ArticleJACC. Case reports2025
Pulmonary Arterial Hypertension After Busulfan Administration During Conditioning Regimen in Neuroblastoma: Key Role of Rescue Treatment.
Article in JACC. Case reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundPulmonary arterial hypertension (PH) is a rare and life-threatening complication of high-dose chemotherapy with busulfan (Bu) used for hematopoietic stem cell transplantation. CASE SUMMARY: A 5-year-old male patient with retroperitoneal neuroblastoma developed PH and acute right-sided heart failure 2 months after conditioning regimen that included Bu and melphalan before autologous hematopoietic stem cell transplantation. Once pulmonary embolism and veno-occlusive disease were excluded by computed tomography, rescue treatment, including epoprostenol, bosentan, and sildenafil, was started, with complete regression of acute right-sided heart failure and normalization of pulmonary pressure. DISCUSSION: Bu can cause PH through different pathophysiological mechanisms. The prompt start of rescue treatment is a cornerstone for a good outcome. In our patient, considering the absence of signs of pulmonary veno-occlusive disease on computed tomography, a triple pulmonary vasodilator therapy was started and a rapid recovery was noted. TAKE-HOME MESSAGE: Early work-up for PH in children after Bu administration is essential to recognize a life-threatening condition requiring a rescue treatment.
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