In patients with hemophilia A, primary prophylaxis and pharmacokinetic (PK)-guided prophylaxis appear to be associated with a reduced need for specialist consultations. Further, decreased bleeding, improved joint health outcomes, and a reduced need for joint surgeries have been linked to PK-guided prophylaxis in this population.

The findings are among the results of a recently published study in the Journal of Blood Medicine. The cross-sectional, retrospective, prevalence-based, burden-of-illness CHESS II study incorporated data compiled between November 2018 and October 2020 from 8 European countries. All data were stratified by treatment regimen.

The analysis comprised primary clinical and healthcare resource utilization outcomes associated with the care of patients with hemophilia A. Healthcare resource utilization endpoints included the numbers of hematologist or nurse consultations, bleed-related hospitalizations, and emergency department admissions.


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Clinical endpoints included the annualized bleeding rate (ABR), presence and number of target joints, and occurrence of joint surgeries. A target joint was defined as “a joint in which ≥3 spontaneous bleeds have occurred within a consecutive 6-month period.”

Learn more about the history of hemophilia

To improve the current standard of care for patients with hemophilia, the World Federation of Hemophilia guidelines recommend the use of a more personalized, tailored prophylactic treatment approach in patients who do not achieve satisfactory outcomes with standard prophylaxis.

Patients were eligible for study inclusion if they were male, at least 18 years of age, and diagnosed with hemophilia A of any severity. The researchers included 281 individuals with hemophilia A who were receiving prophylaxis and fulfilled the study criteria.

The mean participant age was 35.7±13.8 years. All of the patients had severe hemophilia A (factor VIII [FVIII] <1%). A specific FVIII trough level was targeted in 42.7% (120 of 281) of the participants; 39.2% (47 of 120) of these individuals received PK-guided dosing. Secondary prophylaxis was defined as “treatment that was previously on-demand but is currently prophylactic.”

Results showed that patients who received primary prophylaxis exhibited a significantly lower mean ABR than those who received secondary prophylaxis (2.9±2.6 vs 4.0±3.0, respectively; P =.001). Additionally, 27.5% of participants receiving primary prophylaxis had target joints, compared with 46.9% of those receiving secondary prophylaxis (P =.001).

Among participants who received PK-guided dosing, the mean ABR was 2.8±2.1 and the target joint number was 0.5±0.7, compared to 3.9±2.7 and 0.9±1.4, respectively, among individuals who received non-PK-guided therapy. The number of hematologist consultations was 7.1±5.3 in the PK-guided arm and 10.7±5.7 in the non-PK-guided arm. Further, a higher percentage of patients in the non-PK-guided group required hospitalization during their lifetime than that in the PK-guided group.

The authors concluded that “Further research is warranted to explore the specific FVIII trough levels targeted, as well as other potential drivers in the differences in clinical and [healthcare] utilization outcomes among patients who receive PK-guided prophylaxis.”

Reference 

Grazzi EF, Sun SX, Burke T, O’Hara J. The impact of pharmacokinetic-guided prophylaxis on clinical outcomes and healthcare resource utilization in hemophilia A patients: real-world evidence from the CHESS II study. J Blood Med. 2022;13:505-516. doi:10.2147/JBM.S363028