Abstract 1723

Introduction and Objective:

A large number of patients with low-risk myelodysplastic syndrome (MDS) need blood transfusions, being iron overload an inevitable consequence that may cause hepatic fibrosis and cirrhosis, diabetes or myocardiopathy unless it is not treated. The main objective of this study is to describe the evolution of iron overload in transfusion-dependent patients with low-risk MDS. Secondary objectives include the evaluation of the impact of iron chelation therapy on Event Free Survival (EFS) (including infections, arthropathy, diabetes mellitus, hepatic and cardiac complications), Overall Survival (OS) and Leukemia Free Survival (LFS).

Methods and Patients:

Retrospective observational study carried out in Haematology Departments of 47 Spanish hospitals between March 2010 and March 2011. Transfusion-dependent patients with low/intermediate-1 IPSS risk who had received ≥10 red blood cell (RBC) transfusion during at least 12 months previous to study entry were eligible.

Results:

A total of 263 patients were evaluated [age at diagnosis, 74 ± 10.5 years]. IPSS classification was available in 82.9% included patients (lack of cytogenetics in 37 patients), classified as low risk (86%) and int-1 risk (14%). At diagnosis, 25.5% of patients had serum ferritin (SF) levels >500ng/ml, 8.7% SF>1,000ng/ml, and 22% TSI>50%. The median number of RBC transfusions per month was 2.35 RBC/month; during the course of the disease, 82.4% of patients reached SF levels >1,000ng/ml. Cardiac complications worsened/appeared in 24.4% of patients, having received a median RBC transfusions of 22 (7–92), and showing a median SF levels of 1,365 (735–3,025) ng/ml. One-hundred forty-seven (55.9%) patients started iron chelation therapy (85.5% with deferasirox) with a median number of RBC transfused of 23 (14–38) and a median SF levels of 1,570 (1,231.5–2,195) ng/ml. 71% of patients with SF>1,000ng/ml were on iron chelation therapy. Table 1 shows the results of univariate analysis of EFS, OS and LFS. As part of this study, we are currently performing a multivariate analysis whose results will be presented in forthcoming congress.

Table 1.

Univariate analyses of EFS, OS and LFS

Non chelatedChelated*p-value
Median EFS arthropathy complications (months), n=236 Not reached Not reached 0.794 
Median EFS diabetes complications (months), n=216 Not reached Not reached 0.559 
Median EFS hepatic complications (months), n=236 208 Not reached 0.303 
Median EFS cardiac complications (months), n=196 90 137 0.004 
Median OS (months), n=228 105 133 0.009 
Median LFS (months), n=228 Not reached Not reached 0.043 
Non chelatedChelated*p-value
Median EFS arthropathy complications (months), n=236 Not reached Not reached 0.794 
Median EFS diabetes complications (months), n=216 Not reached Not reached 0.559 
Median EFS hepatic complications (months), n=236 208 Not reached 0.303 
Median EFS cardiac complications (months), n=196 90 137 0.004 
Median OS (months), n=228 105 133 0.009 
Median LFS (months), n=228 Not reached Not reached 0.043 
*

Analysis including patients with ≥3 months chelation (n=109) (of whom 73.4% received deferasirox as first option therapy with a mean dose of 18 mg/kg/day).

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Conclusions:

The results of this study show that a high percentage of transfusion-dependent patients with low-risk MDS (82.4%) reach SF levels >1,000ng/ml during the course of their disease. Patients on iron chelation therapy show a higher cardiac EFS, OS and LFS compared with those not treated, being deferasirox the most frequently used chelating agent in clinical practice.

Disclosures:

Sanz:Novartis Farmaceutica S.A.: Employment.

Topics:
iron overload, myelodysplastic syndrome, observational studies, iron chelation therapy, transfusion, cardiac complications, deferasirox, diabetes mellitus, erythrocyte transfusion, joint disorders

Author notes

*

Asterisk with author names denotes non-ASH members.

© 2012 by The American Society of Hematology
2012
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