PB1230 - Single centre review of practice for treatment of Acquired Haemophilia A patients

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Georgina Santiapillai, MBBS

Haematology Specialist Trainee
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK
London, England, United Kingdom

Co-Author(s)

RH

Rosemary Haldon

Haemophilia Clinical Nurse Specialist
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK
London, England, United Kingdom
AR

Anne Riddell

Laboratory Head of Department (Special Coagulation, Haemophilia) and HSL Scientific Lead Coagulation
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK
London, England, United Kingdom
PC

Pratima Chowdary, MD

Consultant Haematologist
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, United Kingdom
London, England, United Kingdom
AD

Anja Drebes

Consultant Haematologist
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK
London, England, United Kingdom
KG

Keith Gomez

Consultant Haematologist
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK
London, England, United Kingdom
JK

Janette Kemp

Haemophilia Clinical Nurse Specialist
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK
London, England, United Kingdom
TY

Thynn Thynn Yee

Associate Specialist
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, UK
London, England, United Kingdom
Paul Batty, MBBS, PhD (he/him/his)

Associate Professor
University College London
London, United Kingdom

Background: Acquired haemophilia A (AHA) caused by auto-antibody formation to factor VIII (FVIII) is a life-threatening disorder. Management includes treatment of acute bleeding and immunosuppressive therapy (IST) for inhibitor eradication, with practices varying in different centres. Infections secondary to IST are an important cause of mortality.

Aims: To review treatment practices and outcomes in patients with acquired haemophilia A.

Methods: A single centre retrospective review of management of acquired haemophilia A, between May 2019 and July 2022, was undertaken. FVIII activity (FVIII:C) was measured using a one-stage assay and inhibitor titres using the Nijmegen-Bethesda assay.

Results: A total of 20 patients (male=15, female=5) were diagnosed with AHA during the observation period. Median age at presentation was 78.5 years (range: 43-97), with 60% (12/20) being idiopathic, and the second most common cause was malignancy (n=5). At presentation, median FVIII:C and inhibitor titres were 1IU/dL and 7.8 BU/mL respectively (Table 1). Bleeding was present at diagnosis in 95% (19/20) and 84% (16/19) required haemostatic treatment. Most (74%) bleeding episodes were treated with FEIBA (factor eight inhibitor bypassing activity). Within the cohort, 30% (n=6) patients were treated solely as an outpatient. For those requiring hospital admission (n=14), the median length of stay was 27 days (range: 5-58). All except for one patient, who went into spontaneous remission, received immunosuppression. Rituximab based regimens, were used in 58% (11/19) using a dose of 375mg/m2/week for 4-weeks. Median time to FVIII:C >50IU/dL and a negative inhibitor titre ( < 0.6BU/mL), were 125 days and 137 days. Complete remission was reported in 50% (10/20) patients and one patient relapsed. Mortality was observed in 8/20 (40%) patients, with three due to infection.

Conclusion(s): Immunosuppressive therapies were selected based on age and underlying co-morbidities. Lower remission rates and infection on immunosuppression suggest the need for trials of immunosuppressive combinations and novel agents.