Pulmonary Hypertension in Pediatric Patients with Venous Occlusive Disease after High-Dose Chemotherapy: A Case Study and Treatment Approach

Background: Pulmonary venous-occlusive disease (PH) is a life-threatening complication in pediatric patients, rarely arising secondary to venous occlusive disease (VOD) following high-dose chemotherapy. This study examines the progression and treatment outcomes of a 4-year-old patient with PH, complicated by VOD affecting both the lungs and liver.

Methods: The patient, diagnosed with stage IV neuroblastoma, developed respiratory insufficiency and severe PH secondary to VOD in both the lungs and liver, following high-dose chemotherapy and stem cell transplantation. The treatment protocol included noninvasive ventilation (NIV), inhaled nitric oxide (NO) therapy, and a combination of pulmonary vasodilators—sildenafil, ilomedin, and macitentan. Additionally, defibrotide therapy was initiated to address the VOD in both the lungs and liver. Echocardiographic monitoring was conducted regularly to assess right ventricular pressure (RVP) and guide therapy adjustments.

Results: Initial echocardiography revealed severe PH with RVP nearing systemic levels (three-fourths systemic pressure). The patient’s condition required escalating therapies, including NO, prostacyclin inhalation, and vasodilator infusions. Defibrotide treatment was critical in managing the VOD affecting the lungs and liver, improving the patient’s overall condition. By the end of treatment, a significant reduction in RVP and pulmonary pressure was achieved, allowing successful weaning from ventilatory support.

Conclusion: This case underscores the importance of a comprehensive, multifaceted approach in treating PH secondary to multiorgan VOD. Combining pulmonary vasodilators, NO therapy, respiratory support, and defibrotide to treat VOD in the lungs and liver improved the patient’s clinical outcome. Regular echocardiographic assessments were essential for guiding treatment, ultimately leading to stabilization of pulmonary pressures and resolution of VOD-related complications.

Publication History

Article published online:
11 February 2025