Researchers reviewed records of 21 children aged 18 or younger who had TBX4-associated pulmonary hypertension at a national center. Nineteen of these children received pulsed intravenous methylprednisolone, while the records of two untreated children were also examined for comparison.
The children who received the steroid treatment showed significant improvement. About 78% had a clear or best response, and those with severe breathing issues or lower functional class improved. Measures of heart function and a marker of heart strain also normalized or improved in the treated group. Children on breathing support were able to wean off it.
In contrast, untreated children did not show early improvement. No significant adverse effects were observed in the treated group, and the medication was generally well tolerated. Despite these positive findings, the study design limits what can be concluded. The evidence for using this therapy specifically for interstitial lung disease in this group is currently lacking. A prospective evaluation is warranted before this approach becomes standard practice.