Botulinum toxin (BT) injection is an alternative treatment of achalasia. The aim of the study was to examine outcomes of patients treated with BT in the Czech Republic. Since 1997, 49 patients with achalasia have been treated with BT. We prospectively evaluated the effect of BT injection on 41 patients during a median follow-up of 24 months (range 9-62). Esophageal manometry was performed before and at 3-5 months after the injection. In 16 patients, BT was injected from the antegrade angle only (subgroup A), in 15 patients, BT was injected from both retrograde and antegrade angles (subgroup B) and, in 10 patients, BT injection was combined with subsequent balloon dilatation (subgroup C). Immediate clinical response was achieved in 93% of patients. Clinical remission was sustained beyond 3 months in 83% of patients (responders). Fourteen responders (41%) did not experience a relapse during the median of 22 months. Twenty responders (59%) experienced symptomatic relapse approximately 8 months after the injection. Ten relapsers underwent BT reinjection, five (50%) of them were asymptomatic for another 14 months. The remaining five (50%) patients reported a second relapse approximately 6 months after the reinjection. Median duration of the symptom-free period was 11.5 months after the first BT injection, and 10.5 months after the second (P = 0.21). We did not find any significant predictor of a favorable outcome; responders tended to be older and to have a lower basal lower-esophageal-sphincter pressure. Patients in subgroup C were more likely to be in remission at 1 and 2 years as compared with patients in subgroup A. BT injection is an effective treatment of achalasia in the short term. However, almost 70% of patients experience a relapse within 2 years. BT injection should therefore be reserved for patients at risk for more invasive procedures or for patients who prefer this treatment.

Injection of botulinum toxin (BT) and pneumatic dilatation are available methods in nonsurgical treatment of achalasia. Authors anticipate beneficial effect of prior BT injection on the success of pneumatic dilatation and duration of its effect. There are no long-term data available to assess efficacy of combined treatment. From 1998 to 2007, 51 consecutive patients (20 men and 31 women, age 24-83) with achalasia were included and prospectively followed up. Each patient received injection of 200 IU of BT into the lower esophageal sphincter (LES) during endoscopy and 8 days later pneumatic dilatation (PD) under X-ray control was performed. The follow-up was established every 3 months first year and then annually. The efficacy was evaluated by a questionnaire concerning patient's symptoms and manometry. Results were compared with 40 historical controls (16 men and 24 women, age 26-80) treated by PD alone using the same method and follow-up. Fifty-one patients underwent combined treatment. Four patients failed in follow-up and were not included for analysis. The mean duration of follow-up was 48 months with range 12-96 months. Thirty-four of forty-seven (72%) patients were satisfied with results with none or very rare and mild troubles at the time of the last visit. Forty-one patients were followed up more than 2 years. Effect of therapy lasted in 75% (31/41) of them. In 17 patients, more than 5 years after treatment, effect lasted in 12 (70%). Mean tonus of LES before therapy was 29 mm Hg (10-80), 3 months after therapy decreased to 14 mmHg (5-26). The cumulative 5 years remission rate (+/-95% CI) in combined treated patients 69% +/- 8% was higher than in controls 50% +/- 9%; however it, was not statistically significant (P= 0.07). In control group 1, case of perforation (2.5%) occurred. Eight patients (17%) with relapse of dysphagia were referred to laparoscopic Heller myotomy with no surgical complication. The main adverse effect was heartburn that appeared in 17 patients (36%). Initial injection of BT followed by PD seems to be effective for long-term results with fewer complications. But the combined therapy is not significantly superior to PD alone.

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