Pharvaris N : Poster

Need for Caregiver Support for People Living With

Hereditary Angioedema in European Countries

Andrea Zanichelli1,2, Laurence Bouillet3, Teresa Caballero4, Markus Magerl5,6, Patrick F.K. Yong7, Kieran Wynne-Cattanach8, Joan Mendivil9

1Universita degli Studi di Milano, Dipartimento di Scienze Biomediche per la Salute, Milan, Italy; 2I.R.C.C.S., Policlinico San Donato, Centro Angioedema, UO Medicina, Milan, Italy; 3CHU Grenoble Alpes, Service de Medecine Interne, Centre de reference des Angioedemes, Grenoble, France; 4Hospital Universitario La Paz, Servicio de Alergología, Madrid, Spain; 5Institute of Allergology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; 6Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany; 7Frimley Health NHS Foundation Trust, Frimley, Surrey, UK; 8Adelphi Real World, Bollington, UK; 9Pharvaris GmbH, Zug, Switzerland

Rationale

• Hereditary angioedema (HAE) is a rare genetic condition characterized by painful, often debilitating swelling attacks that can affect multiple locations in the body.1,2
• The need for caregiver support among people with HAE has not been extensively studied in the literature and the requirements for such care are not well understood.
• This analysis aims to examine caregiver support requirements among people living with HAE in some European countries, as well as the impact of their condition on their health-related quality of life (HRQoL) and ability to work.

Methods

• Data were collected through the Adelphi HAE Disease Specific Programme (DSP )* conducted between January 2023-January 2024 in France, Germany, Italy, Spain, and the UK.

• DSPs are real-world,cross-sectional surveys with retrospective data collection.3 A geographically representative sample of physicians were recruited to participate in the DSP.
• Recruited HAE-treating physicians utilized medical charts (from their next ≤10 consecutive consulting patients) and their diagnostic and clinical judgment to provide data on patient demographics and need for caregivers.
• Patients and their caregivers were recruited via the patient's physician and were eligible for inclusion if the patient had a physician-confirmed diagnosis of HAE and both provided informed consent.
• Patients with HAE and caregivers voluntarily reported impact on their work activity (patients only) and HRQoL using self-reportforms:
• • Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI:SHP)4: higher scores indicate higher percent work impairment.
  • EQ-5D-VisualAnalogue Scale Questionnaire5: records general health and functionality calibrated from "worst health you can imagine" (0) to "best health you can imagine" (100).
  • EQ-5D-5LQuestionnaire6: measures severity of problems across mobility, self-care, usual activities, pain/discomfort, and anxiety/depression (0 bad health-1 good health, relative to the Italy Tariff). The Italy Tariff is a set of values representative of the general adult population in Italy.7
• Institutional review board approval was obtained. Descriptive statistics were reported.

Results

• In this analysis, 119 physicians reported data for 593 adult patients with HAE (53% female; mean ± SD age [years], 35.5 ± 14.0) (Table 1).
• Of the 334 (56%) patients currently receiving long-term prophylactic treatment (LTP), 187 (56%) were only using injectable LTP and 135 (40%) only oral LTP. Caregiver support use was reported for 108 patients (18%), 55 of which were receiving only injectable (68%) or oral (28%) LTP (Table 1).

Table 1. Physician-reported patient demographics and baseline characteristics

	Patients without caregiver	Patients with caregiver support
	support (n=485)	(n=108)
Age (years), mean ± SD [range]	36.4 ± 13.8 [18-82]	31.1 ± 14.1 [18-79]
Female, n (%)	255 (53)	58 (54)
Time (years) since diagnosis, mean ± SD [range]	8.1 ± 8.2 [0-47.5] (n=455)	7.9 ± 7.7 [0-49.4] (n=104)

Results (continued)

• The activity of daily living (ADL) for which patients most required support was managing medications (53%), followed by transportation (30%), and house cleaning/home maintenance (30%) (Figure 2).

Figure 2. Physician-reported ADL requirements for caregiver supporta

Managing medications										53%
Transportation					30%
House cleaning and					30%	Of these 39 patients, 85%
				were receiving injectable
home maintenance
Shopping and					21%		treatments as LTP or ODT.
meal preparation
Managing finances			16%
0%	10%	20%	30%	40%	50%	60%

ADL, activity of daily living; LTP, long-term prophylactic treatment; ODT, on-demand treatment. an=73 physician-reported patient ADL assessments.

• Patient-reporteddata (n=187) showed that overall work impairment was higher in patients with caregiver support (mean percent score: 20.0%) vs those with no additional caregiver support (10.1%) (Figure 3).

Figure 3. Patient-reported work productivity and activity impairment

size)	30%				Those without caregiver support			Those with caregiver support
					20.9%				23.5%
(sample	25%						20.0%	(n=46)
20%						(n=23)			(n=4a)
impairment	15%						9.8%	10.1%	12.1%
					(n=140)
	10%						(n=90)			(n=69)
Percent	5%		2.3%	0.0%
	(n=69)
0%		(n=4a)
			Absenteeism	Presenteeism	Overall work impairment	Overall activity impairment

HAE, hereditary angioedema. Absenteeism was defined as percent work time missed due to HAE. Presenteeism was defined as percent impairment while working due to HAE. aLow base number.

• EQ-5D-VASand EQ-5D-5L assessments yielded worse mean scores in patients with caregiver support (69.3 and 0.89, respectively) vs patients without caregiver support (82.1 and 0.95, respectively) (Figure 4).
• Caregivers (n=31) reported mean EQ-5D-VAS and EQ-5D-5L scores of 80.4 and 0.94, respectively (Figure 4).

Figure 4. Patient- and caregiver-reported HRQoL

HAE type,a n (%)	(n=473)	(n=108)
Type 1	359	(76)	92	(85)
Type 2	91 (19)	9	(8)
HAE with normal C1-INH	23	(5)	7	(6)
Current HAE treatment, n (%)
Prescribed LTP and ODT	188	(39)	36	(33)
Prescribed LTP only	89 (18)	21	(19)
Prescribed ODT only	189	(39)	47	(44)
Not currently prescribed any treatment	19	(4)	4	(4)
LTP by route of administration,b n (%)	(n=267)	(n=55)
Injectable LTP	148	(53)	39	(68)
Oral LTP	119	(43)	16	(28)
Number of HAE attacks in 12 months prior	2.1 ± 3.2 [0-30]	3.5 ± 4.4 [0-26]
to data collection, mean ± SD [range]

C1-INH,C1-inhibitor; HAE, hereditary angioedema; LTP, long-term prophylactic treatment; ODT, on-demand treatment; SD, standard deviation. aUnknown HAE type excluded from base. bPatients on multiple LTP or LTP plus other treatment excluded from base.

EQ-5D-VAS

100

90

80

70

60

50

40

30

20

10

0

EQ-5D-VAS
82.1	80.4
69.3

Those without	Those with	Caregiver
caregiver support	caregiver support	self-reported
(n=139)	(n=46)	(n=31)

j5L- 5D-EQ

1.0

0.9

0.8

0.7

0.6

0.5

0.4

0.3

0.2

0.1

0.0

EQ-5D-5L

0.95	0.89	0.94

Those without	Those with	Caregiver
caregiver support	caregiver support	self-reported
(n=140)	(n=46)	(n=31)

• For the 108 patients with caregiver support, assistance was most frequently provided by their partner/spouse or parent/guardian (Figure 1).
• The mean ± SD time that patients received caregiver support per week was 6.6 ± 9.7 hours (Figure 1).

Figure 1. Physician-reported caregiver utilization by relationship to patienta

															Mean ± SD hours of care
															provided per week
Partner/spouse											52%
												3.1 ± 2.6
Parent/guardian											45%			9.7 ± 13.7
Other relative			10%											3.0 ± 0
Adult child			2%												NR
Friend/neighbor			2%												NR
Other non-professional caregiver			2%												4.0 ± 0
Professional caregiver			1%												NR
0%	10%	20%	30%	40%	50%	60%

NR, not recorded; SD, standard deviation. aRespondents could select more than one category.

			Q3			Range		Mean
EQ-5D-5L, EuroQol 5-Dimension5-Level;EQ-5D-VAS, EuroQol 5-Dimension Visual Analogue Scale;			Median
HRQoL, health-related quality of life.			Q1

Conclusions

• In this study, patients with HAE who reported a greater need for caregiver support also experienced reduced health-related quality of life and reduced work productivity.
• Medication management was reported to be a major factor in the need for caregiver support for people living with HAE in some European countries.

References

1. Bernstein JA. Am J Manag Care. 2018;24(suppl 14):S292-8.2. Banerji A, et al. Ann Allergy Asthma Immunol. 2020;124:600-7.3. Anderson P, et al. Curr Med Res Opin. 2023;39:1707-15.4. Morrisroe K, et al. Clin Exp Rheumatol. 2017;35(suppl 106)4:130-7.5. Rabin R, et al. Ann Med. 2001;33:337-43.6. Devlin N, et al. Cham (CH): Springer; March 24, 2022. https://doi.org/10.1007/978-3-030-89289-0_1. 7. Meregaglia M, et al. Appl Health Econ Health Policy. 2023;21:289-303.

COI: Research grant support, consultancy fees, speaker fees, advisory board, investigator, and/or clinical trial fees - A.Z.: BioCryst, CSL Behring, KalVista, Pharming, Takeda; L.B.: BioCryst, Blueprint, CSL Behring, Novartis, Shire/Takeda; T.C.: Astria, BioCryst, CSL Behring, KalVista, Novartis, Pharming, Pharvaris, Takeda; researcher from the IdiPAZ program for

promoting research activities; M.M.: BioCryst, CSL Behring, Intellia, KalVista, Novartis, Octapharma, Pharming, Pharvaris, Shire/Takeda; P.F.K.Y.: Astria, BioCryst, CSL Behring, KalVista, Pharming, Pharvaris, Takeda; K.W-C.: employee of Adelphi Real World; J.M.: employee of Pharvaris, holds stock in Pharvaris.

Acknowledgments: Medical writing support was provided by Cara Bertozzi, PhD, of Two Labs Pharma Services.

*Funding statement: Funding towards this study was provided by Pharvaris; Adelphi Real World retains ownership of these data.