Efficacy of aprepitant as a prophylactic medication in adults with cyclic vomiting syndrome.

BACKGROUND: Aprepitant is a neurokinin-1 receptor antagonist, and recent guidelines by the American Neurogastoenterology and Motility Society recommend its use as prophylaxis in moderate-to severe cyclic vomiting syndrome (CVS). Data are limited to small studies in children. We aimed to determine its efficacy in adults with CVS. METHODS: A retrospective review of CVS patients diagnosed using Rome criteria at a tertiary referral center was conducted. Drug response was defined as >50% reduction in symptoms and/or healthcare utilization. An intent-to-treat (ITT) analysis was conducted. KEY RESULTS: Of 96 patients prescribed aprepitant, 26 (27%) were unable to start due to cost/lack of insurance coverage. Of 70 receiving therapy, mean age was 33 ± 11 years; 51 (73%) were female and 56 (80%) Caucasian. The majority (93%) were refractory to other prophylactic medications. Aprepitant was taken thrice weekly in 51 (73%), daily in 16 (23%) and a few times a month in 3 (4%) due to cost. Fifty (71.4%) had a global symptom response to aprepitant. There was significant reduction in the number of CVS episodes (14.5 ± 11.7 to 6.2 ± 8.0, p < 0.0001), emergency department visits (4.2 ± 7.7 to 1.8 ± 3.4, p = 0.006), and hospital admissions (1.6 ± 3.9 to 0.8 ± 2.1, p = 0.02) in patients treated with aprepitant. Seven (10%) discontinued the drug due to minor side effects. CONCLUSIONS AND INFERENCES: Aprepitant is a safe and effective prophylactic medication in adults with refractory CVS. Adequate insurance coverage is a major barrier preventing its use.

Retrospective review of patients treated for cyclic vomiting syndrome with topiramate.

BACKGROUND: Practice guidelines recommend topiramate as second-line treatment for the prevention of moderate-severe cyclic vomiting syndrome (CVS) in adults. However, data are limited to small studies in children. AIM: To characterise the response to topiramate as prophylactic therapy in adults with CVS. METHODS: We conducted a retrospective review of patients with CVS. Clinical characteristics, number of CVS episodes, emergency department (ED) visits, and hospitalisations the year before and after initiating topiramate were recorded. Response was defined as a global improvement in symptoms or >50% reduction in the number of CVS episodes, ED visits or hospitalisations. RESULTS: Sixty-five percent (88/136) of patients responded to topiramate in an intent-to-treat analysis. There was a significant decrease in the annual number of CVS episodes (18.1 vs 6.2, P < 0.0001), CVS-related ED visits (4.3 vs 1.6, P = 0.0029), and CVS-related hospitalisations (2.0 vs 1.0, P = 0.035). Logistic regression revealed that higher doses of topiramate, longer use of topiramate (≥12 months) and topiramate as monotherapy were associated with a response to treatment. Anxiety was associated with non-response to topiramate. Fifty-five percent of patients experienced side effects, and 32% discontinued the medication as a result. The most common side effects were cognitive impairment (13%), fatigue (11%) and paresthesia (10%). This represented a refractory group with topiramate being initiated in patients (92%) who had failed treatment with tricyclic antidepressants (TCAs). CONCLUSIONS: Topiramate may be an effective second-line prophylaxis for patients with moderate-severe CVS, but its use is limited by side effects. Efforts to develop better-tolerated therapies for CVS are warranted.

Screening Women Aged 50-59 for CRC Using Fecal Occult Blood Test Produces Outcomes Similar to Men Undergoing Screening Colonoscopy.

BACKGROUND: Average-risk women aged 50-59 years have a lower incidence and mortality of colorectal cancer relative to age-matched men, calling into question the benefit of screening colonoscopy in this age group. AIMS: We aimed to determine whether FOBT is an effective initial screening test in 50-59-year-old women. METHODS: We conducted a cross-sectional study using a computerized endoscopic report generator. We identified 320,906 individuals who had average-risk screening colonoscopy and 32,369 who had colonoscopy for positive FOBT. The primary outcome was the positive predictive value (PPV) of FOBT for large polyp(s) greater than 9 mm, as a surrogate for advanced neoplasia. RESULTS: Among patients aged 50-59 years undergoing screening colonoscopy, men were more likely than women to have large polyps (6.3 vs 4.2%, p < 0.0001). Black women undergoing screening colonoscopy had higher rates of large polyps compared to non-Black women. The PPV in FOBT-positive men aged 50-54 (11.5%) and 55-59 (14.4%) was higher than in women aged 50-54 (6.1%) and 55-59 (5.4%). Despite this lower PPV, women aged 50-54 with a positive FOBT had a similar rate of large polyps as 50-54-year-old men undergoing screening colonoscopy (6.1 vs 6.3%, p = 0.626). CONCLUSIONS: CRC screening with FOBT identifies 50-59-year-old men and women with a higher risk of large polyps. Since younger women have a lower risk of large polyps than men, screening with FOBT in 50-59-year-old non-Black women could be an effective screening strategy, with outcomes similar to the use of screening colonoscopy in 50-59-year-old men.

Characteristics of hypertension in premature infants with and without chronic lung disease: a long-term multi-center study.

BACKGROUND: Many causes for neonatal hypertension in premature infants have been described; however in some cases no etiology can be attributed. Our objectives are to describe such cases of unexplained hypertension and to compare hypertensive infants with and without chronic lung disease (CLD). METHODS: We reviewed all cases of hypertension in premature infants referred from 18 hospitals over 16 years. Inclusion criteria were hypertension occurring at <6 months of age and birth at <37 weeks gestation; the main exclusion criterion was known secondary hypertension. Continuous variables were compared using analysis of variance. Nominal variables were compared using chi-square tests. RESULTS: A total of 97 infants met the inclusion criteria, of whom 37 had CLD. Among these infants, hypertension presented at a mean of 11.3 ± 3.2 chronological weeks of age and a postmenstrual age of 39.6 ± 3.6 weeks. Diagnostic testing was notable for plasma renin activity (PRA) being <11 ng/mL/h in 98% of hypertensive infants. Spironolactone was effective monotherapy in 51 of 56 cases of hypertension. Hypertension resolved in all infants, with an average treatment duration of 25 weeks. Significant differences between the two groups of infants were a 0.4 kg lower birthweight and a 2.5 weeks younger gestational age at birth in those with CLD (p < 0.01, p < 0.01, respectively). Hypertension presented in those with CLD 1.8 weeks later, but at the same postmenstrual age as those without CLD (p < 0.01, p = 0.45, respectively). CONCLUSION: Premature infants with unexplained hypertension, with and without CLD, presented at a postmenstrual age of 40 weeks with low PRA, transient time course, and a favorable response to spironolactone treatment.