Treatment trends for haemophilia A and haemophilia B in the United States: Results from the 2010 practice patterns survey

Haemophilia

Volumn 18, Issue 3, 2012, Pages

  Zappa, S ^a   Mcdaniel, M ^b   Marandola, J ^c   Allen, G ^c  

^a Cook Children's Medical Center ^*  (United States)

^b CHILDREN S HOSPITAL OF ORANGE COUNTY   (United States)

^c RHODE ISLAND HOSPITAL   (United States)

Author keywords

Adherence; Haemophilia; On demand treatment; Prophylaxis

Indexed keywords

BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 9 CONCENTRATE; BLOOD CLOTTING INHIBITOR;

ADOLESCENT; ADULT; AGED; ARTICLE; BLEEDING; CHILD; CLINICAL DECISION MAKING; CLINICAL PRACTICE; COMORBIDITY; CONTINUOUS INFUSION; DISEASE SEVERITY; DOSE RESPONSE; DRUG DOSE REDUCTION; HEALTH CARE ACCESS; HEALTH CARE FACILITY; HEALTH SURVEY; HEMOPHILIA A; HEMOPHILIA B; HUMAN; INFANT; LONG TERM CARE; MAJOR CLINICAL STUDY; NURSE; PATIENT CARE; PATIENT COMPLIANCE; PRESCHOOL CHILD; PRESCRIPTION; PRIMARY PREVENTION; PRIORITY JOURNAL; PROPHYLAXIS; SCHOOL CHILD; SECONDARY PREVENTION; SUBSTITUTION THERAPY; TREATMENT DURATION; TREATMENT PLANNING; UNITED STATES;

ADOLESCENT; ADULT; AGED; BLOOD COAGULATION FACTORS; CHILD; CHILD, PRESCHOOL; DRUG ADMINISTRATION SCHEDULE; FEMALE; HEMOPHILIA A; HEMOPHILIA B; HUMANS; INFANT; MALE; MEDICATION ADHERENCE; MIDDLE AGED; PHYSICIAN'S PRACTICE PATTERNS; UNITED STATES; YOUNG ADULT;

EID: 84860351981     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2012.02770.x     Document Type: Article

References (35)

1. Oldenburg J, Dolan G, Lemm G. Haemophilia care then, now and in the future. Haemophilia 2009; 15: 2-7.
2. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
3. Astermark J, Santagostino E, Keith Hoots W. Clinical issues in inhibitors. Haemophilia 2010; 16: 54-60.
4. Coppola A, Santoro C, Tagliaferri A, Franchini M, G DIM. Understanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategies. Haemophilia 2010; 16: 13-9.
5. Butler RB, McClure W, Wulff K. Practice patterns in haemophilia A therapy - a survey of treatment centres in the United States. Haemophilia 2003; 9: 549-54.
6. 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.
7. Khair K, Lawrence K, Butler R, O'Shea E, Christie BA. Assessment of treatment practice patterns for severe hemophilia A: a global nurse perspective. Acta Haematol 2008; 119: 115-23.
8. Konkle BA, Kessler C, Aledort L et al. Emerging clinical concerns in the ageing haemophilia patient. Haemophilia 2009; 15: 1197-209.
9. Department of Health and Human Services. Centers for Disease Control and Prevention Website. Bleeding and Clotting Disorders Surveillance. Universal Data Collection Summary Reports.
10. Hoots WK, Nugent DJ. Evidence for the benefits of prophylaxis in the management of hemophilia A. Thromb Haemost 2006; 96: 433-40.
11. Hoots WK, Rodriguez N, Boggio L, Valentino LA. Pathogenesis of haemophilic synovitis: clinical aspects. Haemophilia 2007; 13: 4-9.
12. Hacker MR, Page JH, Shapiro AD, Rich-Edwards JW, Manco-Johnson MJ. Central venous access device infections in children with hemophilia: a comparison of prophylaxis and episodic therapy. J Pediatr Hematol Oncol 2007; 29: 458-64.
13. Maclean PS, Richards M, Williams M et al. Treatment related factors and inhibitor development in children with severe haemophilia A. Haemophilia 2011; 17: 282-7.
14. Lindvall K, Colstrup L, Wollter IM et al. Compliance with treatment and understanding of own disease in patients with severe and moderate haemophilia. Haemophilia 2006; 12: 47-51.
15. Srivastava A. Dose and response in haemophilia - optimization of factor replacement therapy. Br J Haematol 2004; 127: 12-25.
16. Escobar MA. Treatment on demand -in vivo dose finding studies. Haemophilia 2003; 9: 360-7.
17. Aronstam A, Wassef M, Hamad Z. Low doses of factor VIII for selected ankle bleeds in severe haemophilia A. Br Med J (Clin Res Ed) 1982; 284: 790.
18. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Int Med 1994; 236: 391-9.
19. Stirling ML, Prescott RJ. Minimum effective dose of intermediate factor-VIII concentrate in haemophiliacs on home therapy. Lancet 1979; 1: 813-4.
20. Shapiro AD, Di Paola J, Cohen A et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood 2005; 105: 518-25.
21. Rocca A, Pizzinelli S, Oliovecchio E, Santagostino E, Rocino A, Iorio A. Replacement therapy with recombinant factor IX. A multicentre evaluation of current dosing practices in Italy. Blood Transfus 2011; 9: 60-9.
22. Aledort LM. Optimizing the treatment of haemophilia B: laboratory and clinical perspectives. Haemophilia 2010; 16: 1-2.
23. Poon M-C. Pharmacokinetics of factors IX, recombinant human activated factor VII and factor XIII. Haemophilia 2006; 12: 61-9.
24. Steen Carlsson K, Hojgard S, Glomstein A et al. On-demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome. Haemophilia 2003; 9: 555-66.
25. Linney D. Managing your health Insurance lifetime limits. Available at Hemaware, 2010.
26. HealthReform.Gov web site. Fact sheet: the affordable care act's new patient's bill of rights. Available at Accessed April 26, 2011.
27. Hooiveld MJ, Roosendaal G, Jacobs KM et al. Initiation of degenerative joint damage by experimental bleeding combined with loading of the joint: a possible mechanism of hemophilic arthropathy. Arthritis Rheum 2004; 50: 2024-31.
28. Roosendaal G, Jansen NW, Schutgens R, Lafeber FP. Haemophilic arthropathy: the importance of the earliest haemarthroses and consequences for treatment. Haemophilia 2008; 14: 4-10.
29. Øvlisen K, Kristensen AT, Jensen AL, Tranholm M. IL-1 beta, IL-6, KC and MCP-1 are elevated in synovial fluid from haemophilic mice with experimentally induced haemarthrosis. Haemophilia 2009; 15: 802-10.
30. Carcao M, Lambert T. Prophylaxis in haemophilia with inhibitors: update from international experience. Haemophilia 2010; 16: 16-23.
31. Santagostino E, Morfini M, Auerswald GK et al. Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs. Haemophilia 2009; 15: 983-9.
32. Allen KN, Kachalsky E. Aging with hemophilia: implications for social work practice. Soc Work Health Care 2010; 49: 327-44.
33. Franchini M, Mannucci PM. Co-morbidities and quality of life in elderly persons with haemophilia. Br J Haematol 2010; 148: 522-33.
34. Globe DR, Cunningham WE, Andersen R et al. The Hemophilia Utilization Group Study (HUGS): determinants of costs of care in persons with haemophilia A. Haemophilia 2003; 9: 325-31.
35. Soucie JM, Nuss R, Evatt B et al. Mortality among males with hemophilia: relations with source of medical care. The Hemophilia Surveillance System Project Investigators. Blood 2000; 96: 437-42.