PB1244 - Emicizumab Prophylaxis: a Single Center Clinical Experience.

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

AT

Ambra Taglietti

Medical Doctor
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy.
roma, Lazio, Italy

Co-Author(s)

fp

francesca paoletti

MD
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy.
roma, Lazio, Italy

Presenting Author(s)

EB

Erminia Baldacci

Hematologist, Medical Doctor
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy
roma, Lazio, Italy

Co-Author(s)

AF

Anna Laura Faccini

Medical Doctor
(1) Department of Translational and Precision Medicine, Sapienza University, Rome, Italy
Rome, Lazio, Italy
MM

Maria Chiara Molinari

Hematologist, Medical Doctor
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy.
ROMA, Lazio, Italy
RM

Rosaria Mormile

Biologist
(1) Hematology, Department of Translational and precision Medicine, Sapienza University, Rome, Italy
Rome, Lazio, Italy
AP

Agnese Pallotta

Biologist
(2) Hematology, AOU Policlinico Umberto I, Rome, Italy
Rome, Lazio, Italy
MP

Mauro Passucci

Hematologist, Medical Doctor
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy
Rome, Lazio, Italy
AC

Antonio Chistolini

Professor, Hematologist, Medical Doctor
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy
Rome, Lazio, Italy
CS

Cristina Santoro

Hematologist, Medical Doctor
(1) Hematology, Department of Translational and Precision Medicine, Sapienza University, Rome, Italy
roma, Lazio, Italy

Background: Emicizumab for severe hemophilia A (HA) has significantly changed current clinical practices.

Aims: We describe our experience in severe HA patients, with or without inhibitors.

Methods: In this retrospective, single-center study, patients’ data were collected from clinical charts. Participants signed an informed consent.

Results: We enrolled 22 patients receiving emicizumab prophylaxis (table 1). The only adverse event was rash at the injection site.
Non-inhibitor patients: 17. Before emicizumab, 14 were on recombinant factor VIII (rFVIII) prophylaxis, 2 were treated on-demand, 1 was treatment-naive. Median age at was 8.42 years (1.0-54.9) and median follow-up is 17.43 months (1.13-38.20). The reason to switch was facilitating therapy administration. During follow-up, 8 patients experienced traumatic bleeds, and 1 spontaneous hematuria. A patient underwent central venous catheter removal. In all instances, rFVIII was administered. 1 patient developed a de novo rFVIII inhibitor. We observed an 83,10% reduction in the annual bleeding rate (ABR), whereas the annual joint bleeding rate (AJBR) did not differ in the year before and after emicizumab.
Inhibitor patients: 5. Before emicizumab, 4 performed on-demand recombinant factor VII (rFVII) therapy, 1 switched upon inhibitor development. Median age was 58.1 years (27.3-67.7) and median follow-up is 49.67 months (20.20-66.40). The reason to switch was achieving better disease control. During follow-up, 3 patients experienced traumatic events; 2 required rFVII treatment. A colonoscopy and 4 dental extractions were performed without rFVII treatment. Both ABR and AJBR reduced by 100% in the year after emicizumab.

Conclusion(s): We confirmed the efficacy and safety of emicizumab prophylaxis. The differences in median age and follow-up are related to prescription regulations and previous clinical study participation. Bleedings were easily managed and no significant adverse events were observed. In the non-inhibitor population, the stable AJBR may be related to an increasingly confident approach to physical activity. An improvement in quality of life was recorded.