2003; baxter - Supplements - Haematologica

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74E. Berntorpadverse events of this regimen and those of tohigh-dose, long-term protocols.AcknowledgementsThis study was supported by funds from MalmöUniversity Hospital and the region of Scania, Sweden.References1. Ehrenforth S, Kreuz W, Scharrer I, Linde R, Funk M,Gungor T, et al. Incidence of development of factor VIIIand factor IX inhibitors in haemophiliacs. Lancet1992;339:594-8.2. Sultan Y. Prevalence of inhibitors in a population of3435 hemophilia patients in France. French HemophiliaStudy Group. Thromb Haemost 1992;67:600-2.3. Katz J. Prevalence of factor IX inhibitors among patientswith haemophilia B: results of a large-scale North Americansurvey. Haemophilia 1996;2:28-31.4. Soucie JM, Evatt B, Jackson D. Occurrence of hemophiliain the United States. The Hemophilia SurveillanceSystem Project Investigators. Am J Hematol 1998; 59:288-94.5. Gruppo R, Bray GL, Schroth P, Perry M, Gomperts ED.Safety and immunogenicity of recombinant factor VIII(Recombinate) in previously untreated patients (PUPs):a 6.5 year update. The Recombinate PUP Study Group.Thromb Haemost 1997;162 Suppl 6:a[abstract PD-663].6. Rothschild C, Laurian Y, Satre EP, Borel Derlon A,Chambost H, Moreau P, et al. French previously untreatedpatients with severe hemophilia A after exposureto recombinant factor VIII : incidence of inhibitorand evaluation of immune tolerance. Thromb Haemost1998;80:779-83.7. Lusher JM, Shapiro A, Gruppo R, Bedrosian CL, NguyenK. Safety and efficacy in previously untreated patients(PUPs) treated exclusively with B-domain deleted factorVIII (BDD rFVIII). The ReFacto PUP Study Group.Thromb Haemost 2001;Suppl:2558a[abstract].8. Warrier I, Ewenstein BM, Koerper MA, Shapiro A, KeyN, DiMichele D, et al. Factor IX inhibitors and anaphylaxisin hemophilia B. J Pediatr Hematol Oncol1997; 19:23-7.9. Katz J. Prevalence of factor IX inhibitors among patientswith haemophilia B: results of a large-scale North Americansurvey. Haemophilia 1996;2:28-31.10. Warrier I. Factor IX inhibitors and anaphylaxis. In:Rodriguez-Merchan, Lee CA, editors. Inhibitors inPatients with Haemophilia. Oxford: Blackwell ScienceLtd. 2002. p. 87-91.11. Nilsson IM, Berntorp E, Zettervall O. Induction of splittolerance and clinical cure in high-responding hemophiliacswith factor IX antibodies. Proc Natl Acad SciUSA 1986;83:9169-73.12. Nilsson IM, Berntorp E, Rickard KA. Results in threeAustralian haemophilia B patients with high-respondinginhibitors treated with the Malmö model. Haemophilia1995;1:59-66.13. Freiburghaus C, Berntorp E, Ekman M, Gunnarsson J,Kjellberg BM, Nilsson IM. Immunoadsorption forremoval of inhibitors: update on treatments in Malmö-Lund between 1980 and 1995. Haemophilia 1998; 4:16-20.14. Tengborn L, Berntorp E. Continuous infusion of factorIX concentrate to induce immune tolerance in twopatients with haemophilia B. Haemophilia 1998;4:56-9.haematologica vol. 88(supplement n. 12):september 2003
[New Aspects in Treatment of Hemophilia B Patients]Factor IX mutations andinhibitor development inhemophilia Breview paperhaematologica 2003; 88(suppl. n. 12):75-77http://www.haematologica.org/free/immunotolerance2001.pdfDAVID LILLICRAPDepartments of Pathology and Medicine, Queen’s University,Kingston, Ontario, CanadaThe development of neutralizing antibodies(inhibitors) to the infused therapeutic coagulationprotein in hemophilia represents themost serious therapy-related complicationencountered in the clinical management ofhemophilia. In severe hemophilia A, inhibitorshave been reported in 15-50% of patients surveyedin different populations. 1,2 In contrast, theincidence of anti-factor IX allo-antibody generationin severe hemophilia B patients has beendocumented to be 30 nucleotides),21 patients with factor IX inhibitors are detailed.These 21 patients have eight different factor IXmutations: four different nonsense mutations,three frameshift mutations and a single missensemutation. In addition to the information derivedfrom the Mutation Database, several other studieshave also documented the association of partialor complete factor IX deletion mutations withthe development of inhibitors (Table 1 and Figure1). Thus, as with hemophilia A, the propensityfor inhibitor development is far greater in patientswith factor IX mutations that eliminate or severelydisrupt protein synthesis. In contrast, hemophiliaB patients with missense mutations veryrarely develop inhibitors.This pattern of genotype/phenotype associationis well illustrated by recent studies fromSweden where all of the hemophilia B patientswith inhibitor had either deletion or nonsensehaematologica vol. 88(supplement n. 12):september 2003
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