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

Scully, Marie; Gattens, Michael; Khair, Kate; Liesner, Ri

doi:10.1111/j.1365-2141.2006.06264.x

Cited by 47 publications

(39 citation statements)

References 14 publications

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“…Bolsa et al [17] reported that the mean ADAMTS13 activity of 8Y V R (13 lots) was 3.5 U/mL, approximately three-fold lower than that of Koate V R -DVI. Thus, the ADAMTS13:Act dosages utilized by Scully et al [5] were substantially lower than that utilized by Naik and Mahoney [16] according to the levels of ADAMTS13 activity in Koate V R -DVI reported herein.…”

Section: Discussioncontrasting

confidence: 57%

“…On the basis of the dose of FVIII:C IU infused by Naik and Mahoney [16] (30-35 IU/kg every 3-4 days) and the results of the present analysis, the effective dose for their patient should be approximately 3-3.5 U ADAMTS13:Act/kg body weight every 3-4 days. The patients treated by Scully et al [5] with 8Y V R received 15-30 FVIII:C IU/kg and the dosing interval varied between every other day to every week, perhaps depending on observed changes in platelet counts and hemoglobin. Bolsa et al [17] reported that the mean ADAMTS13 activity of 8Y V R (13 lots) was 3.5 U/mL, approximately three-fold lower than that of Koate V R -DVI.…”

Section: Discussionmentioning

confidence: 99%

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ADAMTS13 content in plasma‐derived factor VIII/von Willebrand factor concentrates

et al. 2013

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Thrombotic thrombocytopenic purpura (TTP) is a microangiopathy syndrome caused by a congenital or acquired deficiency of ADAMTS13, a plasma metalloprotease that cleaves von Willebrand factor (VWF) and thus prevents the formation of platelet-rich thrombi in the microcirculation. TTP can be fatal if not appropriately and timely treated with the infusion of fresh frozen plasma (FFP) or exchange plasmapheresis, that reverse the process of microangiopathy by removing anti-ADAMTS13 autoantibodies and replacing functional ADAMTS13. The treatment of TTP with FFP is not free from risks and must be administered in hospitals or clinics, owing to the substantial amount of plasma volume infused or exchanged and the frequent need of catheter application. Moreover, most FFPs are not subjected to treatments to remove or inactivate blood-borne infectious agents. A number of recent reports indicate that certain plasma-derived VWF-factor VIII (FVIII) concentrates are clinically effective in the treatment of congenital TTP. In this study, we measured ADAMTS13 levels in various plasma-derived VWF-FVIII concentrates, showing that Koate V R -DVI (Grifols), contained relatively high amounts of ADAMTS13 and that Alphanate V R (Grifols) was the closest other product in terms of protease content. Koate V R -DVI contains, on average (five lots tested), 0.091 6 0.007 Units of ADAMTS13 activity per IU of FVIII. On the basis of this analysis and other reports of VWF-FVIII concentrate utilization in congenital TTP, potential dosing, and future clinical developments are discussed. Am. J.

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Section: Discussioncontrasting

confidence: 57%

Section: Discussionmentioning

confidence: 99%

ADAMTS13 content in plasma‐derived factor VIII/von Willebrand factor concentrates

et al. 2013

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“…There have been an additional 12 cases of acute idiopathic TTP in childhood where ADAMTS13 assays have been performed in the literature and the data is summarized in Table 3 [16][17][18][19][20][21]. Combining the data with our series, the median age is 11 years (range 9 months-17 years).…”

Section: Resultsmentioning

confidence: 92%

Acquired, noncongenital thrombotic thrombocytopenic purpura in children and adolescents: clinical management and the use of ADAMTS 13 assays

McDonald

Liesner²,

Grainger³

et al. 2010

Blood Coagulation &Amp; Fibrinolysis

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Thrombotic thrombocytopenic purpura (TTP) in children is rare and is often thought to be due to congenital ADAMTS13 deficiency. We report seven new cases of noncongenital TTP in children and adolescents and perform a review of the literature where ADAMTS13 assays have been performed in paediatric acquired TTP. All new cases were female and the median age was 13 years. Presenting clinical features included bruising/petechiae or bleeding, fever, neurological, and renal impairment. Median Hb and platelet counts on admission were 66 g/l and 10 x 10(9)/l respectively. Two cases had raised Troponin T levels and one had an abnormal ECG. All cases had ADAMTS13 activity less than 5% and an inhibitor to ADAMTS13. The median number of plasma exchange to remission was 22.5. Six cases received rituximab. Three achieved normal ADAMTS13 activity and remain in remission. Two had persistently low ADAMTS13 activity with high anti-ADAMTS13 IgG levels and one of these relapsed. One had moderately reduced levels of ADAMTS13 in remission with no inhibitor, however, a fall in ADAMTS13 activity and increase in anti-ADAMTS13 IgG heralded clinical relapse. The literature review identified 12 acquired cases showing low ADAMTS13 activity and inhibition of ADAMTS13 (in 95%). In children and adolescents TTP may be due to acquired deficiency of ADAMTS13, associated with an inhibitor/Anti-ADAMTS13 IgG antibodies. Treatment of acquired disease requires PEX and usually immunosuppressive treatment. Rituximab appears to be an effective adjunctive treatment modality.

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“…Recently, virally inactivated intermediate purity factor VIII concentrate that contains a large amount of VWF, and, therefore, ADAMTS13, has been used successfully to treat congenital TTP [34,35]. It appears to be the treatment of choice.…”

Section: Atypical Congenital Ttpmentioning

confidence: 98%

Atypical presentations of thrombotic thrombocytopenic purpura: A review

Sarode

2008

J of Clinical Apheresis

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Thrombotic thrombocytopenic purpura (TTP) is diagnosed by the presence of microangiopathic hemolytic anemia and thrombocytopenia in a patient who frequently presents with central nervous system involvement and, to a lesser extent, renal dysfunction. Recent understanding of the pathophysiology of TTP due to severe deficiency of von Willebrand factor cleaving protease, known as ADAMTS13, has improved diagnosis of TTP. Once the diagnosis is suspected, life-saving therapeutic plasma exchange therapy is initiated. Occasionally, an unusual clinical presentation makes TTP diagnosis difficult, thus resulting in a delay in the management of TTP. This review highlights a variety of atypical TTP presentations described in the literature. It is intended to bring unusual scenarios to the clinician's awareness, so that timely treatment can be delivered.

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

Cited by 47 publications

References 14 publications

ADAMTS13 content in plasma‐derived factor VIII/von Willebrand factor concentrates

ADAMTS13 content in plasma‐derived factor VIII/von Willebrand factor concentrates

Acquired, noncongenital thrombotic thrombocytopenic purpura in children and adolescents: clinical management and the use of ADAMTS 13 assays

Atypical presentations of thrombotic thrombocytopenic purpura: A review

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