Clinical and laboratory features and subsequent events in 200 patients with myelodysplastic syndromes and ≥ 1% ringed sideroblasts without excess blasts, stratified by the ring sideroblast percentage
| Variable . | Myelodysplastic syndromes without excess blasts . | P . | |||||
|---|---|---|---|---|---|---|---|
| RS > 1% (n = 200) . | RS < 5% (n = 56) . | RS 5%-14% (n = 32) . | RS 15%-50% (n = 79) . | RS > 50% (n = 33) . | RS%, categories compared . | RS%, continuous variable . | |
| Median age, y (range) | 71 (17-90) | 70 (17-87) | 66 (25-88) | 71 (44-90) | 74 (49-89) | .05 | .02 |
| Males, n (%) | 140 (70) | 41 (73) | 23 (72) | 51 (65) | 25 (76) | .58 | .76 |
| Median hemoglobin, g/dL (range) | 10 (6-16) | 10 (7-14) | 10.5 (7-15) | 10 (6-16) | 9 (7-12) | .02 | .006 |
| Median MCV, femtoliter (range) | 97 (72-120) | 95 (72-116) | 96 (82-118) | 98 (77-120) | 98 (78-118) | .7 | .07 |
| Median WBC, × 109 cells/L (range) | 4 (1-37) | 4 (1-37) | 4 (1-12) | 4 (1-13) | 6 (1-18) | .20 | .43 |
| Median ANC, × 109 cells/L (range) | 2.1 (.2-33) | 3 (1-33) | 2.8 (0.2-8) | 2.8 (1-9) | 3.4 (2.2-13) | .76 | .45 |
| Median platelets, × 109 cells/L (range) | 133 (6-819) | 86 (18-505) | 84 (15-416) | 183 (6-607) | 237 (7-819) | < .0001 | < .0001 |
| Median BM cellularity, % (range) | 70 (10-100) | 60 (15-95) | 62 (30-100) | 70 (10-100) | 80 (40-100) | .0006 | .0025 |
| Median BM ring sideroblast, % (range) | 19 (1-78) | 1 (1-4) | 8 (5-14) | 30 (15-50) | 60 (52-78) | < .0001 | NA |
| WHO histologic category, n (%) | |||||||
| RARS | 56 (20) | 0 | 0 | 43 (54) | 13 (39) | < .0001 | < .0001 |
| RCMD | 130 (47) | 50 (89) | 29 (91) | 34 (43) | 17 (51) | ||
| MDS-U | 14 (7) | 6 (11) | 3 (9) | 2 (3) | 3 (9) | ||
| Cytogenetics | |||||||
| Normal/Diploid (%) | 109 (55) | 22 (39) | 15 (47) | 51 (65) | 21 (64) | .09 | .12 |
| Abnormal (%) | 91 (45) | 34 (61) | 17 (53) | 28 (35) | 12 (36) | ||
| Cytogenetic risk category per IPSS-R, n (%) | |||||||
| Very good | 11 (6) | 5 (10) | 3 (9) | 0 (0) | 3 (9) | .01 | .008 |
| Good | 128 (64) | 26 (46) | 17 (53) | 62 (79) | 23 (70) | ||
| Intermediate | 20 (10) | 7 (13) | 5 (16) | 7 (9) | 1 (3) | ||
| Poor | 20 (10) | 10 (18) | 4 (13) | 5 (6) | 1 (3) | ||
| Very poor | 21 (11) | 8 (14) | 3 (9) | 5 (6) | 5 (15) | ||
| Molecular profile, n (%) | |||||||
| IDH1 | 3 (2) | 0 (0) | 2 (6) | 1 (1) | 0 (0) | .84 | .9 |
| IDH2 | 17 (9) | 6 (11) | 3 (9) | 7 (9) | 1 (3) | .69 | .25 |
| JAK2 | 5 (3) | 0 (0) | 2 (6) | 1 (1) | 2 (6) | ||
| MPL | 1 (1) | 1 (2) | 0 (0) | 0 (0) | 0 (0) | ||
| SF3B1/n* | 64/110 (58) | 0/10 (0) | 5/17 (29) | 33/50 (66) | 26/33 (79) | .04 | .04 |
| IPSS risk categories, n (%) | |||||||
| Low | 15 (8) | 3 (4) | 2 (6) | 7 (9) | 3 (9) | .67 | .19 |
| Intermediate-1 | 154 (77) | 40 (71) | 24 (75) | 63 (80) | 27 (81) | ||
| Intermediate-2 | 29 (15) | 12 (21) | 6 (19) | 8 (10) | 3 (9) | ||
| High | 2 (1) | 1 (2) | 0 | 1 (1) | 0 (0) | ||
| IPSS-R risk categories, n (%) | |||||||
| Very good | 39 (20) | 4 (7) | 7 (22) | 22 (28) | 6 (18) | .04 | .13 |
| Good | 113 (57) | 33 (60) | 18 (56) | 42 (53) | 20 (60) | ||
| Intermediate | 21 (11) | 6 (11) | 5 (16) | 9 (11) | 1 (3) | ||
| Poor | 9 (5) | 5 (10) | 2 (6) | 0 (0) | 2 (6) | ||
| Very poor | 18 (9) | 8 (14) | 0 (0) | 6 (8) | 4 (8) | ||
| Transfusion dependent, n (%) | 34 (17) | 5 (9) | 4 (13) | 16 (21) | 9 (27) | .12 | .01 |
| Leukemic transformation, n (%) | 24 (12) | 7 (12) | 7 (22) | 7 (9) | 3 (9) | .26 | .48 |
| Deaths, n (%) | 156 (73) | 49 (82) | 22 (85) | 60 (38) | 25 (76) | .23 | .19 |
| Median F/U, mo (range) | 33.7 (0-185) | 14 (0-121) | 23 (1-72) | 59 (1-75) | 43 (0-185) | .20 | .10 |
| Variable . | Myelodysplastic syndromes without excess blasts . | P . | |||||
|---|---|---|---|---|---|---|---|
| RS > 1% (n = 200) . | RS < 5% (n = 56) . | RS 5%-14% (n = 32) . | RS 15%-50% (n = 79) . | RS > 50% (n = 33) . | RS%, categories compared . | RS%, continuous variable . | |
| Median age, y (range) | 71 (17-90) | 70 (17-87) | 66 (25-88) | 71 (44-90) | 74 (49-89) | .05 | .02 |
| Males, n (%) | 140 (70) | 41 (73) | 23 (72) | 51 (65) | 25 (76) | .58 | .76 |
| Median hemoglobin, g/dL (range) | 10 (6-16) | 10 (7-14) | 10.5 (7-15) | 10 (6-16) | 9 (7-12) | .02 | .006 |
| Median MCV, femtoliter (range) | 97 (72-120) | 95 (72-116) | 96 (82-118) | 98 (77-120) | 98 (78-118) | .7 | .07 |
| Median WBC, × 109 cells/L (range) | 4 (1-37) | 4 (1-37) | 4 (1-12) | 4 (1-13) | 6 (1-18) | .20 | .43 |
| Median ANC, × 109 cells/L (range) | 2.1 (.2-33) | 3 (1-33) | 2.8 (0.2-8) | 2.8 (1-9) | 3.4 (2.2-13) | .76 | .45 |
| Median platelets, × 109 cells/L (range) | 133 (6-819) | 86 (18-505) | 84 (15-416) | 183 (6-607) | 237 (7-819) | < .0001 | < .0001 |
| Median BM cellularity, % (range) | 70 (10-100) | 60 (15-95) | 62 (30-100) | 70 (10-100) | 80 (40-100) | .0006 | .0025 |
| Median BM ring sideroblast, % (range) | 19 (1-78) | 1 (1-4) | 8 (5-14) | 30 (15-50) | 60 (52-78) | < .0001 | NA |
| WHO histologic category, n (%) | |||||||
| RARS | 56 (20) | 0 | 0 | 43 (54) | 13 (39) | < .0001 | < .0001 |
| RCMD | 130 (47) | 50 (89) | 29 (91) | 34 (43) | 17 (51) | ||
| MDS-U | 14 (7) | 6 (11) | 3 (9) | 2 (3) | 3 (9) | ||
| Cytogenetics | |||||||
| Normal/Diploid (%) | 109 (55) | 22 (39) | 15 (47) | 51 (65) | 21 (64) | .09 | .12 |
| Abnormal (%) | 91 (45) | 34 (61) | 17 (53) | 28 (35) | 12 (36) | ||
| Cytogenetic risk category per IPSS-R, n (%) | |||||||
| Very good | 11 (6) | 5 (10) | 3 (9) | 0 (0) | 3 (9) | .01 | .008 |
| Good | 128 (64) | 26 (46) | 17 (53) | 62 (79) | 23 (70) | ||
| Intermediate | 20 (10) | 7 (13) | 5 (16) | 7 (9) | 1 (3) | ||
| Poor | 20 (10) | 10 (18) | 4 (13) | 5 (6) | 1 (3) | ||
| Very poor | 21 (11) | 8 (14) | 3 (9) | 5 (6) | 5 (15) | ||
| Molecular profile, n (%) | |||||||
| IDH1 | 3 (2) | 0 (0) | 2 (6) | 1 (1) | 0 (0) | .84 | .9 |
| IDH2 | 17 (9) | 6 (11) | 3 (9) | 7 (9) | 1 (3) | .69 | .25 |
| JAK2 | 5 (3) | 0 (0) | 2 (6) | 1 (1) | 2 (6) | ||
| MPL | 1 (1) | 1 (2) | 0 (0) | 0 (0) | 0 (0) | ||
| SF3B1/n* | 64/110 (58) | 0/10 (0) | 5/17 (29) | 33/50 (66) | 26/33 (79) | .04 | .04 |
| IPSS risk categories, n (%) | |||||||
| Low | 15 (8) | 3 (4) | 2 (6) | 7 (9) | 3 (9) | .67 | .19 |
| Intermediate-1 | 154 (77) | 40 (71) | 24 (75) | 63 (80) | 27 (81) | ||
| Intermediate-2 | 29 (15) | 12 (21) | 6 (19) | 8 (10) | 3 (9) | ||
| High | 2 (1) | 1 (2) | 0 | 1 (1) | 0 (0) | ||
| IPSS-R risk categories, n (%) | |||||||
| Very good | 39 (20) | 4 (7) | 7 (22) | 22 (28) | 6 (18) | .04 | .13 |
| Good | 113 (57) | 33 (60) | 18 (56) | 42 (53) | 20 (60) | ||
| Intermediate | 21 (11) | 6 (11) | 5 (16) | 9 (11) | 1 (3) | ||
| Poor | 9 (5) | 5 (10) | 2 (6) | 0 (0) | 2 (6) | ||
| Very poor | 18 (9) | 8 (14) | 0 (0) | 6 (8) | 4 (8) | ||
| Transfusion dependent, n (%) | 34 (17) | 5 (9) | 4 (13) | 16 (21) | 9 (27) | .12 | .01 |
| Leukemic transformation, n (%) | 24 (12) | 7 (12) | 7 (22) | 7 (9) | 3 (9) | .26 | .48 |
| Deaths, n (%) | 156 (73) | 49 (82) | 22 (85) | 60 (38) | 25 (76) | .23 | .19 |
| Median F/U, mo (range) | 33.7 (0-185) | 14 (0-121) | 23 (1-72) | 59 (1-75) | 43 (0-185) | .20 | .10 |
MDS indicates myelodysplastic syndromes; RS, ring sideroblasts; RARS, refractory anemia with ring sideroblasts; RCMD, refractory cytopenias with multilineage dysplasia; MDS-U, myelodysplastic syndrome unclassifiable; IPSS, International Prognostic Scoring System; IPSS-R, revised IPSS; and WHO, World Health Organization.
Please note that SF3B1 mutational analysis was only performed in 110 of the 200 patients due to limited availability of DNA.