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Journal Articles

Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

Blood (2024) 144 (5): 565–580.

https://doi.org/10.1182/blood.2024024038

Published: 2024

Includes: Supplemental data

Journal Articles

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Tanja C. Vallée, Jannik S. Glasmacher, Hannes Buchner, Peter D. Arkwright, Uta Behrends, Anastasia Bondarenko, Michael J. Browning, David Buchbinder, Alessandro Cattoni, Liudmyla Chernyshova, Peter Ciznar, Theresa Cole, Wojciech Czogała, Gregor Dueckers, John David M. Edgar, Fatih Erbey, Anders Fasth, Francesca Ferrua, Renata Formankova, Eleonora Gambineri, Andrew R. Gennery, Frederick D. Goldman, Luis I. Gonzalez-Granado, Carsten Heilmann, Tarja Heiskanen-Kosma, Hanna Juntti, Leena Kainulainen, Hirokazu Kanegane, Neslihan E. Karaca, Sara S. Kilic, Christoph Klein, Sylwia Kołtan, Irina Kondratenko, Isabelle Meyts, Gulnara M. Nasrullayeva, Lucia D. Notarangelo, Srdjan Pasic, Isabelle Pellier, Claudio Pignata, Siraj Misbah, Ansgar Schulz, Gesmar R. Segundo, Anna Shcherbina, Mary Slatter, Robert Sokolic, Pere Soler-Palacin, Polina Stepensky, Joris M. van Montfrans, Samppa Ryhänen, Beata Wolska-Kuśnierz, John B. Ziegler, Xiaodong Zhao, Alessandro Aiuti, Hans D. Ochs, Michael H. Albert

Blood (2024) 143 (24): 2504–2516.

https://doi.org/10.1182/blood.2023021411

Published: 2024

Includes: Supplemental data

Journal Articles

Improved transplant survival and long-term disease outcome in children with MHC class II deficiency

Blood (2020) 135 (12): 954–973.

https://doi.org/10.1182/blood.2019002690

Published: 2020

Includes: Supplemental data

Journal Articles

Two decades of excellent transplant survival for chronic granulomatous disease: a supraregional immunology transplant center report

Su Han Lum, Terence Flood, Sophie Hambleton, Peter McNaughton, Helen Watson, Mario Abinun, Stephen Owens, Nevenka Cigrovski, Andrew Cant, Andrew R. Gennery, Mary Slatter

Blood (2019) 133 (23): 2546–2549.

https://doi.org/10.1182/blood.2019000021

Published: 2019

Includes: Supplemental data

Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adults with Chronic Granulomatous Disease (CGD): A Study of the Inborn Errors Working Party (IEWP) of the EBMT

Blood (2018) 132 (Supplement 1): 970.

https://doi.org/10.1182/blood-2018-99-114841

Published: 2018

Journal Articles

Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD)

Reem Elkeky, MBBS, David A. Jacobsohn, MD, Rajni Agarwal, MD, Swati Naik, MBBS, Neena Kapoor, MD, Lakshmanan Krishnamurti, MD, Mary Slatter, MD, Federica Galaverna, MD, Pietro Merli, MD, Melissa Aldinger, PharmD, Franco Locatelli

Blood (2018) 132 (Supplement 1): 2207.

https://doi.org/10.1182/blood-2018-99-119792

Published: 2018

Journal Articles

Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen in Patients with Wiskott-Aldrich Syndrome Given Allogeneic Hematopoietic Cell Transplantation — an EBMT Inborn Errors Working Party and Scetide Retrospective Analysis

Blood (2018) 132 (Supplement 1): 2175.

https://doi.org/10.1182/blood-2018-99-112394

Published: 2018

Journal Articles

Administration of BPX-501 Cells Following Αβ T and B-Cell-Depleted HLA-Haploidentical HSCT (haplo-HSCT) in Children with Malignant or Non-Malignant Disorders

Mattia Algeri, MD, Pietro Merli, MD, Waseem Qasim, MD PhD, Mary Slatter, MD, Melissa Kuhn, PharmD, Franco Locatelli

Blood (2018) 132 (Supplement 1): 2171.

https://doi.org/10.1182/blood-2018-99-119635

Published: 2018

Journal Articles

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

Blood (2017) 129 (21): 2928–2938.

https://doi.org/10.1182/blood-2016-11-745638

Published: 2017

Includes: Supplemental data

Journal Articles

Brincidofovir is highly efficacious in controlling adenoviremia in pediatric recipients of hematopoietic cell transplant

Prashant Hiwarkar, Persis Amrolia, Ponni Sivaprakasam, Su Han Lum, Hemalatha Doss, Ciara O’Rafferty, Toni Petterson, Katharine Patrick, Juliana Silva, Mary Slatter, Sarah Lawson, Kanchan Rao, Colin Steward, Adam Gassas, Paul Veys, Robert Wynn

Blood (2017) 129 (14): 2033–2037.

https://doi.org/10.1182/blood-2016-11-749721

Published: 2017

Includes: Supplemental data

Journal Articles

Outcome of Children with Primary Immune-Deficiencies (PIDs) Enrolled in a Phase I-II Trial Based on the Infusion of BPX-501 Donor T Cells Genetically Modified with a Novel Suicide Gene (inducible Caspase 9, iC9) after T-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (haplo-HSCT)

Neena Kapoor, MD, Alice Bertaina, MD PhD, Pietro Merli, MD, Swati Naik, MD, Waseem Qasim, MBBS PhD, Mary Slatter, MD, Reem Elfeky, MD, Barbarella Lucarelli, MD, Monika Smogorzewska, PhD, Giuseppina Li Pira, PhD, Valentina Bertaina, PhD, Annemarie Moseley, MD PhD, Franco Locatelli, MD

Blood (2016) 128 (22): 72.

https://doi.org/10.1182/blood.V128.22.72.72

Published: 2016

Journal Articles

Contemporary Conditioning Regimen before Allogeneic Stem Cell Transplantation for Children with Non-Malignant Diseases

Christina Peters, MD, Mary Slatter, MD, Paul Veys, MD Prof., Franco Locatelli, MD, Adriana Balduzzi, MD, M. Akif Yesilipek, Prof. Dr., Jean-Hugues Dalle, MD PhD Prof., Marina Cavazzana, MD PhD, Jaap Jan Boelens, MD PhD, Isaac Yaniv, MD Prof., Ansgar S Schulz, MD PhD Prof., Jacek Wachowiak, Prof MD PhD, Manfred Hoenig, MD, Arjan C Lankester, MD PhD, Benedicte Neven, MD PhD, Eric Beohou, Arnaud Dallisier, Peter Bader, MD, Andrew Gennery, MD

Blood (2016) 128 (22): 3398.

https://doi.org/10.1182/blood.V128.22.3398.3398

Published: 2016

Journal Articles

Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience

Blood (2016) 128 (3): 440–448.

https://doi.org/10.1182/blood-2016-03-704015

Published: 2016

Journal Articles

Clinical Outcome after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Results of a Phase I-II Trial

Franco Locatelli, Prof, MD PhD, Pietro Merli, MD, Giuseppina Li Pira, PhD, Valentina Bertaina, PhD, Barbarella Lucarelli, MD, Letizia Pomponia Brescia, MD, Mauro Montanari, MD, Daniela Pende, MD, Michela Falco, PhD, Daria Pagliara, MD, Maria Giuseppina Cefalo, MD, Alessandro Moretta, MD, Concetta Quintarelli, PhD, Brigitte Strahm, MD, Waseem Qasim, MBBS PhD, Mary Slatter, Lorenzo Moretta, MD, Annemarie Moseley, MDPhD, Alice Bertaina, MDPhD

Blood (2015) 126 (23): 1931.

https://doi.org/10.1182/blood.V126.23.1931.1931

Published: 2015

Journal Articles

Outcome of hematopoietic stem cell transplantation for adenosine deaminase–deficient severe combined immunodeficiency

Blood (2012) 120 (17): 3615–3624.

https://doi.org/10.1182/blood-2011-12-396879

Published: 2012

Includes: Supplemental data

Journal Articles

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

Blood (2012) 119 (12): 2949–2955.

https://doi.org/10.1182/blood-2011-06-363572

Published: 2012

Includes: Supplemental data

Journal Articles

Reduced Intensity Hematopoietic Stem Cell Transplant Rescues Immune Function and Corrects Pulmonary Alveolar Proteinosis in DCML Deficiency/GATA 2 Mutation

Blood (2011) 118 (21): 2045.

https://doi.org/10.1182/blood.V118.21.2045.2045

Published: 2011

Journal Articles

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

Blood (2011) 118 (6): 1675–1684.

https://doi.org/10.1182/blood-2010-11-319376

Published: 2011

Includes: Supplemental data

Journal Articles

Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening

Lucinda Brown, Jinhua Xu-Bayford, Zoe Allwood, Mary Slatter, Andrew Cant, E. Graham Davies, Paul Veys, Andrew R. Gennery, H. Bobby Gaspar

Blood (2011) 117 (11): 3243–3246.

https://doi.org/10.1182/blood-2010-08-300384

Published: 2011

Journal Articles

Comparison of Outcomes of Mismatched Related Stem Cell and Unrelated Cord Blood Transplants in Children with Severe T-Cell Deficiencies.

Blood (2009) 114 (22): 664.

https://doi.org/10.1182/blood.V114.22.664.664

Published: 2009

NARROW

Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Improved transplant survival and long-term disease outcome in children with MHC class II deficiency

Two decades of excellent transplant survival for chronic granulomatous disease: a supraregional immunology transplant center report

Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adults with Chronic Granulomatous Disease (CGD): A Study of the Inborn Errors Working Party (IEWP) of the EBMT

Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD)

Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen in Patients with Wiskott-Aldrich Syndrome Given Allogeneic Hematopoietic Cell Transplantation — an EBMT Inborn Errors Working Party and Scetide Retrospective Analysis

Administration of BPX-501 Cells Following Αβ T and B-Cell-Depleted HLA-Haploidentical HSCT (haplo-HSCT) in Children with Malignant or Non-Malignant Disorders

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

Brincidofovir is highly efficacious in controlling adenoviremia in pediatric recipients of hematopoietic cell transplant

Contemporary Conditioning Regimen before Allogeneic Stem Cell Transplantation for Children with Non-Malignant Diseases

Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience

Clinical Outcome after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Results of a Phase I-II Trial

Outcome of hematopoietic stem cell transplantation for adenosine deaminase–deficient severe combined immunodeficiency

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

Reduced Intensity Hematopoietic Stem Cell Transplant Rescues Immune Function and Corrects Pulmonary Alveolar Proteinosis in DCML Deficiency/GATA 2 Mutation

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening

Comparison of Outcomes of Mismatched Related Stem Cell and Unrelated Cord Blood Transplants in Children with Severe T-Cell Deficiencies.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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