A 36-month analysis of treatment patterns and outcomes in patients with lower-risk myelodysplastic syndromes from a prospective observational study.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 7122-7122
Author(s):  
Roger M. Lyons ◽  
Billie J. Marek ◽  
Carole S. Paley ◽  
Jason Esposito ◽  
Lawrence E. Garbo ◽  
...  
Keyword(s):  
Blood Cell ◽  
Iron Overload ◽  
Red Blood Cell ◽  
Myelodysplastic Syndromes ◽  
Lower Risk ◽  
Cardiac Events ◽  
Risk Status ◽  
Red Blood Cell Transfusions ◽  
Lost To Follow Up ◽  
Ophthalmologic Disorders

7122 Background: Many patients (pts) with lower-risk myelodysplastic syndromes (MDS) require chronic red blood cell transfusions for symptomatic anemia, which can result in iron overload. We present a 36-month interim analysis of a 5-year US registry that prospectively collected data on clinical events and survival in chelated vs non-chelated, transfused, lower-risk MDS pts. Methods: This multicenter, non-interventional registry enrolled 600 pts ≥18 yr old with lower-risk MDS (WHO, FAB, and/or IPSS risk stratification criteria) and transfusional iron overload (serum ferritin ≥1,000 ng/mL and/or ≥20 packed red blood cell units and/or ≥6 units every 12 wks). The chelated group included pts who had received any chelation. Results: Median age was 76 yr (range, 21–99), 57.8% were male, and risk status was 38.6% IPSS low risk and 61.4% IPSS INT-1 risk. Baseline demographics and IPSS risk status were similar between groups, although transfusion burden trended higher in chelated pts. As of April 30, 2012, 169 pts continued on the registry, and 431 discontinued (345 died, 57.5%; 61 lost to follow-up, 10.2%; and 25 other, 4.2%). In all, 264 pts (44%) received chelation therapy; 200 had ≥6 mos chelation. Overall survival (OS) and time to acute myeloid leukemia (AML) transformation were significantly longer, and the percentage of deaths was significantly lower, in chelated ≥6 mos vs non-chelated pts (P<0.0001, P=0.011 [median not reached in either group], P=0.0002, respectively. AML transformations appeared to be lower in chelated ≥6 mos pts (not significant [NS]). At baseline in non-chelated vs chelated ≥6 mos pts, there was a higher prevalence of vascular, cardiac, endocrine, and ophthalmologic disorders; this trend continued at 36 mos. Most frequent causes of death were MDS/AML, cardiac events, and infection. Use of MDS therapy was lower among non-chelated pts (non-chelated, 88.4%; ≥6 mos chelation, 93.5%; NS). Conclusions: At 36 mos,chelated pts had significantly longer OS and time to AML, as well as significantly fewer deaths. Trends toward fewer AML transformations and fewer vascular, cardiac, endocrine, and ophthalmologic disorders were observed in chelated pts.

Relationship Between Chelation and Clinical Outcomes in 600 Lower-Risk MDS Patients: Registry Analysis At 36 Months

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3800-3800
Author(s):  
Roger M. Lyons ◽  
Billie J. Marek ◽  
Carole Paley ◽  
Jason Esposito ◽  
Lawrence Garbo ◽  
...  
Keyword(s):  
Blood Cell ◽  
Clinical Outcomes ◽  
Red Blood Cell ◽  
Lower Risk ◽  
Research Funding ◽  
Causes Of Death ◽  
Disease Status ◽  
Red Blood Cell Transfusion ◽  
Cardiac Events ◽  
Risk Status

Abstract Abstract 3800 Introduction: Treatment of anemia in pts with myelodysplastic syndromes (MDS) may require packed red blood cell transfusion. Transfusion dependence in MDS is associated with poorer clinical outcomes and reduced overall survival (OS). This US registry prospectively collected data on clinical outcomes in chelated and non-chelated, transfused, lower-risk MDS pts. OS, leukemic transformation, and clinical events are reported for non-chelated and chelated pts at 36 mos on study. Methods: This 5-year, non-interventional registry enrolled 600 pts from 107 US centers. Pts were ≥18 years old with lower-risk MDS (WHO, FAB, and/or IPSS) and transfusional iron overload (serum ferritin ≥1000 μg/L and/or ≥20 packed red blood cell units and/or ≥6 units every 12 weeks). The chelated group included all pts who had ever used iron chelation; sub-analysis was performed on pts with ≥6 mos chelation. Assessments were every 6 mos for 5 years or until death and included demographics, survival, disease status, comorbidities, causes of death, and MDS therapy. Results: Baseline demographics and IPSS risk status were similar between groups, although transfusion burden trended higher in chelated pts (Table 1). As of April 30, 2012, 169 pts continued on registry, and 431 discontinued (345 died, 57.5%; 61 lost to follow-up, 10.2%; and 25 other, 4.2%). In all, 264 (44%) pts received chelation therapy; 200 had ≥6 mos chelation. OS and time to acute myeloid leukemia (AML) transformation were significantly longer, and percentage of deaths was significantly lower in chelated ≥6 mos vs. non-chelated pts (P<0.0001, P=0.011 [median not reached in either group], P=0.0002, respectively; Table 2). AML transformations were also lower in chelated ≥6 mos pts (not significant [NS]). Cardiac (non-chelated, 51.5%; ≥6 mos chelation, 30.5%) and vascular disorders (non-chelated, 59.2%; ≥6 mos chelation, 45.5%) were more prevalent in non-chelated pts at baseline; this trend continued on study: cardiac (non-chelated, 49.7%; ≥6 mos chelation, 42.5%); vascular (non-chelated, 55.7%; ≥6 mos chelation, 48.5%; NS, all comparisons). Most frequent causes of death were MDS/AML, cardiac events, and infection. The percentage of pts who had ever received MDS therapy was lower among non-chelated pts (non-chelated, 88.4%; ≥6 mos chelation, 93.5%; NS). Conclusions: At 36 mos, chelated pts had significantly longer OS and time to AML transformation, as well as significantly fewer deaths. Trends toward fewer AML transformations and cardiac disorders were observed in chelated pts. Baseline characteristics and IPSS risk status were similar between groups, with the exception of more prevalent cardiac and vascular comorbidities in non-chelated pts. Additional assessments over the 5-year duration of this registry will provide further information on the association between chelation and clinical outcomes. Disclosures: Lyons: Novartis: Research Funding; Amgen: Consultancy, Research Funding; Incyte: Consultancy, Research Funding; Telik: Research Funding. Paley:Novartis: Employment. Esposito:Novartis: Employment. Garcia-Manero:Novartis: Research Funding.

scholarly journals Red blood cell transfusions and iron overload in the treatment of patients with myelodysplastic syndromes

Cancer ◽  
2008 ◽  
Vol 112 (5) ◽  
pp. 1089-1095 ◽  
Author(s):  
Elias Jabbour ◽  
Hagop M. Kantarjian ◽  
Charles Koller ◽  
Ali Taher
Keyword(s):  
Blood Cell ◽  
Iron Overload ◽  
Red Blood Cell ◽  
Myelodysplastic Syndromes ◽  
Red Blood Cell Transfusions

scholarly journals Iatrogenic Iron Overload in an End Stage Renal Disease Patient

2020 ◽  
Vol 13 (2) ◽  
pp. 760-763
Author(s):  
Majd M. Aldwairi ◽  
Mohamed A. Yassin
Keyword(s):  
Renal Failure ◽  
Blood Cell ◽  
Iron Overload ◽  
Red Blood Cell ◽  
Disease Patient ◽  
Intravenous Iron ◽  
Human Erythropoietin ◽  
Red Blood Cell Transfusions ◽  
Stage Renal Disease ◽  
End Stage

Iron overload is a common complication in patients with chronic renal failure treated with dialysis prior to the availability of recombinant human erythropoietin therapy. Iron overload was the result of hypoproliferative erythroid marrow function coupled with the need for frequent red blood cell transfusions to manage symptomatic anemia. The repetitive use of intravenous iron with or without the use of red blood cell transfusions also contributed to iron loading and was associated with iron deposition in liver parenchymal and reticuloendothelial cells. Here we report a 56-year-old female with end-stage renal failure who underwent kidney transplant twice and found to have iatrogenic iron overload with excess intravenous iron treated conservatively.

Impact of Red Blood Cell Transfusions on Survival in Lower-Risk MDS Patients Included in the European Leukemianet MDS (EUMDS) Registry

2017 ◽  
Vol 55 ◽  
pp. S160-S161
Author(s):  
L. de Swart ◽  
S. Crouch ◽  
A. Smith ◽  
P. Fenaux ◽  
A. Symeonidis ◽  
...  
Keyword(s):  
Blood Cell ◽  
Red Blood Cell ◽  
Lower Risk ◽  
Red Blood Cell Transfusions

Multiple red blood cell transfusions and iron overload in very low birthweight infants

Vox Sanguinis ◽  
2017 ◽  
Vol 112 (5) ◽  
pp. 453-458 ◽  
Author(s):  
J. D. Treviño-Báez ◽  
E. Briones-Lara ◽  
J. Alamillo-Velázquez ◽  
M. I. Martínez-Moreno
Keyword(s):  
Blood Cell ◽  
Iron Overload ◽  
Red Blood Cell ◽  
Low Birthweight ◽  
Very Low Birthweight ◽  
Red Blood Cell Transfusions ◽  
Very Low Birthweight Infants

scholarly journals Marrow transplantation for patients with thalassemia: results in class 3 patients

Blood ◽  
1996 ◽  
Vol 87 (5) ◽  
pp. 2082-2088 ◽  
Author(s):  
G Lucarelli ◽  
RA Clift ◽  
M Galimberti ◽  
P Polchi ◽  
E Angelucci ◽  
...  
Keyword(s):  
Blood Cell ◽  
Iron Overload ◽  
Liver Damage ◽  
Red Blood Cell ◽  
Marrow Transplantation ◽  
Proportional Hazards ◽  
Conditioning Regimen ◽  
Rejection Rate ◽  
Red Blood Cell Transfusions ◽  
Related Mortality

Thalassemia patients can be categorized as class 1 (minimal liver damage and iron overload), class 3 (extensive liver damage from iron overload), and class 2 (intermediate). These categories are prognostic for treatment outcome after marrow transplantation. Class 3 patients have more transplant-related mortality than other patients. This study examines transplantation outcome for class 3 patients. Records were reviewed of 215 patients in class 3 who received transplants in Pesaro from HLA-identical related donors between May 1, 1984 and May 1, 1994. The influence of pretransplant, peritransplant, and posttransplant variables on survival, relapse, and transplant-related mortality was examined by product-limit and proportional-hazards multivariate analysis. Age and conditioning regimen were influential on survival, and regimens with less than 200 mg/kg cyclosporine (CY) were associated with 5-year survival probabilities of .74 and .63 patients younger than 17 years and older patients, respectively. Transfusion history and regimen were influential on rejection with 5 year probabilities of .53 and .24 in patients who received less than or greater than 100 red blood cell transfusions before transplantation and regimens containing less than 200 mg/kg CY. Results of transplantation for patients with advanced thalassemia treatment have improved with the introduction of conditioning regimens with less CY. This has been associated with an increase in rejection (particularly in patients who have received < 100 red blood cell transfusions before transplant). Efforts at reducing the rejection rate by modifying the conditioning regimen should be concentrated on younger patients who have received a small number of transfusions. Patients with thalassemia who have HLA-identical family members should be transplanted before they are in class 3.

48-Month Update On Survival and AML Transformation In a 600-Patient Registry Of Lower-Risk MDS Patients

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2775-2775 ◽  
Author(s):  
Roger M. Lyons ◽  
Billie J. Marek ◽  
Carole S. Paley ◽  
Jason Esposito ◽  
Katie McNamara ◽  
...  
Keyword(s):  
Blood Cell ◽  
Clinical Outcomes ◽  
Red Blood Cell ◽  
Lower Risk ◽  
Research Funding ◽  
Causes Of Death ◽  
Pharmaceutical Research ◽  
Comorbid Conditions ◽  
Leukemic Transformation ◽  
Risk Status

Abstract Introduction Packed red blood cell transfusion is often required for the treatment of anemia in patients (pts) with myelodysplastic syndromes (MDS). Transfusion requirement is associated with poorer clinical outcomes and reduced overall survival (OS) in MDS. We prospectively collected data on clinical outcomes in chelated and non-chelated, transfused, lower-risk MDS pts. OS, leukemic transformation, and clinical events are reported for these groups at 48 mos on study. Methods This 5-year, non-interventional registry enrolled 599 pts from 107 US centers. Pts were ≥18 years old with lower-risk MDS (WHO, FAB classification, and/or IPSS) and transfusional iron overload (serum ferritin ≥1000 μg/L and/or ≥20 packed red blood cell units and/or ≥6 units every 12 weeks). The chelated group included all pts who had ever used iron chelation; sub-analysis was performed on pts with ≥6 mos chelation. Assessments were every 6 mos for 5 years or until death and included demographics, survival, disease status, comorbidities, causes of death, and MDS therapy. Results We present results for non-chelated pts and those chelated ≥6 mos. Baseline demographics and IPSS risk status were similar between groups, although transfusion burden appeared higher in chelated pts (Table 1). At 48 mos, 120 pts continued on registry and 479 had discontinued (379 died [63.3%]; 64 lost to follow-up [10.7%]; 25 other [4.2%]; and 11 completed the study [1.8%]). In all, 269 pts (44.9%) received chelation therapy; 202 had ≥6 mos lifetime chelation. The percentage of pts who had ever received MDS therapy was lower among non-chelated (88.2%) vs chelated ≥6 mos pts (93.6%; P=0.0425). At enrollment, cardiac and vascular comorbid conditions were higher and endocrine conditions trended higher in non-chelated vs chelated ≥6 mos pts (52.1% vs 30.7% [P<0.0001], 59.4% vs 45.0% [P=0.0013], and 43.9% vs 35.6% [P=0.0588], respectively). While on registry, cardiac, vascular, and endocrine comorbid conditions all trended higher in non-chelated vs chelated ≥6 mos pts (50.9% vs 44.1%, 56.4% vs 49.0%, and 40.3% vs 38.6%, respectively; P>0.05 all comparisons). Presence of cardiovascular comorbidities was associated with a significantly shorter mean (SE) OS (89.1 [5.83] mos vs 85.2 [4.43] mos; P<0.01); however this association was not seen with endocrine comorbidities. Median OS was longer in chelated ≥6 mos vs non-chelated pts (P<0.0001; Table 2). Most frequent causes of death were MDS/acute myeloid leukemia (AML), cardiac events, and infection. Time from diagnosis to leukemic transformation was longer in chelated ≥6 mos vs non-chelated pts (P<0.0001). Conclusions At 48 mos, chelated pts had significantly longer OS and time to AML transformation. At baseline, fewer chelated ≥6 mos vs non-chelated pts had cardiac and vascular comorbidities. Baseline characteristics and IPSS risk status were similar between groups. Additional assessments over the 5-year duration of this registry will provide further information on the association between chelation and clinical outcomes. Disclosures: Lyons: Incyte: Consultancy, Research Funding; Amgen: Consultancy, Research Funding; Novartis Pharmaceutical: Research Funding; Telik: Research Funding. Paley:Novartis Pharmaceuticals: Employment. Esposito:Novartis Pharmaceuticals: Employment. McNamara:Novartis Pharmaceutical: Employment. Garcia-Manero:Novartis Pharmaceutical: Research Funding.

scholarly journals Marrow transplantation for patients with thalassemia: results in class 3 patients

Blood ◽  
1996 ◽  
Vol 87 (5) ◽  
pp. 2082-2088 ◽  
Author(s):  
G Lucarelli ◽  
RA Clift ◽  
M Galimberti ◽  
P Polchi ◽  
E Angelucci ◽  
...  
Keyword(s):  
Blood Cell ◽  
Iron Overload ◽  
Liver Damage ◽  
Red Blood Cell ◽  
Marrow Transplantation ◽  
Proportional Hazards ◽  
Conditioning Regimen ◽  
Rejection Rate ◽  
Red Blood Cell Transfusions ◽  
Related Mortality

Abstract Thalassemia patients can be categorized as class 1 (minimal liver damage and iron overload), class 3 (extensive liver damage from iron overload), and class 2 (intermediate). These categories are prognostic for treatment outcome after marrow transplantation. Class 3 patients have more transplant-related mortality than other patients. This study examines transplantation outcome for class 3 patients. Records were reviewed of 215 patients in class 3 who received transplants in Pesaro from HLA-identical related donors between May 1, 1984 and May 1, 1994. The influence of pretransplant, peritransplant, and posttransplant variables on survival, relapse, and transplant-related mortality was examined by product-limit and proportional-hazards multivariate analysis. Age and conditioning regimen were influential on survival, and regimens with less than 200 mg/kg cyclosporine (CY) were associated with 5-year survival probabilities of .74 and .63 patients younger than 17 years and older patients, respectively. Transfusion history and regimen were influential on rejection with 5 year probabilities of .53 and .24 in patients who received less than or greater than 100 red blood cell transfusions before transplantation and regimens containing less than 200 mg/kg CY. Results of transplantation for patients with advanced thalassemia treatment have improved with the introduction of conditioning regimens with less CY. This has been associated with an increase in rejection (particularly in patients who have received < 100 red blood cell transfusions before transplant). Efforts at reducing the rejection rate by modifying the conditioning regimen should be concentrated on younger patients who have received a small number of transfusions. Patients with thalassemia who have HLA-identical family members should be transplanted before they are in class 3.

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