Utility of Minimum Dataset for Generation of Real-World Data in a Context With Evidence Gaps: The Left Ventricular Assist Devices as Destination Therapy
Author(s)
Janet Puñal-Rioboo, MPH, Ana Maria Vázquez Castelo, MSc, Ewa Pawlowska Pawlowska, PhD, Ignacio José López-Loureiro, PhD, María-José Faraldo Vallés, PhD.
Avalia-t, Galician Health Knowledge Agency (ACIS), Santiago de Compostela, Spain.
Avalia-t, Galician Health Knowledge Agency (ACIS), Santiago de Compostela, Spain.
OBJECTIVES: Patients with advanced heart failure who are not candidates for cardiac transplant could benefit from left ventricular assist device (LVAD) implantation as destination therapy (DT). However, key issues exist regarding the selection of suitable patients. In order to solve these uncertainties, a Delphi consensus-based minimum dataset (MDS), composed of 18 outcomes and 47 variables/measures divided into 7 domains was developed. This MDS was included in a real-world prospective multicenter registry (Monitoring study of LVAD as DT) in the Spanish National Health System (NHS). The aim is to evaluate its implementation and utility in working out evidence gaps identified previously.
METHODS: A registry protocol was designed including criteria of patient’s selection, follow-up intervals (3, 6, 12, 24 months), sample size, quality data assurance plan and the MDS created previously. This protocol was agreed by all participant hospitals (n=26). A qualitative evaluation addressing the MDS usefulness solving evidence gaps was done.
RESULTS: In the first year of follow-up, the main identified challenge was the high rate of adverse events. Regarding the effectiveness of LVAD as DT, it was reported an improvement of clinical/hemodynamic outcomes (NYHA class, INTERMACS profile and LVEF). In addition to that, patient-centered outcomes as quality of life (QoL)(EuroQoL and KCCQ) improved both in overall scores and by dimensions. Moreover, patients reported feeling satisfied with intervention and would undergo surgery again. The safety and clinical/hemodynamic findings were aligned with previously published results. However, data about QoL and satisfaction/acceptability of patients provided relevant insights into these outcomes considered evidence gaps, as they are not well reported in published literature.
CONCLUSIONS: The development of MDS based on identified evidence gaps and its implementation in a real-world registry can be a feasible strategy for generating valuable additional information for better-informed decisions. A long-term follow-up is needed to confirm these initial findings.
METHODS: A registry protocol was designed including criteria of patient’s selection, follow-up intervals (3, 6, 12, 24 months), sample size, quality data assurance plan and the MDS created previously. This protocol was agreed by all participant hospitals (n=26). A qualitative evaluation addressing the MDS usefulness solving evidence gaps was done.
RESULTS: In the first year of follow-up, the main identified challenge was the high rate of adverse events. Regarding the effectiveness of LVAD as DT, it was reported an improvement of clinical/hemodynamic outcomes (NYHA class, INTERMACS profile and LVEF). In addition to that, patient-centered outcomes as quality of life (QoL)(EuroQoL and KCCQ) improved both in overall scores and by dimensions. Moreover, patients reported feeling satisfied with intervention and would undergo surgery again. The safety and clinical/hemodynamic findings were aligned with previously published results. However, data about QoL and satisfaction/acceptability of patients provided relevant insights into these outcomes considered evidence gaps, as they are not well reported in published literature.
CONCLUSIONS: The development of MDS based on identified evidence gaps and its implementation in a real-world registry can be a feasible strategy for generating valuable additional information for better-informed decisions. A long-term follow-up is needed to confirm these initial findings.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO262
Topic
Clinical Outcomes, Patient-Centered Research, Real World Data & Information Systems
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), No Additional Disease & Conditions/Specialized Treatment Areas