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http://repositorio.pediatria.gob.mx:8180/handle/20.500.12103/2232| Título : | A randomized trial of factor VIII and neutralizing antibodies in hemophilia A |
| Creador: | Peyvandi F. |
| Nivel de acceso: | Open access |
| Palabras clave : | Anticuerpos Neutralizantes - sangre Relación Dosis-Respuesta a Droga Quimioterapia Combinada Factor VIII - antagonistas & inhibidores Factor VIII - inmunología Factor VIII - uso terapéutico Hemofilia A - complicaciones Hemofilia A - tratamiento farmacológico Hemofilia A - inmunología Hemorragia - etiología Humanos Isoanticuerpos - análisis Factor de von Willebrand - uso terapéutico Antibodies, Neutralizing - blood Dose-Response Relationship, Drug Drug Therapy, Combination Factor VIII - antagonists & inhibitors Factor VIII - immunology Factor VIII - therapeutic use Hemophilia A - complications Hemophilia A - drug therapy Hemophilia A - immunology Hemorrhage - etiology Humans Isoantibodies - analysis von Willebrand Factor - therapeutic use |
| Descripción : | BACKGROUND: The development of neutralizing anti-factor VIII alloantibodies (inhibitors) in patients with severe hemophilia A may depend on the concentrate used for replacement therapy. METHODS We conducted a randomized trial to assess the incidence of factor VIII inhibitors among patients treated with plasma-derived factor VIII containing von Willebrand factor or recombinant factor VIII. Patients who met the eligibility criteria (male sex, age <6 years, severe hemophilia A, and no previous treatment with any factor VIII concentrate or only minimal treatment with blood components) were included from 42 sites. RESULTS Of 303 patients screened, 264 underwent randomization and 251 were analyzed. Inhibitors developed in 76 patients, 50 of whom had high-titer inhibitors (≥5 Bethesda units). Inhibitors developed in 29 of the 125 patients treated with plasma-derived factor VIII (20 patients had high-titer inhibitors) and in 47 of the 126 patients treated with recombinant factor VIII (30 patients had high-titer inhibitors). The cumulative incidence of all inhibitors was 26.8% (95% confidence interval [CI], 18.4 to 35.2) with plasma-derived factor VIII and 44.5% (95% CI, 34.7 to 54.3) with recombinant factor VIII; the cumulative incidence of high-titer inhibitors was 18.6% (95% CI, 11.2 to 26.0) and 28.4% (95% CI, 19.6 to 37.2), respectively. In Cox regression models for the primary end point of all inhibitors, recombinant factor VIII was associated with an 87% higher incidence than plasma-derived factor VIII (hazard ratio, 1.87; 95% CI, 1.17 to 2.96). This association did not change in multivariable analysis. For high-titer inhibitors, the hazard ratio was 1.69 (95% CI, 0.96 to 2.98). When the analysis was restricted to recombinant factor VIII products other than second-generation full-length recombinant factor VIII, effect estimates remained similar for all inhibitors (hazard ratio, 1.98; 95% CI, 0.99 to 3.97) and high-titer inhibitors (hazard ratio, 2.59; 95% CI, 1.11 to 6.00). CONCLUSIONS Patients treated with plasma-derived factor VIII containing von Willebrand factor had a lower incidence of inhibitors than those treated with recombinant factor VIII. Copyright © 2016 Massachusetts Medical Society. All rights reserved. |
| Colaborador(es) u otros Autores: | Mannucci P.M. Garagiola I. El-Beshlawy A. Elalfy M. Ramanan V. Eshghi P. Hanagavadi S. Varadarajan R. Karimi M. Manglani M.V. Ross C. Young G. Seth T. Apte S. Nayak D.M. Santagostino E. Mancuso M.E. Sandoval Gonzalez A.C. Mahlangu J.N. Bonanad Boix S. Cerqueira M. Ewing N.P. Male C. Owaidah T. Arellano S.V. Kobrinsky N.L. Majumdar S. Perez Garrido R. Sachdeva A. Simpson M. Thomas M. Zanon E. Antmen B. Kavakli K. Manco-Johnson M.J. Martinez M. Marzouka E. Mazzucconi M.G. Neme D. Palomo Bravo A. Paredes Aguilera R. Prezotti A. Schmitt K. Wicklund B.M. Zulfikar B. Rosendaal F.R. |
| Fecha de publicación : | 2016 |
| Tipo de publicación: | Artículo |
| Formato: | |
| Identificador del Recurso : | 10.1056/NEJMoa1516437 |
| Fuente: | New England Journal of Medicine 374(21):2054 - 2064 |
| URI : | http://repositorio.pediatria.gob.mx:8180/handle/20.500.12103/2232 |
| Idioma: | eng |
| Aparece en las colecciones: | Artículos |
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