Bodge, M., Shillingburg, A., Paul, S., & Biondo, L. (2014). Safety and efficacy of aprepitant for chemotherapy-induced nausea and vomiting in pediatric patients: A prospective, observational study. Pediatric Blood & Cancer, 61(6), 1111–1113. 

To evaluate the effectiveness and safety of aprepitant related to the incidence and severity of chemotherapy-induced nausea and vomiting (CINV) when added to a standard antiemetic regimen for highly or moderately emetogenic chemotherapy in pediatric patients

Both male and female patients received a standard antiemetic regimen of ondansetron 0.45 mg/kg and dexamethasone 7 mg/². In addition, they also received oral (solution or capsule) aprepitant with weight-based dosing based on the facility’s dosing table. Issues related to quality of life, such as severity of nausea, frequency of vomiting, appetite, and effect on activities of daily living (ADLs) were assessed using a survey. Since there are few studies using aprepitant in young patients weighing < 40 kg, the safety and tolerability of aprepitant in this population was the focus of this study.

• N = 11 patients (20 encounters)  
• MEAN AGE = 9.55 (4.85) years (range = 12 months–17 years)
• MALES: Not identified, FEMALES: Not identified
• KEY DISEASE CHARACTERISTICS: Confirmed malignancy
• OTHER KEY SAMPLE CHARACTERISTICS: Patients received a cycle of highly or moderately emetogenic chemotherapy.

• SITE: Single-site  
• SETTING TYPE: Not specified  
• LOCATION: Not indicated

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

Prospective, observational study

A patient survey was created by the investigators using the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) grading system v4.0 and a 4-point Likert scale. It was completed at baseline, every day in which chemotherapy was administered, and for five days after the completion of chemotherapy. The survey evaluated the occurrence and severity of nausea, vomiting, appetite, and affect on ADLs during the phases of acute (0–24 hours), delayed (1–5 days), and overall phase (acute and delayed) after chemotherapy. The survey was administered by a study investigator or sent home with the patient’s caregiver. Complete response (CR) was defined as no nausea, vomiting, or no use of rescue drugs during the acute phase.

A total of 7 out of 16 (38.9%) CRs were achieved. No vomiting was reported in the overall (acute and delayed) phase in 16 out of 18 encounters (88.9%). In 5 out of 17 encounters (29.4%), no nausea was experienced in the overall phase. There was no interference in ADLs in 47.1% and no interference with appetite in 10 out of 18 (55.6%) patients. Rescue medications were not required in the acute phase for 7 out of 11 patients. There were no directly attributable adverse effects related to the systemic absorption of aprepitant, and it was found to be well-tolerated in this pediatric study population.

Oral aprepitant administration was found to be safe in patients as young as 12 months in moderate and highly emetogenic chemotherapy. During the study period, complete responses (no nausea, vomiting, or use of rescue drugs) were experienced in 33.3% of patients.

• Small sample (< 30)
• Risk of bias (no control group)
• Risk of bias (no blinding)
• Risk of bias (no random assignment) 
• Risk of bias (sample characteristics)
• Selective outcomes reporting
• Measurement/methods not well described
• Measurement validity/reliability questionable
• Findings not generalizable
• Other limitations/explanation: The range of ages from 12 months to 17 years in a small sample makes it difficult to come to valid conclusions about the results. No tool was used to measure the safety of aprepitant in this population.

In the study population, weight-based dosing of oral aprepitant was well-tolerated and did not cause any systemic adverse reactions.