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Original research
Systematic literature review of efficacy/effectiveness and safety of current therapies for the treatment of cryopyrin-associated periodic syndrome, hyperimmunoglobulin D syndrome and tumour necrosis factor receptor-associated periodic syndrome
  1. Jasmin Beate Kuemmerle-Deschner1,
  2. Raju Gautam2,
  3. Aneesh Thomas George2,
  4. Syed Raza2,
  5. Kathleen Graham Lomax3 and
  6. Peter Hur4
  1. 1 Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tuebingen, Tübingen, Germany
  2. 2 Patient Access Services, Novartis Healthcare Ltd, Hyderabad, India
  3. 3 Medical Affairs, Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA
  4. 4 Health Economics and Outcomes Research (HEOR), Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA
  1. Correspondence to Jasmin B Kuemmerle-Deschner;kuemmerle.deschner{at}uni-tuebingen.de

Abstract

Objectives Several therapies are used for the treatment of rareautoinflammatory conditions like cryopyrin-associated periodic fever syndromes (CAPS), hyperimmunoglobulin Dsyndrome (HIDS)/mevalonate kinase deficiency (MKD) and tumour necrosis factor receptor-associated periodic syndrome (TRAPS). However, reviews reporting on treatment outcomes of these therapies are lacking.

Methods A systematic literature review was conducted using Embase, MEDLINE, MEDLINE-In Process and Cochrane databases to identify the randomised/non-randomised controlled trials (RCTs/non-RCTs) and real-world observational studies of CAPS, HIDS/MKD and TRAPS published as full-texts (January 2000–September 2017) or conference abstracts (January 2014–September 2017). Studies with data for ≥1 biologic were included. Studies with <5 patients were excluded.

Results Of the 3 342 retrieved publications, 72 studies were included (CAPS, n=43; HIDS/MKD, n=9; TRAPS, n=7; studies with ≥2 cohorts, n=13). Most studies were full-text (n=56), published after 2010 (n=56) and real-world observational studies (n=58). Among included studies, four were RCTs (canakinumab, n=2 (CAPS, n=1; HIDS/MKD and TRAPS, n=1); rilonacept, n=1 (in CAPS); simvastatin, n=1 (in HIDS/MKD)). Canakinumab and anakinra were the most commonly used therapies for CAPS and HIDS/MKD, whereas etanercept, canakinumab and anakinra were the most common for TRAPS. The available evidence suggested the efficacy or effectiveness of canakinumab and anakinra in CAPS, HIDS/MKD and TRAPS, and of etanercept in TRAPS; asingle RCT demonstrated the efficacy of rilonacept in CAPS.

Conclusions Canakinumab, anakinra, etanercept and rilonacept were reported to be well tolerated; however, injection-site reactions were observed frequently with anakinra, rilonacept and etanercept. Data on the use of tocilizumab, infliximab and adalimumab in these conditions were limited; thus, further research is warranted.

  • Fever Syndromes
  • Outcomes research
  • Treatment
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Footnotes

  • Contributors Conception and design: JBK-D, PH, KGL, RG, SR, ATG. Generation of data: RG, SR, ATG. Analysis and interpretation of data: PH, RG, SR, ATG. Drafting of the manuscript or revising it critically for important intellectual content: JBK-D, PH, KGL, RG, SR, ATG. Final approval of the submitted manuscript: JBK-D, PH, KGL, RG, SR, ATG. Some part of the work reported in this manuscript has been previously presented at the 2018 Annual Congress of the American College of Rheumatology and published as a conference abstract (Abstract number 1412).

  • Funding This work was supported by Novartis Pharmaceuticals Corporation, USA.

  • Competing interests JBK-D is an employee of University of Tuebingen, Germany, and received consultants/speakers fees from Novartis and SOBI pharmaceuticals and grant support from SOBI and Novartis. RG and ATG are employees of Novartis healthcare Pvt. Ltd., India. PH is an employee of Novartis Pharmaceuticals Corporation, USA. SR was an employee of Novartis at the time of conduct of this study and currently is an independent freelance consultant. KGL was an employee of Novartis at the time of conduct of this study and currently is an employee at Glenmark Pharmaceuticals, USA.

  • Patient consent for publication Not required.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data sharing statement All data relevant to the study are included in the article or uploaded as supplementary information.

  • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.

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