Duquesne, A

Galeotti, C

Guillaume, S

Hachulla, E

Koné-Paut, I

Pillet, P

Richer, O

Rossi-Semerano, L

English

PubMed

Hachulla E

Richer O

Pillet P

Duquesne A

Guillaume S

Rossi-Semerano L

Galeotti C

Koné-Paut I

Directory of Open Access Journals

Pediatric Rheumatology

Interleukin-1 antagonists in Mevalonate Kinase Deficiency

C Galeotti

L Rossi-Semerano

S Guillaume

A Duquesne

P Pillet

O Richer

E Hachulla

I Koné-Paut

Springer - Publisher Connector

POSTER PRESENTATION Open Access
Interleukin-1 antagonists in Mevalonate Kinase
Deficiency
C Galeotti1*, L Rossi-Semerano1, S Guillaume1, A Duquesne2, P Pillet3, O Richer3, E Hachulla4, I Koné-Paut1
From 18th Pediatric Rheumatology European Society (PReS) Congress
Bruges, Belgium. 14-18 September 2011
Background
Mevalonate Kinase Deficiency (MKD) is one of the
autoinflammatory fever syndromes, caused by mutations
in the MKD gene. Systemic inflammatory symptoms
may be mild to severe leading to early death, and recur-
rent bacterial infections frequently develop in the dis-
ease course. On demand NSAID and steroids are the
most commonly used. Few case-reports suggested that
interleukin-1 (IL-1) antagonists could alleviate MKD
symptoms.
Aim
We describe efficacy and safety of IL-1 targeting drugs,
anakinra and canakinumab, in seven children with
MKD.
Methods
A questionnaire containing items of disease activity was
sent to French pediatric and adult rheumatologists. We
collected information from 7 MKD patients treated with
IL-1 antagonists. MKD was suspected clinically then
confirmed genetically in all cases.
Results
The use of IL-1 antagonists was beneficial to all patients
on a long term. Anakinra was daily administered and
canakinumab every 8 weeks, with dose adjustments. The
median clinical score based on fever and symptoms dur-
ing MKD crisis before and after treatment was 9 and 0,5
respectively. The number of days of fever during crisis
before and after treatment was 5,25 and 1,75 respec-
tively. These treatments alleviated biological inflamma-
tory markers. The side effects with anakinra were local
pain, inflammatory signs at the injection site, infections
(bacterial pneumopathy and rhinopharyngitis) and acne.
There was no side effect with canakinumab. Three
patients switched from anakinra to canakinumab.
Conclusion
IL-1 targeting drugs appear effective and safe in our
MKD patients. Controlled trials to further assess their
clinical benefit and safety in MKD patients seem
necessary.
Author details
1Department of Pediatric Rheumatology, CEREMAI, CHU Bicêtre, university of
Paris Sud, France. 2Department of Pediatric Rheumatology, CHU Lyon,
France. 3Department of Pediatric Rheumatology, CHU Bordeaux, France.
4Department of Internal Medicine, CHRU Lille, France.
Published: 14 September 2011
doi:10.1186/1546-0096-9-S1-P7
Cite this article as: Galeotti et al.: Interleukin-1 antagonists in
Mevalonate Kinase Deficiency. Pediatric Rheumatology 2011 9(Suppl 1):P7.
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* Correspondence: caroline.galeotti@gmail.com
1Department of Pediatric Rheumatology, CEREMAI, CHU Bicêtre, university of
Paris Sud, France
Full list of author information is available at the end of the article
Galeotti et al. Pediatric Rheumatology 2011, 9(Suppl 1):P7
http://www.ped-rheum.com/content/9/S1/P7
© 2011 Galeotti et al; licensee BioMed Central Ltd. This is an open access article distributed under the terms of the Creative Commons
Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in
any medium, provided the original work is properly cited.


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Interleukin-1 antagonists in Mevalonate Kinase Deficiency

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