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. Author manuscript; available in PMC: 2024 Jan 10.
Published in final edited form as: N Engl J Med. 2020 Aug 27;383(9):825–835. doi: 10.1056/NEJMoa2005651

Table 1.

Baseline Characteristics

RET-mutant MTC RET Fusion-Positive Thyroid Cancer
Characteristic Vandetanib and/or Cabozantinib pre-treated Vandetanib/Cabozantinib Naïve
(N=55) (N=88) (N=19)
Age, Median (range) – yr 57 (17–84) 58 (15–82) 54 (25–88)
Sex – no. (%)
    Male 36 (66) 58 (66) 9 (47)
    Female 19 (35) 30 (34) 10 (53)
Race – no. (%) *
    White 49 (89) 75 (85) 14 (74)
    Asian 0 4 (5) 2 (11)
    Black or African American 1 (2) 1 (1) 1 (5)
    Other 5 (9) 8 (9) 2 (11)
ECOG performance status – no. (%)
    0 11 (20) 43 (49) 5 (26)
    1 41 (75) 42 (48) 12 (63)
    2 3 (6) 3 (3) 2 (11)
Thyroid cancer tumor type
    Medullary 55 (100) 88 (100)
    Papillary 13 (68)
    Poorly differentiated 3 (16)
    Hürthle cell 1 (5)
    Anaplastic 2 (11)
Median prior systemic regimens (range) – no. 2 (1–8) 0 (0–2) 4 (1–7)
Prior cabozantinib and/or vandetanib 55 (100) 0
    Vandetanib only 18 (33) 0
    Cabozantinib only 13 (24) 0
    Cabozantinib and vandetanib 24 (44) 0
Prior radioactive iodine (RAI) 16 (84)
Prior sorafenib and/or lenvatinib 13 (68)
Prior multikinase inhibitor therapy – no. (%) 55 (100) 7 (8) 15 (79)
    1 26 (47) 6 (7) 7 (37)
    ≥2 29 (53) 1 (1) 8 (42)
Prior non-multikinase inhibitor therapy– no. (%) 17 (31) 9 (10) 14 (74)
Brain metastases – no. (%) 4 (7) 2 (2) 6 (32)
RET alteration – no. (%)  
    RET M918T mutation 33 (60) 49 (56) -
    RET V804M/L mutation 5 (9) 6 (7) -
    RET extracellular cysteine mutation** 7 (13) 20 (23) -
    Other mutations 10 (18) 13 (15) -
    CCDC6-RET fusion - - 9 (47)
    NCOA4-RET fusion - - 6 (32)
    Other RET fusion - - 4 (21)
*

Includes 1 RET fusion-positive patient with missing race

**

Extracellular cysteine mutation defined as mutation including at least 1 of the following cysteine residues: 609, 611, 618, 620, 630, and 634.

Other includes: D631-L633delinsE, E632-L633del, A883F, D631-L633delinsV, L790F, D898-E901del, D898_E901del + D903_S904delinsEP, K666N, T636-V637insCRT, D378-G385delinsE.

Fusions identified in single tumors included CCDC186-RET, ERC1-RET, KTN1-RET, and RUFY3-RET.

Eastern Cooperative Oncology Group (ECOG) performance status scores range from 0 to 5, with higher scores indicating greater disability. Total % may be different than the sum of the individual components due to rounding.