French national reference center for major sickle cell syndromes and other rare pathologies of the red blood cell and erythropoiesis
The reference center for major sickle cell syndromes and other rare pathologies of the red blood cell and erythropoiesis is attached to the general pediatrics and infectious diseases department and the biotherapy department at Necker-Enfants malades hospital in Paris.
It takes care of more than 1,300 patients with red blood cell and erythropoiesis pathologies from birth to adulthood. The center diagnoses anemias and monitors the main red blood cell and erythropoiesis pathologies (notably sickle cell disease, thalassemia and hereditary spherocytosis). Care can be provided in consultation, conventional hospitalization or day hospitalization.
The general pediatrics and infectious diseases department works closely with all departments at Necker-Enfants malades hospital (emergency and intensive care, medical and surgical specialties, radiology), including the immunohematology department (marrow transplant activity). Strong links have also been developed with local medical services, school medicine and patient associations. The biotherapy department carries out exchange transfusions by erythrapheresis and collects stem cells for sickle cell and thalassemia gene therapy. It is also a reference center for the follow-up of adults with sickle cell disease and thalassemia, and for the care of pregnant women with sickle cell disease.
The reference center also provides teaching at the Université Paris Cité, as well as clinical, translational and fundamental research in several laboratories of excellence, including Dr Isabelle André and Pr Marina Cavazzana’s research laboratory, and Pr Olivier Hermine’s « Cellular and molecular mechanisms of hematological disorders and therateutical implications » laboratory at the Institut Imagine.
This reference center is affiliated with the MCGRE rare diseases healthcare network and the EuroBloodnet European reference network (ERN).
Keywords : sickle cell disease, thalassemia, red blood cell and erythropoiesis pathologies, transfusion exchanges, marrow transplantation, gene therapy, innovative treatments, translational research
Recommendations for patients with sickle-cell syndromes during heatwaves :
As hot weather can cause dehydration, leading to vaso-occlusive crises, we recommend that you drink regularly and in greater quantities than usual.
Due to a current epidemic of parvovirus B19 (a virus that is usually benign in the general population but can cause very severe anaemia in patients with red blood cell disease), we recommend that you watch carefully for any signs of anaemia (pallor, fatigue, increased heart rate, increased jaundice or dark urine) and that you seek medical advice immediately if any such signs occur, so that a clinical examination and laboratory tests can be carried out.
It should be noted that in patients with sickle cell disease, this virus can also cause vaso-occlusive attacks with or without fever.
Medical
team
Dr Slimane Allali
MD, PhD
Contact us
Children : Phone +33 (0)1 44 49 57 43 or 44 or 45
Adults : Phone +33 (0)1 44 49 52 74
In case of emergency
- Sickle cell disease
- Thalassemia
- G6PD deficiency
- Pyruvate kinase deficiency
- Hereditary spherocytosis
- Hereditary elliptocytosis
- Blackfan-Diamond anemia
- Iron deficiency and overload
Equipe médicale :
- Dr Slimane Allali, pediatrician – head of the reference center
- Associate Professor Mariane de Montalembert, pediatrician
- Dr Melissa Taylor, pediatrician
- Dr Josephine Brice, pediatrician
- Pr Marina Cavazzana, pediatrician – head of the biotherapy department
- Dr Laure Joseph, adult hematologist
- Dr Fabian Zanchetta, adult chief resident
Equipe paramédicale :
- Cyliane Badzoukoula, coordinating nurse
- Agathe Lanzeray, therapeutic education nurse (RoFSED)
- Laetitia Dessaint et Sylvie Glemarec, pediatric medical secretaries
- Alexandra Bensadi, adult medical secretary
- Floriana Pacelli, pediatric psychologist
- Ariane Krakovitch, adult psychologist
- Marie-Léonie Rousseau et Mélanie Denis-Guerber, pediatric social workers
- Laurence Boismaigre, adult social worker
- Pain management
- Physical and sports activities
- The transition to adult service
- The trip to Africa
Research of early prognostic factors of severity in sickle cell disease
Role of innate immunity in sickle cell disease
Gene therapy for hemoglobin diseases
Sickle cell disease online university degree – Paris-Descartes university
2021
– Effect of hydroxyurea exposure before puberty on sperm parameters in males with sickle cell disease.
L Joseph, C Jean, S Manceau, C Chalas, C Arnaud, A Kamdem, C Pondarré, A Habibi, F Bernaudin, S Allali, M de Montalembert, B Boutonnat-Faucher, J-B Arlet, B Koehl, M Cavazzana, J-A Ribeil, F Lionnet, I Berthaut, V Brousse
Blood, 2021 Feb 11, PMID: 32976551 DOI: 10.1182/blood.2020006270
– Chronic organ injuries in children with sickle cell disease.
Slimane Allali, Melissa Taylor, Joséphine Brice, Mariane de de Montalembert
Haematologica, 2021 Jun 1, PMID: 33626864 PMCID: PMC8168494 DOI: 10.3324/haematol.2020.271353
– Real-Life experience with hydroxyurea in patients with sickle cell disease: Results from the prospective ESCORT-HU cohort study.
Mariane de Montalembert, Ersi Voskaridou, Lena Oevermann, Giovanna Cannas, Anoosha Habibi, Gylna Loko, Laure Joseph, Raffaella Colombatti, Pablo Bartolucci, Valentine Brousse, Frédéric Galactéros, All ESCORT HU Investigators
Am J Hematol, 2021 Oct 1, PMID: 34224583 DOI: 10.1002/ajh.26286
– Young children formula consumption and iron deficiency at 24 months in the general population: A national-level study.
Anne-Sylvia Sacri, Alain Bocquet, Mariane de Montalembert, Serge Hercberg, Laurent Gouya, Béatrice Blondel, Amandine Ganon, Pascale Hebel, Catherine Vincelet, Franck Thollot, Massimiliano Rallo, Piotr Gembara, Corinne Levy, Martin Chalumeau
Clin Nutr, 2021 Jan, PMID: 32507584 DOI: 10.1016/j.clnu.2020.04.041
– CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Haydar Frangoul, David Altshuler, M Domenica Cappellini, Yi-Shan Chen, Jennifer Domm, Brenda K Eustace, Juergen Foell, Josu de la Fuente, Stephan Grupp, Rupert Handgretinger, Tony W Ho, Antonis Kattamis, Andrew Kernytsky, Julie Lekstrom-Himes, Amanda M Li
N Engl J Med, 2021 Jan 21, PMID: 33283989 DOI: 10.1056/NEJMoa2031054
– Distinctive Features of Kawasaki Disease Following SARS-CoV-2 Infection: a Controlled Study in Paris, France.
Julie Toubiana, Jérémie F Cohen, Joséphine Brice, Clément Poirault, Fanny Bajolle, William Curtis, Florence Moulin, Soraya Matczak, Marianne Leruez, Jean-Laurent Casanova, Martin Chalumeau, Melissa Taylor, Slimane Allali
J Clin Immunol, 2021 Apr, PMID: 33394320 PMCID: PMC7780601 DOI: 10.1007/s10875-020-00941-0
– Epidemiology and disease burden of sickle cell disease in France: A descriptive study based on a French nationwide claim database.
Henri Leleu, Jean Benoit Arlet, Anoosha Habibi, Maryse Etienne-Julan, Mehdi Khellaf, Yolande Adjibi, France Pirenne, Marine Pitel, Anna Granghaud, Cynthia Sinniah, Mariane De Montalembert, Frédéric Galacteros
PLoS One, 2021 Jul 9, PMID: 34242255 PMCID: PMC8270152 DOI: 10.1371/journal.pone.0253986
– Hydroxyurea does not affect the spermatogonial pool in prepubertal patients with sickle cell disease.
Anne-Sophie Gille, Corinne Pondarré, Jean-Hugues Dalle, Françoise Bernaudin, Céline Chalas, Mony Fahd, Camille Jean, Harry Lezeau, Lydia Riou, Véronique Drouineaud
Blood, 2021 Feb 11, PMID: 33259585 DOI: 10.1182/blood.2020008146
– Impact of sickle cell disease on patients » daily lives, symptoms reported, and disease management strategies: Results from the international Sickle Cell World Assessment
Ifeyinwa Osunkwo, Biree Andemariam, Caterina P Minniti, Baba P D Inusa, Fuad El Rassi, Beverley Francis-Gibson, Alecia Nero, Cassandra Trimnell, Miguel R Abboud, Jean-Benoît Arlet, Raffaella Colombatti, Mariane de Montalembert et al.
Am J Hematol, 2021 Apr 1, PMID: 33264445 PMCID: PMC8248107 DOI: 10.1002/ajh.26063
2020
– Mortality in children with sickle cell disease in mainland France from 2000 to 2015.
Emilie Desselas, Isabelle Thuret, Florentia Kaguelidou, Malika Benkerrou, Mariane de Montalembert, Marie-Hélène Odièvre, Emmanuelle Lesprit, Eva Rumpler, Arnaud Fontanet, Corinne Pondarre, Valentine Brousse
Haematologica, 2020 Sep 1, PMID: 33054059 PMCID: PMC7556503 DOI: 10.3324/haematol.2019.237602
– Innate immune cells, major protagonists of sickle cell disease pathophysiology.
Slimane Allali, Thiago Trovati Maciel, Olivier Hermine, Mariane de Montalembert
Haematologica, 2020 Jan 31, PMID: 31919091 PMCID: PMC7012475 DOI: 10.3324/haematol.2019.229989
– One-Fifth of Children with Sickle Cell Anemia Show Exercise-Induced Hemoglobin Desaturation: Rate of Perceived Exertion and Role of Blood Rheology.
Valentine Brousse, Corinne Pondarre, Cecile Arnaud, Annie Kamden, Mariane de Montalembert, Benedicte Boutonnat-Faucher, Hélène Bourdeau, Keyne Charlot, David Grévent, Suzanne Verlhac, Lydie da Costa, Philippe Connes
J Clin Med, 2020 Jan 3, PMID: 31947773 PMCID: PMC7019952 DOI: 10.3390/jcm9010133
– Vasomotor hyperresponsiveness in SCD.
Valentine Brousse, Philippe Connes
Blood, 2020 Sep 3, PMID: 32882017 DOI: 10.1182/blood.2020007070
– Mortality in children with sickle cell disease in mainland France from 2000 to 2015.
Emilie Desselas, Isabelle Thuret, Florentia Kaguelidou, Malika Benkerrou, Mariane de Montalembert, Marie-Hélène Odièvre, Emmanuelle Lesprit, Eva Rumpler, Arnaud Fontanet, Corinne Pondarre, Valentine Brousse
Haematologica, 2020 Sep 1, PMID: 33054059 PMCID: PMC7556503 DOI: 10.3324/haematol.2019.237602
– IL-6 levels are dramatically high in the sputum from children with sickle cell disease during acute chest syndrome.
Slimane Allali, Mariane de Montalembert, Rachel Rignault-Bricard, Melissa Taylor, Joséphine Brice, Valentine Brousse, Jean-Marc Talbot, Florence Moulin, Claire Heilbronner, Olivier Hermine, Thiago Trovati Maciel
Blood Adv, 2020 Dec 22, PMID: 33351109 PMCID: PMC7756999 DOI: 10.1182/bloodadvances.2020003519
– Emerging therapies in sickle cell disease.
Amina Nardo-Marino, Valentine Brousse, David Rees
Br J Haematol, 2020 Jul, PMID: 32142156 DOI: 10.1111/bjh.16504
– Patients with sickle cell disease and suspected COVID-19 in a paediatric intensive care unit.
Claire Heilbronner, Laureline Berteloot, Pierre Tremolieres, Laurent Dupic, Laure de Saint Blanquat, Fabrice Lesage, Marie-Hélène Odièvre, Charles de Marcellus, Jacques Fourgeaud, Marianne de Montalembert, Marion Grimaud, Florence Moulin, Sylvain Renolleau, Slimane Allali, Mehdi Oualha
Br J Haematol, 2020 Jul, PMID: 32420608 PMCID: PMC7276717 DOI: 10.1111/bjh.16802
– Appropriate thresholds for accurate screening for β-thalassemias in the newborn period: results from a French center for newborn screening.
Bichr Allaf, Corinne Pondarre, Slimane Allali, Mariane De Montalembert, Cécile Arnaud, Catherine Barrey, Malika Benkerrou, Patricia Benhaim, Philippe Bensaid, Valentine Brousse, Catherine Dollfus, Stéphanie Eyssette-Guerreau, Frédéric Galacteros, Vincent Gajdos, Nathalie Garrec, Cécile Guillaumat, Corinne Guitton, Marie Monfort-Gouraud, François Gouraud, Laurent Holvoet, Ghislaine Ithier, Annie Kamdem, Bérengère Koehl, Aurore Malric, Florence Missud, Brigitte Monier, Marie-Hélène Odièvre, Philippe Joly, Céline Renoux, Franck Patin, Serge Pissard, Nathalie Couque
Clin Chem Lab Med, 2020 Aug 19, PMID: 32813673 DOI: 10.1515/cclm-2020-0803
2019
– The Liver in Sickle Cell Disease.
Eleni Theocharidou, Abid R Suddle
Clin Liver Dis, 2019 May, PMID: 30947870 DOI: 10.1016/j.cld.2018.12.002
2018
– Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, de Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M.
N Engl J Med. 2018 Apr 19;378(16):1479-1493
2017
– Sickle cell disease.
Ware RE, de Montalembert M, Tshilolo L, Abboud MR.
Lancet. 2017 Jul 15;390(10091):311-323. doi: 10.1016/S0140-6736(17)30193-9
– Prevalence and risk factors for red blood cell alloimmunization in 175 children with sickle cell disease in a French university hospital reference centre.
Allali S, Peyrard T, Amiranoff D, Cohen JF, Chalumeau M, Brousse V, de Montalembert M.
Br J Haematol. 2017 May;177(4):641-647. doi: 10.1111/bjh.14609
– Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M.
N Engl J Med. 2017 Mar 2;376(9):848-855.
– Are the risks of treatment to cure a child with severe sickle cell disease too high?
de Montalembert M, Brousse V, Chakravorty S, Pagliuca A, Porter J, Telfer P, Vora A, Rees DC.
BMJ. 2017 Nov 23;359:j5250.
Imagine Institute: curing rare diseases
France Culture | 08.08.2022
At the Imagine Institute, located in the Necker hospital in Paris, researchers are working on the genetic causes of rare diseases. Two teams present their progress concerning eye and blood diseases.
> Read more
Le Blob takes a look at sickle cell disease with Professor Mariane de Montalembert.
Contact information
Necker-Enfants malades university hospital
> General pediatric department
149 rue de Sèvres
75743 PARIS Cedex 15
> Welcome booklet for hospitalized adults
In Necker, the reference center for major sickle cell syndromes and other rare pathologies of the red blood cell and erythropoiesis in brief …
* data valid for 2022