Table 4.
Summary of the Evidence Supporting Botulinum Toxin Injection in Patients With Achalasia
| Study | Country | Study design | Participants | Intervention | Comparator | Follow-up duration | Outcome |
|---|---|---|---|---|---|---|---|
| 1996, Pasricha et al52 | USA | Prospective cohort study | Treatment-naïve patients with achalasia | Botox, n = 31 | None | Median 2.4 yr | Sustained improvement (beyond 3 mo): 53.0% Symptom relapse (within a median of 1.3 yr): 95.0% |
| Symptom relapse (within a median of 1.3 yr): 95.0% | |||||||
| 1999, Muehldorfer et al53 | Germany | RCT | Patients with achalasia with/without prior treatment | Botox, n = 12 | PBD, n = 12 | 2.5 yr | Symptom remission: Botox vs PBD, 0.0% vs 60.0% |
| 1999, Vaezi et al54 | USA | RCT | Treatment-naïve patients with achalasia | Botox, n = 22 | PBD, n = 20 | 1 yr | Symptom remission: Botox vs PBD, 32.0% vs 70.0%, P = 0.017 |
| 2000, Annese et al51 | Italy | Prospective cohort study | Treatment-naïve patients with achalasia | Botox 100 U, 2 injection, n = 38 | Botox 50 U, 2 injection, n = 40 | 2 yr | Symptom relapse: Botox 100 U vs 50 U vs 200 U, 19.0% vs 47.0% vs 43.0% |
| Botox 200 U, 2 injection, n = 40 | |||||||
| 2001, Mikaeli et al55 | Iran | RCT | Treatment-naïve patients with achalasia | Botox, n = 20 | PBD, n = 20 | 1 yr | Symptom remission: Botox vs PBD, 15.0% vs 53.0%, P < 0.01 |
| 2002, D'Onofrio et al56 | Italy | Prospective cohort study | Treatment-naïve patients with achalasia | Botox, n = 37 | None | 1 yr | Symptom remission period: mean 15.6 mo (range, 2-30 mo) |
| 2003, Bansal et al57 | USA | RCT | Treatment-naïve patients with achalasia | Botox, n = 16 | PBD, n = 18 | 4 mo | Symptom remission: Botox vs PBD, 38.0% vs 89.0% |
| 2004, Zaninotto et al58 | Italy | RCT | Treatment-naïve patients with achalasia | Botox, n = 40 | Heller myotomy, n = 40 | 6 mo | Symptom score improvement rate: Botox vs Heller myotomy, 65.0% vs 82.0%, P < 0.05 |
| Symptom relapse rate in Botox group: 65.0% | |||||||
| Symptom remission (2 yr): Botox vs Heller myotomy, 34.0% vs 88.0%, P < 0.05 | |||||||
| 2009, Zhu et al59 | China | RCT | Treatment-naïve patients with achalasia | Botox, n = 29 | PBD, n = 28 | 2 yr | Symptom remission: Botox vs PBD vs PBD + Botox, 14.0% vs 36.0% vs 57.0% |
| PBD + Botox, n = 30 |
PBD, pneumatic balloon dilatation; RCT, randomized controlled trial.