PB1307 - Management of severe Hemophilia A patients: Low-dose prophylaxis Vs on- demand treatment.

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Munira Borhany, MBBS, MCPS, FCPS, FRCP (Edin), FRCPath

Consultant Haematologist
Haemophilia Welfare Society, Karachi
Karachi, Sindh, Pakistan

Co-Author(s)

RA

Rabeea Ali

Clinical fellow
National Institute of Blood Diseases
Karachi, Sindh, Pakistan
Madiha Abid, MSPH

Clinical Research Associate
National Institute of Blood diseases and Bone Marrow Transplantation, Street 2/A, Block 17, Gulshan-e-Iqbal, KDA Scheme 24, Karachi, Sindh, Pakistan.
Karachi, Sindh, Pakistan
SZ

Sidra Zafar

Clinical Research Associate
National Institute of Blood Diseases and Bone Marrow Transplantation
Karachi, Sindh, Pakistan
RN

Rukhshanda Nadeem

Physiotherapist
Haemophilia Welfare Society, Karachi
Karachi, Sindh, Pakistan
RA

Raheel Ahmed

Data operator
Haemophilia Welfare Society, Karachi
Karachi, Sindh, Pakistan

Background: Prophylactic clotting factor infusion regimens to prevent bleeding and joint deformity has become the standard of care in severe hemophilia A patients.

Aims: We aim to assess low-dose prophylaxis as an alternative approach in our population.

Methods: A study that included 68 hemophilia A patients were divided into two groups i.e. prophylaxis and on-demand compared for annualized bleeding rate (ABR), hospitalization, units of factor VIII infused, or plasma products transfused (FFP and CP) and development of FVIII inhibitors.

Results: Out of the 68 patients recruited in this study, 25 were in the prophylaxis group and 43 in the on- demand group. Clinical profile of the patients on prophylaxis and on demand therapy is presented in Table 1. The on-demand group presented a higher median-range in ABR [8(33) vs 5(15), p-value 0.024] while no of hospitalization (39.7%, p-value 0.001) and inhibitors (9.3%, p-value 0.289) compared to the prophylaxis group. Whereas the prophylaxis approach demonstrated a significant negative correlation of ABR with FVIII prophylaxis (r=-0484, p=value=0.014) as presented in Figure 1. Moreover, no hospitalizations or inhibitor development was observed in the prophylaxis group. The estimated annual consumption of FVIII was 328 IU/kg/year in the on-demand group and 1662.6 IU/kg/year in the prophylaxis group. However, highly significant difference in plasma product utilization was observed between the two groups i.e., p-value < 0.001 and 0.038 for FFP and CP respectively.

Conclusion(s): Low-dose factor prophylaxis resulted in improved outcomes in terms of ABR, joint bleeding, hospitalization, and development of inhibitors. This treatment approach may be adopted as an economically feasible alternative to high dose prophylaxis.