C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks - final results of the IMPACT2 study

被引:127
作者
Craig, T. J. [1 ]
Bewtra, A. K. [2 ]
Bahna, S. L. [3 ]
Hurewitz, D. [4 ]
Schneider, L. C. [5 ]
Levy, R. J. [6 ]
Moy, J. N. [7 ]
Offenberger, J.
Jacobson, K. W. [8 ]
Yang, W. H. [9 ]
Eidelman, F. [10 ]
Janss, G. [11 ]
Packer, F. R. [12 ]
Rojavin, M. A. [13 ]
Machnig, T. [14 ]
Keinecke, H-O. [15 ]
Wasserman, R. L. [16 ]
机构
[1] Penn State Univ, Coll Med, Hershey, PA 17033 USA
[2] Creighton Univ, Sch Med, Omaha, NE USA
[3] Louisiana State Univ, Hlth Sci Ctr, Shreveport, LA 71105 USA
[4] Allergy Clin Tulsa Inc, Tulsa, OK USA
[5] Childrens Hosp Boston, Boston, MA USA
[6] Family Allergy & Asthma Ctr PC, Atlanta, GA USA
[7] Univ Consultants Allergy & Immunol, Chicago, IL USA
[8] Allergy & Asthma Res Ctr, Eugene, OR USA
[9] Allergy & Asthma Res Ctr, Ottawa, ON, Canada
[10] Cleveland Clin Florida, Weston, FL USA
[11] Allergy Clin, Rapid City, SD USA
[12] Family First Med Ctr, Idaho Falls, ID USA
[13] CSL Behring LLC, King Of Prussia, PA USA
[14] CSL Behring GmbH, Marburg, Germany
[15] Accovion GmbH, Marburg, Germany
[16] DallasAllergyImmunology, Dallas, TX USA
基金
美国国家卫生研究院;
关键词
Berinert; C1 esterase inhibitor concentrate; C1-inhibitor replacement therapy; Hereditary Angioedema; C1-INHIBITOR CONCENTRATE; AUTOANTIBODIES; RELIEF;
D O I
10.1111/j.1398-9995.2011.02702.x
中图分类号
R392 [医学免疫学];
学科分类号
100102 ;
摘要
Background: The placebo-controlled study International Multicentre Prospective Angioedema C1-INH Trial 1 (I.M.P.A.C.T.1) demonstrated that 20 U/kg C1 esterase inhibitor (C1-INH) concentrate (Berinert (R); CSL Behring, Marburg, Germany) is effective in treating acute abdominal and facial Hereditary Angioedema (HAE) attacks. Methods: I.M.P.A.C.T.2 was an open-label extension study of I.M.P.A.C.T.1 to evaluate the safety and efficacy of long-term treatment with 20 U/kg C1-INH for successive HAE attacks at any body location. Efficacy outcomes included patient-reported time to onset of symptom relief (primary) and time to complete resolution of all symptoms (secondary), analysed on a per-patient and per-attack basis. Safety assessments included adverse events, vital signs, viral safety and anti-C1-INH antibodies. Results: During a median study duration of 24 months, 1085 attacks were treated in 57 patients (10-53 years of age). In the per-patient analysis, the median time to onset of symptom relief was 0.46 h and was similar for all types of attacks (0.39-0.48 h); the median time to complete resolution of symptoms was 15.5 h (shortest for laryngeal attacks: 5.8 h; 12.8-26.6 h for abdominal, peripheral and facial attacks). Demographic factors, type of HAE, intensity of attacks, time to treatment, use of androgens and presence of anti-C1-INH antibodies had no clinically relevant effect on the efficacy outcomes. There were no treatment-related safety concerns. No inhibitory anti-C1-INH antibodies were detected in any patient. Conclusions: A single dose of 20 U/kg C1-INH concentrate is safe and provides reliable efficacy in the long-term treatment of successive HAE attacks at any body location.
引用
收藏
页码:1604 / 1611
页数:8
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