Immune tolerance induction using a factor VIII/von Willebrand factor concentrate (BIOSTATE®), with or without immunosuppression, in Australian paediatric severe haemophilia A patients with high titre inhibitors: A multicentre, retrospective study

Thrombosis Research

Volumn 134, Issue 5, 2014, Pages 1046-1051

  Robertson, Jeremy D ^a   Higgins, Pauline ^a   Price, Jamie ^b   Dunkley, Scott ^c   Barrese, Giulio ^d   Curtin, Julie ^e  

^a ROYAL CHILDREN'S HOSPITAL   (Australia)

^b PRINCESS MARGARET HOSPITAL FOR CHILDREN   (Australia)

^c ROYAL PRINCE ALFRED HOSPITAL   (Australia)

^d CSL LIMITED   (Australia)

^e CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

Author keywords

BIOSTATE; Factor VIII; Haemophilia A; Immune tolerance induction; Paediatric; Von Willebrand factor

Indexed keywords

BLOOD CLOTTING FACTOR 8 PLUS VON WILLEBRAND FACTOR; IMMUNOGLOBULIN; METHYLPREDNISOLONE; RAPAMYCIN; RITUXIMAB; VINCRISTINE; BLOOD CLOTTING FACTOR 8; DRUG COMBINATION; FACTOR VIII, VON WILLEBRAND FACTOR DRUG COMBINATION; VON WILLEBRAND FACTOR;

ABSENCE OF SIDE EFFECTS; ADJUVANT THERAPY; ADOLESCENT; ARTICLE; AUSTRALIA; AUSTRALIAN; CATHETER INFECTION; CATHETER OCCLUSION; CENTRAL VENOUS CATHETER; CHILD; CLINICAL ARTICLE; CLINICAL ASSESSMENT; COHORT ANALYSIS; DISEASE SEVERITY; DRUG MEGADOSE; DRUG TOLERABILITY; DRUG TREATMENT FAILURE; DRUG USE; FOLLOW UP; HEMOPHILIA A; HUMAN; IMMUNE TOLERANCE INDUCTION; IMMUNOLOGICAL TOLERANCE; IMMUNOSUPPRESSIVE TREATMENT; MALE; MEDICATION COMPLIANCE; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; OBSERVATIONAL STUDY; PATIENT COMPLIANCE; PRIORITY JOURNAL; PROGNOSIS; RETROSPECTIVE STUDY; TREATMENT DURATION; TREATMENT RESPONSE; CLINICAL TRIAL; DRUG COMBINATION; DRUG EFFECTS; EPIDEMIOLOGY; FEMALE; IMMUNOLOGY; INFANT; PRESCHOOL CHILD;

ADOLESCENT; AUSTRALIA; CHILD; CHILD, PRESCHOOL; DRUG COMBINATIONS; FACTOR VIII; FEMALE; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; INFANT; MALE; RETROSPECTIVE STUDIES; VON WILLEBRAND FACTOR;

EID: 84927691030     PISSN: 00493848     EISSN: 18792472     Source Type: Journal    
DOI: 10.1016/j.thromres.2014.09.010     Document Type: Article

References (25)

1. Manco-Johnson MJ, Abshire TC, Shapiro A.D., Riske B., Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357:535-44.
2. van den Berg HM, Gouw SC, van der Bom JG. Factor VIII products and inhibitors in severe hemophilia A. N Engl J Med 2013; 368:1457.
3. Astermark J. Prevention and prediction of inhibitor risk. Haemophilia 2012; 18(Suppl. 4):38-42.
4. Witmer C, Young G. Factor VIII inhibitors in hemophilia A: rationale and latest evidence. Ther Adv Hematol 2013; 4:59-72.
5. Collins PW, Chalmers E, Hart D.P., Liesner R., Rangarajan S, Talks K, et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). UK Haemophilia Centre Doctors Organization. Br J Haematol 2013; 160:153-70.
6. Soucie JM, Cianfrini C, Janco R.L., Kulkarni R., Hambleton J, Evatt B, et al. Joint range-of-motion limitations among young males with hemophilia: prevalence and risk factors. Blood 2004; 103:2467-73.
7. Di Minno G, Coppola A Management of patients with long-term inhibitors: is immune tolerance an underestimated life-long solution? Haemophilia 2013; 19(Suppl. 1): 18-23.
8. Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet 1977; 2:933.
9. DiMichele DM, Hoots WK, Pipe S.W., Rivard GE, Santagostino E. International workshop on immune tolerance induction: consensus recommendations. Haemophilia 2007; 13(Suppl. 1):1-22.
10. Benson G, Auerswald G, Elezovic I., Lambert T, Ljung R, Morfini M., et al. Immune tolerance induction in patients with severe hemophilia with inhibitors: expert panel views and recommendations for clinical practice. Eur J Haematol 2012; 88:371-9.
11. Franchini M, Lippi G. Von Willebrand factor-containing factor VIII concentrates and inhibitors in haemophilia A. A critical literature review. Thromb Haemost 2010; 104: 931-40.
12. Ettingshausen CE, Kreuz W. Role of von Willebrand factor in immune tolerance induction. Blood Coagul Fibrinolysis 2005; 16(Suppl. 1):S27-31.
13. Gringeri A, Musso R, Mazzucconi M.G., Piseddu G., Schiavoni M, Pignoloni P, et al. Immune tolerance induction with a high purity von Willebrand factor/VIII complex concentrate in haemophilia A patients with inhibitors at high risk of a poor response. Haemophilia 2007; 13:373-9.
14. Kreuz W. The role of VWF for the success of immune tolerance induction. Thromb Res 2008; 122(Suppl. 2):S7-S12.
15. Hay CR, DiMichele DM, International Immune Tolerance S. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119:1335-44.
16. Gensana M, Altisent C, Aznar J.A., Casana P., Hernandez F, Jorquera JI, et al. Influence of von Willebrand factor on the reactivity of human factor VIII inhibitors with factor VIII. Haemophilia 2001; 7:369-74
17. Kallas A, von Talpsep T. Willebrand factor in factor VIII concentrates protects against neutralization by factor VIII antibodies of haemophilia A patients. Haemophilia 2001; 7:375-80.
18. Gringeri A. VWF/FVIII concentrates in high-risk immunotolerance: the RESIST study. Haemophilia 2007; 13(Suppl. 5):73-7.
19. Rothschild C, D'Oiron R, Borel-Derlon A, Gruel Y., Navarro R, Negrier C. Use of Haemate® P as immune tolerance induction in patients with severe haemophilia A who failed previous induction attempts: a multicentre observational study. Haemophilia 2013; 19:281-6.
20. Greninger DA, Saint-Remy JM, Jacquemin M., Benhida A, DiMichele DM. The use of factor VIII/von Willebrand factor concentrate for immune tolerance induction in haemophilia A patients with high-titre inhibitors: association of clinical outcome with inhibitor epitope profile. Haemophilia 2008; 14:295-302.
21. Kurth M, Puetz J, Kouides P., Sanders J, Sexauer C, Bernstein J., et al. The use of a single von Willebrand factor-containing, plasma-derived FVIII product in hemophilia A immune tolerance induction: the US experience. Journal of thrombosis and haemostasis. J Thromb Haemost 2011; 9:2229-34.
22. Bidlingmaier C, Kurnik K, Escuriola-Ettingshausen C, Jager R., Klamroth R, Male C, et al. Immune tolerance induction with a factor VIII concentrate containing von Willebrand factor (Haemoctin SDH®) in 14 patients with severe haemophilia A. Haemophilia 2011; 17:e837-40.
23. Orsini F, Rotschild C, Beurrier P., Faradji A, Goudemand J, Polack B. Immune tolerance induction with highly purified plasma-derived factor VIII containing von Willebrand factor in hemophilia A patients with high-responding inhibitors. Haematologica 2005; 90:1288-90.
24. Grangl G, Gallistl S, Leschnik B., Muntean W. Port-A-Cath infection causes inhibitor recurrence after initial successful ITI. Haemophilia 2013; 19:e174-5.
25. Collins PW, Mathias M, Hanley J., Keeling D, Keenan R, Laffan M., et al. Rituximab and immune tolerance in severe hemophilia A: a consecutive national cohort. Journal of thrombosis and haemostasis. J Thromb Haemost 2009; 7:787-94.