PO0035 - Emicizumab prophylaxis: experience of a single center.

Wednesday, July 13, 2022

1:00 PM – 2:00 PM

Location: ExCel Center, ICC Capital Suite Room 1

Presenting Author(s)

GM

Giacomo Maestrini

Hematologist
Hematology, Sapienza University of Rome
Rome, Lazio, Italy

Co-Author(s)

EB

Erminia Baldacci

Hematologist
Hematology, University Hospital Policlinico Umberto I, Rome
Rome, Lazio, Italy
AF

Antonietta Ferretti

Hematologist
Hematology, Sapienza University of Rome
Rome, Lazio, Italy
MC

Maria Gabriela Chavez Orellana

MD
Hematology, Sapienza University of Rome
Rome, Lazio, Italy
MM

Maria Rosaria Mormile

Biologist
Hematology, University Hospital Policlinico Umberto I, Rome
Rome, Lazio, Italy
FB

Francesco Barone

Biologist
Hematology, Sapienza University of Rome
Rome, Lazio, Italy
SO

Sandra Olivieri

Technician
Hematology, University Hospital Policlinico Umberto I, Rome, Italy
Rome, Lazio, Italy
AP

Agnese Pallotta

Technician
Hematology, University Hospital Policlinico Umberto I, Rome, Italy
Rome, Lazio, Italy
AS

Alessandra Serrao, MD

MD
Hematology, Sapienza University, Rome, Italy
Rome, Lazio, Italy
FV

Federica Viola

Hematologist
Hematology, Sapienza University of Rome
Rome, Lazio, Italy
AC

Antonio Chistolini

Director
Dipartimento di Medicina Traslazionale e di Precisione Sapienza Università di Roma
Roma, Lazio, Italy
CS

Cristina Santoro

Medical Doctor
Ematologia, Azienda Ospedaliera Universitaria Policlinico Umberto I, Roma
Rome, Lazio, Italy

Background: The use of emicizumab as prophylaxis for severe HA with or without inhibitor, is increasing in routine clinical practice.

Aims: To describe our center experience with emicizumab prophylaxis in severe hemophilia A (HA) patients without and with inhibitors.

Methods: All the participants signed an informed consent; data were collected from clinical charts.

Results: 10 severe HA patients received emicizumab prophylaxis (table 1). We observed 1 drug-related adverse event, a relapsing orticarioid reaction at the injection site.
Non-inhibitor patients: 6, 4 on rFVIII prophylaxis, 1 on demand therapy, 1 never treated. Reason for starting emicizumab: need to facilitate the therapy administration in young children. Just one traumatic tongue lesion occurred which was treated with one dose of rFVIII. None surgical or invasive procedures were performed.
Inhibitor patients: 4. Before switching to emicizumab: 3 on bypassing agents on demand; one patient on rFVIII prophylaxis developed a high titer inhibitor. Two patients received ITI. All patients switched to emicizumab for a better control of the disease. No spontaneous bleeding were reported. A traumatic fracture of the homerus occurred, treated with 4 doses of rFVIIa (90mcg/kg/dose) without any complication. One patient underwent a colonoscopy without additional prophylaxis (no biopsies performed). Two patients underwent dental extraction: just one received prophylaxis with tranexamic acid; no hemorrhagic complications were observed.

Conclusion(s): We confirm the efficacy and safety of emicizumab prophylaxis. No spontaneous bleedings occurred. Two traumatic bleedings were easily managed with rFVIIa or FVIII concentrate. Dental extractions in inhibitor patients were performed without rFVIIa therapy. No serious adverse events observed. The different median age and follow-up between inhibitor and non-inhibitor patients depends on timelines of regulatory indications for prescription and previous participation in clinical studies. We observed an improvement in quality of life of patients and their families.