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MANAGEMENT OF MYELODYSPLASTIC SYNDROMES WITH ERYTHROPOIESIS STIMULATING AGENTS (ESAS) : A REAL-LIFE EXPERIENCE
Author(s): ,
Claudio Cerchione
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
,
Orsola Vitagliano
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
,
Anna Emanuele Pareto
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
,
Maria Di Perna
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
,
Giuseppe Cerciello
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
,
Ilaria Soriente
Affiliations:
Hematology,Ospedale Umberto I,Nocera Inferiore (SA),Italy
,
Fiorella Alfinito
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
Fabrizio Pane
Affiliations:
Hematology,Ematologia e trapianto/au federico ii,Napoli,Italy
(Abstract release date: 05/19/16) EHA Library. Cerchione C. 06/09/16; 135014; PB2114
Dr. Claudio Cerchione
Dr. Claudio Cerchione
Contributions
Abstract
Abstract: PB2114

Type: Publication Only

Background
ESAs are the frontline treatment in low-risk anemic MDS patients and an employment of this therapy in the earlier stage of the disease can delay the need for RBC transfusion, hypothetically by slowing the disease course. It’s matter of debate whether the clinical response is a result of proliferation and maturation of the dysplastic clone or stimulation of residual normal erythropoiesis by ESAs. 

Aims
Macrocytosis is one of the cytological hallmarks of dyserithropoiesis in MDS : an analysis of the erythropoietic response to ESAs therapy in a cohort of anemic non trasfusion-dependent MDS patients, enrolled in a retrospective register, RECAMDS, subgroup of Italian register, was performed.

Methods
159 patients, treated with standard-dose ESAs, have been retrospectively analyzed (Table 1). Data analysis was performed, according to IWG 2006 criteria, at the baseline, after 3 and 6 months of continuous treatment, with a subanalysis of the patients according to WHO and R-IPSS risk stratification. ESAs were started at mean Hb concentration of 9.43 g/dl, mean serum EPO concentration : 42 mU/L, after a mean time from diagnosis of 6 months (r.1-118). 

Results
Overall response rate (ORR) was 83.6% (133/159), no difference among WHO and IPSS subgroups was found :75.6% achieved response after 3 months of treatment, while other 8% after 6 months. 2 patients with SD (non responders IWG), in which treatment was continued, achieved response after 9 months. In the macrocytic-responders group 85.5% exhibits again macrocytosis after 3 months, while 13% become normocytic. In the normocytic-responders group 91.7% exhibits again normocytosis, while 4/52 (8%) become macrocytic : in these 4 patients after 3 months there was a contemporary worsening in neutropenia and thrombocytopenia, with transfusion-dependence, regarded as first signs of progression of disease. Non responders were 27/159 (16.9%) : in the macrocytic-non responders group 89% exhibit again macrocytosis after 3 months, while 11% become normocytic; in the normocytic group 80% exhibits again macrocytosis, while 20% become normocytic (r.1-23). 

Conclusion
These preliminary data can suggest that, in the majority of MDS patients responsive to ESAs, the increase of Hb concentration occurs mainly stimulating erythroid production in MDS clones; in the minority of patients probably it happens recruiting residual polyclonal erythropoiesis. It is interesting to note that stimulating effects of ESAs last even when the expression of dysplasia progresses.

Session topic: E-poster

Keyword(s): Epoetin, Hemoglobin, Myelodysplasia
Abstract: PB2114

Type: Publication Only

Background
ESAs are the frontline treatment in low-risk anemic MDS patients and an employment of this therapy in the earlier stage of the disease can delay the need for RBC transfusion, hypothetically by slowing the disease course. It’s matter of debate whether the clinical response is a result of proliferation and maturation of the dysplastic clone or stimulation of residual normal erythropoiesis by ESAs. 

Aims
Macrocytosis is one of the cytological hallmarks of dyserithropoiesis in MDS : an analysis of the erythropoietic response to ESAs therapy in a cohort of anemic non trasfusion-dependent MDS patients, enrolled in a retrospective register, RECAMDS, subgroup of Italian register, was performed.

Methods
159 patients, treated with standard-dose ESAs, have been retrospectively analyzed (Table 1). Data analysis was performed, according to IWG 2006 criteria, at the baseline, after 3 and 6 months of continuous treatment, with a subanalysis of the patients according to WHO and R-IPSS risk stratification. ESAs were started at mean Hb concentration of 9.43 g/dl, mean serum EPO concentration : 42 mU/L, after a mean time from diagnosis of 6 months (r.1-118). 

Results
Overall response rate (ORR) was 83.6% (133/159), no difference among WHO and IPSS subgroups was found :75.6% achieved response after 3 months of treatment, while other 8% after 6 months. 2 patients with SD (non responders IWG), in which treatment was continued, achieved response after 9 months. In the macrocytic-responders group 85.5% exhibits again macrocytosis after 3 months, while 13% become normocytic. In the normocytic-responders group 91.7% exhibits again normocytosis, while 4/52 (8%) become macrocytic : in these 4 patients after 3 months there was a contemporary worsening in neutropenia and thrombocytopenia, with transfusion-dependence, regarded as first signs of progression of disease. Non responders were 27/159 (16.9%) : in the macrocytic-non responders group 89% exhibit again macrocytosis after 3 months, while 11% become normocytic; in the normocytic group 80% exhibits again macrocytosis, while 20% become normocytic (r.1-23). 

Conclusion
These preliminary data can suggest that, in the majority of MDS patients responsive to ESAs, the increase of Hb concentration occurs mainly stimulating erythroid production in MDS clones; in the minority of patients probably it happens recruiting residual polyclonal erythropoiesis. It is interesting to note that stimulating effects of ESAs last even when the expression of dysplasia progresses.

Session topic: E-poster

Keyword(s): Epoetin, Hemoglobin, Myelodysplasia

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