Shreyaskumar R. Patel, MD: Additional Options Available for Durable Response

What additional options are available for durable response in this patient to stabilize her metastatic disease?

The critical word in that question may be "durable." Durable is going to be variably defined by each individual. If you look at durable responses in the traditional definition, it would last at least a few months. That is a very conservative definition to begin with, and I don't think the patient has any standard therapy options. She could try ifosamide, though the pros and cons of the treatment are many.

The current practice treatment tends to be to take this patient's tumor tissue to see if you can find a target that can be addressed with a specific inhibitor. If so, then a medical professional could try to match that patient to a phase I clinical trial, which may be the best option. There is data out there that suggests that rather than trying an empiric phase I clinical trial, which could be a reasonable option for this patient if she was willing to consider it, is matching them to a targeted therapy rather than picking one empirically.

CASE: Soft-Tissue Sarcoma (Part 1)

Rachel F is a 58-year-old school teacher from Roanoke, Virginia. Her medical history is notable for mild hypertension and total knee replacement in 2011

• In March of 2013, she presented to her PCP with abdominal fullness and distension of several months’ duration; physical exam showed mild abdominal discomfort on palpation; she denied any recent weight loss
• Initial abdominal sonography was inconclusive; subsequent CT scan showed a heterogeneously enhancing retroperitoneal mass along segment I of the inferior vena cava (IVC) and central necrosis
• She underwent contrast-enhanced CT with coronal and sagittal reconstructions, which showed encasement of the aorta and multiple hepatic metastases
• CT guided biopsy of the mass showed leiomyosarcoma that was immunohistochemically positive for desmin, smooth muscle actin, and vimentin, with a high proliferative rate (Ki67 > 60%)
• She underwent chemotherapy with gemcitabine and docetaxel for a total of 6 cycles, and experienced a minor response. Therapy was discontinued however, in November 2013 due to cumulative toxicity

Follow-up CT scan in January 2014 showed progression at multiple sites; at the time of follow up, her ECOG performance status was 1, with renal and hepatic function within normal limits

• She underwent six cycles of chemotherapy with anthracycline and dacarbazine, and her disease stabilized

In September of 2014 she returns for follow-up, unable to work with increasing fatigue and abdominal pain, and her CT scan was consistent with progressive disease

• She received treatment with pazopanib at 800 mg daily for metastatic disease
• Patient tolerated the treatment well, with mild fatigue and diarrhea, and her symptoms improved

After 4 months of therapy, she presents with worsening abdominal pain and declining performance status

• CT showed extensive progression of the primary tumor and hepatic metastases
• At progression, CBC, liver, and renal function were within normal limits, ECOG performance status was 2