326-697X (Linking)
VI  - 1
IP  - 2
DP  - 2013
TI  - A Retrospective Patient Chart Review and Survey in Patients with 
      Cryopyrin-associated Periodic Syndromes Treated with Anakinra.
PG  - 123-133
LID - 10.36469/9860 [doi]
AB  - Background: Cryopyrin-associated periodic syndromes (CAPS) is a group of rare 
      autoinflammatory diseases. Little is known about the burden of disease, patients' 
      views on treatment, and adverse events (AEs) with current therapy. Objectives: 
      The main study objective was to quantify the patients' burden of disease in terms 
      of flares and resource use and to characterize patient symptomatology and 
      tolerability of treatment with anakinra. A secondary objective included comparing 
      chart review and patient recall of symptoms and AEs. Methods: A retrospective 
      medical chart review and concurrent online patient survey was conducted in four 
      European countries. Data 12 months prior to initiation of/during anakinra 
      treatment were entered into web-based case report forms by study groups. Results: 
      Forty-two patients received/were receiving anakinra as primary treatment for at 
      least 12 months. Patients experienced a 79.5% reduction in flares after 
      commencing anakinra treatment. During the past 12 months on anakinra, four of 
      five (80%) patients who recalled experiencing flares reported cancelling social 
      activities and staying home as the most common courses of action. Most common AEs 
      were injection site pain upon treatment initiation and weight gain. According to 
      patient recall, 12 of 21 patients (57.1%) discontinued anakinra to enter another 
      clinical trial; of the 12, eight (38%) specifically discontinued anakinra only 
      for that reason, and four patients cited entering a clinical trial as one of many 
      reasons for discontinuing anakinra. Conclusions: To our knowledge, this is the 
      most comprehensive survey of patient experience with CAPS. Although improved, 
      CAPS treatment remains suboptimal and a significant burden is placed upon 
      patients, caregivers, and the healthcare system. With new agents available, it 
      will be important to compare outcomes in patients using all therapeutic options.
FAU - Lachmann, Helen J
AU  - Lachmann HJ
AD  - UCL Medical School, London, UK.
FAU - Arnold, Renee J G
AU  - Arnold RJG
AD  - Arnold Consultancy & Technology LLC, New York, NY USA; Mount Sinai School of 
      Medicine, New York, NY USA.
FAU - Gattorno, Marco
AU  - Gattorno M
AD  - UO Pediatria II, G. Gaslini Scientific Institute, Italy.
FAU - Kone-Paut, Isabelle
AU  - Kone-Paut I
AD  - Pediatric Rheumatology, CEREMAI, Hopital Kremlin Bicetre, University of Paris 
      SUD, France.
FAU - Ferreira, Alberto
AU  - Ferreira A
AD  - Novartis Pharma AG, Basel, Switzerland.
FAU - Kummerle-Deschner, Jasmin
AU  - Kummerle-Deschner J
AD  - Division of Pediatric Rheumatology, Department of Pediatrics University Hospital 
      Tubingen, Germany.
LA  - eng
PT  - Journal Article
DEP - 20130821
PL  - United States
TA  - J Health Econ Outcomes Res
JT  - Journal of health economics and outcomes research
JID - 101648581
PMC - PMC10471388
OTO - NOTNLM
OT  - anakinra
OT  - caps
OT  - cryopyrin-associated periodic syndromes
OT  - flares
OT  - observational
OT  - perceptions
OT  - retrospective
EDAT- 2013/08/21 00:00
MHDA- 2013/08/21 00:01
PMCR- 2013/08/21
CRDT- 2023/09/04 04:35
PHST- 2013/08/21 00:01 [medline]
PHST- 2013/08/21 00:00 [pubmed]
PHST- 2023/09/04 04:35 [entrez]
PHST- 2013/08/21 00:00 [pmc-release]
AID - 9860 [pii]
AID - 10.36469/9860 [doi]
PST - epublish
SO  - J Health Econ Outcomes Res. 2013 Aug 21;1(2):123-133. doi: 10.36469/9860. 
      eCollection 2013.