Chemotherapy induced nausea and vomiting (CINV) is a serious complication of treatments of hematological malignancies. Although 5HT3 blocker achieved modest control of acute phase CINV (<24 h of chemotherapy), prevention of delayed phase CINV (>24 h after chemotherapy) remained a challenge. Aprepitant, the oral neurokinin-1 receptor antagonist, has been revealed to have anti-emetic efficacy against both acute and delayed phase CINV in highly emetogenic therapies for solid tumors. However, its effect in the treatment of hematological malignancies has not been established. Thus we conducted a randomized controlled study to evaluate the efficacy of aprepitant in patients receiving chemotherapeutic agents for hematological malignancies.

Patients and methods

We examined the additional effect of aprepitant to conventional 5HT3 blocker-based prophylaxis for CINV in induction/consolidation of acute leukemia (n=18), platinum containing regimens for malignant lymphoma (n=18), and preparative regimens of autologous stem cell transplantation (n=5). The primary endpoint of this study was the overall complete response (CR), which was defined as no emetic episodes or no administration of rescue medications during the 10 days after the start of chemotherapies (overall period). Response rates in the acute and delayed phase were also analyzed. Secondary endpoints were the rate of (1) emesis, (2) rescue medications, and (3) significant nausea scored in visual analog scale (VAS) by patients themselves during the total study period or in acute or delayed phase. Oral food intake was also evaluated by patients.


A total of 49 patients were enrolled and 41 of whom (n=22 in the aprepitant arm and n=19 in the control arm) were eligible for analysis. There was no withdrawal due to the adverse effects of aprepitant. CR rates of the aprepitant and control arms were 82% and 68% in the acute phase (p=0.47) and 82% and 58% in the delayed phase (p=0.17), respectively. Overall CR was significantly higher in the aprepitant arm (82% versus 47%, p=0.026). Emetic episodes during the entire observation period were seen at a higher rate in the control arm (9% in the aprepitant arm versus 42% in the control arm, p=0.026) and the control arm tended to have more emetic episodes than the aprepitant arm both in the acute (5% in the aprepitant arm versus 26% in the control arm, p=0.08) and delayed phases (9% in the aprepitant arm versus 37% in the control arm, p=0.057). Whilst the administration rate of salvage anti-emetic therapies was almost the same between the two arms in the early phase (14% in the aprepitant arm versus 16% in the control arm, p=1), this rate tended to be lower in the aprepitant arm in the delayed phase (18% in the aprepitant arm versus 26% in the control arm, p=0.71). In the evaluation of nausea sensation by VAS, the control arm had a trend toward higher score in the late phase. The self-assessed daily oral food intake revealed that about 50% of the patients in the aprepitant arm maintained usual amounts of food intake throughout the observation period whereas this rate dropped significantly in the control arm at day 6 (p=0.049). In the sub-group analyses to find the traits that would potentially favor aprepitant use, patients with therapies for acute leukemia had little benefit from aprepitant use (Odds ratio=0.74, p=1.0). This is probably due to moderate emetogenicity of this group, as substantiated by high CR rate in the control arm (80%).


Aprepitant confers additional anti-emetic effect to conventional 5HT3 blocker-based regimens in moderately/highly emetic chemotherapies for hematological malignancies.


Nannya: Ono Pharmaceutical Co., Ltd: Research Funding. Kurokawa:Ono Pharmaceutical Co., Ltd: Research Funding.

Author notes


Asterisk with author names denotes non-ASH members.

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