PB0193 - Financial impact of switching to emicizumab from conventional haemostatic agents in acquired haemophilia A

Sunday, June 25, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Praveen Gounder, MBBS FRACP FRCPA

Staff Specialist Haematologist
Concord Repatriation General Hospital, Sydney
Baulkham Hills, New South Wales, Australia

Co-Author(s)

JC

Julianne Chong, n/a

VTE/Anticoagulation Stewardship and QUM Pharmacist
Concord Repatriation General Hospital
Macquarie Park, New South Wales, Australia
Caroline Dix, MBBS MSc FRACP FRCPA

Haematology Fellow
Department of Clinical Haematology, The Alfred Hospital, Melbourne, VIC, Australia
London, England, United Kingdom
ME

Marc L. Ellis, MBBS, PhD, FRACP, FRCPA

Haematologist
Concord Repatriation General Hospital
Concord, New South Wales, Australia
CD

Camille Durivage

Thrombosis Fellow
Concord Repatriation General Hospital
Concord, New South Wales, Australia
NW

Nicole Wong Doo

Staff Specialist Haematologist
Concord Repatriation General Hospital
Concord, New South Wales, Australia
UW

Uyen Walker

Blood Bank Senior Scientist
Concord Repatriation General Hospital
Concord, New South Wales, Australia
HT

Huyen Tran

Professor
Australian Centre for Blood Diseases, Monash University
Melbourne, Victoria, Australia
Vivien M. Chen, MBBS PhD FRACP FRCPAA (she/her/hers)

Group Leader, Staff Specialist Haematologist
ANZAC Research Institute, University of Sydney
Sydney, New South Wales, Australia

Background: Acquired haemophilia A (AHA) is a life-threatening bleeding disorder often requiring prolonged use of bypassing agents (BPAs). Conventional haemostatic therapy with the BPAs NovoSeven (rFVIIa) and Factor-Eight-Inhibitor-Bypassing-Activity (FEIBA), are available to Australian patients through the National Blood Authority but costs can escalate rapidly. Emicizumab has been used successfully in patients with AHA, though it is not registered by the Australian Therapeutic Goods Administration for this indication.

Aims: -

Methods: We present three cases of AHA treated with emicizumab, with rapid achievement of haemostasis and decreased consumption of BPAs.

Results: CASE 1: An 83-year-old man presented with haematuria, purpura and typical laboratory findings of AHA (Table 1). He commenced prednisolone, intravenous immunoglobulin and cyclophosphamide. rFVIIa and FEIBA were used sequentially, though the latter was abandoned as it potentiated DIC. Emicizumab was introduced on day 9, facilitating discontinuation of rFVIIa (Figure 1).
CASE 2: A 92-year-old man presented with a fall, chest wall haematoma, increased APTT and subacute DIC. He developed colonic pseudo-obstruction and extensive purpura. AHA was confirmed (Table 1), immune suppression commenced and rFVIIa utilised to control bleeding. FEIBA was abandoned as it potentiated DIC. Emicizumab was introduced on day 8, allowing rapid reduction and then cessation of rFVIIa (Figure 1).
CASE 3: A 51-year-old male with multiply relapsed AHA had four admissions over a 3-month period with severe bleeding requiring haemostatic treatment and no response to immunosuppressive therapy. He required 944mg, 526mg, 684mg and 803mg of rFVIIa for each of these episodes. He was subsequently treated with emicizumab. He had no bleeds during emicizumab treatment and no rFVIIa requirement. Three months after cessation of emicizumab, he presented with recurrent bleeding requiring further rFVIIa.

Conclusion(s): Emicizumab achieved haemostasis rapidly, did not potentiate DIC and appeared to significantly reduce the cumulative financial burden of rFVIIa and FEIBA. Full cost effectiveness studies are warranted.