Contributions
Abstract: PB1932
Type: Publication Only
Background
Although several recent guidelines recommend iron chelation therapy (ICT) for iron overload in transfusion-dependent patients (pts) with lower-risk myelodysplastic syndromes (MDS), several barriers may limit the initiation or the continuance of ICT: older age, comorbidities, poor tolerance and compliance.
Aims
Therefore, with the aim of assessing the safety and efficacy of ICT in the daily clinical practice, we retrospectively analyzed our single-center experience on ICT in MDS and other chronic anemias.
Methods
From October 1997, in our Institution, 69 pts (48 males), median age: 74 (23-96) yrs, with transfusion-dependent anemia, received ICT, because of a diagnosis of iron overload, i.e. both a transfusion history of at least 20 units of RBC and a serum ferritin (SF) higher than 1000 ng/ml.
Results
40 pts (58%) were affected by lower-risk MDS (IPSS risk: low or intermediate-1), while 13 pts (18.8%) showed a higher-risk MDS (IPSS risk: high or intermediate-2) but were considered for ICT because of responsiveness to hypomethylating therapy and/or elegibility for allogeneic SCT. 16 pts (23.2%) were affected by other diseases (chronic myelomonocytic leukemia: 2 pts; idiopathic myelofibrosis: 3 pts; aplastic anemia: 9 pts; pure red cell aplasia (PRCA): 2 pts). 45 pts (65.2%) received deferasirox (DFX) as first-line treatment, 12 pts (17.4%) received DFX after a previous treatment with deferoxamine (DFO), while 9 pts (13%) received DFO and 3 pts (4.3%) received DFO after DFX because of contraindications to DFX or toxicity. Median time from diagnosis to the start of ICT: 18 months. Median number of RBC transfusions before the start of ICT: 37.5. Median SF level pre-ICT: 1964 ng/ml; median SF after ICT (last value): 1858 ng/ml; median duration of ICT: 12 (range 1-230) months. 36 pts (52.2%) continued ICT for a period ≥ 12 months, and 25 pts (36.2%) for a period ≥ 24 months. 27 pts (39.1%) showed a drop of SF ≥ 500 ng/ml, 11 pts (15.9%) showed a drop of SF < 500, 13 pts (18.8%) showed an increase of SF < 500, in spite of ICT, and 18 pts (26.1%) showed an increase of SF ≥ 500. 12 pts (17.4%) achieved a SF value < 1.000, and 48 pts (69.6%) a SF value < 2.500. Adverse events possibly related to DFX were observed in 30 pts (43.5%): renal (increase of serum creatinine): 14 pts (20.3%) (grade > 2: 1 pt: 1.4%); gastrointestinal : 14 pts (20.3%) (grade > 2: 1 pt: 1.4%); cutaneous: 2 pts (2.9%) (grade > 2: no pts). Permanent discontinuation of ICT: 40 pts (58%), because of toxicity (16 pts: 23.2%), worsening of clinical condition (6 pts: 8.7%), discontinuation of transfusions (9 pts: 13%), allogeneic transplantation (9 pts: 13%). 5 pts (7.2%) (4 MDS and 1 PRCA) (with DFX: 4 pts; with DFO: 1 pt) showed an erythroid response following ICT, after 2, 4, 7, 32 and 112 months, respectively, and one of them (with PRCA) achieved complete remission. 35 pts (50.7%) died, because of infection (9 pts), AML (4 pts), cachexia (4 pts), other neoplastic diseases (3 pts), hemorrhage (2 pts), heart failure (2 pts), stroke (2 pts) and other causes (9 pts). 10 pts (14.5%) are still receiving ICT. With a median follow-up of 34 (2-230) months, median overall survival (OS) was 64 months for all pts, 51 months for MDS pts, 87 months for lower-risk MDS (IPSS risk: low and intermediate-1) and 24 months for higher-risk MDS (IPSS risk: intermediate-2 and high).
Conclusion
In conclusion, in our experience ICT appears feasible and effective, in terms of reduction of SF and OS, even in a population of elderly pts, if carefully selected.
Session topic: 10. Myelodysplastic syndromes - Clinical
Keyword(s): Myelodysplasia, iron overload, iron chelation, Deferasirox
Abstract: PB1932
Type: Publication Only
Background
Although several recent guidelines recommend iron chelation therapy (ICT) for iron overload in transfusion-dependent patients (pts) with lower-risk myelodysplastic syndromes (MDS), several barriers may limit the initiation or the continuance of ICT: older age, comorbidities, poor tolerance and compliance.
Aims
Therefore, with the aim of assessing the safety and efficacy of ICT in the daily clinical practice, we retrospectively analyzed our single-center experience on ICT in MDS and other chronic anemias.
Methods
From October 1997, in our Institution, 69 pts (48 males), median age: 74 (23-96) yrs, with transfusion-dependent anemia, received ICT, because of a diagnosis of iron overload, i.e. both a transfusion history of at least 20 units of RBC and a serum ferritin (SF) higher than 1000 ng/ml.
Results
40 pts (58%) were affected by lower-risk MDS (IPSS risk: low or intermediate-1), while 13 pts (18.8%) showed a higher-risk MDS (IPSS risk: high or intermediate-2) but were considered for ICT because of responsiveness to hypomethylating therapy and/or elegibility for allogeneic SCT. 16 pts (23.2%) were affected by other diseases (chronic myelomonocytic leukemia: 2 pts; idiopathic myelofibrosis: 3 pts; aplastic anemia: 9 pts; pure red cell aplasia (PRCA): 2 pts). 45 pts (65.2%) received deferasirox (DFX) as first-line treatment, 12 pts (17.4%) received DFX after a previous treatment with deferoxamine (DFO), while 9 pts (13%) received DFO and 3 pts (4.3%) received DFO after DFX because of contraindications to DFX or toxicity. Median time from diagnosis to the start of ICT: 18 months. Median number of RBC transfusions before the start of ICT: 37.5. Median SF level pre-ICT: 1964 ng/ml; median SF after ICT (last value): 1858 ng/ml; median duration of ICT: 12 (range 1-230) months. 36 pts (52.2%) continued ICT for a period ≥ 12 months, and 25 pts (36.2%) for a period ≥ 24 months. 27 pts (39.1%) showed a drop of SF ≥ 500 ng/ml, 11 pts (15.9%) showed a drop of SF < 500, 13 pts (18.8%) showed an increase of SF < 500, in spite of ICT, and 18 pts (26.1%) showed an increase of SF ≥ 500. 12 pts (17.4%) achieved a SF value < 1.000, and 48 pts (69.6%) a SF value < 2.500. Adverse events possibly related to DFX were observed in 30 pts (43.5%): renal (increase of serum creatinine): 14 pts (20.3%) (grade > 2: 1 pt: 1.4%); gastrointestinal : 14 pts (20.3%) (grade > 2: 1 pt: 1.4%); cutaneous: 2 pts (2.9%) (grade > 2: no pts). Permanent discontinuation of ICT: 40 pts (58%), because of toxicity (16 pts: 23.2%), worsening of clinical condition (6 pts: 8.7%), discontinuation of transfusions (9 pts: 13%), allogeneic transplantation (9 pts: 13%). 5 pts (7.2%) (4 MDS and 1 PRCA) (with DFX: 4 pts; with DFO: 1 pt) showed an erythroid response following ICT, after 2, 4, 7, 32 and 112 months, respectively, and one of them (with PRCA) achieved complete remission. 35 pts (50.7%) died, because of infection (9 pts), AML (4 pts), cachexia (4 pts), other neoplastic diseases (3 pts), hemorrhage (2 pts), heart failure (2 pts), stroke (2 pts) and other causes (9 pts). 10 pts (14.5%) are still receiving ICT. With a median follow-up of 34 (2-230) months, median overall survival (OS) was 64 months for all pts, 51 months for MDS pts, 87 months for lower-risk MDS (IPSS risk: low and intermediate-1) and 24 months for higher-risk MDS (IPSS risk: intermediate-2 and high).
Conclusion
In conclusion, in our experience ICT appears feasible and effective, in terms of reduction of SF and OS, even in a population of elderly pts, if carefully selected.
Session topic: 10. Myelodysplastic syndromes - Clinical
Keyword(s): Myelodysplasia, iron overload, iron chelation, Deferasirox