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

  • Christian Patry (Geteilte/r Erstautor/in)
  • Lukas D Sauer (Geteilte/r Erstautor/in)
  • Anja Sander
  • Kai Krupka
  • Alexander Fichtner
  • Jolanda Brezinski
  • Yvonne Geissbühler
  • Elodie Aubrun
  • Anna Grinienko
  • Luca Dello Strologo
  • Dieter Haffner
  • Jun Oh
  • Ryszard Grenda
  • Lars Pape
  • Rezan Topaloğlu
  • Lutz T Weber
  • Antonia Bouts
  • Jon Jin Kim
  • Agnieszka Prytula
  • Jens König
  • Mohan Shenoy
  • Britta Höcker (Geteilte/r Letztautor/in)
  • Burkhard Tönshoff (Geteilte/r Letztautor/in)

Abstract

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 m2 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.

Bibliografische Daten

OriginalspracheEnglisch
ISSN0931-041X
DOIs
StatusVeröffentlicht - 05.2023

Anmerkungen des Dekanats

© 2022. The Author(s).

PubMed 36264431