Quality of life is an extremely important aspect of patient care and an important consideration not only in clinical trials (eg, in patient-reported outcomes) but also in our daily practice, when we are seeing new patients diagnosed with GIST. Some individuals with GIST are diagnosed incidentally and may not have associated symptoms. However, patients often present with symptoms that may be dependent on the location of the GIST (eg, painful swallowing in esophageal GIST or chronic anemia from occult bleeding). GIST arising in the small intestine tend to have less of a symptom burden and may present as larger tumors. In terms of the symptom burden from GIST, you really have to get to the root cause of the symptoms, which is the GIST itself. The best treatment for a symptom caused by GIST is to treat the GIST itself rather than focusing only on treating the symptoms. The standard approach when initiating treatment is either systemic therapy with a tyrosine kinase inhibitor (TKI) or surgical resection.

Both approaches have their own adverse effects. Surgical resection may result in the removal of significant amounts of bowel, which can alter bowel function and nutrition and can cause chronic abdominal discomfort, nausea, and diarrhea. The TKIs can cause their own side effects. For instance, fatigue is one of the more common symptoms of patients with GIST, but fatigue can be multifactorial. Thus, when a patient presents with fatigue, we do a complete workup that includes assessing thyroid function and making sure that the patient is not anemic, and, if they are anemic, determining the cause of the anemia and then treating the underlying cause to optimize the hemoglobin. And we generally try to address the fatigue as best we can. We do have psychiatrists and psychologists with whom our patients meet to help them cope with this symptom when their fatigue is severe.

With any treatment, we are balancing benefits and risks. Survival prior to TKIs for patients with metastatic GIST was a median of approximately 9 months. In the first early studies that enrolled very high-volume, very sick patients with GIST, the initial signal with TKI therapy was a 5-year median overall survival. More contemporary data that have emerged indicate that median overall survival is 7 years or longer in many patients with GIST. So, these patients are living for a relatively long time. I have patients in my practice who have been living with metastatic GIST for more than 20 years; in fact, some of these individuals were in the original phase 2 and 3 studies. Management of the disease and of the side effects of these agents is critical for our patients with GIST.