1,720,984 research outputs found
Managing unresponsiveness or intolerance to deferasirox therapy: a tale of two doses
No full textEvaluation of: Chang HH, Lu MY, Liao YM et al. Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia. Pediatr. Blood Cancer 56(3), 420-424 (2011). Chronic transfusional iron overload leads to significant morbidity and mortality in patients with β-thalassemia major. The once-daily oral iron chelator, deferasirox, opened new horizons for the management of transfusional siderosis. A large body of evidence is now available regarding its efficacy and safety. Nevertheless, some patients remain unresponsive or intolerant to the adverse events of the drug. Chang et al. evaluated the benefit of twice-daily dosing in this setting. The authors concluded that twice-daily deferasirox improves responsiveness and tolerability. Even though the study included only a small number of patients, it offers promising insights that should be interpreted with caution. © 2011 Expert Reviews Ltd.Angelucci E, 2000, NEW ENGL J MED, V343, P327, DOI 10.1056-NEJM200008033430503; Berdoukas V, 2010, ANN HEMATOL, V89, P1177, DOI 10.1007-s00277-010-0933-1; Cappellini MD, 2007, CLIN THER, V29, P909, DOI 10.1016-j.clinthera.2007.05.007; Cappellini MD, 2010, HAEMATOL-HEMATOL J, V95, P557, DOI 10.3324-haematol.2009.014696; Cappellini MD, 2008, EXPERT OPIN PHARMACO, V9, P2391, DOI [10.1517-14656566.9.13.2391 , 10.1517-14656560802335333]; CAPPELLINI MD, 2009, BLOOD, V114, P4063; Cappellini MD, 2006, BLOOD, V107, P3455, DOI 10.1182-blood-2005-08-3430; Chang HH, 2011, PEDIATR BLOOD CANCER, V56, P420, DOI 10.1002-pbc.22826; Chirnomas D, 2009, BLOOD, V114, P4009, DOI 10.1182-blood-2009-05-222729; Farmaki K, 2011, BLOOD CELL MOL DIS, V47, P33, DOI 10.1016-j.bcmd.2011.03.007; Galanello R, 2010, ANN NY ACAD SCI, V1202, P79, DOI 10.1111-j.1749-6632.2010.05591.x; Pennell DJ, 2011, HAEMATOL-HEMATOL J, V96, P48, DOI 10.3324-haematol.2010.031468; Ponticelli C, 2010, BLOOD REV, V24, P239, DOI 10.1016-j.blre.2010.08.004; Rienhoff HY, 2011, HAEMATOL-HEMATOL J, V96, P521, DOI 10.3324-haematol.2010.034405; Taher A, 2010, ACTA HAEMATOL-BASEL, V123, P220, DOI 10.1159-000313447; Taher A, 2009, BRIT J HAEMATOL, V147, P752, DOI 10.1111-j.1365-2141.2009.07908.x; Telfer PT, 2000, BRIT J HAEMATOL, V110, P971, DOI 10.1046-j.1365-2141.2000.02298.x; Trachtenberg F, 2011, AM J HEMATOL, V86, P433, DOI 10.1002-ajh.21993; Vichinsky E, 2008, AM J HEMATOL, V83, P398, DOI 10.1002-ajh.21119; Wood JC, 2008, BLOOD REV, V22, pS14, DOI 10.1016-S0268-960X(08)70004-30
Mechanisms of renal disease in β-thalassemia
No full textAlthough advances in the care of patients with β-thalassemia translate into better patient survival, this success has allowed previously unrecognized complications to emerge, including several renal abnormalities. Clinical studies continue to show that mild tubular dysfunction and abnormalities in GFR are common in patients with β-thalassemia. Chronic anemia and iron overload are believed to lie behind these abnormalities. Nonprogressive increases in levels of serum creatinine have also been observed after exposure to some iron chelators. Longitudinal studies are needed to understand the true burden of renal dysfunction in patients with β-thalassemia. Copyright © 2012 by the American Society of Nephrology.ALFREY AC, 1994, AM J KIDNEY DIS, V23, P183; Cappellini MD, 2011, BLOOD, V118, P884, DOI 10.1182-blood-2010-11-316646; Cappellini MD, 2006, BLOOD, V107, P3455, DOI 10.1182-blood-2005-08-3430; Clajus C, 2008, NEPHROL DIAL TRANSPL, V23, P1061, DOI 10.1093-ndt-gfm824; DAVIS LE, 1991, AM J PHYSIOL, V261, pR1542; Fibach E, 2008, CURR MOL MED, V8, P609, DOI 10.2174-156652408786241384; KAISSLING B, 1993, AM J PHYSIOL, V264, pF608; Kassab-Chekir A, 2003, CLIN CHIM ACTA, V338, P79, DOI 10.1016-j.cccn.2003.07.010; King SM, 2008, EXP BIOL MED, V233, P701, DOI 10.3181-0708-RM-233; Kokoszko A, 2008, EXP TOXICOL PATHOL, V60, P453, DOI 10.1016-j.etp.2008.04.012; Koliakos G, 2003, CLIN LAB HAEMATOL, V25, P105, DOI 10.1046-j.1365-2257.2003.00507.x; LAFFERTY HM, 1991, AM J KIDNEY DIS, V17, P2; LANDING B H, 1989, Pediatric Pathology, V9, P479; Michelakakis H, 1997, EUR J PEDIATR, V156, P602, DOI 10.1007-s004310050673; Nagababu E, 2008, FREE RADICAL RES, V42, P824, DOI 10.1080-10715760802459879; Nangaku M, 2006, J AM SOC NEPHROL, V17, P17, DOI 10.1681-ASN.2005070757; Papassotiriou I, 2010, BLOOD CELL MOL DIS, V44, P152, DOI 10.1016-j.bcmd.2010.01.001; Ponticelli C, 2010, BLOOD REV, V24, P239, DOI 10.1016-j.blre.2010.08.004; Quinn CT, 2011, BRIT J HAEMATOL, V153, P111, DOI 10.1111-j.1365-2141.2010.08477.x; Rienhoff HY, 2011, HAEMATOL-HEMATOL J, V96, P521, DOI 10.3324-haematol.2010.034405; Sadeghi-Bojd S, 2008, SINGAP MED J, V49, P410; Steinberg MH, 2009, DISORDERS OF HEMOGLOBIN: GENETICS, PATHOPHYSIOLOGY, AND CLINICAL MANAGEMENT, 2ND EDITION, P1, DOI 10.1017-CBO9780511596582; Sumboonnanonda A, 1998, PEDIATR NEPHROL, V12, P280; Sumboonnanonda A, 2009, PEDIATR NEPHROL, V24, P183, DOI 10.1007-s00467-008-0949-0; Taher A, 2009, BRIT J HAEMATOL, V147, P752, DOI 10.1111-j.1365-2141.2009.07908.x; Taher AT, 2011, BRIT J HAEMATOL, V152, P512, DOI 10.1111-j.1365-2141.2010.08486.x; Vichinsky E, 2007, BRIT J HAEMATOL, V136, P501, DOI 10.1111-j.1365-2141.2006.06455.x; Vichinsky E, 2008, AM J HEMATOL, V83, P398, DOI 10.1002-ajh.21119; Weatherall DJ, 2010, BLOOD, V115, P4331, DOI 10.1182-blood-2010-01-251348; Zager RA, 2004, KIDNEY INT, V65, P2123, DOI 10.1111-j.1523-1755.2004.00638.x; Zager RA, 2004, KIDNEY INT, V66, P144, DOI 10.1111-j.1523-1755.2004.00716.x; Zhou XJ, 2000, LAB INVEST, V80, P1905, DOI 10.1038-labinvest.378020075
Clinical Images: Severe photosensitive skin reaction secondary to an herbal treatment in a patient with systemic lupus erythematosus
No full text[No abstract available]0
Iron chelation therapy for transfusional iron overload: A swift evolution
No full textChronic transfusional iron overload leads to significant morbidity and mortality. While deferoxamine (DFO) is an effective iron chelator with over four decades of experience, it requires tedious subcutaneous infusions that reflect negatively on patient compliance. The novel oral iron chelators deferiprone (L1) and deferasirox (DFRA) opened new horizons for the management of transfusional siderosis. A large body of evidence is now available regarding their efficacy and safety in various populations and settings. Nevertheless, experience with both drugs witnessed some drawbacks, and the search for an ideal and cost-effective iron chelator continues. © 2011 Informa Healthcare USA, Inc.Anderson LJ, 2002, LANCET, V360, P516, DOI 10.1016-S0140-6736(02)09740-4; Borgna-Pignatti C, 2004, HAEMATOLOGICA, V89, P1187; Borgna-Pignatti C, 2006, BLOOD, V107, P3733, DOI 10.1182-blood-2005-07-2933; Brittenham GM, 2011, NEW ENGL J MED, V364, P146, DOI 10.1056-NEJMct1004810; Cappellini Maria D, 2009, Hemoglobin, V33 Suppl 1, pS58, DOI 10.3109-03630260903346924; Cappellini MD, 2007, CLIN THER, V29, P909, DOI 10.1016-j.clinthera.2007.05.007; Cappellini MD, 2010, HAEMATOL-HEMATOL J, V95, P557, DOI 10.3324-haematol.2009.014696; Cappellini MD, 2011, BLOOD, V118, P884, DOI 10.1182-blood-2010-11-316646; Cappellini MD, 2009, ACTA HAEMATOL-BASEL, V122, P165, DOI 10.1159-000243801; Cappellini MD, 2008, EXPERT OPIN PHARMACO, V9, P2391, DOI [10.1517-14656566.9.13.2391 , 10.1517-14656560802335333]; Cappellini MD, 2006, BLOOD, V107, P3455, DOI 10.1182-blood-2005-08-3430; Delea TE, 2007, TRANSFUSION, V47, P1919, DOI 10.1111-j.1537-2995.2007.01416.x; Gabutti V, 1996, ACTA HAEMATOL-BASEL, V95, P26; Galanello R, 2010, ANN NY ACAD SCI, V1202, P79, DOI 10.1111-j.1749-6632.2010.05591.x; KONTOGHIORGHES GJ, 1987, LANCET, V1, P1294; Lai ME, 2010, BLOOD CELL MOL DIS, V45, P136, DOI 10.1016-j.bcmd.2010.05.005; Maggio A, 2009, BLOOD CELL MOL DIS, V42, P247, DOI 10.1016-j.bcmd.2009.01.002; Modell B, 2008, J CARDIOVASC MAGN R, V10, DOI 10.1186-1532-429X-10-42; Musallam K, 2008, PEDIATRICS, V121, pE1426, DOI 10.1542-peds.2007-1944; Musallam Khaled M, 2011, Haematologica, V96, pe5, DOI 10.3324-haematol.2010.036061; OLIVIERI NF, 1994, NEW ENGL J MED, V331, P574, DOI 10.1056-NEJM199409013310903; Pennell DJ, 2006, BLOOD, V107, P3738, DOI 10.1182-blood-2005-07-2948; Pennell DJ, 2010, BLOOD, V115, P2364, DOI 10.1182-blood-2009-04-217455; Pennell DJ, 2011, HAEMATOL-HEMATOL J, V96, P48, DOI 10.3324-haematol.2010.031468; PENNELL DJ, 2010, BLOOD, V116, P4276; Pepe A, 2011, HAEMATOL-HEMATOL J, V96, P41, DOI 10.3324-haematol.2009.019042; Piga A, 2003, HAEMATOLOGICA, V88, P489; Piga A, 2010, ANN NY ACAD SCI, V1202, P75, DOI 10.1111-j.1749-6632.2010.05586.x; Ponticelli C, 2010, BLOOD REV, V24, P239, DOI 10.1016-j.blre.2010.08.004; Porter JB, 2008, BLOOD, V112, P5423; Porter J, 2008, EUR J HAEMATOL, V80, P168, DOI 10.1111-j.1600-0609.2007.00985.x; Rienhoff HY, 2011, HAEMATOL-HEMATOL J, V96, P521, DOI 10.3324-haematol.2010.034405; Taher A, 2010, ACTA HAEMATOL-BASEL, V123, P220, DOI 10.1159-000313447; Taher A, 2009, EUR J HAEMATOL, V82, P458, DOI 10.1111-j.1600-0609.2009.01228.x; Taher A, 2009, BRIT J HAEMATOL, V147, P752, DOI 10.1111-j.1365-2141.2009.07908.x; Taher Ali T, 2009, Hemoglobin, V33 Suppl 1, pS46, DOI 10.3109-03630260903346676; Tanner MA, 2007, CIRCULATION, V115, P1876, DOI 10.1161-CIRCULATIONAHA.106.648790; Telfer PT, 2009, HAEMATOL-HEMATOL J, V94, P1777, DOI 10.3324-haematol.2009.009118; Vichinsky E, 2007, BRIT J HAEMATOL, V136, P501, DOI 10.1111-j.1365-2141.2006.06455.x; Wonke B, 1998, BRIT J HAEMATOL, V103, P361; Wood JC, 2010, BLOOD, V116, P537, DOI 10.1182-blood-2009-11-25030856
Thrombosis in thalassemia: Why are we so concerned?
No full textAlthough life expectancy of thalassemia patients has markedly improved over the last few decades, patients still suffer from many complications of this congenital disease. The presence of a high incidence of thromboembolic events (TEE), mainly in β-thalassemia intermedia (β-TI), has led to the identification of a hypercoagulable state in these patients. In this review, the molecular and cellular mechanisms leading to hypercoagulability in thalassemia are highlighted, with a special focus on thalassemia intermedia being the group with the highest incidence of thrombotic events as compared to other types of thalassemia. Clinical experience and available clues on optimal management are also discussed. © 2011 Informa Healthcare USA, Inc.Atichartakarn V, 2002, BRIT J HAEMATOL, V118, P893, DOI 10.1046-j.1365-2141.2002.03711.x; Atichartakarn V, 2003, INT J HEMATOL, V77, P299, DOI 10.1007-BF02983790; Yashar Vered Borenstain-Ben, 1993, American Journal of Hematology, V44, P63, DOI 10.1002-ajh.2830440114; Pignatti CB, 1998, ACTA HAEMATOL-BASEL, V99, P76; Borgna-Pignatti C, 2004, HAEMATOLOGICA, V89, P1187; BUTTHEP P, 1995, THROMB HAEMOSTASIS, V74, P1045; Butthep P, 1997, SE ASIAN J TROP M S3, V28, P141; Cadili A, 2008, AM J MED, V121, P371, DOI 10.1016-j.amjmed.2008.02.014; Cappellini MD, 2010, ANN N Y ACAD SCI, V1202, P231; Cappellini MD, 2000, BRIT J HAEMATOL, V111, P467, DOI 10.1046-j.1365-2141.2000.02376.x; Cappellini MD, 2005, ANN N Y ACAD SCI, V1054, P317; Crary SE, 2009, BLOOD, V114, P2861, DOI 10.1182-blood-2009-04-210112; DELPRINCIPE D, 1993, BRIT J HAEMATOL, V84, P111; ELDOR A, 1991, BLOOD, V77, P1749; ELDOR A, 1989, AM J HEMATOL, V32, P94, DOI 10.1002-ajh.2830320204; Habib A, 2008, HAEMATOL-HEMATOL J, V93, P941, DOI 10.3324-haematol.12460; Helley D, 1996, THROMB HAEMOSTASIS, V76, P322; HERSHKO C, 1978, BRIT J HAEMATOL, V40, P255, DOI 10.1111-j.1365-2141.1978.tb03662.x; Hovav T, 1999, BRIT J HAEMATOL, V106, P178; Iolascon A, 2001, HAEMATOLOGICA, V86, P1112; Karimi M, 2010, THROMB HAEMOSTASIS, V103, P989, DOI 10.1160-TH09-09-0661; Kuypers FA, 2004, CELL MOL BIOL, V50, P147; Manfre L, 1999, AM J ROENTGENOL, V173, P1477; Ruf A, 1997, BRIT J HAEMATOL, V98, P51, DOI 10.1046-j.1365-2141.1997.1502965.x; Rund D, 2005, NEW ENGL J MED, V353, P1135, DOI 10.1056-NEJMra050436; Steinberg MH, 2009, DISORDERS OF HEMOGLOBIN: GENETICS, PATHOPHYSIOLOGY, AND CLINICAL MANAGEMENT, 2ND EDITION, P1, DOI 10.1017-CBO9780511596582; Sumiyoshi A, 1992, SE ASIAN J TROP M S2, V23, P29; Taher A, 2006, THROMB HAEMOSTASIS, V96, P488, DOI 10.1160-TH06-05-0267; Taher A, 2009, BRIT J HAEMATOL, V147, P634, DOI 10.1111-j.1365-2141.2009.07848.x; Taher AT, 2008, BLOOD REV, V22, P283, DOI 10.1016-j.blre.2008.04.001; Taher AT, 2010, BRIT J HAEMATOL, V150, P486, DOI 10.1111-j.1365-2141.2010.08220.x; Taher AT, 2011, BRIT J HAEMATOL, V152, P512, DOI 10.1111-j.1365-2141.2010.08486.x; Taher AT, 2010, J THROMB HAEMOST, V8, P2152, DOI 10.1111-j.1538-7836.2010.03940.x; Taher AT, 2010, J THROMB HAEMOST, V8, P54, DOI 10.1111-j.1538-7836.2009.03651.x; Taher AT, 2010, BLOOD, V115, P1886, DOI 10.1182-blood-2009-09-243154; Tavazzi D, 2001, BRIT J HAEMATOL, V112, P48, DOI 10.1046-j.1365-2141.2001.02482.x; Tripodi A, 2009, HAEMATOL-HEMATOL J, V94, P1520, DOI 10.3324-haematol.2009.010546; WINICHAGOON P, 1981, Southeast Asian Journal of Tropical Medicine and Public Health, V12, P556; Zalloua PA, 2003, THROMB HAEMOSTASIS, V89, P767; ZURLO MG, 1989, LANCET, V2, P2716191
Sickle cell disease at the dawn of the molecular era
No full textRecent investigations have identified a syndrome of hemolysis-associated vasculopathy in patients with sickle cell disease (SCD), which features severe hemolytic anemia that leads to scavenging of nitric oxide (NO) and its biochemical precursor arginine. This diminished bioavailability of NO promotes several clinical sequelae, which includes pulmonary hypertension, cutaneous leg ulceration, priapism, and ischemic stroke. Additional correlates of this vasculopathy include activation of endothelial cell adhesion molecules and leukocytes, as well as oxidative stress-related pathways. Some known risk factors for atherosclerosis are also associated with sickle cell vasculopathy, including low levels of apolipoprotein AI and high levels of asymmetric dimethylarginine, an endogenous inhibitor of NO synthase. Our current understanding of the dysregulated vascular biology pathways in SCD provides a basis for new clinical trials investigating promising targeted therapeutics. © 2009 Informa UK, Ltd.
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