Improving current immunoglobulin therapy for patients with primary immunodeficiency: quality of life and views on treatment

Figures & data

Table 1 Respondent demographics

	All respondents (n=300)	Patients (n=216)	Caregivers answering on behalf of patients they care fora (n=84)
Patient age (years)
1–10	44 (15%)	0 (0%)	44 (52%)
11–20	50 (17%)	17 (8%)	33 (39%)
21–30	30 (10%)	27 (13%)	3 (4%)
31–50	90 (30%)	89 (41%)	1 (1%)
51–60	42 (14%)	40 (19%)	2 (2%)
61+	37 (12%)	37 (17%)	0 (0%)
Declined to answer	7 (2%)	6 (3%)	1 (1%)
Sex
Male/female	143 (48%)/157 (52%)	88 (41%)/128 (59%)	55 (65%)/29 (35%)
Diagnosis
CVID	152 (51%)	125 (58%)	27 (32%)
Hypogammaglobulinemia	38 (13%)	29 (13%)	9 (11%)
X-linked agammaglobulinemia	36 (12%)	22 (10%)	14 (17%)
IgG subclass deficiency	26 (9%)	14 (6%)	12 (14%)
Hyper IgM syndrome	9 (3%)	6 (3%)	3 (4%)
Other	34 (11%)	19 (9%)	15 (18%)
Unknown	5 (2%)	3 (1%)	2 (2%)

Note:

a Demographics shown for caregivers refer to the patient they care for rather than themselves.

Abbreviations: CVID, common variable immunodeficiency; Ig, immunoglobulin.

Table 2 Respondents experiencing immunoglobulin-related injection site reactions and headaches and the impact and importance of reducing these adverse events

	Intravenous respondents (n=160)			Subcutaneous respondents (n=134)
	Experiencing AE, n (%)	Large impact,n (%)	Rating reduction as important, n (%)	Experiencing AE, n (%)	Large impact, n (%)	Rating reduction as important, n (%)
Swelling/bumps at infusion site(s)	45 (28)	6 (4)	28 (18)	125 (95)a	9 (7)	42 (31)
Pain at infusion site(s)	70 (44)	8 (5)	37 (23)	100 (75)a	8 (6)	47 (35)
Itching at infusion site(s)	34 (21)	3 (2)	25 (16)	73 (54)a	13 (10)	31 (23)
Hardness at infusion site(s)	37 (23)	8 (5)	–	74 (55)a	10 (7)	–
Headaches	81 (51)a	18 (11)	55 (34)	41 (31)	10 (7)	33 (25)
Other AEs	72 (45)a	26 (16)	–	44 (33)	20 (15)	–

Note:

a P<0.05 for intravenous versus subcutaneous group.

Abbreviation: AE, adverse event.

Figure 1 Health of patients with primary immunodeficiency disease by country and route of immunoglobulin administration (visual analog scale).^a

Notes: ^aEQ-5D questionnaire; ^bEU5 indicates UK, France, Germany, Spain, and Italy.
Abbreviations: Ig, immunoglobulin; IV, intravenous; SC, subcutaneous; EQ-5D, EuroQoL 5 Dimensions.

Figure 2 12-Item Short Form Health Survey physical and mental norm-based^a summary and component scores for patients with primary immunodeficiency diseases treated with immunoglobulin.

Notes: ^aUsing norm-based scoring, each scale is scored to have the same mean (50) and standard deviation (10) as the general US population; ^boriginal scale is 0–100, with 100 being better health; ^cincludes several elements (ie, limitations in self care, physical, social, and role activities; physical well-being; and rating of health); ^dincludes several elements (ie, psychological distress, social and role disability due to emotional problems and general health rating).

Figure 3 Preference weights for treatment attributes (point estimates and 95% confidence intervals) decided by patients (n=216) and caregivers (n=84).