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A fourth-generation recombinant factor VIII (FVIII) therapy has demonstrated “convincing” efficacy and tolerability as well as low immunogenicity in previously untreated patients (PUPs) with severe hemophilia A, according to researchers.
The therapy, simoctocog alfa, is a B-domain-deleted recombinant FVIII product derived from a human cell line.
In the NuProtect study (also known as GENA-05), researchers are evaluating simoctocog alfa in PUPs with severe hemophilia A.
Interim results from this study were recently published in Haemophilia. The research is sponsored by Octapharma, makers of simoctocog alfa.
Thus far, NuProtect has enrolled 110 PUPs who will receive simoctocog alfa for up to 100 exposure days (EDs).
The Haemophilia article describes interim results for 66 PUPs treated for at least 20 EDs, the time by which most inhibitors arise. The patients’ median age at first treatment was 13 months (range, 3 months to 135 months).
The patients received simoctocog alfa for standard prophylaxis, surgical prophylaxis, or on-demand treatment.
Forty-five (68.2%) patients received standard prophylaxis, 13 (19.7%) received only on-demand treatment, 8 (12.1%) were initially treated on-demand but later received prophylaxis, and 13 (19.7%) patients received surgical prophylaxis (for 14 procedures).
The median number of EDs was 43.0 (range, 4-120).
Results
The primary objective of this study is to assess the immunogenicity of simoctocog alfa by determining inhibitor activity using the Nijmegen-modified Bethesda assay at a central laboratory.
After a median of 11.5 EDs (range, 6-24), 8 patients had developed high-titer anti-FVIII inhibitors, and 5 patients had developed low-titer inhibitors, 4 of them transient.
The cumulative incidence of all inhibitors was 20.8%—12.8% for high-titer and 8.4% for low-titer inhibitors.
For patients who received prophylaxis, the median annual bleeding rate, during inhibitor-free periods, was 2.40 for all bleeds and 0 for spontaneous bleeds.
When simoctocog alfa was used on-demand, 92.4% of bleeds were controlled with 1 or 2 infusions. In addition, simoctocog alfa was said to demonstrate “excellent” or “good” efficacy in 89% of surgical procedures.
Three patients experienced adverse events (other than inhibitor development) that were considered related to simoctocog alfa.
One patient developed a mild fever. Another had a mild allergic reaction after 3 consecutive infusions of simoctocog alfa (but not after subsequent infusions).
The third patient developed a rash that was described as mild but considered serious due to hospitalization. This patient continued treatment and completed the study. 
A fourth-generation recombinant factor VIII (FVIII) therapy has demonstrated “convincing” efficacy and tolerability as well as low immunogenicity in previously untreated patients (PUPs) with severe hemophilia A, according to researchers.
The therapy, simoctocog alfa, is a B-domain-deleted recombinant FVIII product derived from a human cell line.
In the NuProtect study (also known as GENA-05), researchers are evaluating simoctocog alfa in PUPs with severe hemophilia A.
Interim results from this study were recently published in Haemophilia. The research is sponsored by Octapharma, makers of simoctocog alfa.
Thus far, NuProtect has enrolled 110 PUPs who will receive simoctocog alfa for up to 100 exposure days (EDs).
The Haemophilia article describes interim results for 66 PUPs treated for at least 20 EDs, the time by which most inhibitors arise. The patients’ median age at first treatment was 13 months (range, 3 months to 135 months).
The patients received simoctocog alfa for standard prophylaxis, surgical prophylaxis, or on-demand treatment.
Forty-five (68.2%) patients received standard prophylaxis, 13 (19.7%) received only on-demand treatment, 8 (12.1%) were initially treated on-demand but later received prophylaxis, and 13 (19.7%) patients received surgical prophylaxis (for 14 procedures).
The median number of EDs was 43.0 (range, 4-120).
Results
The primary objective of this study is to assess the immunogenicity of simoctocog alfa by determining inhibitor activity using the Nijmegen-modified Bethesda assay at a central laboratory.
After a median of 11.5 EDs (range, 6-24), 8 patients had developed high-titer anti-FVIII inhibitors, and 5 patients had developed low-titer inhibitors, 4 of them transient.
The cumulative incidence of all inhibitors was 20.8%—12.8% for high-titer and 8.4% for low-titer inhibitors.
For patients who received prophylaxis, the median annual bleeding rate, during inhibitor-free periods, was 2.40 for all bleeds and 0 for spontaneous bleeds.
When simoctocog alfa was used on-demand, 92.4% of bleeds were controlled with 1 or 2 infusions. In addition, simoctocog alfa was said to demonstrate “excellent” or “good” efficacy in 89% of surgical procedures.
Three patients experienced adverse events (other than inhibitor development) that were considered related to simoctocog alfa.
One patient developed a mild fever. Another had a mild allergic reaction after 3 consecutive infusions of simoctocog alfa (but not after subsequent infusions).
The third patient developed a rash that was described as mild but considered serious due to hospitalization. This patient continued treatment and completed the study.
A fourth-generation recombinant factor VIII (FVIII) therapy has demonstrated “convincing” efficacy and tolerability as well as low immunogenicity in previously untreated patients (PUPs) with severe hemophilia A, according to researchers.
The therapy, simoctocog alfa, is a B-domain-deleted recombinant FVIII product derived from a human cell line.
In the NuProtect study (also known as GENA-05), researchers are evaluating simoctocog alfa in PUPs with severe hemophilia A.
Interim results from this study were recently published in Haemophilia. The research is sponsored by Octapharma, makers of simoctocog alfa.
Thus far, NuProtect has enrolled 110 PUPs who will receive simoctocog alfa for up to 100 exposure days (EDs).
The Haemophilia article describes interim results for 66 PUPs treated for at least 20 EDs, the time by which most inhibitors arise. The patients’ median age at first treatment was 13 months (range, 3 months to 135 months).
The patients received simoctocog alfa for standard prophylaxis, surgical prophylaxis, or on-demand treatment.
Forty-five (68.2%) patients received standard prophylaxis, 13 (19.7%) received only on-demand treatment, 8 (12.1%) were initially treated on-demand but later received prophylaxis, and 13 (19.7%) patients received surgical prophylaxis (for 14 procedures).
The median number of EDs was 43.0 (range, 4-120).
Results
The primary objective of this study is to assess the immunogenicity of simoctocog alfa by determining inhibitor activity using the Nijmegen-modified Bethesda assay at a central laboratory.
After a median of 11.5 EDs (range, 6-24), 8 patients had developed high-titer anti-FVIII inhibitors, and 5 patients had developed low-titer inhibitors, 4 of them transient.
The cumulative incidence of all inhibitors was 20.8%—12.8% for high-titer and 8.4% for low-titer inhibitors.
For patients who received prophylaxis, the median annual bleeding rate, during inhibitor-free periods, was 2.40 for all bleeds and 0 for spontaneous bleeds.
When simoctocog alfa was used on-demand, 92.4% of bleeds were controlled with 1 or 2 infusions. In addition, simoctocog alfa was said to demonstrate “excellent” or “good” efficacy in 89% of surgical procedures.
Three patients experienced adverse events (other than inhibitor development) that were considered related to simoctocog alfa.
One patient developed a mild fever. Another had a mild allergic reaction after 3 consecutive infusions of simoctocog alfa (but not after subsequent infusions).
The third patient developed a rash that was described as mild but considered serious due to hospitalization. This patient continued treatment and completed the study.