Type and intensity of FVIII exposure on inhibitor development in PUPs with haemophilia A: A patient-level meta-analysis

Thrombosis and Haemostasis

Volumn 113, Issue 5, 2015, Pages 958-967

  Marcucci, Maura ^a,b   Mancuso, Maria Elisa ^c   Santagostino, Elena ^c   Kenet, Gili ^d   Elalfy, Mohssen ^e   Holzhauer, Susanne ^f   Bidlingmaier, Christoph ^g   Ettingshausen, Carmen Escuriola ^h   Iorio, Alfonso ^a   Nowak Göttl, Ulrike ⁱ  

^a MCMASTER UNIVERSITY   (Canada)

^b UNIVERSITY OF MILAN   (Italy)

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

^d TEL AVIV UNIVERSITY   (Israel)

^e AIN SHAMS UNIVERSITY   (Egypt)

^f CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^g LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^h HZRM Haemophilia Centre Rhein Main   (Germany)

ⁱ UNIVERSITY HOSPITAL SCHLESWIG HOLSTEIN   (Germany)

Author keywords

Factor VIII inhibitors; Haemophilia A B; Metaanalysis; Risk factors

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 INHIBITOR; BLOOD CLOTTING INHIBITOR; RECOMBINANT PROTEIN;

AGE; ARTICLE; DISEASE SEVERITY; DRUG DOSE REGIMEN; DRUG EXPOSURE; FAMILY HISTORY; GENE MUTATION; GENOTYPE; HEMOPHILIA A; HUMAN; META ANALYSIS; MULTICENTER STUDY; PREDICTOR VARIABLE; PRIORITY JOURNAL; PROPENSITY SCORE; RISK FACTOR; ADOLESCENT; ADULT; AGED; ALGORITHM; ANTAGONISTS AND INHIBITORS; BLOOD; CHEMISTRY; CHILD; COHORT ANALYSIS; GENETICS; MIDDLE AGED; MUTATION; PROPORTIONAL HAZARDS MODEL; TREATMENT OUTCOME; VERY ELDERLY; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; ALGORITHMS; BLOOD COAGULATION FACTOR INHIBITORS; CHILD; COHORT STUDIES; FACTOR VIII; GENOTYPE; HEMOPHILIA A; HUMANS; MIDDLE AGED; MUTATION; PROPORTIONAL HAZARDS MODELS; RECOMBINANT PROTEINS; RISK FACTORS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84931261706     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1160/TH14-07-0621     Document Type: Article

References (31)

1. Gringeri A, Mantovani LG, Scalone L, et al. Cost of care and quality of life for patients with haemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003; 102: 2358–2363.
2. Guyatt GH, Haynes RB, Jaeschke RZ, et al. Users’ Guides to the Medical Literature: XXV. Evidence-based medicine: principles for applying the Users’ Guides to patient care. Evidence-Based Medicine Working Group. J Am Med Assoc 2000; 284: 1290–1296.
3. Mannucci PM, Gringeri A, Peyvandi F, et al. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers). Haemophilia 2007; 13 (Suppl 5): 65–68.
4. Gouw SC, van der Bom JG, Ljung R, et al.; PedNet and RODIN Study Group. Factor VIII products and inhibitor development in severe haemophilia A. N Engl J Med 2013; 368: 231–239.
5. Gouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe haemophilia A: the RODIN study. Blood 2013; 121: 4046-4055.
6. Iorio A, Skinner MW, Makris M. Factor VIII products and inhibitors in severe haemophilia A. N Engl J Med 2013; 368: 1456.
7. van den Berg HM, Gouw SC, van der Bom JG. Factor VIII products and inhibitors in severe haemophilia A. N Engl J Med 2013; 368: 1457.
8. Kessler CM, Iorio A. The Rodin (Research Of Determinants of INhibitor Development among PUPs with haemophilia) study: the clinical conundrum from the perspective of haemophilia treaters. Haemophilia 2013; 19: 351–354.
9. Iorio A, Halimeh S, Holzhauer S et al. Rate of inhibitor development in previously untreated haemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost 2010; 8: 1256–1265.
10. Sutton AJ, Higgins JPI. Recent developments in meta-analysis. Stat Med 2008; 27: 625–650.
11. Berlin JA, Santanna J, Schmid CH, et al. Individual patient- vs group-level data meta-regressions for the investigation of treatment effect modifiers: ecological bias rears its ugly head. Stat Med 2002; 21: 371–387.
12. Marcucci M, Smith CT, Douketis JD, et al. Patient-level compared with studylevel meta-analyses demonstrate consistency of D-dimer as predictor of venous thromboembolic recurrences. J Clin Epidemiol 2013; 66: 415–425.
13. Boekhorst J, Lari GR, D’Oiron R, et al. Factor VIII genotype and inhibitor development in patients with haemophilia A: highest risk in patients with splice site mutations. Haemophilia 2008; 14: 729–735.
14. Gouw SC, van den Berg HM, Oldenburg J, et al. F8 gene mutation type and inhibitor development in patients with severe haemophilia A: systematic review and meta-analysis. Blood 2012; 119: 2922–2934.
15. Rosembaum PL, Rubin DB. The central role of the propensity score in observational studies for causal effects. Biometrika 1983; 70: 41–55.
16. Austin PC, Grootendorst P, Anderson GM. A comparison of the ability of different propensity score models to balance measured variables between treated and untreated subjects: a Monte Carlo study. Stat Med 2007; 26: 734–753.
17. Zhang H, Holford T, Bracken MB. A tree-based method of analysis for prospective studies. Stat Med 1996 15: 37–49.
18. Klukowska A, Komrska V, Jansen M, et al. Low incidence of factor VIII inhibitors in previously untreated patients during prophylaxis, on-demand treatment and surgical procedures, with Octanate®: interim report from an ongoing prospective clinical study. Haemophilia 2011; 17: 399–406.
19. Shirahata A, Fukutake K, Higasa S, et al.; STUDY GROUP ON FACTORS INVOLVED IN FORMATION OF INHIBITORS TO FACTOR VIII AND IX PREPARATIONS. An analysis of factors affecting the incidence of inhibitor formation in patients with congenital haemophilia in Japan. Haemophilia 2011; 17: 771–776.
20. Mancuso ME, Mannucci PM, Rocino A, et al. Source and purity of factor VIII products as risk factors for inhibitor development in patients with haemophilia A. J Thromb Haemost 2012; 10: 781–790.
21. Auerswald G, Thompson AA, Recht M, et al. Experience of Advate rAHF-PFM in previously untreated patients and minimally treated patients with haemophilia A. Thromb Haemost 2012; 107: 1072–1082.
22. Elalfy MS, Elbarbary NS, Eldebeiky MS, et al. Risk of bleeding and inhibitor development after circumcision of previously untreated or minimally treated severe haemophilia A children. Pediatr Hematol Oncol 2012; 29: 485–493.
23. Elalfy MS, Tantawy AA, Ahmed MH, et al. Frequency of inhibitor development in severe haemophilia A children treated with cryoprecipitate and low-dose immune tolerance induction. Haemophilia 2000; 6: 635–638.
24. Kreuz W, Ettinghausen CE, Zyschka A, et al. Inhibitor development in previously untreated patients with haemophilia A: A prospective long-term followup comparing plasma-derived and recombinant products. Semin Thromb Haemost 2002; 28: 285–290.
25. Bidlingmaier C, Manner D, Halimeh S, et al. Influence of factor VIII products, viral inactivation and dosage regimens on meaningful inhibitor development in children with severe haemophilia A: Results of a non-concurrent cohort study. Abstracts of the XXII Congress of the International Society of Thrombosis and Haemostasis. J Thromb.Haemost 2009; 7 (Suppl s2): 525.
26. Halimeh S, Kenet G, Bidlingmaier C, et al. Abstracts of the XXIXth International Congress of the World Federation of Haemophilia. Validation of a predictive model for identifying an increased risk for clinical meaningful inhibitor development in children with haemophilia A – results of a multicentre cohort study. Haemophilia 2010; 16 (Suppl 4): 20.
27. Kenet G, Bidlingmaier C, Ettingshausen CE, et al. Influence of Factor V G1691A or Prothrombin G20210A Mutation On Inhibitor Development in Patients with Severe Haemophilia A – an Israeli-German Database Study. American Society of Hematology 54th Annual Meeting Abstracts. Blood 2012; 120: 2234.
28. Strauss T, Ravid B, Martinowitz U, et al. Early exposure to recombinant factor concentrates may increase inhibitor development: a single centre cohort study. Abstracts of the XXVIIIth International Congress of the World Federation of Haemophilia. Haemophilia 2008; 14 (Suppl s2): 52.
29. Collins PW, Palmer BP, Chalmers EA, et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe haemophilia A, 2000–2011. Blood 2014; Epub ahead of print.
30. Calvez T, Chambost H, Claeyssens-Donadel S, et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe haemophilia A. Blood 2014; Epub ahead of print.
31. Matino D, Lillicrap D, Astermark J, et al. Switching clotting factor concentrates: considerations in estimating the risk of immunogenicity. Haemophilia 2014; 20: 200–206.