Figure 1.
Robust and dose-dependent erythroid response of hyporegenerative anemia during DFX treatment. (A) BM morphology of the aspiration specimen obtained on initial consultation shows erythroid hyperplasia and impaired erythroid maturation without evidence of MDS or other malignancies (original magnification ×40). (B) Overview of the kinetics of Hb (green line) and ferritin (purple line) levels, transfusion requirements, and DFX dosing. Each red arrow denotes the transfusion of 1RBC unit. Within the first 8 months, a total of 29 RBC units were transfused. Periods of DFX treatment are indicated at the top. Changes in line thickness reflect changes in drug dose. The asterisk marks the start of the DFX rechallenge, a closer view of which is provided in panel C. Ferritin levels were already increased on initial presentation and continued to rise during transfusion therapy until DFX treatment was initiated. Subsequently, ferritin levels declined and stabilized at slightly elevated levels of >200 ng/mL. (C) Rapid increase in Hb levels within a few weeks after restart of DFX treatment. The corresponding time point in panel B is indicated by an asterisk. Of note, CRP was elevated to 57.9 mg/L at week 0 and normalized to <3 mg/L (without any antibiotics) at week +7. (D) Dose-dependent distribution of Hb levels during DFX treatment. Standard-dose (SD) DFX was administered between months 13 and 19, and low-dose (LD) DFX between months 25 and 49 after initial presentation. Hb levels were significantly higher during SD (17 mg/kg/d) than during LD (3 mg/kg/d or even every other day) DFX treatment (median Hb of 14.1 g/dL vs 12.6 g/dL; P = .02 by unpaired t test). Horizontal lines represent the median; asterisk indicates statistical significance. (E) (Left) Representative immunoblot of ABCB6 protein in the membrane of RBC lysate of the proband and a pool of 3 healthy controls. β-actin is the loading control. (Right) Quantification by densitometric analysis from 3 separate western blots with similar results. Data are means ± standard deviation (*P < .05). OD, optical density.

Robust and dose-dependent erythroid response of hyporegenerative anemia during DFX treatment. (A) BM morphology of the aspiration specimen obtained on initial consultation shows erythroid hyperplasia and impaired erythroid maturation without evidence of MDS or other malignancies (original magnification ×40). (B) Overview of the kinetics of Hb (green line) and ferritin (purple line) levels, transfusion requirements, and DFX dosing. Each red arrow denotes the transfusion of 1RBC unit. Within the first 8 months, a total of 29 RBC units were transfused. Periods of DFX treatment are indicated at the top. Changes in line thickness reflect changes in drug dose. The asterisk marks the start of the DFX rechallenge, a closer view of which is provided in panel C. Ferritin levels were already increased on initial presentation and continued to rise during transfusion therapy until DFX treatment was initiated. Subsequently, ferritin levels declined and stabilized at slightly elevated levels of >200 ng/mL. (C) Rapid increase in Hb levels within a few weeks after restart of DFX treatment. The corresponding time point in panel B is indicated by an asterisk. Of note, CRP was elevated to 57.9 mg/L at week 0 and normalized to <3 mg/L (without any antibiotics) at week +7. (D) Dose-dependent distribution of Hb levels during DFX treatment. Standard-dose (SD) DFX was administered between months 13 and 19, and low-dose (LD) DFX between months 25 and 49 after initial presentation. Hb levels were significantly higher during SD (17 mg/kg/d) than during LD (3 mg/kg/d or even every other day) DFX treatment (median Hb of 14.1 g/dL vs 12.6 g/dL; P = .02 by unpaired t test). Horizontal lines represent the median; asterisk indicates statistical significance. (E) (Left) Representative immunoblot of ABCB6 protein in the membrane of RBC lysate of the proband and a pool of 3 healthy controls. β-actin is the loading control. (Right) Quantification by densitometric analysis from 3 separate western blots with similar results. Data are means ± standard deviation (*P < .05). OD, optical density.

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