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

Jasmin Beate Kuemmerle-Deschner; Raju Gautam; Aneesh Thomas George; Syed Raza; Kathleen Graham Lomax; Peter Hur

doi:10.1136/rmdopen-2020-001227

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

RMD Open. 2020 Jul;6(2):e001227. doi: 10.1136/rmdopen-2020-001227.

Authors

Jasmin Beate Kuemmerle-Deschner¹, Raju Gautam², Aneesh Thomas George², Syed Raza², Kathleen Graham Lomax³, Peter Hur⁴

Affiliations

¹ Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tuebingen, Tübingen, Germany kuemmerle.deschner@uni-tuebingen.de.
² Patient Access Services, Novartis Healthcare Ltd, Hyderabad, India.
³ Medical Affairs, Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA.
⁴ Health Economics and Outcomes Research (HEOR), Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA.

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.

Keywords: Fever Syndromes; Outcomes research; Treatment.

Publication types

Meta-Analysis
Research Support, Non-U.S. Gov't
Systematic Review

MeSH terms

Clinical Decision-Making
Combined Modality Therapy
Cryopyrin-Associated Periodic Syndromes / therapy*
Disease Management
Disease Susceptibility
Drug Substitution
Fever / therapy*
Hereditary Autoinflammatory Diseases / therapy*
Humans
Mevalonate Kinase Deficiency / therapy*
Publication Bias
Treatment Outcome

Supplementary concepts

Periodic fever, familial, autosomal dominant