Multi-centre, randomised, open-label, blinded endpoint assessed, trial of corticosteroids plus intravenous immunoglobulin (IVIG) and aspirin, versus IVIG and aspirin for prevention of coronary artery aneurysms (CAA) in Kawasaki disease (KD): the KD CAA prevention (KD-CAAP) trial protocol.


Journal

Trials
ISSN: 1745-6215
Titre abrégé: Trials
Pays: England
ID NLM: 101263253

Informations de publication

Date de publication:
26 Jan 2023
Historique:
received: 28 10 2022
accepted: 23 12 2022
entrez: 26 1 2023
pubmed: 27 1 2023
medline: 31 1 2023
Statut: epublish

Résumé

Kawasaki disease (KD) is an acute self-limiting inflammatory vasculitis affecting predominantly medium-sized arteries, particularly the coronary arteries. A number of recent studies conducted in different European countries have demonstrated alarmingly high coronary complications despite treatment with intravenous immunoglobulin (IVIG). These high complication rates now emphasize the need for an urgent reappraisal of IVIG as the sole primary therapeutic agent for KD. The Kawasaki disease CAA prevention (KD-CAAP) trial will test the hypothesis that immediate adjunctive corticosteroid treatment to standard of care IVIG and aspirin will reduce coronary artery aneurysm (CAA) rates in unselected KD patients across Europe. KD-CAAP is a multicentre, randomised, controlled, open-label, blinded endpoint assessed trial that will be conducted across Europe supported by the conect4children pan-European clinical trials network. Patients with KD who satisfy the eligibility criteria will be randomised (1:1) to receive either oral prednisolone 2 mg/kg/day plus standard of care therapy IVIG (2 g/kg) and aspirin (40 mg/kg/day); or IVIG and aspirin alone. Further management is dictated by temperature and C-reactive protein (CRP) responses. Co-primary outcomes are as follows: (i) any CAA within the 3 months of trial follow-up; (ii) average estimate of maximum coronary Z-score at weeks 1, 2 and 6 adjusting for rescue treatment. Additional outcomes will be assessed including cost effectiveness, quality of life, corticosteroid toxicity and other safety outcomes. Several recent studies have indicated that coronary complications associated with KD across Europe are much higher than early trials of IVIG had initially suggested. KD-CAAP directly addresses this issue by exploring the therapeutic benefit of adjunctive corticosteroids in unselected KD cases. If we find that corticosteroids prevent CAA and are safe, this is a cheap and widely available intervention that could be implemented immediately for the benefit of children. ISRCTN71987471- March 31, 2020; Eudract 2019-004433-17.

Sections du résumé

BACKGROUND BACKGROUND
Kawasaki disease (KD) is an acute self-limiting inflammatory vasculitis affecting predominantly medium-sized arteries, particularly the coronary arteries. A number of recent studies conducted in different European countries have demonstrated alarmingly high coronary complications despite treatment with intravenous immunoglobulin (IVIG). These high complication rates now emphasize the need for an urgent reappraisal of IVIG as the sole primary therapeutic agent for KD. The Kawasaki disease CAA prevention (KD-CAAP) trial will test the hypothesis that immediate adjunctive corticosteroid treatment to standard of care IVIG and aspirin will reduce coronary artery aneurysm (CAA) rates in unselected KD patients across Europe.
METHODS METHODS
KD-CAAP is a multicentre, randomised, controlled, open-label, blinded endpoint assessed trial that will be conducted across Europe supported by the conect4children pan-European clinical trials network. Patients with KD who satisfy the eligibility criteria will be randomised (1:1) to receive either oral prednisolone 2 mg/kg/day plus standard of care therapy IVIG (2 g/kg) and aspirin (40 mg/kg/day); or IVIG and aspirin alone. Further management is dictated by temperature and C-reactive protein (CRP) responses. Co-primary outcomes are as follows: (i) any CAA within the 3 months of trial follow-up; (ii) average estimate of maximum coronary Z-score at weeks 1, 2 and 6 adjusting for rescue treatment. Additional outcomes will be assessed including cost effectiveness, quality of life, corticosteroid toxicity and other safety outcomes.
DISCUSSION CONCLUSIONS
Several recent studies have indicated that coronary complications associated with KD across Europe are much higher than early trials of IVIG had initially suggested. KD-CAAP directly addresses this issue by exploring the therapeutic benefit of adjunctive corticosteroids in unselected KD cases. If we find that corticosteroids prevent CAA and are safe, this is a cheap and widely available intervention that could be implemented immediately for the benefit of children.
TRIAL REGISTRATION BACKGROUND
ISRCTN71987471- March 31, 2020; Eudract 2019-004433-17.

Identifiants

pubmed: 36703139
doi: 10.1186/s13063-022-07051-9
pii: 10.1186/s13063-022-07051-9
pmc: PMC9879235
doi:

Substances chimiques

Adrenal Cortex Hormones 0
Aspirin R16CO5Y76E
Immunoglobulins, Intravenous 0

Types de publication

Clinical Trial Protocol Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

60

Subventions

Organisme : Medical Research Council
ID : MR/M008665/1
Pays : United Kingdom
Organisme : Innovative Medicines Initiative
ID : 777389

Informations de copyright

© 2023. The Author(s).

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Auteurs

Despina Eleftheriou (D)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK. d.eleftheriou@ucl.ac.uk.

Yolanda Collaco Moraes (YC)

Medical Research Council (MRC) Clinical Trials Unit (CTU) at University College London (UCL), London, UK.

Cara Purvis (C)

Medical Research Council (MRC) Clinical Trials Unit (CTU) at University College London (UCL), London, UK.

Molly Pursell (M)

Medical Research Council (MRC) Clinical Trials Unit (CTU) at University College London (UCL), London, UK.

Marta Merida Morillas (MM)

Medical Research Council (MRC) Clinical Trials Unit (CTU) at University College London (UCL), London, UK.

Robin Kahn (R)

Department of Paediatrics, Lund University, Clinical Sciences, Lund, Sweden.

Maria Mossberg (M)

Department of Paediatrics, Lund University, Clinical Sciences, Lund, Sweden.

Filip Kucera (F)

Great Ormond Street Hospital, London, UK.

Robert Tulloh (R)

Bristol Heart Institute, Bristol, UK.

Joseph F Standing (JF)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.

Veronica Swallow (V)

Sheffield Hallam University, Sheffield, UK.

Rachael McCormack (R)

Societi Foundation CIO, The UK Foundation for Kawasaki Disease, Newark, UK.

Jethro Herberg (J)

Section of Paediatric Infectious Diseases, Imperial College London, London, UK.

Michael Levin (M)

Section of Paediatric Infectious Diseases, Imperial College London, London, UK.

Mandy Wan (M)

Pharmacy Department, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK.
Institute of Pharmaceutical Science, King's College London, London, UK.

Nigel Klein (N)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.

Roisin Connon (R)

Medical Research Council (MRC) Clinical Trials Unit (CTU) at University College London (UCL), London, UK.

Ann Sarah Walker (AS)

Medical Research Council (MRC) Clinical Trials Unit (CTU) at University College London (UCL), London, UK.

Paul Brogan (P)

UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.

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