Hematologic characterization of 8 patients with transfusion dependent β-thalassemia during 3 months of pretreatment and 8 months (nonresponders) to 13 months (responders) of treatment with oral IBT
| Component . | Patient type . | Values before IBT treatment* . | Values during IBT treatment† . | Pvalue . |
|---|---|---|---|---|
| Median (Interquartile range) . | Median (Interquartile range) . | |||
| HbF‡(%) | Responders | 5.2 (4.8-6.0) | 9.3 (7.1-16.4) | P = .0016 |
| Nonresponders | 2.4 (1.8-4.3) | 4.0 (2.9-6.6) | P = .0089 | |
| P = .0007 | P < .0001 | |||
| Absolute HbF‡(g/L) | Responders | 4.5 (4.0-5.1) | 7.1 (6.1-13.6) | P = .0018 |
| Nonresponders | 1.9 (1.5-2.4) | 3.4 (2.5-5.4) | P = .0071 | |
| P = .0011 | P < .0001 | |||
| MCV‡(fL) | Responders | 79.5 (76.5-81.3) | 83.8 (82.2-87.8) | P = .0029 |
| Nonresponders | 79.0 (77.0-81.0) | 86.1 (83.0-88.0) | P < .0001 | |
| P = .92 | P = .29 | |||
| MCH‡(pg) | Responders | 27.5 (26.7-28.1) | 28.8 (28.2-29.0) | P = .0056 |
| Nonresponders | 27.6 (27.2-27.9) | 29.2 (28.3-29.9) | P < .0001 | |
| P = .76 | P = .073 | |||
| Free Hb‡(g/L) | Responders | 0.51 (0.43-0.75) | 0.21 (0.17-0.27) | P = .0003 |
| Nonresponders | 0.45 (0.38-0.87) | 0.18 (0.15-0.22) | P < .0001 | |
| P = .59 | P = .20 | |||
| Erythropoietin‡(IU/L) | Responders | ND | 340 (180-480) | — |
| Nonresponders | ND | 66 (30-103) | — | |
| P < .0001 | ||||
| Iron load2-153 (μg/kg per day) | Responders | 539 (454-685) | 360 (214-564) | P = .0322 |
| Nonresponders | 444 (392-658) | 458 (385-647) | P = .96 | |
| P = .19 | P = .013 | |||
| Hemoglobin‡(g/L) | Responders | 84 (82-87) | 85 (82-86) | P > .1 |
| Nonresponders | 85 (81-87) | 85 (81-87) | P > .1 | |
| P > .1 | P > .1 | |||
| nRBCs/nL‡ | Responders | 0.67 (0.0-1.43) | 0.28 (0.0-0.74) | P > .1 |
| Nonresponders | 0.285 (0.075-3.77) | 0.18 (0.0-0.55) | P > .1 | |
| P > .1 | P > .1 |
| Component . | Patient type . | Values before IBT treatment* . | Values during IBT treatment† . | Pvalue . |
|---|---|---|---|---|
| Median (Interquartile range) . | Median (Interquartile range) . | |||
| HbF‡(%) | Responders | 5.2 (4.8-6.0) | 9.3 (7.1-16.4) | P = .0016 |
| Nonresponders | 2.4 (1.8-4.3) | 4.0 (2.9-6.6) | P = .0089 | |
| P = .0007 | P < .0001 | |||
| Absolute HbF‡(g/L) | Responders | 4.5 (4.0-5.1) | 7.1 (6.1-13.6) | P = .0018 |
| Nonresponders | 1.9 (1.5-2.4) | 3.4 (2.5-5.4) | P = .0071 | |
| P = .0011 | P < .0001 | |||
| MCV‡(fL) | Responders | 79.5 (76.5-81.3) | 83.8 (82.2-87.8) | P = .0029 |
| Nonresponders | 79.0 (77.0-81.0) | 86.1 (83.0-88.0) | P < .0001 | |
| P = .92 | P = .29 | |||
| MCH‡(pg) | Responders | 27.5 (26.7-28.1) | 28.8 (28.2-29.0) | P = .0056 |
| Nonresponders | 27.6 (27.2-27.9) | 29.2 (28.3-29.9) | P < .0001 | |
| P = .76 | P = .073 | |||
| Free Hb‡(g/L) | Responders | 0.51 (0.43-0.75) | 0.21 (0.17-0.27) | P = .0003 |
| Nonresponders | 0.45 (0.38-0.87) | 0.18 (0.15-0.22) | P < .0001 | |
| P = .59 | P = .20 | |||
| Erythropoietin‡(IU/L) | Responders | ND | 340 (180-480) | — |
| Nonresponders | ND | 66 (30-103) | — | |
| P < .0001 | ||||
| Iron load2-153 (μg/kg per day) | Responders | 539 (454-685) | 360 (214-564) | P = .0322 |
| Nonresponders | 444 (392-658) | 458 (385-647) | P = .96 | |
| P = .19 | P = .013 | |||
| Hemoglobin‡(g/L) | Responders | 84 (82-87) | 85 (82-86) | P > .1 |
| Nonresponders | 85 (81-87) | 85 (81-87) | P > .1 | |
| P > .1 | P > .1 | |||
| nRBCs/nL‡ | Responders | 0.67 (0.0-1.43) | 0.28 (0.0-0.74) | P > .1 |
| Nonresponders | 0.285 (0.075-3.77) | 0.18 (0.0-0.55) | P > .1 | |
| P > .1 | P > .1 |
ND indicates not done.
Data obtained prestudy, spanning 3 transfusion intervals (3 months).
Data obtained during 8 months of IBT treatment in nonresponders and during 12 to 13 months in responders.
For calculation of the median, pretransfusion values were used.
Assuming that 1 mL of RBC concentrate contains 1 mg of iron, daily iron load was calculated from the volume of RBC concentrate necessary to maintain total Hb not below 85 g/L before and during IBT therapy.