Gene therapy for hemophilia A and B: Patient selection and follow-up, requirements for a cure

Thrombosis and Haemostasis

Volumn 82, Issue 2, 1999, Pages 572-575

  Lusher, Jeanne M ^a,b  

^a WAYNE STATE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b CHILDREN'S HOSPITAL OF MICHIGAN   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9;

CLINICAL RESEARCH; CONFERENCE PAPER; DISEASE SEVERITY; FOLLOW UP; GENE THERAPY; HEMOPHILIA A; HEMOPHILIA B; HUMAN; PATIENT SELECTION; PRIORITY JOURNAL; QUALITY OF LIFE;

EID: 0032725075     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-0037-1615882     Document Type: Conference Paper

References (19)

1. Mannucci PM. Desmopressin: a nontransfusional hemostatic agent. Annu Rev Med. 1990;41:55-64.
2. 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.
3. Petrini P, Lindvall N, Egberg N, Blombäck M. Prophylaxis with factor concentrates in preventing hemophilic arthropathy. Am J Pediatr Hematol Oncol. 1991;13:280-287.
4. van den Berg HM, Nieuwenhuis HK, Mauser-Bunschoten EP, Roosendaal G. Haemophilia prophylaxis in The Netherlands. Semin Hematol. 1994;31:13-15.
5. Berntorp E. Methods of haemophilia care delivery: regular prophylaxis versus episodic treatment. Haemophilia. 1995;1(Suppl 1):3-7.
6. Lofqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients: a long-term follow-up. J Intern Med. 1997;241:395-400.
7. Berntorp E. Guidelines on treatment of haemophilia in Sweden. Haemophilia. 1998;4:421-430.
8. Medical and Scientific Advisory Council. Recommendations concerning prophylaxis. Medical Advisory No. 193, Chapter Advisory No. 197, 1994, The National Hemophilia Foundation.
9. Lee CA. Transfusion-transmitted disease. Baillieres Clin Haematol. 1996;9:369-394.
10. Lusher JM. Recombinant clotting factor concentrates. Baillieres Clin Haematol. 1996;9:291-303.
11. Kaufman RJ, Pipe SW. Can we improve on nature? "Super molecules" of factor VIII. Haemophilia. 1998;4:370-379.
12. Bird A, Isarangkura P, Almagro D, Gonzaga A, Srivastava A. Factor concentrates for haemophilia in the developing world. Haemophilia. 1998;4:481-485.
13. Roberts HR, Hoffman M. Hemophilia and related conditions -inherited deficiencies of prothrombin (factor II), factor V, and factors VII to XII. In: Beutler E, Lichtman MA, Coller BS, Kipps TJ, eds. Williams Hematology, 5th Ed., McGraw-Hill, New York, NY, 1995;1413-1439.
14. Ahlberg Å. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand. 1965;77(Suppl 1):7-80.
15. Nilsson IM, Blombäck M, Ahlberg Å. Our experience in Sweden with prophylaxis on haemophilia. Bibl Haematol. 1968;34:111-124.
16. Ljung RCR. Prophylactic treatment in Sweden: overtreatment or optimal model? Haemophilia. 1998;4:409-412.
17. Pettersson H, Gilbert M. Diagnostic Imaging in Hemophilia: Musculoskeletal and Other Hemorrhagic Complications. Springer-Verlag; Berlin, 1985;56-65.
18. Pettersson H, Ahlberg Å, Nilsson IM. A radiologic classification of hemophilic arthropathy. Clin Orthop. 1980;149:153-159.
19. Verbruggen B, Novakova I, Wessels H, Boezeman J, van den Berg M, Mauser-Bunschoten E. The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitors: improved specificity and reliability. Thromb Haemost. 1995;73:247-251.