The cost-effectiveness of treatment strategies for achalasia

Achalasia is a disorder characterized by abnormal motility of the esophageal body and the lower esophageal sphincter, resulting in dysphagia, regurgitation, and chest pain. Treatment options for achalasia include Botulinum toxin injection, pneumatic balloon dilation, and surgical esophagomyotomy. The aim of this study was to determine the cost-effectiveness of these three strategies in the treatment of achalasia in adults. We constructed a Markov cost-effectiveness model comparing Botox injection, pneumatic balloon dilation, and laparoscopic esophagomyotomy as initial treatments of achalasia. Costs and probabilities were derived from the published literature. The utility for symptomatic achalasia was derived from a sample of patients with achalasia. Sensitivity analyses were performed. Over a five-year time horizon, pneumatic dilation was the most cost-effective treatment strategy for achalasia, with an incremental cost-effectiveness ratio of $1348 per quality-adjusted life-year compared to Botox. Although laparoscopic esophagomyotomy was more effective than the other treatment options, it was not cost-effective because of its high initial cost. In conclusion, pneumatic dilation is the most cost-effective treatment option for adults with achalasia. Further studies should examine the long-term relapse rates following treatment with Botox and more precisely determine the quality of life of symptomatic achalasia.     

Nonoperative management of esophageal strictures following esophagomyotomy for achalasia

The optimal management of reflux-induced esophageal strictures that occur after esophagomytomy for achalasia is uncertain. This paper presents our experience with the nonsurgical treatment of postesophagomyotomy strictures in achalasia patients using endoscopic dilation and gastric acid suppression. Six patients with achalasia who had undergone prior esophagomyotomy subsequently developed recurrent dysphagia and were found to have an esophageal stricture. Esophagrams typically showed a markedly dilated esophagus with a narrowed, sharply angulated gastroesophageal junction. Esophageal manometry confirmed esophageal aperistalsis and, when measured, the LES pressure was<5 mm Hg. Endoscopy showed esophageal inflammation and a fixed stricture at the gastroesophageal junction. Strictures were dilated under direct visualization using through-the-scope balloon dilators. Patients began gastric acid suppressive treatment at the same time. Five patients who remained symptomatic underwent repeat endoscopy, which demonstrated improvement in esophagitis. Dilation was then repeated with a larger balloon dilator. Over a mean follow-up period of 3.8 years, the average number of repeat dilations per patient was 3.6 (range: 0–12). All patients had symptomatic improvement and weight gain. No patient required surgery. We conclude that esophageal strictures after esophagomyotomy for achalasia can be safely treated using endoscopic dilation and gastric acid suppression, thus avoiding the need for reoperation.     

Pneumatic dilatation or esophagomyotomy treatment for idiopathic achalasia: Clinical outcomes and cost analysis

The choice between pneumatic dilatation and surgical esophagomyotomy as the initial treatment for achalasia is controversial. The aims of this study were to determine the long term clinical outcome and costs of treating achalasia initially with pneumatic dilatation as compared to esophagomyotomy. Of 123 patients undergoing an initial pneumatic dilatation for achalasia at our institution from 1976 to 1986, 71 (58%) received no further treatment for achalasia during a mean follow up of 4.7±2.8 years. Only 15 of these 123 patients (12%) eventually underwent surgical esophagomyotomy, (two for perforation during pneumatic dilatation, 13 for persistent or recurrent symptoms). The degree of dysphagia at follow up was improved to a similar degree in patients treated with an initial pneumatic dilatation as compared to patients treated with an initial esophagomyotomy. Patients with age45, years at time of initial pneumatic dilatation had fewer subsequent treatments for persistent or recurrent symptoms and had less dysphagia on follow up as compared to patients <45 years. Subsequent pneumatic dilatations to treat persistent or recurrent symptoms were less beneficial than an initial pneumatic dilation. The cost of esophagomyotomy was 5 times greater than the cost of pneumatic dilatation. When costs were analyzed to include subsequent treatments of symptomatic patients, the total expectant costs of treating with an initial esophagomyotomy remained 2.4 times greater than treating with an initial pneumatic dilatation. This study suggests that an initial pneumatic dilatation will be the only treatment needed for the majority of patients with achalasia. A treatment regimen starting with penumatic dilatation has less overall costs than starting with esophagomyotomy. For each subsequent pneumatic dilatation, however, the clinical benefit leans toward, surgery.     

Complications during pneumatic dilation for achalasia or diffuse esophageal spasm

A retrospective cohort study was performed to assess risk factors, early clinical characteristics, and outcome of complications in patients undergoing pneumatic dilation. Of 178 patients with achalasia or diffuse esophageal spasm who underwent 236 dilations with a Browne-McHardy dilator, 16 patients experienced a complication (9.0%). Nine major complications developed: perforations (4), hematemesis (2), fever (2), and angina (1). A prior pneumatic dilation and use of inflation pressure ≥11 PSI were independent risk factors by multivariate analysis for developing a complication. An esophagram immediately following the dilation identified three of the four perforations. Three postdilation findings were identified as indicators of patients with an increased risk of having developed a perforation: blood on the dilator, tachycardia, and prolonged chest pain lasting >4 hr after dilation. In all patients incurring a major complication, one of the three indicators, or the complication itself was recognized within 5 hr of dilation. All patients with complications, including the four with perforation who received prompt surgical repair and esophagomyotomy, recovered uneventfully. The symptomatic relief of dysphagia in patients with perforation undergoing emergent surgical repair and esophagomyotomy was similar to patients undergoing elective esophagomyotomy. Conclusions: (1) Pneumatic dilation is a safe treatment of achalasia, with a 1.7% risk of perforation. (2) The risk of developing a complication is increased by having had a previous pneumatic dilation or by use of inflation pressures ≥11 psi. (3) All patients with a major complication were identified within 5 hr after dilation. (4) Complications following pneumatic dilation, if recognized and treated promptly, were not associated with adverse, long-term sequelae.     

Pneumatic dilation in achalasia.

Pneumatic dilation is the most common first-line therapy for the treatment of achalasia. The aim of dilation is a controlled disruption of circular muscle fibres of the lower esophageal sphincter to reduce the functional obstruction. Several types of dilators and different dilation techniques are used, but the achieved results are similar. The mean success rate is about 80% in the short term, but some patients need redilation in the further course (particularly young patients). Best long term results are obtained if the lower esophageal sphincter pressure can be reduced below 10 mmHg. Major complications are rare after pneumatic dilation; the most serious complication is esophageal perforation, which occurs at a mean rate of about 2.5%. Considering the pros and cons of other effective forms of treatment of achalasia (esophagomyotomy and intrasphincteric injection of botulinum toxin), pneumatic dilation is still the treatment of choice in the majority of patients with achalasia.     

Esophageal diameter and the treatment of achalasia

The effect of pneumatic dilation and Heller esophagomyotomy on esophageal diameter was studied in 49 patients with achalasia. Standardized radiologic technic was used with the patient in a horizontal position and with a 36-in. target-to-film distance. Fifteen normal subjects had a mean esophageal diameter (± SD) of 1.50±0.37 cm, while that in untreated patients was 4.85±1.44 cm. When pneumatic dilation resulted in a fall in mean diameter to 2.27±0.49 cm, symptoms improved; they persisted in those with a diameter of 4.99±1.23 cm. Heller esophagomyotomy led to a decrease in diameter to 2.29±0.62 cm in those with relief of symptoms, while symptoms continued in those with a diameter of 4.35±0.80 cm. In a longitudinal study of individual patients, it was noted that when treatment resulted in a diameter of less than 3.0 cm, symptoms improved. If esophageal size remained over 3.0 cm after treatment, however, dysphagia or pain recurred and persisted. Serial measurements of esophageal diameter, therefore, may be valuable in the clinical management of achalasia.The authors are indebted to Mr. Joseph Soussou of the Massachusetts Institute of Technology for statistical and data-processing assistance.Presented in part at the Multiple Discipline Research Forum, Meeting of the American Medical Association, San Francisco, June 20, 1968.     

胃镜下气囊扩张治疗贲门失弛缓症的疗效及随访观察

目的 评价胃镜下用Rigiflex气囊扩张器治疗原发性贲门失弛缓症的近期及远期疗效.方法 经临床症状、食管钡餐、食管测压和胃镜检查确诊的125例贲门失弛缓症患者在胃镜下用直径为3.5 cm的Rigiflex气囊扩张器进行扩张,术后根据临床症状积分、食管体正常蠕动率（EPR）、下食管括约肌静息压（LESBP）、下食管括约肌松弛率（LESRR）、下食管括约肌松弛度（LESRD）、食管钡餐和胃镜进行术后1月、6月、1年、3年和5年的随访.结果 治疗后1月内所有患者临床症状积分、LESBP、食管钡餐和胃镜均显著改善（P＜0.01）,EPR、LESRR和LESRD均无显著改善（P＞0.05）,5年随访中有7例复发,有效率达92.14%.治疗中1例出现食管穿孔.结论 在胃镜下用直径3.5 cm的Rigiflex气囊扩张器治疗原发性贲门失弛缓症是非常有效的方法,疗效较持久.随访中间歇性出现的临床症状可能与失调的食管蠕动和LES的松弛有密切关系.     

Long-term outcome of pneumatic dilation in the treatment of achalasia

OBJECTIVE: Achalasia is a well-defined esophageal motor disorder for which pneumatic dilation is an established therapeutic method. Even though it has been used for several years, there are limited data on the long-term outcomes of patients treated with this procedure. Hence, we aimed to evaluate the long-term efficacy of pneumatic dilation to control the symptoms of achalasia. METHODS: The medical records of all patients treated in our unit for achalasia with pneumatic dilation were reviewed. We identified the long-term result of the initial procedure, the date of the first dilation, and the time interval between dilation and retreatment. RESULTS: Of 260 patients who were treated with pneumatic dilation, 153 (67 men, 86 women) were followed up for more than 5 yr. The mean follow-up period was 11.09 +/- 3.91 yr, and the success rate of the dilation was 75.8%. Among these patients, 35 (19 men, 16 women) had follow-up periods of more than 15 yr. The mean follow-up time of those patients was 16.56 +/- 1.09 yr, and the success rate was 51.4%. Kaplan-Meier survival analysis showed that, overall, 50% of patients develop recurring symptoms after 10.92 yr. CONCLUSIONS: Although 51.4% of patients continued to be in clinical remission more than 15 yr after the initial pneumatic dilation, the long-term success rate of pneumatic dilation seems to drop progressively with time.     

Comparison of temporary stent insertion with pneumatic dilation of the same diameter in the treatment of achalasia patients: A retrospective study

Background and Aim: To retrospectively analyze and compare the clinical efficacy of temporary stent insertion with pneumatic dilation of the same diameter in the treatment of achalasia based on a long‐term follow up. Methods: A total of 101 treated achalasia patients were divided into a pneumatic dilation group (Group A, n = 38) and a temporary stent insertion group (Group B, n = 63). The diameter of the balloon or stent used was 30 mm. The total symptom scores (TSS) and esophageal manometry were used to assess the symptoms and lower esophageal sphincter (LES) pressure improvement. Barium swallow–esophageal studies were used to assess esophageal emptying objectively. TSS and LES pressure improvement were assessed, recorded, and compared during the regular interval follow up. Results: Forty‐nine pneumatic dilations and 65 stent insertions were successfully performed in all patients under fluoroscopy. Complications included pain, reflux, and bleeding, which occurred in nine (23.6%), eight (21.1%), and three (8%) patients in Group A, and in 27 (42.9%), eight (12.7%), and 10 (15.9%) patients in Group B, respectively. The stent was retained approximately 4–7 days and was retrieved via endoscope. TSS, esophageal manometry, and barium esophagram post‐treatment significantly improved compared to those given pretreatment (P < 0.0001). At the end of follow up, TSS and LES pressure in Group B were 4.00 ± 1.00 and 43.67 ± 12.66 mmHg, compared to 10.20 ± 0.45 (P = 0.0096) and 58.60 ± 8.65 mmHg (P = 0.1687) in Group A. The Kaplan–Meier method revealed better symptom remission in Group B compared to Group A (log–rank test, P = 0.0212). Conclusion: Retrievable stent placement is more effective than the same diameter pneumatic dilation for the treatment of achalasia with a long‐term follow up.     

Barrett's esophagus and achalasia

Two unusual cases of achalasia with endoscopic and histologic documentation of Barrett's esophagus are presented. One patient had Barrett's esophagus at the time of initial endoscopy for achalasia, before any treatment. The other patient developed specialized columnar epithelia in the esophagus after treatment with pneumatic dilation. Each patient had evidence of low-grade dysplasia. Including these two patients, 30 cases of Barrett's esophagus in patients with achalasia have been reported in the literature. In 73% (22 of 30) of the cases, Barrett's esophagus was detected after esophagomyotomy. In 20% (6 of 30) of the cases of achalasia and Barrett's esophagus, adenocarcinoma developed. The current two cases are unusual because Barrett's esophagus in achalasia generally develops from gastroesophageal reflux after esophagomyotomy. No other patients have been reported to develop Barrett's esophagus after pneumatic dilation alone. Patients with achalasia and Barrett's esophagus may be at a particularly high risk for developing dysplasia and adenocarcinoma.     

Incomplete Lower Esophageal Sphincter Relaxation in Subjects with Peristalsis: Prevalence and Clinical Outcome

Incomplete lower esophageal sphincter relaxation is recognized in achalasia and has been reported in subjects with esophageal spasm. We reviewed 500 consecutive manometric studies from a 3-yr period to determine the prevalence of this manometric finding, its association with other motility abnormalities, and the clinical outcome of subjects without associated aperistalsis (i.e., without achalasia). We identified 60 subjects with incomplete lower sphincter relaxation, 17 of whom had at least some normal peristalsis (3.4% of the total). Mean lower sphincter residual pressure for these 17 subjects (4.5 +/- 2.8 mm Hg) was intermediate between those with achalasia (11.7 +/- 6.8 mm Hg) and those with normal relaxation (0.1 +/- 0.2 mm Hg). Both peristaltic and contraction abnormalities in the esophageal body were prevalent in the 17 subjects compared with those who had normal relaxation. Outcome with conservative medical therapy after a mean follow-up of 3.3 yr was not significantly related to presence of peristaltic or contraction abnormalities at presentation, and 71% of subjects with or without these concomitant findings had improvement or complete resolution of symptoms. Only one subject worsened and was treated with pneumatic dilation. We conclude that incomplete relaxation of the lower esophageal sphincter without aperistalsis is uncommon, symptom regression occurs with conservative therapy, and pneumatic dilation appears rarely required over a modest follow-up period.     

Assessment of esophageal emptying post-pneumatic dilation: use of the timed barium esophagram

OBJECTIVES: The reported success rate of pneumatic dilation in patients with achalasia varies from 50% to 93%. This wide variability may be due to using symptom relief post-dilation as the only assessment of success. There are no studies comparing subjective symptom improvements to objective improvement in esophageal emptying after pneumatic dilation. METHODS: Patients with achalasia undergoing pneumatic dilation from 1995 to 1997 were evaluated. Pre- and post-dilation symptoms were recorded using a standardized scoring system. Barium column height was measured 1 min and 5 min after upright ingestion to assess esophageal emptying. Based on percentage of total symptom and barium height improvement post-dilation, patients were grouped according to one of nine outcomes; the association between subjective and objective parameters of improvement was tested. RESULTS: A total of 37 patients underwent 53 pneumatic dilations. There was a significant association (p < 0.001) between improvement in patient symptoms and barium height. In 38 of 53 (72%) pneumatic dilations, the degree of symptom and barium height improvement was similar. Near complete symptom resolution was reported after 26 dilations. In eight of 26 (31%) patients however, there was < 50% improvement in barium height (group A). Compared with the 16 patients with 91-100% improvement in both symptoms and barium height (group B), forward stepwise regression identified age as the only difference between the two groups, with group A patients being significantly (p = 0.04) older. CONCLUSIONS: Objective assessment of esophageal emptying pre- and post-dilation identifies an important subset of patients with poor esophageal emptying who report near complete symptom resolution. This group may benefit from any early repeat pneumatic dilation.     

Timed barium oesophagram: better predictor of long term success after pneumatic dilation in achalasia than symptom assessment

BACKGROUND: Symptom relief post pneumatic dilation is traditionally used to assess treatment success in achalasia patients. Recently, we showed that symptom relief and objective oesophageal emptying are concordant in about 70% of patients, while up to 30% of achalasia patients report near complete symptom relief despite poor oesophageal emptying of barium. AIMS: We now report the results of long term clinical follow up in these two groups of achalasia patients, assessing differences in symptomatic remission rates. METHODS: Achalasia patients undergoing pneumatic dilation since 1995 were evaluated both symptomatically and objectively at regular intervals. Pre and post dilation symptoms were recorded. Barium column height was measured five minutes after ingesting a fixed volume of barium per patient to assess oesophageal emptying. Patients who initially reported near complete symptom relief were divided into two groups based on objective findings on barium study: (1) complete oesophageal emptying (concordant group), and (2) poor oesophageal emptying (discordant group). Patients were followed prospectively for symptom recurrence. RESULTS: Thirty four patients with complete symptom relief post pneumatic dilation were identified. In 22/34 (65%) patients, the degree of symptom and barium height improvements was similar (concordant group). In 10/34 (30%) patients, there was < 50% improvement in barium height (discordant group). Significantly (p<0.001) more discordant (9/10; 90%) than concordant (2/22; 9%) patients failed therapy at the one year follow up. Seventeen of 22 (77%) concordant patients were still in remission while all discordant patients had failed therapy by six years of follow up. Length of time in symptom remission (mean (SEM)) post pneumatic dilation was significantly (p=0.001) less for the discordant group (18.0 (3.6) months) compared with the concordant group (59.0 (4.8) months). CONCLUSIONS: (1) Poor oesophageal emptying is present in nearly 30% of achalasia patients reporting complete symptom relief post pneumatic dilation. (2) The majority (90%) of these patients will fail within one year of treatment. (3) Timed barium oesophagram is an important tool in the objective evaluation of achalasia patients post pneumatic dilation.     

Management of dysphagia in suspected esophageal motor disorders

Fifty-three patients suffering from dysphagia because of suspected esophageal motor disorders were treated by pneumatic dilatation using the Rider-Moeller technique. Fifteen had achalasia demonstrated by manometric studies. Forty-nine of them had remarkable clinical improvement after the procedure. During the mean period of follow-up (average 5 years, range 1-11), 75% of the patients needed a new dilatation, with a delay of two years. The results of the dilatation were excellent or good in 80% of the cases. Early complications consisted in two esophageal perforations surgically treated. There was no mortality. We did not observe late complications of the procedure. We conclude that pneumatic dilatation should be the initial procedure in the treatment of dysphagia in suspected esophageal motor disorders.     

Long-term results of pneumatic dilation for achalasia： A 15 years＇ experience

锘緼IM: Although most patients with achalasia respond to pneumatic dilation, one-third experienced recurrence, and prolonged follow-up studies on parameters associated with various outcomes are scanty. In this retrospective study, we reported a 15-years' experience with pneumatic dilation treatment in patients with primary achalasia, and determined whether previously described predictors of outcome remain significant after endoscopic dilation. METHODS: Between September 1989 and September 2004, 39 consecutive patients with primary symptomatic achalasia (diagnosed by clinical presentation, esophagoscopy, barium esophagogram, and manometry) who received balloon dilation were followed up at regular intervals in person or by phone interview. Remission was assessed by a structured interview and a previous symptoms score. The median dysphagia-free duration was calculated by Kaplan-Meier analysis. RESULTS: Symptoms were dysphagia (n=39, 100%), regurgitation(n=23, 58.7%), chest pain (n=4, 10.2%), and weight loss (n=26, 66.6%). A total of 74 dilations were performed in 39 patients; 13 patients (28%) underwent a single dilation, 17 patients (48.7%) required a second procedure within a median of 26.7 mo (range 5-97 mo), and 9 patients (23.3%) underwent a third procedure within a median of 47.8 mo (range 37-120 mo). Post-dilation lower esophageal sphincter (LES) pressure, assessed in 35 patients, has decreased from a baseline of 35.8卤10.4-10.0卤7.1 mmHg after the procedure. The median follow-up period was 9.3 years (range 0.5-15 years). The dysphagiafree duration by Kaplan-Meier analysis was 78%, 61% and 58.3% after 5, 10 and 15 years respectively. CONCLUSION: Balloon dilation is a safe and effective treatment for primary achalasia. Post-dilation LES pressure estimation may be useful in assessing response.     

Modern management of achalasia

Opinion statement The goals in the treatment of achalasia are threefold: 1) relieving the symptoms, particularly dysphagia and bland regurgitation; 2) improving esophageal emptying by disrupting the poorly relaxing lower esophageal sphincter (LES); and 3) preventing the development of megaesophagus. Although achalasia cannot be permanently cured, excellent palliation is available in over 90% of patients, especially those with pneumatic dilation and laparoscopic Heller myotomy. The efficacy for short- and long-term therapy seems to be similar when performed by experts. Pneumatic dilation done as an outpatient surgery disrupts the LES muscle from within by using balloons of progressively larger diameter (3.0, 3.5, and 4.0 cm). Repeat dilations may be required; secondary severe gastroesophageal reflux disease (GERD) is rare, but approximately 2% of patients will have an esophageal perforation. A surgical Heller myotomy is now being done laparoscopically through the abdomen that cuts the LES and extends the myotomy 2 to 3 cm onto the stomach. Usually 2 days of hospitalization is required, and patients can normally return to work in 1 to 2 weeks. Severe GERD with esophagitis and peptic stricture is a common complication; therefore, most surgeons combine the myotomy with an incomplete fundoplication. Medical therapy is much less effective than these invasive procedures. Smooth muscle relaxants (nitrates and calcium channel blockers) taken immediately before meals improve dysphagia, but side effects and drug tolerance are common. The injection of botulinum toxin (100 to 200 units) endoscopically into the LES gives short-term relief of symptoms and improves esophageal emptying. This treatment is most effective in the elderly, as symptom relief can last up to 1 to 2 years with a single injection. Several studies suggest the most cost-effective management of achalasia is initial treatment with pneumatic dilation.     

Controlled trial of botulinum toxin injection versus placebo and pneumatic dilation in achalasia

BACKGROUND & AIMS: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: 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. (Gastroenterology 1996 Dec;111(6):1418-24)     

Witzel pneumatic dilation for achalasia: safety and long-term efficacy

Forceful dilation of the lower esophageal sphincter is considered primary therapy for achalasia. The Witzel pneumatic balloon dilator, unlike fluoroscopically placed dilators, is placed over a standard gastroscope allowing positioning and dilation under direct vision. We report our experience with the Witzel dilator in 45 patients with achalasia over a 5-year period. All patients had at least one major symptom score of 8 out of 10 for dysphagia and/or regurgitation before dilation. After Witzel dilation, symptomatic response was graded as excellent (score 0 to 2), good (score 3 to 5), fair (score 6 to 8), and poor (no improvement). Symptom response was assessed after 1 week, 1 month, 6 month, 1 year, and present. The mean period of follow-up was 25 months (range, 3 to 85 months). Passage of the balloon across the gastroesophageal junction was technically unsuccessful in three patients. Esophageal perforation occurred in two patients (4%) and transient chest pain greater than 2 days in three patients (7%). There was no bleeding or death. Symptomatic long-term improvement was excellent in 25 patients (63%), good in 6 patients (15%), fair in 4 patients (10%), and poor in 5 patients (12%). A repeat Witzel dilation was performed in five patients but resulted in good/excellent improvement in only one patient. We conclude that pneumatic dilation with the Witzel balloon is a safe, effective procedure for achalasia.     

Use of botulinum toxin as a diagnostic/therapeutic trial to help clarify an indication for definitive therapy in patients with achalasia

OBJECTIVE: Intrasphincteric injection of botulinum toxin is useful in achalasia but is limited by its short term efficacy. The aim of this study was to evaluate the use of botulinum toxin in selected patients in whom its short duration of action may be useful in guiding therapy before considering more invasive procedures that might not be indicated. METHODS: Over a 3 yr period, botulinum toxin was injected into the lower esophageal sphincter in patients with: 1) symptoms consistent with achalasia but insufficient manometric criteria to make the diagnosis; 2) complex clinical situations in which there were factors in addition to achalasia that may be contributing to the patient's symptoms and that required different treatment; 3) atypical manifestations of achalasia; 4) advanced achalasia in which it was unclear that sphincter-directed therapy (vs esophagectomy) would be of benefit; and 5) after Heller myotomy. Clinical response was assessed mostly by symptom improvement, but in some patients follow-up barium swallow or radioscintigraphy was available. RESULTS: Eleven patients were identified. Ten had complete symptomatic response to the injection. Two patients have undergone subsequent successful pneumatic dilation, one a successful laparoscopic myotomy, and another currently scheduled for surgical myotomy. The only patient without response had advanced achalasia requiring esophagectomy. CONCLUSIONS: Intrasphincteric injection of botulinum toxin into the lower esophageal sphincter is a useful and safe means of guiding therapy in those patients with a variant of achalasia, atypical achalasia, or complex achalasia in which it is unclear that more invasive procedures such as pneumatic dilation or surgical myotomy are the correct therapy.     

Long-term efficacy of Botulinum toxin in classical achalasia: a prospective study

OBJECTIVE: We sought to examine the long-term efficacy of intrasphincteric Botulinum toxin A injection in a prospective cohort study of 30 patients with achalasia. METHODS: Thirty patients with classical achalasia were treated with intrasphincteric Botulinum toxin A injection. Follow-up consisted of clinical assessment, symptom scoring, and postinjection manometry. RESULTS: Symptomatic improvement for >3 months was seen in 23 of 30 patients (77%). Of the 23 initial responders, seven (30%) experienced a sustained symptomatic response after a single Botulinum toxin injection (mean follow-up, 21 months). The remaining 16 initial responders (70%) eventually relapsed (mean initial response, 11 months). Nine received a 2nd Botulinum toxin injection, and seven experienced an ongoing response (mean duration, 9 months); two patients eventually required a 3rd injection with good effect (mean duration, 22 months). The remaining seven patients who relapsed after Botulinum toxin opted for pneumatic dilation or surgical myotomy. Five of the seven patients who had no initial response received a 2nd injection but again did not respond. A residual lower esophageal sphincter pressure <18 mm Hg after the first Botulinum toxin injection predicted a good response to Botulinum therapy (single or multiple injections, p < 0.002, positive predictive value = 0.71, negative predictive value = 1.0). Neither initial nor sustained response to Botulinum toxin could be predicted based on gender, age, duration of illness, previous pneumatic dilation, or esophageal motility before treatment. CONCLUSIONS: We found that 77% of patients with classical achalasia experienced a good symptomatic response after Botulinum toxin and 30% of initial responders achieve sustained symptomatic relief after a single treatment with Botulinum toxin. The initial responders who relapsed did well with subsequent Botulinum toxin A. Lack of an initial symptomatic response and residual lower esophageal sphincter pressure > or =18 mm Hg after Botulinum toxin are associated with a poor response.