Susoctocog-alfa (Obizur
Journal
Blood transfusion = Trasfusione del sangue
ISSN: 2385-2070
Titre abrégé: Blood Transfus
Pays: Italy
ID NLM: 101237479
Informations de publication
Date de publication:
07 2020
07 2020
Historique:
received:
10
01
2020
accepted:
19
03
2020
entrez:
24
7
2020
pubmed:
24
7
2020
medline:
7
5
2021
Statut:
ppublish
Résumé
In 2016, a new recombinant B-domain deleted porcine FVIII (rpFVIII) was licensed in Italy for the treatment of acquired haemophilia A (AHA), but only a few cases of patients receiving this have been reported in the literature. Here we report the largest registry of the use of rpFVIII for the treatment of AHA. The objective of this retrospective study was to describe the efficacy and the safety of susoctocog-alfa for AHA. We studied a population of nine patients, recruited in five Italian haemophilia centres presenting AHA, and treated with Obizur rpFVIII was used as a first-line therapy in one-third of the patients. The median delay between clinical onset and diagnosis was 16 days. Initial bolus of infused susoctocog-alfa was 100 IU/kg, lower than the recommended dose. The treatment was maintained for a median of four days. Only one patient with serious co-morbidities and recurrent infections was treated for 32 days. All patients reached a complete resolution of AHA, and no recurrences were reported. Two patients developed a low-titre inhibitor against rpFVIII, neither experienced any complications. In our real world experience, susoctocog-alfa was proven to be an effective and safe therapeutic option for patients with AHA, also at a lower than recommended dosage. In our report, the appearance of low-titre inhibitors against rpFVIII, was not found to be clinically significant.
Sections du résumé
BACKGROUND
In 2016, a new recombinant B-domain deleted porcine FVIII (rpFVIII) was licensed in Italy for the treatment of acquired haemophilia A (AHA), but only a few cases of patients receiving this have been reported in the literature. Here we report the largest registry of the use of rpFVIII for the treatment of AHA. The objective of this retrospective study was to describe the efficacy and the safety of susoctocog-alfa for AHA.
MATERIAL AND METHODS
We studied a population of nine patients, recruited in five Italian haemophilia centres presenting AHA, and treated with Obizur
RESULTS
rpFVIII was used as a first-line therapy in one-third of the patients. The median delay between clinical onset and diagnosis was 16 days. Initial bolus of infused susoctocog-alfa was 100 IU/kg, lower than the recommended dose. The treatment was maintained for a median of four days. Only one patient with serious co-morbidities and recurrent infections was treated for 32 days. All patients reached a complete resolution of AHA, and no recurrences were reported. Two patients developed a low-titre inhibitor against rpFVIII, neither experienced any complications.
DISCUSSION
In our real world experience, susoctocog-alfa was proven to be an effective and safe therapeutic option for patients with AHA, also at a lower than recommended dosage. In our report, the appearance of low-titre inhibitors against rpFVIII, was not found to be clinically significant.
Identifiants
pubmed: 32698943
pii: 2020.00006-20
doi: 10.2450/2020.00006-20
pmc: PMC7375884
doi:
Substances chimiques
Recombinant Proteins
0
Factor VIII
9001-27-8
Types de publication
Case Reports
Journal Article
Multicenter Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
312-321Références
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