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Table 2 List of final questionnaire items and source

From: Development of a patient-reported outcome questionnaire for aplastic anemia and paroxysmal nocturnal hemoglobinuria (PRO-AA/PNH)

Questionnaire item

Source

Fatiguea, b

PRO-CTCAE Symptom Term: Fatigue

Fevera

Center specific diagnostic and treatment guidelines based on Petersdorf et al. [34]

Bleedinga, b

Modified WHO Bleeding Scale [13]

Dyspneaa, b

PRO-CTCAE Symptom Term: Shortness of breath

Paina

According to PRO-CTCAE Symptom Term: General pain

Mooda, b

According to PRO-CTCAE Symptom Term: Sad; and based on an instrument by Whooley et al. [35]

Concentration/memorya, b

Combination of PRO-CTCAE Symptom Term: Concentration and PRO-CTCAE Symptom Term: Memory

Palpitationsa, b

PRO-CTCAE Symptom Term: Heart palpitations

Gastro-intestinal problemsb

PRO-CTCAE Symptom Terms: Nausea, Vomiting, Heartburn, Gas, Bloating, Constipation and Diarrhea

Open question on other symptom

N/A

Items only for AA questionnaire

 Palpitationsa

PRO-CTCAE Symptom Term: Heart palpitations

 Tremora

According to CTCAE V5.0 Term: Tremor and adopted to PRO-CTCAE style

 Muscle crampsa, b

According to CTCAE V5.0 Term: Muscle cramp and adopted to PRO-CTCAE style.

 Paresthesia, numbnessa

PRO-CTCAE Symptom Term: Numbness & tingling

 Trouble doing strenuous activitiesb

EORTC-QLQ-C30

 Dizzinessb

PRO-CTCAE Symptom Term: Dizziness

Items only for PNH questionnaire

 Hemoglobinuriaa, b

N/A

 Jaundicea

N/A

 Dysphagiaa, b

According to CTCAE V5.0 Term: Dysphagia and adopted to the PRO-CTCAE Symptom Term: Difficulty swallowing

 Erectile dysfunctiona

PRO-CTCAE Symptom Term: Achieve and maintain erection

 Itchingb

PRO-CTCAE Symptom Term: Itching

 Painb

According to PRO-CTCAE Symptom Term: General pain

  1. aexperts choice; bpatients choice WHO World Health Organization, PRO-CTCAE patient-reported outcome version of the Common Terminology Criteria for Adverse Events, EORTC-QLQ-C30 European Organization for Research and Treatment of Cancer Quality of Life of Cancer Patients, N/A not applicable