Botulinum toxin versus pneumatic dilatation in the treatment of achalasia: a randomised trial. Intrasphincteric botulinum toxin versus pneumatic balloon dilation for treatment of primary achalasia. Controlled trial of botulinum toxin injection versus placebo and pneumatic dilation in achalasia. Esophageal achalasia: intrasphincteric injection of botulinum toxin A versus balloon dilation. Randomized controlled trial of intrasphincteric botulinum toxin A injection versus balloon dilatation in treatment of achalasia cardia. Randomized controlled trial comparing botulinum toxin injection to pneumatic dilatation for the treatment of achalasia.
Intrasphincteric injection of botulinum toxin is a new treatment option for achalasia.
                To compare the immediate and long term efficacy of botulinum toxin with that of pneumatic dilatation.
                Symptomatic patients with achalasia were randomised to botulinum toxin (22 patients, median age 57 years) or pneumatic dilatation (20 patients, median age 56 years). Symptom scores were assessed initially, and at one, three, six, nine, and 12 months after treatment. Objective assessment included oesophageal manometry initially and at one month, and barium oesophagram initially and at one, six, and 12 months post-treatment.
                Pneumatic dilatation resulted in a significantly (p=0.02) higher cumulative remission rate. At 12 months, 14/20 (70%) pneumatic dilatation and 7/22 (32%) botulinum toxin treated patients were in symptomatic remission (p=0.017). Failure rates were similar initially, but failure over time was significantly (p=0.01) higher after botulinum toxin (50%) than pneumatic dilatation (7%). Pneumatic dilatation resulted in significant (p<0.001) reduction in symptom scores, and lower oesophageal sphincter pressure, oesophageal barium column height, and oesophageal diameter. Botulinum toxin produced significant reduction in symptom scores (p<0.001), but no reduction in objective parameters.
                At one year pneumatic dilatation is more effective than botulinum toxin. Symptom improvement parallels objective oesophageal measurements after pneumatic dilatation but not after botulinum toxin treatment for achalasia. Despite a high success rate, pneumatic dilatation for achalasia is accompanied by a significant risk of esophageal perforation. Injection of botulinum toxin (botox) into the lower esophageal sphincter (LES) can lead to improvement in symptoms with reduced risk of complications. Direct comparisons of the two techniques are needed to define their role in clinical management.
                We compared pneumatic dilatation to botox for patients with achalasia using a double blind, randomized study design. Patients underwent clinical, manometric, radiographic and endoscopic evaluation to confirm primary achalasia. They were randomized to receive either 80 units of botox into the LES or Witzel balloon dilatation. Patients also received sham dilatation or injection, respectively. The patients and investigators assessing symptom response were blinded to therapy. Symptoms and esophageal function were assessed at 3 weeks, 3 months and 1 year after therapy. Treatment failure was defined as the lack of decrease in symptom grade more than 1 or recurrence of symptoms. Patients with treatment failure crossed over to the alternative treatment.
                Thirty four patients were studied, and 31 completed the trial. Of the 18 patients randomized to Witzel dilatation, 16 (89%) of 18 remained in clinical remission. Of the two patients with treatment failure, one responded to botox injection. Of the 16 patients randomized to botox, (38%) 6 of 16 remained in clinical remission. Four patients had initial failure, and 6 relapsed at a mean of 4 months after therapy. Of the nine patients who crossed over to dilatation, seven responded well, but two required surgical management of perforation. Although both treatments had excellent initial clinical improvement, patients randomized to Witzel dilatation had superior long-term success ( < 0.01).
                Initial therapy with Witzel dilatation is associated with better long-term outcome than a single injection of botox. Because of the risk of endoscopic perforation, botox remains a viable alternative to dilatation. Intrasphincteric injection of botulinum toxin has been suggested as an alternative treatment modality in esophageal achalasia. A controlled trial comparing botulinum toxin, placebo, and pneumatic dilation is reported.
                Sixteen patients received random intrasphincteric injections of either botulinum toxin or saline. The efficacy of treatment was assessed by symptom score, esophageal manometry, and scintigraphy. In case of failure, pneumatic dilation was performed.
                One month after injection, symptoms had improved in all patients treated with botulinum toxin (symptom score, 0.9 +/- 0.6 vs. 5.5 +/- 1.4; P < 0.02). In the placebo group, symptoms were unchanged in all patients, who were all dilated. Lower esophageal sphincter pressure decreased by 49% after treatment with botulinum toxin (P < 0.03) and by 72% after dilation (P < 0.01). Similarly, esophageal retention decreased by 47% after treatment with botulinum toxin (P < 0.02) and by 59% after dilation (P < 0.02). No significant difference in symptom score and esophageal function test results was found between patients treated with botulinum toxin injections and those undergoing dilation. However, 7 of the 8 patients in the botulinum toxin group required a second injection because of recurrent dysphagia.
                Treatment of achalasia with botulinum toxin was as effective as pneumatic dilation in relieving symptoms and improving esophageal function. The effect of the first injection was temporary, but the effect of the second injection lasted longer. In patients with esophageal achalasia, pneumatic dilation is the treatment of choice, but it bears the risk of perforation in about 4% of cases. A new nonsurgical method, intrasphincteric injection of botulinum toxin A, has shown promising initial results, and we thus undertook this study to compare the long-term outcome of these two methods.
                In a prospective randomized study, 24 patients with definitive esophageal achalasia were divided into two equal groups and underwent either balloon dilation or injection of botulinum toxin (20 U injected into each of the four quadrants in the lower esophageal sphincter). The outcome was assessed on the basis of a symptom score documented before treatment and at regular intervals for two and a half years thereafter. Complications associated with the two techniques were also documented.
                No relevant complication occurred in either of the treatment groups. Initially, dilation was successful in 10 of 12 patients (83%), and botulinum toxin injection in 9 of 12 patients (75%). The symptom scores showed no significant differences between the two groups before and one month after treatment. Over the two and a half year follow-up, however, all nine successfully treated patients in the botulinum toxin group experienced recurrence of symptoms, but only four of the ten patients (40%) in the dilation group.
                The two treatment methods initially had equal success rates, but in the long term the effect of the botulinum toxin injection was statistically significantly shorter than that of balloon dilation. As fewer risks are associated with the injection treatment, studies should be undertaken either to identify patient subgroups in whom botulinum toxin can be effective long-term or to test substances with longer-lasting effects. As the few randomized controlled trials available in the literature comparing botulinum toxin (BT) injection with established endoscopic treatment of achalasia cardia, i.e. pneumatic dilatation, showed conflicting results, we conducted a prospective randomized trial. Seventeen consecutive patients with achalasia cardia diagnosed during a period between December 1997 and February 2000 were randomized into two treatment groups [pneumatic dilatation by Rigiflex dilator (n=10), BT injection by sclerotherapy needle into four quadrants of lower esophageal sphincter (LES) (n=7) 80 units in five cases, 60 units in two cases] after dysphagia grading, endoscopy, barium esophagogram, and manometry, all of which were repeated 1 week after treatment. Patients were followed up clinically for 35.2+/-14 weeks. Chi-squares, Wilcoxon rank-sum test, Kaplan-Meier method and log-rank tests were used for statistical analysis. After 1 week, 6/7 (86%) BT-treated vs. 8/10 (80%) dilatation-treated patients improved (P=NS). There was no difference in LES pressure and maximum esophageal diameter in the barium esophagogram in the two groups before therapy. Both therapies resulted in significant reduction in LES pressure. The cumulative dysphagia-free state using the Kaplan-Meier method decreased progressively in BT-treated compared with dilatation-treated patients (P=0.027). Two patients with tortuous megaesophagus, one of whom had failed dilatation complicated by perforation previously, improved after BT. One other patient in whom pneumatic dilatation had previously failed improved in a similar manner. BT is as good as pneumatic dilatation in achieving an initial improvement in dysphagia of achalasia cardia. It is also effective in patients with tortuous megaesophagus and previous failed pneumatic dilatation. However, dysphagia often recurs during 1-year follow up. Therapeutic options for achalasia include pharmacological therapy, surgical myotomy, pneumatic dilatation and intrasphincteric botulinum toxin injection.
                To compare botulinum toxin injection with pneumatic dilatation in a randomized trial.
                Forty adults with newly diagnosed achalasia were randomized to receive botulinum toxin (n=20) or pneumatic dilatation (n=20). Symptom scores were evaluated at 1, 6 and 12 months. Clinical relapse was defined as a symptom score greater than 50% of baseline. Relapsers received a second botulinum toxin injection or pneumatic dilatation.
                The cumulative 12-month remission rate was significantly higher after a single pneumatic dilatation (53%) compared to a single botulinum toxin injection (15%)(P < 0.01). The 12-month estimated adjusted hazard for relapse and need for retreatment for the botulinum toxin group was 2.69 times that of the pneumatic dilatation group (95% confidence interval; 1.18-6.14). When a second treatment was administered to the relapsers in each group, the cumulative remission rate 1 year after initial treatment was significantly higher in the pneumatic dilatation group (100%) compared to the botulinum toxin group (60%) (P < 0.01). There were no major complications in either group.
                Pneumatic dilatation is more efficacious than botulinum toxin in providing sustained symptomatic relief in patients with achalasia. The efficacy of a single pneumatic dilatation is similar to that of two botulinum toxin injections.