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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