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Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever.

Gül A, Ozdogan H, Erer B, Ugurlu S, Kasapcopur O, Davis N, Sevgi S - Arthritis Res. Ther. (2015)

Bottom Line: Primary efficacy outcome measure was the proportion of patients with 50 % or more reduction in attack frequency.All 9 patients achieved a 50 % or more reduction in attack frequency, and only one patient had an attack during the treatment period.Five patients had an attack during the 2-month follow-up, occurring median 71 (range, 31 to 78) days after the last dose.

View Article: PubMed Central - PubMed

Affiliation: Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology, 34093 Fatih, Istanbul, Turkey. agul@istanbul.edu.tr.

ABSTRACT

Introduction: This open-label pilot study aimed to investigate the efficacy of canakinumab in colchicine-resistant familial Mediterranean fever (FMF) patients.

Method: Patients with one or more attacks in a month in the preceding 3 months despite colchicine were eligible to enter a 30-day run-in period. Patients who had an attack during the first run-in period advanced to a second 30-day period. At the first attack, patients started to receive three canakinumab 150 mg subcutaneous injections at 4-week intervals, and were then followed for an additional 2 months. Primary efficacy outcome measure was the proportion of patients with 50 % or more reduction in attack frequency. Secondary outcome measures included time to next attack following last canakinumab dose and changes in quality of life assessed by SF-36.

Results: Thirteen patients were enrolled in the run-in period and 9 advanced to the treatment period. All 9 patients achieved a 50 % or more reduction in attack frequency, and only one patient had an attack during the treatment period. C-reactive protein and serum amyloid A protein levels remained low throughout the treatment period. Significant improvement was observed in both physical and mental component scores of the Short Form-36 at Day 8. Five patients had an attack during the 2-month follow-up, occurring median 71 (range, 31 to 78) days after the last dose. Adverse events were similar to those observed in the previous canakinumab trials.

Conclusion: Canakinumab was effective at controlling the attack recurrence in patients with FMF resistant to colchicine. Further investigations are warranted to explore canakinumab's potential in the treatment of patients with colchicine resistant FMF.

Trial registration: ClinicalTrials.gov NCT01088880 . Registered 16 March 2010.

No MeSH data available.


Related in: MedlinePlus

Number and severity of familial Mediterranean fever attacks observed throughout the study period. Severity of the attack was assessed according to patients’ assessment in relation to their previous experiences
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Fig1: Number and severity of familial Mediterranean fever attacks observed throughout the study period. Severity of the attack was assessed according to patients’ assessment in relation to their previous experiences

Mentions: Five patients, all p.Met694Val homozygous and receiving 2 mg/day colchicine, subsequently experienced an attack within the 2-month follow up, which occurred at a median 71 days (range 31 to 78 days) after the last canakinumab injection (Fig. 1). Median baseline CRP and SAA levels (58 mg/L and 162 mg/L, respectively) on day 1 of canakinumab administration normalized (2.5 mg/L and 5.8 mg/L, respectively) by day 8, and remained low throughout the study (Additional file 2: Figure S1). No other significant laboratory abnormalities were noted.Fig. 1


Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever.

Gül A, Ozdogan H, Erer B, Ugurlu S, Kasapcopur O, Davis N, Sevgi S - Arthritis Res. Ther. (2015)

Number and severity of familial Mediterranean fever attacks observed throughout the study period. Severity of the attack was assessed according to patients’ assessment in relation to their previous experiences
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC4559892&req=5

Fig1: Number and severity of familial Mediterranean fever attacks observed throughout the study period. Severity of the attack was assessed according to patients’ assessment in relation to their previous experiences
Mentions: Five patients, all p.Met694Val homozygous and receiving 2 mg/day colchicine, subsequently experienced an attack within the 2-month follow up, which occurred at a median 71 days (range 31 to 78 days) after the last canakinumab injection (Fig. 1). Median baseline CRP and SAA levels (58 mg/L and 162 mg/L, respectively) on day 1 of canakinumab administration normalized (2.5 mg/L and 5.8 mg/L, respectively) by day 8, and remained low throughout the study (Additional file 2: Figure S1). No other significant laboratory abnormalities were noted.Fig. 1

Bottom Line: Primary efficacy outcome measure was the proportion of patients with 50 % or more reduction in attack frequency.All 9 patients achieved a 50 % or more reduction in attack frequency, and only one patient had an attack during the treatment period.Five patients had an attack during the 2-month follow-up, occurring median 71 (range, 31 to 78) days after the last dose.

View Article: PubMed Central - PubMed

Affiliation: Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology, 34093 Fatih, Istanbul, Turkey. agul@istanbul.edu.tr.

ABSTRACT

Introduction: This open-label pilot study aimed to investigate the efficacy of canakinumab in colchicine-resistant familial Mediterranean fever (FMF) patients.

Method: Patients with one or more attacks in a month in the preceding 3 months despite colchicine were eligible to enter a 30-day run-in period. Patients who had an attack during the first run-in period advanced to a second 30-day period. At the first attack, patients started to receive three canakinumab 150 mg subcutaneous injections at 4-week intervals, and were then followed for an additional 2 months. Primary efficacy outcome measure was the proportion of patients with 50 % or more reduction in attack frequency. Secondary outcome measures included time to next attack following last canakinumab dose and changes in quality of life assessed by SF-36.

Results: Thirteen patients were enrolled in the run-in period and 9 advanced to the treatment period. All 9 patients achieved a 50 % or more reduction in attack frequency, and only one patient had an attack during the treatment period. C-reactive protein and serum amyloid A protein levels remained low throughout the treatment period. Significant improvement was observed in both physical and mental component scores of the Short Form-36 at Day 8. Five patients had an attack during the 2-month follow-up, occurring median 71 (range, 31 to 78) days after the last dose. Adverse events were similar to those observed in the previous canakinumab trials.

Conclusion: Canakinumab was effective at controlling the attack recurrence in patients with FMF resistant to colchicine. Further investigations are warranted to explore canakinumab's potential in the treatment of patients with colchicine resistant FMF.

Trial registration: ClinicalTrials.gov NCT01088880 . Registered 16 March 2010.

No MeSH data available.


Related in: MedlinePlus