Sarcomas/TGCT

Ripretinib did not result in a significant improvement in progression-free survival over sunitinib in patients with gastrointestinal stromal tumor who were previously treated with imatinib, missing the primary end point of the phase 3 INTRIGUE trial.

Ifosfamide induced small improvements in terms of prolonging event-free survival and overall survival compared with topotecan plus cyclophosphamide in patients with relapsed/refractory Ewing sarcoma.

Treatment with larotrectinib elicited robust and durable responses, had a favorable safety profile, and sustained survival benefit in patients with central nervous system TRK fusion cancers.

Nirogacestat was found to significantly improve progression-free survival over placebo when used in the treatment of adult patients with progressing desmoid tumors, meeting the primary end point of the phase 3 DeFi trial.

Selinexor monotherapy prolonged progression-free survival, time to next treatment, and reduced pain in patients with advanced, refractory dedifferentiated liposarcoma.

The FDA has accepted for filing the new drug application resubmission for a unique formulation of sodium thiosulfate for the prevention of platinum-induced ototoxicity in patients between the ages of 1 month and 18 years who have localized, nonmetastatic, solid tumors.

For patients with osteosarcoma or Ewing sarcoma who develop pathologic fractures, the prognosis remains poor; however, there is still the possibility for limb salvage therapy.

The FDA has granted an orphan drug designation to Veyonda, a novel proprietary formulation of idronoxil, for use as a potential therapeutic option in patients with soft tissue sarcoma.

R. Lor Randall, MD, discusses takeaways from the American Academy of Orthopaedic Surgeons 2022 Annual Meeting.

A new drug application for a unique formulation of sodium thiosulfate was resubmitted to the FDA for the second time for the prevention of platinum-induced ototoxicity in patients between the ages of 1 month and 18 years who have localized, nonmetastatic, solid tumors.

Nab-sirolimus became a treatment option for a rare, aggressive soft-tissue sarcoma—malignant perivascular epithelioid cell tumor—following encouraging data in the first prospective clinical trial for this patient population.

William D. Tap, MD, discusses the key challenges faced in the treatment of patients with soft tissue sarcoma and efforts being made to overcome them.

Version 1.2022 of the National Comprehensive Cancer Network guidelines now include ripretinib as a category 1 fourth-line treatment option for patients with advanced gastrointestinal stromal tumor and as an additional TKI for consideration prior to surgery.

New agents for rare cancers make up approximately 35% of drugs in the pipeline for all rare diseases, according to a report from the Pharmaceutical Research and Manufacturers of America.

Individuals with Li-Fraumeni Syndrome, a rare hereditary condition, are at increased risk for a wide spectrum of malignancies, including breast cancer and sarcomas. Investigators are working to determine whether metformin, a drug indicated for diabetes and infertility caused by polycystic ovarian syndrome, can prevent cancers in LFS carriers.

R. Lor Randall, MD, discusses the importance of collaborations between orthopedic surgeons and interventional radiologists, and how they are beginning to pick up steam in the United States for patients with metastatic bone cancer.

Although brachytherapy is rarely utilized as a treatment for pediatric and adolescent/young adult patients in the United States, AYA patients with rhabdomyosarcoma treated with BT had favorable survival outcomes.

The combination of the TKI inhibitor imatinib and the MEK inhibitor binimetinib elicited encouraging responses in patients with treatment-naïve advanced gastrointestinal stromal tumors.

Brian A. Van Tine, MD, PhD, sheds light on the promise of SPEAR T-cell therapy in patients with synovial sarcoma and MRCLS, the safety and efficacy observed with afami-cel in the SPEARHEAD-1 trial, and next steps for research.

William D. Tap, MD, discusses developing treatment opportunities in soft tissue sarcomas.

The FDA has granted a rare pediatric disease designation to the bone-targeting radiopharmaceutical, Samarium-153-DOTMP, for use as a potential therapeutic option for patients with osteosarcoma.

R. Lor Randall, MD, speaks to the findings from a report that said multiple sex-related, racial/ethnic, and socioeconomic status disparities are associated with an increased incidence of metastatic bone disease originating from cancers in the prostate, renal, colon, lung, and breast.

Christian Frederick Meyer, MD, PhD, MS, discusses standard treatment options in cardiac angiosarcoma.

The diffusing alpha-emitter radiation therapy, Alpha DaRT, was found to elicit complete responses per RECIST v1.1 criteria in 10 patients with malignant skin and soft tissue cancers who are enrolled to an ongoing single-institution pilot feasibility trial.

No dose-limiting toxicities have been reported in the first cohort of patients with relapsed clear cell sarcoma who received the combination of devimistat and hydroxychloroquine in the dose-escalation portion of the ongoing phase 1/2 APOLLO 613 trial.

R. Lor Randall, MD, FACS, discusses methods of preventing early failures with compressive osseointegrative endoprosthetic devices of the femur in bone sarcomas.

In patients with advanced uterine leiomyosarcoma whose tumors harbor a BRCA-like phenotype, the combination of olaparib with temozolomide could represent a new standard of care.

The FDA has granted a rare pediatric disease designation to IMX-110 for the treatment of rhabdomyosarcoma.

Cardiac angiosarcoma is an exceptionally rare cancer that has become national news because of the recent death of acclaimed fashion designer, Virgil Abloh. He was 41 years old when he died in November 2021, following a 2-year battle with the disease, which is an aggressive subtype of soft-tissue sarcoma.

Despite their rarity, the management of desmoid tumors has been an area of significant clinical change as the field has shifted away from aggressive surgical interventions to active surveillance and systemic therapies that are less morbid for patients.