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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]