Selexipag for the treatment of children with pulmonary arterial hypertension: First multicenter experience in drug safety and efficacy

Hansmann, Georg; Meinel, Katharina; Bukova, Mila; Chouvarine, Philippe; Wåhlander, Håkan; Koestenberger, Martin

doi:10.1016/j.healun.2020.03.029

Cited by 38 publications

(45 citation statements)

References 23 publications

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“…However, longitudinal assessment of NT-proBNP in combination with echocardiography is very useful to assess disease progression and/or response to treatment. [41][42][43][44] Meanwhile, additional biomarkers are under consideration and may contribute to future PH management. 12,40 TREATMENT Supportive measures Supplemental oxygen should be supplied when target oxygen saturations are >93% for infants with suspected and >95% for infants with proven PH (Fig.…”

Section: Diagnosis Of Bpd and Bpd-phmentioning

confidence: 99%

“…The first prospective, pediatric multicenter study on PAH-combination therapy indicates that selexipag can be used safely even in small children (4-10 kg bodyweight), and that a positive drug response can be expected in half of the children treated with selexipag. 43 Interventional and surgical procedures In children with BPD, large left-to-right atrial or ventricular shunts are not well tolerated, and may be closed early if PVD is mild (i.e., no or only mild PVR elevation at cardiac catheterization). 59 However, aorto-pulmonary connections (e.g., a patent ductus arteriosus, PDA) may facilitate decompressing a pressure-loaded RV in severe PH/PVD.…”

Section: Pharmacotherapymentioning

confidence: 99%

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Pulmonary hypertension in bronchopulmonary dysplasia

et al. 2020

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Bronchopulmonary dysplasia (BPD) is a major complication in prematurely born infants. Pulmonary hypertension (PH) associated with BPD (BPD-PH) is characterized by alveolar diffusion impairment, abnormal vascular remodeling, and rarefication of pulmonary vessels (vascular growth arrest), which lead to increased pulmonary vascular resistance and right heart failure. About 25% of infants with moderate to severe BPD develop BPD-PH that is associated with high morbidity and mortality. The recent evolution of broader PH-targeted pharmacotherapy in adults has opened up new treatment options for infants with BPD-PH. Sildenafil became the mainstay of contemporary BPD-PH therapy. Additional medications, such as endothelin receptor antagonists and prostacyclin analogs/mimetics, are increasingly being investigated in infants with PH. However, pediatric data from prospective or randomized controlled trials are still sparse. We discuss comprehensive diagnostic and therapeutic strategies for BPD-PH and briefly review the relevant differential diagnoses of parenchymal and interstitial developmental lung diseases. In addition, we provide a practical framework for the management of children with BPD-PH, incorporating the modified definition and classification of pediatric PH from the 2018 World Symposium on Pulmonary Hypertension, and the 2019 EPPVDN consensus recommendations on established and newly developed therapeutic strategies. Finally, current gaps of knowledge and future research directions are discussed.

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Section: Diagnosis Of Bpd and Bpd-phmentioning

confidence: 99%

Section: Pharmacotherapymentioning

confidence: 99%

Pulmonary hypertension in bronchopulmonary dysplasia

et al. 2020

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“…Limited data exist in children; no relevant side effects were reported (78,79). Only recently, selexipag was used in a prospective observational study as add on therapy in 15 children with PAH with variable efficacy improving outcome-relevant variables in about 50% of patients (25). Another small study in 10 patients reported at least improved clinical parameters after switching from IV treprostinil to PO selexipag (80).…”

Section: Prostacyclin Receptor Agonistmentioning

confidence: 99%

Treatment of pulmonary arterial hypertension in children

Gorenflo

Ziesenitz

2021

Cardiovasc Diagn Ther

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Pulmonary arterial hypertension (PAH) is a devastating illness causing already significant morbidity in childhood. Currently approved treatment options for children comprise the endothelin receptor antagonist bosentan, as well as the phosphodiesterase-5 inhibitor sildenafil. But PAH treatment has advanced significantly over the past decade, and new classes of targeted drug therapies, such as stimulators of the soluble guanylate cyclase (riociguat) or prostacyclin receptor agonists (selexipag), are currently evaluated regarding their efficacy and safety in children, in order to limit off-label use. Due to the different etiologies in children, such as PAH-CHD, there is no evidence that initial combination therapy in children is superior to a mono-therapy with respect to survival. Special attention should also be paid to the pharmacology of PAH drugs in children, which might be impacted by ontogeny or drug-drug-interactions. Therapeutic drug monitoring may be useful in pediatric patients. There is a clear need for more controlled studies of PAH medications, alone or in combination therapy in the pediatric age group. Data from clinical trials as well as from patient registries should be pooled to optimize drug development and evaluation, trial design, and evidence-based pharmacotherapy in pediatric patients with PAH. In this review, the current treatment options of pediatric PAH are summarized, and an overview of new treatment concepts, which are already evaluated in adults, is presented.

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“…While selexipag demonstrated improvements in invasive hemodynamic parameters, RV systolic function, and functional class from baseline, this was not seen uniformly across all patients, with patients who were less sick at baseline demonstrating better drug response to selexipag. Fifty percent of the patients improved, while 25% were stabilized, and 20% deteriorated (two patients proceeded to lung transplant, and one later died after being transitioned to IV treprostinil) 81 …”

Section: Newer Pah Medicationsmentioning

confidence: 99%

Opportunities and challenges of pharmacotherapy for pulmonary arterial hypertension in children

Kam

Ruiz

2020

Pediatric Pulmonology

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Pediatric pulmonary hypertension (PAH) is a rare disease that carries a poor prognosis if left untreated. Although there are published guidelines for the treatment of children with pulmonary hypertension, due to the limited number of robust pediatric clinical trials, recommendations are often based on limited data or clinical experience. Furthermore, many practical aspects of care, particularly for the pediatric patient, are learned through experience and best navigated with a multidisciplinary team. While newer PAH therapies have been approved for adults, there is still limited but expanding experience in pediatrics. This new information will help improve the targets of goal‐oriented therapy. Lastly, this review highlights practical aspects in the use of the different therapies available for the treatment of pediatric pulmonary hypertension.

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Selexipag for the treatment of children with pulmonary arterial hypertension: First multicenter experience in drug safety and efficacy

Cited by 38 publications

References 23 publications

Pulmonary hypertension in bronchopulmonary dysplasia

Pulmonary hypertension in bronchopulmonary dysplasia

Treatment of pulmonary arterial hypertension in children

Opportunities and challenges of pharmacotherapy for pulmonary arterial hypertension in children

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