RESUMO
BACKGROUND: Embryonal tumor with multilayer rosettes (ETMR) is a rare CNS malignancy affecting young children that carries a very poor prognosis. Treatment with intensive surgical resection, radiotherapy, and high-dose chemotherapy is insufficient treatment in the vast majority of cases. Effective, biologically based therapies for this tumor are therefore desperately needed. The Dana-Farber Cancer Institute-modified IRS-III protocol incorporates preclinically active agents, such as doxorubicin and actinomycin D, into the treatment regimen for ETMR and may improve patient outcomes. METHODS: The authors present a case series of 5 children with ETMR treated with an IRS-III-based chemotherapy backbone. RESULTS: All 5 patients received a gross-total tumor resection. Patients received between 12 and 51 weeks of IRS-III therapy at the discretion of their treating physician. Four patients received focal radiation therapy, with the fifth patient instead receiving a cycle of high-dose chemotherapy with autologous stem cell rescue. Four patients have progression-free survival of more than 18 months. Chemotherapy treatment was reasonably tolerated by all 5 patients with one case of mild sinusoidal obstructive syndrome and one case of Grade 3 peripheral neuropathy. CONCLUSIONS: The patient outcomes in this small cohort are far better than would be expected based on the historical survival for this tumor. Given the tremendous need for effective therapy for ETMR, further investigation of this approach is warranted. An international consensus protocol based on the IRS-III regimen has been developed and will be available through a multicenter clinical trial and a global treatment registry.
RESUMO
Introduction: The techniques used to fixate osteotomized segments of bone have evolved alongside the treatment of craniosynostosis. The use of nonresorbable metal plates and screws offered a method of rigidly stabilizing repositioned segments of bone. Several reports specify the tendency for these fixation systems to "migrate" transcranially. Methods: We present a unique case of a patient who initially underwent treatment of multisuture craniosynostosis utilizing titanium miniplates at 6 months of age. At 16 years of age, the patient was returned to the operating room with complaints of pain and contour irregularities, and intracranial migration of the screws and plates was observed. Results: The hardware was extracted and the cranium reconstructed. Symptoms resolved and bony contour was improved. Conclusion: The craniofacial surgeon considering metal plate fixation in the pediatric population should be aware of the possibility for transcranial plate and screw migration.
