Pexidartinib Upfront in a Case of Tenosynovial Giant Cell Tumor: Proof of Concept for a Treatment Paradigm Shift.

BACKGROUND: Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive tumor of the joints, bursa, and tendon sheath that can cause considerable pain and substantial morbidity. Although surgery is the primary treatment for patients with TGCT, surgical resection is associated with high rates of recurrence, particularly for patients with diffuse TGCT. Pexidartinib, a colony-stimulating factor 1 receptor inhibitor, is approved by the US Food and Drug Administration for the treatment of adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. CASE DESCRIPTION: A 32-year-old man presented with intra-articular diffuse TGCT with pain and received noncurative treatment for 5 years (2014-2019). In 2019, the patient was found to have extensive disease accompanied by pain and limited range of motion. The patient's case was presented to a sarcoma multidisciplinary tumor board, who determined that surgery would cause significant morbidity and macroscopic residual tumor. As a result of the extent of disease, young age, and otherwise good health, treatment with pexidartinib was started through a compassionate use program at 800 mg/day. After dose reductions to pexidartinib at 400 mg/day and then 200 mg/day as a result of creatine phosphokinase elevations, the patient achieved a complete response after 2 years of treatment; pain was reduced and mobility was restored. The patient reported no side effects related to pexidartinib treatment. Treatment was stopped in 2022 for future family planning. After pexidartinib therapy was interrupted, the patient's wife had a successful pregnancy and delivery; however, the disease showed a slow but constant clinical deterioration, with a reduction in the range of movement of the affected knee and an apparent increase in widespread TGCT nodules. CONCLUSION: Our case is unique because it provides support for pexidartinib use as upfront therapy for TGCT, instead of surgery, in selected cases.

Transpedicular Contrast-enhanced CT-guided biopsy of the body and dens of the axis avoiding the trans-oral approach: Technical report and literature review.

This technical report illustrates the technique to perform computed tomography (CT)-guided bone biopsies in the body and dens of the axis (C2 vertebra) through a posterior transpedicular approach with the use of preoperative contrast-enhanced scans to highlight the course of the vertebral artery. The technique is presented through two exemplification cases: a pediatric patient with osteoblastoma and secondary aneurysmal bone cyst and one adult patient with melanoma metastasis. This case highlights the potential of the CT-guided posterolateral/transpedicular approach for performing safe and effective biopsies in the body and dens of C2, even in pediatric patients.

Electrochemotherapy in Aggressive Hemangioma of the Spine: A Case Series and Narrative Literature Review.

(1) Background: this case series and literature review aims to evaluate the efficacy and safety of electrochemotherapy in the management of aggressive spinal hemangiomas, presenting two distinct cases. (2) Methods: we present two cases of spinal aggressive hemangioma which were refractory to conventional treatments and underwent electrochemotherapy. Case 1 involves a 50-year-old female who presented with an aggressive spinal hemangioma of L1, who previously underwent various treatments including surgery, radio-chemotherapy, and arterial embolization. Case 2 describes a 16-year-old female with a T12 vertebral hemangioma, previously treated with surgery and stabilization, who faced limitations in treatment options due to her young age and the location of the hemangioma. (3) Results: in Case 1, electrochemotherapy with bleomycin was administered following the failure of previous treatments and resulted in the reduction of the lesion size and improvement in clinical symptoms. In Case 2, electrochemotherapy was chosen due to the risks associated with other treatments and was completed without any adverse events. Both cases demonstrated the potential of electrochemotherapy as a viable treatment option for spinal hemangiomas, especially in complex or recurrent cases. (4) Conclusions: electrochemotherapy with bleomycin is a promising treatment for aggressive spinal hemangiomas when conventional therapies are not feasible or have failed. Further research is needed to establish definitive protocols and long-term outcomes of electrochemotherapy in spinal hemangioma management.