Pulmonary Hypertensive Vasculopathy Following Tandem Autologous Transplantation in Pediatric Patients with Central Nervous System Tumors

作者：Schechter Tal^*; Leucht Stephan; Bouffet Eric; Cutz Ernest; Gassas Adam; Huang Annie; Bartels Ute; Humpl Tilman; Doyle John

来源：Biology of Blood and Marrow Transplantation, 2013, 19(2): 235-239.

DOI：10.1016/j.bbmt.2012.09.011

• Pulmonary hypertension
• Children
• Stem-Cell Transplantation

Tandem cycles of high-dose chemotherapy are an increasingly being used as alternative to radiation treatment in the management of infants and young children with central nervous system (CNS) tumors. Most of these protocols have a carboplatinum and thiotepa backbone. The toxicities of these regimens have been reported extensively; however, pulmonary arterial vasculopathy (PAV) with pulmonary arterial hypertension (PAH) has not been previously documented in patients treated with this approach. PAH is a disorder of the pulmonary vasculature characterized by a progressive increase in pulmonary vascular resistance, leading to right heart failure and potentially death. We evaluated PAH as a complication after high-dose chemotherapy and autologous stem cell transplantation (SCT). We performed a retrospective evaluation of all pediatric patients diagnosed with a CNS tumor between 2001 and 2010 scheduled to receive 3 cycles of high-dose chemotherapy with carboplatinum (17 mg/kd/day for 2 days) and thiotepa (10 mg/kg/day for 2 days), followed by autologous SCT. Our primary objective was to evaluate the incidence of PAV and PAH in this population, as well as patient outcomes after the development of PAH. Our cohort comprised 20 patients with a median age at diagnosis of 28 months (range, 3-131 months). Three patients developed biopsy-confirmed PAV with PAH, the 2 patients who developed PAV with PAH at the end of the third cycle succumbed to PAH. Death due to PAV and PAH was the sole toxic mortality observed during the study period. PAV with PAH is a major and possibly fatal complication after high-dose chemotherapy and sequential autologous SCT using carboplatinum and thiotepa in a tandem fashion. There is an urgent need to evaluate PAH as' a potential complication after each cycle of high-dose chemotherapy when using such regimens in pediatric patients with CNS tumors.

• 出版日期2013-2