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Table 1 Patient demographic and clinical characteristics

From: A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia

Sample characteristics

Stage 1 (n = 14)

Stage 2 (n = 18)

Recruitment source

 MDS Foundation

6 (43%)

11 (61%)

 Physician referral

8 (57%)

7 (39%)

Diagnosis type

 Higher-risk MDS

11 (79%)

14 (78%)

 Low-blast count AML

1 (7%)

1 (6%)

 CMML

2 (14%)

3 (17%)

Age, years

 Mean (SD)

68.8 (±8.48)

68.1 (±10.1)

 Minimum

54

50

 Maximum

83

83

Gender

 Female

9 (64%)

10 (56%)

 Male

5 (36%)

8 (44%)

Education level

 Post-graduate degree

5 (36%)

5 (28%)

 Undergraduate degree

1 (7%)

1 (6%)

 Some college

3 (21%)

3 (17%)

 Trade/technical degree

0 (0%)

1 (6%)

 High school/General Educational Development equivalent

4 (29%)

6 (33%)

 Some high school or less

1 (7%)

2 (12%)

Employment status

 Retired

8 (57%)

10 (56%)

 Part-time

3 (21%)

3 (17%)

 Full-time

2 (14%)

1 (6%)

 Disability

1 (7%)

3 (17%)

 Not employed

0 (0%)

1 (6%)

Eastern Cooperative Oncology Group status

 Clinician-reported

0 – fully active

1 (7%)

1 (6%)

1 – restricted in physically strenuous activity

7 (50%)

6 (33%)

 Patient-reported

1 – difficulty with physically strenuous activity

1 (17%)

5 (28%)

2 – able to walk and care for self, restricted in work activities

5 (36%)

6 (33%)

Classification (clinician-reported only)

n = 8

n = 7

 FAB- Refractory anemia

1 (13%)

1 (14%)

 WHO-RAEB1

3 (38%)

2 (29%)

 WHO-RAEB2

1 (13%)

1 (14%)

 FAB- Refractory anemia, WHO RAEB1

2 (25%)

2 (29%)

 FAB-CMML and WHO-CMML1

1 (12%)

1 (14%)

Prognostic risk categoryb (MDS only)

n = 11

n = 10

 Very high

4 (36%)

3 (30%)

 High

3 (27%)

3 (30%)

 Intermediate

2 (18%)

2 (20%)

 Unknown

2 (18%)

2 (20%)

Hemoglobin level

 7.0–9.9

6 (43%)

9 (50%)

 10.0–11.9

6 (43%)

6 (33%)

 12.0–13.0

2 (14%)

3 (17%)

% myeloblasts in bone marrow (patient-reported only)

n = 6

n = 10

 < 1%

1 (17%)

1 (10%)

 1–9.9%

3 (50%)

5 (50%)

 10–11%

1 (17%)

1 (10%)

 Missing data

1 (17%)

3 (30%)

Treatmenta

 Azacitidine

9 (64%)

12 (67%)

 Decitabine

2 (14%)

2 (11%)

 G-CSF; copper gluconate; ondansetron; sulfamethoxazole / trimethoprim; levofloxacin; valacyclovir; acetaminophen/hydrocodone; oxycodone; prochlorperazine

1 (7%) each

1 (6%) each

 Unidentified clinical trial study drug

0 (0%)

1 (6%)

 No treatment

0 (0%)

1 (6%)

  1. Abbreviations: AML Acute myeloid leukemia, CMML Chronic myelomonocytic leukemia, FAB French American British, G-CSF Granulocyte-colony stimulating factor, MDS Myelodysplastic syndromes, RAEB Refractory anemia with excess blasts, SD Standard deviation, WHO World health organization
  2. aNot mutually exclusive, missing treatment data for 1 patient
  3. bPer International Prognostic Scoring System-Revised [30]