P3802 - Patient Voice on Adherence and Satisfaction Following Switch in Therapy to Trientine Tetrahydrochloride for Wilson Disease: The ASTRA Study

Tuesday, October 24, 2023

10:30 AM – 4:00 PM PT

Location: Exhibit Hall

Presenting Author(s)

Valentina Medici (she/her/hers)

University of California Davis
Sacramento, CA

Valentina Medici, ¹, Massimo Zuin, ², Nora Cazzagon, ³, Alberto Civolani, ⁴, Fabio Tedone, ⁵, Stefania Lopatriello, ⁵, Pier Maria Battezzati, ⁶
¹University of California Davis, Sacramento, CA; ²University of Milan, Milan, Lombardia, Italy; ³University of Padua; UOC Gastroenterology, University Hospital of Padua, Padova, Veneto, Italy; ⁴AOU Cagliari, UOC Gastroenterologia, Cagliari, Sardegna, Italy; ⁵Helaglobe srl, Firenze, Toscana, Italy; ⁶ASST Santi Paolo e Carlo, Polo universitario ospedale San Paolo, Università degli Studi di Milano, Milan, Lombardia, Italy

Introduction: Wilson disease (WD) is an inherited disorder of copper transport resulting in copper accumulation predominantly affecting the liver and brain. Treatment of WD is life-long with the aim of controlling circulating free copper. All therapies must be taken separate from food and multiple times a day. Side effects are common, all potentially negatively impacting adherence which may lead to poorer outcomes. No study has been performed to evaluate WD patient adherence and satisfaction following a change in therapy.

Methods: An Italian multicenter, prospective observational study of WD adults prescribed trientine tetrahydrochloride (TETA4; Orphalan SA, France). Demographic data, 24-hour urinary copper excretion (UCE), posology and pill count at each visit were collected. Patient reported outcomes were obtained on adherence (Morisky Medication Adherence Scale; MMAS-8), satisfaction (Patient Experiences and Satisfaction with Medication questionnaire; PESaM) and quality of life (QoL) questionnaire (SF36). Data were collected at two-time points; baseline (T0) and first visit (minimum 3 months) after switching to TETA4 (T1).

Results: Of the 25 patients enrolled, 14 were female. Median (IQR) age and time from diagnosis was 43 (30,52) and 24 (14,30) years, respectively. The majority were employed (18/25) and 8/25 had co-morbidities. Pre-switch therapies were, zinc (13), penicillamine (6), trientine dihydrochloride (TETA2; 4) and combination TETA2/zinc (2). Median (range) UCE for zinc treated patients prior to switch 94 (19, 422) mcg/day with 6/13 suggesting non-adherence (UCE >100mcg/day). Of the 12 patients receiving chelation (alone or in combination), median (range) UCE was 118 (26, 757) compared with at first follow up of 222 (21, 610) mcg/day. Mean (range) of pills of TETA4 prescribed was 4 (2-6). Based on returned pills at first follow up, 16/23 were adherent, 6/23 moderately- and 1/23 non-adherent. Change in MMAS-8 scores (16/16; 100% response) are shown in figure. PESaM and SF36 questionnaires are summarized in table.

Discussion: Patient reported adherence improved with TETA4 compared with previous therapy. Patient satisfaction with TETA4 improved through perceived ease of use and fewer reported adverse events and improvements in the QoL domains of vitality, social functioning and general health compared with prior therapy. Long term follow-up to assess continued adherence and QoL are needed.

Figure: MMAS-8 Scores Represented As Very, Moderately and Poorly Adherent Before and After Switch to TETA4

Variable	Respondents T0 (n/N)	Mean	SD	Median	IQR	Respondents T1 (n/N)	Mean	SD	Median	IQR
PESaM
Effectiveness (1 to 5)	25/25	3.97	0.95	4	3.5, 5	24/25	3.95	0.9	3.88	3.4, 4.8
Satisfaction Effectiveness (-5 to +5)	25/25	2.28	3.15	4	3.1, 5	24/25	3.29	2.3	4	3, 5
Adverse Events Burden (1 to 5)	13/25	3.2	1.3	3.25	2, 4.5	4/25	3.06	1.6	3.25	2.5, 3.8
Satisfaction of Safety (-5 to +5)	13/25	2.28	3.15	4	1,4	4/25	2	2.3	2	2.5, 3.8
*Ease of Use (1 to 5)	25/25	4.04	0.94	4.3	3.7, 4.7	24/25	4.6	0.6	4	4.3, 5
Overall Satisfaction (-5 to +5)	25/25	2.04	3.62	4	-1, 5	24/25	3.6	2.2	4	3.5, 5
SF-36
*Physical Limitations	25/25	86.6	25.2	100	90, 100	25/25	91.4	17.9	100	91.3, 100
Role- Physical Limitations	25/25	88	29.9	100	100, 100	25/25	83.3	17.9	100	75, 100
Body Pain	25/25	81.2	21.1	100	67.5, 100	25/25	83	33.5	100	71.3, 100
General Health	25/25	53.9	23.1	56.2	31.3, 75	25/25	56.1	20.5	62.5	39, 75
*Role- emotional limitations	25/25	76	40.3	56.2	33.3, 100	24/25	86.1	29.3	100	100, 100
Vitality	25/25	52.3	22.8	46.6	33.3, 73.3	25/25	55.3	16.4	56.7	46.7, 66.7
Wellbeing	25/25	63.7	20.1	68	54, 78	25/25	64.7	15.5	68	48, 76
Social Functioning	25/25	73.5	20.1	75	54, 78	25/25	77.6	23.0	87.5	62.5, 100

Disclosures:

Valentina Medici: Arbormed – Advisory Committee/Board Member. Arbormed – Grant/Research Support. Oprhalan – Advisory Committee/Board Member. Orphalan – site PI. Ultragenix – site PI. Vivet – Site PI.

Massimo Zuin: Alexion – Advisory Committee/Board Member. Orphalan – Advisory Committee/Board Member. Orphalan – Speaker.

Nora Cazzagon: Adavanz Pharma – Consultant. Intercept – Consultant. Ipsen – Consultant. Orphalan – Consultant.

Alberto Civolani indicated no relevant financial relationships.

Fabio Tedone indicated no relevant financial relationships.

Stefania Lopatriello indicated no relevant financial relationships.

Pier Maria Battezzati: AbbVie – Consultant. CYMABAY – Consultant. Gilead Sciences, Inc. – Consultant. Intercept Pharmaceuticals – Consultant. Orphalan – Consultant. Roche – Consultant. Salix Pharmaceuticals – Consultant. Tobira Therapeutics – Consultant. Ultragenyx Pharmaceutical Inc. – Consultant.

Valentina Medici, ¹, Massimo Zuin, ², Nora Cazzagon, ³, Alberto Civolani, ⁴, Fabio Tedone, ⁵, Stefania Lopatriello, ⁵, Pier Maria Battezzati, ⁶. P3802 - Patient Voice on Adherence and Satisfaction Following Switch in Therapy to Trientine Tetrahydrochloride for Wilson Disease: The ASTRA Study, ACG 2023 Annual Scientific Meeting Abstracts. Vancouver, BC, Canada: American College of Gastroenterology.