Gudrun Bernhard

Predictors of outcome in patients with achalasia treated by pneumatic dilation.

This prospective study investigates whether the effect of pneumatic dilation in patients with achalasia can be predicted on the basis of patient characteristics, type of treatment, or results of postdilation investigations. Over a period of 10 years, 54 consecutive patients with newly diagnosed achalasia were treated with pneumatic dilation and underwent pretreatment and posttreatment manometric, radiographic, and scintigraphic investigations. They were followed up every 2 years until the fall of 1991. Among the factors evaluated in the initial examination, only young age adversely affected outcome (P < 0.05). With the exception of the diameter of the dilating balloon, the treatment characteristics had a low predictive value. Postdilation lower esophageal sphincter pressure was the single most valuable factor for predicting the long-term clinical response (P < 0.0005). However, patients with high sphincter pressures and poor treatment results benefited from repeated dilations by having progressively longer remissions. It is concluded that young patients are poor candidates for pneumatic dilation, that treatment should be aimed at near complete inflation of the dilating bag, and that postdilation sphincter pressure may guide further treatment.

Pneumatic dilation for achalasia: late results of a prospective follow up investigation

Background and aims: In this prospective study, we determined the long term clinical course of patients with achalasia who were treated by pneumatic dilation using the Browne-McHardy dilator, and determined whether previously described predictors of outcome remain significant after prolonged follow up. Methods: Between 1981 and 1991, 54 consecutive patients were treated by pneumatic dilation and followed up at regular intervals for a median of 13.8 years. Remission was determined with the use of a structured interview and a previously described symptom score. Duration of remission was evaluated by Kaplan-Meier estimates of time to recurrence. Predictors of outcome were determined using the log rank test. Results: Complete follow up until 2002 was obtained in 98% of all patients. Seven patients had died and were censored. A single pneumatic dilation resulted in a five year remission rate of 40% and a 10 year remission rate of 36%. Repeated dilations only mildly improved the clinical response. Patients who were older than 40 years had a significantly better outcome than younger patients (log rank test, p = 0.0014). However, the most significant predictive factor for a favourable long term outcome was a post-dilation lower oesophageal sphincter pressure of less than 10 mm Hg (log rank test, p = 0.0001). Conclusions: Long term results of pneumatic dilation are less favourable than previously thought. Young patients and those not responding to a single pneumatic dilation should be offered alternative therapy. Patients who remain in remission for five years are likely to benefit from the longlasting treatment effect of pneumatic dilation.

Treatment of soft tissue sarcoma in childhood and adolescence. A report of the German Cooperative Soft Tissue Sarcoma Study.

Background. In the first German soft tissue sarcoma (STS) study, CWS‐81, 344 patients younger than 19 years of age who had previously untreated soft tissue sarcoma were studied. For this analysis, there were 218 patients with chemosensitive STS (Group A: rhabdomyosarcoma [RMS], synovial sarcoma, extraosseous Ewing sarcoma, leiomyosarcoma, undifferentiated sarcoma, and malignant peripheral neuroectodermal tumor) who could be studied for a minimum potential follow‐up time of 6 years.

Heller Myotomy for Failed Pneumatic Dilation in Achalasia: How Effective Is It?

Objectives:This long-term prospective study describes the effect of myotomy in patients who fail to respond to repeated pneumatic dilations and compares their clinical course with that of patients responding to dilation therapy. Methods:Nineteen consecutive patients who had never reached a clinical remission after repeated pneumatic dilation underwent myotomy. Their clinical course was compared with that of patients who had reached a clinical remission after a single (n = 34) or multiple (n = 14) pneumatic dilation(s). Symptoms were graded with a previously described symptom score ranging from 0 to 12. Remission was defined as a score of 3 or less persisting for at least 6 months. Duration of remission was summarized using Kaplan Meier survival curves. Association between baseline factors and the need for surgery was evaluated using logistic regression. Results:Complete follow-up was obtained for 98.5% of the patients. The median duration of follow-up was similar in patients treated by myotomy (10.0 years), in patients reaching a clinical remission after a single dilation (10.6 years), but differed in patients undergoing repeated dilations (6.9 years). The 10-year remission rate was 77% (95% CI 53–100%) in patients undergoing myotomy, 72% (95% CI: 56–87%) in patients “successfully” treated with a single pneumatic dilation and 45% (95% CI: 16–73%) in patients undergoing several dilations. Among all baseline factors investigated, young age was associated with an increased need of surgery. Conclusions:Myotomy is an effective treatment modality in patients with achalasia who have failed to respond to pneumatic dilation. Young patients may benefit from primary surgical therapy.

Anorectal function and morphology in patients with sporadic proctalgia fugax

PURPOSE: The pathophysiology of sporadic proctalgia fugax remains unknown. This study investigates whether patients with this syndrome exhibit alterations in anal function and morphology. METHODS: Eighteen patients with sporadic proctalgia fugax and 18 sex-matched and age-matched healthy controls were studied. Manometric studies investigated anal resting and squeeze pressures, the rectoanal inhibitory reflex, rectal compliance, and smooth muscle response to edrophonium chloride administration. External and internal sphincter thickness was measured endosonographically. RESULTS: Patients had slightly higher (P=0.0291) anal resting pressures (65.5±11.4 mmHg) than controls (56±9.9 mmHg). However, anal squeeze pressure, sphincter relaxation during rectal distention, and rectal compliance were similar in both groups, and no alterations were detected in external and internal anal sphincter thickness. Edrophonium chloride administration was followed by sharp postrelaxation contractions in two patients, whereas anal function remained unaltered in controls. Acute episodes of proctalgia, which occurred in two patients while under study, were associated with a rise in anal resting tone and an increase in slow wave amplitude. CONCLUSIONS: In the resting state, patients with proctalgia fugax have normal anorectal function and morphology. However, they may exhibit a motor abnormality of the anal smooth muscle during an acute attack.

Life expectancy, complications, and causes of death in patients with achalasia: results of a 33-year follow-up investigation

Background Patients with achalasia require repeated invasive therapies and may experience multiple complications. The objectives of this study were to determine the incidence of such complications, causes of death, and life expectancy in 253 patients. Methods Patients consisted of two groups: group A comprised 177 patients with newly diagnosed achalasia; group B consisted of 76 patients in whom the diagnosis had been established in external institutions. All patients regularly underwent structured interviews and were reinvestigated if changes in health status occurred. Survival rates were determined by Kaplan–Meier estimates and were compared with those of an average German population. Causes of death were determined from hospital records, information supplied by private physicians, and from death certificates. Results Complete follow-up was obtained in 98.9% (group A) and in 100% (group B) of all patients. The observation period for group A ranged from 2 to 33 years and for group B from 2 to 26 years (disease duration: 4–68 years). The most frequent complications were reflux esophagitis (group A: 6.2%, group B: 19.7%) and megaesophagus (group A: 6.2%, group B: 21.0%). Thirty-six patients had died during follow-up. Five of these deaths were related to achalasia. In group A, the estimated 20-year survival rates in patients with achalasia [76% (95% confidence interval (CI): 66–85%)] did not significantly differ from those in controls 80% (95% CI: 71–89%). In group B, 25-year survival rates were also similar in patients [87% (95% CI: 78–97%)] and controls [86% (95% CI: 76–97%)]. Conclusion Patients with achalasia experience a significant number of complications. Causes of death and life expectancy, however, do not differ from those of the average population.

Multiple Hypotheses Testing

The paper is mainly concerned with multiple testing procedures which control a given multiple level α. General concepts for this purpose are the closure test and a modification which is independent of the special structure of hypotheses and tests. We consider improvements of this modification using information about the logical dependences (redundancies) within the system of hypotheses and present an efficient algorithm. Finally, we discuss some problems which are specific for hierarchical systems of hypotheses, e.g. in model search.

Colonoscopy without premedication versus barium enema: a comparison of patient discomfort

BACKGROUND Colonoscopy is considered a painful procedure requiring routine intravenous sedation. We investigated whether unsedated colonoscopy causes more discomfort than barium enema. METHODS Procedure-related discomfort was determined in 100 consecutive patients undergoing colonoscopy without premedication and in an equal number of patients referred for sigmoidoscopy and barium enema. All patients underwent such examinations for the first time and had no history of previous bowel surgery. During colonoscopy, sedation was offered if significant pain or discomfort occurred. RESULTS In patients without stenosis and with satisfactory preparation, the completion rate of colonoscopy was 95%. Five percent of all patients undergoing endoscopy required sedation. On an analog scale ranging from 1 to 9, patients undergoing colonoscopy and barium enema reported similar ratings for procedure related discomfort (3.2 +/- 1.7 and 3.1 +/- 1.9) and for discomfort caused by bowel preparation (3.2 +/- 2.1 and 3.1 +/- 1.8). Eighty-seven percent of all patients undergoing colonoscopy stated that they would prefer no premedication in the event of repeated examinations. CONCLUSIONS Colonoscopy with sedation on demand does not cause more discomfort than barium enema and will be accepted by the vast majority of patients undergoing this procedure.

Anal ultra slow waves: a smooth muscle phenomenon associated with dyschezia.

Anal ultra slow waves (USWs) have been describedin a variety of anorectal disorders, all of which may beassociated with constipation. We investigated whetherthey represent a marker for dyschezia and whether their occurrence can be modified.Manometric and endosonographic studies were performed in25 patients with dyschezia, in 25 age- and sex-matchedcontrols, and in an equal number of patients with hemorrhoids. Patients exhibiting ultra slowwaves were repeatedly studied with and without localadministration of isosorbide dinitrate. In addition, wedetermined whether stimulatory maneuvers modify the occurrence of USWs. Anal USWs were persistentlyfound in 56% of patients with dyschezia, in 8% ofpatients with hemorrhoids, and in none of the healthycontrols. They were stimulated by anal squeeze and completely abolished by local administration ofisosorbide dinitrate. Ultra slow waves always occurredin conjunction with an increase in anal resting pressureand were tightly associated with a fluctuation in slow wave amplitude. Anal sphinctermorphology was similar in patients with dyschezia and incontrols. We conclude that anal USWs occur mostfrequently in patients with dyschezia and indicatesmooth muscle dysfunction. Treatment directed atabolishing this motor phenomenon may represent a novelapproach to the management of patients withdyschezia.

A rapid algorithm and a computer program for multiple test procedures using logical structures of hypotheses

It is demonstrated how improvements of general multiple test procedures can be obtained using information about the logical structures among the hypotheses. Based on a procedure of Bergmann and Hommel (B. Bergmann and G. Hommel, Improvements of general multiple test procedures for redundant systems of hypotheses, in Multiple Hypothesenprüfung--Multiple Hypotheses Testing, Eds. P. Bauer, G. Hommel and E. Sonnemann, pp. 100-115 (Springer-Verlag, Berlin, 1988)), a computer program was written by Bernhard (G. Bernhard, Computerunterstützte Durchführung von multiplen Testprozeduren--Algorithmen und Powervergleich, Doctoral thesis (Mainz, 1992)) using this information. It is applicable for a general class of systems of hypotheses which can be expressed in a linear way. By means of a simulation study it is shown that the proposed procedure is often substantially more powerful than other usual multiple test procedures.