Pilot trial of a novel two-step therapy protocol using nebulized tranexamic acid and recombinant factor VIIa in children with intractable diffuse alveolar hemorrhage

Hind Bafaqih, May Chehab, Suliman Almohaimeed, Farah Thabet, Abdulrahman Alhejaily, Mohammed AlShahrani, Mohammed A Zolaly, Abeer A Abdelmoneim, Ehab S Abd
Annals of Saudi Medicine 2015, 35 (3): 231-9

BACKGROUND AND OBJECTIVES: Diffuse alveolar hemorrhage (DAH) is a life threatening condition with very limited, often unsuccessful, therapeutic options. This study aimed at exploring the feasibility and efficacy of nebulized tranexamic acid TXA (n-TXA) and nebulized recombinant factor VIIa (n-rFVIIa) when used in a two-step therapy protocol in children with intractable DAH in a pediatric intensive care unit.

METHODS: In a prospective trial, n-TXA (250 mg/dose for children < 25 kg and 500 mg/dose for children > 25 kg) was administered to 18 children (median age [interquartile range]; 24.0 months [11.3, 58.5]) with intractable DAH. N-rFVIIa (35 micro g/kg/dose for children < 25 kg, and 50 micro g/kg/dose for children > 25 kg) was added if no or minimal response was seen after 3 to 4 doses (18 to 24 hours) of n-TXA.

RESULTS: DAH was stopped in 10 (55.6%) children with n-TXA alone within 24 hours of therapy. Documented concomitant respiratory infection showed a significant negative association with response to n-TXA in a step.wise regression analysis (OR=0.06; 95% CI=0.01-0.74). In the other 8 (44.4%) children, n-rFVIIa was added due to n-TXA failure. Six (75.0%) showed complete cessation of DAH, while two children failed to respond with the addition of n-rFVIIa (25.0%). None of the children who responded to therapy showed recurrence of DAH after therapy termination. No complications related to therapy were recorded.

CONCLUSIONS: n-TXA and n-rFVIIa were effective and safe when used in a two-step-therapy protocol to control intractable DAH in pediatric patients in intensive care settings. This therapy modality warrants further exploration through larger multicenter clinical trials.

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