• Je něco špatně v tomto záznamu ?

Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

F. Ferrua, S. Galimberti, V. Courteille, MA. Slatter, C. Booth, D. Moshous, B. Neven, S. Blanche, M. Cavazzana, A. Laberko, A. Shcherbina, D. Balashov, E. Soncini, F. Porta, H. Al-Mousa, B. Al-Saud, H. Al-Dhekri, R. Arnaout, R. Formankova, Y....

. 2019 ; 143 (6) : 2238-2253. [pub] 20190117

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články, Research Support, N.I.H., Extramural, práce podpořená grantem

Perzistentní odkaz   https://www.medvik.cz/link/bmc20023903

Grantová podpora
U54 AI082973 NIAID NIH HHS - United States
R13 AI094943 NIAID NIH HHS - United States

BACKGROUND: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation (HSCT). OBJECTIVE: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics. METHODS: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure. RESULTS: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow-derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%. CONCLUSION: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy.

Biotherapy Clinical Investigation Center Groupe Hospitalier Universitaire Ouest AP HP INSERM Paris France

Biotherapy Department Necker Children's Hospital AP HP Paris France

Cancer Centre for Children Children's Hospital at Westmead Sydney Australia

Center for Chronic Immunodeficiency Medical Center Faculty of Medicine University of Freiburg Freiburg Germany

Center for Pediatrics and Center for Chronic Immunodeficiency Medical Center University of Freiburg Freiburg Germany

Center of Biostatistics for Clinical Epidemiology School of Medicine and Surgery University of Milano Bicocca Monza Italy

College de France Paris France

Department of Allergy and Immunology Royal Children's Hospital Melbourne Australia

Department of BMT Great Ormond Street Hospital for Children NHS Trust London United Kingdom

Department of Clinical Immunology and Transplantology Jagiellonian University Medical Collage Transplantation Center University Children's Hospital Cracow Poland

Department of Hematology Karolinska University Hospital Stockholm Sweden

Department of Pediatric Hematology and Oncology University Hospital Motol Prague Prague Czech Republic

Department of Pediatric Hematology and Oncology Wroclaw Medical University Wrocław Poland

Department of Pediatric Hematology and Stem Cell Transplantation United St István and St László Hospital Budapest Hungary

Department of Pediatric Immunology Allergy and BMT Unit Ankara University Medical School Ankara Turkey

Department of Pediatric Immunology and Allergy Ankara University School of Medicine Ankara Turkey

Department of Pediatric Immunology and HSCT Great North Children's Hospital Newcastle upon Tyne United Kingdom

Department of Pediatric Immunology Great Ormond Street Hospital London United Kingdom

Department of Pediatrics and Adolescent Medicine Division of Pediatric Hematology and Oncology Medical Center Faculty of Medicine University of Freiburg Freiburg Germany

Department of Pediatrics Division of Allergology Clinical Immunology Respiratory Diseases and Rheumatology University Hospital Center Zagreb Zagreb Croatia

Department of Pediatrics King Faisal Specialist Hospital and Research Center Riyadh Saudi Arabia

Department of Pediatrics Memorial Sloan Kettering Cancer Center BMT and Cell Therapies Program New York NY

Department of Pediatrics Sahlgrenska Academy at University of Gothenburg and Queen Silvia Children's Hospital Gothenburg Sweden

Department of Pediatrics University Hospitals Leuven Division of Pediatric Immunology Department of Immunology and Microbiology Catholic University Leuven Leuven Belgium

Department of Pediatrics University Medical Center Ulm Ulm Germany

Department of Pediatrics University Medical Centre Utrecht Utrecht University Utrecht The Netherlands

Department of Pediatrics University of Texas Southwestern Medical Center Dallas Dallas Tex

Department of Pediatrics Willem Alexander Children's hospital Leiden University Medical Center Leiden The Netherlands

Division of Allergy Immunology Department of Pediatrics Children's Hospital of Philadelphia Philadelphia Pa

Division of Pediatric Allergy Immunology and Bone Marrow Transplantation University of California San Francisco Calif

Division of Pediatrics CLINTEC Karolinska Institutet Stockholm Sweden

Dmitry Rogachev Federal Research Centre of Pediatric Hematology Oncology and Immunology Moscow Russia

French National Reference Center for Primary Immune Deficiencies Necker Enfants Malades University Hospital AP HP Paris France

Hématologie Adulte Hôpital Necker AP HP Paris France

Immunology Department Children's Memorial Health Institute Warsaw Poland

INSERM UMR 1163 Laboratory of Human Genetics of Infectious Diseases Necker Branch Paris France

INSERM UMR 1163 Laboratory of Human Lymphohematopoiesis Paris France

Institut d'Hematologie et d'Oncologie Pediatrique Hospices Civils de Lyon Lyon France

Institute of Cellular Medicine Newcastle University Newcastle upon Tyne United Kingdom

L Hirszfeld Institute of Immunology and Experimental Therapy Polish Academy of Sciences Wrocław Poland

Laboratory for Translational Immunology Tumor immunology University Medical Center Utrecht Utrecht The Netherlands

Lower Silesian Center for Cellular Transplantation and National Bone Marrow Donor Registry Wrocław Poland

Nationwide Children's Hospital Columbus Ohio

Paris Descartes Sorbonne Paris Cité University Imagine Institute Paris France

Pediatric Blood and Marrow Transplant University of Minnesota Minneapolis Minn

Pediatric Blood Disorders Immunodeficiency and SCT Astrid Lindgren Children's Hospital Karolinska University Hospital Stockholm Sweden

Pediatric Clinic Rigshospitalet Copenhagen Denmark

Pediatric Hematology and Oncology Department Hospital Universitario MaternoInfantil Vall d'Hebron Barcelona Spain

Pediatric Hematology Immunology and Rheumatology Unit Necker Enfants Malades Hospital Assistance Publique Hôpitaux de Paris Paris France

Pediatric Hematology Oncology and Stem Cell Transplantation Ghent University Hospital Ghent Belgium

Pediatric Hematology Oncology Dr von Hauner University Children's Hospital Munich Germany

Pediatric Oncology and Hematology Unit Children Hospital University Hospital Nancy Vandoeuvre les Nancy France

Pediatric Oncology Hematology and BMT Unit Spedali Civili di Brescia Brescia Italy

Pediatric Stem Cell Transplantation Columbia University College of Physicians and Surgeons New York NY

Princess Maxima Center for Pediatric Oncology Utrecht The Netherlands

San Raffaele Telethon Institute for Gene Therapy Pediatric Immunohematology and Bone Marrow Transplantation Unit San Raffaele Scientific Institute Milan Italy

Service d'hématologie pédiatrique Hôpital de la Timone Enfants Marseille France

University Children's Hospital of Cracow Cracow Poland

University of Sydney Medical Program Sydney Australia

Vita Salute San Raffaele University Milan Italy

Citace poskytuje Crossref.org

000      
00000naa a2200000 a 4500
001      
bmc20023903
003      
CZ-PrNML
005      
20201214131633.0
007      
ta
008      
201125s2019 xxu f 000 0|eng||
009      
AR
024    7_
$a 10.1016/j.jaci.2018.12.1010 $2 doi
035    __
$a (PubMed)30660643
040    __
$a ABA008 $b cze $d ABA008 $e AACR2
041    0_
$a eng
044    __
$a xxu
100    1_
$a Ferrua, Francesca $u Department of Pediatric Immunology and HSCT, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. Electronic address: ferrua.francesca@hsr.it.
245    10
$a Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study / $c F. Ferrua, S. Galimberti, V. Courteille, MA. Slatter, C. Booth, D. Moshous, B. Neven, S. Blanche, M. Cavazzana, A. Laberko, A. Shcherbina, D. Balashov, E. Soncini, F. Porta, H. Al-Mousa, B. Al-Saud, H. Al-Dhekri, R. Arnaout, R. Formankova, Y. Bertrand, A. Lange, J. Smart, B. Wolska-Kusnierz, VM. Aquino, CC. Dvorak, A. Fasth, F. Fouyssac, C. Heilmann, M. Hoenig, C. Schuetz, J. Kelečić, RGM. Bredius, AC. Lankester, CA. Lindemans, F. Suarez, KE. Sullivan, MH. Albert, K. Kałwak, V. Barlogis, M. Bhatia, V. Bordon, W. Czogala, L. Alonso, F. Dogu, J. Gozdzik, A. Ikinciogullari, G. Kriván, P. Ljungman, I. Meyts, P. Mustillo, AR. Smith, C. Speckmann, M. Sundin, SJ. Keogh, PJ. Shaw, JJ. Boelens, AS. Schulz, P. Sedlacek, P. Veys, N. Mahlaoui, A. Janda, EG. Davies, A. Fischer, MJ. Cowan, AR. Gennery, SCETIDE, PIDTC, EBMT & ESID IEWP,
520    9_
$a BACKGROUND: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation (HSCT). OBJECTIVE: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics. METHODS: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure. RESULTS: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow-derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%. CONCLUSION: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy.
650    _2
$a ligand CD40 $x nedostatek $7 D023201
650    _2
$a dítě $7 D002648
650    _2
$a předškolní dítě $7 D002675
650    12
$a transplantace hematopoetických kmenových buněk $7 D018380
650    _2
$a lidé $7 D006801
650    _2
$a kojenec $7 D007223
650    _2
$a novorozenec $7 D007231
650    _2
$a výsledek terapie $7 D016896
650    _2
$a kombinované imunodeficience vázané na chromozom X $x mortalita $x terapie $7 D053632
655    _2
$a časopisecké články $7 D016428
655    _2
$a Research Support, N.I.H., Extramural $7 D052061
655    _2
$a práce podpořená grantem $7 D013485
700    1_
$a Galimberti, Stefania $u Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy.
700    1_
$a Courteille, Virginie $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Slatter, Mary Anne $u Department of Pediatric Immunology and HSCT, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom; Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
700    1_
$a Booth, Claire $u Department of Pediatric Immunology, Great Ormond Street Hospital, London, United Kingdom.
700    1_
$a Moshous, Despina $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Neven, Benedicte $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Blanche, Stephane $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Cavazzana, Marina $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Biotherapy Department, Necker Children's Hospital, AP-HP, Paris, France; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, Paris, France; INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, Paris, France.
700    1_
$a Laberko, Alexandra $u Dmitry Rogachev Federal Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
700    1_
$a Shcherbina, Anna $u Dmitry Rogachev Federal Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
700    1_
$a Balashov, Dmitry $u Dmitry Rogachev Federal Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
700    1_
$a Soncini, Elena $u Pediatric Oncology-Hematology and BMT Unit, Spedali Civili di Brescia, Brescia, Italy.
700    1_
$a Porta, Fulvio $u Pediatric Oncology-Hematology and BMT Unit, Spedali Civili di Brescia, Brescia, Italy.
700    1_
$a Al-Mousa, Hamoud $u Department of Pediatrics, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.
700    1_
$a Al-Saud, Bandar $u Department of Pediatrics, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.
700    1_
$a Al-Dhekri, Hasan $u Department of Pediatrics, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.
700    1_
$a Arnaout, Rand $u Department of Pediatrics, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.
700    1_
$a Formankova, Renata $u Department of Pediatric Hematology and Oncology, University Hospital Motol Prague, Prague, Czech Republic.
700    1_
$a Bertrand, Yves $u Institut d'Hematologie et d'Oncologie Pediatrique, Hospices Civils de Lyon, Lyon, France.
700    1_
$a Lange, Andrzej $u L. Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Wrocław, Poland; Lower Silesian Center for Cellular Transplantation & National Bone Marrow Donor Registry, Wrocław, Poland.
700    1_
$a Smart, Joanne $u Department of Allergy and Immunology, Royal Children's Hospital, Melbourne, Australia.
700    1_
$a Wolska-Kusnierz, Beata $u Immunology Department, Children's Memorial Health Institute, Warsaw, Poland.
700    1_
$a Aquino, Victor M $u Department of Pediatrics, University of Texas Southwestern Medical Center Dallas, Dallas, Tex.
700    1_
$a Dvorak, Christopher C $u Division of Pediatric Allergy, Immunology & Bone Marrow Transplantation, University of California, San Francisco, Calif.
700    1_
$a Fasth, Anders $u Department of Pediatrics, Sahlgrenska Academy at University of Gothenburg and Queen Silvia Children's Hospital, Gothenburg, Sweden.
700    1_
$a Fouyssac, Fanny $u Pediatric Oncology and Hematology Unit, Children Hospital, University Hospital Nancy, Vandoeuvre-les-Nancy, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Heilmann, Carsten $u Pediatric Clinic, Rigshospitalet, Copenhagen, Denmark.
700    1_
$a Hoenig, Manfred $u Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
700    1_
$a Schuetz, Catharina $u Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
700    1_
$a Kelečić, Jadranka $u Department of Pediatrics, Division of Allergology, Clinical Immunology, Respiratory Diseases and Rheumatology, University Hospital Center Zagreb, Zagreb, Croatia.
700    1_
$a Bredius, Robbert G M $u Department of Pediatrics/Willem-Alexander Children's hospital, Leiden University Medical Center, Leiden, The Netherlands.
700    1_
$a Lankester, Arjan C $u Department of Pediatrics/Willem-Alexander Children's hospital, Leiden University Medical Center, Leiden, The Netherlands.
700    1_
$a Lindemans, Caroline A $u Department of Pediatrics, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands; Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands.
700    1_
$a Suarez, Felipe $u Hématologie Adulte, Hôpital Necker, AP-HP, Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Sullivan, Kathleen E $u Division of Allergy Immunology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pa.
700    1_
$a Albert, Michael H $u Pediatric Hematology/Oncology, Dr. von Hauner University Children's Hospital, Munich, Germany.
700    1_
$a Kałwak, Krzysztof $u Department of Pediatric Hematology and Oncology, Wroclaw Medical University, Wrocław, Poland.
700    1_
$a Barlogis, Vincent $u Service d'hématologie pédiatrique, Hôpital de la Timone Enfants, Marseille, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France.
700    1_
$a Bhatia, Monica $u Pediatric Stem Cell Transplantation, Columbia University College of Physicians and Surgeons, New York, NY.
700    1_
$a Bordon, Victoria $u Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium.
700    1_
$a Czogala, Wojciech $u University Children's Hospital of Cracow, Cracow, Poland.
700    1_
$a Alonso, Laura $u Pediatric Hematology and Oncology Department, Hospital Universitario MaternoInfantil Vall d'Hebron, Barcelona, Spain.
700    1_
$a Dogu, Figen $u Department of Pediatric Immunology and Allergy, Ankara University School of Medicine, Ankara, Turkey.
700    1_
$a Gozdzik, Jolanta $u Department of Clinical Immunology and Transplantology, Jagiellonian University, Medical Collage, Transplantation Center, University Children's Hospital, Cracow, Poland.
700    1_
$a Ikinciogullari, Aydan $u Department of Pediatric Immunology-Allergy and BMT Unit, Ankara University Medical School, Ankara, Turkey.
700    1_
$a Kriván, Gergely $u Department of Pediatric Hematology and Stem Cell Transplantation United St. István and St László Hospital, Budapest, Hungary.
700    1_
$a Ljungman, Per $u Department of Hematology, Karolinska University Hospital, Stockholm, Sweden.
700    1_
$a Meyts, Isabelle $u Department of Pediatrics, University Hospitals Leuven, Division of Pediatric Immunology, Department of Immunology and Microbiology, Catholic University Leuven, Leuven, Belgium.
700    1_
$a Mustillo, Peter $u Nationwide Children's Hospital, Columbus, Ohio.
700    1_
$a Smith, Angela R $u Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, Minn.
700    1_
$a Speckmann, Carsten $u Center for Chronic Immunodeficiency, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany; Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
700    1_
$a Sundin, Mikael $u Division of Pediatrics, CLINTEC, Karolinska Institutet, Stockholm, Sweden; Pediatric Blood Disorders, Immunodeficiency and SCT, Astrid Lindgren Children's Hospital, Karolinska University Hospital, Stockholm, Sweden.
700    1_
$a Keogh, Steven John $u Cancer Centre for Children, Children's Hospital at Westmead, Sydney, Australia.
700    1_
$a Shaw, Peter John $u Cancer Centre for Children, Children's Hospital at Westmead, Sydney, Australia; University of Sydney Medical Program, Sydney, Australia.
700    1_
$a Boelens, Jaap Jan $u Department of Pediatrics, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands; Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, BMT and Cell Therapies Program, New York, NY; Laboratory for Translational Immunology, Tumor-immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
700    1_
$a Schulz, Ansgar S $u Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
700    1_
$a Sedlacek, Petr $u Department of Pediatric Hematology and Oncology, University Hospital Motol Prague, Prague, Czech Republic.
700    1_
$a Veys, Paul $u Department of BMT, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom.
700    1_
$a Mahlaoui, Nizar $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France; INSERM UMR 1163, Laboratory of Human Genetics of Infectious Diseases, Necker Branch, Paris, France.
700    1_
$a Janda, Ales $u Center for Pediatrics and Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg, Germany.
700    1_
$a Davies, E Graham $u Department of Pediatric Immunology, Great Ormond Street Hospital, London, United Kingdom.
700    1_
$a Fischer, Alain $u Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France; College de France, Paris, France.
700    1_
$a Cowan, Morton J $u Division of Pediatric Allergy, Immunology & Bone Marrow Transplantation, University of California, San Francisco, Calif.
700    1_
$a Gennery, Andrew Richard $u Department of Pediatric Immunology and HSCT, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom; Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
710    2_
$a SCETIDE, PIDTC, EBMT & ESID IEWP
773    0_
$w MED00002505 $t The Journal of allergy and clinical immunology $x 1097-6825 $g Roč. 143, č. 6 (2019), s. 2238-2253
856    41
$u https://pubmed.ncbi.nlm.nih.gov/30660643 $y Pubmed
910    __
$a ABA008 $b sig $c sign $y a $z 0
990    __
$a 20201125 $b ABA008
991    __
$a 20201214131629 $b ABA008
999    __
$a ok $b bmc $g 1596222 $s 1114579
BAS    __
$a 3
BAS    __
$a PreBMC
BMC    __
$a 2019 $b 143 $c 6 $d 2238-2253 $e 20190117 $i 1097-6825 $m Journal of allergy and clinical immunology $n J Allergy Clin Immunol $x MED00002505
GRA    __
$a U54 AI082973 $p NIAID NIH HHS $2 United States
GRA    __
$a R13 AI094943 $p NIAID NIH HHS $2 United States
LZP    __
$a Pubmed-20201125

Najít záznam

Citační ukazatele

Nahrávání dat ...

Možnosti archivace

Nahrávání dat ...