OBJECTIVES: To measure the cost of prophylactic treatment and bleeding management in a 12-year-old child weighing 42kg¹ with severe hemophilia with or without inhibitors from the perspective of the Brazilian healthcare system.

For non-inhibitor treatment, we compared emicizumab vs FVIII and for inhibitor treatment, emicizumab vs bypass agents.

METHODS: This study used local guidelines regimens^2,3, prices⁴ and annual bleeding rates (ABR): for bypass agents (21,1), FVIII (4,8), emicizumab (0,3) for inhibitor treatment and (1,3) non-inhibitor^5-7.

For the annual treatment, we considered the weekly-recommended dose of 7020 mcg/kg for recombinant activated factor VII (rFVIIa), 78mg/kg for emicizumab, 4151U/kg for FVIII, multiplied by 52 weeks. Except for rFVIIa, which can only be used for 6 months (26 weeks) and FVIII that is based on a cohort study⁸.

For the annual bleeding treatment, we considered the ABR and a mean consumption for each breakthrough bleeding was 270mgc/kg of rFVIIa to treat inhibitor patients on emicizumab or rFVIIa. For non-inhibitor patients, 46,7U/kg of FVII was considered when using FVIII and 43U/kg of FVIII when using emicizumab⁹.

The unitary costs were: R$3,59/mcg for rFVIIa⁴; R$174,05/mg for emicizumab⁴ and R$1,10 for FVIII⁴.

RESULTS: Costs for inhibitor treatment were:

* Inhibitor patient:

rFVIIa - R$1,139,896.80 (Prophylaxis - P), R$858,993,66 (Bleed treatment - BT), R$1,998,890,46 (Total - T)

Emicizumab - R$540,947,40 (P), R$12,213,18 (BT), R$553,160,58 (T)

* Non inhibitor patient

FVIII - R$238,848,54 (P), R$12,898,17 (BT), R$251,746,71 (T)

Emicizumab - R$540,947,40 (P), R$3,246,49 (BT), R$544,163,89 (T)

CONCLUSIONS: Despite the more expensive unitary cost, because of the lower quantity and greater efficacy in bleeding control, emicizumab costs less to treat when compared to rFVIIa and immune tolerance, when the high-dose protocol is used.

Emicizumab represents a saving of R$1,445,729,88 when compared to rFVIIa, which can pay up to 2 non inhibitor patients to switch froFVIII to emicizumab.