Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study
Abstract Background Consequences of distal renal tubular acidosis (dRTA) on growth, bone and kidney, sometimes associated with hearing loss, may significantly affect quality of life (QoL). This descriptive qualitative study explores QoL linked to dRTA and gathers the impressions of patients with thi...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2022-03-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-022-02294-w |
| _version_ | 1811260480021331968 |
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| author | Michaël Acquadro Alexia Marrel Maria A. Manso-Silván Catherine Guittet Sophie Joukoff Aurélia Bertholet-Thomas |
| author_facet | Michaël Acquadro Alexia Marrel Maria A. Manso-Silván Catherine Guittet Sophie Joukoff Aurélia Bertholet-Thomas |
| author_sort | Michaël Acquadro |
| collection | DOAJ |
| description | Abstract Background Consequences of distal renal tubular acidosis (dRTA) on growth, bone and kidney, sometimes associated with hearing loss, may significantly affect quality of life (QoL). This descriptive qualitative study explores QoL linked to dRTA and gathers the impressions of patients with this rare disease (and caregivers) 5 years after enrolment in a clinical study, during which patients were treated with ADV7103, a prolonged-release granule formulation combining potassium citrate and potassium bicarbonate. Semi-structured, one-hour interviews with 6 adult and 13 paediatric patients with a confirmed diagnosis of dRTA and with parents of paediatric patients were performed using an interview guide. Qualitative analysis of anonymized interview transcripts based on grounded theory was conducted. Results The main QoL domains impacted by dRTA and its treatment were education/work, social/family life, and emotional and physical well-being. ADV7103 (administered twice daily) was compared with the standard of care (SoC) taken before study entry (more than twice daily). Patients/parents reported that switching from previous SoC to ADV7103 had changed their lives: Difficulties at school due to burdensome administrative issues and need to explain disease and treatment affecting all families of paediatric patients (n = 13) disappeared, facilitating parents who had stopped working (to deal with their child’s treatment) to return to work, Family functioning was improved (n = 18), as travel and holidays became easier to organise and patients/parents stopped thinking about managing treatment daily/nightly, reducing tension in the family or couple, The emotional burden of disease perceived was relieved (n = 12) in the absence of treatment-related invasive questions from others, Gastro-intestinal adverse events and taste problems improved with ADV7103 (n = 18) and better compliance led to milder physical impacts and less need to be hospitalised. The mean satisfaction score with ADV7103 compared to SoC was 9 out of 10 (10 = very satisfied). ADV7103 exceeded or met the expectations of 14 out of 17 patients that commented on that. 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 satisfaction of the patients and their families. Trial registration EU Clinical Trials Register, EudraCT 2013-003828-36 on the 3rd of September 2013. |
| first_indexed | 2024-04-12T18:47:59Z |
| format | Article |
| id | doaj.art-414cef9ae7f847e5ab5de297029975e9 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-04-12T18:47:59Z |
| publishDate | 2022-03-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-414cef9ae7f847e5ab5de297029975e92022-12-22T03:20:33ZengBMCOrphanet Journal of Rare Diseases1750-11722022-03-011711810.1186/s13023-022-02294-wLived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative studyMichaël Acquadro0Alexia Marrel1Maria A. Manso-Silván2Catherine Guittet3Sophie Joukoff4Aurélia Bertholet-Thomas5ICON PlcICON PlcAdvicenne S.A.Advicenne S.A.Advicenne S.A.Centre de Référence des Maladies Rénales Rares–Néphrogones–Hôpital Femme Mère Enfant, Hospices Civils de Lyon–Filière ORKiDAbstract Background Consequences of distal renal tubular acidosis (dRTA) on growth, bone and kidney, sometimes associated with hearing loss, may significantly affect quality of life (QoL). This descriptive qualitative study explores QoL linked to dRTA and gathers the impressions of patients with this rare disease (and caregivers) 5 years after enrolment in a clinical study, during which patients were treated with ADV7103, a prolonged-release granule formulation combining potassium citrate and potassium bicarbonate. Semi-structured, one-hour interviews with 6 adult and 13 paediatric patients with a confirmed diagnosis of dRTA and with parents of paediatric patients were performed using an interview guide. Qualitative analysis of anonymized interview transcripts based on grounded theory was conducted. Results The main QoL domains impacted by dRTA and its treatment were education/work, social/family life, and emotional and physical well-being. ADV7103 (administered twice daily) was compared with the standard of care (SoC) taken before study entry (more than twice daily). Patients/parents reported that switching from previous SoC to ADV7103 had changed their lives: Difficulties at school due to burdensome administrative issues and need to explain disease and treatment affecting all families of paediatric patients (n = 13) disappeared, facilitating parents who had stopped working (to deal with their child’s treatment) to return to work, Family functioning was improved (n = 18), as travel and holidays became easier to organise and patients/parents stopped thinking about managing treatment daily/nightly, reducing tension in the family or couple, The emotional burden of disease perceived was relieved (n = 12) in the absence of treatment-related invasive questions from others, Gastro-intestinal adverse events and taste problems improved with ADV7103 (n = 18) and better compliance led to milder physical impacts and less need to be hospitalised. The mean satisfaction score with ADV7103 compared to SoC was 9 out of 10 (10 = very satisfied). ADV7103 exceeded or met the expectations of 14 out of 17 patients that commented on that. 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 satisfaction of the patients and their families. Trial registration EU Clinical Trials Register, EudraCT 2013-003828-36 on the 3rd of September 2013.https://doi.org/10.1186/s13023-022-02294-wdRTAPotassium citrate and potassium bicarbonateQualitative researchPatient-reported outcomes |
| spellingShingle | Michaël Acquadro Alexia Marrel Maria A. Manso-Silván Catherine Guittet Sophie Joukoff Aurélia Bertholet-Thomas Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study Orphanet Journal of Rare Diseases dRTA Potassium citrate and potassium bicarbonate Qualitative research Patient-reported outcomes |
| title | Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study |
| title_full | Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study |
| title_fullStr | Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study |
| title_full_unstemmed | Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study |
| title_short | Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study |
| title_sort | lived experiences of patients with distal renal tubular acidosis treated with adv7103 and of their caregivers a qualitative study |
| topic | dRTA Potassium citrate and potassium bicarbonate Qualitative research Patient-reported outcomes |
| url | https://doi.org/10.1186/s13023-022-02294-w |
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