Efficacy and Safety of Canakinumab in Patients with CAPS Aged

Paul Brogan¹, Michaël Hofer², Jasmin B. Kuemmerle-Deschner³, Bernard R. Lauwerys⁴, Antonio Speziale⁵, K. Abrams⁶, Karolynn Leon⁷, Xiaoling Wei⁸ and Ronald Laxer⁹, ¹Department of Paediatric Rheumatology, UCL Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom, ²Unité Romande de Rhumatologie Pédiatrique, Hospitalier Universitaire Vaudois, Lausanne, Switzerland, ³University Hospital Tuebingen, Tuebingen, Germany, ⁴Cliniques Universitaires Saint-Luc and Université Catholique de Louvain, Brussels, Belgium, ⁵Novartis Pharma AG, Basel, Switzerland, ⁶Novartis Pharmaceutical Corporation, East Hanover, NJ, ⁷Novartis Pharmaceuticals Corporation, East Hanover, NJ, ⁸Novartis Pharma, Beijing, NU, China, ⁹University of Toronto, The Hospital for Sick Children, Toronto, ON, Canada

Meeting: 2015 ACR/ARHP Annual Meeting

Date of first publication: September 29, 2015

Keywords: Autoinflammatory Disease, canakinumab and interleukins (IL)

Session Information

Date: Sunday, November 8, 2015

Title: Miscellaneous Rheumatic and Inflammatory Diseases Poster I

Session Type: ACR Poster Session A

Session Time: 9:00AM-11:00AM

Background/Purpose: Canakinumab (CAN) is indicated for
the treatment of cryopyrin-associated periodic syndrome (CAPS) in patients aged
≥2 years.^1,2 However, the efficacy of
CAN has not yet been studied in infants. Here we present the efficacy and
safety of CAN in patients with CAPS aged <24 months.

Methods: Patients aged 28 days to 4 years
with CAPS received CAN at 2 to 12 mg/kg every 4 or 8 weeks for 56 weeks. Efficacy
was evaluated by complete response (clinical response and normal C-reactive
protein [CRP] levels) and subsequent relapse. Safety assessments included
adverse events (AEs).

Results: Of 17 patients enrolled, 6 were aged
<24 months (44 days to 5 months). The phenotypic distribution of patients
aged <24 months was: familial cold autoinflammatory syndrome (FCAS, n=1),
Muckle–Wells syndrome (MWS, n=4), and neonatal-onset multisystem inflammatory
disease (NOMID, n=1). All 17 patients achieved a clinical response, and 16
achieved a complete response, with 5/6 patients <24 months of age achieving
a complete response. One patient aged 1 year with persistently elevated CRP
levels did not achieve complete response (Table). Of the patients with a
complete response, 4 (2 MWS and 2 NOMID) subsequently relapsed; however, all
regained complete response 2 (1 MWS; 1 NOMID) with and 2 (1 MWS; 1 NOMID)
without dose escalation. Seven patients required dose escalation to achieve
and/or maintain their responses. The proportion of complete responders without
a relapse was higher in the <24 months age group (5/5, 88.9%) versus ≥24
months (7/11, 63.6%). The most common AEs were infections, typically involving
the upper respiratory tract. Four patients experienced a serious AE (SAE), with
no SAE occurring more than once. No patient discontinued due to an AE.

Table. Response assessment

n (%)

Age <24 months

n=6

Age ≥24 months n=11

Total

N=17

Patients achieving complete response

5 (83.3)

11 (100.0)

16 (94.1)

Patients not achieving complete response

1* (16.7)

0 (0.0)

1 (5.9)

*One-year-old patient with persistently elevated CRP

Conclusion: Canakinumab is an effective treatment
for patients with CAPS aged as young as 44 days. The safety profile of
canakinumab was acceptable in younger patients and similar to that observed for
older patients.

References:

1.
ILARIS
(summary of product characteristics). Novartis Europharm Limited; 2014.

2.
Chioato A, et al. Clin
Vaccine Immunol. 2010;17:1952-57.

Disclosure: P. Brogan, Roche Pharmaceuticals and SOBI, 5; M. Hofer, Abbvie, 2,Abbvie and Novartis Pharmaceutical Corporation, 5; J. B. Kuemmerle-Deschner, Novartis Pharmaceutical Corporation, 8,Novartis and Sobi, 5; B. R. Lauwerys, None; A. Speziale, Novartis, 3; K. Abrams, Novartis Pharmaceutical Corporation, 1,Novartis Pharmaceutical Corporation, 3; K. Leon, Novartis Pharmaceutical Corporation, 3; X. Wei, Novartis Pharmaceutical Corporation, 3; R. Laxer, None.

To cite this abstract in AMA style:

Brogan P, Hofer M, Kuemmerle-Deschner JB, Lauwerys BR, Speziale A, Abrams K, Leon K, Wei X, Laxer R. Efficacy and Safety of Canakinumab in Patients with CAPS Aged <24 Months: Results from an Open-Label, Multicenter, Phase III Trial [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/efficacy-and-safety-of-canakinumab-in-patients-with-caps-aged/. Accessed .

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