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Differential response to FEIBA is strongly associated with the prothrombotic microparticles
Puja Soni1, Chandrakala Shanmukhaiah2, Rucha Patil1
1National Institute of Immunohematology (NIIH-ICMR), 13th floor, KEM Hospital, Parel, Mumbai 400012, India.
Background:
Treatment of patients with hemophilia with an inhibitor is generally done using bypassing agents (BPA), wherein variability in response is observed. Due to lack of validated laboratory assays, monitoring is being carried out by clinical response only. Emerging biomarkers like procoagulant microparticles (MPs) may prove to be promising.
Aim:
To analyze whether procoagulant MP levels correlate with clinical response to FEIBA therapy.
Materials And Methods:
Total phosphatidylserine (PS) expressing MPs along with different cell derived MPs were measured in blood samples obtained prior and 2 hour post-FEIBA infusion in 64 bleeding episodes associated with 43 severe hemophilia patients.
Results And Discussion:
Patients with excellent response showed statistically significant increase in %MP of PS-MPs (p < 0.0001; 95.0% CI Range: -64.33 to -24.42) when compared to those with moderate response; platelet %MP change was also found significantly associated (p < 0.05) with clinical response. In search of an assay for monitoring FEIBA, results though preliminary seem to be promising with increase in %PS-MP correlating well with the clinical response. Coagulation being multifactorial process involves multiple factors for balanced hemostasis, which needs to be accounted. Larger studies in this line may provide indications for usage of MPs as monitoring and dose adjustment tool of FEIBA therapy.
Insights
Procoagulant microparticles (MPs) show promise as a biomarker for monitoring response to FEIBA therapy in hemophilia patients. Increased levels of phosphatidylserine (PS)-MPs correlate with better clinical outcomes, suggesting a potential role in dose adjustment.
Area of Science:
- Hematology
- Hemostasis and Thrombosis
- Biomarker Discovery
Background:
- Hemophilia treatment with bypassing agents (BPAs) like FEIBA shows variable patient response.
- Current monitoring relies solely on clinical assessment due to a lack of validated laboratory assays.
- Procoagulant microparticles (MPs) are emerging as potential biomarkers for treatment efficacy.
Purpose of the Study:
- To investigate the correlation between procoagulant microparticle (MP) levels and clinical response to FEIBA therapy in hemophilia patients.
- To explore the potential of MPs as a monitoring tool for FEIBA treatment.
Main Methods:
- Measurement of total phosphatidylserine (PS)-expressing MPs and cell-derived MPs in blood samples.
- Samples were collected before and 2 hours after FEIBA infusion in 64 bleeding episodes from 43 severe hemophilia patients.
- Analysis of the association between MP levels and clinical response to FEIBA.
Main Results:
- Patients with excellent clinical response to FEIBA showed a statistically significant increase in %PS-MP levels (p < 0.0001).
- Platelet %MP change was also significantly associated with clinical response (p < 0.05).
- Preliminary findings suggest a promising correlation between increased %PS-MP and positive clinical response.
Conclusions:
- Procoagulant MPs, particularly PS-MPs, may serve as a valuable biomarker for monitoring FEIBA therapy effectiveness in hemophilia.
- Further larger studies are warranted to establish MPs as a reliable tool for monitoring and dose adjustment of FEIBA.
- Understanding the multifactorial nature of coagulation is crucial for developing effective monitoring strategies.
