Rationale for individualizing haemophilia care

Blood Coagulation and Fibrinolysis

Volumn 26, Issue 8, 2015, Pages 849-857

  Sørensen, Benny ^a   Auerswald, Günter ^b   Benson, Gary ^c   Elezović, Ivo ^d   Felder, Markus ^e   Lambert, Thierry ^f   Morfini, Massimo ^g   Remor, Eduardo ^h   Salaj, Peter ⁱ   Santagostino, Elena ^j   Šalek, Silva Z ^k   Ljung, Rolf ^l  

^a GUY'S AND ST THOMAS' NHS FOUNDATION TRUST   (United Kingdom)

^b KLINIKUM BREMEN MITTE   (Germany)

^c Northern Ireland Haemophilia Comprehensive Care Centre   (United Kingdom)

^d UNIVERSITY OF BELGRADE   (Serbia)

^e Faculté de Médecine Paris XI ^*  (Switzerland)

^f ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^g CAREGGI UNIVERSITY HOSPITAL   (Italy)

^h UNIVERSIDAD AUTÓNOMA DE MADRID   (Spain)

ⁱ INSTITUTE OF HEMATOLOGY AND BLOOD TRANSFUSION   (Czech Republic)

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

^k KBC Zagreb   (Croatia)

^l LUND UNIVERSITY   (Sweden)

more..

Author keywords

Haemophilia; Health economics; Individualized care; Inhibitors; On demand; Prophylaxis

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; ANALGESIC AGENT; RECOMBINANT BLOOD CLOTTING FACTOR 7A; BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; COAGULATING AGENT; PROTHROMBIN COMPLEX CONCENTRATES;

BLEEDING; CLINICAL FEATURE; DISEASE SEVERITY; GENETIC SUSCEPTIBILITY; HEMOPHILIA; HUMAN; PAIN; PAIN ASSESSMENT; PERSONALIZED MEDICINE; PHENOTYPE; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROPHYLAXIS; REVIEW; RISK FACTOR; ADULT; BLOOD; CHILD; DISEASE MANAGEMENT; FEMALE; HEMOPHILIA A; HEMOPHILIA B; HEMORRHAGE; MALE; METABOLISM; PATHOLOGY; PROCEDURES; QUALITY OF LIFE; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

ADULT; BLOOD COAGULATION FACTORS; CHILD; COAGULANTS; DISEASE MANAGEMENT; FACTOR IX; FACTOR VIII; FEMALE; HEMOPHILIA A; HEMOPHILIA B; HEMORRHAGE; HUMANS; MALE; PRECISION MEDICINE; QUALITY OF LIFE; RISK FACTORS; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 84947126707     PISSN: 09575235     EISSN: 14735733     Source Type: Journal    
DOI: 10.1097/MBC.0000000000000225     Document Type: Review

References (59)

1. White GC, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 2001; 85:560.
2. Den Uijl IE, Mauser Bunschoten EP, Roosendaal G, Schutgens RE, Biesma DH, Grobbee DE, et al. Clinical severity of haemophilia A: does the classification of the 1950s still stand? Haemophilia 2011; 17:849-853.
3. Franchini M, Montagnana M, Targher G, Veneri D, Zaffanello M, Salvagno GL, et al. Interpatient phenotypic inconsistency in severe congenital hemophilia: a systematic review of the role of inherited thrombophilia. Semin Thromb Hemost 2009; 35:307-312.
4. Jayandharan GR, Srivastava A. The phenotypic heterogeneity of severe hemophilia. Semin Thromb Hemost 2008; 34:128-141.
5. Astermark J, Oldenburg J, Pavlova A, Berntorp E, Lefvert AK. Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. Blood 2006; 107:3167-3172.
6. Oldenburg J, Brackmann HH, Schwaab R. Risk factors for inhibitor development in hemophilia A. Haematologica 2000; 85:7-13.
7. Santagostino E, Mancuso ME, Tripodi A, Chantarangkul V, Clerici M, Garagiola I, et al. Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile. J Thromb Haemost 2010; 8:737-743.
8. Ahnström J, Berntorp E, Lindvall K, Björkman S. A 6-year follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia. Haemophilia 2004; 10:689-697.
9. Sørensen B, Ingerslev J. Whole blood clot formation phenotypes in hemophilia A and rare coagulation disorders. Patterns of response to recombinant factor VIIa. J Thromb Haemost 2004; 2:102-110.
10. Björkman S, Blanchette VS, Fischer K, Oh M, Spotts G, Schroth P, et al. Comparative pharmacokinetics of plasma- and albumin-free recombinant factor VIII in children and adults: the influence of blood sampling schedule on observed age-related differences and implications for dose tailoring. J Thromb Haemost 2010; 8:730-736.
11. Anthony LM, Rea CJ, Sorensen B. Inter and intra-individual variation of factor VIII half-life in severe haemophilia A. Blood 2010; 116:Abstract544.
12. Kenet G, Stenmo CB, Blemings A, Wegert W, Goudemand J, Krause M, et al. Intra-patient variability of thromboelastographic parameters following in vivo and ex vivo administration of recombinant activated factor VII in haemophilia patients. A multicentre, randomised trial. Thromb Haemost 2010; 103:351-359.
13. Ling M, Heysen JP, Duncan EM, Rodgers SE, Lloyd JV. High incidence of ankle arthropathy in mild and moderate haemophilia A. Thromb Haemost 2011; 105:261-268.
14. Pettersson H, Nilsson IM, Hedner U, Noréhn K, Ahlberg A. Radiologic evaluation of prophylaxis in severe haemophilia. Acta Paediatr Scand 1981; 70:565-570.
15. Manco-Johnson MJ, Abshire TC, Shapiro AD, 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; 357:535-544.
16. Valentino LA. Blood-induced joint disease: the pathophysiology of hemophilic arthropathy. J Thromb Haemost 2010; 8:1895-1902.
17. Lundin B, Ljung R, Pettersson H. MRI scores of ankle joints in children with haemophilia - comparison with clinical data. Haemophilia 2005; 11:116-122.
18. Soreff J, Blombäck M. Arthropathy in children with severe hemophilia A. Acta Paediatr Scand 1980; 69:667-673.
19. CDC Hemophilia A Mutation Project (CHAMP). CHAMP F8 mutation list. Centers for Disease Control and Prevention. http://www.cdc.gov/ncbddd/hemophilia/champs.html.Accessed 14 April 2014.
20. Knobe KE, Villoutreix BO, Tengborn LI, Petrini P, Ljung RC. Factor VIII inhibitors in two families with mild haemophilia A: structural analysis of the mutations. Haemostasis 2000; 30:268-279.
21. Sørensen B, Rangarajan S, McCorkell S, Ferris S, Mitchell M, Bevan D. Effective haemostasis without FVIII: major cardiac surgery in a patient with mild haemophilia A and a history of inhibitor development. J Thromb Haemost 2009; 7:510.
22. Plug I, Mauser-Bunschoten EP, Bröcker-Vriends AH, van Amstel HK, van der Bom JG, van Diemen-Homan JE, et al. Bleeding in carriers of hemophilia. Blood 2006; 108:52-56.
23. Larsen OH, Fenger-Eriksen C, Christiansen K, Ingerslev J, Sørensen B. Diagnostic performance and therapeutic consequence of thromboelastometry activated by kaolin versus a panel of specific reagents. Anesthesiology 2011; 115:294-302.
24. Dargaud Y, Lienhart A, Negrier C. Prospective assessment of thrombin generation test for dose monitoring of bypassing therapy in hemophilia patients with inhibitors undergoing elective surgery. Blood 2010; 116:5734-5737.
25. Berntorp E, Collins P, D'Oiron R, Ewing N, Gringeri A, Négrier C, et al. Identifying nonresponsive bleeding episodes in patients with haemophilia and inhibitors: a consensus definition. Haemophilia 2011; 17:e202-e210.
26. Rea CJ, Ezban M, Foley JH, Sorensen B. rFVIIa-analogue and rFVIIa enhance clot stability more than FVIII in haemophilia A. J Thromb Haemost 2011; 9 (Suppl 2); Abstract 466.
27. Sørensen B, Ingerslev J. Platelet infusion supports recombinant factor VIIa in a patient with severe haemophilia A and inhibitor - clinical outcome and laboratory observations. Thromb Haemost 2010; 103:1275-1276.
28. European Medicines Agency. Report of expert meeting on Factor VIII products and inhibitor development. European Medicines Agency. http://www.emea.europa.eu/docs/en-GB/document-library/Report/2009/11/WC500015512.pdf.Accessed 14 April 2014.
29. Astermark J, Altisent C, Batorova A, Diniz MJ, Gringeri A, Holme PA, et al. Nongenetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report. Haemophilia 2010; 16:747-766.
30. ter Avest PC, Fischer K, Mancuso ME, Santagostino E, Yuste VJ, van den Berg HM, et al. Risk stratification for inhibitor development at first treatment for severe hemophilia A: a tool for clinical practice. J Thromb Haemost 2008; 6:2048-2054.
31. Gouw SC, van der Bom JG, Marijke van den Berg H. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood 2007; 109:4648-4654.
32. 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; 16:256-262.
33. Morado M, Villar A, Jiménez Yuste V, Quintana M, Hernandez NF. Prophylactic treatment effects on inhibitor risk: experience in one centre. Haemophilia 2005; 11:79-83.
34. Santagostino E, Mancuso ME, Rocino A, Mancuso G, Mazzucconi MG, Tagliaferri A, et al. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol 2005; 130:422-427.
35. van den Berg HM, Fischer K, van der Bom JG. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia 2003; 9 (Suppl 1):27-31.
36. Fischer K, Steen Carlsson K, Petrini P, Holmström M, Ljung R, van den Berg HM, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood 2013; 122:1129-1136.
37. Farrugia A, Cassar J, Kimber MC, Bansal M, Balboni S. Treatment for life: Prophylaxis in haemophilia A is more cost-effective than on-demand therapy in a cost-utility model. Haemophilia 2012; 18:828-829.
38. Feldman BM, Pai M, Rivard GE, Israels S, Poon MC, Demers C, et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost 2006; 4:1228-1236.
39. Collins PW, Blanchette VS, Fischer K, Bjorkman S, Oh M, Fritsch S, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost 2009; 7:413-420.
40. Leissinger C, Gringeri A, Antmen B, Berntorp E, Biasoli C, Carpenter S, et al. Antiinhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med 2011; 365:1684-1692.
41. Konkle BA, Ebbesen LS, Erhardtsen E, Bianco RP, Lissitchkov T, Rusen L, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007; 5:1904-1913.
42. Young G, Auerswald G, Jimenez-Yuste V, Lambert T, Morfini M, Santagostino E, et al. PRO-PACT: Retrospective observational study on the prophylactic use of recombinant factor VIIa in hemophilia patients with inhibitors. Thromb Res 2012; 130:846-870.
43. Geraghty S, Dunkley T, Harrington C, Lindvall K, Maahs J, Sek J. Practice patterns in haemophilia A therapy - global progress towards optimal care. Haemophilia 2006; 12:75-81.
44. Srivastava A, You SK, Ayob Y, Chuansumrit A, de Bosch N, Perez Bianco R, et al. Hemophilia treatment in developing countries: products and protocols. Semin Thromb Hemost 2005; 31:495-500.
45. Salek SZ, Benson GM, Elezovic I, Krenn V, Ljung RC, Morfini M, et al. The need for speed in the management of haemophilia patients with inhibitors. Haemophilia 2011; 17:95-102.
46. Gouw SC, van den Berg HM, le Cessie S, van der Bom JG. Treatment characteristics and the risk of inhibitor development: a multicenter cohort study among previously untreated patients with severe hemophilia A. J Thromb Haemost 2007; 5:1383-1390.
47. Fischer K, van der Bom JG, Prejs R, Mauser-Bunschoten EP, Roosendaal G, Grobbee DE, et al. Discontinuation of prophylactic therapy in severe haemophilia: incidence and effects on outcome. Haemophilia 2001; 7:544-550.
48. Richards M, Altisent C, Batorova A, Chambost H, Dolan G, de Moerloose P, et al. Should prophylaxis be used in adolescent and adult patients with severe haemophilia? A European survey of practice and outcome data. Haemophilia 2007; 13:473-479.
49. Auerswald G, Šalek SZ, Benson G, Elezovic I, Lambert T, Morfini M, et al. Beyond patient benefit: clinical development in hemophilia. Hematology 2012; 17:1-8.
50. Lambert T, Auerswald G, Benson G, Hedner U, Jiménez-Yuste V, Ljung R, et al. Joint disease, the hallmark of haemophilia: what issues and challenges remain despite the development of effective therapies? Thromb Res 2014; 133:967-971.
51. Collins PW. Personalized prophylaxis. Haemophilia 2012; 18 (Suppl 4):131-135.
52. Björkman S, Oh M, Spotts G, Schroth P, Fritsch S, Ewenstein BM, et al. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood 2012; 119:612-618.
53. Othman M, Powell S, Hopman WM, Lillicrap D. Variability of thromboelastographic responses following the administration of rFVIIa to haemophilia A dogs supports the individualization of therapy with a global test of haemostasis. Haemophilia 2010; 16:919-925.
54. Elander J, Robinson G. A brief haemophilia pain coping questionnaire. Haemophilia 2008; 14:1039-1048.
55. Morfini M, Haya S, Tagariello G, Pollmann H, Quintana M, Siegmund B, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007; 13:606-612.
56. Witkop M, Lambing A, Kachalsky E, Divine G, Rushlow D, Dinnen J. Assessment of acute and persistent pain management in patients with haemophilia. Haemophilia 2011; 17:612-619.
57. Wallny T, Hess L, Seuser A, Zander D, Brackmann HH, Kraft CN. Pain status of patients with severe haemophilic arthropathy. Haemophilia 2001; 7:453-458.
58. Remor E. Predictors of treatment difficulties and satisfaction with haemophilia therapy in adult patients. Haemophilia 2011; 17:e901-e905.
59. Miller WR, Rollnic S. Motivational interviewing. Preparing people for change, 2nd ed. New York: The Guilford Press; 2002.