Frequency of inhibitor development in severe haemophilia A children treated with cryoprecipitate and low‐dose immune tolerance induction

Alfy, Mohsen S. El; Tantawy, Azza Abdel Gawad; Ahmed, Mona A.; Abdin, Iman

doi:10.1046/j.1365-2516.2000.00449.x

Cited by 16 publications

(13 citation statements)

References 14 publications

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“…In this study, positive factor VIII inhibitor was found in 16 patients (35%). The prevalence of factor VIII inhibitor in this study was higher than that in studies by El Alfy et al 16 and Kavakli et al 17 The difference between these studies, apparently, was caused by different methodologies that were applied. In our study, determination of the presence of inhibitor was done only once, hence we were not able to rule out the presence of transient inhibitor.…”

Section: Prevalence Of Factor VIII Inhibitorcontrasting

confidence: 81%

“…El Alfy et al 16 obtained a low prevalence, 10%, of inhibitor among severe hemophilia-A patients who received cryoprecipitate. The study was a prospective study and the Bethesda assay examination was conducted every six months in order to rule out any transient inhibitor.…”

Section: Prevalence Of Factor VIII Inhibitormentioning

confidence: 99%

“…The administration of cryoprecipitate as prophylaxis was also done in studies by El Alfy et al 16 and Kavakli et al, 17 only the objective of cryoprecipitate administration in those studies was to induce immune tolerance. El Alfy et al 16 used cryoprecipitate for immune tolerance induction at a dose of 25 IU/kg three times a week for 1-4 months if inhibitor titre was <40 BU and 50 IU/kg if inhibitor titre was >40 BU. Immune tolerance induction at the mentioned dose gave satisfying results with the disappearance of inhibitor in 8 out of 10 patients.…”

Section: Pattern and Type Of Factor VIII Treatmentmentioning

confidence: 99%

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The prevalence of factor VIII inhibitor in patients with severe hemophilia-A and its clinical characteristics

Harijadi¹,

Gatot²,

Akib³

2016

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Background Hemophilia is a hereditary blood-clotting disorderdue to factor VIII deficiency. Up to this date, the administration offactor VIII in preventing and managing bleeding has been the maintreatment. One of the complications, which may occur due to re-peated administrations of factor VIII, is the formation of factor VIIIantibody (factor VIII inhibitor).Objective To find out the prevalence of severe hemophilia-A withfactor VIII inhibitor and its clinical characteristics.Methods A cross-sectional descriptive study was performed onchildren with severe hemophilia-A at the National Hemophilia CareCentre, Cipto Mangunkusumo Hospital, Jakarta, Indonesia, in June-August 2004.Results Out of 45 children studied, 16 had factor VIII inhibitor withaverage inhibitor titre of 1.15 Bethesda units (BU) (range 0.15-15BU). Most of them (12 patients) had inhibitor titre <5 BU. Chronicarthropathy was found in 17 out of 45 (37%) children with severehemophilia-A, consisting of nine patients from positive inhibitorgroup and 8 patients from negative inhibitor group. Thirty-ninepatients (86%) used an on-demand treatment pattern, among whom15 had positive inhibitor. Among patients receiving prophylactictreatment pattern, only one had positive inhibitor. There were 39patients (86%) treated using cryoprecipitate, among whom factorVIII inhibitor was found in 12, while among those treated with fac-tor VIII concentrate, the inhibitor was positive in 4/6. The averageamount of factor VIII transfused in positive and negative factor VIIIinhibitor groups was similar.Conclusion The prevalence of factor VIII inhibitor in severe he-mophilia-A patients was 35%. Chronic arthropathy occurred moreoften in patients with positive factor VIII inhibitor. Factor VIII inhibi-tor was found more frequently in patients with an on-demand treat-ment pattern and in those using factor VIII concentrate

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Section: Prevalence Of Factor VIII Inhibitorcontrasting

confidence: 81%

Section: Prevalence Of Factor VIII Inhibitormentioning

confidence: 99%

Section: Pattern and Type Of Factor VIII Treatmentmentioning

confidence: 99%

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The prevalence of factor VIII inhibitor in patients with severe hemophilia-A and its clinical characteristics

Harijadi¹,

Gatot²,

Akib³

2016

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“…A Turkish study showed only 26% achieving success (half‐life > 6 hours). Early Egyptian study results suggested that patients with pre‐ITI inhibitor titer < 30 BU usually had a good response. The success rate in patients with pre‐ITI inhibitor titer > 40 BU was lower, at 66%.…”

Section: Discussionmentioning

confidence: 99%

Low‐dose immune tolerance induction for children with hemophilia A with poor‐risk high‐titer inhibitors: A pilot study in China

Chen

Cheng

et al. 2019

Research and Practice in Thrombosis and Haemostasis

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BackgroundImmune tolerance induction (ITI) therapy is currently unaffordable in China. Management of hemophilia A children with high‐titer inhibitor is therefore a challenge.AimTo describe the ITI strategy using plasma‐derived factor VIII/von Willebrand factor concentrate (pdFVIII/VWF) +/− immunosuppression and to report its efficacy in children with hemophilia A having poor‐risk status for ITI success.MethodsA prospective pilot study on children with hemophilia A having poor‐risk status (all with at least inhibitor titer > 10 BU pre‐ITI initiation). Patients received ~50 IU/kg FVIII every other day using domestic intermediate purity pdFVIII/VWF products, either alone or in combination with rituximab +/− prednisone.ResultsSixteen patients with median age 2.9 (range, 2.2‐13.2) years and median pre‐ITI inhibitor titer 30.7 (range, 10.4‐128) BU were enrolled. Analysis at median 14.7 (range, 12.4‐22.6) months’ follow‐up showed a total response rate of 87.5%. This included success (achieving inhibitor < 0.6 BU) in 13 patients (81.3%) in a median of 8.8 (range, 3.2‐11.8) months, and partial success (achieving inhibitor < 5 BU but > 0.6BU) in 1 (6.3%). Compared to the pre‐ITI period, the mean bleeds/month during ITI was 0.51 (64.0% reduction), and joint bleeds/month was 0.34 (64.3% reduction). This low‐dose ITI strategy cost less by 70% to 87% than that for the high‐dose FVIII regimen. No severe adverse events were observed.ConclusionThis low‐dose ITI strategy of pdFVIII/VWF +/− immunosuppression achieved relatively satisfactory outcomes in children with hemophilia A inhibitor having poor‐risk status. This low‐dose regimen showed economic advantages and is therefore suitable for using in China. However, further study in a larger cohort with a longer follow‐up time is needed.

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“…In six of the studies [17,36,[38][39][40][41], patients were treated with a variety of FVIII preparations; in the remaining 15 studies, only one preparation was used. Nine studies [18,31,32,34,[36][37][38]42,43] were restricted to patients with severe haemophilia (FVIII:C < 2% or less), although others did report the incidence separately for patients with different grades of severity of disease (see Table 7). Almost all studies recruited predominantly infants and children.…”

Section: Studies Of the Incidence Of Inhibitor Developmentmentioning

confidence: 99%

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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Frequency of inhibitor development in severe haemophilia A children treated with cryoprecipitate and low‐dose immune tolerance induction

Cited by 16 publications

References 14 publications

The prevalence of factor VIII inhibitor in patients with severe hemophilia-A and its clinical characteristics

The prevalence of factor VIII inhibitor in patients with severe hemophilia-A and its clinical characteristics

Low‐dose immune tolerance induction for children with hemophilia A with poor‐risk high‐titer inhibitors: A pilot study in China

The epidemiology of inhibitors in haemophilia A: a systematic review

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