Treatment of achalasia: Botulinum toxin injection vs. pneumatic balloon dilation. A prospective study with long-term follow-up

Background and Study Aims: In patients with achalasia; intrasphincteric inj ection of botulinum toxin (BTX) has been suggested as an alternative regime n to balloon dilation and has been shown to be superior to placebo injectio n. The aim of the present study was to test the effectiveness, the long-ter m outcome and the cumulative costs of BTX injection in consecutive patients with symptomatic achalasia in comparison with pneumatic balloon dilation. Patients and Methods: 37 patients, who presented with symptomatic achalasia between January 1994 and December 1996 were treated with either BTX inject ion (n = 23) or pneumatic dilation (n = 14). Patients with short-term or lo ng-term symptomatic failures of the initial procedure were treated again, e ither with the same or with the alternative method, depending on the initia l response and on the patient's wish. Symptoms were assessed using a global symptom score (0-10) which was evaluated before treatment and 1 week, 1 mo nth and then every 6 months after the treatment. In addition, body weight a nd recurrence of symptoms were noted and manometry was carried out before a nd after treatment. The patients were regularly contacted for the long-term follow-up. Results: There were significant improvements in the global symptom scores o f all patients treated, in both the BTX injection group (before 8.2 +/- 1.3 , after 3.0 +/- 1.6) and the dilation group (before 8.3 +/- 1.1, after 2.3 +/- 1.9). There was also a significant decrease of lower esophageal sphinct er pressure after treatment in the BTX group and the dilation group. There were no significant differences with regard to overall treatment failure an d long-term outcome between patients who had or had not received previous t reatment. No major complications ere encountered in either group. An actuar ial analysis over 48 months comparing patients receiving BTX injection or b alloon dilation demonstrated that after 12 months neither therapy was signi ficantly superior. After 24 months a single pneumatic dilation was superior to a single BTX injection, and after 48 months all patients treated by BTX injection had experienced a symptomatic relapse. In contrast, 35 % of all patients treated by dilation and 45 % of patients treated successfully by d ilation were still symptom-free in an intention-to-treat analysis after 48 months. When the overall costs of treatment and further treatment after rec urrence were compared, dilation and BTX injection showed a similar cost-eff ectiveness (costs per symptom-free day) after 48 months. Conclusions: BTX injection, which can be performed in an outpatient setting , is as safe and cost-effective as balloon dilation in symptomatic achalasi a. Taking into account the lower long-term efficacy of BTX injection therap y, however, it is an alternative only in a minority of older or high-risk p atients.