Retrospective review of methylprednisolone response in children with TBX4-associated pulmonary hypertension and interstitial lung disease.

This publication is a review of a retrospective study involving 21 children aged ≤18 years with TBX4-associated pulmonary hypertension and interstitial lung disease. The setting was a national centre evaluating pulsed intravenous methylprednisolone against untreated children. Follow-up duration was not reported. Primary outcomes included response using ChILD-EU categories. Secondary outcomes included respiratory severity score, functional class, echocardiographic measures, and NT-proBNP. 7/9 patients demonstrated clear or best response to corticosteroids. Respiratory severity scores improved in 6/9 patients (p=0.02). all on respiratory support achieved partial or complete weaning.

Functional class improved in all with FC III/IV at baseline (p=0.02). Right ventricular function measured by TAPSE z-score improved by +1.65 (p=0.04). NT-proBNP levels normalised. Early improvement was not observed in untreated children. The study compares treated subjects to untreated children regarding these specific clinical metrics. Authors note this is a retrospective study and evidence for ILD-directed therapy in this group is lacking. No significant adverse effects were observed. Serious adverse events and discontinuations were not reported. Further prospective evaluation is warranted. Corticosteroids were associated with meaningful improvements, but causality is not established. Do not overstate association versus causation or surrogate versus clinical outcomes. Certainty of evidence was not reported. Funding or conflicts were not reported.