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Treatment Patterns in Pediatric Pulmonary Hypertension (PH) - Results from the First Pediatric Global Registry: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP)

Tilman Humpl

Robyn Barst


R. A. Kronmal

R. M. F. Berger


Donald Moore

Fasnacht Boillat

Ingram Schulze-Neick


Maurice Beghetti


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Conference: 2012 International PHA Conference and Scientific Sessions

Release Date: 06.22.2012

Presentation Type: Abstracts

Humpl T1; Barst RJ2; Kronmal RA3; Berger RMF4; Moore DJ5; Fasnacht Boillat MS6; Schulze-Neick I8; Beghetti M9

1. Hospital for Sick Children, Toronto, Canada
2. Columbia University College of Physicians and Surgeons, New York, United States
3. University of Washington, Seattle, United States
4. Beatrix Children's Hospital, Groningen, Netherlands
5. Vanderbild Children´s Hospital, Nashville, United States
6. Universitäts-Kinderklinik, Zürich, Switzerland
7. Children´s Memorial Health Institute, Warszawa, Poland
8. Great Ormond Street Hospital, London, United Kingdom
9. Children's University Hospital, Geneva, Switzerland.

BACKGROUNDThere is limited data on pediatric PH. The TOPP registry (31 sites, 19 countries) was designed to provide current demographic, clinical and outcome data in pediatric PH (diagnosis on/after January 2001, enrollment 2008-2010). One of the primary objectives was to describe current treatment.

METHODSPatients diagnosed with PH and increased pulmonary vascular resistance (by right heart catheterization, RHC) between 3 m and 18 y were eligible for enrollment. Diagnostic methods and treatment decisions were made by site clinicians without TOPP guidance.

PH targeted therapy (PHTT) included: prostacyclin and its analogs (PGI2s), endothelin receptor antagonists (ERAs), phosphodiesterase inhibitors type 5 (PDE5Is); calcium channel blockers (CCBs) were considered as PHTT in responders to acute vasodilator testing. Supportive therapy (ST) included: anticoagulation, oxygen, diuretics and/or digitalis.

RESULTSA total of 456 patients were enrolled (n=135 incident patients, IP: diagnostic RHC <3 m prior to enrollment; n=321 prevalent patients, PP: RHC >3 m prior to enrollment). At diagnosis 10% (n=14) of IP were on PHTT, 73% (n=98) were treatment nave, 17% (n=23) had incomplete data regarding treatment; data was similar for PP: 9% (n=29) on PHTT, 77% (n=246) treatment nave and 14% (n=46) incomplete data. Treatment with PDE5Is: 44% (n=60) IP and 37% (n=118) PP, with ERAs: 23% (n=31) IP and 34% (n=108) PP, with: PGI2s 13% (n=17) IP and 15% (n=48) PP. CCBs for PHTT were used in 10% (n=13) IP and 12% (n=38) PP.

With respect to ST, 16% (n=21) IP and 31% (n=100) PP received anticoagulation; 22% (n=29) IP and 32% (n=104) PP oxygen; 34% (n=46) IP and 31% (n= 98) PP diuretics; 23% (n=31) IP and 57% (n=18) PP digitalis. Of IP, 70% started on PHTT monotherapy at diagnosis and 14% started dual therapy. Similarly, 68% of PP started monotherapy at diagnosis, 14% dual therapy and 1% triple therapy.

CONCLUSIONSThe largest proportion of pediatric PH patients enrolled in TOPP (regardless of whether IP or PP) start on PDE5Is (as monotherapy) at diagnosis. Long-term follow-up should provide invaluable data on how treatment patterns affect outcomes.