Kidney dysfunction after heart transplantation (HT) is associated with significant morbidity and mortality. Recipient and perioperative factors may all influence the risk of kidney injury. Furthermore, data suggest that the incidence of kidney dysfunction, both acute and chronic, is increasing after the implementation of the United States' 2018 allocation system due to increasing use of temporary mechanical circulatory support and changing recipient characteristics. While data are robust regarding nephroprotective therapies such as renin-angiotensin-aldosterone system inhibition and SGLT2i (sodium-glucose cotransporter 2 inhibitors) to minimize the progression of chronic kidney disease in patients with heart failure, data in HT recipients are beginning to emerge. This state-of-the-art review will critically examine the existing literature regarding the epidemiology of kidney dysfunction after HT, mitigation strategies for acute kidney injury and chronic kidney disease, including pharmacotherapeutics, the need for kidney transplantation after HT, and practical next steps for the larger HT community.

Valosin-containing protein (VCP)-associated multisystem proteinopathy is a rare, autosomal dominant disease that affects skeletal muscle, bone, central nervous system, and the heart. While VCP mutations are well established as causing inclusion body myopathy, Paget's disease of bone, frontotemporal dementia, and amyotrophic lateral sclerosis, their role in cardiomyopathy remains underrecognized. This review aims to evaluate the pathophysiology, diagnostic approach, and management of VCP-associated cardiomyopathy to provide a framework for clinical care and future research. Emerging evidence from animal models and human case studies suggests that VCP dysfunction disrupts cardiomyocyte homeostasis, impairs protein degradation, and alters mitochondrial function, leading to maladaptive cardiac remodeling and susceptibility to dilated or hypertrophic cardiomyopathy. Echocardiographic studies in patients with VCP variants reveal a significant prevalence of diastolic dysfunction, conduction abnormalities, and variable degrees of systolic impairment. Despite these findings, there are no standardized guidelines for the diagnosis and management of VCP-associated cardiomyopathy. Current treatment strategies are extrapolated from heart failure guidelines, incorporating neurohormonal blockades with angiotensin-converting enzyme inhibitors, beta-blockers, and mineralocorticoid receptor antagonists. Our review highlights the need for systematic screening protocols, genotype-phenotype correlation studies, and the development of targeted therapies. Future research should focus on identifying biomarkers for early detection, elucidating the molecular mechanisms underlying cardiac dysfunction, and assessing the efficacy of novel treatment strategies. Recognizing VCP-associated cardiomyopathy as a distinct clinical entity will facilitate earlier diagnosis, improve patient outcomes, pave the way for disease-specific therapeutic interventions and insights into the mechanism for isolated cardiomyopathy.

Purpose: The Minnesota Pectoralis Risk Score (MPRS) utilizes CT quantified thoracic muscle measures and clinical variables to predict survival after left ventricular assist device (LVAD) implantation. The model has not been assessed as a predictor of right ventricular failure (RVF) after LVAD.

Purpose: Published data on therapeutic plasma exchange (TPE) in patients with heparin induced thrombocytopenia (HIT) prior to left ventricular assist device (LVAD) implantation is limited. Here we describe a case series of patients with acute HIT who underwent TPE prior to LVAD implantation allowing the use of intraoperative heparin.

BACKGROUND: The Minnesota Pectoralis Risk Score (MPRS) utilizes computed tomography-quantified thoracic muscle and clinical variables to predict survival after left ventricular assist device (LVAD) implantation. The model has not been prospectively tested in HeartMate 3 recipients. METHODS: A single-center HeartMate 3 cohort from July 2016 to July 2021 (n = 108) was utilized for this analysis. Cohort subjects with complete covariates for MPRS calculation (pectoralis muscle measures, Black race, creatinine, total bilirubin, body mass index, bridge to transplant status, and presence/absence of contrast) implanted after MPRS development were included. MPRS were calculated on each subject. Receiver operating characteristic curves were generated to test model discrimination at 30-day, 90-day, and 1-year mortality post-LVAD. Next, the performance of the 1-year post-LVAD outcome was compared to the HeartMate 3 survival risk score (HM3RS). RESULTS: The mean age was 58 (15 years), 80% (86/108) were male, and 26% (28/108) were destination therapy. The area under the curve (AUC) for the MPRS model to predict post-LVAD mortality was 0.73 at 30 days, 0.78 at 90 days, and 0.81 at 1 year. The AUC for the HM3RS for the 1-year outcome was 0.693. Each 1-unit point of the MPRS was associated with a significant increase in the hazard rate of death after LVAD (hazard ratio 2.1, 95% confidence interval 1.5-3.0, p < 0.0001). CONCLUSIONS: The MPRS had high performance in this prospective validation, particularly with respect to 90-day and 1-year post-LVAD mortality. Such a tool can provide additional information regarding risk stratification to aid informed decision-making. (c) 2023 International Society for Heart and Lung Transplantation. All rights reserved.

Superior vena cava syndrome is a constellation of clinical signs and symptoms resulting from the obstruction of blood flow through the superior vena cava. Although it rarely occurs in the setting of orthotopic heart transplantation, here we report a case that developed in the early postoperative setting. Our goal is to raise awareness of this entity, given the associated high fatality rate and potential challenges of timely recognition in the setting of sedation and mechanical ventilation.

Hypertrophic cardiomyopathy (HCM) is characterized by myocyte hypertrophy, sarcomere disarray, and myocardial fibrosis, leading to significant morbidity and mortality. As the most common inherited cardiomyopathy, HCM largely results from mutations in sarcomeric protein genes. Current treatments for HCM primarily focus on alleviating late-stage symptoms, with a critical gap in the detailed understanding of early-stage deficiencies that drive disease progression. We recently showed, in monolayers of cardiomyocytes derived from human induced pluripotent stem cells (hiPSCs) with MYH7 R723C and MYH6 R725C mutations, altered expression of several extracellular matrix (ECM)-related genes with associated defects in cardiomyocyte-ECM adhesion. To better evaluate the cardiomyocyte-ECM interface and pathological ECM dynamics in early-stage HCM, here we adopted a 3D engineered heart tissue (EHT) model containing both cardiomyocytes and fibroblasts, the primary contributor to ECM remodeling. Mutant EHTs showed aberrant cardiomyocyte distribution, augmented calcium handling, and force generation compared to controls. Altered proteoglycan deposition and increased phosphorylated focal adhesion kinase (pFAK) further indicated changes in ECM composition and connectivity. Elevated transforming growth factor beta-1 (TGF-β1) secretion and a higher proportion of activated fibroblasts were identified in mutant EHTs, along with sustained TGF-β1 transcription specifically in mutant cardiomyocytes. Remarkably, blocking TGF-β1 receptor signaling reduced fibroblast activation and contraction force to control levels. This study underscores the early interplay of mutant hiPSC-CMs with fibroblasts, wherein mutant cardiomyocytes initiate fibroblast activation via TGF-β1 overexpression, independent of the immune system. These findings provide a promising foundation for developing and implementing novel strategies to treat HCM well before the manifestation of clinically detectable fibrosis and cardiac dysfunction. ### Competing Interest Statement The authors have declared no competing interest.

Purpose: Low thoracic muscle mass and tissue quantified on preoperative chest CTs are associated with increased post LVAD mortality. The serum sarcopenia index (SI), a ratio between serum creatinine and cystatin C, correlates with CT quantified muscle measurements in several disease states. The purpose of this analysis was to determine whether the SI itself is associated with post LVAD mortality.

Human induced pluripotent stem cell cardiomyocytes (hiPSC-CMs) are based on ground-breaking technology that has significantly impacted cardiovascular research. They provide a renewable source of human cardiomyocytes for a variety of applications including in vitro disease modeling and drug toxicity testing. Cardiac calcium regulation plays a critical role in the cardiomyocyte and is often dysregulated in cardiovascular disease. Due to the limited availability of human cardiac tissue, calcium handling and its regulation have most commonly been studied in the context of animal models. hiPSC-CMs can provide unique insights into human physiology and pathophysiology, although a remaining limitation is the relative immaturity of these cells compared to adult cardiomyocytes Therefore, this field is rapidly developing techniques to improve the maturity of hiPSC-CMs, further establishing their place in cardiovascular research. This review briefly covers the basics of cardiomyocyte calcium cycling and hiPSC technology, and will provide a detailed description of our current understanding of calcium in hiPSC-CMs.

OBJECTIVES/GOALS: We will use control- and DMD-engineered heart tissues to better model and investigate DMD cardiomyopathy. We will primarily assess cardiac calcium handling, mitochondrial function, and mitochondrial calcium handling, as calcium regulation and mitochondrial function are known to be affected in DMD. METHODS/STUDY POPULATION: We will use patient-derived stem cells, differentiated into cardiomyocytes in bioprinted 3D heart tissue muscle chambers to better model DMD cardiomyopathy. We will look at calcium handling and general mitochondrial function, as well as mitochondrial calcium handling, using a novel multifunctional genetic probe I previously developed allowing for simultaneous observation of cytosolic and mitochondrial calcium in real time. Optical mapping will also be used for tissue-level analysis. We will establish the functional differences at baseline, and then progress heart failure in the tissues to see how the abnormalities seen in the DMD tissues may get worse. Finally, we will investigate the effects of early restoration of dystrophin function on the effects of DMD cardiomyopathy development. RESULTS/ANTICIPATED RESULTS: We anticipate that DMD tissues will show more irregular/abnormal calcium handling, as seen in 2D hiPSC-CMs, as well as disruptions to mitochondrial function and ultrastructural development, as well as a decreased synchronization between cytosolic and mitochondrial calcium dynamics. We anticipate that these abnormalities will be exacerbated as the disease state progresses, but at least partially ameliorated with the restoration of dystrophin function. DISCUSSION/SIGNIFICANCE: DMD is a fatal disease with no known cure. Patients develop heart failure in their teens and die in their 20s, so any new insight that may prolong life and improve quality of life for patients is drastically needed. This would be the most accurate preclinical model of DMD cardiomyopathy to date and would investigate yet-untapped aspects of the disease state.

Heart failure (HF) affects more than 64 million individuals worldwide, including 6.5 million adults in the United States.1,2 In addition to its escalating global prevalence, HF is a leading cause of mortality and morbidity worldwide, resulting in increasing hospitalizations and significant health care expendi-tures. Despite considerable advances in medical and device therapies for patients across the spectrum of HF, the burden of HF continues to grow; thus, there is an urgent global priority to address further the pathogenesis and treatment of HF. HF is a complex and heterogeneous clinical syn-drome that requires an understanding of the under-lying disease etiology, severity, congestive profile, and pathogenesis so as to optimize treatment and management. Additionally, from a global perspec-tive, the distribution of HF etiologies varies by geo-graphic region, necessitating local investigation to address the unique HF features of that population. In addition to ongoing clinical efforts, further unrav-eling of the molecular mechanisms of HF is needed in order to accelerate the development of novel eti-ology-specific therapies. Translational research in HF can play an integral role in facilitating a personalized approach to HF, including care of spe-cial populations in whom differences have been documented increasingly.

More than 60% of hypertrophic cardiomyopathy (HCM)-causing mutations are found in the gene loci encoding cardiac myosin-associated proteins including myosin heavy chain (MHC) and myosin binding protein C (MyBP-C). Moreover, patients with more than one independent HCM mutation may be at increased risk for more severe disease expression and adverse outcomes. However detailed mechanistic understanding, especially at early stages of disease progression, is limited. To identify early-stage HCM triggers, we generated single ( MYH7 c . 2167C>T [R723C] with a known pathogenic significance in the MHC converter domain) and double ( MYH7 c . 2167C>T [R723C]; MYH6 c . 2173C>T [R725C] with unknown significance) myosin gene mutations in human induced pluripotent stem cells (hiPSCs) using a base-editing strategy. Cardiomyocytes (CMs) derived from hiPSCs with either single or double mutation exhibited phenotypic characteristics consistent with later-stage HCM including hypertrophy, multinucleation, altered calcium handling, metabolism and arrhythmia. We then probed mutant CMs at time points prior to the detection of known HCM characteristics. We found MYH7/MYH6 dual mutation dysregulated extracellular matrix (ECM) remodeling, altered integrin expression, and interrupted cell-ECM adhesion by limiting the formation of focal adhesions. These results point to a new phenotypic feature of early-stage HCM and reveal novel therapeutic avenues aimed to delay or prohibit disease onset.

A single-center continuous-flow left ventricular assist device (LVAD) cohort (n = 503) was reviewed for patients with information on cardiac rehabilitation (CR) participation (n = 273) over a 13-year period. The analysis was then limited LVAD recipients who fit into three main CR categories: those who graduated CR (n = 138), those who were able to but declined participation (n = 61), and those who were too sick to complete or start CR (n = 28). To assess the association between CR categories and mortality and hospitalizations on LVAD support, multivariate cox regression and negative binomial regression analyses were performed, respectively. Among those who started CR and had the opportunity to finish (enough follow-up time, insurance coverage), 79% graduated. Those who graduated CR had a 96% survival at 1 year (95% confidence interval [CI], 91–98). Compared with the graduated group, those in the too sick group had an increased hazards rate of mortality (hazard ratio, 2.85; 95% CI, 1.49–5.44; p < 0.01) and an increase in the incidence rate of hospitalizations (incidence rate ratio, 1.74; 95% CI, 1.14–2.66, p = 0.01). This study is the largest to date to report outcomes of LVAD recipients referred for CR. The lower readmission rates and high survival in the group that graduated CR provides further evidence for the safety of CR in LVAD recipients.

The Kidney Disease Improving Global Outcome guidelines recommend the use of cystatin-C over serum creatinine in patients with muscle wasting conditions. 1 Stevens PE Levin A Kidney Disease Improving Global Outcomes Chronic Kidney Disease Guideline Development Work Group MembersEvaluation and management of chronic kidney disease: synopsis of the kidney disease: improving global outcomes 2012 clinical practice guideline. Ann Intern Med. 2013; 158: 825-830 Crossref PubMed Scopus (1917) Google Scholar Muscle wasting is common in heart failure (HF); however, HF guidelines have not yet shifted away from the use of serum creatinine to estimate renal function. 2 Heidenreich PA Bozkurt B Aguilar D Allen LA Byun JJ Colvin MM et al. 2022 AHA/ACC/HFSA guideline for the management of heart failure: executive summary: a report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. J Am Coll Cardiol. 2022; 79: 1757-1780 Crossref PubMed Scopus (142) Google Scholar A recent prospective left ventricular assist device (LVAD) cohort analysis found that 52% of LVAD recipients were sarcopenic before implantation, with a gain of 2.3 kg change in fat-free mass (ie, muscle) by dual X-ray absorptiometry in the first 3 months after LVAD implantation. 3 Vest AR Wong WW Chery J Coston A Telfer L Lawrence M et al. Skeletal muscle mass recovery early after left ventricular assist device implantation in patients with advanced systolic heart failure. Circ Heart Fail. 2022; 15e009012 Crossref PubMed Scopus (5) Google Scholar In another LVAD cohort, serial cystatin-C and creatinine measurements over time suggested an overestimation of renal function using serum creatinine in the postoperative period owing to muscle wasting. 4 Pinsino A Mondellini GM Royzman EA Hoffman KL D'Angelo D Mabasa M et al. Cystatin C- versus creatinine-based assessment of renal function and prediction of early outcomes among patients with a left ventricular assist device. Circ Heart Fail. 2020; 13e006326 Crossref PubMed Scopus (14) Google Scholar In this analysis, we build on this work by using a single-center, well-characterized advanced HF cohort with preoperative quantitative thoracic muscle measurements, cystatin-C, and serial body mass index (BMI) and creatinine values to investigate the hypotheses that (1) BMI decreases before LVAD and this decrease will be associated with thoracic muscle mass and quality, (2) that pre-LVAD serum cystatin-C and creatinine will differ significantly, and (3) post-LVAD BMI and creatinine estimated glomerular filtration rate (eGFR) trends will be inversely related (creatinine will rise as body weight increases). If these hypotheses are true, this would support the need for alternative, non–muscle-dependent measures of renal function in the advanced HF population.

Purpose The HeartMate 3 left ventricular assist device (LVAD) has lower stroke rates compared to the HeartMate 2 in a clinical trial setting. Here we report real world stroke rates in patients implanted with HeartMate 2 and HeartMate 3 LVADs. Methods Data from a single center, first time continuous-flow LVAD cohort was utilized for this analysis (n=549). Patients with complete stroke data implanted with HeartMate2 (n=151) or HeartMate3(n=167) were included (n=318). Time to first stroke and stroke type were recorded. Freedom from all cause stroke by device type was estimated using the Kaplan Meir method and compared with the log rank test. To assess the adjusted difference in the rate of stroke by pump type, a multivariable cox regression was performed Results The mean age of the study cohort was 59 +/- 14 years, 83 % (265/318) were men, 63 % (197/318) were destination therapy and 31 % (97/318) were INTERMACS profiles 1 and 2. Freedom from all cause stroke to 1 year of LVAD support was similar between pumps (HeartMate 3: 88 % vs. HeartMate 2: 89 %, p = 0.76, adjusted HR 1.12, 95% CI 0.56-2.26, p=0.74), however the odds of thrombotic stroke in the first 60 days post-implantation was higher in the HeartMate 3 recipients (OR 2.5, 95 % CI 0 .86- 7.12, p=0.093). Ninety-five percent (17/18) of stroke events in HeartMate 3 recipients in the first 60 days post-implantation were ischemic in etiology. Conclusion The wider gaps within the HeartMate 3 may increase the risk of early post-operative embolic events. Special attention to prevent any material ingestion into the pump during HeartMate 3 implant is likely important and may reduce strokes in the early post-operative period.

Duchenne muscular dystrophy (DMD) affects approximately 1 in 5,000 boys worldwide. 1 Kamdar F Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016; 67: 2533-2546 Crossref PubMed Scopus (173) Google Scholar ,2 Cox GF Kunkel LM. Dystrophies and heart disease. Curr Opin Cardiol. 1997; 12: 329-343 Crossref PubMed Scopus (226) Google Scholar This X-linked recessive disease is a result of spontaneous or inherited mutations in the dystrophin gene. 3 Hoffman EP Brown Jr., RH Kunkel LM Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987; 51: 919-928 Abstract Full Text PDF PubMed Scopus (3603) Google Scholar The dystrophin gene is the longest gene in the human genome as it spans 2.5 megabases, includes 79 exons, and encodes a 427-kDa cytolinker protein. 1 Kamdar F Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016; 67: 2533-2546 Crossref PubMed Scopus (173) Google Scholar ,3 Hoffman EP Brown Jr., RH Kunkel LM Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987; 51: 919-928 Abstract Full Text PDF PubMed Scopus (3603) Google Scholar The dystrophin protein connects the interior of the cell (skeletal myocyte, cardiomyocyte, or neuron) by binding to actin via its amino terminal domain to the sarcolemma as it binds to β-dystroglycan via its carboxyterminal domain. 1 Kamdar F Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016; 67: 2533-2546 Crossref PubMed Scopus (173) Google Scholar ,3 Hoffman EP Brown Jr., RH Kunkel LM Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987; 51: 919-928 Abstract Full Text PDF PubMed Scopus (3603) Google Scholar ,4 Ervasti JM Campbell KP. A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin. J Cell Biol. 1993; 122: 809-823 Crossref PubMed Scopus (1159) Google Scholar In this fashion, dystrophin is part of a larger complex also referred to the dystrophin glycoprotein complex, which interacts with a number proteins and signaling factors. 1 Kamdar F Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016; 67: 2533-2546 Crossref PubMed Scopus (173) Google Scholar ,3 Hoffman EP Brown Jr., RH Kunkel LM Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987; 51: 919-928 Abstract Full Text PDF PubMed Scopus (3603) Google Scholar ,4 Ervasti JM Campbell KP. A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin. J Cell Biol. 1993; 122: 809-823 Crossref PubMed Scopus (1159) Google Scholar In the absence of dystrophin, the dystrophin glycoprotein complex is destabilized, and the sarcolemma develops micropunctures resulting in an influx of calcium leading to myocyte necrosis and fibrosis. 1 Kamdar F Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016; 67: 2533-2546 Crossref PubMed Scopus (173) Google Scholar ,3 Hoffman EP Brown Jr., RH Kunkel LM Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987; 51: 919-928 Abstract Full Text PDF PubMed Scopus (3603) Google Scholar These cellular events result in loss of ambulation and ultimately premature death. Although pediatric neurologists, pulmonologists, and orthopedic surgeons have improved the quality of life and extended survival through the use of steroid therapy, nocturnal ventilation, and spinal stabilization, essentially all the patients now develop a cardiomyopathy that results in their demise. 1 Kamdar F Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016; 67: 2533-2546 Crossref PubMed Scopus (173) Google Scholar

Purpose To evaluate the hypothesis that weight gain after left ventricular assist device (LVAD) occurs after the nadir of natriuretic peptides (nt-proBNP), and that persistently elevated natriuretic peptides is associated with failure to gain weight after LVAD implantation. Methods Body mass index (BMI) and nt-proBNP trends before and after LVAD in a single center continuous flow cohort (n=505) were computed and plotted using Locally Estimated Scatterplot Smoothing (LOESS). Linear mixed effects modeling was performed to test the association between nt-proBNP and BMI. CT measures of sarcopenia (pectoralis muscle mass index to body surface area and tissue attenuation, n=194) was correlated with pre-LVAD BMI trend. Results The plots of BMI and log nt-proBNP for the entire cohort before and after LVAD are demonstrated (Figure). The rise in BMI did not occur until after the nadir of post implant nt-proBNP. Downward slope of BMI prior to LVAD was associated with lower pectoralis muscle measures prior to implantation. Patients who failed to gain weight post LVAD had the highest 6 month post LVAD natuetic peptides (lowest pecent BMI gain tertile nt-proBNP: 2,208 vs. highest 1,635 pg/mL, p <0.001). Post LVAD nt-pro BNP and BMI trends were significantly associated (a decrease of 1 unit log nt-proBNP was associated with an increase in 0.81 BMI, 95 % CI: 0.53-1.09, p < 0.001). Conclusions Weight gain post LVAD did not occur until the nadir of natriuretic peptides. Failure to gain weight post LVAD was associated with persistently elevated natriuretic peptides. These data collectively suggest that gains in body mass may be dependent on resolution of heart failure. To evaluate the hypothesis that weight gain after left ventricular assist device (LVAD) occurs after the nadir of natriuretic peptides (nt-proBNP), and that persistently elevated natriuretic peptides is associated with failure to gain weight after LVAD implantation. Body mass index (BMI) and nt-proBNP trends before and after LVAD in a single center continuous flow cohort (n=505) were computed and plotted using Locally Estimated Scatterplot Smoothing (LOESS). Linear mixed effects modeling was performed to test the association between nt-proBNP and BMI. CT measures of sarcopenia (pectoralis muscle mass index to body surface area and tissue attenuation, n=194) was correlated with pre-LVAD BMI trend. The plots of BMI and log nt-proBNP for the entire cohort before and after LVAD are demonstrated (Figure). The rise in BMI did not occur until after the nadir of post implant nt-proBNP. Downward slope of BMI prior to LVAD was associated with lower pectoralis muscle measures prior to implantation. Patients who failed to gain weight post LVAD had the highest 6 month post LVAD natuetic peptides (lowest pecent BMI gain tertile nt-proBNP: 2,208 vs. highest 1,635 pg/mL, p <0.001). Post LVAD nt-pro BNP and BMI trends were significantly associated (a decrease of 1 unit log nt-proBNP was associated with an increase in 0.81 BMI, 95 % CI: 0.53-1.09, p < 0.001). Weight gain post LVAD did not occur until the nadir of natriuretic peptides. Failure to gain weight post LVAD was associated with persistently elevated natriuretic peptides. These data collectively suggest that gains in body mass may be dependent on resolution of heart failure.

Advanced heart failure (HF) therapies improve survival in patients with stage D HF. We sought to evaluate differences by race/ethnicity and sex in advanced HF therapy referrals and decision-making across a multicenter survey. We performed a retrospective analysis of patients referred for evaluation for advanced HF therapies at 9 centers (n = 515) across the United States. The median age was 58 years, and 73% were male. White patients comprised 55.7% of referrals, whereas non-White patients comprised 44.3%. Non-ischemic etiology was more common in non-White patients (66.6% vs 47.4% p = 0.0005), and ischemic etiology was more common in men (37.8% vs 20.4% p = 0.0005). The primary reason for referral differed by race/ethnicity but not sex, with ventricular arrhythmias (7.6% vs 3%, p = 0.024) and pulmonary hypertension (3.4% vs 0.4% p = 0.018) being more common in White patients, whereas worsening HF was less common (25.4% vs 35.9%; p = 0.009). White patients were offered left ventricular assist devices (LVADs) (60.3% vs 54.7 p = 0.039) and heart transplants (51.8% vs 33.1% p = 0.0007) more often than non-White patients. The preference not to pursue LVAD therapy was more common in non-White patients (17.6% vs 9.6%; p = 0.049). Men were more often declined for a heart transplant because of psychosocial contraindications (34% vs 15%, p = 0.005). In conclusion, in this multicenter analysis of referrals for advanced HF therapies, we observed significant differences by race, ethnicity, and sex in both referral characteristics and evaluation outcomes. Further investigation is warranted to better understand why rates of LVAD and transplantation may be lower in non-White patients who are referred for advanced therapies.

Background:Myotonic dystrophies (DM) are multi-systemic diseases characterized by muscle weakness and myotonia. Despite a growing appreciation for the cardiovascular manifestations in myotonic dystrophy type 1 (DM1), cardiac involvement in myotonic dystrophy type 2 (DM2) has been less well characterized. In patients with DM2, cardiomyopathy has rarely been described.Case summary:This case report describes a rare case of DM2 associated cardiomyopathy. A 56-year-old male with DM2 who presented with palpitations and fatigue. Cardiac magnetic resonance (CMR) imaging confirmed a severely enlarged left ventricular cavity with a left ventricular ejection fraction of 28% consistent with severely reduced global systolic function. The lateral wall epicardium exhibited late gadolinium enhancement in a pattern seen in myotonic dystrophy-related cardiomyopathy.Discussion:This case highlights the potential for significant cardiovascular involvement in DM2, as well as the importance of screening, including CMR imaging, and therapy in the myotonic dystrophy patient population.

The overall goal of our previous reports was to emphasize the need to establish dedicated clinics focused on dystrophic cardiomyopathy [1,2]. We believe this is essential as Duchenne (DMD) and Becker muscular dystrophy (BMD) patient populations are underserved as the incidence of cardiomyopathy is nearly universal and results in heart failure progression [1,2]. We also emphasized the need for increased clinical and basic science research funding focused on dystrophic cardiomyopathy, the need for increased education related to this topic at national meetings such as the Heart Failure Society of America (HFSA) Annual Scientific Meeting and the AHA Annual Scientific Sessions and the need for a registry database that will enroll and longitudinally follow all patients with muscular dystrophies [2].