Improved survival in red blood cell transfusion dependent patients with primary myelofibrosis (PMF) receiving iron chelation therapy

2009 ◽  
pp. n/a-n/a ◽  
Author(s):  
Heather A. Leitch ◽  
Jocelyn M. Chase ◽  
Trisha A. Goodman ◽  
Hatoon Ezzat ◽  
Meaghan D. Rollins ◽  
...  
Keyword(s):  
Blood Cell ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Primary Myelofibrosis ◽  
Red Blood Cell Transfusion ◽  
Iron Chelation Therapy

scholarly journals Durable Red Blood Cell Transfusion Independence in a Patient with an MDS/MPN Overlap Syndrome Following Discontinuation of Iron Chelation Therapy

2015 ◽  
Vol 2015 ◽  
pp. 1-7 ◽  
Author(s):  
Harpreet Kochhar ◽  
Chantal S. Leger ◽  
Heather A. Leitch
Keyword(s):  
Blood Cell ◽  
Iron Overload ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Red Blood Cell Transfusion ◽  
Favorable Effect ◽  
Iron Chelation Therapy ◽  
Transfusion Independence ◽  
Rbc Transfusion

Background. Hematologic improvement (HI) occurs in some patients with acquired anemias and transfusional iron overload receiving iron chelation therapy (ICT) but there is little information on transfusion status after stopping chelation.Case Report. A patient with low IPSS risk RARS-T evolved to myelofibrosis developed a regular red blood cell (RBC) transfusion requirement. There was no response to a six-month course of study medication or to erythropoietin for three months. At 27 months of transfusion dependence, she started deferasirox and within 6 weeks became RBC transfusion independent, with the hemoglobin normalizing by 10 weeks of chelation. After 12 months of chelation, deferasirox was stopped; she remains RBC transfusion independent with a normal hemoglobin 17 months later. We report the patient’s course in detail and review the literature on HI with chelation.Discussion. There are reports of transfusion independence with ICT, but that transfusion independence may be sustained long term after stopping chelation deserves emphasis. This observation suggests that reduction of iron overload may have a lasting favorable effect on bone marrow failure in at least some patients with acquired anemias.

Improved survival with iron chelation therapy for red blood cell transfusion dependent lower IPSS risk MDS may be more significant in patients with a non-RARS diagnosis

2012 ◽  
Vol 36 (11) ◽  
pp. 1380-1386 ◽  
Author(s):  
Heather A. Leitch ◽  
Christopher Chan ◽  
Chantal S. Leger ◽  
Lynda M. Foltz ◽  
Khaled M. Ramadan ◽  
...  
Keyword(s):  
Blood Cell ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Red Blood Cell Transfusion ◽  
Iron Chelation Therapy

Quality of Life and Economic Impact of Red Blood Cell (RBC) Transfusions on Patients with Myelodysplastic Syndromes (MDS).

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4716-4716 ◽  
Author(s):  
S. Brechignac ◽  
E. Hellstrom-Lindberg ◽  
D. T. Bowen ◽  
T. M. DeWitte ◽  
M. Cazzola ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Blood Cell ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Subcutaneous Administration ◽  
Iron Chelation ◽  
International Prognostic Scoring System ◽  
Blood Collection ◽  
Iron Chelation Therapy

Abstract Background: Supportive care with blood product transfusions is the primary management strategy for the majority of patients with MDS. Approximately 80% of MDS patients are anemic at the time of presentation and more than 40% require regular RBC transfusions at some stage of disease, while platelet transfusions are less often required. Methods: In an effort to systematically study quality of life and economic cost associated with transfusion dependency (especially RBC transfusions), The MDS Foundation has disseminated a practices and treatment survey to its Centers of Excellence and is also accumulating transfusion data. Retrospective and prospective data collected include hematologic parameters defining transfusion need; percentage (%) of MDS patients requiring transfusion; % of transfusion-dependent MDS patients by subtype and International Prognostic Scoring System (IPSS) risk group; per patient frequency of transfusions; % of patients requiring iron chelation therapy. Results: A total of 30 Centers have replied to the survey to date, and responses reveal that a substantial proportion of MDS patients receive multiple RBC transfusions with most of these patients needing chelation therapy with desferoxamine (generally subcutaneous administration, 4-times weekly): Table 1. In addition, detailed data are available from 4 European Centers that have provided transfusion records from randomly selected multiply-transfused MDS patients: 38 patients (median age: 73) received a median of 42 transfusions over the last 24 months (range: 11–207). The average per transfusion costs calculated from estimates provided by the 4 European centers is 436 euros or $ 526 ($1 US dollar = 0.83 euros), where the per transfusion cost includes 2 filtered red blood cell units, blood collection, administrative costs, and staff time, resulting in a median per patient cost over the last 24 months of 11,118 euros (range: 5668–21,800 euros). This does not include the cost of chelation therapy (300 euros/month for desferioxamine SC) and indirect costs (e.g., time spent at transfusion facility, travel time for patient to facility, travel and wait time for private caretaker or family member). Conclusion: Preliminary data analysis from the ongoing retrospective study suggests that the transfusion burden to MDS patients and to society, in terms of quality of life and cost, is much greater than generally appreciated. Updated data of this study will be presented. Table 1: RBC Transfusion-dependent MDS patients Mean % IPSS low risk 39 IPSS intermediate-1 risk 50 IPSS intermediate-2 risk 63 IPSS high risk 79 Iron chelation therapy 28

Improved Survival in Red Blood Cell Transfusion Dependent Patients with Primary Myelofibrosis (PMF) Receiving Iron Chelation Therapy.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 1748-1748 ◽  
Author(s):  
Heather Leitch ◽  
Hatoon Ezzat ◽  
Meaghan D Rollins ◽  
Trisha A Goodman ◽  
Chantal S Leger ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Blood Cell ◽  
Red Blood Cell ◽  
Clinical Evidence ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Monocyte Count ◽  
Iron Chelation Therapy ◽  
Wbc Count ◽  
Rbc Transfusion

Abstract Patients (pts) with PMF and iron overload (IOL) may receive iron chelation therapy (ICT), although there are no data demonstrating that this improves clinical outcome. Red blood cell (RBC) transfusion dependent (TD) pts with thalassemia receiving ICT have improved survival and decreased end-organ toxicities and RBC-TD pts with myelodysplastic syndrome (MDS) receiving ICT have improved survival. We performed a review of 41 pts seen from January 1987 to April 2007 with a bone marrow biopsy confirmed diagnosis (Dx) of PMF. Clinical data were collected from the practice database, the Provincial Home Hemosiderosis Program of British Columbia database, and by chart review. Pts receiving ICT were treated with desferrioxamine (DFO) 0.5–3g by subcutaneous infusion over 12 hours, 35 days per week or with deferasirox (DFX) 20mg/kg/day orally, dose adjusted to response and pt tolerance. 29 were male and 12 female. Median age at PMF Dx was 64 (43–86) years (y) and 24 pts were >60y. White blood cell (WBC) count at Dx was <4 or >30x109/L in 8, hemoglobin (Hgb) <100 G/L in 5, platelet count <100x109/L in 5, monocyte count >1x109/L in 7. Karyotype analysis was: normal, n=16; del(6)(q25), n=1; tri(14), str12p, n=1; complex, n=1. Lille, Strasser and Mayo prognostic scores were: low risk, n=15, 8, 11; intermediate, n=15, 19, 9; high, n=5, 11, 5 respectively. Primary PMF treatment was: supportive care, n=23; hydroxyurea, n=10; immunomodulatory, n=4; splenectomy, n=2. Clinical evidence of IOL was documented in 21 pts; number of RBC units (NRBCU) received, n=18; ferritin >2000ug/l, n=6 (and ferritin >1000ug/l, n=1); CHF, n=5; liver disease, n=3; endocrine, n=3. 16 pts were RBC transfusion–independent (TI) and 25 were TD; of these 10 received ICT. Median duration of ICT was 18.3 (0.1–117) months (mo) and reasons for initiating ICT were: NRBCU received, n=9; elevated ferritin, n=6; clinical evidence of IOL, n=3. Five pts received DFO, 4 DFX, and 1 DFO followed by DFX. In ICT pts, initial/Pre-ICT ferritin levels were significantly higher than in TD-NO ICT pts at a median of 2318 (range 263–8400) and 527 (120–934) mg/L respectively (p=0.05) and decreased significantly in TD-ICT pts at most recent follow-up to 1571 (1005–3211) mg/L (p=0.01). Causes of death were: TI patients, no deaths; TD-NO ICT patients, 11 deaths (73%): probably PMF-related, n=9; progression to PMF-blast phase (BP), n=3; sepsis, n=3; cardiac, n=2; bleeding, n=1, unknown, n=2 ; TD-ICT patients, 2 deaths (20%); PMF-BP, n=1; bleeding, n=1. Kaplan- Meier analysis showed a median overall survival (OS) for all pts of 126.5 (14.4–293.2) mo. In a univariate analysis of TD pts, factors significant for OS (and 5y OS) were: WBC count at Dx (4.0–30x109/L, 69%; <4.0 or >30x109/L, 0%; p=0.002); monocyte count at Dx (<1.0x109/L, 74%; >1.0x109/L, 0%; p=0.0001); Mayo prognostic score (low, 67%; intermediate, 50%; high, 0%; p=0.05); NRBCU transfused (<20U, 30%; 21–50U, 27%; >50U, 12%; p=0.02) and receiving ICT (ICT, 89%; NO-ICT, 34%; p=0.003). In Cox regression analysis of TD pts, factors significant for OS were: NRBCU (p=0.001) and ICT (p=0.0001). For TI, TD-NO ICT and TD-ICT pts respectively the median OS was not reached (NR) at 200 mo, 58 mo and NR at 293 mo respectively (p=0.01 for TD-NO ICT vs TI and NS for TD-ICT vs. TI). The hazard ratio (HR) for pts receiving >20 RBCU was increased at 7.6 (95% CI 1.2–49.3) and the HR for pts receiving ICT was improved at 0.15 (0.03–0.77). In conclusion, 61% of PMF pts developed RBC-TD and had inferior OS, however TD pts receiving ICT had superior OS compared to TD pts not receiving ICT and the OS of TD-ICT pts was comparable to the OS of TI patients, suggesting a benefit to ICT. These are to our knowledge the first data documenting improved clinical outcome in pts with PMF receiving ICT. Prospective studies of IOL and the impact of ICT in pts with PMF are warranted.

scholarly journals Iron chelation therapy with deferiprone improves oxidative status and red blood cell quality and reduces redox-active iron in β-thalassemia/hemoglobin E patients

2022 ◽  
Vol 145 ◽  
pp. 112381
Author(s):  
Noppawan Phumala Morales ◽  
Supot Rodrat ◽  
Pannaree Piromkraipak ◽  
Paveena Yamanont ◽  
Kittiphong Paiboonsukwong ◽  
...  
Keyword(s):  
Blood Cell ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Oxidative Status ◽  
Iron Chelation Therapy ◽  
Hemoglobin E ◽  
Active Iron ◽  
Redox Active

scholarly journals Red blood cell transfusion-dependency implies a poor survival in primary myelofibrosis irrespective of IPSS and DIPSS

Haematologica ◽  
2010 ◽  
Vol 96 (1) ◽  
pp. 167-170 ◽  
Author(s):  
C. Elena ◽  
F. Passamonti ◽  
E. Rumi ◽  
L. Malcovati ◽  
L. Arcaini ◽  
...  
Keyword(s):  
Blood Cell ◽  
Red Blood Cell ◽  
Primary Myelofibrosis ◽  
Red Blood Cell Transfusion ◽  
Poor Survival ◽  
Transfusion Dependency

scholarly journals Imaging of Body Iron Stores in Transfusion-Dependent Patients By Liver Dual- Energy CT

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2677-2677
Author(s):  
Hironori Kobayashi ◽  
Norihiko Yoshimura ◽  
Takashi Ushiki ◽  
Yasuhiko Shibasaki ◽  
Masato Moriyama ◽  
...  
Keyword(s):  
Blood Cell ◽  
Serum Ferritin ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Deposition ◽  
Liver Iron ◽  
Body Iron ◽  
Iron Stores ◽  
Red Blood Cell Transfusions

Abstract [Background] Chronic red blood cell transfusions, leading to iron overload, cause hepatic, cardiac, and endocrine dysfunction. It is very important to monitor body iron stores and to start optimal iron chelation therapy. Serum ferritin, which is widely used as a surrogate marker of body iron stores, elevate under inflammation or liver injury. Therefore, reliable techniques to evaluate body iron stores are needed. The liver iron concentration (LIC) is thought to be an indicator of total body iron stores and measurement of the T2* value by MRI has been a standard noninvasive technique to evaluate LIC. It should be worthwhile using CT, which is lower cost and widely applied in clinical setting. Dual-energy CT (DECT) is a technique to obtain additional information regarding tissue composition compared with what single-energy CT can provide. This technique is based on the fact that substances show different densities by two different energies. However, the role of DECT in monitoring LIC remains to be clarified. We examined whether a DECT could be a new technique for the measurement of LIC. [Patients and Methods] Eight transfusion-dependent patients underwent DECT. Patient 1 was a 54-year-old male with MDS (RCMD-RS). He received 66 U red blood cell transfusions in our hospital, and depended on transfusion in another hospital, but the total doses were not available. Patient 2 was a 37-year-old male with AML in 2nd relapse. His total red blood cell transfusions were 54 U. Patient 3 was a 66-year-old female with AML with MRC in 1st CR. She received 37 U red blood cell transfusions in our hospital, and depended on transfusion in another hospital, but total doses were not available. Patient 4 was a 47-year-old female who had received renal transplantation for chronic renal failure. She received 12 U red blood cell transfusions in our hospital, and had a long history of transfusion dependence in another hospital, but total doses were not available. Patient 5 was a 57-year-old male with MDS (RCMD). His total red blood cell transfusions were 148 U, and he received iron chelation therapy. Patient 6 was a 65-year-old male with AML with MRC. His total red blood cell transfusions were 82 U, and he received iron chelation therapy. Patient 7 was a 47-year-old male with AML in 3rd CR. He received 28 U red blood cell transfusions in our hospital, and depended on transfusion in another hospital, but total doses were not available. Patient 8 was a 52-year-old female with AA. Her total blood cell transfusions were 92 U. [Results] All patients were examined for serum ferritin and patients 1, 3, 4, 6, 7, and 8 also underwent liver MRI. Serum ferritin levels of patients 1, 3, 4, 6, 7, and 8 were 961, 2168, 7875, 795, 1921, and 5104 ng/ml, respectively. These patients showed hypointensity on MRI T2*-weighted images, and also showed liver iron deposition by DECT. Serum ferritin of patient 5 was 4042 ng/ml, and he showed liver iron deposition by DECT. Serum ferritin of patient 2 was 6113 ng/ml, and he did not show liver iron deposition by DECT. [Conclusion] Our results suggest that liver DECT could visualize liver iron deposition of transfusion-dependent patients and could be a new technique for the measurement of LIC instead of MRI. Disclosures No relevant conflicts of interest to declare.

Erythropoietin Therapy Does Not Benefit Transfusion-Dependent Primary Myelofibrosis Patients and Treatment Response Is Infrequent with a Baseline Hemoglobin Level ≥ 10 g/dL.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 3555-3555
Author(s):  
Jocelin Huang ◽  
Terra L. Lasho ◽  
Chin Yang Li ◽  
Animesh D. Pardanani ◽  
Ruben A. Mesa ◽  
...  
Keyword(s):  
Blood Cell ◽  
Treatment Response ◽  
Red Blood Cell ◽  
Primary Myelofibrosis ◽  
Hemoglobin Level ◽  
World Health ◽  
Red Blood Cell Transfusion ◽  
Treatment History ◽  
Baseline Hemoglobin

Abstract BACKGROUND: The potential risks of tumor growth promotion and thromboembolism associated with erythropoietin (Epo) therapy warrant cautious use of erythropoiesis stimulating agents (ESA). Recent data suggest a surprising and significant association between ESA use and increased incidence of leukemic transformation in primary myelofibrosis (PMF) (data from an analysis separately submitted for American Society of Hematology meeting, 2007). It is prudent, therefore, to avoid unnecessary ESA exposure in PMF, especially in patients with low likelihood of treatment response. METHODS: Diagnosis of PMF was based on World Health Organization criteria. Study inclusion required availability of treatment history with ESA and adequate follow-up. We excluded patients who were receiving hemoglobin-improving agents concomitantly with an ESA, such as prednisone, androgens, danazol, or thalidomide. Transfusion dependency was defined by a history of minimum 2 units of red blood cell transfusions in a 1-month period for a hemoglobin < 8.5 g/dL, not associated with bleeding. An adequate treatment response was defined as a minimum 2.0 g/dL increase in hemoglobin level or becoming transfusion-independent over a minimum 1-month period. RESULTS: A master database of PMF patients seen at the Mayo Clinic from 1976 through 2006 was queried to identify 43 patients (median age 67 years; 35% females) treated with ESAs and in whom adequate follow-up was available to determine response. Prior to ESA treatment, median hemoglobin was 9.2 g/dL (range 7.0–11.9), and 16 (37%) were red blood cell transfusion-dependent. In 25 patients, pre-treatment serum Epo level was available (median 34 mU/mL; range 2–323). Among the 43 study patients, 16 (37%) were previously treated with other PMF-specific therapies, and 18 (42%) were receiving cytoreductive treatment concurrent with an ESA. Overall, 23% of patients (10 of 43) responded to treatment with ESA. The response rate was 0% (0 of 16) in the transfusion-dependent group compared to 37% (10 of 27) in the transfusion-independent group (p=0.007). Among the transfusion-independent patients, the proportion of ESA responders was significantly higher in the presence of a baseline hemoglobin level < 10 g/dL prior to treatment (p=0.009). ESA response was not correlated with baseline serum Epo level in the context of either all 43 study patients (p=0.68) or the 27 transfusion-independent patients (p=0.39). ESA response was not affected by PMF-specific treatment history (p=0.46), use of concurrent cytoreductive therapy (p=0.28), cytogenetic findings (p=0.62), JAK2V617F presence (p=0.69) or its allele burden at time of initial PMF diagnosis (p=0.16). CONCLUSIONS: Clear benefit of ESAs in PMF is limited to red blood cell transfusion-independent patients and those with baseline hemoglobin levels less than 10 g/dL.

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