Effective Use of Sirolimus and Zoledronic Acid for Multiosteotic Pseudomyogenic Hemangioendothelioma of the Bone in a Child: Case Report and Review of Literature.

摘要: Pseudomyogenic hemangioendothelioma (PMH) is a rare neoplasm with vascular and sarcomatous elements, unpredictable course, uncommon metastatic or fatal potential. Although systemic chemotherapy has been reported variable success, generally accepted treatment aggressive surgery wide margins. Evidence-based options are lacking, lack of clear prognostic features poses risk undertreatment overtreatment associated morbidity mortality. We report the use initial therapy oral sirolimus (SIR) IV zoledronic acid (ZA) to induce sustained clinical response avoidance amputation in 6-year-old boy. At 37 months after diagnosis, our patient remains remission as documented by x-ray, MRI, PET-CT return normal mobility/activity resolution swelling pain. Literature review identified 20 cases pediatric young adult patients PMH, which 7 received some form therapy. To best knowledge, represents youngest case PMH first successful limb-sparing utilization primary for PMH.