Peer-reviewed article
Health-related quality of life, mental health and caregiver burden in children with autosomal recessive polycystic kidney disease.
Charlotte Gimpel, Susanne Schaefer, Franz Schaefer ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This study examined health-related quality of life, mental health, and caregiver burden in 43 children with autosomal recessive polycystic kidney disease (ARPKD), a rare inherited kidney condition. Children with ARPKD showed significantly impaired quality of life and psychosocial problems compared to healthy peers, with effects comparable to or worse than children with advanced kidney failure. Treatment type (dialysis, transplant, or early-stage disease) and developmental delay were the most important factors affecting outcomes. Caregivers also experienced substantial burden and reduced quality of life.
Key points
- All four measures of quality of life and psychosocial functioning showed significant differences by treatment modality, with worst scores in dialysis patients
- Developmental delay of the child was the most significant extra-renal risk factor affecting all four quality of life and mental health measures
- Children with ARPKD had elevated mental health problems (SDQ scores) compared to both contemporary and historical healthy controls
- Children with ARPKD showed particular difficulties with peer relationship problems
- ARPKD caused equal or greater caregiver burden and impaired quality of life compared to caregivers of children with more advanced kidney failure
Limitations
The study does not specify which countries were included, limiting understanding of generalizability. The cross-sectional design with historical controls cannot establish causation or track changes over time.
Abstract
BACKGROUND: Pediatric chronic kidney disease (CKD) causes significantly impaired health-related quality of life (hrQOL) and caregiver burden, but no studies focus specifically on autosomal recessive polycystic kidney disease (ARPKD). METHODS: This prospective case-control study assessed hrQOL (using PedsQL®ESRD) and screened for psychosocial problems (strength and difficulties questionnaire (SDQ)) in 43 children with ARPKD. Fifty-eight caregivers reported on the disease's impact on family (FaBel) and their own QOL (Ulm inventory of parental caregiver QOL (ULQIE)). As controls, we questioned 36 matched healthy children and 57 parents under similar pandemic restrictions and used published historical controls (healthy and with advanced CKD). RESULTS: Patients were aged 9.0 ± 4.8 years with CKD stage G1-4 (45%), on dialysis (14%) or after kidney transplantation (26%). Nine patients had developmental delay secondary to medical complications. PedsQL®ESRD total scores correlated significantly to kidney function, but could not capture liver-specific symptoms. All 4 measures showed significant differences between treatment modalities with best scores in patients during CKD stages G1-4 and worst on dialysis, except SDQ, which was worst after transplantation. The most significant extra-renal risk factor for all 4 scores was developmental delay of the child. SDQ scores were elevated in contemporary vs. historical controls, but even further in ARPKD especially for peer relationship problems. CONCLUSION: In summary, ARPKD causes significantly impaired hrQOL, psychosocial problems and caregiver burden, which were equal to, if not greater than, that of controls with more advanced kidney failure. Treatment modality and developmental delay were the most important risk factors. TRIAL REGISTRATION: Trial registered 06/2020 DRKS S00021059.