Publications Aplasie médullaire/Hémoglobinurie paroxystique nocturne/Anémie de Fanconi

Hematopoietic stem cell transplantation for patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a retrospective study of 21 patients from SFGM-TC centers.

The effect of age in patients with acquired aplastic anaemia treated with immunosuppressive therapy: comparison of Adolescents and Young Adults with children and older adults. Nationwide survey in France on the use of romiplostim in patients with refractory severe aplastic anemia.

Aplastic anemia in the elderly: a nationwide survey on behalf of the French Reference Center for Aplastic Anemia.

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study.

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study.

Familial predisposition to TP53/complex karyotype MDS and leukemia in DNA repair-deficient xeroderma pigmentosum.

Regulation of globin-heme balance in Diamond-Blackfan anemia by HSP70/GATA1.

The effect of age in patients with acquired aplastic anaemia treated with immunosuppressive therapy: comparison of Adolescents and Young Adults with children and older adults.

Mutations in the SRP54 gene cause severe congenital neutropenia as well as Shwachman-Diamond-like syndrome.

Nationwide survey in France on the use of romiplostim in patients with refractory severe aplasticanemia.

Aplastic anemia in the elderly: a nationwide survey on behalf of the French Reference Center for Aplastic Anemia.

Aplastic anemia in the elderly: a nationwide survey on behalf of the French Reference Center for Aplastic Anemia.

Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia: a nationwide phase 2 study.

Nationwide survey on the use of horse antithymocyte globulins (ATGAM) in patients with acquired aplastic anemia: A report on behalf of the French Reference Center for Aplastic Anemia.

Transplant outcome for patients with acquired aplastic anemia over the age of 40: has the outcomeimproved? Giammarco S et al. Blood. 2018 Mar 16.

First line treatment of aplastic anemia with thymoglobuline in Europe and Asia: Outcome of 955 patients treated 2001-2012. Bacigalupo A et al. Am J Hematol. 2018 May.

Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia. Lengline E et al. Haematologica. 2018 Feb.

Nationwide survey on the use of horse antithymocyte globulins (ATGAM) in patients with acquired aplastic anemia: A report on behalf of the French Reference Center for Aplastic Anemia. Peffault de Latour R et al. Am J Hematol. 2018 May.

A landscape of germ line mutations in a cohort of inherited bone marrow failure patients. Bluteau O et al. Blood. 2018 Feb 15.

Transplant results in adults with Fanconi anaemia. Bierings M et al. Br J Haematol. 2018 Jan.

Hematopoietic stem cell transplantation for patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a retrospective study of 21 patients from SFGM-TC centers. Vallet N, et al. Haematologica. 2018 Mar.

Association of aplastic anaemia and lymphoma: a report from the severe aplastic anaemia working party of the European Society of Blood and Bone Marrow Transplantation. Rovó A et al. Br J Haematol. 2017 Dec 19.

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. Townsley DM et al. N Engl J Med. 2017 Apr 20;376(16):1540-1550.​

Transplantation for bone marrow failure: current issues. Peffault de Latour R. Hematology Am Soc Hematol Educ Program. 2016

Avancée majeure dans le traitement de patientes enceintes atteintes d’Hémoglobinurie Paroxystique Nocturne (HPN): Eculizumab in Pregnant Patients with Paroxysmal Nocturnal Hemoglobinuria, N Engl J Med 2015; 373:1032-1039September 10, 2015

Paroxysmal nocturnal hemoglobinuria (PNH) and T cell large granular lymphocyte (LGL) leukemia–an unusual association: another cause of cytopenia in PNH. Boyer T et al. Annals of Hematology, October 2015, Volume 94, Issue 10, pp 1759-1760.
 
Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant. Dufour C et al. British Journal of Haematology, 14 FEB 2015.
 
HLA-matched related-donor HSCT in Fanconi anemia patients conditioned with cyclophosphamide and fludarabine. Benajiba L et al. Blood 2015 125: 417-418.

Meppiel E et al., Cerebral venous thrombosis in paroxysmal nocturnal hemoglobinuria: a series of 15 cases and review of the literature. Medicine, 2015 Jan; 94(1): e362.

Assessing complement blockade in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumab. Peffault de Latour et al. Blood 2015; 125(5):775-783.
 
Paroxysmal nocturnal hemoglobinuria and hematopoietic stem cell transplantation: Better results in 2015? Sicre de Fontbrune et al. Leukemia & Lymphoma Volume 57, Issue 4, 2016.
 
Second allogeneic stem cell transplant for aplastic anaemia: a retrospective study by the Severe Aplastic Anaemia Working Party of the European Society for Blood and Marrow Transplantation. Cesaro S et al. British Journal of Haematology Volume 171, Issue 4, pages 606–614, November 2015.
 
Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Dufour C et al. British Journal of Haematology Volume 171, Issue 4, pages 585–594, November 2015.
 
[Paroxysmal nocturnal hemoglobinuria: An unknown cause of thrombosis?]. Doutrelon C et al. Journal des Maladies Vasculaires, Vol 40 – N° 6 P. 384-390 – décembre 2015.

[Paroxysmal nocturnal hemoglobinuria]. Peffault de Latour R, Socié G.

Hémoglobinurie Paroxystique Nocturne Socié G., R. Peffault de Latour, Mary J.Y. (EMC 2008).

Eculizumab in paroxysmal nocturnal hemoglobinuria. Socié G, Varoqueaux N, Peffault de Latour R. Med Sci (Paris) 2009; 25(12), 1126-9.

[Dyskeratosis congenita: an update]. Mialou V, Leblanc T, Peffault de Latour R, Dalle JH, Socié G. Arch Pediatr.2013 Mar;20(3), 299-306.