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Kurth M.,Childrens Hospitals and Clinics of Minnesota | Puetz J.,Cardinal Glennon Childrens Medical Center | Kouides P.,University of Rochester | Sanders J.,Cook Childrens Hospital | And 9 more authors.
Journal of Thrombosis and Haemostasis | Year: 2011

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. © 2011 International Society on Thrombosis and Haemostasis.


Lindsay H.,Childrens Center for Cancer and Blood Diseases | Jubran R.F.,Childrens Center for Cancer and Blood Diseases | Wang L.,Saban Research Institute | Kipp B.R.,Mayo Medical School | May W.A.,Childrens Center for Cancer and Blood Diseases
Journal of Pediatrics | Year: 2013

We describe a 12-year-old girl, simultaneously presenting with colonic adenocarcinoma and medulloblastoma from bialleic deletions in the mismatch repair gene PMS2. Her distinctive physical and clinical findings are characteristic of constitutional mismatch repair deficiency syndrome. Earlier recognition of such findings may permit better screening and more effective treatment. Copyright © 2013 Mosby Inc. All rights reserved.

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