PB1241 - Successfully retinoblastoma surgery in a Severe Hemophilia A with an incomplete impregnation dose of Emicizumab, Instituto Nacional de Pediatria, Mexico city, Mexico

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Mara Nunez Toscano, Medical Doctor,

Pediatric Hematologist,
Instituto Nacional de Pediatria
Del. Coyoacan, Distrito Federal, Mexico

Co-Author(s)

NL

Norma Candelaria Lopez Santiago

Head Department
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO, Distrito Federal, Mexico
JC

JENIFER COLOP

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
MF

MARILYN FLORES

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
IC

IRLANDA CAMPOS

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
AG

ALMA GURROLA

Attending Physcian
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
DG

DULCE GRIJALVA

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
YE

YAMILE ESQUIVEL

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
JR

JOB ROMERO

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico
MH

MONTSERRAT HERNANEZ

fellow
INSTITUTO NACIONAL DE PEDIATRIA
MEXICO CITY, Distrito Federal, Mexico

Background: Emicizumab is a mimetic FVIII, Initially approved for patients with hemophilia and inhibitors, it is recommended to use a weekly dose for 4 weeks as an impregnation dose to continue weekly, biweekly, or monthly maintenance doses.
We present the case of a patient who, after only two doses of emicizumab, underwent ocular extraction for retinoblastoma, with follow-up with ROTEM, which did not present hemorrhagic data only with administration of a single dose of FVIII.

Aims: To report the experience with incomplete doses of emicizumab in the control of bleeding in the face of a hemostatic challenge

Methods: CASE
11-month-old male, Severe Hemophilia A (SHA), Factor VIII 0.1%, with previous untreated patient (PUP), no history of hemarthrosis events, diagnosis of stage E left unilateral retinoblastoma requiring enucleation, decided to initiate primary prophylaxis-based on Emicizumab was initiated at a dose of 3mg/kg/weekly dose subcutaneously.
At 24 hours after the surgical event, a dose of Factor VIII at 30mg/kg/dose was administered, at 48 hours a rotational thromboelastogram was performed, which was observed within normal parameters, so the administration of Factor VIII was suspended. Figure 1.

Results: The bleeding during the surgery reported was 5 ml, the patient continued with the impregnation doses and now in a cancer remission

Conclusion(s): Risk factors for developing inhibitors include high doses of factor VIII concentrated, in pro-inflammatory states such as surgery, type of mutation, race, siblings, or family history of inhibitors.
The management in a PUP patient with a non-replacement therapy is not clear until now, however after two doses of emicizumab we were able to verify through viscoelastic tests the effect on hemostasis in this patient, making us a reflection that if it’s impregnation doses are really necessary in the patient with hemophilia, this opens the opportunity to create new therapeutic strategies in the patient with SHA