Background: Pediatric patients with relapsed or refractory
sarcomas have poor outcome and need novel
therapies that provide disease control while maintaining an acceptable quality of life. The safety of
vincristine,
irinotecan, and
pazopanib (VIPaz) association has not yet been published in this population. Methods: A chart review was conducted in children and adolescents with relapsed or refractory bone and
soft tissue sarcomas who received VIPaz in our institution. Results: One hundred sixty-six patients with a diagnosis of soft or bone
sarcoma were admitted to our hospital in the period between March 2015 and August 2018, 30 were relapsed or resistant. Seventeen out of 30 resistant or relapsed patients (median age, 14 years) received 114 VIPaz cycles (median six cycles per patient, range 1-17). Sixteen courses (15%) resulted in gastrointestinal toxicity with Grade two
diarrhea; 35 courses (30%) resulted in Grade ≥3
neutropenia. One patient presented Grade two
hypothyroidism after nine courses, and another one had Grade two
hyperbilirubinemia after 12 courses. Two and five patients required a 25%
dose reduction of
irinotecan (because of
diarrhea) and
pazopanib (because of
neutropenia four and
hyperbilirubinemia 1), respectively. No patient experienced
heart failure,
hypertension, nor
posterior reversible encephalopathy syndrome.
Pneumothorax was not reported in any case even in lung metastatic patients. After two and four VIPaz cycles, we observed one complete response (CR), five partial responses (PRs), seven stable diseases (SDs), and four progressive diseases (PDs). With a median follow-up of 15 months (range 3-32), five out of 17 (29%) patients were alive, and four patients were in continuous CR after 12 VIPaz cycles. Conclusions: The VIPaz regimen might be a safe option in children and adolescents with relapsed or refractory
sarcomas otherwise unable to be enrolled in other clinical trials; on the other hand, the efficacy of
pazopanib observed cannot be sustained from the current study.