scholarly journals PATTERN AND CLINICAL PROFILE OF PATIENTS WITH Β- THALASSEMIA IN REPEATED BLOOD TRANSFUSION

2020 ◽  
pp. 32-34
Author(s):  
Ashok Badakali ◽  
Deepti Shetty ◽  
Manohar MR
Keyword(s):  
Blood Transfusion ◽  
Iron Overload ◽  
Serum Calcium ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Serum Phosphate ◽  
Clinical Profile ◽  
Beta Thalassemia ◽  
Serum Phosphate Level ◽  
The Mean

Chronic transfusions inevitably lead to iron overload as humans cannot actively remove excess iron. The cumulative effects of iron overload lead to significant morbidity and mortality, if untreated. The combination of transfusion and chelation therapy has dramatically extended the life expectancy of thalassemia patients, but with complications like hypocalcaemia. Hence, present study was undertaken to determine pattern and clinical profile of patients with β- thalassemia who are receiving repeated blood transfusion Methods: Hospital based study conducted at S. Nijalingappa Medical College and Hanagal Shri Kumareshwar hospital, Bagalkot. The study period was one and half year from 2015 to 2016. 53 beta thalassemia major cases fulfilling inclusion criteria were investigated after an informed consent, for serum calcium, serum phosphorous, serum ALP and paratharmone levels. Result: Among 53 transfusion dependent children studied, the mean age is 5.249 years. The study consisted of 32 (60.4%) males and 21 (39.6%) females. Maximum number of cases i.e. 29 (54.7%) were diagnosed at the age of 4-6 months. 50 (94.3%) were on iron chelation therapy. The mean serum calcium is 8.28 + 0.89 mg/dl. The mean serum phosphate is 6.40 + 0.80mg/dl, mean PTH is 14.96 + 15.49ng/L. The mean value of serum phosphate level is 14.96 + 15.49 ng/L. The mean ALP is 166.789 U/L. Conclusion: To get better results, regular testing is needed to detect the complications of the early stages with proper treatment of the factors and complications. Therefore, should be monitored to avoid complication related to hypocalcemia.

Effective and Safe Deferasirox Treatment of Patients with Various anemia's and Transfusional Iron-Overload in the Medical Practice: Results From Two German Observational Studies

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5165-5165
Author(s):  
Christian Junghanss ◽  
Rudolf Schlag ◽  
Bernd Gaede ◽  
Matthias Moelle ◽  
Steffen Doerfel ◽  
...  
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Observational Studies ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelator ◽  
Beta Thalassemia ◽  
Final Visit ◽  
Iron Chelation Therapy ◽  
The Mean

Abstract Abstract 5165 Background: Progressive anaemia is highly prevalent amongst many malignant diseases leading to RBC transfusion-dependency. Therefore transfusion-related iron overload (IOL) is common in these patients (pts) and can result in multiple organ failure. Iron chelation therapy prevents organ failure, reduces the risk of infections and can improve hematopoesis in some diseases. The once-daily oral iron chelator deferasirox has been shown to reduce iron overload in pts with various transfusion-dependent anaemias assessed by serum ferritin (SF). Despite extensive knowledge of iron chelation in MDS or beta-thalassemia pts, data in pts with other anaemias is limited. Here, we present data from a subgroup of transfusion-related IOL pts that were included two non-interventional studies (EXTEND, EXJANGE) performed in Germany and who suffered from diseases other than MDS or beta thalassemia. Methods: 130 pts with various malignant diseases such as myeloproliferative disorders (43 pts, including 31 pts particular specified as myelofibrosis), acute myeloid leukaemia (14 pts), sickle cell anaemia (6 pts), aplastic anaemia (11), congenital aplastic anaemia (5) or Non-Hodgkin's lymphoma (6 pts) were treated with deferasirox in the daily-routine setting of office-based physicians and included in either the EXTEND or EXJANGE study. Patient with MDS or beta-thalassemia were also included in the studies, but are excluded from this analysis. Analysis is based on 1-year pooled data of these two, multicenter, non-interventional observational studies. Transfusion-dependent pts with IOL with or without prior chelation were enrolled and received the iron chelator deferasirox. Prescription of deferasirox, just as inclusion and exclusion criteria was in accordance with the terms of Exjade marketing authorization in the EU. Efficacy and safety parameters, including serum ferritin and adverse events (AEs), were collected in 2-monthly intervals. Results: 98 pts had no prior chelation therapy (51 M, 45 F, 2 missing; mean age 63.3, range 3.2–91.9 yrs) and a median baseline SF of 2,968 (range 561–11, 423) ng/mL. 32 pts had prior received prior chelation therapy (mainly with desferal; 17 M, 15 F; mean age 50.1, range 3.5–80.9 yrs) and a median baseline SF of 2,635 (range 539–19, 540) ng/mL. The mean number of prior red blood cell transfusions was 55. The mean prescribed daily dose of deferasirox at the first visit was 16.3 mg/kg/d rising up to 18.1 mg/kg/d after 12 months. During treatment, median SF levels clearly decreased from first to final visit [-806 ng/mL; p<0.0001 (explorative analysis)] in the chelation-naïve and also in the pre-chelated population [-300 ng/ml; p = 0.1705 (explorative analysis)]. The median observation period and days on therapy was 349 and 343 days, respectively. At final visit 74 pts (56.9%) were still on deferasirox therapy. Reasons for discontinuation by the final visit included 19 AEs (35.2%). 45 pts (34.6%) experienced an investigator assessed drug-related AE. The most common drug-related AEs were diarrhea (n=17; 37.8%), nausea (n=11; 24.4%) and blood creatinine increased (n=6; 13.3%). As in previous clinical trials, serum creatinine clearances showed a minor decrease over the study period (median decrease until final visit: 4 ml/min). Conclusion: Our analysis confirmed that deferasirox is effective and well tolerated in chelation-naïve as well as in previously chelated pts with transfusion-related IOL and diseases other than MDS or beta thalassemia. As baseline serum ferritin values were >2,500 ng/mL even in pts with prior chelation therapy, adequate chelation treatment should be considered earlier at a serum ferritin >1,000 ng/mL in pts with transfusion-dependent IOL for adequate iron chelation therapy. Disclosures: Junghanss: Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Haus:Novartis Pharma: Employment. Junkes:Novartis: Employment. Leismann:Novartis: Employment.

The Correlation Of Hepatic Iron Loading Determined By Magnetic Resonance Image and Serum Ferritin In Patients With Beta-Thalassemia Intermedia

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2203-2203
Author(s):  
Ampaiwan Chuansumrit ◽  
Jiraporn Laothamathat ◽  
Nongnuch Sirachainan ◽  
Pakawan Wongwerawattanakoon ◽  
Praguywan Kadekasem ◽  
...  
Keyword(s):  
Blood Transfusion ◽  
Serum Ferritin ◽  
Iron Chelation ◽  
Dry Weight ◽  
Beta Thalassemia ◽  
Hepatic Iron ◽  
Iron Loading ◽  
Thalassemia Intermedia ◽  
The Mean ◽  
Mri Study

Abstract Background Current practice of starting iron chelation when ferritin values reach 1,000 mcg/l has been given to patients with thalassemia disease. The body iron load in patients with thalassemia intemedia can be different from other severe thalassemia disease due to the increased intestinal iron absorption. Objective The correlation of hepatic iron loading determined by magnetic resonance image study (MRI) and various parameters was calculated. Materials and Methods MRI study, using CRMtool to determine the myocardial and hepatic iron loading, was performed in pediatric patients with beta-thalassemia intermedia. The amount of blood transfusion and duration of iron chelation were recorded. The levels of serum ferritin as well as the transfusion-transmitted diseases were checked twice yearly. Results In all, 40 patients (19 males, 23 females) manifested as beta-thalassemia intermedia were enrolled in the study. They included beta thalassemia/HbE disease (n=37) and beta thalassemia major (n=3) with the mean age of 14.9±3.6 years. Three patients with beta-thalassemia major behaved as beta-thalassemia intermedia since two patients carried the combination of beta-thalassemia0 and beta-thalassemia+ genes while another patient had an additional alpha-thalassemia gene. The remaining patients possessed the combination of beta-thalassemia genes at codon 41/42 (4 base pair deletion) and HbE gene at codon 26 (GAG>AAG). All patients received routine hepatitis B vaccination. None had positive serological testing for HBsAg, antiHCV or antiHIV. They required regular transfusion to maintain their pre-transfusion hematocrit at 24% starting at the mean age of 4.1±3.3 years with the mean duration of 10.1±4.6 years. Nineteen were splenectomized at the mean age of 8.0±3.1 years. They all received iron chelation of 10-12 hours of desferrioxamine subcutaneous injection, oral deferiprone ingestion or the combination of desferrioxamine and deferiprone starting at the mean age of 9.1±3.8 years with the mean duration of 6.2 ±4.3 years. At the mean age of 14.9±3.6 years, they underwent MRI study and revealed that the mean T2* of myocardium was 38.6±8.1 milliseconds (ms) and mean T2* of liver was 3.2±2.0 ms. Neither patients had myocardial iron loading while 36 patients had hepatic iron loading varying from severe degree of <1.4 ms (iron >10 mg/g dry weight, n=4), moderate degree of >1.4-2.7 ms (iron >5-10 mg/g dry weight, n=16) and mild degree of >2.7-6.3 ms (iron 2-5 mg/g dry weight, n=16). The results revealed no correlation of the hepatic T2* and the duration of blood transfusion (p=0.157), duration of iron chelation (p=0.071), total blood transfusion and total iron loading from transfusion (p=0.471) one-year blood transfusion and one-year iron loading from transfusion (p=0.321) except for the serum ferritin (p=0.001). The geographic mean of ferritin was 1584.9 mcg/l. The hepatic iron loading by MRI was shown in the equation of T2*(ms) = 4.663-0.001ferritin (mcg/l) (r=-0.503, p=0.001). Patients with serum ferritin ≥1,000 mcg/l risked hepatic iron loading (T2*<2.7 ms, iron 5-10 mcg/g dry weight) with an odds ratio of 5.07 (95% CI 1.09-23.44). Therefore, patients with beta-thalassemia intermedia were at risk of hepatic iron overloading if the initiation of iron chelation started at the serum ferritin of 1,000 mcg/l. Conclusion The current practice of starting iron chelation when ferritin values reach 1,000 mcg/l risks hepatic iron loading in patients with beta-thalassemia intermedia. Advanced technology for evaluating hepatic iron loading is suggested. Thus, where MRI study is not feasible, serum ferritin can be used to estimate the hepatic T2*. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Correlation between serum ferritin level, cardiac and hepatic T2-star MRI in patients with β-thalassemia major in Al-Diwaniya thalassemia center

2018 ◽  
Vol 9 (SPL1) ◽  
Author(s):  
Alaa Mutter Jabur Al-Shibany ◽  
AalanHadi AL-Zamili
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Chelation Therapy ◽  
Ferritin Level ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Iron Level ◽  
The Mean ◽  
T2 Mri

Patients with transfusion dependent thalassemia major is often associated with iron overload. Proper use of iron chelators to treat iron overload requires an accurate measurement of iron levels. Magnetic resonance T2-star (T2* MRI) is the preferred method to measure iron level in the liver andthe heart. The goal of our study was to see if there is an association exists between serum ferritin level and T2* MRI results in patients with beta thalassemia major.This study was done in Al-Diwaniya Thalassemia center,Maternity and children teaching hospital,Iraq. During the period from 1st of January to 31st of October. Fifty eight patients with a diagnosis of beta thalassemia major were enrolled in the study. They were older than five years old,transfusion dependent and on chelation therapy. Hepatic and Myocardial T2*MRI and the mean serum ferritin levels were measured during the study period for all patients.There is a significant correlation was observed between serum ferritin level and cardiac T2*MRI (p=0.018 ). also a significant correlation was observed between serum ferritin and hepatic T2*MRI (p=0.02). Neither cardiac T2* MRI nor hepatic T2* MRI show any correlation with the mean age.our study also showa positive correlation between the patients withcardiac T2* MRI and the development of diabetes mellitus in contrast to hepatic T2* MRI in which there is no any correlation. Hypothyroidism was observedno correlation with either cardiac or hepatic T2* MRI.Our results showed a positiveassociation between hepatic, cardiac T2*MRI and serum ferritin levels.

scholarly journals Serum calcium and serum phosphate levels in transfusion dependent beta thalassemia

2018 ◽  
Vol 13 (2) ◽  
pp. 54-58
Author(s):  
Mst Ariza Sultana ◽  
Qazi Shamima Akhter
Keyword(s):  
Serum Calcium ◽  
Calcium Level ◽  
High Serum ◽  
Serum Phosphate ◽  
Phosphate Level ◽  
Beta Thalassemia ◽  
Control Group ◽  
Serum Phosphate Level ◽  
Ethical Aspect ◽  
Cross Sectional

Background: Patients with transfusion dependent beta thalassemia with severeanemia require regular blood transfusion to improve quality of life. This can lead to iron overload which might cause various complications including hypocalcaemia. Objective: To estimate the serum calcium and phosphate levels in transfusion dependent beta thalassemia patients. Methods: This cross-sectional study was conducted in the Department of Physiology, Dhaka Medical College, Dhaka from July 2016 to June 2017. After fulfilling the ethical aspect, a total number of 60 subjects were selected with the age ranging from 5 to 25 years. Among them, 40 transfusion dependent beta thalassemia patients were selected as the study group and 20 age and sex matched apparently healthy individuals were considered as control group for comparison. The study population were selected from Thalassemia foundation hospital, Dhaka. Theserum calcium and phosphate levels were estimated by autoanalyzer.. For statistical analysis, unpaired Student’s‘t’ test, Chi-square test were performed as applicable. Results: In this study, serum calcium level were significantly (p < 0.001) lower and serum phosphate level were significantly (p < 0.001) higher in transfusion dependent beta thalassemia patients as compared to healthy controls. In addition, 67.5% thalassemia patients had hypocalcemia (calcium level < 8.5 mg/dl) and 85% of thalassemia patients had hyperphosphatemia(phosphate level > 4.7mg/dl). Conclusions: This study concludes transfusion dependent beta thalassemiapatients have low calcium level and high serum phosphate level which should be monitored to avoid complications related to hypocalcaemia and hyperphosphatemia. J Bangladesh Soc Physiol. 2018, December; 13(2): 54-58

Leptin Is Associated with the Degree of Anemia and the Erythropoietin Levels in β Thalassemia Patients

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 950-950
Author(s):  
Lila Mahagna ◽  
Osama Tanous ◽  
Tal Dujovny ◽  
Harel Eitam ◽  
Refaat Masalha ◽  
...  
Keyword(s):  
Adipose Tissue ◽  
Iron Overload ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Healthy Controls ◽  
Fat Percentage ◽  
Anthropometric Parameters ◽  
Chronic Anemia ◽  
Serum Leptin ◽  
The Mean

Abstract Background: β-thalassemia (BT) is a hereditary hemolytic anemia. The imbalance between α- and β-globin chain synthesis results in ineffective erythropoiesis, severe microcytic hypochromic anemia and iron overload. Although regular transfusions and iron-chelation therapy markedly improve the survival and quality of life of BT patients, they have also led to the emergence of previously unrecognized complications. Patients with thalassemia major often present with endocrine abnormalities due to dysfunction of the hypothalamic-pituitary axis (Poggi, 2016), and are frequently underweight with abnormally low body-fat percentage (Fung, 2010). Adipose tissue stores lipids, but it also has an endocrine function through the production of leptin, an adipocyte-derived hormone that regulates food intake, metabolic and endocrine functions by activating its receptor in the central nervous system. Leptin plays a regulatory role in immunity and inflammation, and also seems to act synergistically with erythropoietin on mammalian hematopoiesis (Bennett, 1996). For example, a stimulatory effect of leptin on erythropoiesis among patients with end-stage renal disease was observed (Axelsson 2005). Previous studies on leptin in BT patients have shown lower levels than in healthy controls, and a negative correlation with the level of soluble transferrin receptor in transfused patients (Dedoussis, 2002). The reduced leptin levels were explained by a possible toxic effect of iron on adipocytes (Chaliasos, 2010). Study aims: In this study, we investigated the correlation between leptin level and anthropometric parameters in BT patients compared to heathy controls. We also explored the relationship between leptin and hematological and erythropoietic parameters, as well as iron-overload status in BT patients. Patients and methods: Transfusion-dependent BT patients (n = 33; 16 females, 17 males, mean age 23.5 ± 8.6 [range 8-41] years), treated at the Pediatric Hematology Unit of Emek Medical Center, and 11 healthy controls (6 females, 5 males, mean age 26.4 ± 10.4 [range 8-39] years) were studied. Patients were treated by regular blood transfusions and iron-chelation therapy. Anthropometric assessments included height, weight, fat percentage and BMI calculation. Blood samples were obtained at fasting and before blood transfusion. Serum leptin, complete blood count, hemoglobin and reticulocyte counts and serum erythropoietin were analyzed. These studies were performed twice, 3 months apart, and the mean values were utilized for the statistical analysis. Serum leptin levels were analyzed by radioimmunoassay, and leptin-normalized values were calculated (ng/dL leptin per % body fat). Results: BT patients were found to have lower leptin levels than healthy controls (5.4 ± 5.9 vs. 13 ± 10.1 ng/dL; p= 0.0006). Leptin levels were higher in females than in males (mean leptin 7.2 ± 6.5 vs. 3.7 ± 4.7 ng/dL; p= 0.01), and increased with age (r= 0.4635, p= 0.0066). A negative correlation was found between leptin and erythropoietin (r= -0.43473, p= 0.0115), and a positive correlation between leptin and hemoglobin, as well as the mean pre-transfusion hemoglobin levels in the previous 5 years (r= 0.66884, p &lt; 0.001; r= 0.40261, p= 0.0202, respectively). No correlation was observed between leptin levels and anthropometric parameters (weight, height, BMI), iron-overload parameters or reticulocyte count. Fasting and normalized leptin showed similar patterns. Conclusions: This study confirms that BT patients are unable to maintain adequate leptin production, suggesting that adipose tissue dysfunction may be related to the chronic anemia. Our results correlate well with previous studies of lower leptin levels in BT patients. The positive correlation of leptin level with hemoglobin, together with the inverse correlation with erythropoietin provide further evidence of the effect of leptin on erythropoiesis. Additional studies are needed to examine the intricate interplay between adipose tissue, leptin and erythropoiesis in the environment of chronic anemia and iron overload present in BT patients. Disclosures No relevant conflicts of interest to declare.

Supportive care and chelation therapy in MDS: are we saving lives or just lowering iron?

Hematology ◽  
2009 ◽  
Vol 2009 (1) ◽  
pp. 664-672 ◽  
Author(s):  
Heather A. Leitch ◽  
Linda M. Vickars
Keyword(s):  
Iron Overload ◽  
Supportive Care ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Left Ventricular ◽  
Left Ventricular Ejection ◽  
Beta Thalassemia ◽  
Ventricular Ejection Fraction ◽  
The Impact

AbstractThe myelodysplastic syndromes (MDS) are characterized by cytopenias and risk of transformation to acute myeloid leukemia (AML). Although new treatments are available, a mainstay in MDS remains supportive care, which aims to minimize the impact of cytopenias and transfusion of blood products. Red blood cell (RBC) transfusions place patients at risk of iron overload (IOL). In beta-thalassemia major (BTM), IOL from chronic RBC transfusions inevitably leads to organ dysfunction and death. With iron chelation therapy (ICT), survival in BTM improved from the second decade to near normal and correlated with ICT compliance. Effects of ICT in BTM include reversal of cardiac arrhythmias, improvement in left ventricular ejection fraction, arrest of hepatic fibrosis, and reduction of glucose intolerance.It is not clear whether these specific outcomes are applicable to MDS. Although retrospective, recent studies in MDS suggest an adverse effect of transfusion dependence and IOL on survival and AML transformation, and that lowering iron minimizes this impact. These data raise important points that warrant further study. ICT is potentially toxic and cumbersome, is costly, and in MDS patients should be initiated only after weighing potential risks against benefits until further data are available to better justify its use. Since most MDS patients eventually require RBC transfusions, the public health implications both of transfusion dependence and ICT in MDS are considerable. This paper summarizes the impact of cytopenias in MDS and treatment approaches to minimize their impact, with a focus on RBC transfusions and their complications, particularly with respect to iron overload.

Assessment of the Relationship Between Iron Overload Based on Cardiac T2* MRI and Fragmented QRS in Beta-Thalassemia Major Patients

2020 ◽  
Vol 21 (8) ◽  
Author(s):  
Yazdan Ghandi ◽  
Danial Habibi ◽  
Aziz Eghbali
Keyword(s):  
Iron Overload ◽  
Chelation Therapy ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Cardiac Iron ◽  
Fragmented Qrs ◽  
Beta Thalassemia Major ◽  
Cardiac Iron Overload ◽  
The Mean ◽  
T2 Mri

Background: Cardiac involvement in beta-thalassemia major patients is an important cause of mortality. Therefore, in these patients, timely diagnosis of cardiac disorder is essential. Objectives: The present study aimed at determining the association between cardiac iron overload and fragmented QRS (fQRS). Methods: This cross-sectional study was conducted on 40 β-TM patients, aged 5 - 40 years. The presence of fQRS was evaluated in 12-lead surface electrocardiograms. Cardiac T2* MRI was performed to determine the iron overload. The patients were divided into four groups of chelation therapy. Results: The mean age of patients was reported to be 22.50 ± 6.75 years. The groups showed no significant difference regarding gender, age, or left ventricular ejection fraction. The presence of fQRS was detected in 10 patients (25%), while T2* value was lower than 20 ms in 10 patients (25%). The mean age of patients with and without fQRS was 26.23 ± 2.71 and 19.40 ± 2.61 years, respectively (P = 0.001). The univariate analysis indicated that fQRS had a significant relationship with cardiac iron overload (OR = 5; 95% CI: 1.04 - 23.99; P < 0.044). The multiple logistic regression analysis represented a significant association between iron overload and fQRS (OR = 5.556; 95% CI: 1.027 - 30.049). The sensitivity and specificity of the fQRS against MRI were equal to 50% and 83.3% respectively. Conclusions: The absence of fQRS on ECGs could be a good predictor of the lack of cardiac iron overload in β-TM patients. The results showed that fQRS might indicate the no need for close monitoring for cardiac overload with cardiac MRI and aggressive chelation therapy.

Latin American Registry of Patients (Pts) with Transfusional Hemosiderosis (Th): The RELATH Study.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4013-4013 ◽  
Author(s):  
Rodolfo Cancado ◽  
Ivan Angulo ◽  
Zaida Plumacher ◽  
Mariela Moreno ◽  
Adriana Linares ◽  
...  
Keyword(s):  
Iron Overload ◽  
Latin American ◽  
African Ancestry ◽  
Tertiary Care ◽  
Iron Chelation ◽  
Refractory Anemia ◽  
Beta Thalassemia ◽  
Liver Iron ◽  
The Mean ◽  
Rbc Transfusion

Abstract In Latin America (LA), miscegenation of populations of Mediterranean and African ancestry has occurred for several centuries, thus facilitating the spread of hemoglobin variants. The prevalence of thalassemias, sickle-cell disease (SCD) and other hemoglobinopathies, as well as of myelodysplastic syndromes (MDS) and other diseases associated with TH is largely unknown in LA. As a retrospective registry of pts with TH in LA, the RELATH Study provides a unique opportunity to gain insight about the prevalence of TH and the patterns of care for these pts in LA. Participating countries include Brazil, Colombia, Mexico, Peru, and Venezuela. Target accrual is approximately 1,000 patients. Cases are accrued by large-volume, tertiary-care hematology centers located in large cities through a CRF designed for the study. Treatment and pt evaluation are not influenced by the study. Eligible pts have age >2 yr, consultation in the participating institutions at least once since 01/04, any disorder requiring chronic red-blood-cell (RBC) transfusion, receipt of >9 RBC units, at least one value of serum ferritin >1000 mcg/L, and/or a liver iron content (LIC) >2 mg/g dry weight (pts. with leukemia are excluded). Between Jan/06 and May/07, 239 pts have been accrued, 236 of which are evaluable. The mean age was 29.2 +/− 20.4 (range, 2 to 92), and 53.8% of pts were female. Ethnic distribution was Hispanic (41.0%), African (34.6%), and Caucasian ancestry (24.4%). The most frequent diagnoses were SCD (43.6%), beta-thalassemia major (17.4%), aplastic anemia (13.6%), and MDS (7.2%, 53.3% of which had refractory anemia). RBC transfusion was > 9 in 100% and >19 in 89.8% of pts, and mean ferritin was 2564 +/− 1834 mcg/L. LIC determination was not available or not done in 90.6% of cases. The level of hemoglobin at which transfusion was indicated was 7 to 10 g/dL in 60.7%, and 6 g/dL or less in 38.1% (N/A in 1.3%). The mean number of transfusions received was 12.2 +/− 8.8/yr (range, 1 to 80). Iron overload was assessed using ferritin (93.2%), and TH related complications were evaluated with echocardiogram (42.8%), and liver US (27.5%). TH-related complications were reported in 82.2% of cases (66.5% of pts had hepatic complications, 32.6% endocrine, 16.1% cardiac). Iron-chelation therapy was given to 39.8% of pts, more frequently on the basis of ferritin (27.5%), number of transfusions (18.2%), and complication from iron overload (5.5%). Deferoxamine (93.6%) and deferasirox (6.4%) were the most frequent chelators. In most cases, treatment was still ongoing, but reasons for discontinuation were pt refusal (4.7%), drug no longer available (4.2%), and poor compliance (3.8%). In conclusion, this preliminary report from the ongoing RELATH study shows that a registry is feasible and may provide valuable information regarding TH in various countries of LA. In addition, the study suggests so far that most LA patients undergoing chronic transfusion develop TH, whose complications could be prevented by more effective use of iron chelation, which was relatively low in this sample.

Serum Ferritin Elevation and Use of Iron Chelation In Chronically Versus Sporadically Transfused Sickle Cell Patients

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2671-2671
Author(s):  
Ismael Shaukat ◽  
Faraz Khan ◽  
Andrew Eisenberger ◽  
Marcus Stevenson ◽  
Alice J. Cohen
Keyword(s):  
Sickle Cell Disease ◽  
Iron Overload ◽  
Renal Dysfunction ◽  
Sickle Cell ◽  
Serum Ferritin ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Cell Disease ◽  
The Mean

Abstract Abstract 2671 Background: Red cell transfusions play an integral role in the treatment and prevention of serious complications related to sickle cell disease. It has been shown that in other hemoglobinopathies, such as β-Thalassemia, patients (pts) suffer from iron overload which can result in end organ damage. There is concern that heavily transfused sickle cell pts may also develop iron overload with consequent morbidity and mortality. While pediatric pts routinely receive blood transfusions and iron chelation therapy, adult pts often discontinue chronic transfusion programs and are transfused sporadically. These pts may not receive routine iron chelation therapy. Methods: A retrospective review of our sickle cell database from 1988–2010 which also included those pts who were not routinely followed at the comprehensive sickle cell clinic. Adult pts (>18 yrs of age) with serum ferritin (SF) levels >1000 ng/ml (criteria for iron overload in our institution) were identified and use of iron chelation was reviewed in this population. Clinical characteristics evaluated were age, type of sickle cell disease, frequency of transfusions (chronic vs. sporadic), total units transfused, use and type of chelation, as well as reasons for non-use of chelation therapy. Results: 65/170(38%) pts were identified with SF >1000. The mean age is 33 years (range 19–70). 38/65 (59%) have the SS phenotype, 25/65 (38%) have the Sβ phenotype and 2/65 (3%) have the SC phenotype. The mean SF is 3697 ng/ml (range 1012–14312). Of those pts considered to have iron overload, 28/65 (43%) were treated with iron chelation: 27/65 (42%) received deferasirox and 1/65 (2%) received deferoxamine. Of the untreated pts, 24/37 (65%) had no identifiable reason for lack of chelation therapy, 10/37 (27%) had renal dysfunction, 1/37(3%) had hepatic impairment. 16/65 (25%) were transfused chronically, while 49/65 (75 %) were transfused sporadically. Chronically transfused pts received a mean of 81 units throughout their lifetime, while sporadically transfused pts received 30 units (p=0.01). The mean SF for chronically transfused pts was 5891, while the mean SF for pts transfused sporadically was 2981 (p=0.01). Of pts transfused chronically, 11/16 (69%) were on chelation therapy. Of the pts receiving sporadic transfusions, only 16/49 (33%) were on iron chelation (p= 0.01). In all pts chronically transfused, the reason for non-use of chelation therapy was renal dysfunction. In sporadically transfused pts, 33/49 (51%) had no identifiable reason for lack of chelation therapy. Conclusion: SF levels are significantly lower in pts who are sporadically transfused, though levels are high. Adult pts receiving sporadic transfusions are not routinely receiving iron chelation therapy despite elevated SF. The need for chelation therapy in both sporadically and chronically transfused pts remains to be determined. Disclosures: No relevant conflicts of interest to declare.

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