Real-World Experience of People with Hemophilia A Receiving Turoctocog Alfa Pegol (N8-GP): Results from a Patient Experience Survey

Figures & data

Table 1 Patient Demographic Details

Characteristics	Value
Number of Patients	n=46
Age, years [mean (range)]	29 (13–60)
Gender, n (%)
Male	61 (98)
Female	1 (2)
Time since diagnosis (mean years)	28
Severity, n (%)
Non-severe (1–40% FVIII activity)	37 (60)
Severe (<1% FVIII activity)	25 (40)
Mode of N8-GP treatment, n (%)
Prophylactic	47 (76)
On-demand	15 (24)
Employment status, n (%)
Working (Net)	28 (61)
Working full-time	17 (37)
Working part-time	7 (15)
Self-employed / contract worker	4 (9)
Student (Net)	12 (26)
Full-time student	9 (20)
Part-time student	3 (7)
Retired	4 (9)
Full-time caregiver	1 (2)
Unemployed	1 (2)
Receiving disability financial assistance	1 (2)
Region (n)
IT	16
ES	9
DE	10^a
PT	5
UK	2
US	4

Notes: Table is based on survey questions S2, S4, Q24, D1, D3, Q30. ^aOne patient aged 16 in DE completed the survey themself. Refer to Appendix 2, for questionnaires. Base: All patients (n=46). DE, Germany; ES, Spain; FVIII, factor VIII; IT, Italy; PT Portugal; UK, United Kingdom; US, United States.

Table 2 Caregiver Demographic Details

Characteristics	Value
Number of caregivers	n=16
Age, years (mean)	44
Gender, n (%)
Female	15 (94)
Male	1 (6)
Caregiver living with patient (%)	100
Relationship to patient, n (%)
Mother	15 (94)
Legal guardian	1 (6)
Employment status, n (%)
Working full-time	4 (25)
Working part-time	4 (25)
Unemployed	4 (25)
Self-employed	3 (19)
Volunteering	1 (6)
Full-time caregiver	1 (6)
Part-time student	1 (6)
Region (n)
IT	9
ES	2
DE	1
PT	0
UK	1
US	3

Notes: The questions for caregiver demographic details were S2, S7a, S6, C1, C2. Please refer to the Appendix 2, for the questionnaires. Base: All caregivers (n=16). DE, Germany; ES, Spain; IT, Italy; PT Portugal; UK, United Kingdom; US, United States.

Table 3 Mode of Treatment

Treatment	Participants
	Non-severe	Severe	Adult	Caregiver
Prophylaxis, n (%)	23 (62)	24 (96)	36 (78)	11 (69)
On-demand, n (%)	14 (38)	1 (4)	10 (22)	5 (31)

Notes: Table is based on survey question S8b. Please refer to the Appendix 2, for the questionnaires. Base: Non-severe: n=37; Severe: n=25; Adult: n=46; Caregiver (caring for adolescent patients): n=16.

Figure 1 Patient health status according to EQ-5D-5L. (A) EQ-5D-5L responses and overall health score. (Q1, Q2, Q3, Q4, Q5). (B) EQ-5D-5L responses and overall health score for non-severe and severe patients. (Q1, Q2, Q3, Q4, Q5). (C) Overall EQ VAS response. Whiskers represent minimum and maximum values (Range 61–80). (Q6). Base: All patients completing the survey themselves (n=46). EQ-5D-5L: EuroQol-5 Dimension. EQ VAS: EuroQol-visual analogue scales. Data represent percentage (%) of patients completing survey themselves. Please refer to the supplementary materials, for the questionnaires.

Figure 2 Patient treatment satisfaction. (A) Percentage of participants satisfied with N8-GP and previous treatments measured on a 7-point scale (Q36). Patients rating 5 to 7 were considered satisfied with the treatment and are represented as combined percentage of patients satisfied with N8-GP. Patients satisfied with N8-GP, n=59, 95%; patients satisfied with previous treatment: n=26, 42%. Base: All patients (n=62). (B) All reasons and main reasons for satisfaction with N8-GP (Q37). Base: All patients who are satisfied (n=59). (C) Main reasons for satisfaction for non-severe and severe patients (Q37). Non-severe: Less frequent injections (n=22, 61%) and improved QoL (n=29, 81%). Severe: Less frequent injections (n=20, 87%) and improved QoL (n=13, 57%). Base: Non-severe patients (n=37), all severe patients (n=25). Tx: Treatment. (D) Main reasons for satisfaction for patients on PPX and on-demand (Q37). Base: PPX (n=47), on-demand (n=15). PPX: Prophylaxis. ^†Indicate values are rounded.

Figure 3 Preference for N8-GP over previous treatments. (A) Preference for N8-GP over previous treatments (n=53, 91%). (Patients rating 4 or 5 on a 5-point scale; Q38a). (B) Main unprompted reasons for preferring N8-GP (Q38b). Net indicates combined scores for parameters falling within main reasons. Base: patients who preferred N8-GP to previous treatments (n=53) (Please refer to Appendix 1 for examples of data analysis). (C) Preference for N8-GP for patient subgroups: Non-severe (n=29, 88%) and severe (n=24, 96%); prophylaxis (n=41, 93%) and on-demand (n=12, 86%). (Please refer to supplementary materials for examples of data analysis). Tx: Treatment. Base: All patients with a previous treatment (n=58).

Table 4 Impact of Hemophilia on Day-to-Day Life

	Adult (n;%)	Caregivers (n;%)	p-value	Non-Severe (n;%)	Severe (n;%)	p-value	Prophylaxis (n;%)	On-demand (n;%)	p-value
Total, n	46	16	–	37	25	–	47	15	–
Overall QoL	16;36	12;71	p=0.017	23;62	5;20	p=0.001	20;43	8;53	p=0.471
Day-to-day routine/activities	11;24	12;71	p=0.001	20;54	3;12	p=0.001	18;38	5;33	p=0.732
Levels of stress and anxiety	11;24	9;53	p=0.037	15;41	5;20	p=0.097	15;32	5;33	p=0.919
Social life/relationships	8;18	9;53	p=0.007	14;38	3;12	p=0.030	13;28	4;27	p=0.940
Education/work	5;11	8;47	p=0.003	9;24	4;16	p=0.436	10;21	3;20	p=0.916

Notes: The table is based on the survey question Q22 on the impact of hemophilia on aspects of day-to-day life. Percentage of responses with p-values from different participant subgroups on various elements related to day-to-day life are presented. QoL: Quality of life.

Figure 4 Impact of N8-GP treatment on QoL and QoL score. (A) Agreement with statements for overall QoL (n=52, 84%), daily living (n=51, 82%) and joint protection and stable joint health (n=40, 65%), rated on a scale from 1 to 5 with 4 or 5 rating considered as agreement to the statements. Percentages are combined from 4 and 5 ratings. (Q39) (n=62). (B) QoL score currently and prior to N8-GP for all patients with 100 representing average person’s QoL score. (Q26 & Q35) (n=62).

Figure 5 Performance of N8-GP compared to previous treatment. (A) Percentage of responses from non-severe (n=37) and severe (n=25) groups for performance of N8-GP compared to previous product. (B) Percentage of responses from prophylaxis (n=47) and on-demand (n=15) patient groups for performance of N8-GP compared to previous treatment. ^†Indicate values are rounded.

Figure 6 Long-term impact of N8-GP on patients’ lives. (A) Percentage of responses for various aspects of living. Base: n=62 (Q41). (B) Percentage of responses for aspects of feelings for N8-GP compared to previous treatments. Base: n=62 (Q44).