ESPN 53rd Annual Meeting

ESPN 2021


 
ASSESSMENT OF QUALITY OF LIFE THROUGH A PATIENT-CENTRED APPROACH IN CHILDREN AND ADULTS WITH DRTA TREATED WITH A NEW PROLONGED-RELEASE ALKALISING DRUG (ADV7103) FOR 5 YEARS
Aurélia Bertholet-Thomas 1 Michael Acquadro 2 Catherine Guittet 3 Sophie Joukoff 3 Maria A. Manso-Silván 3 Victor Navas 3 Alexia Marrel 2

1- HOSPICES CIVILS DE LYON
2- ICON plc
3- Advicenne SA
 
Introduction:

Patients with distal renal tubular acidosis (dRTA) have negative impacts on growth, bone and kidney, sometimes with hearing loss, which may significantly affect their quality of life (QoL). This study explores their lived experience about QoL linked to dRTA and its treatment.

Material and methods:

Individual 1-hour interviews (n=19) were performed to gather the impressions of patients with dRTA, (and/or their parents) 5 years after enrolment in a clinical study. ADV7103 (potassium citrate and potassium bicarbonate prolonged-release granules, twice daily) was compared with the standard of care (SoC) taken before study entry. Interviewers trained in qualitative research conducted the interviews using a semi-structured interview guide and performed thematic analysis with MAXDA software.

Results:

The main QoL domains impacted by dRTA and its treatment were difficulties studying, social challenges, and negative emotional and physical impacts. Difficulties associated to hearing impairment remained, but switching from previous SoC to ADV7103 was experienced as “life-changing” by patients/parents (Table 1): 

  • The emotional burden of disease perceived was relieved in the absence of treatment-related invasive questions from others,
  • Difficulties at school due to burdensome administrative issues and need to explain disease and treatment disappeared, facilitating parents who had stopped working to return to work,
  • Social/family issues improved: travel and holidays became easier to organise, patients/parents stopped thinking about managing treatment daily/nightly, reducing tension in the family/couple,
  • Bad taste and gastro-intestinal adverse events improved with ADV7103. Better compliance led to milder physical impacts and less fear to be hospitalised.

Mean satisfaction score with ADV7103 vs. SoC was 9 out of 10 and ADV7103 exceeded or met the expectations of 82% patients.

 

Table 1. Number of patients (%) concerned by each QoL domain after switching from SoC to ADV7103 (n=19)

Reported impacts with SoC

Reported impacts with ADV7103

Emotional functioning difficulties, 13 patients (68%)

Improved emotional functioning, 12 patients (63%)

Administrative complications at school and difficulties for parents to work, 13 paediatric patients (100%)

No more organisation problems for families at school and work, 13 paediatric patients (100%)

Social functioning and family problems, 6 patients (31%)

Improved family functioning, 18 patients (95%)

Health and product acceptability issues, 16 patients (84%)

Improved health and product acceptability, 18 patients (95%)

 

Conclusions:

 Qualitative interviews show that dRTA and its treatment have a significant impact on QoL of patients and parents and that ADV7103 helps improve daily-life and reduces treatment burden, resulting in greater overall patients’ and parents’ satisfaction.