The use of a single von Willebrand factor-containing, plasma-derived FVIII product in hemophilia A immune tolerance induction: the US experience

被引：43

作者：

Kurth, M. ^{[1
]}

Puetz, J. ^{[2
]}

Kouides, P. ^{[3
]}

Sanders, J. ^{[4
]}

Sexauer, C. ^{[5
]}

Bernstein, J. ^{[6
]}

Gruppo, R. ^{[7
]}

Manco-Johnson, M. ^{[8
,9
]}

Neufeld, E. J. ^{[10
]}

Rodriguez, N. ^{[11
]}

Wicklund, B. ^{[12
]}

Quon, D. ^{[13
]}

Aledort, L. ^{[14
]}

机构：

[1] Childrens Hosp & Clin Minnesota, Dept Pediat, Minneapolis, MN 55404 USA

[2] Cardinal Glennon Childrens Med Ctr, Dept Pediat, St Louis, MO USA

[3] Univ Rochester, Med Ctr, Dept Med, Rochester, NY 14642 USA

[4] Cook Childrens Hosp, Dept Pediat, Ft Worth, TX USA

[5] Univ Oklahoma, Hlth Sci Ctr, Dept Pediat, Oklahoma City, OK 73190 USA

[6] Childrens Ctr Canc & Blood Dis, Las Vegas, NV USA

[7] Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH USA

[8] Univ Colorado, Dept Pediat, Aurora, CO USA

[9] Childrens Hosp Colorado, Hlth Sci Ctr, Aurora, CO USA

[10] Childrens Hosp Boston, Dept Pediat, Boston, MA USA

[11] Univ Texas Texas Med Ctr, Dept Pediat, Houston, TX USA

[12] Childrens Mercy Hosp, Dept Pediat, Kansas City, MO 64108 USA

[13] Orthaped Hosp, Dept Med, Los Angeles, CA USA

[14] Mt Sinai Med Ctr, Dept Med, New York, NY 10029 USA

来源：

JOURNAL OF THROMBOSIS AND HAEMOSTASIS | 2011年 / 9卷 / 11期

关键词：

factor concentrate; hemophilia; immune tolerance; inhibitor; von Willebrand factor; FACTOR-VIII INHIBITORS; HIGH-RISK; REGISTRY; THERAPY; COMPLEX; PURITY; ALLOANTIBODIES; CONCENTRATE; MECHANISMS; RITUXIMAB;

D O I：

10.1111/j.1538-7836.2011.04493.x

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Background: Inhibitors are a serious complication for patients with severe hemophilia A. Immune tolerance induction (ITI) is the primary method for eradicating these inhibitors. The role of type of concentrate and in particular the use of von Willebrand factor-containing, plasma-derived factor VIII (VWF/pd-FVIII) concentrate in primary or rescue ITI remains unclear. Objectives: To report retrospective collection of data on the use of a single VWF/pd-FVIII concentrate in primary and rescue ITI. Methods: Retrospective chart review of hemophilia A inhibitor patients at 11 US institutions who received VWF/pd-FVIII concentrate in primary or rescue ITI. Results: Primary ITI was carried out in eight inhibitor patients with a 75% complete and partial success. Secondary ITI was carried out in 25 inhibitor patients, with 52% attaining complete or partial success. Conclusions: This report represents the largest group of primarily pediatric, high-titer inhibitor patients treated with a single VWF/pd-FVIII concentrate. It adds retrospective data to the use of VWF-containing plasma-derived factor VIII concentrate in primary and rescue ITI, particularly in those patients with characteristics of poor response to ITI.

引用

页码：2229 / 2234

页数：6

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