Medullary thyroid cancer

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Note: these are regimens tested in histology-specific populations, please see the main thyroid cancer page for other regimens.

6 regimens on this page
7 variants on this page


Guidelines

ATA

  • 2015: Guidelines for the management of medullary thyroid carcinoma PubMed

ESMO

Older

NCCN

All lines of therapy

Cabozantinib monotherapy

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Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Kurzrock et al. 2011 (XL184-001) 2005-2008 Non-randomized
Elisei et al. 2013 (EXAM) 2008-2011 Phase III (E-RT-esc) Placebo Superior PFS

Targeted therapy

Continued indefinitely

References

  1. XL184-001: Kurzrock R, Sherman SI, Ball DW, Forastiere AA, Cohen RB, Mehra R, Pfister DG, Cohen EE, Janisch L, Nauling F, Hong DS, Ng CS, Ye L, Gagel RF, Frye J, Müller T, Ratain MJ, Salgia R. Activity of XL184 (Cabozantinib), an oral tyrosine kinase inhibitor, in patients with medullary thyroid cancer. J Clin Oncol. 2011 Jul 1;29(19):2660-6. Epub 2011 May 23. link to original article contains verified protocol link to PMC article PubMed NCT00215605
  2. EXAM: Elisei R, Schlumberger MJ, Müller SP, Schöffski P, Brose MS, Shah MH, Licitra L, Jarzab B, Medvedev V, Kreissl MC, Niederle B, Cohen EE, Wirth LJ, Ali H, Hessel C, Yaron Y, Ball D, Nelkin B, Sherman SI. Cabozantinib in progressive medullary thyroid cancer. J Clin Oncol. 2013 Oct 10;31(29):3639-46. Epub 2013 Sep 3. Erratum in: J Clin Oncol. 2014 Jun 10;32(17):1864. link to original article contains verified protocol link to PMC article PubMed NCT00704730
    1. Update: Schlumberger M, Elisei R, Müller S, Schöffski P, Brose M, Shah M, Licitra L, Krajewska J, Kreissl MC, Niederle B, Cohen EEW, Wirth L, Ali H, Clary DO, Yaron Y, Mangeshkar M, Ball D, Nelkin B, Sherman S. Overall survival analysis of EXAM, a phase III trial of cabozantinib in patients with radiographically progressive medullary thyroid carcinoma. Ann Oncol. 2017 Nov 1;28(11):2813-2819. link to original article link to PMC article PubMed

Placebo

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Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Wells et al. 2011 (ZETA) 2006-2007 Phase III (C) Vandetanib Inferior PFS
Elisei et al. 2013 (EXAM) 2008-2011 Phase III (C) Cabozantinib Inferior PFS

No active antineoplastic treatment. Included for reference purposes only.

References

  1. ZETA: Wells SA Jr, Robinson BG, Gagel RF, Dralle H, Fagin JA, Santoro M, Baudin E, Elisei R, Jarzab B, Vasselli JR, Read J, Langmuir P, Ryan AJ, Schlumberger MJ. Vandetanib in patients with locally advanced or metastatic medullary thyroid cancer: a randomized, double-blind phase III trial. J Clin Oncol. 2012 Jan 10;30(2):134-41. Epub 2011 Oct 24. link to original article contains verified protocol link to PMC article PubMed NCT00410761
  2. EXAM: Elisei R, Schlumberger MJ, Müller SP, Schöffski P, Brose MS, Shah MH, Licitra L, Jarzab B, Medvedev V, Kreissl MC, Niederle B, Cohen EE, Wirth LJ, Ali H, Hessel C, Yaron Y, Ball D, Nelkin B, Sherman SI. Cabozantinib in progressive medullary thyroid cancer. J Clin Oncol. 2013 Oct 10;31(29):3639-46. Epub 2013 Sep 3. Erratum in: J Clin Oncol. 2014 Jun 10;32(17):1864. link to original article contains verified protocol link to PMC article PubMed NCT00704730

Sorafenib monotherapy

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Regimen

Study Evidence
Lam et al. 2010 (OSU 06054) Phase II

Targeted therapy

Continued indefinitely

References

  1. OSU 06054: Lam ET, Ringel MD, Kloos RT, Prior TW, Knopp MV, Liang J, Sammet S, Hall NC, Wakely PE Jr, Vasko VV, Saji M, Snyder PJ, Wei L, Arbogast D, Collamore M, Wright JJ, Moley JF, Villalona-Calero MA, Shah MH. Phase II clinical trial of sorafenib in metastatic medullary thyroid cancer. J Clin Oncol. 2010 May 10;28(14):2323-30. Epub 2010 Apr 5. link to original article contains verified protocol link to PMC article PubMed NCT00390325

Sunitinib monotherapy

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Regimen

Study Years of enrollment Evidence
Carr et al. 2010 2007-2009 Phase II

Targeted therapy

Continued indefinitely

References

  1. Carr LL, Mankoff DA, Goulart BH, Eaton KD, Capell PT, Kell EM, Bauman JE, Martins RG. Phase II study of daily sunitinib in FDG-PET-positive, iodine-refractory differentiated thyroid cancer and metastatic medullary carcinoma of the thyroid with functional imaging correlation. Clin Cancer Res. 2010 Nov 1;16(21):5260-8. Epub 2010 Sep 16. link to original article contains verified protocol link to PMC article PubMed

Vandetanib monotherapy

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Regimen variant #1, 100 mg/day

Study Evidence
Robinson et al. 2010 Phase II, <20 patients

Targeted therapy

Continued indefinitely

Subsequent treatment

  • At progression, eligible patients could increase the dose to 300 mg PO once per day; see paper for details

Regimen variant #2, 300 mg/day

Study Years of enrollment Evidence Comparator Comparative Efficacy
Wells et al. 2010 2004-2006 Phase II
Wells et al. 2011 (ZETA) 2006-2007 Phase III (E-RT-esc) Placebo Superior PFS

Targeted therapy

Continued indefinitely

References

  1. Wells SA Jr, Gosnell JE, Gagel RF, Moley J, Pfister D, Sosa JA, Skinner M, Krebs A, Vasselli J, Schlumberger M. Vandetanib for the treatment of patients with locally advanced or metastatic hereditary medullary thyroid cancer. J Clin Oncol. 2010 Feb 10;28(5):767-72. Epub 2010 Jan 11. link to original article contains verified protocol link to PMC article PubMed
  2. Robinson BG, Paz-Ares L, Krebs A, Vasselli J, Haddad R. Vandetanib (100 mg) in patients with locally advanced or metastatic hereditary medullary thyroid cancer. J Clin Endocrinol Metab. 2010 Jun;95(6):2664-71. Epub 2010 Apr 6. link to original article contains verified protocol link to PMC article PubMed content property of HemOnc.org
  3. ZETA: Wells SA Jr, Robinson BG, Gagel RF, Dralle H, Fagin JA, Santoro M, Baudin E, Elisei R, Jarzab B, Vasselli JR, Read J, Langmuir P, Ryan AJ, Schlumberger MJ. Vandetanib in patients with locally advanced or metastatic medullary thyroid cancer: a randomized, double-blind phase III trial. J Clin Oncol. 2012 Jan 10;30(2):134-41. Epub 2011 Oct 24. link to original article contains verified protocol link to PMC article PubMed NCT00410761

Advanced or metastatic disease

Axitinib monotherapy

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Regimen

Study Evidence
Locati et al. 2014 (A4061027) Phase II

Targeted therapy

Continued indefinitely

References

  1. A4061027: Locati LD, Licitra L, Agate L, Ou SH, Boucher A, Jarzab B, Qin S, Kane MA, Wirth LJ, Chen C, Kim S, Ingrosso A, Pithavala YK, Bycott P, Cohen EE. Treatment of advanced thyroid cancer with axitinib: Phase 2 study with pharmacokinetic/pharmacodynamic and quality-of-life assessments. Cancer. 2014 Sep 1;120(17):2694-703. Epub 2014 May 20. link to original article contains verified protocol PubMed NCT00389441

Sunitinib monotherapy

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Regimen

Study Evidence Efficacy
Carr et al. 2010 Phase II ORR: 31% (95% CI: 16-47)

Targeted therapy

Continued indefinitely

References

  1. Carr LL, Mankoff DA, Goulart BH, Eaton KD, Capell PT, Kell EM, Bauman JE, Martins RG. Phase II study of daily sunitinib in FDG-PET-positive, iodine-refractory differentiated thyroid cancer and metastatic medullary carcinoma of the thyroid with functional imaging correlation. Clin Cancer Res. 2010 Nov 1;16(21):5260-8. Epub 2010 Sep 16. link to original article contains verified protocol link to PMC article PubMed