Core Outcome Measures in Effectiveness Trials

Toward Establishing Core Outcome Domains For Trials in Kidney Transplantation: Report of the Standardized Outcomes in Nephrology — Kidney Transplantation Consensus Workshops.

General Information

Abstract:
Background.

Treatment decisions in kidney transplantation requires patients and clinicians to weigh the benefits and harms of a broad range of medical and surgical interventions, but the heterogeneity and lack of patient-relevant outcomes across trials in transplantation makes these trade-offs uncertain, thus, the need for a core outcome set that reflects stakeholder priorities.

Methods.

We convened 2 international Standardized Outcomes in Nephrology-Kidney Transplantation stakeholder consensus workshops in Boston (17 patients/caregivers; 52 health professionals) and Hong Kong (10 patients/caregivers; 45 health professionals). In facilitated breakout groups, participants discussed the development and implementation of core outcome domains for trials in kidney transplantation.

Results.

Seven themes were identified. Reinforcing the paramount importance of graft outcomes encompassed the prevailing dread of dialysis, distilling the meaning of graft function, and acknowledging the terrifying and ambiguous terminology of rejection. Reflecting critical trade-offs between graft health and medical comorbidities was fundamental. Contextualizing mortality explained discrepancies in the prioritization of death among stakeholders — inevitability of death (patients), preventing premature death (clinicians), and ensuring safety (regulators). Imperative to capture patient-reported outcomes was driven by making explicit patient priorities, fulfilling regulatory requirements, and addressing life participation. Specificity to transplant; feasibility and pragmatism (long-term impacts and responsiveness to interventions); and recognizing gradients of severity within outcome domains were raised as considerations.

Conclusions.

Stakeholders support the inclusion of graft health, mortality, cardiovascular disease, infection, cancer, and patient-reported outcomes (ie, life participation) in a core outcomes set. Addressing ambiguous terminology and feasibility is needed in establishing these core outcome domains for trials in kidney transplantation.

Aim:
This workshop report describes stakeholder reflec-
tions and deliberations on the core outcomes domains in kidney
transplantation, and recommendations for the way forward.

Authors:
Allison Tong, PhD,

John Gill, MD,

Klemens Budde, MD,

Lorna Marson, MD, FRCS,

Peter P. Reese, MD, MSCE,

David Rosenbloom,

Lionel Rostaing, MD, PhD,

Germaine Wong, MBBS, PhD,

Michelle A. Josephson, MD,

Timothy L. Pruett, MD,

Anthony N. Warrens, MD,

Jonathan C. Craig, MBChB, PhD,

Benedicte Sautenet, MD, PhD,

Nicole Evangelidis, BSocSc,

Angelique F. Ralph, BPsych (Hons),

Camilla S. Hanson, BPsych (Hons),

Jenny I. Shen, MD,

Kirsten Howard, PhD,

Klemens Meyer, MD,

Ronald D. Perrone, MD,

Daniel E. Weiner, MD,

Samuel Fung, MBBS, FRCP,

MaggieK.M.Ma,MBBS,MPH,

Caren Rose, PhD,

Jessica Ryan, MBBS, PhD,

Ling-Xin Chen, MD,

Martin Howell, PhD,

Nicholas Larkins, MBBS, MMed (Clin Epi),

Siah Kim, MBBS, PhD,

Sobhana Thangaraju, MD,

Angela Ju, BSc (Hons),

and Jeremy R. Chapman, FRACP

on behalf of the SONG-Tx Investigators

Publication

Journal:
Transplantation
Volume:
Issue:
101
Pages:
1887 - 1896
Year:
2017
DOI:
Further Study Information

Date:
Funding source(s):
None stated

Health Area

Disease Category
Kidney disease

Disease Name
Transplant surgery
Kidney Transplant

Target Population

Age Range
0 - 110

Sex
Either


Nature / type of Intervention
Surgery

Method(s)

Consensus conference
Consensus meeting
Delphi process

Stakeholders Involved

Clinical experts
Consumers (caregivers)
Consumers (patients)

Study Type

COS for clinical trials or clinical research

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