An unexpected but interesting response to a novel therapy for malignant extragastrointestinal stromal tumor of the mesoileum: a case report and review of the literature

BACKGROUND: Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the gastrointestinal tract. Extragastrointestinal stromal tumors (eGISTs) of the mesoileum are extremely rare and are usually treated with surgery combined with imatinib therapy. CASE PRESENTATION: We present the case of a 43-year-old man who developed a large eGIST in the mesoileum. Abdominal/pelvic computed tomography revealed a large heterogeneous mass with cystic and solid components that measured 20.0 × 12.0 × 8.0 cm. Three cycles of neoadjuvant chemotherapy with epirubicin, cyclophosphamide and hydroxycamptothecin; en bloc resection; and three more cycles of adjuvant chemotherapy with the same regimen and drugs resulted in five years of disease-free survival without any symptoms. CONCLUSIONS: Although imatinib treatment is usually chosen for eGISTs, resistance to imatinib remains a concern; these patients may receive neoadjuvant or adjuvant chemotherapy. In case of the former, further treatment, that is, surgery or adjuvant chemotherapy, depends on tumor response to the neoadjuvant chemotherapy. In addition, this treatment for eGIST is not only beneficial but also economical for patients compared with imatinib. A novel treatment approach that combined neoadjuvant chemotherapy, surgery and adjuvant chemotherapy resulted in long-term survival in our patient, thus showing promise as a potential therapy for eGISTs.