Flore Sicre-de-Fontbrune

POSTER: MDS-748: Danicopan Add-On Therapy to Ravulizumab or Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis Stratified by Bone Marrow Failure History

Morag Griffin, Jin Seok Kim, Yasutaka Ueda, Flore Sicre-de-Fontbrune et autres

gb, kr, jp, fr, us (code pays fourni par la source)

MDS-748: Danicopan Add-On Therapy to Ravulizumab or Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis Stratified by Bone Marrow Failure History

Morag Griffin, Jin Seok Kim, Yasutaka Ueda, Flore Sicre-de-Fontbrune et autres

gb, kr, jp, fr, us (code pays fourni par la source)

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Austin Kulasekararaj, Morag Griffin, Robert A. Brodsky, Jun‐ichi Nishimura et autres

Abstract Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by intravascular hemolysis, thrombosis, and organ damage. Add-on therapy with danicopan, a first-in-class oral factor D inhibitor, has shown significant improvements to clinical outcomes in adult patients with ravulizumab- or …

gb, us, jp, ca, de, it, es, br, fr, kr (code pays fourni par la source)

Serum ferritin changes in iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria

Antonio M. Risitano, Camilla Frieri, Bing Han, Richard J. Kelly et autres

Abstract Introduction: Patients with paroxysmal nocturnal hemoglobinuria (PNH) often develop iron deficiency (ID) due to chronic intravascular hemolysis (IVH), which can lead to iron loss in the urine and, consequently, require iron supplementation. Conversely, when IVH is controlled by terminal complement component …

nl, it, us, cn, gb, de, br, jp, ch, fr (code pays fourni par la source)

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

Antonio M. Risitano, Camilla Frieri, Bor-Sheng Ko, Lily Wong Lee Lee et autres

Abstract Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis and consequent anemia. Despite treatment with anti-C5 therapy, many patients with PNH remain anemic and may continue to experience symptoms of PNH and extravascular hemolysis. Iptacopan is …

nl, it, tw, my, de, fr, sg, kr, ch, es, us (code pays fourni par la source)

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial)

Britta Hoechsmann, Philippe Schafhausen, Fabian Beier, Michael Heuser et autres

Abstract Introduction: Eltrombopag (EPAG) combined with horse antithymocyte globulin and cyclosporine A (CSA) when given as first-line treatment of patients (pts) with severe aplastic anemia (SAA) improves rate, rapidity, and strength of hematologic response. No consensus exists on the optimal treatment for …

de, us, ch, fr (code pays fourni par la source)

Thymoglobuline and grafalon show comparable transplant outcomes in patients with aplastic anemia undergoing allogeneic stem cell transplantation: A multicenter EBMT saawp study of 1603 patients

Fabian Beier, Edgar Jost, Dirk-Jan Eikema, Joe Tuffnell et autres

Abstract Introduction Rabbit anti-thymocyte globulin (ATG) is a key component of conditioning regimens for patients with aplastic anemia (AA) undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Thymoglobuline (Thymo) is widely regarded as the standard formulation due to its extensive clinical adoption and …

de, nl, Algérie, us, fr, ir, sa, jo, dk, pt, lb, no, il, cz, gb, it (code pays fourni par la source)

Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

Fabiana Cacace, Filomena Pierri, Francesca Fioredda, Laura Van Hussen-Daenen et autres

Abstract Introduction Dyskeratosis congenita (DKC) is a rare telomere disorder causing multi-organ dysfunction and bone marrow failure, with allogeneic hematopoietic cell transplantation (HCT) being the only curative option for hematologic complications. We previously reported 3- and 5-years overall survival (OS) rates of …

it, nl, ru, fr, gb, au, sa, es, il, ch, pl (code pays fourni par la source)

Deep immune signature of immune-mediated aplastic anemia patients shows distinct subsets of regulatory T cells associated with response to treatment: Results from the phase 3, randomized EBMT race clinical trial

Sıla Gerlevik, Giorgio Napolitani, Deniz Ece Kaya, Riley Cook et autres

Abstract Introduction: Immune-mediated aplastic anaemia (AA) is bone marrow failure syndrome, where T-cell mediated destruction of hematopoietic stem and progenitor cells (HSPCs) results in pancytopenia. Overall, two third of patient respond to immune suppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine …

gb, nl, it, fr, ch, jp, us (code pays fourni par la source)

Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dl on anti-C5 therapy: Subgroup analysis of the appulse-pnh Phase 3b trial

Austin Kulasekararaj, Flore Sicre-de-Fontbrune, Anna Gayà, Ilene Weitz et autres

Abstract Introduction: Despite achieving a ‘good response’ with anti-C5 therapy (defined as hemoglobin [Hb] levels 10 to <12 g/dL), some patients with paroxysmal nocturnal hemoglobinuria (PNH) remain anemic and continue to experience symptoms due to extravascular hemolysis. Iptacopan, the first oral selective …

gb, fr, es, us, it, tr, ch, cn (code pays fourni par la source)

The CSMD1 gene is involved in hematopoietic stem cell HSC pruning in patients with acquired immune-mediated aplastic anemia

Antonio M. Risitano, Deniz Ece Kaya, S. Iacobelli, Riley Cook et autres

Abstract Introduction Somatic mutations in Hematopoietic Stem Cells and Progenitors (HSC/HCP) are very frequent in patients with acquired Immune-mediated Aplastic Anemia (IAA) being found in about a third of patients at diagnosis and up to 65-75% after Immuno-Suppressive Treatment (IST). In our …

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Erratum: Sorafenib promotes graft-versus-leukemia activity in mice and humans through IL-15 production in FLT3-ITD-mutant leukemia cells

Nimitha R. Mathew, Francis Baumgartner, Lukas M. Braun, David O’Sullivan et autres