Canakinumab Excels For CAPS Treatment

Major Finding: Of 38 canakinumab-naive patients, 68% achieved a complete response based on physician global assessment of disease activity and skin assessment, and normalization of C-reactive protein and serum amyloid A. More than 80% of the canakinumab-naive patients were relapse-free after a median of 290 days of exposure to treatment, and normal levels were also maintained throughout the study period in the rollover patients.

Data Source: From an open-label, single treatment arm, phase III extension study of canakinumab for CAPS.

Disclosures: Dr. Kuemmerle-Deschner disclosed that she has received research grants and consulting fees from Novartis Pharmaceuticals, the maker of canakinumab (Ilaris).

ATLANTA — Canakinumab was safe, well tolerated, and highly effective in the long-term treatment of cryopyrin-associated periodic syndromes in pediatric patients in an open-label phase III extension study.

Treatment of cryopyrin-associated periodic syndromes (CAPS) in 47 patients, aged 3–17 years, was associated with a rapid and sustained clinical and biochemical remission across varying disease severity phenotypes.

Five patients had familial cold autoinflammatory syndrome (FCAS), 23 had Muckle-Wells syndrome (MWS), and 18 had chronic infantile neurologic, cutaneous, and articular syndrome/neonatal-onset multisystem inflammatory disease (CINCA/NOMID). One child was found to not have a CAPS disease phenotype, said Dr. Jasmin B. Kuemmerle-Deschner.

Of the 47 patients, 38 had not previously been treated with the human interleukin-1 beta monoclonal antibody, and 9 were “rolled over” from earlier phase II or III studies. Of the 38 canakinumab-naive patients, 68% achieved a complete response based on physician global assessment of disease activity and skin assessment, plus normalization of C-reactive protein (CRP) and lowering of serum amyloid A (SAA) to less than 10 mg/L for both measures. More than 80% of the canakinumab-naive patients were relapse-free after a median of 290 days of exposure to treatment, and normal CRP and SAA levels were also maintained throughout the study period in the rollover patients, said Dr. Kuemmerle-Deschner, a pediatric rheumatologist at Universitaetsklinikum Tuebingen (Germany).

The response in the treatment-naive patients was rapid, with CRP and SAA falling within 7 days from 14.8 to 2.5 mg/L, and from 40.6 to 7.4 mg/L, respectively. In addition, 8 of the 11 patients who had an incomplete response within 7 days developed a complete response by 14 days, and the remaining patients al a partial response, she said.

All patients were treated with 150 mg (or 2 mg/kg for those weighing 40 kg or less) of subcutaneous canakinumab once every 8 weeks. In cases of incomplete response more frequent dosing or anindditional dose of 300 mg (or 4 mg/kg for thoients weighing 40 kg or less) was allowed, orsesadjus requiradjusted in 17 patients, she said.

Major Finding: Of 38 canakinumab-naive patients, 68% achieved a complete response based on physician global assessment of disease activity and skin assessment, and normalization of C-reactive protein and serum amyloid A. More than 80% of the canakinumab-naive patients were relapse-free after a median of 290 days of exposure to treatment, and normal levels were also maintained throughout the study period in the rollover patients.

Data Source: From an open-label, single treatment arm, phase III extension study of canakinumab for CAPS.

Disclosures: Dr. Kuemmerle-Deschner disclosed that she has received research grants and consulting fees from Novartis Pharmaceuticals, the maker of canakinumab (Ilaris).

ATLANTA — Canakinumab was safe, well tolerated, and highly effective in the long-term treatment of cryopyrin-associated periodic syndromes in pediatric patients in an open-label phase III extension study.

Treatment of cryopyrin-associated periodic syndromes (CAPS) in 47 patients, aged 3–17 years, was associated with a rapid and sustained clinical and biochemical remission across varying disease severity phenotypes.

Five patients had familial cold autoinflammatory syndrome (FCAS), 23 had Muckle-Wells syndrome (MWS), and 18 had chronic infantile neurologic, cutaneous, and articular syndrome/neonatal-onset multisystem inflammatory disease (CINCA/NOMID). One child was found to not have a CAPS disease phenotype, said Dr. Jasmin B. Kuemmerle-Deschner.

Of the 47 patients, 38 had not previously been treated with the human interleukin-1 beta monoclonal antibody, and 9 were “rolled over” from earlier phase II or III studies. Of the 38 canakinumab-naive patients, 68% achieved a complete response based on physician global assessment of disease activity and skin assessment, plus normalization of C-reactive protein (CRP) and lowering of serum amyloid A (SAA) to less than 10 mg/L for both measures. More than 80% of the canakinumab-naive patients were relapse-free after a median of 290 days of exposure to treatment, and normal CRP and SAA levels were also maintained throughout the study period in the rollover patients, said Dr. Kuemmerle-Deschner, a pediatric rheumatologist at Universitaetsklinikum Tuebingen (Germany).

The response in the treatment-naive patients was rapid, with CRP and SAA falling within 7 days from 14.8 to 2.5 mg/L, and from 40.6 to 7.4 mg/L, respectively. In addition, 8 of the 11 patients who had an incomplete response within 7 days developed a complete response by 14 days, and the remaining patients al a partial response, she said.

All patients were treated with 150 mg (or 2 mg/kg for those weighing 40 kg or less) of subcutaneous canakinumab once every 8 weeks. In cases of incomplete response more frequent dosing or anindditional dose of 300 mg (or 4 mg/kg for thoients weighing 40 kg or less) was allowed, orsesadjus requiradjusted in 17 patients, she said.

Major Finding: Of 38 canakinumab-naive patients, 68% achieved a complete response based on physician global assessment of disease activity and skin assessment, and normalization of C-reactive protein and serum amyloid A. More than 80% of the canakinumab-naive patients were relapse-free after a median of 290 days of exposure to treatment, and normal levels were also maintained throughout the study period in the rollover patients.

Data Source: From an open-label, single treatment arm, phase III extension study of canakinumab for CAPS.

Disclosures: Dr. Kuemmerle-Deschner disclosed that she has received research grants and consulting fees from Novartis Pharmaceuticals, the maker of canakinumab (Ilaris).

ATLANTA — Canakinumab was safe, well tolerated, and highly effective in the long-term treatment of cryopyrin-associated periodic syndromes in pediatric patients in an open-label phase III extension study.

Treatment of cryopyrin-associated periodic syndromes (CAPS) in 47 patients, aged 3–17 years, was associated with a rapid and sustained clinical and biochemical remission across varying disease severity phenotypes.

Five patients had familial cold autoinflammatory syndrome (FCAS), 23 had Muckle-Wells syndrome (MWS), and 18 had chronic infantile neurologic, cutaneous, and articular syndrome/neonatal-onset multisystem inflammatory disease (CINCA/NOMID). One child was found to not have a CAPS disease phenotype, said Dr. Jasmin B. Kuemmerle-Deschner.

Of the 47 patients, 38 had not previously been treated with the human interleukin-1 beta monoclonal antibody, and 9 were “rolled over” from earlier phase II or III studies. Of the 38 canakinumab-naive patients, 68% achieved a complete response based on physician global assessment of disease activity and skin assessment, plus normalization of C-reactive protein (CRP) and lowering of serum amyloid A (SAA) to less than 10 mg/L for both measures. More than 80% of the canakinumab-naive patients were relapse-free after a median of 290 days of exposure to treatment, and normal CRP and SAA levels were also maintained throughout the study period in the rollover patients, said Dr. Kuemmerle-Deschner, a pediatric rheumatologist at Universitaetsklinikum Tuebingen (Germany).

The response in the treatment-naive patients was rapid, with CRP and SAA falling within 7 days from 14.8 to 2.5 mg/L, and from 40.6 to 7.4 mg/L, respectively. In addition, 8 of the 11 patients who had an incomplete response within 7 days developed a complete response by 14 days, and the remaining patients al a partial response, she said.

All patients were treated with 150 mg (or 2 mg/kg for those weighing 40 kg or less) of subcutaneous canakinumab once every 8 weeks. In cases of incomplete response more frequent dosing or anindditional dose of 300 mg (or 4 mg/kg for thoients weighing 40 kg or less) was allowed, orsesadjus requiradjusted in 17 patients, she said.