What is the safety of GLP1-RAs in patients with a family history of thyroid cancer?

Comment by InpharmD Researcher

The current literature regarding the risk of thyroid cancer in patients with a family history of thyroid cancer who use GLP-1 RA medications is limited. Some studies suggest potential links between GLP-1 RA use and thyroid disorders, including thyroid cancer. However, none of the identified studies take into account the risks associated with family history. Moreover, most studies do not differentiate between cases of medullary and papillary thyroid cancer. It has been emphasized that established risk factors for thyroid cancer, such as familial history and other medical conditions, should be taken into account. Contraindications for the use of Wegovy and Saxenda exist for individuals with a personal or family history of medullary thyroid carcinoma. Communication from the manufacturer also confirmed a significant lack of data regarding the use in patients with a family history of papillary thyroid cancer. Studies investigating the correlation between GLP-1 RA use and thyroid cancer have not discovered a substantial causal relationship between the two, but findings should be interpreted cautiously in the absence of more robust evidence.

Background

A 2022 meta-analysis evaluated the association between the use of glucagon-like peptide-1 receptor agonists (GLP-1 RAs) and the risk of different thyroid disorders. A total of 45 randomized controlled trials (N= 94,063) were analyzed and the results indicated that the use of GLP-1 RAs was associated with a 28% increased risk of overall thyroid disorders (relative risk [RR] 1.28; 95% confidence interval [CI] 1.03 to 1.60; p= 0.027). However, when examining different GLP-1 RAs, only liraglutide, and dulaglutide showcased significant increases in the risk of overall thyroid disorders. Liraglutide increased the risk by 37% (RR 1.37; 95% CI 1.01 to 1.86; p= 0.044)), and dulaglutide showed a 96% increase (RR 1.96; 95% CI 1.11 to 3.45; p= 0.020). On the other hand, other GLP-1 receptor agonists such as semaglutide, lixisenatide, exenatide, and albiglutide, did not show significant effects on the occurrence of overall thyroid disorders. When evaluating specific types of thyroid disorders, GLP-1 RAs did not show significant effects on the occurrence of thyroid cancer (RR 1.30; 95% CI 0.86 to 1.97; p= 0.608), hyperthyroidism (RR 1.19; 95% CI 0.61 to 2.35; p= 0.608), hypothyroidism (RR 1.22; 95% CI 0.80 to 1.87; p= 0.359), thyroiditis (RR 1.83; 95% CI 0.51 to 6.57; p= 0.353), thyroid mass (RR 1.17; 95% CI 0.43 to 3.20; p= 0.759), and goiter (RR 1.17; 95% CI 0.74 to 1.86; p= 0.503). Based on these findings, it was concluded that GLP-1 RAs did not show any significant impact on the risk of thyroid cancer, hyperthyroidism, hypothyroidism, thyroiditis, thyroid mass, and goiter. However, it is important to note that the mechanism responsible for the unfavorable effects of GLP-1 RAs on thyroid disorders still remains unclear. For thyroid cancer, some of the included studies did not specify the type of thyroid cancer, which would affect the accuracy of the results and further limit the findings. Additionally, the study lacked any discussion regarding risk factors, including family history, associated with the development of thyroid cancer. Researchers emphasize the need for further prospective studies to confirm the potential effects of GLP-1 RAs on overall thyroid disorders, and specifically in thyroid cancer. [1]

Following the release of a case-control analysis, which revealed an elevated risk of thyroid cancer associated with the usage of GLP-1 RA for a duration of 1 to 3 years (adjusted hazard ratio [HR] 1.58; 95% CI 1.27 to 1.95), a commentary published in 2023 further delved into the connection between GLP-1 RAs and thyroid cancer risk. The article emphasized that well-established risk factors for thyroid cancer encompass conditions such as goiter, nodules, familial history, prior exposure to radiation, obesity, and genetic syndromes. Notably, while certain factors can be addressed through claims data, it’s important to highlight that the study was unable to account for family history in its adjustment. While it remains plausible that GLP-1 RAs may lead to a modest relative increase in thyroid cancer risk, it's important to acknowledge that the presence of detection bias cannot be outright dismissed as an alternative explanation. It's worth noting that thyroid cancer is an infrequent outcome, thereby resulting in an exceedingly marginal escalation in absolute risk. Consequently, it was recommended that healthcare practitioners and patients alike adopt a discerning approach in weighing the benefits against the potential drawbacks of treatment options while taking into account available alternatives. In populations devoid of specific risk factors associated with thyroid cancer, the favorable impacts of GLP-1 RAs are anticipated to substantially outweigh any potential adverse effects. [2], [3]

A 2022 study embarked on an investigation into the potential association between GLP-1RA and tumor development by analyzing data from the FDA Adverse Event Reporting System (FAERS) database spanning from the first quarter of 2004 to the second quarter of 2020. This comprehensive analysis revealed a total of 8,718 reported cases of tumors associated with GLP-1RA usage. Significant correlations emerged between GLP-1RA and specific tumor types, notably thyroid cancer, including medullary thyroid cancer (with a proportional reporting ratio [PRR] of 27.43) and papillary thyroid cancer (PTC, with a PRR of 8.68). Among cases of malignant thyroid neoplasms, PTC constituted 28.9% of the occurrences. However, the precise reasons behind the elevated reporting of PTC cases linked to GLP-1RA remain uncertain and multifaceted. One plausible explanation is that the awareness of GLP-1RA elevating the risk of medullary thyroid cancer could potentially lead to an attribution of PTC reports to GLP-1RA. Furthermore, studies have indicated the presence of GLP-1 receptors (GLP-1R) in PTC cells. Specifically, one study found that 32.1% of PTC cases displayed immunoreactivity to GLP-1R. Additionally, research revealed that the expression of GLP-1R was more pronounced in papillary thyroid cancer cells than in normal thyroid cells. Intriguingly, though GLP-1RA did not exhibit a significant impact on the proliferation of papillary thyroid cancer cells in vitro, the activation of GLP-1R by GLP-1RA could still hold implications for the potential development of PTC. Therefore, the relationship between GLP-1RA and PTC may require long-term observation. [4]

Due to the limited available data regarding the potential relationship between a family history of PTC and the utilization of GLP-1 RAs, we initiated communication with Novo Nordisk, the manufacturer responsible for the production of Wegovy and Saxenda. In response, the medical information correspondent conveyed that while administration of these medications is contraindicated for individuals with a family or personal history of medullary thyroid cancer, they regrettably lack sufficient data to offer a comprehensive assessment of the risk associated with their use in patients affected by PTC. [5]

References:

[1] Hu W, Song R, Cheng R, et al. Use of GLP-1 Receptor Agonists and Occurrence of Thyroid Disorders: a Meta-Analysis of Randomized Controlled Trials. Front Endocrinol (Lausanne). 2022;13:927859. Published 2022 Jul 11. doi:10.3389/fendo.2022.927859
[2] Thompson CA, Stürmer T. Putting GLP-1 RAs and Thyroid Cancer in Context: Additional Evidence and Remaining Doubts. Diabetes Care. 2023;46(2):249-251. doi:10.2337/dci22-0052
[3] Bezin J, Gouverneur A, Pénichon M, et al. GLP-1 Receptor Agonists and the Risk of Thyroid Cancer. Diabetes Care. 2023;46(2):384-390. doi:10.2337/dc22-1148
[4] Yang Z, Lv Y, Yu M, et al. GLP-1 receptor agonist-associated tumor adverse events: A real-world study from 2004 to 2021 based on FAERS. Front Pharmacol. 2022;13:925377. Published 2022 Oct 25. doi:10.3389/fphar.2022.925377
[5] Personal Correspondence. Novo Nordisk. Medical Information. August 7, 2023.

Relevant Prescribing Information

Wegovy [6]
Contraindication:
A personal or family history of medullary thyroid carcinoma (MTC) or in patients with Multiple Endocrine Neoplasia syndrome type 2 (MEN 2)

Saxenda [7]:
Contraindication:
Patients with a personal or family history of medullary thyroid carcinoma (MTC) or patients with Multiple Endocrine Neoplasia syndrome type 2 (MEN 2)

Clinical Trials Experience:
Papillary Thyroid Cancer: In Saxenda clinical trials in adults, papillary thyroid carcinoma confirmed by adjudication was reported in 8 (0.2%) of 3291 Saxenda-treated patients compared with no cases among 1843 placebo-treated patients. Four of these papillary thyroid carcinomas were less than 1 cm in greatest diameter and 4 were diagnosed in surgical pathology specimens after thyroidectomy prompted by findings identified prior to treatment.

References:

[6] Wegovy (semaglutide injection). Prescribing information. Novo Nordisk; 2023.
[7] Saxenda (liraglutide injection). Prescribing information. Novo Nordisk; 2023.

Literature Review

A search of the published medical literature revealed 1 study investigating the researchable question:

What is the safety of GLP1-RAs in patients with a family history of thyroid cancer?

Level of evidence

D - Case reports or unreliable data  Read more→



Please see Table 1 for your response.


 

Risk of Thyroid Cancer Associated with Use of Liraglutide and Other Antidiabetic Drugs in a US Commercially Insured Population

Design

Prospective cohort study

N= 390,096 

Objective

To quantify the association between the glucagon-like peptide-1 receptor agonist liraglutide and the risk of thyroid cancer (TC) compared to other antidiabetic drugs (AD)

Study Groups

Liraglutide (n= 27,287)

Comparators (n= 362,809)

Inclusion Criteria

Patients aged 18–89 years who initiated liraglutide or another AD following at least 6 months of continuous health plan enrollment with complete medical and pharmacy benefits; patients who were naïve to AD treatments, those who switched to cohort-defining therapy from another specific AD or drug class, and those who started cohort-defining therapy as an add-on treatment to existing therapy

Exclusion Criteria

Patients with claims for International Classification of Diseases, 9th Revision (ICD-9) diagnosis codes 193 (Malignant neoplasm of the thyroid gland) or V10.87 (Personal history of malignant neoplasm of the thyroid) during baseline 

Methods

Patients were selected from the Optum Research Database (ORD), a private research repository that houses eligibility information, medical claims, and pharmacy claims from a sizable, Optum-affiliated commercial health plan. Liraglutide was one of the study's ADs, along with the comparators exenatide, metformin, pioglitazone, a sulfonylurea (SU; glyburide, glipizide, glimepiride), and a DPP-4i (sitagliptin, saxagliptin, linagliptin). For all combined comparators as well as liraglutide initiators, baseline distributions of covariates were calculated. To quantify the risks related to starting liraglutide, using liraglutide recently, and cumulative exposure history, several analytic methods were used due to the complexity of drug use patterns and the various mechanisms of cancer occurrence.

Duration

Enrollment: February 2010 to November 2014

Follow-up: 17 months

Outcome Measures

Incidence rates of TC among liraglutide and comparators were assessed using relative risks estimated within propensity score-matched cohorts using intention to treat (ITT) and time on drug analyses. Latency effects and potential surveillance bias were evaluated

Baseline Characteristics

 

Liraglutide (n= 34,902)

All comparators (n= 34,902)

 

Age, years 

53 (46.0 to 60.0) 53 (46.0 to 59.0)  

Female

18,544 (53.1%) 18,507 (53%)  

Black

4,256 (12.2%) 4,941 (14.2%)  

The size of matched cohorts varied considerably due to patient characteristics and the prevalence of drugs on the market. All matched Initiators (initial and subsequent initiations) were included in these totals.

Results

Endpoint

Liraglutide (n= 34,902)

All comparators (n= 34,902)

Relative Risk 95% CI 

Time to Thyroid Cancer Diagnosis, days

257 (115 to 560) 348 (189 to 498)

1.66 (0.73 to 3.79)

Histology of Confirmed Thyroid Cancer Cases by Exposure

9

Exenatide; 2

DPP-4 Inhibitors: 4

Metformin: 37

Sulfonylureas: 14

Pioglitazone; 6

-- 

Abbreviation: CI, confidence interval

There was not much variance in the histology according to exposure: 85% had papillary cancer, follicular variant papillary cancer, or both; 7% had follicular cancer; 4% had other papillary, follicular, and follicular variants papillary cancer; and 4% had no known histology. All of the cases of microcarcinoma (PTMC) were papillary or a follicular variation of papillary. Patients who started taking liraglutide were more likely to have a PTMC among confirmed instances than all other comparators (67% versus 43%).

The analyses of time on the drug indicated no heightened risk associated with either prolonged duration or a higher cumulative dose of liraglutide.

Adverse Events

See above. 

Study Author Conclusions

Improved study designs are warranted to ascertain any true causal associations for newly marketed drugs, like liraglutide, in the presence of surveillance bias. A number of analyses were conducted and were generally consistent with no increase in the risk of TC among liraglutide initiators. Sensitivity analyses support the interpretation that the observed elevated point estimates likely resulted from surveillance bias.

InpharmD Researcher Critique

While all patients started an AD, many were already receiving other therapies, some of which continued throughout the study period. Even many naive initiators were likely to add or switch AD therapies during follow-up, which limits the evaluation of single therapy. Moreover, the study failed to address the potential correlation between certain risk factors, such as a family history of papillary thyroid cancer, and the likelihood of developing TC.  



References:

Funch D, Mortimer K, Ziyadeh NJ, et al. Risk of Thyroid Cancer Associated with Use of Liraglutide and Other Antidiabetic Drugs in a US Commercially Insured Population. Diabetes Metab Syndr Obes. 2021;14:2619-2629. Published 2021 Jun 10. doi:10.2147/DMSO.S305496