Haplo-identical or mismatched unrelated donor hematopoietic cell transplantation for Fanconi anemia: results from the Severe Aplastic Anemia Working Party of the EBMT.

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Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for bone marrow failure or hematopoietic malignant diseases for Fanconi anemia (FA) patients. Although results have improved over the last […]

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Prediction of postpartum hemorrhage in pregnant women with immune thrombocytopenia: development and validation of the MONITOR model in a nationwide multicenter study.

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Globally, postpartum hemorrhage (PPH) is the leading cause of maternal death. Women with immune thrombocytopenia (ITP) are at increased risk of developing PPH. Early identification of PPH helps to prevent […]

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Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data.

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Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine. In 2009 it was identified as a pharmacological chaperone for mutant glucocerebrosidase, albeit in a […]

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BH3 profiling identifies ruxolitinib as a promising partner for venetoclax to treat T-cell prolymphocytic leukemia.

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Conventional therapies for patients with T-cell prolymphocytic leukemia (T-PLL) such as cytotoxic chemotherapy and alemtuzumab have limited efficacy and considerable toxicity. Several novel agent classes have demonstrated preclinical activity in […]

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Light chain proteinuria revealing mu-heavy chain disease: an atypical presentation of Waldenström macroglobulinemia in two cases.

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Not available. View the full article @ Haematologica Get PDF with LibKey Authors: Hélène Vergneault, Djaouida Bengoufa, Aline Frazier-Mironer, Isabelle Brocheriou, Samuel Bitoun, Camille Villesuzanne, Alexis Talbot, Stéphanie Harel, Bertrand […]

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A mutation in the iron-responsive element of ALAS2 is a modifier of disease severity in a patient suffering from CLPX associated erythropoietic protoporphyria.

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Not available. View the full article @ Haematologica Get PDF with LibKey Authors: Sarah Ducamp, Sara Luscieti, Xènia Ferrer-Cortès, Gaël Nicolas, Hana Manceau, Katell Peoc’h, Yvette Y Yien, Caroline Kannengiesser, […]

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Factor XIII cross-links fibrin(ogen) independent of fibrin polymerization in experimental acute liver injury.

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Intravascular fibrin clot formation follows a well-ordered series of reactions catalyzed by thrombin cleavage of fibrinogen leading to fibrin polymerization and cross-linking by factor XIIIa (FXIIIa). Extravascular fibrin(ogen) deposits are […]

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Methotrexate-related central neurotoxicity: clinical characteristics, risk factors and genome-wide association study in children treated for acute lymphoblastic leukemia.

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Symptomatic methotrexate-related central neurotoxicity, ‘MTX neurotoxicity’, is a severe toxicity experienced during acute lymphoblastic leukemia (ALL) therapy with potential long-term neurologic complications. Risk factors and long-term outcomes require further study. […]

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Sequence-specific 2′-O-methoxyethyl antisense oligonucleotides activate human platelets through glycoprotein VI, triggering formation of plateletleukocyte aggregates.

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Antisense oligonucleotides (ASOs) are DNA-based, disease-modifying drugs. Clinical trials with 2′-O-methoxyethyl (2’MOE) ASOs have reported dose- and sequencespecific lowering of platelet counts according to two phenotypes. Phenotype 1 is a […]

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Sulfated non-anticoagulant heparin derivative modifies intracellular hemoglobin, inhibits cell sickling in vitro, and prolongs survival of sickle cell mice under hypoxia.

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Sickle cell disease (SCD) is an autosomal recessive genetic disease caused by a single point mutation, resulting in abnormal sickle hemoglobin (HbS). During hypoxia or dehydration, HbS polymerizes to form […]

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