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document
Effectiveness and safety of inhaled treprostinil for the treatment of pulmonary arterial hypertension in children . The introduction of prostanoid therapy has revolutionized the treatment of pulmonary arterial hypertension ( PAH ) . However , continuous intravenous prostacyclin infusion poses significant risks and challenges , particularly in children . Inhaled treprostinil has been shown to be safe and efficacious in adults . This study describes the safety and efficacy of inhaled treprostinil in children with PAH . A retrospective analysis of 29 children treated with inhaled treprostinil for ≥6 weeks was performed . Effects of inhaled treprostinil on exercise capacity , functional class , and echocardiographic and hemodynamic data were evaluated . Adverse events were documented . Patients received 3 to 9 breaths ( 6 μg/breath ) of inhaled treprostinil 4 times/day . All were receiving background PAH therapy ; 12 had previously received parenteral prostanoid . Inhaled treprostinil was discontinued in 4 patients because of symptoms including cough and bronchospasm ( n = 3 ) and progression of PAH ( n = 1 ) . Mild side effects including cough ( n = 9 ) and sore throat ( n = 6 ) did not require discontinuation of therapy . World Health Organization functional class improved in 19 and was unchanged in 10 ; exercise capacity significantly improved with the 6-minute walk distance , improving on follow-up from 455.7 ± 71.5 to 498 ± 70 m ( p = 0.01 ) and peak oxygen consumption increasing from 25.5 ± 10.2 to 27.4 ± 10 ( p = 0.04 ) . In conclusion , inhaled treprostinil was associated with improvement in exercise capacity and World Health Organization functional class when added to background targeted PAH therapy in children and had an acceptable safety profile . Based on these early data , further study of inhaled treprostinil appears warranted in pediatric patients with PAH .
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Intervention_Pharmacological
treprostinil
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Intervention_Pharmacological
prostanoid therapy
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Intervention_Pharmacological
prostacyclin
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Intervention_Pharmacological
treprostinil
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Intervention_Pharmacological
treprostinil
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Intervention_Pharmacological
treprostinil
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Outcome_Adverse-effects
cough and bronchospasm
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Outcome_Physical
World Health Organization functional class
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Outcome_Physical
exercise capacity
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Outcome_Physical
peak oxygen consumption
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Participant_Condition
pulmonary arterial hypertension in children .
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Participant_Age
children .
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Participant_Age
children with PAH .