Cost-effectiveness and efficacy of a novel combination regimen in acromegaly: a prospective, randomized trial

Vivien Bonert, James Mirocha, John Carmichael, Kevin C J Yuen, Takako Araki, Shlomo Melmed
Journal of Clinical Endocrinology and Metabolism 2020 July 10

CONTEXT: Combination therapy with somatostatin receptor ligand (SRL) plus pegvisomant for acromegaly patients is recommended after maximizing dose on monotherapy. Lower-dose combination regimens are not well-studied.

OBJECTIVE: To compare cost-effectiveness and efficacy of three lower-dose combination regimens in controlled and uncontrolled acromegaly.

DESIGN AND SETTING: Prospective, randomized, open-label, parallel arm study at a tertiary referral pituitary center.

PATIENTS: Adults with acromegaly regardless of response to prior SRL and biochemical control status at baseline, stratified by SRL dose required for IGF-I normalization during any 3-month period within 12 months preceding enrollment.

INTERVENTION: Combination therapy for 24-32 weeks on Arm A, high-dose SRL (lanreotide 120 mg/octreotide LAR 30 mg) plus weekly pegvisomant (40-160 mg/week); Arm B, low-dose SRL (lanreotide 60 mg/octreotide LAR 10 mg) plus weekly pegvisomant; or Arm C, low-dose SRL plus daily pegvisomant (15-60 mg/day).

MAIN OUTCOME MEASURE: Monthly treatment cost in each Arm in subjects completing ≥24 weeks of therapy.

RESULTS: Sixty patients were enrolled and 52 evaluable. Fifty of 52 (96%) demonstrated IGF-I control regardless of prior SRL responsiveness (Arm A, 14/15 [93.3%]; Arm B, 22/23 [95.7%]; Arm C, 14/14 [100%]). Arm B was least costly (mean, $9,837±1,375 per month); Arm C was most expensive (mean, $22,543±11,158 per month) and Arm A had an intermediate cost (mean, $14,261±1,645 per month). Approximately 30% required pegvisomant dose uptitration. Rates of adverse events were all <10%.

CONCLUSIONS: Low-dose SRL plus weekly pegvisomant represents a novel dosing option for achieving cost-effective, optimal biochemical control in patients with uncontrolled acromegaly requiring combination therapy.

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