Patients with pulmonary arterial hypertension (PAH) and iron deficiency in the absence of anemia who received iron supplements did not show significant clinical benefit at 12 weeks, according to recent study results published in the Annals of the American Thoracic Society.

Patients with PAH and iron deficiency in the absence of anemia received parenteral iron supplementation in the treatment groups in 2 randomized, double-blind, placebo-controlled 12-week crossover studies ( Identifier: NCT01447628). In Europe, patients were randomly assigned 1:1 to a single infusion of ferric carboxymaltose 1000 mg or saline given over the course of 15 minutes, with cross over after 12 weeks. Meanwhile, in China, patients received iron dextran 20 mg iron/kg body weight or saline. The studies were analyzed separately and combined.

Outcomes of interest included changes in iron status as well as exercise capacity (measured via cardiopulmonary exercise testing or 6-minute walk test) and cardiopulmonary hemodynamics (measured via right heart catheterization, cardiac magnetic resonance or plasma N terminal pro-brain natriuretic peptide [NT-proBNP]) at 12 weeks.

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Among the 39 patients in Europe and 17 patients in China in the studies, supplementation with iron led to increases in blood ferritin levels. Furthermore, the treatment was well tolerated, and no significant side effects were reported. However, no significant clinical benefit was seen in either study for exercise capacity or cardiopulmonary hemodynamics at 12 weeks.

“Iron replacement in the absence of overt anaemia has no clinically significant impact on markers of disease severity or quality of life at 12 weeks,” the study authors wrote. “Patients with iron deficiency should not be denied iron replacement at the discretion of the physician but this report should temper the expectation of therapeutic benefit from iron replacement in PAH patients with iron deficiency in the absence of anaemia.”

Disclosure: A product used in the study was donated by a pharmaceutical company.


Howard LSGE, He J, Watson GMJ, et al. Supplementation with iron in pulmonary arterial hypertension: two randomized crossover trials. Ann Am Thorac Soc. Published online March 18, 2021. doi:10.1513/AnnalsATS.202009-1131OC