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Thomas F Imperiale

Publications and source records attributed to Thomas F Imperiale.

At least 19 recordsLinked to original sources

Comparison of endoscopic ultrasound and computed tomography for the preoperative evaluation of pancreatic cancer: a systematic review.

BACKGROUND & AIMS: It is uncertain whether computed tomography (CT) or endoscopic ultrasound (EUS) is superior for the detection, staging, and resectability of pancreatic cancer. We therefore performed a systematic literature review to determine which test is more accurate. METHODS: We identified relevant studies from MEDLINE (1986-2004) and evaluated study quality, which was measured on the basis of guidelines for assessing studies of diagnostic tests. Quantitative outcomes data were abstracted from the studies. RESULTS: Eleven studies with 678 patients satisfied inclusion criteria. Nine studies assessed tumor detection, all of which concluded that the sensitivity of EUS was superior to CT. Four of 5 studies that assessed tumor staging accuracy and 5 of 8 that assessed nodal staging accuracy concluded that EUS was superior to CT. Among the 4 studies that assessed resectability, 2 showed no difference between EUS and CT, and 1 favored each modality. Three of 11 studies met all but one of the quality criteria. The most important and frequent study limitations were lack of a consecutive series of patients and biased patient selection for surgery. Quantitative comparisons among studies were precluded by differences in tumor staging classifications, surgical selection, CT and EUS techniques, and reporting of operating characteristics. CONCLUSIONS: The published literature comparing EUS and CT for preoperative assessment of pancreatic cancer is heterogeneous in study design, quality, and results. All studies have methodologic limitations that potentially affect validity. Prospective studies with state-of-the-art imaging are needed to further define the role of each test.

Endosonography↗

Elevated prevalence of hepatitis C infection in users of United States veterans medical centers.

Several studies suggest veterans have a higher prevalence of hepatitis C virus infection than nonveterans, possibly because of military exposures. The purpose of this study was to estimate the prevalence of anti-hepatitis C antibody and evaluate factors associated with infection among users of Department of Veterans Affairs medical centers. Using a two-staged cluster sample, 1288 of 3863 randomly selected veterans completed a survey and underwent home-based phlebotomy for serological testing. Administrative and clinical data were used to correct the prevalence estimate for nonparticipation. The prevalence of antihepatitis C antibody among serology participants was 4.0% (95% CI, 2.6%-5.5%). The estimated prevalence in the population of Veterans Affairs medical center users was 5.4% (95% CI, 3.3%-7.5%) after correction for sociodemographic and clinical differences between participants and nonparticipants. Significant predictors of seropositivity included demographic factors, period of military service (e.g., Vietnam era), prior diagnoses, health care use, and lifestyle factors. At least one traditional risk factor (transfusion or intravenous drug use) was reported by 30.2% of all subjects. Among those testing positive for hepatitis C antibody, 78% either had a transfusion or had used injection drugs. Adjusting for injection drug use and nonparticipation, seropositivity was associated with tattoos and incarceration. Military-related exposures were not found to be associated with infection in the adjusted analysis. In conclusion, the prevalence of hepatitis C in these subjects exceeds the estimate from the general US population by more than 2-fold, likely reflecting more exposure to traditional risk factors among these veterans.

Adult↗

Endoscopic therapy versus medical therapy for bleeding peptic ulcer with adherent clot: a meta-analysis.

BACKGROUND & AIMS: The optimal management of bleeding peptic ulcer with adherent clot is controversial and may include endoscopic therapy or medical therapy. METHODS: We searched MEDLINE, BIOSIS, EMBASE, and the Cochrane Library to identify all randomized controlled trials comparing the 2 interventions. Outcomes evaluated in the meta-analysis were recurrent bleeding, need for surgical intervention, length of hospitalization, transfusion requirement, and mortality. RESULTS: Six studies were identified that included 240 patients from the United States, Hong Kong, South Korea, and Spain. Patients in the endoscopic therapy group underwent endoscopic clot removal and treatment of the underlying lesion with thermal energy, electrocoagulation, and/or injection of sclerosants. Rebleeding occurred in 5 of 61 (8.2%) patients in the endoscopic therapy group, compared with 21 of 85 (24.7%) in the medical therapy group (P = .01), for a pooled relative risk of 0.35 (95% confidence interval, 0.14-0.83; number needed to treat, 6.3). There was no difference between endoscopic therapy and medical therapy in length of hospital stay (mean, 6.8 vs 5.6 days; P = .27), transfusion requirement (mean, 3.0 vs 2.8 units of packed red blood cells; P = .75), or mortality (9.8% vs 7%; P = .54). Patients in the endoscopic therapy group were less likely to undergo surgery (pooled relative risk, 0.43; 95% confidence interval, 0.19-0.98; number needed to treat, 13.3); however, this outcome became nonsignificant when only peer-reviewed studies were considered. CONCLUSIONS: Endoscopic therapy is superior to medical therapy for preventing recurrent hemorrhage in patients with bleeding peptic ulcers and adherent clots. The interventions are comparable with respect to the need for surgical intervention, length of hospital stay, transfusion requirement, and mortality.

Blood Transfusion↗

Fecal DNA versus fecal occult blood for colorectal-cancer screening in an average-risk population.

BACKGROUND: Although fecal occult-blood testing is the only available noninvasive screening method that reduces the risk of death from colorectal cancer, it has limited sensitivity. We compared an approach that identifies abnormal DNA in stool samples with the Hemoccult II fecal occult-blood test in average-risk, asymptomatic persons 50 years of age or older. METHODS: Eligible subjects submitted one stool specimen for DNA analysis, underwent standard Hemoccult II testing, and then underwent colonoscopy. Of 5486 subjects enrolled, 4404 completed all aspects of the study. A subgroup of 2507 subjects was analyzed, including all those with a diagnosis of invasive adenocarcinoma or advanced adenoma plus randomly chosen subjects with no polyps or minor polyps. The fecal DNA panel consisted of 21 mutations. RESULTS: The fecal DNA panel detected 16 of 31 invasive cancers, whereas Hemoccult II identified 4 of 31 (51.6 percent vs. 12.9 percent, P=0.003). The DNA panel detected 29 of 71 invasive cancers plus adenomas with high-grade dysplasia, whereas Hemoccult II identified 10 of 71 (40.8 percent vs. 14.1 percent, P<0.001). Among 418 subjects with advanced neoplasia (defined as a tubular adenoma at least 1 cm in diameter, a polyp with a villous histologic appearance, a polyp with high-grade dysplasia, or cancer), the DNA panel was positive in 76 (18.2 percent), whereas Hemoccult II was positive in 45 (10.8 percent). Specificity in subjects with negative findings on colonoscopy was 94.4 percent for the fecal DNA panel and 95.2 percent for Hemoccult II. CONCLUSIONS: Although the majority of neoplastic lesions identified by colonoscopy were not detected by either noninvasive test, the multitarget analysis of fecal DNA detected a greater proportion of important colorectal neoplasia than did Hemoccult II without compromising specificity.

Adenocarcinoma↗

Do aspirin and nonsteroidal anti-inflammatory drugs cause false-positive fecal occult blood test results? A prospective study in a cohort of veterans.

PURPOSE: To determine whether use of regular aspirin or nonsteroidal anti-inflammatory drugs (NSAIDs) is a risk factor for a false-positive fecal occult blood test result. METHODS: Consecutive patients referred for colonoscopy for a positive fecal occult blood test result at a Veterans Affairs hospital were eligible. Patients with hematochezia, peptic ulcer disease, or unevaluated dyspepsia requiring antacids, or who used warfarin, were excluded. Regular aspirin and NSAID use was defined as at least one daily dose for at least 3 days per week. Colonoscopic findings unlikely to explain a positive test result alone were defined a priori as diverticulosis, hemorrhoids, or polyps <1.0 cm with no villous histology. Findings likely to explain a positive test result included cancer and advanced polyps. RESULTS: The sample comprised 193 veterans with a mean (+/- SD) age of 66 +/- 10 years; 98% were male and 86% were white. No colonoscopic findings explained the positive fecal occult blood test result in 153 patients (79%). One hundred and thirty-five patients (70%) were regular aspirin or NSAID users, of whom 21% (n = 29) had findings to explain the positive test results, compared with 19% (11/58) of nonusers (P = 0.7). There was no relation between aspirin dose and colonoscopic findings unlikely to explain a positive test result. Multivariate analysis found no association between regular aspirin or NSAID use and a false-positive test result (odds ratio = 0.85; 95% confidence interval: 0.39 to 1.84). CONCLUSION: Aspirin and NSAID use were not risk factors for a false-positive fecal occult blood test result in this study.

Aged↗

Comparison of endoscopic ultrasonography and multidetector computed tomography for detecting and staging pancreatic cancer.

BACKGROUND: Accurate preoperative detection and staging of pancreatic cancer may identify patients with locoregional disease that is amenable to surgical resection. OBJECTIVE: To compare endoscopic ultrasonography and multidetector computed tomography (CT) for the detection, staging, and resectability of known or suspected locoregional pancreatic cancer. DESIGN: Prospective, observational, cohort study. SETTING: Single, tertiary referral hospital in Indianapolis, Indiana. PATIENTS: 120 participants with known or suspected locoregional pancreatic cancer. INTERVENTIONS: Endoscopic ultrasonography followed by multidetector CT was performed in all patients. Patients with known or suspected pancreatic cancer deemed potentially resectable by 1 or both tests were considered for surgery. MEASUREMENTS: Detection, staging, and resectability of pancreatic cancer. Surgically resected pancreatic cancer with negative microscopic histologic margins was considered resectable. RESULTS: Of 120 patients enrolled, 104 (87%) underwent endoscopic ultrasonography and CT. Of the 80 patients with pancreatic cancer, 27 (34%) were managed nonoperatively, and 53 (66%) treated surgically had resectable (n = 25) or unresectable (n = 28) cancer. For the 80 patients with cancer, the sensitivity of endoscopic ultrasonography (98% [95% CI, 91% to 100%]) for detecting a pancreatic mass was greater than that of CT (86% [CI, 77% to 93%]; P = 0.012). For the 53 surgical patients, endoscopic ultrasonography was superior to CT for tumor staging accuracy (67% vs. 41%; P < 0.001) but equivalent for nodal staging accuracy (44% vs. 47%; P > 0.2). Of the 25 resectable pancreatic tumors in patients recommended for surgery, endoscopic ultrasonography and CT correctly identified 88% and 92%, respectively, as resectable. Of the 28 unresectable pancreatic tumors in patients recommended for surgery, endoscopic ultrasonography and CT correctly identified 68% and 64%, respectively, as unresectable. LIMITATIONS: Radiologists who read the scans and endosonographers were not blinded to previous radiographic information. Because of the modest sample size, CIs of the sensitivity estimates were sometimes wide. CONCLUSION: Compared with multidetector CT, endoscopic ultrasonography is superior for tumor detection and staging but similar for nodal staging and resectability of preoperatively suspected nonmetastatic pancreatic cancer.

Adenocarcinoma↗

Endoscopic ultrasound in non-small cell lung cancer and negative mediastinum on computed tomography.

Despite technical advances in staging non-small cell lung cancer (NSCLC), accurate staging remains a challenge. Endoscopic ultrasound is useful in staging NSCLC when lymphadenopathy is present on a computed tomography (CT), but its role in the absence of lymphadenopathy on CT has not been well defined. Therefore, we sought to determine the clinical impact of endoscopic ultrasound (EUS) in staging NSCLC in absence of mediastinal lymphadenopathy on CT. Seventy-six patients with NSCLC with absence of mediastinal lymphadenopathy on CT were enrolled and followed prospectively. EUS-guided fine-needle aspiration was performed on sites that were suspicious for metastases. Surgical pathology after thoracotomy was used as the reference standard for assessing accuracy. Sixty-two (86%) patients underwent surgery, and 10 (13%) did not. EUS precluded surgery in 9 patients (12%) and influenced management in 18 (25%) of all patients in this study. EUS detected malignant mediastinal lymphadenopathy more frequently in patients with lower lobe and hilar cancers combined compared with upper lobe cancers (p = 0.004). EUS played a significant role in identifying patients with unresectable (N3) NSCLC when adenopathy was not present on CT imaging and appears to be more sensitive in detecting lymph node metastases in lower lobe and hilar NSCLC compared with upper lobe NSCLC.

Adult↗

Is diverticulosis associated with colorectal neoplasia? A cross-sectional colonoscopic study.

OBJECTIVE: To determine the relationship between distal diverticulosis and risk for colorectal neoplasia. METHODS: Patients undergoing first-time colonoscopy for any indication were eligible if they had no prior polypectomy, colonic resection, or inflammatory bowel disease. Patients completed a survey about risk factors for colorectal cancer (CRC) prior to colonoscopy. Endoscopists, blinded to study objective and survey results, recorded the size, extent (none, few, or many), and location of diverticuli and polyps. RESULTS: The 502 participants were 67% male with a mean age of 58.6 yr. Twenty-three percent had extensive distal diverticulosis (EDD), 36% had > or =1 adenoma, and 14% had advanced neoplasia. Overall comparison of those with EDD versus few or no diverticuli revealed no differences in the risks of any neoplasia or advanced neoplasia, either distally (26.7%vs 25.4%; 12.9%vs 8.8%, respectively) or proximally (25%vs 18.4%; 6.0%vs 4.9%). Compared to women with few or no distal diverticuli, however, women with EDD were more likely to have any neoplasia and advanced neoplasia, both distally (34.6%vs 16.3%; p= 0.03, and 23.1%vs 5.7%; p= 0.003) and proximally (30.8%vs 14.9%; p= 0.049, and 11.5%vs 4.3%, p= 0.13). Adjustment for age did not affect results for advanced distal neoplasia (OR = 3.92; CI: 1.18-13); however, adjustment for the presence of a distal neoplasm eliminated the increased risk of proximal neoplasia associated with EDD (OR = 1.31; CI: 0.43-4.02). CONCLUSION: In this study, women with EDD were more likely to have advanced distal neoplasia. The presence of distal neoplasia in women with EDD accounted for their increased risk of proximal neoplasia. Distal diverticulosis was not independently associated with proximal neoplasia in men or women.

Colonoscopy↗

Risk of pancreatitis with mutation of the cystic fibrosis gene.

BACKGROUND: Between 5% and 15% of patients with recurrent pancreatitis have no identified etiology after routine investigation and advanced endoscopic evaluation. OBJECTIVE: To determine whether mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene is a risk factor for idiopathic pancreatitis. METHODS: We compared the frequency of CFTR mutations as measured by DNA probe analysis in a case group of persons with idiopathic pancreatitis and a control group without pancreatitis, all of whom underwent endoscopic retrograde cholangiopancreatography. A separate analysis compared the prevalence of CFTR mutations between the case group and controls with pancreatitis of known etiology. A subgroup comparison was made between cases of pancreas divisum with pancreatitis and controls with pancreas divisum and no pancreatitis. RESULTS: CFTR mutations were present in 19 (19%) of 96 cases and 7 (3.5%) of 198 controls without pancreatitis (odds ratio, OR = 6.7; 95% CI, 2.8-16.3; p < 0.00001). Compared to the controls with a known cause of pancreatitis (N = 78), cases had a higher prevalence of CFTR mutations (19% vs 2.6%, OR = 9.4; CI, 2.1-41.7; p= 0.0005). Among subjects with pancreas divisum, CFTR mutations were present in 8 (22%) of 37 cases compared to 0 (0%) of 20 controls (OR = 11.8; CI, 8.9-14.7; p= 0.02). CONCLUSION: The risk of idiopathic pancreatitis is greater among persons with CFTR mutations as compared to persons without CFTR mutations. Among persons with pancreas divisum, CFTR mutations appear to increase the risk for pancreatitis.

Adolescent↗

Using risk for advanced proximal colonic neoplasia to tailor endoscopic screening for colorectal cancer.

BACKGROUND: Colonoscopic screening for colorectal cancer has been suggested because sigmoidoscopy misses nearly half of persons with advanced proximal neoplasia. OBJECTIVE: To create a clinical index to stratify risk for advanced proximal neoplasia and to identify a subgroup with very low risk in which screening sigmoidoscopy alone might suffice. DESIGN: Cross-sectional study. SETTING: A company-based program of screening colonoscopy for colorectal cancer. PATIENTS: Consecutive persons 50 years of age or older undergoing first-time screening colonoscopy between September 1995 and June 2001. MEASUREMENTS: A clinical index with 3 variables was created from information on the first 1994 persons. Points were assigned to categories of age, sex, and distal findings. Risk for advanced proximal neoplasia (defined as an adenoma 1 cm or larger or one with villous histology, severe dysplasia, or cancer) was measured for each score. The index was tested on the next 1031 persons from the same screening program. RESULTS: Of 1994 persons, 67 (3.4%) had advanced proximal neoplasia. A low-risk subgroup comprising 37% of the cohort had scores of 0 or 1 and a risk of 0.68% (95% CI, 0.22% to 1.57%). Among the validation group of 1031 persons, risk for advanced proximal neoplasia in the low-risk subgroup (comprising 47% of the cohort) was 0.4% (upper confidence limit of 1.49%). Application of this index detected 92% of persons with advanced proximal neoplasms and, if applied following screening sigmoidoscopy, could reduce the need for colonoscopy by 40%. The marginal benefit of colonoscopy among low-risk persons was small: To detect 7 additional persons with advanced proximal neoplasia, 1217 additional colonoscopies would be required. CONCLUSIONS: This clinical index stratifies the risk for advanced proximal neoplasia and identifies a subgroup at very low risk. If it is validated in other cohorts or groups, the index could be used to tailor endoscopic screening for colorectal cancer.

Age Distribution↗

A randomized trial of yogurt for prevention of antibiotic-associated diarrhea.

Antibiotic-associated diarrhea (AAD) is the most common adverse effect of antibiotic therapy. Our aim was to determine the effectiveness of a dietary supplement of yogurt for prevention of AAD. Two hundred two hospitalized patients receiving oral or intravenous antibiotics were randomized to receive or not receive a dietary yogurt supplement, consisting of 227 grams of commercial yogurt, and followed for 8 days. Mean age of the study group was 70 years and 43% were male. Compliance and 8-day follow-up were 85% and 91%, respectively. Patients receiving yogurt reported less frequent diarrhea (12% vs 24%; P = 0.04), and significantly less total diarrhea] days (23 vs 60). The cumulative proportions of patients without diarrhea were significantly different (P = 0.02) between patients receiving and not receiving yogurt. For conclusion, dietary supplementation with yogurt is a simple, effective, and safe treatment that decreases the incidence and duration of AAD.

Administration, Oral↗

Is the distal hyperplastic polyp a marker for proximal neoplasia?

CONTEXT: The current literature is unclear about the association between distal hyperplastic polyps and synchronous neoplasia (adenomatous polyps and cancer) in the proximal colon. OBJECTIVE: To estimate the prevalence of proximal neoplasia associated with distal hyperplastic polyps. DATA SOURCES: Database searches (medline and embase from 1966 to 2001) and manual search of the bibliographies of included and excluded studies, case reports, editorials, review articles, and textbooks of Gastroenterology. STUDY SELECTION: Studies describing the prevalence of proximal neoplasia in persons with distal hyperplastic polyps. DATA EXTRACTION: Demographics, clinical variables, study design, and prevalence of proximal neoplasia associated with various distal colorectal findings. DATA SYNTHESIS: Of 18 included studies, 12 involved asymptomatic individuals in which the pooled absolute risk of any proximal neoplasia associated with distal hyperplastic polyps was 25% (95% confidence interval [95% CI], 21% to 29%). In 4 studies where colonoscopy was performed irrespective of distal findings, the absolute risk was 21% (95% CI, 14% to 28%). The relative risk of finding any proximal neoplasia in persons with distal hyperplastic polyps was 1.3 (95% CI, 0.9 to 1.8) compared to those with no distal polyps. Among 6 studies of patients with symptoms or risk factors for neoplasia, the absolute risk of proximal neoplasia was 35% (95% CI, 32% to 39%) in persons with distal hyperplastic polyps. In 2 studies of screening colonoscopy, advanced proximal neoplasia (cancer, or a polyp with villous histology or severe dysplasia, or a tubular adenoma >/=1 cm) was present in 4% to 5% of persons with distal hyperplastic polyps, which was 1.5 to 2.6 times greater than in those with no distal polyps. CONCLUSIONS: In asymptomatic persons, a distal hyperplastic polyp is associated with a 21% to 25% risk for any proximal neoplasia and a 4% to 5% risk of advanced proximal neoplasia, and may justify examination of the proximal colon. Further study is needed to determine the risk of advanced proximal neoplasia associated with size and number of distal hyperplastic polyps.

Colonic Neoplasms↗

Measuring the hemodynamic response to primary pharmacoprophylaxis of variceal bleeding: a cost-effectiveness analysis.

OBJECTIVES: The hemodynamic response to ss-blockers alone or with nitrates is highly predictive of efficacy in prevention of variceal bleeding. Hemodynamic monitoring (HDM) requires catheterization of the hepatic vein and measurement of the hepatic venous pressure gradient, the difference between wedged and free hepatic venous pressure. The aim of this study was to compare HDM with no HDM in patients considered for primary pharmacoprophylaxis of esophageal variceal bleeding. METHODS: A decision model was constructed to compare HDM with no HDM in cirrhotic patients with moderate to large esophageal varices. Patients intolerant to beta-blocker therapy would undergo endoscopic variceal ligation; those with an inadequate hemodynamic response (HDR) to beta-blocker therapy could have nitrates added before ligation was considered. Variceal bleeding was treated with ligation, with transjugular intrahepatic portosystemic shunt (TIPS) reserved for refractory bleeding. Probabilities of treatment responses as well as risks of bleeding and mortality were based on published literature. Only direct costs were considered during the 5-yr time horizon. Outcomes were cost in United States dollars, survival length in life-years, and proportions of patients who experienced variceal bleeding, TIPS insertion, and mortality from any cause. RESULTS: In the base case analysis, HDM was either cost-saving ($2,523 US dollars /life-year gained) or cost-effective (incremental cost-effectiveness ratio of $5,200 US dollars/life-year saved) compared with no HDM, depending on whether nitrates were added to beta-blocker therapy. HDM reduced variceal bleeding by nearly 60% and had a small effect on all-cause mortality. In the sensitivity analysis, HDM was sensitive to the time horizon, as it was not cost-effective for a time horizon of <22 months and was not cost-saving before 49 months. The cost-effectiveness of HDM was not sensitive to reasonable variation in the probability of HDR to beta-blocker therapy, risk of bleeding, risk reduction with pharmacotherapy, or to the costs of HDM, bleeding, ligation, or TIPS. Probabilistic sensitivity analysis indicated that HDM was more effective and less costly 100% and 57% of the time, respectively. CONCLUSIONS: Compared with the current standard of no HDM, measuring the hemodynamic response of primary pharmacoprophylaxis substantially reduces the number of bleeding episodes and is cost-effective or cost-saving over a wide range of sensitivity analyses. A randomized trial of HDM is needed to verify its efficacy in clinical practice.

Adrenergic beta-Antagonists↗

Results of screening colonoscopy among persons 40 to 49 years of age.

BACKGROUND: The prevalence of colorectal lesions in persons 40 to 49 years of age, as identified on colonoscopy, has not been determined. METHODS: We reviewed the procedure and pathology reports for 906 consecutive persons 40 to 49 years of age who voluntarily participated in an employer-based screening-colonoscopy program. The histologic features of lesions that were identified and removed on endoscopy were categorized according to those of the most advanced lesion removed proximally (up to the junction of the splenic flexure and the descending colon) and the most advanced lesion removed distally. An advanced lesion was defined as an adenoma at least 1 cm in diameter, a polyp with villous histologic features or severe dysplasia, or a cancer. RESULTS: Among those who underwent colonoscopic screening, 78.9 percent had no detected lesions, 10.0 percent had hyperplastic polyps, 8.7 percent had tubular adenomas, and 3.5 percent had advanced neoplasms, none of which were cancerous (95 percent confidence interval for cancer, 0 to 0.4 percent). Eighteen of 33 advanced neoplasms (55 percent) were located distally and were potentially within reach of a sigmoidoscope. If these results are applicable to the general population, at least 250 persons, and perhaps 1000 or more, would need to be screened to detect one cancer in this age group. CONCLUSIONS: Colonoscopic detection of colorectal cancer is uncommon in asymptomatic persons 40 to 49 years of age. The noncancerous lesions are equally distributed proximally and distally. The low yield of screening colonoscopy in this age group is consistent with current recommendations about the age at which to begin screening in persons at average risk.

Adenoma↗

Effect of growth hormone therapy on height in children with idiopathic short stature: a meta-analysis.

CONTEXT: Use of growth hormone (GH) therapy to promote growth in children with idiopathic short stature is controversial. A fundamental issue underlying the controversy is uncertainty about the magnitude of effectiveness of GH for this condition. OBJECTIVE: To determine the effect of GH on short- and long-term growth in idiopathic short stature. STUDY DESIGN: Systematic review of controlled and uncontrolled studies. DATA SOURCES: MEDLINE (1985-2000), key journals, cross-referencing of bibliographies, abstract booklets, and experts. STUDY SELECTION AND DATA EXTRACTION: We performed a meta-analysis of all studies satisfying the inclusion criteria for idiopathic short stature: initial height below the 10th percentile, normal stimulated GH levels (>10 microg/L), absence of comorbid conditions, no previous GH therapy, treatment with biosynthetic GH, and inclusion of major outcome measures. PRIMARY OUTCOME MEASURES: Growth velocity and height SD score (number of SDs from mean height for age and sex) at baseline and after 1 year to evaluate the short-term effect of GH. Adult height was analyzed to evaluate the long-term effect of GH. DATA SYNTHESIS: Ten controlled trials (434 patients) and 28 uncontrolled trials (655 patients) met the inclusion criteria. While baseline growth velocities were equivalent at baseline, 1-year growth velocity of the GH-treated group significantly exceeded that of controls by 2.86 cm/y. Similarly, in uncontrolled trials, growth velocity increased after 1 year, and height SD score increased from -2.72 at baseline to -2.19. In controlled studies, the adult height of the GH-treated group significantly exceeded controls by 0.84 SD, and in uncontrolled trials the adult height attained after GH treatment (-1.62 SDs) exceeded that predicted at baseline (-2.18 SDs). These results suggest an average gain in adult height of approximately 4 to 6 cm (range, 2.3-8.7 cm) with GH therapy. Given current treatment costs, this corresponds to more than $35 000 per inch (2.54 cm) gained in adult height in idiopathic short stature. CONCLUSIONS: Treatment with GH results in short-term increases in growth for children with idiopathic short stature, and long-term GH can increase adult height. These results are fundamental to decisions about GH use and raise questions about the goals of treatment. Use of GH for idiopathic short stature in clinical practice will depend on its efficacy in promoting growth and the value of this effect to families, physicians, and third-party payers.

Body Height↗

The cost-effectiveness of treatment strategies for achalasia.

Achalasia is a disorder characterized by abnormal motility of the esophageal body and the lower esophageal sphincter, resulting in dysphagia, regurgitation, and chest pain. Treatment options for achalasia include Botulinum toxin injection, pneumatic balloon dilation, and surgical esophagomyotomy. The aim of this study was to determine the cost-effectiveness of these three strategies in the treatment of achalasia in adults. We constructed a Markov cost-effectiveness model comparing Botox injection, pneumatic balloon dilation, and laparoscopic esophagomyotomy as initial treatments of achalasia. Costs and probabilities were derived from the published literature. The utility for symptomatic achalasia was derived from a sample of patients with achalasia. Sensitivity analyses were performed. Over a five-year time horizon, pneumatic dilation was the most cost-effective treatment strategy for achalasia, with an incremental cost-effectiveness ratio of $1348 per quality-adjusted life-year compared to Botox. Although laparoscopic esophagomyotomy was more effective than the other treatment options, it was not cost-effective because of its high initial cost. In conclusion, pneumatic dilation is the most cost-effective treatment option for adults with achalasia. Further studies should examine the long-term relapse rates following treatment with Botox and more precisely determine the quality of life of symptomatic achalasia.

Algorithms↗