scholarly journals Novel Trial Design: CHIEF-HF

2021 ◽  
Vol 14 (3) ◽  
Author(s):  
John A. Spertus ◽  
Mary C. Birmingham ◽  
Javed Butler ◽  
Ildiko Lingvay ◽  
David E. Lanfear ◽  
...  
Keyword(s):  
Heart Failure ◽  
Clinical Trial ◽  
Ejection Fraction ◽  
Randomized Clinical Trial ◽  
Kansas City ◽  
Mobile Technologies ◽  
Patient Centered ◽  
Kansas City Cardiomyopathy Questionnaire ◽  
Diabetes Status ◽  
Patient Reported

Background: The expense of clinical trials mandates new strategies to efficiently generate evidence and test novel therapies. In this context, we designed a decentralized, patient-centered randomized clinical trial leveraging mobile technologies, rather than in-person site visits, to test the efficacy of 12 weeks of canagliflozin for the treatment of heart failure, regardless of ejection fraction or diabetes status, on the reduction of heart failure symptoms. Methods: One thousand nine hundred patients will be enrolled with a medical record-confirmed diagnosis of heart failure, stratified by reduced (≤40%) or preserved (>40%) ejection fraction and randomized 1:1 to 100 mg daily of canagliflozin or matching placebo. The primary outcome will be the 12-week change in the total symptom score of the Kansas City Cardiomyopathy Questionnaire. Secondary outcomes will be daily step count and other scales of the Kansas City Cardiomyopathy Questionnaire. Results: The trial is currently enrolling, even in the era of the coronavirus disease 2019 (COVID-19) pandemic. Conclusions: CHIEF-HF (Canagliflozin: Impact on Health Status, Quality of Life and Functional Status in Heart Failure) is deploying a novel model of conducting a decentralized, patient-centered, randomized clinical trial for a new indication for canagliflozin to improve the symptoms of patients with heart failure. It can model a new method for more cost-effectively testing the efficacy of treatments using mobile technologies with patient-reported outcomes as the primary clinical end point of the trial. Registration: URL: https://www.clinicaltrials.gov ; Unique identifier: NCT04252287.

scholarly journals Empagliflozin in heart failure patients with reduced ejection fraction: a randomized clinical trial (Empire HF)

Trials ◽  
2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Jesper Jensen ◽  
Massar Omar ◽  
Caroline Kistorp ◽  
Mikael Kjær Poulsen ◽  
Christian Tuxen ◽  
...  
Keyword(s):  
Heart Failure ◽  
Clinical Trial ◽  
Ejection Fraction ◽  
Randomized Clinical Trial ◽  
Reduced Ejection Fraction ◽  
Heart Failure Patients

scholarly journals Quality of life and disease experience in patients with heart failure with reduced ejection fraction in Spain: a mixed-methods study

BMJ Open ◽  
2021 ◽  
Vol 11 (12) ◽  
pp. e053216
Author(s):  
Raül Rubio ◽  
Beatriz Palacios ◽  
Luis Varela ◽  
Raquel Fernández ◽  
Selene Camargo Correa ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Heart Failure ◽  
Ejection Fraction ◽  
Kansas City ◽  
Mixed Methods Study ◽  
Kansas City Cardiomyopathy Questionnaire ◽  
Reduced Ejection Fraction ◽  
Patients With Heart Failure ◽  
Disease Experience

ObjectivesTo gather insights on the disease experience of patients with heart failure (HF) with reduced ejection fraction (HFrEF), and assess how patients’ experiences and narratives related to the disease complement data collected through standardised patient-reported outcome measures (PROMs). Also, to explore new ways of evaluating the burden experienced by patients and caregivers.DesignObservational, descriptive, multicentre, cross-sectional, mixed-methods study.SettingSecondary care, patient’s homes.ParticipantsTwenty patients with HFrEF (New York Heart Association (NYHA) classification I–III) aged 38–85 years.MeasuresPROMs EuroQoL 5D-5L (EQ-5D-5L) and Kansas City Cardiomyopathy Questionnaire and patient interview and observation.ResultsA total of 20 patients with HFrEF participated in the study. The patients’ mean (SD) age was 72.5 (11.4) years, 65% were male and were classified inNYHA functional classes I (n=4), II (n=7) and III (n=9). The study showed a strong impact of HF in the patients’ quality of life (QoL) and disease experience, as revealed by the standardised PROMs (EQ-5D-5L global index=0.64 (0.36); Kansas City Cardiomyopathy Questionnaire total symptom score=71.56 (20.55)) and the in-depth interviews. Patients and caregivers often disagreed describing and evaluating perceived QoL, as patients downplayed their limitations and caregivers overemphasised the poor QoL of the patients. Patients related current QoL to distant life experiences or to critical moments in their disease, such as hospitalisations. Anxiety over the disease progression is apparent in both patients and caregivers, suggesting that caregiver-specific tools should be developed.ConclusionsPROMs are an effective way of assessing symptoms over the most recent time period. However, especially in chronic diseases such as HFrEF, PROM scores could be complemented with additional tools to gain a better understanding of the patient’s status. New PROMs designed to evaluate and compare specific points in the life of the patient could be clinically more useful to assess changes in health status.

scholarly journals Extended‐Release Oral Milrinone for the Treatment of Heart Failure With Preserved Ejection Fraction

2020 ◽  
Vol 9 (13) ◽  
Author(s):  
Shane Nanayakkara ◽  
Melissa Byrne ◽  
Vivian Mak ◽  
Kaye Carter ◽  
Eliza Dean ◽  
...  
Keyword(s):  
Heart Failure ◽  
Ejection Fraction ◽  
Kansas City ◽  
Extended Release ◽  
Preserved Ejection Fraction ◽  
Walk Distance ◽  
Kansas City Cardiomyopathy Questionnaire ◽  
Minute Walk Distance ◽  
Minute Walk

Background Heart failure with preserved ejection fraction ( HF p EF ) is an increasingly prevalent form of heart failure, representing half of the total burden of heart failure. We hypothesised that modulation of the phosphodiesterase type 3/cyclic AMP using a novel oral formulation of milrinone might exert favorable effects HF p EF via pulmonary and systemic vasodilation and enhancement of ventricular relaxation. We assessed the safety and efficacy of oral milrinone on quality of life and functional outcomes in patients with HF p EF . Methods and Results The Mil HFPEF (Extended Release Oral Milrinone for the Treatment of Heart Failure With Preserved Ejection Fraction) study was a randomized, double‐blind, placebo‐controlled pilot study in 23 patients with symptomatic HF p EF . Efficacy end points included changes from baseline in Kansas City Cardiomyopathy Questionnaire summary score and 6‐minute walk distance. The primary safety end point was the development of clinically significant arrhythmia. The Kansas City Cardiomyopathy Questionnaire score improved significantly in milrinone‐treated patients compared with placebo (+10±13 versus −3±15; P =0.046). Six‐minute walk distance also tended to improve in the treatment group compared with placebo (+22 [−8 to 49] versus −47 [−97 to 12]; P =0.092). Heart rate (−1±5 versus −2±9 bpm; P =0.9) and systolic blood pressure (−3±18 versus +1±12 mm Hg; P =0.57) were unchanged. Early filling velocity/early mitral annular velocity (−0.3±3.0 versus −1.9±4.8; P =0.38) was unchanged. One patient in the placebo arm was hospitalized for heart failure. Holter monitoring did not demonstrate evidence of a proarrhythmic effect of milrinone. Conclusions In this novel pilot study, extended release oral milrinone was well tolerated and associated with improved quality of life in patients with HF p EF . Further longer‐term studies are warranted to establish the role of this therapeutic approach in  HF p EF . Registration URL : https://www.anzctr.org.au/ ; Unique identifier: ACTRN 12616000619448.

scholarly journals Association of Tubular Solute Clearance with Symptom Burden in Incident Peritoneal Dialysis

2020 ◽  
Vol 15 (4) ◽  
pp. 530-538 ◽  
Author(s):  
Ke Wang ◽  
Michelle Nguyen ◽  
Yan Chen ◽  
Andrew N. Hoofnagle ◽  
Jessica O. Becker ◽  
...  
Keyword(s):  
Heart Failure ◽  
Peritoneal Dialysis ◽  
Confidence Interval ◽  
Kidney Function ◽  
Kansas City ◽  
Summary Score ◽  
Symptom Index ◽  
Kansas City Cardiomyopathy Questionnaire ◽  
Residual Kidney Function ◽  
Patient Reported

Background and objectivesResidual kidney function is important to the health and wellbeing of patients with ESKD. We tested whether the kidney clearances of proximal tubular secretory solutes are associated with burden of uremic and heart failure symptoms among patients on peritoneal dialysis with residual kidney function.Design, setting, participants, & measurementsWe enrolled 29 patients on incident peritoneal dialysis with residual urine output >250 ml daily. We used targeted liquid chromatography-mass spectrometry to quantify plasma, 24-hour urine, and peritoneal dialysate concentrations of ten tubular secretory solutes. We calculated the kidney and peritoneal dialysis clearances of each secretory solute, creatinine, and urea, and we estimated a composite kidney and peritoneal secretion score. We assessed for uremic symptoms using the Dialysis Symptom Index and heart failure–related symptoms using the Kansas City Cardiomyopathy Questionnaire. We used linear regression to determine associations of composite secretory solute clearances and GFRurea+Cr with Dialysis Symptom Index symptom score and Kansas City Cardiomyopathy Questionnaire summary score.ResultsMean residual kidney clearances of creatinine and urea were 8±5 and 9±6 ml/min per 1.73 m2, respectively, and mean GFRurea+Cr was 8±5 ml/min per 1.73 m2. The residual kidney clearances of most secretory solutes were considerably higher than creatinine and urea clearance, and also, they were higher than their respective peritoneal dialysis clearances. After adjustments for age and sex, each SD higher composite kidney secretion score was associated with an 11-point lower Dialysis Symptom Index score (95% confidence interval, −20 to −1; P=0.03) and a 12-point higher Kansas City Cardiomyopathy Questionnaire score (95% confidence interval, 0.5- to 23-point higher score; P=0.04). Composite peritoneal dialysis secretion score was not associated with either symptom assessment.ConclusionsResidual kidney clearances of secretory solutes are higher than peritoneal dialysis clearances. Kidney clearances of secretory solutes are associated with patient-reported uremic and heart failure–related symptoms.

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