Treatment of Childhood Gastrointestinal Stromal Tumors

Observation.

Surgery.

Targeted therapy.

Only 9% of patients experienced a fatal event, whereas 71% (54 patients) developed tumor recurrence or progression at a median of 2.5 years.

For this population, the 1-year event-free survival (EFS) rate was 73%, the 5-year EFS rate was 24%, and the 10-year EFS rate was 16%.

Factors associated with an increased risk of recurrence included metastatic disease and elevated mitotic rate. SDH status and extent of surgical resection did not influence the risk of recurrence.

Among 33 patients who underwent reoperation for recurrent disease, each subsequent resection was associated with a lower EFS rate.

In a review of ten patients who were treated with imatinib mesylate, one patient experienced a partial response and three patients had stable disease.[3]

In the phase III SWOG Cancer Research Network intergroup S0033 (NCT00009906) trial, 20 tumors from patients presumed to have wild-type disease were resequenced.[8]

In the phase III SWOG Cancer Research Network intergroup S0033 (NCT00009906) trial, 20 tumors from patients presumed to have wild-type disease were resequenced.[8]

Twelve of these tumors were identified as having SDH variants, and only one patient (8.3%) experienced a partial response to imatinib.[10]

In another study, sunitinib appeared to show more activity.[11]

In another study, sunitinib appeared to show more activity.[11]

In six children with imatinib-resistant GIST, one patient had a partial response, and five patients had stable disease.

The combination of olaparib and temozolomide produced symptomatic relief and response in one pediatric patient with multiply relapsed SDH-deficient GIST who had osseous and mediastinal disease.[12]

In a phase II trial of guadecitabine, no partial or complete responses were seen in seven patients (aged 18–57 years) with recurrent or refractory SDH-deficient GIST.[13]