Real-World Clinical Profile, Treatment Patterns And Patient-Reported Outcomes for Thyroid Cancer in Japan

Figures & data

Table 1. Patient demographics and treatment history by tumor type (patient-record form).

Characteristic	MTC (n = 56)	PTC (n = 106)
Age, years, median (range)	67.5 (38–88)	68.5 (23–88)
Sex, n (%)
Male	30 (54)	21 (20)
Female	26 (46)	85 (80)
BMI, mean (SD)	22.5 (2.4)	22.4 (3.3)
Ethnic origin, n (%)
Japanese	55 (98)	105 (99)
Korean	1 (2)	0
Chinese	0 (0)	1 (1)
Disease state at data capture, n (%)
Locally advanced	19 (34)	43 (41)
Metastatic	34 (61)	61 (58)
Unknown/not assessed	3 (5)	2 (2)
Sites of metastases at data capture, n (%)^†
Bone	8 (24)	9 (15)
Breast	1 (3)	0 (0)
Brain	2 (6)	2 (3)
Liver	6 (18)	1 (2)
Lungs	8 (24)	31 (51)
Lymph nodes	16 (47)	26 (43)
Skin	1 (3)	5 (8)
Unknown	3 (9)	4 (7)
Life expectancy at data capture, n (%)
<5 years	7 (12)	18 (17)
5–10 years	19 (34)	30 (28)
>10 years	13 (23)	26 (25)
Unknown	17 (30)	32 (30)
ECOG score at data capture
0	21 (38)	48 (45)
1	13 (23)	34 (32)
2	11 (20)	17 (16)
3	6 (11)	3 (3)
4	0 (0)	0 (0)
NA	5 (9)	4 (4)
ECOG score at initial diagnosis
0	29 (52)	70 (66)
1	15 (27)	23 (22)
2	3 (5)	3 (3)
3	0 (0)	1 (1)
4	1 (2)	0 (0)
NA	8 (14)	9 (8)
Received current/prior treatment, n (%)
Surgery (e.g., total thyroidectomy, lymph node removal)	28 (50)	74 (70)
Radiotherapy	10 (18)	46 (43)
Drug treatment	27 (48)	51 (48)
Best supportive care	7 (12)	18 (17)
None of the above	12 (21)	8 (8)

† Among 34 MTC patients and 61 PTC patients with metastatic diseases.

ECOG: Eastern Cooperative Oncology Group; MTC: Medullary thyroid cancer; NA: Not assessed/unknown; PTC: Papillary thyroid cancer; SD: Standard deviation.

Table 2. Patient testing and results for rearranged during transfection alterations by tumor type (patient-record form).

	MTC	PTC
RET testing conducted to aid diagnosis, n (%)^†	n = 56	n = 106
Yes	31 (55)	25 (24)
No	25 (45)	81 (76)
RET test results, n (%)^‡	n = 31	n = 25
Positive	13 (42)^§	4 (16)
Negative	15 (48)	18 (72)
Inconclusive	0 (0)	1 (4)
Awaiting result	3 (10)	2 (8)
Subtype of MEN2, n (%)^¶	n = 13	NA
MEN2A	5 (38)	NA
MEN2B	4 (31)	NA
Familial MTC	3 (23)	NA
Unknown	1 (8)	NA
Patient with positive RET test offered genetic counseling, n (%)	n = 13	NA
Yes	10 (77)	NA
No/unknown	3 (23)	NA
Patient received genetic counseling, n (%)^#	n = 10	NA
Yes	6 (60)	NA
No	4 (40)	NA

† RET tests for MTC patients: RET germline tests for RET mutation. RET tests for PTC patients: RET fusion tests.

‡ Among patients who received RET tests for diagnosis.

§ Germline mutations.

¶ Among patients who received a positive germline RET test.

# Among patients who were offered genetic counseling.

MEN2: Multiple endocrine neoplasia type 2; MTC: Medullary thyroid cancer; NA: Not applicable; PTC: Papillary thyroid cancer; RET: Rearranged during transfection.

Table 3. Systemic antitumor drug treatment by tumor type (patient-record form).

	MTC	PTC
Ever received systemic antitumor treatment	n = 56	n = 106
Yes	27 (48)	51 (48)
No	29 (52)	55 (52)
First-line therapy^†	n = 27	n = 51
Cisplatin	2 (7)	5 (10)
Cyclophosphamide	1 (4)	0 (0)
5-FU	4 (15)	3 (6)
Sorafenib	5 (19)	14 (27)
Lenvatinib	7 (26)	26 (51)
Vandetanib	6 (22)	1 (2)
Clinical trial regimen	4 (15)	0 (0)
Other	0 (0)	1 (2)
Second-line therapy^‡	n = 5	n = 1
Cisplatin	1 (20)	1 (100)
5-FU	1 (20)	0 (0)
Lenvatinib	4 (80)	0 (0)
Third-line therapy^§	n = 2	n = 0
Vandetanib	2 (100)	0 (0)
Time from initial diagnosis to initiation of MKI (months)^¶	n = 16	n = 35
Mean (SD)	23.6 (26.9)	29.3 (45.1)
Range	0.2–101.5	0.5–241.1
Time from recurrence/metastasis to first MKI (months)^#	n = 10	n = 13
Mean (SD)	34.5 (28.5)	18.5 (32.5)
Range	1.4–101.5	0–110.0
Year of first-line of therapy^††	n = 21	n = 50
2013	1 (5)	1 (2)
2014	0 (0)	1 (2)
2015	1 (5)	4 (8)
2016	0 (0)	4 (8)
2017	1 (5)	1 (2)
2018	2 (10)	5 (10)
2019	8 (38)	17 (34)
2020	8 (38)	17 (34)

† Patients who received first-line therapy.

‡ Patients who received second-line therapy.

§ Patients who received third-line therapy.

¶ Patients with a known date of initial diagnosis and date of MKI initiation.

# Patients with a known date of recurrence/metastasis and date of MKI.

†† Patients who received first-line drug treatment and have a known start date.

5-FU: 5-fluorouracil; MKI: Multikinase inhibitor; MTC: Medullary thyroid cancer; PTC: Papillary thyroid cancer; SD: Standard deviation.

Table 4. Physician-reported patient side effects with multikinase inhibitor therapy by tumor type (patient-record form).

Side effect (n, %)	MTC (n = 10^†)	PTC (n = 23^†)
Loss of appetite	5 (50)	11 (48)
Decreased appetite	3 (30)	6 (26)
Constipation	2 (20)	3 (13)
Diarrhea	2 (20)	3 (13)
Hypertension	2 (20)	6 (26)
Nausea	2 (20)	7 (30)
Fatigue	2 (20)	6 (26)
Hand foot syndrome	1 (10)	10 (43)
Anemia	1 (10)	3 (13)
Heart arrhythmias	1 (10)	1 (4)
Headaches	1 (10)	0 (0)
Weight loss	1 (10)	2 (9)
Abdominal pain	1 (10)	0 (0)
QT prolongation	2 (20)	0 (0)
Dyspnea	1 (10)	1 (4)
Cough	1 (10)	1 (4)
Hair loss/thinning	0 (0)	1 (4)
Rash	1 (10)	1 (4)
Easy bruising and bleeding	1 (10)	0 (0)
Mouth sores	0 (0)	0 (0)
Gastrointestinal perforation	0 (0)	0 (0)
Dyspepsia	0 (0)	0 (0)
Dermatitis acneiform/acne	1 (10)	0 (0)
Pruritus	1 (10)	0 (0)
Dysphonia	1 (10)	0 (0)
Myalgia	0 (0)	1 (4)
Vomiting	0 (0)	1 (4)
Stomatitis	0 (0)	3 (13)
Increased AST	0 (0)	2 (9)
Increased ALT	0 (0)	2 (9)
Other	1 (10)	0 (0)

† Patients who received MKIs as the current/most recent therapy and reported side effects.

ALT: Alanine aminotransferase; AST: Aspartate aminotransferase; MKI: Multikinase inhibitor; MTC: Medullary thyroid cancer; PTC: Papillary thyroid cancer.

Table 5. Patient 5-level EuroQol 5 Dimension questionnaire, Functional Assessment of Cancer Therapy-General score, and Work Productivity and Activity Impairment mean activity impairment (patient self-completion form).

Scores at data capture
	MTC	PTC
EQ-5D-5L^†	n = 43	n = 86
Mean (SD)	0.76 (0.20)	0.79 (0.14)
FACT-G^‡	n = 43	n = 83
Mean (SD)	63.2 (18.9)	65.4 (16.2)
WPAI^§	n = 44	n = 85
Mean (SD)	45.0% (29.5)	37.7% (25.5)
Scores while on first-line MKI
	MTC	PTC
EQ-5D-5L	n = 11	n = 32
Mean (SD)	0.80 (0.14)	0.80 (0.15)
FACT-G	n = 11	n = 31
Mean (SD)	75.3 (26.7)	64.5 (21.0)
WPAI	n = 11	n = 33
Mean (SD)	30.9% (31.1)	38.8% (27.4)

† Patients who completed the entire EQ-5D-5L; health utility index scores were generated using specific tariffs for Japan.

‡ Patients who completed the entire FACT-G.

§ Patients who reported their ability to perform normal daily activities.

EQ-5D-5L: 5-level EuroQol 5 Dimension questionnaire; FACT-G: Functional Assessment of Cancer Therapy-General; MKI: Multikinase inhibitor; MTC: Medullary thyroid cancer; PTC: Papillary thyroid cancer; SD: Standard deviation; WPAI: Work Productivity and Activity Impairment.

Data sharing statement

The dataset supporting the conclusions of this article are included within the article (and its additional files).