Zero Incidence of Inhibitors in Previously Untreated Patients who Received Intermediate Purity Factor VIII Concentrate or Factor IX Complex

Schimpf, K.; Schwarz, Peter Hans; Kunschak, M

doi:10.1055/s-0038-1653818

Cited by 19 publications

(23 citation statements)

References 8 publications

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“…The cumulative risk of inhibitor development (reported in the text, calculated from data supplied, or interpreted from printed graphs) varied from 0% [47] to 39% [37]. There are a number of possible explanations for this.…”

Section: Variations In Cumulative Risk Of Inhibitor In Different Studiesmentioning

confidence: 99%

“…The cohorts reported on are relatively small, ranging from 22 patients [47] to 101 [41]. The duration of observation ranged from 6 months median [44] to 23 years [38].…”

mentioning

confidence: 99%

“…This is borne out by the fact that the cumulative risk of high‐responding inhibitors is greater in the former studies than in the recombinant studies. However, the differences between the studies which used a single plasma‐derived product and the others are manifest even when we consider only high‐responding inhibitors, suggesting that they cannot be explained by differences in testing frequency or can they be explained as well as, by differences in the degree of severity of the disease, as three of them [32,45,47] recruited only severe haemophiliacs, and one of the studies of rFVIII [46] recruited both severe and moderately affected haemophiliacs.…”

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confidence: 99%

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The epidemiology of inhibitors in haemophilia A: a systematic review

2003

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This paper emphasizes the importance of distinguishing between the prevalence, incidence and cumulative incidence of inhibitors in haemophilia A. Incidence and cumulative incidence data will include patients with transient inhibitors or whose inhibitors have been eliminated by treatment. As these will not be included in prevalence data, prevalence studies will tend to give rise to lower figures than incidence studies. As a result, the most accurate estimates of the true risk of inhibitor development comes from prospective studies of newly diagnosed haemophiliacs who are tested regularly for the presence of inhibitors. This paper reports a systematic review of the best available evidence relating to the epidemiology of inhibitors in haemophilia A. Cohort studies, registry data reporting incidence or prevalence of inhibitors in patients with haemophilia A, and prospective studies of factor VIII (FVIII) in the treatment of previously untreated patients which reported the development of inhibitors as an outcome, were included in the review. The overall prevalence of inhibitors in unselected haemophiliac populations was found to be 5-7%. The cumulative risk of inhibitor development varied (0-39%). Incidence and prevalence were substantially higher in patients with severe haemophilia. Studies of patients using a single plasma-derived FVIII (pdFVIII) preparation reported lower inhibitor incidence than those using multiple pdFVIII preparations or single recombinant FVIII preparations. Incidence data should be used to estimate the likely demand for treatments aimed at eliminating inhibitors, whereas the best estimates of the overall burden to the National Health Service (NHS) of treating bleeding episodes in patients with continuing inhibitors will come from prevalence studies.

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Section: Variations In Cumulative Risk Of Inhibitor In Different Studiesmentioning

confidence: 99%

“…The cohorts reported on are relatively small, ranging from 22 patients [47] to 101 [41]. The duration of observation ranged from 6 months median [44] to 23 years [38].…”

mentioning

confidence: 99%

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confidence: 99%

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The epidemiology of inhibitors in haemophilia A: a systematic review

2003

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“…[26][27][28] A systematic review by Wight and Paisley 29 also supports the views that the source of FVIII may influence inhibitor development. A disparate cumulative incidence of inhibitors between plasma-derived (0%-12%) 30,31 and recombinant FVIII products (36%-38.7%) 32,33 was found in PUPs. 29 These results were received with some skepticism by the hemophilia community because the studies included in the analysis were very heterogeneous with regards to size, design, population, inhibitor definition, and also inhibitor incidence (Figure 1).…”

Section: Introductionmentioning

confidence: 97%

How we choose factor VIII to treat hemophilia

Mannucci¹,

Mancuso

Santagostino

2012

Blood

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AbstractIn high-income countries, the large availability of coagulation factors for replacement therapy of patients with hemophilia A has raised the life expectancy of these lifelong bleeders to that of males from the general population. The practicing clinician is offered a multitude of choices among several commercial brands of factor VIII extracted from human plasma or engineered from mammalian cell cultures by means of recombinant DNA technology. This article has the goal to offer our opinions on how to choose among the different products, that we consider interchangeable relevant to their clinical efficacy in the control of bleeding and safety from pathogen transmission. Hence, the main determinants of our choices are price and the risk of occurrence of factor VIII inhibitory alloantibodies. With this as background, we present the rationale underlying the choices for different categories of patients with severe hemophilia A: previously untreated patients, multiply treated patients, and patients undergoing immune tolerance induction with large doses of factor VIII to eradicate inhibitors. Mention is also made to the possible strategies that should be implemented to make available coagulation factors for replacement therapy in developing countries.

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“…The cumulative incidence of inhibitors ranged from 20.3% to 33.0% in PUPs exposed to different brands of plasma-derived concentrates [3,4]. For patients treated only with a single plasma-derived concentrate, containing von Willebrand factor (VWF) the cumulative incidence was much lower, ranging from 0% to 12.4% [5,6]. Other as yet unpublished experiences with plasma-derived, VWF-containing FVIII concentrates (VWF/FVIII) in Norway (Glomstein, et al) and UK (Brown, et al), seem to mirror this low incidence.…”

Section: Introductionmentioning

confidence: 99%