A Retrospective Analysis of the SIPPET Study

In the original SIPPET study, researchers found strong evidence that recombinant factor VIII (FVIII) use results in an 87% higher rate of inhibitor development than plasma-derived FVIII/VWF in previously untreated patients with hemophilia A. To assess whether genetic profiling would identify patients who can reduce the risk of inhibitors with recombinant FVIII, investigators conducted a retrospective SIPPET analysis.1,2

PATIENT POPULATION

Genetic risk profiles, combined with treatment type, can play roles in inhibitor development across product classes. In order to determine if genetic risk profiling can help identify the risk of inhibitor frequency, participants in this SIPPET retrospective analysis were further categorized by genetic FVIII mutation.2

Hemophilia A Patients with FVIII mutation Retrospective SIPPET Analysis Chart

RETROSPECTIVE SIPPET ANALYSIS DEMONSTRATES THAT GENETIC PROFILING DOES NOT REDUCE INHIBITOR RISK WITH RECOMBINANT FVIII TREATMENT2


Among high-risk patients, cumulative incidence of inhibitors was 47% with recombinant FVIII. With the low-risk patients the cumulative incidence of inhibitors with recombinant FVIII was 43%. It was determined there was no difference between the two groups treated with recombinant FVIII, regardless of their genetic profile.

Inhibitor Incidence in High Risk Patients Treated with rFVIII Chart 
Inhibitor Incidence in Low Risk Patients Treated with rFVIII Chart
 

IMPLICATIONS OF THE SIPPET RETROSPECTIVE ANALYSIS2

Results of the analysis lend no support for risk profiling of previously untreated patients and prescribing recombinant FVIII to those with a low prior inhibitor risk.

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REFERENCES

  1. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054-2064.
  2. Rosendaal FR, Palla R, Garagiola I, Mannucci PM, Peyvandi F. Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis. Blood. 2017;130(15):1757-1759.