Evidence map›Paper›PMID 34521444›Full record

ArticlePediatric rheumatology online journal2021

Reasons for canakinumab initiation among patients with periodic fever syndromes: a retrospective medical chart review from the United States.

Peter Hur, Kathleen G Lomax, Raluca Ionescu-Ittu, Ameur M Manceur, Jipan Xie, Jordan Cammarota, Raju Gautam, Navneet Sanghera, Nina Kim, Alexei A Grom

Open access · goldAbstract read
In one paragraph

Article in Pediatric rheumatology online journal, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
0.2field-weighted citation impact, top 47% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 1 synthesis or guideline pooled it, 4 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 5 institutions in 2 countries.

Peter HurNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Kathleen G LomaxNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Raluca Ionescu-IttuAnalysis Group, Inc, Montreal, QC, Canada.
Ameur M ManceurAnalysis Group, Inc, Montreal, QC, Canada.
Jipan XieAnalysis Group, Inc, Los Angeles, CA, USA.
Jordan CammarotaAnalysis Group, Inc, Washington, DC, USA.
Raju GautamNovartis Healthcare Pvt. Ltd, Hyderabad, India.
Navneet SangheraNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Nina KimBaylor Scott and White Medical Center Temple, Texas and University of Texas, Austin, Texas, USA.
Alexei A GromDivision of Pediatric Rheumatology, Children's Hospital Medical Center, MLC 4010, 3333 Burnet Avenue, Cincinnati, OH, 45229, USA. Alexi.Grom@cchmc.org.
Novartis (United States) · USAnalysis Group (United States) · USBaylor Scott & White Medical Center - Temple · USCincinnati Children's Hospital Medical Center · USNovartis (India) · IN

Funding

Novartis Pharmaceuticals Corporation Novartis Pharmaceuticals Corporation
6 · The paper itself

Abstract

backgroundAlthough canakinumab has demonstrated efficacy in multiple trials in patients with periodic fever syndromes (PFS), the evidence on initiation of canakinumab among PFS patients in real world setting is not well understood. We aimed to characterize the reasons for canakinumab initiation among patients with PFS, specifically, cryopyrin-associated periodic syndrome (CAPS), hyperimmunoglobulin D syndrome/mevalonate kinase deficiency (HIDS/MKD), TNF receptor-associated periodic syndrome (TRAPS) and familial Mediterranean fever (FMF).

methodsPhysicians retrospectively reviewed the medical charts of PFS patients prescribed canakinumab between 2016 and 2018. Information collected included patient clinical characteristics, reasons for previous treatment discontinuation and canakinumab initiation. The results were summarized for overall patients, and by children (< 18 years) and adults and by subtype of PFS.

resultsFifty-eight physicians in the US (rheumatologists, 44.8 %; allergists/immunologists, 29.3 %; dermatologists, 25.9 %) abstracted information for 147 patients (children, 46.3 %; males, 57.1 %; CAPS, 36.7 %; TRAPS, 26.5 %; FMF, 26.5 %; HIDS/MKD, 6.8 %; Mixed, 3.4 %). Overall, most patients (90.5 %) received treatment directly preceding canakinumab (NSAIDs, 27.8 % [40.0 % in HIDS/MKD]; anakinra, 24.1 % [32.7 % in CAPS]; colchicine, 21.8 % [35.9 % in FMF]), which were discontinued due to lack of efficacy/effectiveness (39.5 %) and availability of a new treatment (36.1 %). The common reasons for canakinumab initiation were physician perceived efficacy/effectiveness (81.0 %; children, 75.0 %; adults, 86.1 %), lack of response to previous treatment (40.8 %; children, 38.2 %; adults, 43.0 %) and favorable safety profile/tolerability (40.1 %; children, 42.6 %; adults, 38.0 %). Within subtypes, efficacy/effectiveness was the most stated reason for canakinumab initiation in HIDS/MKD (90.9 %), lack of response to previous treatment in FMF (52.4 %) and convenience of administration/dosing in CAPS (27.1 %).

conclusionsThis study provided insights into how canakinumab is initiated in US clinical practice among PFS patients, with physician perceived efficacy/effectiveness of canakinumab, lack of response to previous treatment and favorable safety profile/tolerability of canakinumab being the dominant reasons for canakinumab initiation in all patients and in children and adults and PFS subtypes. Notably, the favorable safety profile/tolerability of canakinumab was more often the reason for initiation among children versus adults.

Indexed as

AdolescentAdultAntibodies, Monoclonal, HumanizedChildChild, PreschoolCryopyrin-Associated Periodic SyndromesDrug PrescriptionsFamilial Mediterranean FeverFemaleFeverHereditary Autoinflammatory DiseasesHumansMaleMevalonate Kinase DeficiencyPractice Patterns, Physicians'Retrospective StudiesAntibodies, Monoclonal, HumanizedcanakinumabCanakinumabCryopyrin-associated periodic fever syndromeFamilial Mediterranean feverHyperimmunoglobulin D syndromeMevalonate kinase deficiencyReal worldRetrospective reviewTNF receptor-associated periodic fever syndrome

Identifiers

PMID34521444
PMCPMC8439059
OpenAlexW3200138468

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.