Marilyne Levy1, Maria-Jesus Del Cerro2, Sophie Nadaud3, Karunakar Vadlamudi4, Elizabeth Colgazier5, Jeff Fineman5, Damien Bonnet6, Ian Adatia7. 1. Université Paris Descartes, Paris, France and M3C-Necker, AP-HP, Hôpital Necker-Enfants Malades, Université Paris Descartes, Sorbonne Paris Cité, Paris, France. Electronic address: marilyne.levy@ue3c.fr. 2. Ramon y Cajal University Hospital, Madrid, Spain. 3. Sorbonne Universités, UPMC Univ Paris 06, INSERM, UMR S 1166-ICAN, F-75013 Paris, France. 4. Stollery Children's Hospital, University of Alberta, Edmonton, Canada. 5. Benioff Children's Hospital and University of California at San Francisco, San Francisco, CA, USA. 6. Université Paris Descartes, Paris, France and M3C-Necker, AP-HP, Hôpital Necker-Enfants Malades, Université Paris Descartes, Sorbonne Paris Cité, Paris, France. 7. Stollery Children's Hospital, University of Alberta, Edmonton, Canada; Glenwood Children's Heart Center and Echocardiography Laboratory, Edmonton, Alberta, Canada.
Abstract
BACKGROUND: Continuous intravenous epoprostenol was the first treatment approved for pulmonary arterial hypertension (PAH) but administration through a central venous line carries risks of thrombosis and sepsis, particularly in children. We sought to evaluate the safety, efficacy and management of subcutaneous (SC) treprostinil in children with PAH. METHODS: Fifty-six children (median age 65, range 1-200 months) were treated with SC treprostinil. Clinical status, echocardiography, NT-proBNP, and site pain and infection were evaluated. Right heart catheterization was performed in 54 patients before starting SC treprostinil infusion and was repeated at 6 months in 31 patients. RESULTS: Treatment was well tolerated in 79% of patients. Site pain resistant to simple analgesics occurred in 12 patients (21%), but could be managed in 9/12 children. At 6 months, 3 patients had died, 4 had received a Potts shunt and 1 underwent lung transplantation. Among the 48 treated patients, 40 (83%) showed significant improvement in WHO functional class, 6 minute walk distance, NT-proBNP and pulmonary vascular resistance (p < 0.01 for all parameters). At last follow-up (median 37 months), ten patients had died, 2 underwent a lung transplantation and 8 underwent a Potts shunt. In 30 of the 36 remaining treated patients, improvement of clinical status was sustained. No children developed sepsis and 12 had minor site infections. CONCLUSION: Subcutaneous treprostinil infusion is an effective therapy without serious side effects in children with PAH. Site pain can be managed with simple analgesics in most children.
BACKGROUND: Continuous intravenous epoprostenol was the first treatment approved for pulmonary arterial hypertension (PAH) but administration through a central venous line carries risks of thrombosis and sepsis, particularly in children. We sought to evaluate the safety, efficacy and management of subcutaneous (SC) treprostinil in children with PAH. METHODS: Fifty-six children (median age 65, range 1-200 months) were treated with SC treprostinil. Clinical status, echocardiography, NT-proBNP, and site pain and infection were evaluated. Right heart catheterization was performed in 54 patients before starting SC treprostinil infusion and was repeated at 6 months in 31 patients. RESULTS: Treatment was well tolerated in 79% of patients. Site pain resistant to simple analgesics occurred in 12 patients (21%), but could be managed in 9/12 children. At 6 months, 3 patients had died, 4 had received a Potts shunt and 1 underwent lung transplantation. Among the 48 treated patients, 40 (83%) showed significant improvement in WHO functional class, 6 minute walk distance, NT-proBNP and pulmonary vascular resistance (p < 0.01 for all parameters). At last follow-up (median 37 months), ten patients had died, 2 underwent a lung transplantation and 8 underwent a Potts shunt. In 30 of the 36 remaining treated patients, improvement of clinical status was sustained. No children developed sepsis and 12 had minor site infections. CONCLUSION: Subcutaneous treprostinil infusion is an effective therapy without serious side effects in children with PAH. Site pain can be managed with simple analgesics in most children.
Authors: Joseph B Tella; Thomas J Kulik; Julia E McSweeney; Lynn A Sleeper; Minmin Lu; Mary P Mullen Journal: Pulm Circ Date: 2020-12-07 Impact factor: 3.017
Authors: Erika B Rosenzweig; Steven H Abman; Ian Adatia; Maurice Beghetti; Damien Bonnet; Sheila Haworth; D Dunbar Ivy; Rolf M F Berger Journal: Eur Respir J Date: 2019-01-24 Impact factor: 16.671
Authors: Johannes M Douwes; Willemijn M H Zijlstra; Erika B Rosenzweig; Mark-Jan Ploegstra; Usha S Krishnan; Meindina G Haarman; Marcus T R Roofthooft; Douwe Postmus; Hans L Hillege; D Dunbar Ivy; Rolf M F Berger Journal: Ann Am Thorac Soc Date: 2022-02