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Blood Res (2024) 59:30

Published online October 8, 2024

https://doi.org/10.1007/s44313-024-00034-6

© The Korean Society of Hematology

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization

Valentina Perrone1, Melania Leogrande1, Maria Cappuccilli1 and Luca Degli Esposti1*

1 CliCon S.r.l. Società Benefit, Health, Economics & Outcomes Research, Bologna, Italy

Correspondence to : Luca Degli Esposti
luca.degliesposti@clicon.it

Received: April 15, 2024; Accepted: September 23, 2024

© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.

Abstract

Purpose This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.
Methods From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021–2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021–2022 having at least 12-month follow-up were assessed.
Results Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.
Conclusion These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.

Keywords Healthcare resource utilization, Hemophilia A, Monoclonal antibodies, Plasma-derived FVIII, Treatment patterns

Article

RESEARCH

Blood Res 2024; 59():

Published online October 8, 2024 https://doi.org/10.1007/s44313-024-00034-6

Copyright © The Korean Society of Hematology.

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization

Valentina Perrone1, Melania Leogrande1, Maria Cappuccilli1 and Luca Degli Esposti1*

1 CliCon S.r.l. Società Benefit, Health, Economics & Outcomes Research, Bologna, Italy

Correspondence to:Luca Degli Esposti
luca.degliesposti@clicon.it

Received: April 15, 2024; Accepted: September 23, 2024

© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.

Abstract

Purpose This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.
Methods From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021–2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021–2022 having at least 12-month follow-up were assessed.
Results Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.
Conclusion These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.

Keywords: Healthcare resource utilization, Hemophilia A, Monoclonal antibodies, Plasma-derived FVIII, Treatment patterns

Fig 1.

Figure 1.Scheme for patients’ selection

Fig 2.

Figure 2.Distribution of prophylaxis treatments according to the different types of FVIII

Table 1 . Demographic and clinical characteristics of HA patients stratified by administration purpose (on-demand and prophylaxis).

Patients on-demand (N = 202)Patients on prophylaxis (N = 165)
Demographic and clinical characteristics
Age at index-date (years), mean (± SD)36.7 (± 19.5)35.7 (± 16.8)
Age groups
0–5 years, N (%)8 (4.0%)NR
6–11 years, N (%)12 (5.9%)10 (6.1%)
12–17 years, N (%)25 (12.4%)16 (9.7%)
18–39 years, N (%)61 (30.2%)70 (42.4%)
40–59 years, N (%)65 (32.2%)50 (30.3%)
60–69 years, N (%)25 (5.0%)15 (3.0%)
≥ 70 years, N (%)6 (3.0%)NR
Charlson index, mean (± SD)0.4 (± 1.0)0.3 (± 0.5)
Charlson index = 0, N (%)141 (69.8%)122 (73.9%)
Charlson index = 1, N (%)48 (23.8%)36 (21.8%)
Charlson index ≥ 2, N (%)13 (6.4%)7 (4.2%)
Comorbidities
Hypertension, N (%)41 (20.3%)27 (16.4%)
Dyslipidemia, N (%)18 (8.9%)9 (5.5%)
HCV, N (%)12 (5.9%)10 (6.1%)
HIV, N (%)8 (4.0%)4 (2.4%)
HBV, N (%)0 (0.0%)0 (0.0%)
Arthropathy, N (%)NRNR
Cardiovascular disease, N (%)0 (0.0%)NR
Acute coronary syndrome, N (%)0 (0.0%)0 (0.0%)
Atrial fibrillation, N (%)0 (0.0%)0 (0.0%)
Arterial or venous embolism and thrombosis, N (%)0 (0.0%)0 (0.0%)
Osteoporosis, N (%)0 (0.0%)0 (0.0%)

Abbreviations: HBV Hepatitis B virus, HCV Hepatitis C virus, HIV Human immunodeficiency virus, IU International units, NR not reported for data privacy (< 3 patients), SD Standard deviation.


Table 2 . Annual consumption expressed in IU of FVIII among patients treated with EHL FVIII, overall (N = 145) and on prophylaxis (N = 105).

EHL FVIIIOverall patients (N = 145)On prophylaxis, N (%) (N = 105)Annual consumption in IU of FVIII (mean; median)
Efmoroctocog alfa5138 (74.5%)290,579; 260,000
Damoctocog alfa pegol5040 (80.0%)328,813; 303,500
Turoctocog alfa pegol2517 (68.0%)440,647; 392,000
Rurioctocog alfa pegol1910 (52.6%)366,800; 350,000

Abbreviations: EHL Extended half-life, FVIII Factor VIII, IU International units.


Table 3 . Focus analysis on monthly consumption of EHL FVIII products on HA adults only (N = 88) on prophylaxis.

Adults HA patients only (N = 88) on prophylaxis
Efmoroctocog alfaRurioctocog alfa pegolDamoctocog alfa pegolTuroctocog alfa pegol
N (%)27 (30.7%)9 (10.2%)36 (40.9%)16 (18.2%)
Expecteda
Expected range of IU/month10,645.821,291.718,25026,614.6
Observed
IU/month25,941.430,851.927,10337,265.6
N (%) patients > 25% expected range27 (100.0%)9 (100.0%)36 (100.0%)16 (100.0%)
N (%) patients > 50% expected range27 (100.0%)9 (100.0%)36 (100.0%)16 (100.0%)
N (%) patients > 75% expected range27 (100.0%)9 (100.0%)36 (100.0%)16 (100.0%)
N (%) patients > 100% expected range27 (100.0%)9 (100.0%)36 (100.0%)16 (100.0%)
N (%) patients > 125% expected range23 (85.2%)5 (55.6%)27 (75.0%)8 (50.0%)
N (%) patients > 150% expected range20 (74.1%)4 (44.4%)15 (41.7%)5 (31.3%)
N (%) patients > 175% expected range19 (70.4%)NI8 (22.2%)NI
N (%) patients > 200% expected range16 (59.3%)0 (0.0%)NINI
IU/month (excluded patients with high IU consumption, i.e. > 200% expected range)15,212.130,851.926,118.936,000

Abbreviations: EHL Extended half-life, FVIII Factor VIII, IU International units, NI Not issuable (for data privacy: subgroups of ≤ 3 patients).

a “Expected” refers to the minimum value required to consider a patient in prophylaxis.


Table 4 . Healthcare consumptions (expressed as number of provisions/services per patient) among HA patients treated during 2021–2022 and with at least 12 months of follow-up (n = 462), stratified by category of FVIII.

Number per patientPatients on EHL FVIII (n = 145)Patients on SHL rFVIII (n = 222)Patients on SHL pdFVIII (n = 37)Patients on emicizumab (n = 58)
Prescriptions for HA, mean (± SD)9.2 (± 5.4)7.6 (± 6.9)6.0 (± 5.5)9.9 (± 4.3)
Other drug prescriptions, mean (± SD)5.6 (± 10.0)5.9 (± 10.5)6.5 (± 6.7)3.1 (± 5.5)
Outpatient specialist services, mean (± SD)3.1 (± 4.7)2.3 (± 3.5)3.5 (± 4.5)2.5 (± 3.6)
Hospitalizations, mean (± SD)0.1 (± 0.5)0.1 (± 0.4)0.2 (± 0.5)0.1 (± 0.3)

EHL FVIII includes efmoroctocog alfa, rurioctocog alfa pegol, turoctocog alfa pegol, damoctocog alfa pegol; SHL rFVIII includes octocog alfa, lonoctogoc alfa, turoctocog alfa, simoctocg alfa, moroctocog alfa; SHL pdFVIII include human coagulation FVIII/Von Willebrand factor.

Abbreviations: EHL Extended half-life, HA Hemophilia A, SD Standard deviation, rSHL FVIII Standard half-life recombinant FVIII.


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