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Therapeutic Approaches in Heart Failure with Preserved Ejection Fraction (HFpEF) in Children: Present and Future
Pediatric Drugs ( IF 3.4 ) Pub Date : 2022-05-02 , DOI: 10.1007/s40272-022-00508-z
Bibhuti B Das 1
Affiliation  

For a long time, pediatric heart failure (HF) with preserved systolic function (HFpEF) has been noted in patients with cardiomyopathies and congenital heart disease. HFpEF is infrequently reported in children and instead of using the HFpEF terminology the HF symptoms are attributed to diastolic dysfunction. Identifying HFpEF in children is challenging because of heterogeneous etiologies and unknown pathophysiological mechanisms. Advances in echocardiography and cardiac magnetic resonance imaging techniques have further increased our understanding of HFpEF in children. However, the literature does not describe the incidence, etiology, clinical features, and treatment of HFpEF in children. At present, treatment of HFpEF in children is extrapolated from clinical trials in adults. There are significant differences between pediatric and adult HF with reduced ejection fraction, supported by a lack of adequate response to adult HF therapies. Evidence-based clinical trials in children are still not available because of the difficulty of conducting trials with a limited number of pediatric patients with HF. The treatment of HFpEF in children is based upon the clinician’s experience, and the majority of children receive off-level medications. There are significant differences between pediatric and adult HFpEF pharmacotherapies in many areas, including side-effect profiles, underlying pathophysiologies, the β-receptor physiology, and pharmacokinetics and pharmacodynamics. This review describes the present and future treatments for children with HFpEF compared with adults. This review also highlights the need to urgently test new therapies in children with HFpEF to demonstrate the safety and efficacy of drugs and devices with proven benefits in adults.



中文翻译:

儿童保留射血分数 (HFpEF) 心力衰竭的治疗方法:现在和未来

长期以来,在患有心肌病和先天性心脏病的患者中发现了具有保留收缩功能 (HFpEF) 的小儿心力衰竭 (HF)。HFpEF 在儿童中很少报告,而不是使用 HFpEF 术语,HF 症状归因于舒张功能障碍。由于异质性病因和未知的病理生理机制,识别儿童 HFpEF 具有挑战性。超声心动图和心脏磁共振成像技术的进步进一步增加了我们对儿童 HFpEF 的了解。然而,文献没有描述儿童 HFpEF 的发病率、病因、临床特征和治疗。目前,儿童 HFpEF 的治疗是从成人临床试验中推断出来的。由于对成人 HF 治疗缺乏足够的反应,射血分数降低的儿童和成人 HF 之间存在显着差异。由于难以对数量有限的 HF 儿科患者进行试验,因此仍然无法进行基于证据的儿童临床试验。儿童 HFpEF 的治疗基于临床医生的经验,大多数儿童接受非水平药物治疗。儿童和成人 HFpEF 药物疗法在许多领域存在显着差异,包括副作用概况、潜在的病理生理学、β受体生理学以及药代动力学和药效学。本综述描述了儿童 HFpEF 与成人相比的当前和未来治疗方法。

更新日期:2022-05-04
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