177Lu-DOTATATE Molecular Radiotherapy for Childhood Neuroblastoma

This study tested the principle that Ga-68-DOTATATE PET/CT may be used to select children with primary refractory or relapsed high-risk neuroblastoma for treatment with Lu-177-DOTATATE and evaluated whether this is a viable therapeutic option for those children. Methods: Between 2008 and 2010, 8 children with relapsed or refractory high-risk neuroblastoma were studied with Ga-68-DOTATATE PET/CT. The criterion of eligibility for Lu-177-DOTATATE therapy was uptake on the diagnostic scan equal to or higher than that of the liver. Results: Of the 8 children imaged, 6 had abnormally high uptake on the Ga-68-DOTATATE PET/CT scan and proceeded to treatment. Patients received 2 or 3 administrations of Lu-177-DOTATATE at a median interval of 9 wk and a median administered activity of 7.3 GBq. Of the 6 children treated, 5 had stable disease by the response evaluation criteria in solid tumors (RECIST). Of these 5 children, 2 had an initial metabolic response and reduction in the size of their lesions, and 1 patient had a persistent partial metabolic response and reduction in size of the lesions on CT, although the disease was stable by RECIST. One had progressive disease. Three children had grade 3 and 1 child had grade 4 thrombocytopenia. No significant renal toxicity has been seen. Conclusion: Ga-68-DOTATATE can be used to image children with neuroblastoma and identify those suitable for molecular radiotherapy with Lu-177-DOTATATE. We have shown, for what is to our knowledge the first time, that treatment with Lu-177-DOTATATE is safe and feasible in children with relapsed or primary refractory high-risk neuroblastoma. We plan to evaluate this approach formally in a phase I-II clinical trial.