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Table 5 Progression trend of the ‘Fatigue’ and ‘Pain’ concepts since starting study treatment up to Week 25 and its correspondence with quantitative assessments

From: How to address the challenges of evaluating treatment benefits-risks in rare diseases? A convergent mixed methods approach applied within a Merkel cell carcinoma phase 2 clinical trial

Patient #

Qualitative assessment

Quantitative assessment

Overall response by IERC per RECISTa

Change in FACT-M item related to Fatigue

Change in FACT-M item related to Pain

Fatigue

Pain

HI7. I feel fatigued

GP4. I have pain

M1. I have pain at my melanoma site or surgical site

M5. I have aches and pains in my bones

M13. Movement of my swollen area is painful

1

No change (still tired)

No change (no pain)

Partial/complete responder

No change

Improvement

No change

Worsening

No change

2

Improvement

No change (no pain)

Partial/complete responder

No change

Worsening

Improvement

No change

No change

3

No change (still tired)

No change (no pain)

Partial/complete responder

No change

Worsening

No change

Worsening

No change

4

Improvement

ND

Partial/complete responder

Improvement

No change

No change

No change

No change

5

ND

No change (no pain)

Partial/complete responder

No change

No change

Improvement

Improvement

No change

6

Improvement

Improvement

Partial/complete responder

No change

No change

No change

Worsening

No change

7

Improvement

Improvement

Partial/complete responder

Improvement

Improvement

Improvement

Worsening

No change

8

ND

Improvement

Partial/complete responder

NC

NC

NC

NC

NC

9

ND

No change (still pain)

Progressive disease

Worsening

Worsening

No change

No change

No change

  1. NC Not computed, due to missing baseline data, ND Not determined, trend not possible to determine as data was not reported spontaneously by the patient and was not probed at one of the time-point interviews, IERC Independent Endpoint Review Committee; RECIST: Response Evaluation Criteria In Solid Tumors version 1.1
  2. aOverall response by IERC per RECIST identical at both Week 13 and Week 25 for all patients