Universität zu Köln

Gimpel, Charlotte ORCID: 0000-0003-1296-9081, Liebau, Max Christoph ORCID: 0000-0003-0494-9080 and Schaefer, Franz (2020). Systematic review on outcomes used in clinical research on autosomal recessive polycystic kidney disease-are patient-centered outcomes our blind spot? Pediatr. Nephrol., 36 (12). S. 3841 - 3852. NEW YORK: SPRINGER. ISSN 1432-198X

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Abstract

Background Autosomal recessive polycystic kidney disease (ARPKD) is a rare severe hepatorenal disease. Survivors of pulmonary hypoplasia and patients with milder presentations often achieve long-term survival but frequently require kidney and/or liver transplantation. Objective To examine the use of clinical, surrogate and patient-centered outcomes in studies on ARPKD with special attention to core outcomes of the Standardized Outcomes in NephroloGy project for children with chronic kidney disease (SONG-Kids). Data sources and study eligibility criteria A systematic MEDLINE literature search identified 367 ARPKD studies published since 1990; however, of these 134 were excluded because they did not report any clinical outcomes (e.g. only histopathological, genetic, protein structure or radiological markers), 19 studies because they only included prenatal patients and 138 because they were case reports with <= 3 patients. Study appraisal Seventy-six eligible studies were examined for study type, size, intervention, and reported outcomes by organ system and type, including all SONG-kids tier 1-3 outcomes. Participants There were 3231 patient-reports of children and adults with ARPKD. Results The overwhelming majority of studies reported clinical and surrogate outcomes (75/76 (98%) and 73/76 (96%)), but only 11/76 (14%) examined patient-centered outcomes and only 2/76 (3%) used validated instruments to capture them. Of the SONG-Kids core outcomes, kidney function was reported almost universally (70/76 (92%), infection and survival in three quarters (57/ 76 (75%), 55/76 (72%)) and measures of life participation (including neurological impairment) only rarely and inconsistently (16/76 (21%)). Limitations Thirty studies (39%) were of low quality as they were either narrative case reports (n = 14, 18%) and/or patients with ARPKD were an indistinguishable subgroup (n = 18, 24%). Only 28 trials compared interventions, but none were randomized. Conclusions and implications Studies that reported clinical outcomes in ARPKD usually covered the core outcome domains of kidney function, infections, and survival, but measures of life participation and patient-centered outcomes are distinctly lacking and require more attention in future trials.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Gimpel, Charlotte UNSPECIFIED orcid.org/0000-0003-1296-9081 UNSPECIFIED Liebau, Max Christoph UNSPECIFIED orcid.org/0000-0003-0494-9080 UNSPECIFIED Schaefer, Franz UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-584262
DOI:	10.1007/s00467-021-05192-8
Journal or Publication Title:	Pediatr. Nephrol.
Volume:	36
Number:	12
Page Range:	S. 3841 - 3852
Date:	2020
Publisher:	SPRINGER
Place of Publication:	NEW YORK
ISSN:	1432-198X
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language CONGENITAL HEPATIC-FIBROSIS; LIVER-TRANSPLANTATION; CHILDREN; ADOLESCENTS; OLIGOHYDRAMNIOS; DOMAINS; TRIALS Multiple languages Pediatrics; Urology & Nephrology Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/58426