Peroral endoscopic myotomy provides effective and sustained relief for achalasia in Allgrove syndrome: a long-term comparative cohort study

Background: Allgrove syndrome is a rare autosomal recessive disorder characterized by the triad of alacrima, adrenal insufficiency, and achalasia. Although corticosteroid replacement and artificial tears effectively manage adrenal insufficiency and alacrima, the optimal treatment for achalasia in this syndrome remains challenging and poorly defined. Methods: All patients diagnosed with Allgrove syndrome were identified between 2009 and 2025 at Peking Union Medical College Hospital, which is a national rare disease center in China. Clinical characteristics and genetic mutations were collected and analyzed, and the long-term safety and efficacy of peroral endoscopic myotomy (POEM) in Allgrove syndrome were evaluated through follow-up telephone interviews. A comparison cohort of idiopathic achalasia (IAC) patients undergoing POEM, matched for follow-up duration, was enrolled to delineate disease-specific features. Results: = 0.046). Over a median follow-up of 5.8 years, the mean Eckardt score decreased from 5.3 to 0.8 in Allgrove patients and from 7.6 to 1.9 in idiopathic achalasia patients at the last follow-up. Conclusion: Allgrove syndrome should be taken into consideration in patients with early-onset achalasia. POEM provides effective and sustained symptom relief for achalasia in Allgrove syndrome, with a favorable safety profile.