Identification of the Profile of the Patients with Hemophilia B Eligible for Treatment with Nonacog Alfa Once-Weekly
Abstract
:1. Introduction
2. Methods
- The percentages of their hemophilia B patients undergoing replacement therapy, “On-demand” treatment or continuous prophylaxis, depending on the severity of the disease;
- The percentage distribution of patients, by age, receiving the three treatment above options;
- The criteria guiding the choice to use continuous prophylactic treatment instead of “On-demand” therapy by age group;
- The criteria used to monitor patients on continuous prophylaxis;
- The percentage distribution of patients on once-weekly and twice-weekly prophylaxis and the doses of FIX used;
- The role of Nonacog alfa in early primary prophylaxis;
- The advantages of a once-weekly infusion scheme in patients on continuous prophylaxis;
- The possible obstacles to once-weekly infusion prophylaxis in patients with difficult venous access;
- The age group of patients they believe could benefit most from once-weekly prophylactic treatment,
- To define the profile of patients eligible for once-weekly prophylaxis.
3. Results
3.1. Use of Replacement Therapy, “On-demand” Treatment or Continuous Prophylaxis Based on Disease Severity
3.2. Use and Type of Replacement Therapy According to Age
3.3. The Criteria Guiding the Choice of Continuous Prophylaxis Rather Than “On-Demand” Treatment, by Age Group
3.4. The Criteria Used for Monitoring Patients on Continuous Prophylaxis
3.5. Percentage Distribution of Patients on Once and Twice-A-Week Prophylaxis and Dosage Used
3.6. The Role of Nonacog Alfa in Weekly Prophylaxis in HB
3.7. Possible Advantages of Once-Weekly Infusions in Patients on Continuous Prophylaxis.
3.8. Possible Obstacles to Once-Weekly Infusion Prophylaxis in Patients with Difficult Venous Access
3.9. The Age Group of Patients That Could Benefit Most from Once-Weekly Prophylactic Treatment
3.10. Defining the Profile of Patients Eligible for Once-Weekly Prophylaxis
4. Discussion
5. Conclusions
Author Contributions
Funding
Acknowledgments
Conflict of Interest
References
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Answers of Hemophilia Treatment Center (HTC) Directors | ||||||
---|---|---|---|---|---|---|
Patients on Therapy | HTC | Only On-Demand | Only Prophylaxis | Prophylaxis or On-Demand | ||
Severity Levels of Hemophilia | % | n | n | n | n | Patients’ Ratio Prophy/OD (Mean of %) |
Mild: FIX 5–40 IU/dL | 90–100 | 8/13 | 12 | 0 | 1 | 0.24 |
40–57 | 5/13 | |||||
Moderate: FIX 1–5 IU/dL | 80–100 | 12/13 | 7 | 0 | 6 | 0.70 |
60 | 1/13 | |||||
Severe: <1 IU/dL | 90–100 | 14/14 | 0 | 3 | 11 | 3.02 |
Answers of HTC Directors | ||||||
---|---|---|---|---|---|---|
Patients on Therapy | HTC | Only On-Demand | Only Prophylaxis | Prophylaxis or On-Demand | ||
Severity Levels of Hemophilia | % | n | n | n | n | Patients’ Ratio Prophy/OD (Mean of %) |
<6 years old | 100 | 7/9 | 0 | 2 | 7 | 4.19 |
10 | 2/9 | |||||
6–18 years old | 90–100 | 6/11 | 2 | 3 | 6 | 2.46 |
45–75 | 5/11 | |||||
18–30 years old | 90–100 | 7/11 | 2 | 1 | 9 | 1.21 |
20–85 | 5/11 | |||||
30–65 years old | 90–100 | 9/13 | 0 | 2 | 11 | 1.05 |
15–80 | 4/13 | |||||
>65 years old | 90–100 | 9/13 | 8 | 1 | 4 | 0.35 |
5–65 | 4/13 |
Prophylaxis | Answers of HTC | Patients Treated | Dose | |
---|---|---|---|---|
n | % | % | IU/kg | |
Once a week | 4/7 | 57 | 20–50 | 50–100 |
3/7 | 43 | 10–15 | 90–100 | |
Twice a week | 11/14 | 78 | 80–100 | 25–50 |
3/14 | 22 | 10–50 | 25–40 |
SCORES | ||||||
---|---|---|---|---|---|---|
1 | 2 | 3 | 4 | 5 | ||
Patients’ Group According to Age | Answers of HTCs | Reasons for Decision | ||||
<6 years old | 2 | 1 | 0 | 3 | 7 | Better parents’ compliance; Difficulty in finding venous access; All patients would be prophy-oriented once-weekly |
6–18 years old | 0 | 3 | 2 | 3 | 5 | The age group that often becomes less adherent to the prophylactic treatment; Lower acceptability of therapy in a particularly critical age group; Adolescents’ crisis: prophylaxis rejection; All patients would be oriented to once-a-week |
18–30 years old | 2 | 1 | 6 | 2 | 2 | All patients would be once-weekly prophy oriented. |
30–65 years old | 1 | 6 | 1 | 2 | 3 | All patients would be once-weekly prophy oriented |
>65 years old | 5 | 1 | 1 | 2 | 4 | Better compliance and because patients are less symptomatic and on which aggressive protection of the joints is not necessary; All patients would be once-weekly prophy-oriented. |
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Cultrera, D.; De Cristofaro, R.; Giordano, P.; Linari, S.; Macchi, S.; Marino, R.; Molinari, A.C.; Rocino, A.; Santoro, C.; Schinco, P.; et al. Identification of the Profile of the Patients with Hemophilia B Eligible for Treatment with Nonacog Alfa Once-Weekly. Reports 2020, 3, 3. https://doi.org/10.3390/reports3010003
Cultrera D, De Cristofaro R, Giordano P, Linari S, Macchi S, Marino R, Molinari AC, Rocino A, Santoro C, Schinco P, et al. Identification of the Profile of the Patients with Hemophilia B Eligible for Treatment with Nonacog Alfa Once-Weekly. Reports. 2020; 3(1):3. https://doi.org/10.3390/reports3010003
Chicago/Turabian StyleCultrera, Dorina, Raimondo De Cristofaro, Paola Giordano, Silvia Linari, Silvia Macchi, Renato Marino, Angelo Claudio Molinari, Angiola Rocino, Cristina Santoro, Piercarla Schinco, and et al. 2020. "Identification of the Profile of the Patients with Hemophilia B Eligible for Treatment with Nonacog Alfa Once-Weekly" Reports 3, no. 1: 3. https://doi.org/10.3390/reports3010003