What is Your opinion on incidence of inhibitor formation in patients treated by plasma derived versus recombinant clotting factor concentrates? What is your current practical approach in choosing CF treatment in previously untreated patients (PUPs) and previously treated patients (PTPs)?
Dear Peter,
I have read a publication entitled "Plasma-derived versus recombinant Factor VIII concentrates for the treatment of haemophilia A: recombinant is better" and found it to be quite good.
Herein some important text taken from the article:
Giles et al. reported that no increase in the incidence of inhibitor development was found in 478 previously treated patients followed up for 1 year after switching from plasma-derived FVIII to recombinant FVIII. Overall, the incidence of true inhibitor development (i.e., negative for inhibitors before conversion to recombinant FVIII and positive after conversion) in previously treated patients with haemophilia A was 2% to 3% over 2 years. This is similar to the incidence in patients treated with plasma-derived FVIII39.
In addition, there is no consensus on the preferred FVIII product for achieving inhibitor eradication during ITI. No conclusive evidence demonstrates that using plasma-derived FVIII rich in vWF will improve the likelihood of successful tolerisation; the only data suggesting that high-content vWF factor concentrates play a role in the outcome of ITI regimens are derived from small in vitro studies40,41, small case series42,43 and retrospective studies44,45, and small, uncontrolled studies46.
Meta-analysis of data from the IITR, NAITR and PROFIT trials found no association between ITI outcome and treatment product (the distribution of success and failure was equivalent: 70% success, 30% failure)47. The International Workshop on Immune Tolerance Induction declared, by consensus recommendation, that ITI is successful using FVIII products with and without vWF (level 2B recommendation)47. Moreover, the International Immune Tolerance Induction Study was interrupted prematurely last year because intercurrent bleeding was significantly higher in the low-dose arm than in the high-dose arm regardless of the product used48.
In conclusion, the published data document that inhibitor development in patients with haemophilia A and the outcome of ITI are not influenced by the type of FVIII product. Thus, because of the low risk of pathogen transmission associated with recombinant FVIII and in accordance with current evidence-based guidelines, recombinant FVIII products should be considered the treatment of choice for patients with haemophilia A. Furthermore, the increased use of recombinant FVIII products over the next few years may lead to decreased costs, rendering the comparison with plasma-derived FVIII concentrates more favourable from an economic point of view.
To view the full article, please use the following link:
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2957496/
For further publications on this topic, please use the following links:
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3629762/
http://www.bloodjournal.org/content/120/4/720?sso-checked=true
A Review of Treatment Options for Hemophilia
by: Marina Freydin
Institution: University of Maryland
Date: October 2007
ABSTRACT
Hemophilias are genetic bleeding disorders for which there are still no cures. Treatment of hemophilias are difficult because patients need repeated infusion of missing coagulation factors, some patients develop inhibitors to the infused factors, and gene therapy is still not suitable for mass treatment. The choice between the two most common therapies, plasma-derived products or recombinant factor VIII or factor IX, is still a dilemma for clinicians involved in the care of patients with hemophilia. This review article details three primary types of treatment methods for patients with hemophilias A and B: plasma-derived products, recombinant factors, and gene therapy, as well as the therapies for patients who develop inhibitors: activated Prothrombin complex concentrates, recombinant factor VIIa, and immunosuppressive or immuno-tolerance inducing treatments. The review also examines the available evidence in relative effectiveness of the different treatment options available in hemophilia patients who do and do not develop inhibitors to treatment. Ultimately, it is suggested that recombinant factors are a preferred treatment method for patients free of inhibitors and FVIIa treatments are the suggested method for patients that do develop inhibitors to the aforementioned regimen.
http://www.jyi.org/issue/a-review-of-treatment-options-for-hemophilia/
Expert Review of Hematology
Volume 6, Issue 5, 2013
EditorialsTreatment for hemophilia: recombinant versus plasma-derived coagulation factors – controversy and debate forever? An ethical medical challenge?
Full text HTMLPDF
Free access
DOI:
10.1586/17474086.2013.834798
Antonio Lirasb & Rebecca García-Trenchardapages 489-492
Publishing models and article dates explained
Published online: 10 Jan 2014
http://www.tandfonline.com/doi/full/10.1586/17474086.2013.834798
The Lancet logo
The past and future of haemophilia: diagnosis, treatments, and its complications
Prof Flora Peyvandi, MDcorrespondenceemail, Isabella Garagiola, PhD, Guy Young, MD
http://thelancet.com/pdfs/journals/lancet/PIIS0140-6736(15)01123-X.pdf
Hoping this will contribute to the discussion on this topic,
Rafik
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