Department of Pediatrics, Division of Cardiology, University of Mississippi Medical Center, 2500 N State St., Jackson, MS, 39216, USA.
Paediatr Drugs. 2022 May;24(3):235-246. doi: 10.1007/s40272-022-00508-z. Epub 2022 May 2.
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.
很长一段时间以来，人们一直注意到患有心肌病和先天性心脏病的患者存在收缩功能保留的儿科心力衰竭（HF）（HFpEF）。HFpEF 在儿童中很少见，HF 症状不是归因于 HFpEF，而是归因于舒张功能障碍。由于病因异质性和未知的病理生理机制，儿童 HFpEF 的识别具有挑战性。超声心动图和心脏磁共振成像技术的进步进一步提高了我们对儿童 HFpEF 的认识。然而，文献并未描述儿童 HFpEF 的发病率、病因、临床特征和治疗方法。目前，儿童 HFpEF 的治疗是从成人临床试验中推断出来的。儿科与成人射血分数降低性心力衰竭之间存在显著差异，这与成人 HF 治疗的反应不足有关。由于儿童 HF 患者数量有限，进行试验存在困难，因此仍然缺乏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，大多数儿童接受的是超适应证药物治疗。儿科和成人 HFpEF 药物治疗在许多方面存在显著差异，包括副作用谱、潜在病理生理学、β受体生理学以及药代动力学和药效学。本综述描述了与成人相比，目前和未来针对儿童 HFpEF 的治疗方法。本综述还强调了迫切需要在患有 HFpEF 的儿童中测试新疗法，以证明药物和器械的安全性和有效性，这些药物和器械在成人中已被证明具有益处。
