Patients with relapsed stage 4 neuroblastoma have an extremely poor long-term prognosis, making the investigation of new agents of interest. We report the outcome of the first patient treated in a phase 1 study for relapsed neuroblastoma, using the chemotherapy agent decitabine to upregulate cancer testis antigen expression, followed by a dendritic cell vaccine targeting the cancer testis antigens MAGE-A1, MAGE-A3, and NY-ESO-1. Our patient had persistent tumor in his bone marrow after completion of standard therapy for neuroblastoma, including multiagent chemotherapy, tumor resection, stem cell transplantation, radiation therapy, and anti-GD2 monoclonal antibodies. His marrow disease persisted despite chemotherapy, which was given while the vaccine was being produced. After 3 cycles of decitabine and vaccine, this patient achieved a complete remission and is now 1 year from his last treatment, with no evidence of tumor in his bone marrow or other sites. This patient was noted to have an increase in MAGE-A3-specific T cells. This is the first report combining demethylating chemotherapy to enhance tumor antigen expression followed by a cancer antigen vaccine.
Pulmonary arterial hypertension (PAH) in pediatric patients is associated with significant morbidity and mortality. Few studies exist to evaluate the incidence of PAH in pediatric oncology patients treated with carboplatin and thiotepa followed by hematopoietic stem cell transplant (HSCT). We describe two pediatric patients who developed PAH following high dose chemotherapy with carboplatin and thiotepa followed by autologous HSCT. These cases highlight the need for a surveillance protocol and reinforce the need for cardiologist-oncologists or close collaboration between providers in these sub-specialties who provide care to patients undergoing this type of chemotherapy regimen.
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