Outcomes in children with idiopathic pulmonary arterial hypertension.
BACKGROUND
Treatment for idiopathic pulmonary arterial hypertension in children includes calcium channel blockade (CCB) for acute responders with vasodilator testing and chronic epoprostenol for nonresponders. We sought to determine parameters associated with survival and treatment success.


RESULTS
A previously identified cohort of 77 children diagnosed between 1982 and 1995 with idiopathic pulmonary arterial hypertension was followed up through 2002. For acute responders treated with CCB (n=31), survival at 1, 5, and 10 years was 97%, 97%, and 81%, respectively; treatment success was 84%, 68%, and 47%, respectively. Survival for all children treated with epoprostenol (n=35) at 1, 5, and 10 years was 94%, 81%, and 61%, respectively; treatment success was 83%, 57%, and 37%, respectively. Because of the inconsistent availability of epoprostenol before 1995, we defined a "recent medical era" subset by excluding children from the total 77 patient cohort for whom epoprostenol was recommended but was unavailable. Survival in the recent medical era (n=44) at 1, 5, and 10 years was 97%, 97%, and 78%; treatment success was 93%, 86%, and 60%, respectively. Treatment success on CCB decreased significantly when acute responders became nonresponders. Age at diagnosis predicted treatment success in the recent medical era.


CONCLUSIONS
Survival for children with idiopathic pulmonary arterial hypertension has significantly improved with CCB and epoprostenol. Children who are acute responders are treated with CCB; they are treated with epoprostenol if they become nonresponders. The decrease in survival and in treatment success after 5 years in all children supports the role for transplant evaluation before treatment failure.
Department of Pediatrics, Columbia University College of Physicians & Surgeons, New York, NY 10032, USA.
Delphine
D
Yung
Allison C
AC
Widlitz
Erika Berman
EB
Rosenzweig
Diane
D
Kerstein
Greg
G
Maislin
Robyn J
RJ
Barst
D000293:Adolescent; D000925:Anticoagulants / Q000627:therapeutic use; D002121:Calcium Channel Blockers / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004351:Drug Resistance; D004359:Drug Therapy, Combination; D011464:Epoprostenol / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006346:Heart Septum / Q000601:surgery; D016027:Heart Transplantation / Q000706:statistics & numerical data; D006439:Hemodynamics / Q000187:drug effects; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy* / Q000401:mortality / Q000601:surgery; D007223:Infant; D015993:Life Tables; D008297:Male; D009026:Mortality / Q000639:trends; D011379:Prognosis; D016019:Survival Analysis; D015996:Survival Rate; D016896:Treatment Outcome; D014665:Vasodilator Agents / Q000627:therapeutic use*
D000925:Anticoagulants; D002121:Calcium Channel Blockers; D014665:Vasodilator Agents; D011464:Epoprostenol