Inhibitor development after switching of <scp>FVIII</scp> concentrate in multitransfused patients with severe haemophilia A

Aznar, José A.; Moret, A.; Ibáñez, Francisco Javier Muñoz; Vila, Carmen; Cabrera, Noelia; Mesa, Elvis; Bonanad, Santiago

doi:10.1111/hae.12439

Cited by 11 publications

(20 citation statements)

References 33 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Although in this study two of the three patients who relapsed had switched product, one to a second-generation rFVIII and the other to a pdFVIII/VWF product, it has been described that neither the number of different FVIII products administered nor the switching of products influence the incidence of inhibitors, at least in multitransfused patients [19,20]. In all cases of inhibitor relapse, patients attempted rescue ITI and were successfully tolerized according to the definitions of ITI success and failure currently in use [21] as described in the G-ITI study [14,15], using pdFVIII/VWF products.…”

Section: Discussionmentioning

confidence: 68%

Long‐term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma‐derived FVIII/VWF product: the long‐term ITI study

Jiménez‐Yuste

Oldenburg²,

Rangarajan

et al. 2016

Haemophilia

View full text Add to dashboard Cite

Introduction: Immune tolerance induction (ITI) is a standard intervention to eradicate inhibitors in haemophilic patients. However, information on the long-term condition of patients who eradicated the inhibitor totally or partially after ITI is scarce. Aim: To perform a long-term follow-up to describe the status of patients reported as ITI success in the G-ITI study. Methods: This was an international, multicentre, observational, retrospective study of the 57 haemophilic patients who were reported as ITI success in the G-ITI study. Demographics and post-ITI clinical data recorded until January 2015 were extracted from the medical records. A descriptive analysis was undertaken. Results: Forty-four patients were evaluable. Post-ITI follow-up was 9.1 years in average. Thirtyseven target joints were affected in 21 patients; 38 patients presented bleeding with a mean of 1.0 AE 1.2 episodes year À1, most of them (271/330) treated with Fanhdi â (Grifols). Around half of the patients underwent at least one surgical procedure. Most venous access complications were of expected nature, requiring hospital stay in practically all cases. Fanhdi was used in 42 patients as the regular haemophilia treatment after ITI, mainly prophylactically. Three patients (6.8%) who were being treated with Fanhdi (prophylaxis), Kogenate (prophylaxis) and Emoclot (on demand), respectively, showed inhibitor relapse (at 29, 53 and 13 months after ITI, with 0.9, 3.65 and 12.5 BU respectively). All of them were successfully tolerized after rescue ITI. Conclusion: After ITI success, all patients continued with regular successful FVIII treatment for haemophilia for more than 9 years. The few inhibitor relapses were successfully overcome after rescue ITI.

show abstract

Section: Discussionmentioning

confidence: 68%

Long‐term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma‐derived FVIII/VWF product: the long‐term ITI study

Jiménez‐Yuste

Oldenburg²,

Rangarajan

et al. 2016

Haemophilia

View full text Add to dashboard Cite

show abstract

“…In summary, we welcome the study from Aznar et al [9], and we point it out to readers of Haemophilia as an example of how to report their own experience and contribute answering the questions still open in the treatment of haemophilia.…”

mentioning

confidence: 89%

“…To provide their contribution, Aznar et al [9] transparently showed the numerous different brands used over time and the number of patients treated with one or another class of concentrates in their center. To provide their contribution, Aznar et al [9] transparently showed the numerous different brands used over time and the number of patients treated with one or another class of concentrates in their center.…”

mentioning

confidence: 99%

“…In this perspective, it is interesting to appraise the contribution provided by Aznar and colleagues [9] in this issue of Haemophilia. In brief, the authors performed a single-centre, retrospective in severe haemophilia A patients who were still inhibitor negative after 150 lifetime ED on one or several different FVIII concentrate products.…”

mentioning

confidence: 99%

“…

Although many aspects of inhibitor development have been elucidated, the role of switching FVIII product concentrate in the risk of inhibitors development in previously treated patients is still under discussion. To provide their contribution, Aznar et al [9] transparently showed the numerous different brands used over time and the number of patients treated with one or another class of concentrates in their center. This way of inclusively reporting data as generated in routine clinical practice would need to be adopted more broadly among hemophilia treater and scientists.

…”

mentioning

confidence: 99%

See 2 more Smart Citations

Answering relevant research questions via careful observation of clinical practice: a fresh look at the old way forward

Matino

Iorio

2014

Haemophilia

View full text Add to dashboard Cite

show abstract

Inhibitors in Hemophilia A: A Pharmacoeconomic Perspective

Messori¹

2017

Semin Thromb Hemost

View full text Add to dashboard Cite

A discussion of the main pharmacoeconomic issues related to inhibitors in hemophilia A cannot be separated from an analysis of the most relevant clinical questions. In the field of inhibitors, the clinical evidence includes several controversial topics, such as high-titer versus low-titer inhibitors, the influence of factor VIII products on inhibitor risk, effectiveness of different immune tolerance induction (ITI) treatments, the role of bypassing agents, and development of new non-factor-VIII compounds. In terms of pharmacoeconomic data, numerous cost estimates have been reported in these fields, but this information is strongly influenced by the wide between-country differences in unit costs. Quite reliable data are, however, available regarding expenditure for replacement therapy in patients without inhibitors, increased lifetime costs caused by high-titer inhibitors, and cost of pharmacological interventions aimed at eradicating inhibitors. As regards the cost-effectiveness ratio, the data on ITI are not conclusive; nonetheless, irrespective of the specific treatments employed for inducing tolerance, their costs seem to be offset by the subsequent savings in the cost per patient. Other issues, such as the cost of low-titer inhibitors in patients with hemophilia A and effectiveness of pharmacological interventions aimed at eradicating low-titer inhibitors are not supported by sound data and will require further research. Finally, although the efficacy and safety profiles of novel treatments (e.g., emicizumab, Roche) warrant long-term clinical studies, the economic advantages of these new compounds might be very substantial both in patients with inhibitors and in those at risk of developing inhibitors.

show abstract

Inhibitor development after switching of FVIII concentrate in multitransfused patients with severe haemophilia A

Cited by 11 publications

References 33 publications

Long‐term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma‐derived FVIII/VWF product: the long‐term ITI study

Long‐term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma‐derived FVIII/VWF product: the long‐term ITI study

Answering relevant research questions via careful observation of clinical practice: a fresh look at the old way forward

Inhibitors in Hemophilia A: A Pharmacoeconomic Perspective

Contact Info

Product

Resources

About