Use and Safety of Per-Oral Endoscopic Myotomy for Achalasia in the US

Abstract

Importance: Several professional practice guidelines recommend per-oral endoscopic myotomy (POEM) as a potential first-line therapy for the management of achalasia, yet payers remain hesitant to reimburse for the procedure owing to unanswered questions regarding safety.

Objective: To evaluate the use, safety, health care utilization, and costs associated with the use of POEM for treatment of achalasia relative to laparoscopic Heller myotomy (LHM) and pneumatic dilation (PD).

Design, setting, and participants: This was a retrospective national cohort study of commercially insured patients, aged 18 to 63 years, who underwent index intervention for achalasia with either LHM, PD, or POEM in the US between July 1, 2010, and December 31, 2017. Patient data were obtained from a national commercial claims database. Included in the study were patients with at least 12 months of enrollment after index treatment and a minimum of 6 months of continuous enrollment before their index procedure. Patients 64 years or older were excluded to avoid underestimation of health care claims from enrollment in Medicare supplemental insurance. Data were analyzed from July 1, 2019, to July 1, 2021.

Main outcomes and measures: Changes in the proportion of annual procedures performed for achalasia were evaluated over time. The frequency of severe procedure-related adverse events, including perforation, pneumothorax, bleeding, and death, were compared. Negative binomial regression was used to compare the incidence rates of subsequent diagnostic testing, reintervention, and unplanned hospitalization. Generalized linear models were used to compare differences in 1-year health-related expenditures across procedures.

Results: This cohort study included a total of 1921 patients (median [IQR] age: LHM group, 48 [37-56] years; 737 men [51%]; PD group, 51 [41-58] years; 168 men [52%]; POEM group, 50 [40-57] years; 80 men [56%]). The use of POEM increased 19-fold over the study period, from 1.1% (95% CI, 0.2%-3.2%) of procedures in 2010 to 18.9% in 2017 (95% CI, 13.6%-25.3%; P = .01). Adverse events were rare and did not differ between procedures. Compared with LHM, POEM was associated with more subsequent diagnostic testing (incidence rate ratio [IRR], 2.2; 95% CI, 1.9-2.6) and reinterventions (IRR, 1.9; 95% CI, 1.1-3.3). When compared with PD, POEM was associated with more subsequent diagnostic testing (IRR, 1.5; 95% CI, 1.3-1.8) but fewer reinterventions (IRR, 0.4; 95% CI, 0.2-0.6). The total 1-year health care costs were similar between POEM and LHM, but significantly lower for PD (mean cost difference, $7674; 95% CI, $657-$14 692).

Conclusions and relevance: Results of this cohort study suggest that POEM was associated with higher health care utilization compared with LHM and lower subsequent health care utilization but higher costs compared with PD. The use of POEM is increasing rapidly; payers should recognize the totality of evidence and current treatment guidelines as they consider reimbursement for POEM. Patients should be informed of the trade-offs between approaches when considering treatment.

Figure 1.. Study Flow Diagram of Patients Included in the Final Analysis

^aInclusion criteria included diagnosis code plus definitive procedure and index procedure during the study period.

Figure 2.. Definitive Procedures Performed for Achalasia Among Commercially Insured US Adults From 2010-2017