Emulation of the control cohort of a randomized controlled trial in pediatric kidney transplantation with Real-World Data from the CERTAIN Registry.

Autor: Patry C; Department of Pediatrics I, University Children's Hospital Heidelberg, Heidelberg, Germany. Christian.Patry@med.uni-heidelberg.de., Sauer LD; Institute of Medical Biometry, University of Heidelberg, Heidelberg, Germany., Sander A; Institute of Medical Biometry, University of Heidelberg, Heidelberg, Germany., Krupka K; Department of Pediatrics I, University Children's Hospital Heidelberg, Heidelberg, Germany., Fichtner A; Department of Pediatrics I, University Children's Hospital Heidelberg, Heidelberg, Germany., Brezinski J; Institute of Medical Biometry, University of Heidelberg, Heidelberg, Germany., Geissbühler Y; Novartis Pharma AG, Basel, Switzerland., Aubrun E; Novartis Pharma AG, Basel, Switzerland., Grinienko A; Novartis Pharmaceuticals, East Hanover, NJ, USA., Strologo LD; Renal Transplant Unit, Bambino Gesù Children's Hospital, Pediatric subspecialities, Rome, Italy., Haffner D; Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Hannover, Germany., Oh J; Pediatric Nephrology, University Hospital Hamburg, Hamburg, Germany., Grenda R; Department of Nephrology, Kidney Transplantation and Hypertension, Children's Memorial Health Institute, Warsaw, Poland., Pape L; Clinic for Paediatrics III, Essen University Hospital, Essen, Germany., Topaloğlu R; Department of Pediatric Nephrology, School of Medicine, Hacettepe University, Ankara, Turkey., Weber LT; Pediatric Nephrology, Children's and Adolescents' Hospital, University Hospital Cologne, Medical Faculty University of Cologne, Cologne, Germany., Bouts A; Department of Pediatric Nephrology, Amsterdam University Medical Center, Emma Children's Hospital, Amsterdam, The Netherlands., Kim JJ; Department of Paediatric Nephrology, Nottingham University Hospital, Nottingham, UK., Prytula A; Pediatric Nephrology and Rheumatology Department, Ghent University Hospital, Ghent, Belgium., König J; Department of General Pediatrics, University Children's Hospital, Munster, Germany., Shenoy M; Paediatric Nephrology, Royal Manchester Children's Hospital, Manchester, UK., Höcker B; Department of Pediatrics I, University Children's Hospital Heidelberg, Heidelberg, Germany., Tönshoff B; Department of Pediatrics I, University Children's Hospital Heidelberg, Heidelberg, Germany.
Jazyk: angličtina
Zdroj: Pediatric nephrology (Berlin, Germany) [Pediatr Nephrol] 2023 May; Vol. 38 (5), pp. 1621-1632. Date of Electronic Publication: 2022 Oct 20.
DOI: 10.1007/s00467-022-05777-x
Abstrakt: Background: Randomized controlled trials in pediatric kidney transplantation are hampered by low incidence and prevalence of kidney failure in children. Real-World Data from patient registries could facilitate the conduct of clinical trials by substituting a control cohort. However, the emulation of a control cohort by registry data in pediatric kidney transplantation has not been investigated so far.
Methods: In this multicenter comparative analysis, we emulated the control cohort (n = 54) of an RCT in pediatric kidney transplant patients (CRADLE trial; ClinicalTrials.gov NCT01544491) with data derived from the Cooperative European Paediatric Renal Transplant Initiative (CERTAIN) registry, using the same inclusion and exclusion criteria (CERTAIN cohort, n = 554).
Results: Most baseline patient and transplant characteristics were well comparable between both cohorts. At year 1 posttransplant, a composite efficacy failure end point comprising biopsy-proven acute rejection, graft loss or death (5.8% ± 3.3% vs. 7.5% ± 1.1%, P = 0.33), and kidney function (72.5 ± 24.9 vs. 77.3 ± 24.2 mL/min/1.73 m 2 P = 0.19) did not differ significantly between CRADLE and CERTAIN. Furthermore, the incidence and severity of BPAR (5.6% vs. 7.8%), the degree of proteinuria (20.2 ± 13.9 vs. 30.6 ± 58.4 g/mol, P = 0.15), and the key safety parameters such as occurrence of urinary tract infections (24.1% vs. 15.5%, P = 0.10) were well comparable.
Conclusions: In conclusion, usage of Real-World Data from patient registries such as CERTAIN to emulate the control cohort of an RCT is feasible and could facilitate the conduct of clinical trials in pediatric kidney transplantation. A higher resolution version of the Graphical abstract is available as Supplementary information.
(© 2022. The Author(s).)
Databáze: MEDLINE
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