Botulinum toxin versus pneumatic dilatation in the treatment of achalasia: a randomised trial

Abstract

Background: Intrasphincteric injection of botulinum toxin is a new treatment option for achalasia.

Aims: To compare the immediate and long term efficacy of botulinum toxin with that of pneumatic dilatation.

Methods: 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.

Results: 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.

Conclusions: 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.

Figure 1

Schematic summary of the study protocol comparing pneumatic dilatation with botulinum toxin.

Figure 2

Kaplan-Meier plot showing significantly (p=0.02) higher cumulative one year remission rate for patients treated with pneumatic dilatation compared with botulinum toxin.

Figure 3

Patient symptom scores initially and at one, three, six, nine, and 12 months after pneumatic dilatation (A) and botulinum toxin treatment (B). The medians and interquartile ranges (25% to 75%) are shown with horizontal lines and boxed in rectangles, respectively. The number of patients (n) remaining in the study is shown for each time interval.

Figure 4

Lower oesophageal sphincter pressures (LOSP) initially and at one month after pneumatic dilatation (A) and botulinum toxin treatment (B). The horizontal lines denote group medians and the vertical bars represent interquartile ranges (25% to 75%). The dotted horizontal line represents a lower oesophageal sphincter pressure of 12 mm Hg.

Figure 5

Lower oesophageal sphincter pressures (LOSP) initially and at one month after botulinum toxin treatment in (A) early and (B) late failure groups as well as those in remission at (C) one year.

Figure 6

Oesophageal barium column height representing oesophageal emptying initially and at one, six, and 12 months after pneumatic dilatation (A) and botulinum toxin treatment (B). The horizontal lines denote group medians while the vertical bars represent interquartile ranges (25% to 75%). The number of patients (n) remaining in the study is shown for each time interval.

Figure 7

Barium width representing oesophageal diameter initially and at one, six, and 12 months after pneumatic dilatation (A) and botulinum toxin treatment (B). The horizontal lines denote group medians while the vertical bars represent interquartile ranges (25% to 75%). The number of patients (n) remaining in the study is shown for each time interval.