A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A

Peyvandi, Flora; Mannucci, Pier Mannuccio; Garagiola, Isabella; El‐Beshlawy, Amal; Elalfy, Mohsen Saleh; Ramanan, Vijay; Eshghi, Peyman; Hanagavadi, Suresh; Varadarajan, Ramabadran; Karimi, Mehran; Manglani, Mamta; Ross, Cecil; Young, Guy; Seth, Tulika; Apte, Shashikant; Nayak, Dinesh; Santagostino, Elena; Mancuso, Maria Elisa; Gonzalez, Adriana C Sandoval; Mahlangu, Johnny; Bonanad, Santiago; Cerqueira, M.; Ewing, Nadia P.; Male, Christoph; Owaidah, Tarek; Arellano, Verónica Soto; Kobrinsky, Nathan L.; Majumdar, Suvankar; Garrido, Rosario; Anupam, Sachdeva; Simpson, Mindy L.; Thomas, Mathew; Zanon, Ezio; Antmen, Bülent; Kavaklı, Kaan; Manco‐Johnson, Marilyn J.; Martínez, Mònica; Marzouka, E; Mazzucconi, Maria Gabriella; Neme, Daniela; Bravo, Ángeles Palomo; Aguilera, Rogelio Paredes; Prezotti, Alessandra Nunes Loureiro; Schimrigk, К.; Wicklund, Brian M.; Zülfikar, Bulent; Rosendaal, Frits R.

doi:10.1056/nejmoa1516437

Cited by 424 publications

(495 citation statements)

References 33 publications

Supporting

Mentioning

445

Contrasting

Unclassified

Order By: Relevance

“…40 The formation of inhibitor antibodies against rFVIII/rFIX products is a severe complication, which is frequently observed in hemophilia therapy, and may render replacement therapy ineffective, especially in hemophilia A. 24,41 Although the progress in the development of products and several immune tolerance induction (ITI) studies that have improved inhibitor management, it remains the major issue in the hemophilia treatment considering both the patient's perspective as well as clinician's and healthcare provider's perspective. 42 In a recent publication, reported the results of a randomized trial to assess the incidence of FVIII inhibitors among patients treated with plasma-derived FVIII or recombinant FVIII (SIPPET, Survey of Inhibitors in Plasma-Product Exposed Toddlers).…”

Section: Recombinant Factor VIII Productsmentioning

confidence: 99%

“…42 In a recent publication, reported the results of a randomized trial to assess the incidence of FVIII inhibitors among patients treated with plasma-derived FVIII or recombinant FVIII (SIPPET, Survey of Inhibitors in Plasma-Product Exposed Toddlers). 41 Only rFVIII products produced in CHO and BHK cells were used (Recombinate Ò , Kogenate FS Ò , Advate Ò , and ReFacto AF Ò ). The authors found that rFVIII products had nearly twice the rate of inhibitor development as to plasma-derived products.…”

Section: Recombinant Factor VIII Productsmentioning

confidence: 99%

See 1 more Smart Citation

Production of recombinant coagulation factors: Are humans the best host cells?

2017

View full text Add to dashboard Cite

The main treatment option for Hemophilia A/B patients involves the administration of recombinant coagulation factors on-demand or in a prophylactic approach. Despite the safety and efficacy of this replacement therapy, the development of antibodies against the coagulation factor infused, which neutralize the procoagulant activity, is a severe complication. The production of recombinant coagulation factors in human cell lines is an efficient approach to avoid such complication. Human cell lines can produce recombinant proteins with post translation modifications more similar to their natural counterpart, reducing potential immunogenic reactions. This review provides a brief overview of the most important characteristics of recombinant FVIII and FIX products available on the market and the improvements that have recently been achieved by the production using human cell lines.

show abstract

Section: Recombinant Factor VIII Productsmentioning

confidence: 99%

Section: Recombinant Factor VIII Productsmentioning

confidence: 99%

Production of recombinant coagulation factors: Are humans the best host cells?

2017

View full text Add to dashboard Cite

show abstract

“…Predhodno nezdravljeni otroci s tež-ko HA, mlajši od 6 let, ki so prejeli rekombinantni FVIII, so imeli za 87 % višjo incidenco pojava inhibitorjev kot otroci, zdravljeni s plazemskim FVIII, ki vsebuje tudi VWF (študija SIPPET) (187,188).…”

Section: Inhibitorji Pri Težki Hemofiliji Aunclassified

“…Novorojenček naj prejme rekombinantni FVIII/FIX (275). Zaradi morebitnega povečanega tveganja za razvoj inhibitorjev pri HA se lahko uporabi plazemski FVIII, in sicer na podlagi podatkov randomizirane prospektivne študije o manjši pojavnosti inhibitorjev pri uporabi plazemskih koagulacijskih faktorjev (187,188), čeprav stališča niso enotna (20,96).…”

Section: Plod S Hemofilijounclassified

Nacionalna priporočila za obravnavo bolnikov s hemofilijo

et al. 2017

View full text Add to dashboard Cite

The document presents recommendations for the comprehensive treatment of patients with haemophilia in Slovenia. It enables health workers at all three levels of health care to become well-acquainted with all the possible aspects of treatment based on the best practices of major centres worldwide, and on the studies and experience of health professionals at the National Haemophilia Centre, University Medical Center Ljubljana. The document contains definitions, treatment algorithms and lists of medications with their characteristics and appropriate dosages. It specifically defines indications for the exclusive competence of the tertiary level in Ljubljana due to the actual availability of teams and laboratory options. It also contains an extensive list of the literature on haemophilia.

show abstract

“…One of the major areas in which it is happening are the international clinical trials of many new products that are already in the market or on the horizon. The SIPPET trial is one of the best examples of that Indian collaboration with the international community [5]. The Haemophilia Federation of India did establish a haemophilia patient registry which has stimulated ICMR to take up this registry and introduce similar registries in common genetic disorders.…”

mentioning

confidence: 99%