Bakhshi, S., Batra, A., Biswas, B., Dhawan, D., Paul, R., & Sreenivas, V. (2015). Aprepitant as an add-on therapy in children receiving highly emetogenic chemotherapy: A randomized, double-blind, placebo-controlled trial. Supportive Care in Cancer, 23, 3229–3237.

To evaluate the efficacy of aprepitant in treating chemotherapy-induced vomiting when given as an additional antiemetic to children receiving highly emetic chemotherapy

All patients received ondansetron at 0.15 mg/kg IV then dexamethasone at 0.15 mg/kg IV 30 minutes prior to chemotherapy. Oral ondansetron was given at 0.3 mg/kg every eight hours, and oral dexamethasone at 0.15 mg/kg was given every eight hours until 48 hours after the completion of chemotherapy. In the treatment group, patients weighing 15–40 kg received aprepitant capsules of 80 mg for one hour before chemotherapy on days 1–3, and patients weighing 41–65 kg received 125 mg on day 1 then 80 mg on days 2–3. The control group received placebo capsules.

• N = 93 (50 in treatment group, 43 in control group)  
• MEDIAN AGE = 13 years (range = 5–18 years)
• MALES: 69 (72%), FEMALES: 27 (28%)
• KEY DISEASE CHARACTERISTICS: Hodgkin lymphoma, osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, and adenoid cystic carcinoma
• OTHER KEY SAMPLE CHARACTERISTICS: Received any highly emetic chemotherapy protocol (ABVD, VAdC, or VAC)

• SITE: Single site    
• SETTING TYPE: Inpatient    
• LOCATION: New Delhi, India

• PHASE OF CARE: Active antitumor treatment
• APPLICATIONS: Pediatrics

Randomized, double-blinded, placebo-controlled study

• In a diary, patients or their guardians recorded vomiting episodes, chemotherapy-related toxicities, food and fluid intake, and rescue antiemetic medication use.
• Febrile neutropenia information was obtained from medical records.
• Vomiting was categorized according to the National Cancer Institute's (NCI's) guidelines (mild, moderate, or severe).
• Toxicities were evaluated according to NCI Common Terminology Criteria for Adverse Events.

In the acute phase, significantly less moderate and severe vomiting occurred in treatment group compared to the control group (38% versus 72%, p = 0.001). There was a significantly higher rate of complete response in the treatment group compared to the control group (48% versus 12%, p < 0.001). There was significantly less food and fluid intake in the control group than the treatment group (p = 0.04 and p = 0.03, respectively). In the delayed phase, there was no significant difference between the treatment and control groups in regard to moderate or severe vomiting or complete response. There was significantly less fluid intake in the control group versus the treatment group (p = 0.002), but there was no difference in food intake. In the overall phase, there were no grade 3 or 4 adverse events, and there was no difference in the number of rescue medications used in the groups. The incidence of febrile neutropenia was the same between the treatment and control group.

Oral aprepitant appeared to be safe, and no major adverse events were reported. It significantly reduced vomiting in the acute phase of highly emetic chemotherapy treatment when given in combination with ondansetron and decadron in a pediatric population. However, it did not help in the delayed phase.

• Small sample (< 100)
• Measurement validity/reliability questionable
• Other limitations/explanation: The measurement of all outcomes (vomiting, food and fluid intake, and adverse events) relied on the reports of patients or guardians in a diary, and events may have been over- or under-reported. In addition, the sample size was smaller than the power analysis determination (103 patients were needed per arm to detect a significant difference).

Oral aprepitant may decrease vomiting in pediatric patients during the acute phase of highly emetic chemotherapy, but more studies are needed.