Paediatric pulmonary hypertension: monitoring progress and identifying unmet needs

M Beghetti1

  • 1Hôpital des Enfants, Geneva, Switzerland. maurice.beghetti@hcuge.ch

Insights

Effective management of pediatric pulmonary hypertension (PH) requires more research. The TOPP registry and adapted therapies are crucial for addressing unmet needs in childhood PH.

Area of Science:

  • Pediatric Cardiology
  • Pulmonary Medicine
  • Rare Diseases

Background:

  • Pulmonary hypertension (PH) treatment has advanced, yet effective management in children remains challenging.
  • Pediatric PH lacks comprehensive epidemiological data, hindering optimal clinical decision-making.
  • Current treatment protocols for pediatric PH often adapt adult guidelines, necessitating specific pediatric research.

Purpose of the Study:

  • To address the unmet needs in pediatric pulmonary hypertension (PH) management.
  • To highlight the importance of epidemiological data and registries for childhood PH.
  • To discuss the challenges and future directions for pediatric PH therapies.

Main Methods:

  • Review of current understanding and management strategies for pediatric PH.
  • Discussion of the role of the international pediatric PH registry (TOPP).
  • Analysis of treatment adaptations and research gaps in pediatric PH.

Main Results:

  • Epidemiological data for pediatric PH are scarce, emphasizing the need for resources like the TOPP registry.
  • Treatment studies in children are limited, with adult algorithms often adapted.
  • Invasive treatments pose challenges in pediatric populations, and adapted formulations/doses are required.

Conclusions:

  • Further epidemiological and treatment data are essential for pediatric pulmonary hypertension.
  • The TOPP registry offers valuable insights, but research into adapted pediatric therapies is critical.
  • Specialized childhood PH services are recommended for improved diagnosis and management.

Related Concept Videos

Pulmonary Hypertension: Classification and Pathogenesis01:30

Pulmonary Hypertension: Classification and Pathogenesis

Pulmonary hypertension (PH) is a severe health condition in which the mean pulmonary arterial pressure increases to 25 mmHg or more, even when the body is at rest. This high pressure in the blood vessels that transport blood from the heart to the lungs can cause various symptoms, including shortness of breath, can lead to right heart failure, and significantly affect the overall quality of life.
There are various classifications for PH, each relating to different underlying causes and also...
Treatment for Pulmonary Arterial Hypertension: Oxygen Therapy for Respiratory Failure01:16

Treatment for Pulmonary Arterial Hypertension: Oxygen Therapy for Respiratory Failure

Oxygen therapy has emerged as a significant tool in enhancing the quality of life for patients suffering from pulmonary arterial hypertension (PAH). While this therapy has principally been studied on patients with significant hypoxemia, this therapeutic approach helps prevent potential organ damage and can be administered in the comfort of one's home.
Oxygen therapy is vital in increasing and maintaining blood oxygen levels in PAH patients. As a result, it aids in reducing fatigue, improving...
Pharmacokinetics in Pediatric Patients: Drug Metabolism01:24

Pharmacokinetics in Pediatric Patients: Drug Metabolism

In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses a challenge in...
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists01:18

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme (ECE). Of...
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers01:26

Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers

Receptor tyrosine kinase inhibitors (TKIs) and calcium channel blockers (CCBs) are two critical categories of drugs employed in the treatment of pulmonary artery hypertension (PAH). PAH is a disease that causes high blood pressure in the pulmonary arteries, resulting in chest pain, fatigue, and shortness of breath.
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
Pharmacokinetics in Pediatric Patients: Drug Excretion01:26

Pharmacokinetics in Pediatric Patients: Drug Excretion

In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...