The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura

British Journal of Haematology

Volumn 135, Issue 1, 2006, Pages 101-104

  Scully, Marie ^a   Gattens, Michael ^b   Khair, Kate ^c   Liesner, Ri ^c  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b ADDENBROOKE S HOSPITAL   (United Kingdom)

^c GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

ADAMTS 13; BPL 8Y; Congenital thrombotic thrombocytopenic purpura; Hyperbilirubinaemia; Thrombocytopenia

Indexed keywords

BLOOD CLOTTING FACTOR 8; PROPIOLACTONE; VON WILLEBRAND FACTOR CLEAVING PROTEINASE;

ARTICLE; CHILD; CLINICAL FEATURE; CONTROLLED STUDY; DEMOGRAPHY; FEMALE; FOLLOW UP; HUMAN; HYPERBILIRUBINEMIA; MALE; MICROANGIOPATHY; NEWBORN; PLASMA TRANSFUSION; PRIORITY JOURNAL; PROPHYLAXIS; THROMBOCYTOPENIA; THROMBOTIC THROMBOCYTOPENIC PURPURA;

FACTOR VIII; FEMALE; HUMANS; HYPERBILIRUBINEMIA; INFANT; INFANT, NEWBORN; MALE; PLATELET COUNT; PURPURA, THROMBOTIC THROMBOCYTOPENIC; RECURRENCE; TREATMENT OUTCOME;

EID: 33748342133     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2006.06264.x     Document Type: Article

References (12)

1. Allford, S.L., Harrison, P., Lawrie, A.S., Liesner, R., MacKie, I.J. & Machin, S.J. (2000) Von Willebrand factor-cleaving protease activity in congenital thrombotic thrombocytopenic purpura. British Journal of Haematology, 111, 1215-1222.
2. Assink, K., Schiphorst, R., Allford, S., Karpman, D., Etzioni, A., Brichard, B., van de, K.N., Monnens, L. & van den, H.L. (2003) Mutation analysis and clinical implications of von Willebrand factor-cleaving protease deficiency. Kidney International, 63, 1995-1999.
3. Fujikawa, K., Suzuki, H., McMullen, B. & Chung, D. (2001) Purification of human von Willebrand factor-cleaving protease and its identification as a new member of the metalloproteinase family. Blood, 98, 1662-1666.
4. Furlan, M., Robles, R. & Lamie, B. (1996) Partial purification and characterization of a protease from human plasma cleaving von Willebrand factor to fragments produced by in vivo proteolysis. Blood, 87, 4223-4234.
5. Furlan, M., Robles, R., Morselli, B., Sandoz, P. & Lammle, B. (1999) Recovery and half-life of von Willebrand factor-cleaving protease after plasma therapy in patients with thrombotic thrombocytopenic purpura. Thrombosis and Haemostasis, 81, 8-13.
6. Gerritsen, H.E., Turecek, P.L., Schwarz, H.P., Lammle, B. & Furlan, M. (1999) Assay of von Willebrand factor (vWF)-cleaving protease based on decreased collagen binding affinity of degraded vWF: a tool for the diagnosis of thrombotic thrombocytopenic purpura (TTP). Thrombosis and Haemostasis, 82, 1386-1389.
7. Kentouche, K., Budde, U., Furlan, M., Scharfe, V., Schneppenheim, R. & Zintl, F. (2002) Remission of thrombotic thrombocytopenic purpura in a patient with compound heterozygous deficiency of von Willebrand factor-cleaving protease by infusion of solvent/detergent plasma. Acta Paediatrica, 91, 1056-1059.
8. Lester, W.A., Williams, M.D., Allford, S.L., Enayat, M.S. & Machin, S.J. (2002) Successful treatment of congenital thrombotic thrombocytopenic purpura using the intermediate purity factor VIII concentrate BPL 8Y. British journal of Haematology, 119, 176-179.
9. Levy, G.G., Nichols, W.C., Lian, E.C., Foroud, T., McClintick, J.N., McGee, B.M., Yang, A.Y., Siemieniak, D.R., Stark, K.R., Gruppo, R., Sarode, R., Shurin, S.B., Chandrasekaran, V., Stabler, S.P., Sabio, H., Bouhassira, E.E., Upshaw, J.D. Jr, Ginsburg, D. & Tsai, H.M. (2001) Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature, 413, 488-494.
10. Schiff, D.E., Roberts, W.D., Willert, J. & Tsai, H.M. (2004) Thrombocytopenia and severe hyperbilirubinemia in the neonatal period secondary to congenital thrombotic thrombocytopenic purpura and ADAMTS13 deficiency. Journal of Pediatric Hematology/Oncology, 26, 535-538.
11. Veyradier, A., Obert, B., Haddad, E., Cloarec, S., Nivet, H., Foulard, M., Lesure, F., Delattre, P., Lakhdari, M., Meyer, D., Girma, J.P. & Loirat, C. (2003) Severe deficiency of the specific von Willebrand factor-cleaving protease (ADAMTS 13) activity in a subgroup of children with atypical hemolytic uremic syndrome. Journal of Pediatrics, 142, 310-317.
12. Yarranton, H., Lawrie, A.S., MacKie, I.J., Pinkoski, L., Corash, L. & Machin, S.J. (2005) Coagulation factor levels in cryosupernatant prepared from plasma treated with amotosalen hydrochloride (S-59) and ultraviolet A light. Transfusion, 45, 1453-1458.