Surgical Experience from the STASEY Study of Emicizumab Prophylaxis in People with Hemophilia A with Factor VIII Inhibitors

Abstract

Background Guidelines surrounding emicizumab prophylaxis and perioperative treatment for people with hemophilia A (PwHA) with factor (F)VIII inhibitors undergoing surgeries are limited. The phase IIIb multicenter, single-arm STASEY study evaluated safety and tolerability of emicizumab prophylaxis in PwHA aged ≥12 years with FVIII inhibitors. This analysis assesses surgeries during study conduct, associated hemophilia medications, and postoperative bleeds (treated and untreated).

Methods PwHA with FVIII inhibitors received emicizumab 3.0 mg/kg/week for 4 weeks, then 1.5 mg/kg/week until 2 years. Surgeries were managed and documented by treating physicians. Bleeds and treatments were recorded by physicians and participants.

Results Forty-six participants had ≥1 on-study surgery, 37 underwent 56 minor surgeries, and 13 underwent 22 major surgeries. Four participants underwent both minor and major surgeries. Of 18 (81.8%) and 4 (18.2%) major surgeries managed with/without additional hemostatic medication, 33.3 and 25.0% were associated with a treated postoperative bleed, respectively. Of 24 (42.9%) and 32 (57.1%) minor surgeries managed with/without additional hemostatic medication, 15.6 and 25.0% were associated with a treated postoperative bleed, respectively. Recombinant activated FVII was the most common medication for prophylaxis and bleed treatment. There were no thrombotic microangiopathies (TMAs). One hypertrophic clot, considered unrelated to emicizumab, occurred following tooth extraction.

Conclusion In this challenging population with a high bleeding risk, major surgeries were performed in PwHA receiving emicizumab with/without additional hemostatic medication. Postoperative bleeds occurred following 59.1% of major surgeries; 53.8% were treated. No arterial/venous thrombotic events or TMAs occurred due to concomitant emicizumab and bypassing agents.

Trial registration This trial is registered at ClinicalTrials.gov (NCT03191799).

Keywords

clinical trial - emicizumab - hemophilia A - hemostasis - surgeries

Data Availability Statement

Qualified researchers may request access to individual patient-level clinical data through a data request platform. At the time of writing, this request platform is Vivli. https://vivli.org/ourmember/roche/. For up-to-date details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see https://go.roche.com/data_sharing. Anonymized records for individual patients across more than one data source external to Roche cannot, and should not, be linked due to a potential increase in risk of patient reidentification.

Authors' Contributions

Conception and design of the work: G.C., F.P., J.A.K.H., S.R., V.J-Y.

Study conduct, recruitment and follow-up of participants, and data collection: G.C., F.P., J.A.K.H., R.E.G.S., S.R., V.J-Y.

Data collection: G.C., F.P., J.A.K.H., R.E.G.S., V.J-Y.

Analysis and interpretation of data for the work: G.C., F.P., J.A.K.H., K.M., R.E.G.S., S.R., V.J-Y.

All authors revised the manuscript critically and provided final approval of the version to be published. All authors agreed to be accountable for all aspects of the work.

Publication History

Received: 16 May 2023

Accepted: 28 October 2023

Article published online:
12 January 2024

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