Recent and Upcoming Drug Therapies for Pediatric Heart Failure

Loss, Karla L.; Shaddy, Robert E.; Kantor, Paul F.

doi:10.3389/fped.2021.681224

Cited by 14 publications

(11 citation statements)

References 91 publications

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“…It is crucial to consider that medications and surgical methods for treating cardiomyopathy have mostly been tried and evaluated in adults. Thus, as also stated in a review by Loss et al [180], pediatric management in general follows the guidelines for adult HF treatment. This is due to the lack of clinical trials with children, which are especially challenging due to difficulties in recruiting an adequate number of probands, high costs, and so on.…”

Section: Treatment Of Pediatric Cardiomyopathymentioning

confidence: 84%

Cardiomyopathies in Children: Genetics, Pathomechanisms and Therapeutic Strategies

Cimiotti¹,

Sadat-Ebrahimi²,

Mügge³

et al. 2024

New Insights on Cardiomyopathy

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Despite great advances in cardiovascular medicine, cardiomyopathies in children still are challenging for pediatricians as well as cardiologists. Pediatric cardiomyopathies can manifest in diverse phenotypes but are often life-threatening and have a poor prognosis. However, many therapeutic options available for adult patients do not apply for children, leaving a very limited portfolio to attenuate disease progression to avoid or postpone heart transplantation. Childhood cardiomyopathies can arise from different etiologies, but genetic defects such as mutations, for example, in sarcomeric proteins, which are pivotal for the contractile function, are common. This leads to the demand to identify new variants found by genetic screening as pathogenic and furthermore to allow a prognosis or risk assessment for related carriers, thus increasing the need to uncover molecular pathomechanisms of such mutations. This chapter aims to highlight the unique characteristics of pediatric cardiomyopathies in contrast to adult forms, including etiology, pathophysiology, genetics, as well as molecular mechanisms. We will also tackle currents options, challenges, and perspectives in diagnosis and treatment of pediatric cardiomyopathies.

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Section: Treatment Of Pediatric Cardiomyopathymentioning

confidence: 84%

Cardiomyopathies in Children: Genetics, Pathomechanisms and Therapeutic Strategies

Cimiotti¹,

Sadat-Ebrahimi²,

Mügge³

et al. 2024

New Insights on Cardiomyopathy

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“…Also, newer medications such as ivabradine and sacubitril/valsartan that have proven effective in the adult population are being introduced in pHF management. Many other similar candidate drugs are undergoing clinical trials [ 15 ].…”

Section: Discussionmentioning

confidence: 99%

Pediatric Heart Failure Inpatient Mortality: A Cross-Sectional Analysis

Adebiyi¹,

Edigin²,

Shaka³

et al. 2022

Cureus

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BackgroundHeart failure constitutes significant morbidity and mortality among the pediatric population. Few data exist on the prevalence and mortality rate of pediatric heart failure (pHF) in the United States. ObjectivesThis study aimed to determine the in-hospital mortality and the principal diagnoses in pediatric patients with heart failure who died while being hospitalized in the United States. MethodsThis is a retrospective cross-sectional study using data from the 2019 Kid Inpatient Database (KID). The KID contained data on hospitalized children below 21 years of age. Using Stata 17 software (StataCorp LLC, College Station, Texas), the data were searched for heart failure diagnoses using International Classification of Diseases 10th revision Clinical Modification (ICD-10-CM) codes. By using the "rank" command in Stata, the most common principal diagnoses were placed in descending order of frequency, and these were further divided into different ICD-10 code categories. ResultsThere were 16,206 pHF admissions in 2019. Of these admissions, 1,023 (6.31%) patients died. The top five principal ICD 10 code categories among all pHF deaths in descending order were circulatory system (17.95%), congenital/chromosomal abnormalities (17.43%), respiratory system (10.28%), infectious diseases (9.24%, and perinatal diseases (7.90%). Among all pHF deaths, sepsis of unspecified organisms (5.14%), hypoplastic left heart syndrome (HLHS) (3.19%), and acute respiratory failure with hypoxia (3.14%) were the most common primary diagnoses. Conclusion and significancePediatric heart failure in-hospital overall mortality is 6.31%, and sepsis of unspecified organisms, HLHS, and acute respiratory failure are the most common principal diagnoses among these children. Preventive measures and prompt treatment of infections are paramount to reducing pHF mortality.

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“…Inotropes are used as rescue therapy in patients with ADHF to prevent end-organ failure by increasing perfusion pressure and allowing diuresis to occur [ 32 , 33 ]. These drugs improve myocardial contractility and increase cardiac output.…”

Section: Acute Decompensated Heart Failurementioning

confidence: 99%

Heart failure in children and adolescents: an update on diagnostic approaches and management

Agrawal,

Janjua,

Alsayed Ali Zeyada

et al. 2024

Clin Exp Pediatr

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Cardiac failure is a clinical syndrome that may develop in children owing to cardiac dysfunction or underlying structural heart diseases. Considering the differences in diagnostic and therapeutic approaches for pediatric heart failure (PHF) and adult heart failure, we have reviewed the current literature on PHF. Relevant studies were extracted from MEDLINE/PubMed, Google Scholar, and Clinical Trial Registries using the terms “pediatric heart failure” or “heart failure in children” and “management” or “decongestive therapy.” Recent advances in diagnostic approaches, such as cardiac magnetic resonance, speckle-tracking echocardiography, tissue Doppler imaging, and molecular diagnostic techniques, have increased our under -standing of PHF. It is imperative that clinicians evaluate the interrelated factors responsible for the develop ment of PHF, including myocardial function, pulmonary and systemic blood flow, heart rhythm, valve function, and nutritional status. Although recent advances have demon strated the efficacy of many new drugs in adult heart failure trials, it cannot be concluded that these drugs will show similar efficacy in children, considering the heterogeneous nature of the underlying mechanisms and variable pharmacody-namics and pharmacokinetics. Therefore, the underlying pathophysiology of PHF and the mechanisms of action of different drugs should be considered when selecting appropriate therapies. Further trials are needed to establi sh the efficacy and safety of these drugs, and a combined mul-ti disciplinary strategy will help enhance PHF outcomes.

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Recent and Upcoming Drug Therapies for Pediatric Heart Failure

Cited by 14 publications

References 91 publications

Cardiomyopathies in Children: Genetics, Pathomechanisms and Therapeutic Strategies

Cardiomyopathies in Children: Genetics, Pathomechanisms and Therapeutic Strategies

Pediatric Heart Failure Inpatient Mortality: A Cross-Sectional Analysis

Heart failure in children and adolescents: an update on diagnostic approaches and management

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