Prophylaxis in Patients with Hemophilia a and Its Impact on Quality of Life Using the Specific Index Haem-a-Qol at a Hospital in Paraguay
Authors: Frutos Cristobal A , Konkle Barbara , Batista Jorge , Brizuela Silvia , Enciso Elvira , Fernandez Gustavo , Gaona Adolfo , Martinez Helga , Poisson Alma M , Rivarola Marlene 
Published: October 2019

Prophylactic replacement therapy with FVIII is considered the standard of care for patients with hemophilia although a protocol for administering the prophylaxis has yet to be defined by the World Federation of Hemophilia. Gaona in 2012 demonstrated a significant reduction in the number of hospitalizations due to bleeding episodes in patients with Hemophilia A treated with secondary prophylaxis at the Hematology Service of the Hospital Central del Instituto de Prevision Social (HCIPS) with a regimen of 25 IU/Kg per week in two infusions by reporting results from such regimen that had started at the HCIPS in 2010. This is a lower dose to those most frequently used in high resource countries like the Malmo or Utrecht protocols.

The goal of this study was to assess the impact of our lower dose prophylaxis regimen on bleeding events, hospitalizations and quality of life as an alternative for low resource countries that cannot afford to provide their patients with higher dose treatments.

The Dutch group Haemo-QoL designed the specific questionnaire for adult patients with hemophilia, the Haem-A-QoL with a Spanish version available for patients aged 17 and up.

Results: The HCIPS currently has 36 patients receiving prophylactic treatment with FVIII. From these 18 patients consented to filling out the questionnaire. The mean age of the patients was 28. 3 of the patients were married, 2 were in a stable relationship and 13 were single. 62% of patients lived within 20 km of the Hospital and 38% lived further away going up to 200 km. 17 of the 18 patients had the highest level of education possible for their age. Only one patient, the oldest one, had stopped his education at primary school. All of them had severe disease with one or more affected joints.

The average FVIII use per year was 68,000 IU ranging from 8,000 IU to 96,000 IU or 917 IU/kg/year. The strongest determinant for receiving less FVIII was missing appointments. Distance to the Hospital was not of significance. Bleeding episodes prior to prophylactic treatment was 2-3 minor traumatic bleeding episodes per week (after brushing their teeth, easy bruising from minor trauma), 2-3 joint bleeds per month at which point they would seek medical assistance and 1-2 major bleeding episodes per year requiring hospitalization. In the last year 5 patients were hospitalized once. One for pneumonia, one for phlebitis, one for dengue fever and two for hemathrosis. They reported a decrease in bleeding episodes at home from 2-3 joint bleeds per month to 1 every other month. Patients reported after traumatic events their bleeding was "normal" (i.e. when brushing their teeth if they started bleeding they would continue bleeding for days at a time whereas when in prophylaxis they would bleed right after the brushing and then it stopped which they took to be normal).

Converting the results from the Haem-A-QoL questionnaire to a scoring system of 1-100 being 1 the best QoL and 100 the worst, the average QoL was 52. With scores of 60 for physical health, 50 for feelings, 52 for view of themselves, 48 for work/school, 53 for treatment, 56 for future, 47 for family planning and 45 for dating. Scores for sports and leisure though only amounting to 64, 33% of patients said that category did not apply to them since they did not practice any sports and the dealing category with a score of 37 was the lowest of all. When compared to results published by a Blood Center in Brazil with an on demand regimen patients with severe hemophilia in that study showed a physical health score of 55, 40 for feelings, 35 for self-perception, 60 for sports and leisure, 35 for work and school, 20 for coping, 45 for treatment, 45 for future and 25 for dating with an average overall score of 40. With an annual average usage of 63,683 IU on the severely affected patients.

In summary, the proposed dosing of 25IU/kgs/week seems at first glance to reduce bleeding episodes among patients with severe hemophilia as well as hospitalizations. In general patients treated at the HCIPS have a regular to poor quality of life as measured by the Haem-A-QoL questionnaire which contrasts with results from the Brazilian study on quality of life from patients with on demand treatment and roughly same FVIII usage per year showing their patients with severe hemophilia had an overall quality of life from regular to good. Perhaps the psychological factor comes in to play? A follow up study after patients receive proper psychological evaluations may help clarify results.

Disclosures

No relevant conflicts of interest to declare.