Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: A systematic review

Journal of Thrombosis and Haemostasis

Volumn 8, Issue 6, 2010, Pages 1256-1265

  Iorio, A ^a   Halimeh, S ^b   Holzhauer, S ^c   Goldenberg, N ^d   Marchesini, E ^a   Marcucci, M ^a   Young, G ^e   Bidlingmaier, C ^h   Brandao, L R ⁱ   Ettingshausen, C E ^j   Gringeri, A ^f   Kenet, G ^k   Knofler R ^l   Kreuz, W ^j   Kurnik, K ^h   Manner, D ^g   Santagostino, E ^f   Mannucci, P M ^f   Nowak Gottl U ^g  

^a UNIVERSITY OF PERUGIA   (Italy)

^b Medical Thrombosis and Hemophilia Treatment/Blood Transfusion Center Duisburg   (Germany)

^c CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^d Children's Hospital Colorado   (United States)

^e CHILDREN S HOSPITAL LOS ANGELES   (United States)

^f FONDAZIONE IRCCS CA GRANDA OSPEDALE MAGGIORE POLICLINICO   (Italy)

^g UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^h LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

ⁱ HOSPITAL FOR SICK CHILDREN   (Canada)

^j UNIVERSITY HOSPITAL FRANKFURT   (Germany)

^k SHEBA MEDICAL CENTER   (Israel)

^l UNIVERSITY HOSPITAL CARL GUSTAV CARUS   (Germany)

more..

Author keywords

Factor VIII concentrates; Hemophilia A; Inhibitor development; Previously untreated patients; Systematic review

Indexed keywords

ALLOANTIBODY; BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 8;

ARTICLE; CLINICAL TRIAL; DISEASE SEVERITY; FOLLOW UP; HEMOPHILIA A; HUMAN; PRIORITY JOURNAL; SENSITIVITY ANALYSIS; SYSTEMATIC REVIEW;

FACTOR VIII; HEMOPHILIA A; HUMANS; PROSPECTIVE STUDIES; REGRESSION ANALYSIS;

EID: 77954506432     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/j.1538-7836.2010.03823.x     Document Type: Article

References (23)

1. Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992, 232:25-32.
2. Astermark J, Petrini P, Tengborn L, Schulman S, Ljung R, Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 1999, 105:1109-13.
3. Fischer K, van der Bom JG, Mauser-Bunschoten EP, Roosendaal G, Prejs R, Grobbee DE, van den Berg HM. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001, 7:446-52.
4. Ljung R. Paediatric care of the child with haemophilia. Haemophilia 2002, 8:178-82.
5. Blanchette VS, Manco-Johnson M, Santagostino E, Ljung R. Optimizing factor prophylaxis for the haemophilia population: where do we stand? Haemophilia 2004, 10:97-104.
6. Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003, 102:2358-63.
7. Guerois C, Laurian Y, Rothschild C, Parquet-Gernez A, Duclos AM, Negrier C, Vicariot M, Fimbel B, Fressinaud E, Fiks-Sigaud M. Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of highly purified plasma-derived concentrate. Thromb Haemost 1995, 73:215-8.
8. Peerlinck K, Rosendaal FR, Vermylen J. Incidence of inhibitor development in a group of young hemophilia A patients treated exclusively with lyophilized cryoprecipitate. Blood 1993, 81:3332-5.
9. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003, 9:418-35.
10. Goudemand J, Rothschild C, Demiguel V, Vinciguerrat C, Lambert T, Chambost H, Borel-Derlon A, Claeyssens S, Laurian Y, Calvez T. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 2006, 107:46-51.
11. Chalmers EA, Brown SA, Keeling D, Liesner R, Richards M, Stirling D, Thomas A, Vidler V, Williams MD, Young D. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007, 13:149-55.
12. Gouw SC, van der Bom JG, Auerswald G, Ettinghausen CE, Tedgard U, van den Berg HM. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood 2007, 109:4693-7.
13. Dasgupta S, Repesse Y, Bayry J, Navarrete AM, Wootla B, Delignat S, Irinopoulou T, Kamate C, Saint-Remy JM, Jacquemin M, Lenting PJ, Borel-Derlon A, Kaveri SV, Lacroix-Desmazes S. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007, 109:610-2.
14. Waters B, Qadura M, Burnett E, Chegeni R, Labelle A, Thompson P, Hough C, Lillicrap D. Anti-CD3 prevents factor VIII inhibitor development in hemophilia A mice by a regulatory CD4 + CD25 +-dependent mechanism and by shifting cytokine production to favor a Th1 response. Blood 2009, 113:193-203.
15. Qadura M, Waters B, Burnett E, Chegeni R, Bradshaw S, Hough C, Othman M, Lillicrap D. Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine microenvironments in hemophilia A mice. Blood 2009, 114:871-80.
16. Mannucci P, Gringeri A, Peyvandi F, Santagostino E. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers). Haemophilia 2009, 13:65-8.
17. Stroup DF, Berlin JA, Morton SC, Olkin I, Williamson GD, Rennie D, Moher D, Becker BJ, Sipe TA, Thacker SB. Meta-analysis of observational studies in epidemiology: a proposal for reporting. JAMA 2000, 283:2008-12.
18. von Elm E, Altman DG, Egger M, Pocock SJ, Gotzsche PC, Vandenbroucke JP. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies. Ann Intern Med 2007, 147:573-7.
19. Wells G, Shea B, O'Connel D, Robertson J, Peterson J, Welch V, Losos M, Tugwell P. The Newcastle-Ottawa Scale (NOS) for assessing the Quality of Nonrandomized Studies in meta-analysis. 2009
20. Report of expert meeting on factor VIII products and inhibitor development. 2007, DOC Ref:EMEA/CHMP/BPWP/12835/2006
21. Laird NM, Mosteller F. Some statistical methods for combining experimental results. Int J Technol Assess Health Care 1990, 6:5-30.
22. Calvez T, Laurian Y, Goudemand J. Inhibitor incidence with recombinant vs. plasma-derived FVIII in previously untreated patients with severe hemophilia A: homogeneous results from four published observational studies. J Thromb Haemost 2008, 6:390-2.
23. Kurnik K, Bidlingmaier C, Engl W, Chehadeh H, Reipert B, Auerswald G. New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development. Haemophilia 2010, 16:256-62.