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4CPS-001 Emicizumab in acquired haemophilia type A: a case report
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  1. J Esquivel Negrín1,
  2. P Joy Carmona1,
  3. A Martín López1,
  4. MÁ Ocaña Gómez1,
  5. M Ríos De Paz2,
  6. MD De Dios García2,
  7. B Del Rosario García1,
  8. EG Fernández López1,
  9. T Betancor García1,
  10. J Merino Alonso1
  1. 1Hospital Universitario Nuestra Señora De Candelaria, Servicio De Farmacia, Santa Cruz De Tenerife, Spain
  2. 2Hospital Universitario Nuestra Señora De Candelaria, Servicio De Hematología, Santa Cruz De Tenerife, Spain

Abstract

Background and Importance Acquired haemophilia A is a coagulation disorder in which antibodies against factor VIII are produced, interfering with its activity and leading to potentially severe bleeding. Among numerous causes, cancer is a prevailing one. First-line haemostatic treatment until inhibitor eradication consists of bypass agents, including recombinant factor VII activated (rFVIIa) or activated prothrombin complex concentrates (aPCC).

Aim and Objectives We present the case of a 70-year-old male patient diagnosed with metastatic prostate cancer who went to the emergency department of a tertiary referral hospital due to an acute-onset extensive hematoma on the right thigh, with neither personal nor family history of haemophilia.

Material and Methods The patient was diagnosed with paraneoplasic acquired haemophilia. Therefore, immunosuppressive (methylprednisolone + cyclophosphamide) and haemostatic treatment (rFVIIa at 5 mg every 8h) was initiated.

9 days in, off-label use of emicizumab was requested, intended to guarantee a haemostatic level that would allow outpatient management. Emicizumab was administered subcutaneously at 3 mg/kg weekly over 4 weeks and then fortnightly over 16 weeks between January 13th and May 25th, 2022.

Haemostatic was monitored daily during hospitalisation and weekly after discharge through determination of inhibitor activity (Bethesda Units, UB) and FVIII activity (bovine based Chromogenic Factor VIII assay, UI) in blood samples.

Results The patient was successfully treated until the resolution of bleeding and normalised FVIII levels. Over the treatment with emicizumab as the only haemostatic agent (107 days), 8 subcutaneous injections were administered (cost: €51,255.2).

Having used rFVIIa (5 mg every 12 h) would have entailed 214 intravenous infusions, with a direct cost of €618,301.64. Thus, emicizumab treatment meant direct cost saving of €567,046.44.

Moreover, contributing factors to overheads as prolonged hospital stay, expenditure on consumables or staffing should be taken into account. Also risk of vascular access complications and quality of life must be considered.

Conclusion and Relevance Emicizumab has been a safe and cost-effective alternative to rFVIIa in haemorrhage prophylaxis, reducing direct costs by more than 10 times and allowed outpatient management.

Self-administration at home represents a major improvement in acquired haemophilia A quality of life.

Hospital pharmacy and haematology must collaborate to achieve a rational use of resources and an improvement in quality of life.

Conflict of Interest No conflict of interest

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