Insights From Randomized Controlled Trials on the Efficacy and Safety of Pharmacological Therapies for Differentiated Thyroid Cancer: A Systematic Review

Author(s)

Bisen R, Thakur L, Shivsingwale G, Gunde D, Mukta Y
SRS Pharmaceuticals Pvt. Ltd., Mumbai, MH, India

OBJECTIVES: Differentiated thyroid cancer (DTC) patients are offered multiple targeted therapeutics, but it is unclear which pharmaceutical therapies are the most effective and safe. We conducted a systematic review to investigate the efficacy and safety of targeted treatments.

METHODS: A comprehensive searches of MEDLINE®, Embase®, Evidence-based Medicine Reviews, and grey literature were conducted. All records were screened against predefined inclusion criteria. All included studies were extracted and evaluated using NICE's critical appraisal checklist.

RESULTS: A total of 18 studies from 5,334 publications were eligible. Two studies assessed treatments in the DTC population, while 16 studies addressed the radioactive iodine-resistant (RAIR) sub-population. DTC patients treated with nintedanib had a small increase in median progression-free survival (PFS) compared to placebo (3.71 vs 2.86 months). Selumetinib with radioactive iodine had a minor effect on complete remission rates (40 vs. 38%), but its higher adverse event profile raised concerns. However, the landscape for RAIR-DTC changes substantially. Clinical benefits were seen with donafenib, lenvatinib, anlotinib, apatinib, cabozantinib, cediranib, dabrafenib+trametinib, sorafenib, paazopanib, and vandetanib, ranging from 2% (intermittent pazopanib) to 69.9% (lenvatinib), However, five studies failed to reach the median overall survival (OS), suggesting that longer survival is possible. Furthermore, cabozantinib significantly improved PFS when compared to placebo, with anlotinib reaching a remarkable 40.54 months. Adverse events (mainly grade 1-2) were reported across all studies, with hypertension, diarrhea, and hand-foot skin reactions being the most common. In spite of its exceptional efficacy, lenvatinib caused an adverse event rate of 75.9% (grade 3).

CONCLUSIONS: Despite modest progress in the general DTC population, lenvatinib has revolutionized RAIR-DTC treatment by delivering a higher response rate and extended survival, although with a higher adverse event risk. Therefore, further DTC research needs to be conducted both in the broader population and in the RAIR-DTC population.

Conference/Value in Health Info

2024-11, ISPOR Europe 2024, Barcelona, Spain

Value in Health, Volume 27, Issue 12, S2 (December 2024)

Code

CO106

Topic

Clinical Outcomes, Study Approaches

Topic Subcategory

Clinical Outcomes Assessment, Comparative Effectiveness or Efficacy, Literature Review & Synthesis

Disease

Oncology

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