Tafamidis and quality of life in people with transthyretin amyloid cardiomyopathy in the study ATTR-ACT: A plain language summary

This plain language summary describes the results of a study called ATTR-ACT, which was published in the American Journal of Cardiology. In ATTR-ACT, researchers looked at the effects of tafamidis treatment in people with transthyretin amyloid cardiomyopathy (called ATTR-CM for short). Tafamidis is currently available in the USA and other countries as an oral treatment for adults with ATTR-CM. In ATTR-ACT, 441 people with ATTR-CM from 13 different countries took either tafamidis or placebo by mouth for 30 months. First, researchers looked at the effects of tafamidis on the risk of death and hospitalization due to heart problems between the start and the end of the study; they found that these risks were about one-third lower with tafamidis compared with placebo. As described in this summary, researchers also looked at the effects of tafamidis on people’s heart failure symptoms, quality of life, and general health over the 30-month study. People who took part in ATTR-ACT rated these effects using questionnaires filled out before, during, and after the study. More people who took tafamidis saw improvement or no change in their heart failure symptoms and quality of life than people who took placebo. In addition, compared with people taking placebo, people taking tafamidis had less worsening of their general health during the study. These results show the benefits of tafamidis in reducing the declines in quality of life and health that often occur with this debilitating disease. To read the full Plain Language Summary of this article, click on the View Article button above and download the PDF. Clinical Trial Registration: NCT01994889 ( ClinicalTrials.gov )

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MO131THE SHRUNKEN PORE SYNDROME IS ASSOCIATED WITH POOR PROGNOSIS AND LOWER QUALITY OF LIFE IN HEART FAILURE PATIENTS- THE HARVEST-MALMÖ STUDY

Nephrology Dialysis Transplantation ◽

10.1093/ndt/gfab092.009 ◽

2021 ◽

Vol 36 (Supplement_1) ◽

Author(s):

Liana Xhakollari ◽

Anders Grubb ◽

Amra Jujic ◽

Erasmus Bachus ◽

Peter M Nilsson ◽

...

Keyword(s):

Quality Of Life ◽

Heart Failure ◽

Poor Prognosis ◽

Kansas City ◽

Cox Regression ◽

Cardiorenal Syndrome ◽

Risk Of Death ◽

Increased Risk ◽

Health Related Qol

Abstract Background and Aims The cardiorenal syndrome was studied in heart failure (HF) patients with respect to the “Shrunken pore syndrome” (SPS) that is characterized by a difference in renal filtration between cystatin C and creatinine, resulting in a low eGFRcystatin C/eGFRcreatinine-ratio. Method 373 patients hospitalized for HF were retrieved from the HeARt and brain failure inVESTigation trial (HARVEST-Malmö). We used CKD-EPI formulas for estimated glomerular filtration rate (eGFR). Presence of SPS was defined as eGFRcystatinC ≤60% of eGFRcreatinine. In Cox regression multivariate models, associations between SPS, risk of death and risk of 30-day re-hospitalization were studied. Associations between SPS and impaired quality of life (QoL) were studied using multivariate logistic regressions. Results SPS was associated with all-cause mortality (124 events; hazard ratio (HR) 2.35; confidence interval (CI95%) 1.17-4.71; p=0.016 and with 30-day re-hospitalization (70 events; HR 1.82; CI95% 1.04-3.18; p=0.036). Analyses of QoL, based on a Kansas City Cardiomyopathy Questionnaire overall score <50, revealed that SPS was associated with increased risk of low health-related QoL (odds ratios (OR) 2.15 (CI95% 1.03-4.49; p=0.042). Conclusion The results of this observational study show for the first time an association between SPS and poor prognosis in HF. Further studies are needed to confirm the results in HF cohorts and experimental settings to identify pathophysiological mechanisms.

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Relationship Between Residual Mitral Regurgitation and Clinical and Quality-of-Life Outcomes After Transcatheter and Medical Treatments in Heart Failure: The COAPT Trial

Circulation ◽

10.1161/circulationaha.120.053061 ◽

2021 ◽

Author(s):

Saibal Kar ◽

Michael J. Mack ◽

JoAnn Lindenfeld ◽

William T. Abraham ◽

Federico M. Asch ◽

...

Keyword(s):

Quality Of Life ◽

Heart Failure ◽

Mitral Regurgitation ◽

Life Outcomes ◽

Unique Identifier ◽

Clinical Trial Registration ◽

Medical Treatments ◽

Transcatheter Mitral Valve Repair

Background: In the randomized COAPT trial, among 614 heart failure (HF) patients with 3+ or 4+ secondary mitral regurgitation (MR), transcatheter mitral valve repair (TMVr) with the MitraClip reduced MR, HF hospitalizations (HFH), and mortality and improved quality of life compared with guideline-directed medical therapy (GDMT) alone. We sought to examine the prognostic relationship between MR reduction and outcomes after TMVr and GDMT alone. Methods: Outcomes in COAPT between 30 days and 2 years were examined based on the severity of residual MR at 30 days. Results: TMVr-treated patients had less severe residual MR at 30 days than GDMT-treated patients (0/1+, 2+, and 3+/4+: 72.9%, 19.9%, and 7.2% versus 8.2%, 26.1%, and 65.8%, respectively, P<0.0001). The rate of composite death or HFH between 30 days and 2 years was lower in patients with 30-day residual MR of 0/1+ and 2+ compared with 3+/4+ (37.7% versus 49.5% versus 72.2%, respectively, P<0.0001). This relationship was consistent in the TMVr and GDMT arms (P interaction =0.92). The improvement in KCCQ score from baseline to 30 days was maintained between 30 days and 2 years in patients with 30-day MR ≤2+ but deteriorated in those with 30-day MR 3+/4+ (-0.3±1.7 versus -9.4±4.6, P=0.0008) consistently in both groups (P interaction =0.95). Conclusions: In the COAPT trial, reduced MR at 30 days was associated with greater freedom from death or HFH and improved quality of life through 2-year follow-up whether the MR reduction was achieved by TMVr or GDMT. Clinical Trial Registration: https://www.clinicaltrials.gov Unique Identifier: NCT01626079

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Darolutamide and survival in nonmetastatic, castration-resistant prostate cancer: a patient perspective of the ARAMIS trial

Future Oncology ◽

10.2217/fon-2020-1291 ◽

2021 ◽

pp. 1-9

Author(s):

K Fizazi ◽

Ian Blue ◽

Joel T Nowak

Keyword(s):

Prostate Cancer ◽

Hormone Therapy ◽

Bone Fractures ◽

Oral Treatment ◽

The Body ◽

Castration Resistant Prostate Cancer ◽

Plain Language ◽

Clinical Trial Registration ◽

Risk Of Death ◽

Castration Resistant

This is a summary of a publication about the ARAMIS (Androgen Receptor Antagonizing Agent for Metastasis-free Survival) trial, which was published in the New England Journal of Medicine in September 2020. The trial was in adult participants with nonmetastatic, castration-resistant prostate cancer (nmCRPC) who received a trial treatment called darolutamide (brand name Nubeqa®). Darolutamide is currently available as an oral treatment for adults with nmCRPC. The ARAMIS trial looked at darolutamide taken by mouth in 1509 participants from 36 countries with nmCRPC (prostate cancer that has not spread to other parts of the body and no longer responds adequately to initial hormone therapy). The trial showed that darolutamide in addition to hormone therapy increased the length of time that the trial participants were still alive for and lowered the risk of death by 31% compared with placebo (sugar pill) and hormone therapy. The participants who received darolutamide and hormone therapy also had longer time to worsening pain, needing chemotherapy, and having cancer-related bone fractures or symptoms related to cancer-related bone fractures compared with those who received placebo and hormone therapy during the trial. In general, the percentage of participants who experienced medical problems (referred to as adverse events) was similar between those who received darolutamide and those who received placebo, in addition to hormone therapy. This summary also includes insights and perspectives from a participant who was in the ARAMIS trial and from a prostate cancer patient advocate. To read the full Plain Language Summary of this article, click on the View Article button above and download the PDF. Clinical Trial Registration: NCT02200614 ( ClinicalTrials.gov )

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Parent reports of health-related quality of life and heart failure severity score independently predict outcome in children with dilated cardiomyopathy

Cardiology in the Young ◽

10.1017/s1047951116002833 ◽

2017 ◽

Vol 27 (6) ◽

pp. 1194-1202 ◽

Cited By ~ 3

Author(s):

Susanna L. den Boer ◽

Sara J. Baart ◽

Marijke H. van der Meulen ◽

Gabriëlle G. van Iperen ◽

Ad P. Backx ◽

...

Keyword(s):

Quality Of Life ◽

Heart Failure ◽

Dilated Cardiomyopathy ◽

Health Related Quality ◽

Risk Of Death ◽

Failure Index ◽

Related Quality ◽

Health Related ◽

Heart Failure Severity

AbstractBackgroundDilated cardiomyopathy in children causes heart failure and has a poor prognosis. Health-related quality of life in this patient group is unknown. Moreover, results may provide detailed information of parents’ sense of their child’s functioning. We hypothesised that health-related quality of life, as rated by parents, and the paediatric heart failure score, as assessed by physicians, have both predictive value on outcome.Methods and resultsIn this prospective study, health-related quality of life was assessed by parent reports: the Infant Toddler Quality of Life questionnaire (0–4 years) or Child Health Questionnaire-Parent Form 50 (4–18 years) at 3–6-month intervals. We included 90 children (median age 3.8 years, interquartile range (IQR) 0.9–12.3) whose parents completed 515 questionnaires. At the same visit, physicians completed the New York University Pediatric Heart Failure Index. Compared with Dutch normative data, quality of life was severely impaired at diagnosis (0–4 years: 7/10 subscales and 4–18 years: 8/11 subscales) and ⩾1 year after diagnosis (3/10 and 6/11 subscales). Older children were more impaired (p<0.05). After a median follow-up of 3 years (IQR 2–4), 15 patients underwent transplantation. Using multivariable time-dependent Cox regression, “physical functioning” subscale and the Heart Failure Index were independently predictive of the risk of death and heart transplantation (hazard ratio 1.24 per 10% decrease of predicted, 95% confidence interval (CI) 1.06–1.47 and hazard ratio 1.38 per unit, 95% CI 1.19–1.61, respectively).ConclusionPhysical impairment rated by parents and heart failure severity assessed by physicians independently predicted the risk of death or heart transplantation in children with dilated cardiomyopathy.

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