1 - Serviço de Imunohemoterapia, Centro Hospitalar Lisboa Norte
2 - Unidade de Hematologia Pediátrica, Hospital Dona Estefânia, Centro Hospitalar Lisboa Central
3 - Serviço de Imunohemoterapia, Hospital Espírito Santo, Évora
- Publicação em forma de Abstract. ISTH 2023 Congress. Montreal
Background: In Portugal, emicizumab is not reimbursed for patients without inhibitors (PwHAni).
Aims: We present the experience of 2 portuguese centers, where children without inhibitors started prophylaxis with emicizumab after a special request.
Methods: Pediatric PwHAni on emicizumab ≥ 6 months were retrospectively evaluated for annualized and joint bleeding rates (ABR/AJBR) calculated from 12 months before to at least 6 months after initiation of emicizumab.
Results: Four children were on prophylaxis with emicizumab, with a median age of 1.7 (range: 1-3) years. Median age at initiation of emicizumab was 1,2 (range: 0.8–2,15) years. Three patients were younger than 2 years at initiation of emicizumab, and 1 was a previously untreated patient (PUP). In the 3 children previously treated with factor VIII the median exposure days (ED) was 16. None of the children maintained concomitant FVIII administration. Median follow-up time on emicizumab was 12,8 (range: 6.2 – 19.8) months. ABR with emicizumab was 0,0 vs 5,5 (0-10) pre-switch. In the case of AJBR, while before emicizumab was 8 (0-6) after starting prophylaxis with emicizumab no joint bleeds were observed. Three patients (75%) never needed treatment since starting this therapy and only one intestinal bleeding, related to a gastroenteritis, was treated. In one patient, with 3 episodes of head trauma, treatment was decided for bleeding prevention, but no intracranial hemorrhage was confirmed. Also, before emicizumab, 3 of the children were frequently observed in emergency (ER) due to bleeding (n=22) and hospitalized (n=8). After initiation of emicizumab, only in 3 occasions evaluation in the ER was needed, and no hospitalization was required. No adverse events were observed.
Conclusion(s): Prophylaxis with emicizumab led to significantly less bleeding events and no spontaneous bleeding. We hope these results will help sensibilize decision makers about the benefits of this subcutaneous therapy, namely in children where venous access is often a problem.
Palavras Chave: Emicizumab, Haemophilia A, PwHAni