Anakinra for palmoplantar pustulosis: results from a randomized, double-blind, multicentre, two-staged, adaptive placebo-controlled trial (APRICOT).


Journal

The British journal of dermatology
ISSN: 1365-2133
Titre abrégé: Br J Dermatol
Pays: England
ID NLM: 0004041

Informations de publication

Date de publication:
19 Aug 2021
Historique:
accepted: 14 07 2021
pubmed: 20 8 2021
medline: 20 8 2021
entrez: 19 8 2021
Statut: aheadofprint

Résumé

Palmoplantar pustulosis (PPP) is a rare, debilitating, chronic inflammatory skin disease that affects the hands and feet. Clinical, immunological and genetic findings suggest a pathogenic role for interleukin (IL)-1. To determine whether anakinra (an IL-1 receptor antagonist) delivers therapeutic benefit in PPP. This was a randomized (1 : 1), double-blind, two-staged, adaptive, UK multicentre, placebo-controlled trial [ISCRTN13127147 (registered 1 August 2016); EudraCT number: 2015-003600-23 (registered 1 April 2016)]. Participants had a diagnosis of PPP (> 6 months) requiring systemic therapy. Treatment was 8 weeks of anakinra or placebo via daily, self-administered subcutaneous injections. Primary outcome was the Palmoplantar Pustulosis Psoriasis Area and Severity Index (PPPASI) at 8 weeks. A total of 374 patients were screened; 64 were enrolled (31 in the anakinra arm and 33 in the placebo arm) with a mean (SD) baseline PPPASI of 17·8 (10·5) and a PPP investigator's global assessment of severe (50%) or moderate (50%). The baseline adjusted mean difference in PPPASI favoured anakinra but did not demonstrate superiority in the intention-to-treat analysis [-1·65, 95% confidence interval (CI) -4·77 to 1·47; P = 0·30]. Similarly, secondary objective measures, including fresh pustule count (2·94, 95% CI -26·44 to 32·33; favouring anakinra), total pustule count (-30·08, 95% CI -83·20 to 23·05; favouring placebo) and patient-reported outcomes, did not show superiority of anakinra. When modelling the impact of adherence, the PPPASI complier average causal effect for an individual who received ≥ 90% of the total treatment (48% in the anakinra group) was -3·80 (95% CI -10·76 to 3·16; P = 0·285). No serious adverse events occurred. No evidence for the superiority of anakinra was found. IL-1 blockade is not a useful intervention for the treatment of PPP.

Sections du résumé

BACKGROUND BACKGROUND
Palmoplantar pustulosis (PPP) is a rare, debilitating, chronic inflammatory skin disease that affects the hands and feet. Clinical, immunological and genetic findings suggest a pathogenic role for interleukin (IL)-1.
OBJECTIVES OBJECTIVE
To determine whether anakinra (an IL-1 receptor antagonist) delivers therapeutic benefit in PPP.
METHODS METHODS
This was a randomized (1 : 1), double-blind, two-staged, adaptive, UK multicentre, placebo-controlled trial [ISCRTN13127147 (registered 1 August 2016); EudraCT number: 2015-003600-23 (registered 1 April 2016)]. Participants had a diagnosis of PPP (> 6 months) requiring systemic therapy. Treatment was 8 weeks of anakinra or placebo via daily, self-administered subcutaneous injections. Primary outcome was the Palmoplantar Pustulosis Psoriasis Area and Severity Index (PPPASI) at 8 weeks.
RESULTS RESULTS
A total of 374 patients were screened; 64 were enrolled (31 in the anakinra arm and 33 in the placebo arm) with a mean (SD) baseline PPPASI of 17·8 (10·5) and a PPP investigator's global assessment of severe (50%) or moderate (50%). The baseline adjusted mean difference in PPPASI favoured anakinra but did not demonstrate superiority in the intention-to-treat analysis [-1·65, 95% confidence interval (CI) -4·77 to 1·47; P = 0·30]. Similarly, secondary objective measures, including fresh pustule count (2·94, 95% CI -26·44 to 32·33; favouring anakinra), total pustule count (-30·08, 95% CI -83·20 to 23·05; favouring placebo) and patient-reported outcomes, did not show superiority of anakinra. When modelling the impact of adherence, the PPPASI complier average causal effect for an individual who received ≥ 90% of the total treatment (48% in the anakinra group) was -3·80 (95% CI -10·76 to 3·16; P = 0·285). No serious adverse events occurred.
CONCLUSIONS CONCLUSIONS
No evidence for the superiority of anakinra was found. IL-1 blockade is not a useful intervention for the treatment of PPP.

Identifiants

pubmed: 34411292
doi: 10.1111/bjd.20653
pmc: PMC9255857
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Efficacy and Mechanism Evaluation Programme
ID : NIHR EME 13/50/17 APRICOT
Organisme : The Psoriasis Association
ID : RG2/10
Organisme : NIHR BioResource Clinical Research Facility and comprehensive Biomedical Research Centre awards to Guy's and St Thomas' NHS Foundation Trust in partnership with King's College London and King's College Hospital NHS Foundation Trust
ID : guysbrc-2012-1
Organisme : Swedish Orphan Biovitrum

Commentaires et corrections

Type : CommentIn
Type : CommentIn
Type : CommentIn

Informations de copyright

© 2021 British Association of Dermatologists.

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Auteurs

S Cro (S)

Imperial Clinical Trials Unit, Imperial College London, London, W12 7RH, UK.

V R Cornelius (VR)

Imperial Clinical Trials Unit, Imperial College London, London, W12 7RH, UK.

A E Pink (AE)

St John's Institute of Dermatology, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, UK.

R Wilson (R)

St John's Institute of Dermatology, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, UK.

A Pushpa-Rajah (A)

St John's Institute of Dermatology, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, UK.

P Patel (P)

St John's Institute of Dermatology, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, UK.

A Abdul-Wahab (A)

St George's University Hospitals NHS Foundation Trust, London, SW17 0QT, UK.

S August (S)

Poole Hospital NHS Foundation Trust University Hospitals Dorset, Poole, BH15 2JB, UK.

J Azad (J)

South Tees Hospitals NHS Foundation Trust, Middlesbrough, TS4 3BW, UK.

G Becher (G)

West Glasgow Ambulatory Care Hospital, Glasgow, G3 8SJ, UK.

A Chapman (A)

Homerton University Hospital, London, E9 6SR, UK.

G Dunnil (G)

Bristol Royal Infirmary, Bristol, BS2 8HW, UK.

A D Ferguson (AD)

University Hospitals of Derby and Burton NHS Foundation Trust, Derby, DE22 3NE, UK.

A Fogo (A)

Kingston Hospital, Kingston upon Thames, KT2 7QB, UK.

S A Ghaffar (SA)

Ninewells Hospital and Medical School, Dundee, DD1 9SY, UK.

J R Ingram (JR)

Division of Infection and Immunity, School of Medicine, Cardiff University, University Hospital of Wales, Cardiff, CF14 4XN, UK.

S Kavakleiva (S)

Royal Lancaster Infirmary, Lancaster, LA1 4RP, UK.

E Ladoyanni (E)

Russells Hall Hospital, Dudley, DY1 2HQ, UK.

J A Leman (JA)

Kings Park Hospital, Stirling, FK7 9JH, UK.

A E Macbeth (AE)

Norfolk and Norwich University Hospitals NHS Foundation Trust, Norwich, NR4 7UY, UK.

A Makrygeoegou (A)

West Glasgow Ambulatory Care Hospital, Glasgow, G3 8SJ, UK.

R Parslew (R)

Liverpool University Hospitals NHS Foundation Trust, Liverpool, L9 7AL, UK.

A J Ryan (AJ)

King's College Hospital, London, SE5 9RS, UK.

A Sharma (A)

Nottingham University Hospitals NHS Trust, Nottingham, NG7 2UH, UK.

A R Shipman (AR)

Portsmouth Hospitals Universities NHS Trust, St Mary's Community Health Campus, Portsmouth, PO3 6AD, UK.

C Sinclair (C)

Broomfield Hospital, Chelmsford, CM1 7ET, UK.

R Wachsmuth (R)

Royal Devon and Exeter NHS Foundation Trust, Exeter, EX2 5DW, UK.

R T Woolf (RT)

St John's Institute of Dermatology, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, UK.

A Wright (A)

Bradford Teaching Hospitals NHS Foundation Trust, Bradford, BD9 6RJ, UK.

H McAteer (H)

The Psoriasis Association, Northampton, NN4 7BF, UK.

J N W N Barker (JNWN)

St John's Institute of Dermatology, School of Basic and Medical Biosciences, Faculty of Life Sciences and Medicine, King's College London, London, SE1 9RT, UK.

A D Burden (AD)

Institute of Infection, Immunity and Inflammation, University of Glasgow, Glasgow, G12 8TA, UK.

C E M Griffiths (CEM)

Dermatology Centre, Salford Royal NHS Foundation Trust, University of Manchester, NIHR Manchester Biomedical Research Centre, Manchester, M6 8HD, UK.

N J Reynolds (NJ)

Institute of Translational and Clinical Medicine, Medical School, University of Newcastle, Department of Dermatology, Royal Victoria Infirmary and NIHR Newcastle Biomedical Research Centre, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, NE2 4HH, UK.

R B Warren (RB)

National Amyloidosis Centre, University College London, London, NW3 2PF, UK.

H J Lachmann (HJ)

National Amyloidosis Centre, University College London, London, NW3 2PF, UK.

F Capon (F)

Department of Medical and Molecular Genetics, King's College London, London, SE1 9RT, UK.

C H Smith (CH)

St John's Institute of Dermatology, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, UK.

Classifications MeSH