Retrospective Cohort Study
Copyright ©The Author(s) 2017. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Sep 7, 2017; 23(33): 6128-6136
Published online Sep 7, 2017. doi: 10.3748/wjg.v23.i33.6128
Real-world treatment patterns of gastrointestinal neuroendocrine tumors: A claims database analysis
Al B Benson III, Michael S Broder, Beilei Cai, Eunice Chang, Maureen P Neary, Elya Papoyan
Al B Benson III, Northwestern University, Chicago, IL 60208, United States
Michael S Broder, Eunice Chang, Elya Papoyan, Partnership for Health Analytic Research, LLC, Beverly Hills, CA 90212, United States
Beilei Cai, Maureen P Neary, Novartis Pharmaceuticals, East Hanover, NJ 07936, United States
Author contributions: All authors were equally involved in the design of the study; Chang E conducted the statistical analyses and all authors contributed equally in the interpretation of results and writing of the manuscript.
Supported by Novartis Pharmaceuticals, One Health Plaza, East Hanover, No. NJ 07936-1080, United State.
Institutional review board statement: We conducted a retrospective cohort study using the Truven Health Analytics MarketScan Database and the IMS Health Pharmetrics Database, both commercial health insurance claims database for employer-insured beneficiaries in the United States. The databases are fully compliant with the Health Insurance Portability and Accountability Act and meets the criteria for a limited-use dataset. Since the patient and provider data included in this analysis were fully de-identified, this study was exempt from the Institutional Review Board review.
Conflict-of-interest statement: Cai B and Neary MP are employees of Novartis Pharmaceuticals Corporation; Benson III AB was paid as a research consultant for the study as a subject matter expert by Novartis and is an employee of Northwestern University; Broder MS, Chang E and Papoyan E are employees of Partnership for Health Analytic Research, LLC (PHAR, LLC), a health services research company paid by Novartis to conduct this research.
Data sharing statement: The study statistician, Eunice Chang, conducted all statistical analysis for this study using Health Insurance Portability and Accountability Act-compliant commercial-insurance secondary databases MarketScan and PharMetrics.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Michael S Broder, MD, MSHS, Partnership for Health Analytic Research, LLC, 280 S. Beverly Drive, Suite 404, Beverly Hills, CA 90212, United States. mbroder@pharllc.com
Telephone: +1-310-8589555 Fax: +1-310-8589552
Received: February 9, 2017
Peer-review started: February 9, 2017
First decision: April 21, 2017
Revised: May 9, 2017
Accepted: June 18, 2017
Article in press: June 19, 2017
Published online: September 7, 2017
Processing time: 210 Days and 6.3 Hours
Abstract
AIM

To describe real-world treatment patterns of gastrointestinal neuroendocrine tumors (GI NET).

METHODS

In this retrospective cohort study, we used 2009-2014 data from 2 United States commercial claims databases to examine newly pharmacologically treated patients using tabular and graphical techniques. Treatments included somatostatin analogues (SSA), cytotoxic chemotherapy (CC), targeted therapy (TT), interferon (IF) and combinations. We identified patients at least 18 years of age, with ≥ 1 inpatient or ≥ 2 outpatient claims for GI NET who initiated pharmacologic treatment from 7/1/09-6/30/14. A 6 mo clean period prior to first treatment ensured patients were newly treated. Patients were followed until end of enrollment or the study end date, whichever was first.

RESULTS

We identified 2258 newly treated GI NET patients: mean (SD) age was 55.6 years (SD = 9.7), 47.2% of the patients were between 55 and 64 years, and 48.8% were female. All regions of the United States were represented. 59.6% started first-line therapy with SSA monotherapy (964 with octreotide LAR, 380 with octreotide SA, and 1 with lanreotide), 33.3% CC, 3.6% TT, and 0.5% IF. The remainder received combinations. Mean follow up was 576 d. Overall mean first-line therapy duration was 361 d (449 d for SSA, 215 for CC, 267 for TT). 58.9% of patients had no pharmacological treatment beyond first line. The most common second-line was combination therapy with SSA. In graphical pattern analysis, there was no clear pattern visible after first line therapy.

CONCLUSION

In this study, 60% of patients initiated treatment with SSA alone or in combination. The relatively long time to discontinuation suggests possible sustained effectiveness and tolerability.

Keywords: Gastrointestinal neuroendocrine tumors; Treatment patterns; Insurance claims; Somatostatin analogue; Targeted therapy; Chemotherapy

Core tip: In this retrospective study of real-world treatment patterns, somatostatin analogues were the most common initial pharmacologic treatment in patients with gastrointestinal neuroendocrine tumors, and most of the remaining patients began treatment with chemotherapy. However, despite the many treatment options, over half of the patients discontinued treatments after first line and only less than 10% of patients received any second-line pharmacotherapy. Given limitations of claims data to elucidate reasons for this lack of continued treatment, a study using more detailed clinical information such as medical charts or physician surveys is warranted.