Immune tolerance for haemophilia patients with inhibitors: analysis of the western United States experience

Damiano,; Hutter,

doi:10.1046/j.1365-2516.2000.00412.x

Cited by 20 publications

(19 citation statements)

References 26 publications

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“…Forty‐eight studies reported between 1982 and 2003 with 2704 patients receiving a total of 2973 CVADs were included in the meta‐analysis (Table 1) [2–4,6,7,9–22,27–55]. None of the studies was unpublished.…”

Section: Resultsmentioning

confidence: 99%

Central venous access devices in haemophilia

Valentino

Ewenstein

Navickis³

et al. 2004

Haemophilia

139

182

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Central venous access devices (CVADs) can facilitate repeated and/or urgent administration of coagulation factors in haemophilic patients. We conducted a systematic review and meta-analysis of complication rates and risk factors for poor outcome. Forty-eight studies with a total of 2704 patients and 2973 CVADs were included. The primary indications for CVADs were immune tolerance therapy (34.9% of patients), difficult venous access (31.8%) and prophylaxis (29.1%). Fully implanted CVADs were employed in 77.4% of cases and external CVADs in 22.6%. A total of 1190 infections were reported, and the pooled incidence of infection was 0.66 per 1000 CVAD days [confidence interval (CI), 0.44-0.97 per 1000 CVAD days]. Among patients developing infection, the pooled time to first infection was 295 days (CI, 181-479 days). Presence of inhibitors was an independent risk factor for infection with an incidence rate ratio (IRR) of 1.67 (CI, 1.15-2.43). Infection was less likely in patients >6 years of age (IRR, 0.46; CI, 0.27-0.79) and recipients of fully implanted CVADs (IRR, 0.31; CI, 0.12-0.86). Available information on thrombosis was limited, with only 55 cases being reported. Eventually, 31.3% of CVADs were removed, and infection was the reason for removal in 69.9% of cases and thrombosis in 4.1%. The pooled time period CVADs remained indwelling prior to removal or the expiration of the study observation period was 578 days per CVAD (CI, 456-733 days per CVAD). CVADs can confer major benefits in patients with haemophilia requiring long-term venous access, and serious complications are rare.

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Section: Resultsmentioning

confidence: 99%

Central venous access devices in haemophilia

Valentino

Ewenstein

Navickis³

et al. 2004

Haemophilia

139

182

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“…One report of a survey of numerous centres in the western US [31] was excluded as it was not possible to separate out the results for patients with haemophilia A from those with haemophilia B.…”

Section: Populations Studiedmentioning

confidence: 99%

Immune tolerance induction in patients with haemophilia A with inhibitors: a systematic review

2003

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In some patients with haemophilia A, therapeutically administered factor VIII (FVIII) comes to stimulate the production of antibodies (inhibitors) which react with FVIII to render it ineffective. As a result, FVIII cannot be used prophylactically and patients become liable to recurrent bleeds. There are two elements to the management of patients with inhibitors: the treatment of bleeding episodes, and attempts to abolish inhibitor production through the induction of immune tolerance. This paper reports a systematic review of the best available evidence of clinical effectiveness in relation to immune tolerance induction (ITI) in patients with haemophilia A with inhibitors. Owing to the lack of randomized controlled trials on this topic, broad inclusion criteria with regard to study design were applied in order to assess the best available evidence for each intervention. As a result of the clinical and methodological heterogeneity of the evidence, it was not appropriate to pool data across studies; instead, data were synthesized using tabulation and qualitative narrative assessment. The International Registry provides the most reliable estimate of the proportion of successful cases of ITI [48.7%, 95% confidence interval (CI) 42.6-52.7%]. The duration of effect is unclear, but relapses appear to be infrequent. The International Registry shows a rate of relapse of 15% at 15 years. The comparative effectiveness of different protocols is uncertain, as no trials have been undertaken which compare them directly. However, the evidence suggests that the Bonn protocol may be more effective than the Malmö or low-dose protocols. There is no good evidence that immunosuppressive drug regimens are effective.

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“…In numerous studies, an inhibitor titre below 10 BU mL )1 at the start of ITI has been found to correlate with improved outcomes [5,25,[28][29][30]. Based on these findings, published guidelines often suggest delaying ITI until the inhibitor titre has fallen to <10 BU mL )1 [31][32][33][34].…”

Section: Risk Factors For Unsuccessful Itimentioning

confidence: 99%

Immune tolerance induction in 31 children with haemophilia A: is ITI less successful in African Americans?

et al. 2010

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Inhibitor development continues to be a major problem in the treatment of haemophilia. Immune tolerance induction (ITI) continues to be the most effective approach to managing this complication. This study reviews the practice and outcome of ITI at a single centre over a 17-year period. All 31 inhibitor patients have haemophilia A. Two patients with haemophilia A underwent two trials of ITI and a third patient underwent three trials of ITI for a total of 35 courses of ITI in these 31 patients. Most patients had high responding inhibitors, 22 of 31. Seventy-one percent of haemophilia patients achieved tolerance. Courses of ITI in African American (AA) patients with haemophilia A were much less likely to achieve tolerance compared with non-AAs, 57.9% and 92% (P = 0.04) respectively. Most trials of ITI were carried out with recombinant products (25 of 35). While ITI continues to be an effective therapy for patients with inhibitors, it is less effective in AA patients, and patients with higher inhibitor titres. In this refractory group of patients, new approaches are needed.

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Immune tolerance for haemophilia patients with inhibitors: analysis of the western United States experience

Cited by 20 publications

References 26 publications

Central venous access devices in haemophilia

Central venous access devices in haemophilia

Immune tolerance induction in patients with haemophilia A with inhibitors: a systematic review

Immune tolerance induction in 31 children with haemophilia A: is ITI less successful in African Americans?

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