Development and Validation Features of the Patient Benefit Index for the Treatment of Allergic Rhinoconjunctivitis with Allergen Immunotherapy

Figures & data

Table 1 Characteristics of the Participating Patients with ARC at t1

	Starting with Allergen Immunotherapy	Treated with Symptomatic Therapy	p-value
Age (yrs., mean ± SD)	37.1 ± 12.4	38.0 ± 15.2	0.643
Female (%)	55.9	62.1	0.245
BMI > 30 (%)	13.5	11.1	0.605
Pollen allergy: total (%)	96.6	93.8	0.262
Pollen allergy: trees (%)	65.1	62.5	0.69
Pollen allergy: grass and cereals (%)	76.6	59.4	< 0.01
Pollen allergy: herbs (%)	20.2	18.8	0.782
Allergy to feathers (%)	22.4	14.3	0.144
Allergy to dust mites (%)	35.4	41.3	0.366
Confirmed diagnosis of asthma (%)*	25.7	19.4	0.279
Time since ARC diagnosis (yrs., mean ± SD)	10.8 ± 11.2	12.1 ± 10.6	0.489
Months affected by ARC symptoms in the last year (number of months, mean ± SD, estimated by patient)	5.7 ± 3.2	4.9 ± 3.1	0.053
Current ARC severity (assessed by physician, scale from 0 = no ARC to 4 = very strong ARC)	2.0 ± 1.2	1.8 ± 1.0	0.096
Current ARC severity (assessed by patient, scale from 0 = not bothersome at all to 10 = extremely bothersome)	2.8 ± 3.0	3.3 ± 3.0	0.316
RQLQ global score (0 = not at all to 6 = extremely impaired by the disease in QoL)	1.6 ± 1.3	1.6 ± 1.5	0.95
EQ VAS (0 = worst imaginable health state to 100 = best imaginable health state)	77.0 ± 18.0	77.8 ± 17.2	0.751
Satisfaction with previous treatment (1 = very satisfied to 4 = not at all satisfied)	2.2 ± 0.9	1.9 ± 0.7	< 0.01
PBI (0 = no benefit to 4 = maximum benefit)	1.8 ± 1.1	2.4 ± 1.0	< 0.01

Note: Missing values vary per variable.

Abbreviations: BMI, body mass index; EQ VAS, European quality of life visual analogue scale; RQLQ, Rhinoconjunctivitis Quality of Life Questionnaire; SD, standard deviation; yrs., years.

Figure 1 Timeline of the study (t1 to t5).

Figure 2 Patient Benefit Index (PBI) score distribution (median and interquartile range) of patients with allergic rhinoconjunctivitis and either allergen immunotherapy (AIT) or symptomatic medication at t1 and t5 (AIT: n = 279 at t1, n = 333 at t5; symptomatic medication: n = 56 at t1, n = 43 at t5).

Table 2 Patient Needs Questionnaire Records per Item (t1). Only Patients with Allergen Immunotherapy, n = 422

Item No.	Treatment Goal	Mean^a	SD	Missing Values (n)	Missing Values (%)	Does Not Apply to Me (n)	Does Not Apply to Me (%)
1	… not have sneezing impulses anymore	3.3	0.9	10	2.4	27	6.4
2	… no longer have a runny or stuffed up nose	3.6	0.6	7	1.7	12	2.8
3	… be able to breathe through your nose more freely	3.7	0.7	9	2.1	18	4.3
4	… feel less fatigued or groggy	3.3	0.9	8	1.9	58	13.7
5	… be able to stay outdoors without symptoms	3.7	0.6	4	0.9	31	7.3
6	… feel less irritated	3.0	1.1	14	3.3	129	30.6
7	… have an easily applicable treatment	3.3	0.9	13	3.1	19	4.5
8	… not have itching on the eyes, nose or palate anymore	3.7	0.7	8	1.9	30	7.1
9	… not have burning or watery eyes anymore	3.5	0.8	7	1.7	61	14.5
10	… be healed of all symptoms	3.7	0.7	4	0.9	9	2.1
11	… be able to sleep better	3.3	1.0	9	2.1	94	22.3
12	… feel less depressed	3.0	1.2	8	1.9	151	35.8
13	… experience a greater enjoyment of life	3.2	1.1	11	2.6	144	34.1
14	… have no fear that the disease will become worse	3.2	1.1	8	1.9	96	22.7
15	… avoid the occurrence of asthma	3.6	0.9	7	1.7	67	15.9
16	… have less asthma symptoms	3.3	1.1	8	1.9	203	48.1
17	… be more productive in everyday life	3.3	1.0	8	1.9	81	19.2
18	… be able to engage in normal leisure activities	3.4	0.9	6	1.4	82	19.4
19	… be comfortable showing myself more in public	2.5	1.4	11	2.6	213	50.5
20	… be able to concentrate better at work	3.1	1.1	5	1.2	129	30.6
21	… be able to have more contact with other people	2.6	1.4	6	1.4	214	50.7
22	… be able to have a normal sex life	2.5	1.6	15	3.6	245	58.1
23	… be less dependent on doctor and clinic visits	3.1	1.2	9	2.1	84	19.9
24	… need less time for daily treatment	3.0	1.1	8	1.9	53	12.6
25	… have fewer out-of-pocket treatment expenses	3.1	1.2	8	1.9	70	16.6
26	… have fewer side effects	3.2	1.1	10	2.4	87	20.6
27	… have confidence in the therapy	3.5	0.9	7	1.7	40	9.5
28	… be able to engage in physical outdoor activities	3.6	0.7	6	1.4	65	15.4
29	… get permanently by without medication	3.6	0.7	7	1.7	16	3.8
30	… be permanently free of physical complaints	3.7	0.6	7	1.7	6	1.4
31	… be less burdened in my partnership	2.9	1.3	13	3.1	191	45.3
32	… not take health risks	3.5	0.9	7	1.7	38	9.0
33	… not have physical discomfort as a result of the treatment	3.4	1.0	7	1.7	66	15.6
Mean		3.2		8.3	1.9	88.7	21.0
Median		3.4		8.0	1.9	67.0	15.9
Standard deviation		0.6		2.6	0.6	69.3	16.4

Note: ^aScale from 0 = goal is not at all important to 4 = goal is very important.

Table 3 Patient Benefit Questionnaire Records per Item (t5). Only Patients with Allergen Immunotherapy, n = 349

Item No.	Treatment Goal	Mean^a	SD	Missing Values (n)	Missing Values (%)	Did Not Apply to Me (n)	Did Not Apply to Me (%)
1	… not have sneezing impulses anymore	2.4	1.2	12	3.4	20	5.7
2	… no longer have a runny or stuffed up nose	2.4	1.2	11	3.2	14	4.0
3	… be able to breathe through my nose more freely	2.4	1.2	15	4.3	28	8.0
4	… feel less fatigued or groggy	2.3	1.2	12	3.4	80	22.9
5	… be able to stay outdoors without symptoms	2.5	1.2	10	2.9	53	15.2
6	… feel less irritated	2.3	1.2	11	3.2	155	44.4
7	… have an easily applicable treatment	2.9	1.2	13	3.7	35	10.0
8	… not have itching on the eyes, nose or palate anymore	2.4	1.2	12	3.4	34	9.7
9	… not have burning or watery eyes anymore	2.4	1.2	13	3.7	60	17.2
10	… be healed of all symptoms	2.1	1.3	14	4.0	32	9.2
11	… be able to sleep better	2.3	1.2	11	3.2	99	28.4
12	… feel less depressed	2.3	1.2	9	2.6	165	47.3
13	… experience a greater enjoyment of life	2.5	1.3	11	3.2	171	49.0
14	… have no fear that the disease will become worse	2.6	1.3	13	3.7	100	28.7
15	… avoid the occurrence of asthma	2.7	1.2	18	5.2	115	33.0
16	… have less asthma symptoms	2.4	1.3	13	3.7	183	52.4
17	… be more productive in everyday life	2.5	1.3	13	3.7	111	31.8
18	… be able to engage in normal leisure activities	2.6	1.2	11	3.2	85	24.4
19	… be comfortable showing myself more in public	2.3	1.3	10	2.9	201	57.6
20	… be able to concentrate better at work	2.4	1.2	12	3.4	128	36.8
21	… be able to have more contact with other people	2.4	1.3	10	2.9	194	55.6
22	… be able to have a normal sex life	2.5	1.4	16	4.6	219	62.8
23	… be less dependent on doctor and clinic visits	2.2	1.4	11	3.2	94	26.9
24	… need less time for daily treatment	2.4	1.3	15	4.3	52	14.9
25	… have fewer out-of-pocket treatment expenses	2.2	1.4	13	3.7	80	22.9
26	… have fewer side effects	2.5	1.2	13	3.7	81	23.2
27	… have confidence in the therapy	3.0	1.0	15	4.3	43	12.3
28	… be able to engage in physical outdoor activities	2.7	1.2	13	3.7	65	18.6
29	… get permanently by without medication	2.4	1.3	15	4.3	35	10.1
30	… be permanently free of physical complaints	2.4	1.3	17	4.9	29	8.3
31	… be less burdened in my partnership	2.4	1.3	12	3.4	197	56.4
32	… not take health risks	2.6	1.2	13	3.7	95	27.2
33	… not have physical discomfort as a result of the treatment	2.5	1.3	14	4.0	118	33.8
Mean		2.4		12.8	3.7	96.1	27.5
Median		2.4		13.0	3.7	85.0	24.4
Standard deviation		0.9		2.1	0.6	60.3	17.3

Note: ^aScale from 0 = no benefit from the therapy to 4 = maximum benefit from the therapy in this respect.

Table 4 Correlation of the Patient Benefit Index Questionnaire for Allergen Immunotherapy with Other Outcome Variables. Only Patients with Allergen Immunotherapy at t5

	r	n	p
Current severity assessed by physician (0 = no ARC to 4 = very strong ARC)	−0.26	325	0.000
Current severity assessed by the patient (0 = not bothersome at all to 10 = extremely bothersome)	−0.37	329	0.000
RQLQ global score (0 = not at all to 6 = extremely impaired by the disease in QoL)	−0.35	331	0.000
EQ VAS: current health status (0 = worst imaginable health state to 100 = best imaginable health state)	0.27	327	0.000
Satisfaction with previous treatment (1 = very satisfied to 4 = not at all satisfied)	−0.57	314	0.000

Abbreviations: ARC, allergic rhinoconjunctivitis; EQ VAS, European quality of life visual analogue scale; RQLQ, Rhinoconjunctivitis Quality of Life Questionnaire; QoL, quality of life.