Patient characteristics
| Variable . | Baseline n = 2205 . | First visit n = 1861 . | Second visit n = 1506 . |
|---|---|---|---|
| Median (IQR)/n (%) . | Median (IQR)/n (%) . | Median (IQR)/n (%) . | |
| Age, y | 74 (67-80) | 74 (67-80) | 74 (67-80) |
| Age, y (categorical) | |||
| <60 | 228 (10) | 181 (10) | 134 (9) |
| 60-75 | 893 (41) | 763 (41) | 621 (41) |
| ≥75 | 1084 (49) | 917 (49) | 751 (50) |
| Female | 849 (38.5) | 734 (39) | 610 (40) |
| Time since diagnosis, months | 1.4 (0.7-2.3) | 6.5 (5.9-7.6) | 12.8 (11.8-14.4) |
| EQ-5D index value | 0.8 (0.6-1.0) | 0.8 (0.6-1.0) | 0.8 (0.6-1.0) |
| Low EQ-5D index value (<0.779) | 1022 (46) | 643 (35) | 541 (36) |
| Visual analog score | 70 (58-85) | 70 (60-85) | 70 (60-80) |
| Low visual analog score (<0.7) | 914 (42) | 618 (33) | 485 (32) |
| Impaired EQ-5D – mobility∗ | 750 (34) | 484 (26) | 414 (28) |
| Impaired EQ-5D – self-care∗ | 243 (11) | 192 (10) | 166 (11) |
| Impaired EQ-5D – usual activities∗ | 659 (30) | 465 (25) | 402 (27) |
| Impaired EQ-5D – pain/discomfort∗ | 908 (41) | 569 (31) | 479 (32) |
| Impaired EQ-5D – anxiety/depression∗ | 688 (31) | 435 (23) | 346 (23) |
| WHO subtypes | |||
| RA | 381 (17) | 204 (11) | 167 (11) |
| RARS | 345 (16) | 234 (13) | 209 (14) |
| RCMD | 854 (39) | 506 (27) | 398 (26) |
| RCMD-RS | 127 (6) | 60 (3) | 48 (3) |
| RAEB-1 | 264 (12) | 154 (8) | 114 (8) |
| RAEB-2 | 9 (0) | 36 (2) | 32 (2) |
| MDS-U | 94 (4.3) | 51 (3) | 36 (2) |
| Del 5q | 130 (6) | 88 (5) | 76 (5) |
| IPSS-R (continuous, score unit) | 2 (1.5-3.0) | 2 (1.0-3.0) | 2 (1.0-3.0) |
| IPSS-R, (categorical) | |||
| Very low | 559 (25) | 505 (27) | 430 (29) |
| Low | 900 (41) | 675 (36) | 546 (36) |
| Intermediate | 327 (15) | 235 (13) | 199 (13) |
| High | 80 (4) | 78 (4) | 55 (4) |
| Very high | 6 (0) | 17 (1) | 19 (1) |
| Hb, g/dL | 10.2 (9.0-11.4) | 10.6 (9.3-11.8) | 10.5 (9.2-11.9) |
| Hb, g/dL (categorical) | |||
| ≥10 | 1197 (54) | 1140 (61) | 890 (59) |
| 8-10 | 791 (36) | 520 (28) | 438 (29) |
| <8 | 202 (9) | 156 (8) | 136 (9) |
| Ferritin, μg/L (categorical) | |||
| ≥1000 | 164 (7) | 181 (10) | 167 (11) |
| 300-1000 | 624 (28) | 337 (18) | 247 (16) |
| ≤300 | 726 (33) | 409 (22) | 339 (23) |
| Erythropoietin, IU/L (categorical) | |||
| >500 | 75 (3) | 36 (2) | 21 (1) |
| 100-500 | 253 (12) | 112 (6) | 82 (5) |
| <100 | 680 (31) | 303 (16) | 221 (15) |
| Platelets, ×109/L (categorical) | |||
| ≥100 | 1634 (74) | 1293 (70) | 1074 (71) |
| 50-100 | 375 (17) | 321 (17) | 220 (15) |
| <50 | 171 (8) | 186 (10) | 162 (11) |
| Neutrophils, ×109/L | 2.4 (1.0-3.8) | 2.4 (1.3-3.8) | 2.3 (1.3-3.7) |
| Neutrophils, ≤1 × 109/L | 335 (15) | 298 (16) | 235 (16) |
| Therapies received | |||
| ESA | 331 (15) | 514 (28) | 424 (23) |
| ESA with Hb ≤ 10 g/dL | 258 (12) | 239 (13) | 184 (12) |
| Granulocyte colony-stimulating factor | 27 (1) | 54 (3) | 55 (4) |
| Iron chelation | 17 (1) | 59 (3) | 83 (6) |
| Immunosuppressive therapy | 7 (0) | 15 (1) | 17 (1) |
| Demethylating agent | 2 (0) | 28 (2) | 30 (2) |
| Lenalidomide | 7 (0) | 38 (2) | 45 (3) |
| Transfusion dependence† | 500 (23) | 455 (25) | 332 (22) |
| Transfusion density‡ | 0 (0.0-0.5) | 0 (0.0-0.5) | 0 (0.0-0.5) |
| Cumulative number of transfusions | 0 (0.0-1.0) | 0 (0.0-2.0) | 0 (0.0-2.0) |
| MDS progression§ | - | 200 (11) | 186 (12) |
| KPS (continuous) | 90 (80-100) | 90 (80-100) | 90 (80-100) |
| KPS (categorical) | |||
| Able to work (80-100) | 1428 (65) | 1110 (60) | 874 (58) |
| Unable to work (50-80) | 392 (18) | 329 (18) | 248 (17) |
| Unable to care for self (<50) | 28 (1) | 26 (1) | 26 (2) |
| MDS-CI (continuous) | 0 (0.0-2.0) | 0 (0.0-0.0) | 0 (0.0-1.0) |
| MDS-CI (categorical) | |||
| Low risk (0) | 1355 (62) | 1399 (75) | 1091 (72) |
| Intermediate risk (1-2) | 716 (33) | 398 (21) | 352 (23) |
| High risk (≥3) | 126 (6) | 46 (3) | 46 (3) |
| HCT-CI (continuous) | 1 (0.0-3.0) | 0 (0.0-2.0) | 0 (0.0-2.0) |
| HCT-CI (categorical) | |||
| Low risk (0) | 771 (35) | 1009 (54) | 759 (50) |
| Intermediate risk (1-2) | 799 (36) | 545 (29) | 461 (31) |
| High risk (≥3) | 635 (29) | 307 (17) | 286 (19) |
| BMI, kg/m2 | 26 (23.8-29.0) | 26 (23.8-29.1) | 26 (23.5-29.1) |
| BMI, kg/m2 (categorical) | |||
| Underweight <23 | 311 (14) | 268 (14) | 242 (16) |
| Weight range (23-29.9) | 1084 (50) | 911 (49) | 733 (49) |
| Overweight ≥30 | 320 (15) | 282 (15) | 231 (15) |
| Variable . | Baseline n = 2205 . | First visit n = 1861 . | Second visit n = 1506 . |
|---|---|---|---|
| Median (IQR)/n (%) . | Median (IQR)/n (%) . | Median (IQR)/n (%) . | |
| Age, y | 74 (67-80) | 74 (67-80) | 74 (67-80) |
| Age, y (categorical) | |||
| <60 | 228 (10) | 181 (10) | 134 (9) |
| 60-75 | 893 (41) | 763 (41) | 621 (41) |
| ≥75 | 1084 (49) | 917 (49) | 751 (50) |
| Female | 849 (38.5) | 734 (39) | 610 (40) |
| Time since diagnosis, months | 1.4 (0.7-2.3) | 6.5 (5.9-7.6) | 12.8 (11.8-14.4) |
| EQ-5D index value | 0.8 (0.6-1.0) | 0.8 (0.6-1.0) | 0.8 (0.6-1.0) |
| Low EQ-5D index value (<0.779) | 1022 (46) | 643 (35) | 541 (36) |
| Visual analog score | 70 (58-85) | 70 (60-85) | 70 (60-80) |
| Low visual analog score (<0.7) | 914 (42) | 618 (33) | 485 (32) |
| Impaired EQ-5D – mobility∗ | 750 (34) | 484 (26) | 414 (28) |
| Impaired EQ-5D – self-care∗ | 243 (11) | 192 (10) | 166 (11) |
| Impaired EQ-5D – usual activities∗ | 659 (30) | 465 (25) | 402 (27) |
| Impaired EQ-5D – pain/discomfort∗ | 908 (41) | 569 (31) | 479 (32) |
| Impaired EQ-5D – anxiety/depression∗ | 688 (31) | 435 (23) | 346 (23) |
| WHO subtypes | |||
| RA | 381 (17) | 204 (11) | 167 (11) |
| RARS | 345 (16) | 234 (13) | 209 (14) |
| RCMD | 854 (39) | 506 (27) | 398 (26) |
| RCMD-RS | 127 (6) | 60 (3) | 48 (3) |
| RAEB-1 | 264 (12) | 154 (8) | 114 (8) |
| RAEB-2 | 9 (0) | 36 (2) | 32 (2) |
| MDS-U | 94 (4.3) | 51 (3) | 36 (2) |
| Del 5q | 130 (6) | 88 (5) | 76 (5) |
| IPSS-R (continuous, score unit) | 2 (1.5-3.0) | 2 (1.0-3.0) | 2 (1.0-3.0) |
| IPSS-R, (categorical) | |||
| Very low | 559 (25) | 505 (27) | 430 (29) |
| Low | 900 (41) | 675 (36) | 546 (36) |
| Intermediate | 327 (15) | 235 (13) | 199 (13) |
| High | 80 (4) | 78 (4) | 55 (4) |
| Very high | 6 (0) | 17 (1) | 19 (1) |
| Hb, g/dL | 10.2 (9.0-11.4) | 10.6 (9.3-11.8) | 10.5 (9.2-11.9) |
| Hb, g/dL (categorical) | |||
| ≥10 | 1197 (54) | 1140 (61) | 890 (59) |
| 8-10 | 791 (36) | 520 (28) | 438 (29) |
| <8 | 202 (9) | 156 (8) | 136 (9) |
| Ferritin, μg/L (categorical) | |||
| ≥1000 | 164 (7) | 181 (10) | 167 (11) |
| 300-1000 | 624 (28) | 337 (18) | 247 (16) |
| ≤300 | 726 (33) | 409 (22) | 339 (23) |
| Erythropoietin, IU/L (categorical) | |||
| >500 | 75 (3) | 36 (2) | 21 (1) |
| 100-500 | 253 (12) | 112 (6) | 82 (5) |
| <100 | 680 (31) | 303 (16) | 221 (15) |
| Platelets, ×109/L (categorical) | |||
| ≥100 | 1634 (74) | 1293 (70) | 1074 (71) |
| 50-100 | 375 (17) | 321 (17) | 220 (15) |
| <50 | 171 (8) | 186 (10) | 162 (11) |
| Neutrophils, ×109/L | 2.4 (1.0-3.8) | 2.4 (1.3-3.8) | 2.3 (1.3-3.7) |
| Neutrophils, ≤1 × 109/L | 335 (15) | 298 (16) | 235 (16) |
| Therapies received | |||
| ESA | 331 (15) | 514 (28) | 424 (23) |
| ESA with Hb ≤ 10 g/dL | 258 (12) | 239 (13) | 184 (12) |
| Granulocyte colony-stimulating factor | 27 (1) | 54 (3) | 55 (4) |
| Iron chelation | 17 (1) | 59 (3) | 83 (6) |
| Immunosuppressive therapy | 7 (0) | 15 (1) | 17 (1) |
| Demethylating agent | 2 (0) | 28 (2) | 30 (2) |
| Lenalidomide | 7 (0) | 38 (2) | 45 (3) |
| Transfusion dependence† | 500 (23) | 455 (25) | 332 (22) |
| Transfusion density‡ | 0 (0.0-0.5) | 0 (0.0-0.5) | 0 (0.0-0.5) |
| Cumulative number of transfusions | 0 (0.0-1.0) | 0 (0.0-2.0) | 0 (0.0-2.0) |
| MDS progression§ | - | 200 (11) | 186 (12) |
| KPS (continuous) | 90 (80-100) | 90 (80-100) | 90 (80-100) |
| KPS (categorical) | |||
| Able to work (80-100) | 1428 (65) | 1110 (60) | 874 (58) |
| Unable to work (50-80) | 392 (18) | 329 (18) | 248 (17) |
| Unable to care for self (<50) | 28 (1) | 26 (1) | 26 (2) |
| MDS-CI (continuous) | 0 (0.0-2.0) | 0 (0.0-0.0) | 0 (0.0-1.0) |
| MDS-CI (categorical) | |||
| Low risk (0) | 1355 (62) | 1399 (75) | 1091 (72) |
| Intermediate risk (1-2) | 716 (33) | 398 (21) | 352 (23) |
| High risk (≥3) | 126 (6) | 46 (3) | 46 (3) |
| HCT-CI (continuous) | 1 (0.0-3.0) | 0 (0.0-2.0) | 0 (0.0-2.0) |
| HCT-CI (categorical) | |||
| Low risk (0) | 771 (35) | 1009 (54) | 759 (50) |
| Intermediate risk (1-2) | 799 (36) | 545 (29) | 461 (31) |
| High risk (≥3) | 635 (29) | 307 (17) | 286 (19) |
| BMI, kg/m2 | 26 (23.8-29.0) | 26 (23.8-29.1) | 26 (23.5-29.1) |
| BMI, kg/m2 (categorical) | |||
| Underweight <23 | 311 (14) | 268 (14) | 242 (16) |
| Weight range (23-29.9) | 1084 (50) | 911 (49) | 733 (49) |
| Overweight ≥30 | 320 (15) | 282 (15) | 231 (15) |
The numbers represent the observed, nonmissing values for the patient characteristics at each time point. Reported percentages relate to all patients (including those with missing values). The complementary missing values are represented detail in supplemental Table 1.
Del 5q, MDS associated with isolated del(5q); EQ-5D, EQ-5D 3-Levels questionnaire; HCT-CI, hematopoietic cell transplantation-specific comorbidity index; IQR, interquartile range; MDS-U, MDS-unclassified; RA, refractory anemia; RAEB, refractory anemia with excess blasts; RARS, refractory anemia with ringed sideroblasts; RCMD, refractory cytopenia with multilineage dysplasia; RCMD-RS, refractory cytopenia with multilineage dysplasia and ringed sideroblasts.
Any problems (answer category 1 and 2) versus no problems (answer category 3).
At least 1 unit RBC transfusion for a surveillance time of 8 weeks before the HRQoL assessment.
Average number of RBC transfusions per month since the last visit or up to 8 weeks before MDS diagnose.
Progression to higher IPSS-R risk group.