Conditioning regimens for inborn errors of immunity: current perspectives and future strategies

[1]  E. Macintyre,et al.  Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome , 2022, Nature medicine.

[2]  T. Morio,et al.  Hematopoietic Cell Transplantation for Inborn Errors of Immunity Other than Severe Combined Immunodeficiency in Japan: Retrospective Analysis for 1985–2016 , 2021, Journal of clinical immunology.

[3]  A. Fischer,et al.  Hematopoietic cell transplantation in severe combined immunodeficiency: the SCETIDE 2006-2014 European cohort. , 2021, The Journal of allergy and clinical immunology.

[4]  T. Morio,et al.  Hematopoietic Cell Transplantation for Severe Combined Immunodeficiency Patients: a Japanese Retrospective Study , 2021, Journal of Clinical Immunology.

[5]  Michael J. Eckrich,et al.  A Comparison of Hematopoietic Cell Transplant Conditioning Regimens for Hemophagocytic Lymphohistiocytosis Disorders. , 2021, The Journal of allergy and clinical immunology.

[6]  A. Schulz,et al.  EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity , 2021, Bone Marrow Transplantation.

[7]  H. Ochs,et al.  Hematopoietic Stem Cell Therapy for Wiskott–Aldrich Syndrome: Improved Outcome and Quality of Life , 2021, Journal of blood medicine.

[8]  I. Sultan,et al.  Haploidentical Hematopoietic Cell Transplantation Using Post-transplant Cyclophosphamide for Children with Non-malignant Diseases , 2021, Journal of Clinical Immunology.

[9]  S. Choo,et al.  Reduced-Intensity/Reduced-Toxicity Conditioning Approaches Are Tolerated in XIAP Deficiency but Patients Fare Poorly with Acute GVHD , 2021, Journal of Clinical Immunology.

[10]  H. Kanegane,et al.  Hematopoietic Cell Transplantation with Reduced Intensity Conditioning Using Fludarabine/Busulfan or Fludarabine/Melphalan for Primary Immunodeficiency Diseases , 2021, Journal of Clinical Immunology.

[11]  A. Fischer,et al.  Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation , 2020, Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology.

[12]  H. Kanegane,et al.  A prospective study of allogeneic hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donors for non-malignant diseases. , 2020, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[13]  T. Morio,et al.  Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan , 2020, Frontiers in Immunology.

[14]  J. Garcia,et al.  Outcomes after Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Patients with Primary Immunodeficiency Diseases. , 2020, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[15]  M. Roncarolo,et al.  Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions. , 2020, The Journal of allergy and clinical immunology.

[16]  Yao-Hsu Yang,et al.  Chronic Granulomatous Disease: a Comprehensive Review , 2020, Clinical Reviews in Allergy & Immunology.

[17]  M. Ayas,et al.  Hematopoietic stem cell transplantation in children with Griscelli syndrome type 2: a single-center report on 35 patients , 2020, Bone Marrow Transplantation.

[18]  L. Notarangelo,et al.  Excellent Outcomes Following Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: A PIDTC Report. , 2020, Blood.

[19]  J. Casanova,et al.  Human Inborn Errors of Immunity: 2019 Update of the IUIS Phenotypical Classification , 2020, Journal of Clinical Immunology.

[20]  J. Casanova,et al.  Human Inborn Errors of Immunity: 2019 Update on the Classification from the International Union of Immunological Societies Expert Committee , 2020, Journal of Clinical Immunology.

[21]  A. Fischer,et al.  Gene therapy for severe combined immunodeficiencies and beyond , 2019, The Journal of experimental medicine.

[22]  Stephanie N. Hicks,et al.  Prospective study of a novel, radiation-free, reduced-intensity bone marrow transplantation platform for primary immunodeficiency diseases. , 2020, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[23]  E. Gambineri,et al.  Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Immune Dysregulatory Disorders , 2019, Front. Pediatr..

[24]  R. Mitchell,et al.  TCR α+β+/CD19+ cell–depleted hematopoietic stem cell transplantation for pediatric patients , 2019, Pediatric transplantation.

[25]  A. Fischer,et al.  Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplant Cyclophosphamide for Primary Immunodeficiencies and Inherited Disorders in Children. , 2019, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[26]  A. Gennery,et al.  New graft manipulation strategies improve the outcome of mismatched stem cell transplantation in children with primary immunodeficiencies. , 2019, The Journal of allergy and clinical immunology.

[27]  T. Morio,et al.  Long-term outcome and chimerism in patients with Wiskott–Aldrich syndrome treated by hematopoietic cell transplantation: a retrospective nationwide survey , 2019, International journal of hematology.

[28]  A. Fischer,et al.  Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study. , 2019, The Journal of allergy and clinical immunology.

[29]  P. Fu,et al.  Role of Allogeneic Hematopoietic Stem Cell Transplant for Chronic Granulomatous Disease (CGD): a Report of the United States Immunodeficiency Network , 2019, Journal of Clinical Immunology.

[30]  A. Worth,et al.  How I manage patients with Wiskott Aldrich syndrome , 2019, British journal of haematology.

[31]  L. Notarangelo,et al.  SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. , 2018, Blood.

[32]  E. Inoue,et al.  A prospective study of allogeneic transplantation from unrelated donors for chronic granulomatous disease with target busulfan-based reduced-intensity conditioning , 2018, Bone Marrow Transplantation.

[33]  Rong Liu,et al.  Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott–Aldrich syndrome , 2018, Pediatric blood & cancer.

[34]  S. Kozlovskaya,et al.  A Conditioning Regimen with Plerixafor Is Safe and Improves the Outcome of TCRαβ+ and CD19+ Cell-Depleted Stem Cell Transplantation in Patients with Wiskott-Aldrich Syndrome. , 2018, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[35]  G. Lucchini,et al.  T‐cell receptor &agr;&bgr;+ and CD19+ cell–depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency , 2017, The Journal of allergy and clinical immunology.

[36]  H. Kanegane,et al.  Population Pharmacokinetics of Intravenous Busulfan in Japanese Pediatric Patients With Primary Immunodeficiency Diseases , 2018, Journal of clinical pharmacology.

[37]  Scott Montgomery,et al.  Confirmed efficacy of etoposide and dexamethasone in HLH treatment: long-term results of the cooperative HLH-2004 study. , 2017, Blood.

[38]  I. Tezcan,et al.  Hematopoietic stem cell transplantation in children with Griscelli syndrome: A single‐center experience , 2017, Pediatric transplantation.

[39]  L. Notarangelo,et al.  Long‐term outcomes of 176 patients with X‐linked hyper‐IgM syndrome treated with or without hematopoietic cell transplantation , 2017, The Journal of allergy and clinical immunology.

[40]  H. Kanegane,et al.  Hematopoietic Stem Cell Transplantation for XIAP Deficiency in Japan , 2016, Journal of Clinical Immunology.

[41]  G. Lucchini,et al.  Effect of stem cell source on long-term chimerism and event-free survival in children with primary immunodeficiency disorders after fludarabine and melphalan conditioning regimen. , 2016, The Journal of allergy and clinical immunology.

[42]  O. Ohara,et al.  Novel compound heterozygous mutations in a Japanese girl with Janus kinase 3 deficiency , 2016, Pediatrics international : official journal of the Japan Pediatric Society.

[43]  A. Schulz,et al.  Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience. , 2016, Blood.

[44]  B. Burkhardt,et al.  The minimum required level of donor chimerism in hereditary hemophagocytic lymphohistiocytosis. , 2016, Blood.

[45]  S. Adachi,et al.  Allogeneic hematopoietic stem cell transplantation for Chediak–Higashi syndrome , 2016, Pediatric transplantation.

[46]  A. Ghavamzadeh,et al.  Long-term outcomes of fludarabine, melphalan and antithymocyte globulin as reduced-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiency disorders: a prospective single center study , 2016, Bone Marrow Transplantation.

[47]  T. Fleisher Transplantation Outcomes for Severe Combined Immunodeficiency 2000–2009 , 2015, Pediatrics.

[48]  Y. Skvortsova,et al.  Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRαβ and CD19 Depletion in Children with Primary Immunodeficiency Syndromes. , 2015, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[49]  T. Morio,et al.  Clinical features and hematopoietic stem cell transplantations for CD40 ligand deficiency in Japan. , 2015, The Journal of allergy and clinical immunology.

[50]  J. Seo Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: recent advances and controversies , 2015, Blood research.

[51]  A. Schulz,et al.  Haploidentical T-cell alpha beta receptor and CD19-depleted stem cell transplant for Wiskott-Aldrich syndrome. , 2014, The Journal of allergy and clinical immunology.

[52]  S. Davies,et al.  Reduced-intensity conditioning hematopoietic cell transplantation is an effective treatment for patients with SLAM-associated protein deficiency/X-linked lymphoproliferative disease type 1. , 2014, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[53]  H. Kanegane,et al.  Pneumothorax in patients with severe combined immunodeficiency , 2014, Pediatrics international : official journal of the Japan Pediatric Society.

[54]  A. Fischer,et al.  Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study , 2014, The Lancet.

[55]  P. Veys,et al.  How I treat severe combined immunodeficiency. , 2013, Blood.

[56]  A. Gennery,et al.  Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation. , 2013, The Journal of allergy and clinical immunology.

[57]  H. Kanegane,et al.  Clinical characteristics and outcomes of chédiak–Higashi syndrome: A nationwide survey of Japan , 2013, Pediatric blood & cancer.

[58]  A. Ghavamzadeh,et al.  Hematopoietic stem cell transplantation with a reduced‐intensity conditioning regimen in pediatric patients with Griscelli syndrome type 2 , 2013, Pediatric transplantation.

[59]  E. Haddad,et al.  B-cell reconstitution for SCID: should a conditioning regimen be used in SCID treatment? , 2013, The Journal of allergy and clinical immunology.

[60]  A. Fischer,et al.  Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes. , 2013, Blood.

[61]  A. Fischer,et al.  Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. , 2012, Blood.

[62]  H. Kanegane,et al.  Clinical features and outcome of X‐linked lymphoproliferative syndrome type 1 (SAP deficiency) in Japan identified by the combination of flow cytometric assay and genetic analysis , 2012, Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology.

[63]  R. Egeler,et al.  Chemoimmunotherapy for hemophagocytic lymphohistiocytosis: long-term results of the HLH-94 treatment protocol. , 2011, Blood.

[64]  A. Fischer,et al.  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. , 2011, Blood.

[65]  A. Fischer,et al.  X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. , 2011, Blood.

[66]  S. Davies,et al.  Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. , 2010, Blood.

[67]  A. Fischer,et al.  Immune deficiencies , infection , and systemic immune disorders Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe : Entering a new century , do we do better ? , 2010 .

[68]  T. Hara,et al.  Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein–Barr virus‐associated hemophagocytic lymphohistiocytosis in Japan , 2010, Pediatric blood & cancer.

[69]  A. Towbin,et al.  Chronic granulomatous disease , 2010, Pediatric Radiology.

[70]  A. Fischer,et al.  Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients. , 2009, Blood.

[71]  A. Fischer,et al.  Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. , 2008, Blood.

[72]  J. Kurtzberg,et al.  Hematopoietic cell transplantation for Chediak–Higashi syndrome , 2007, Bone Marrow Transplantation.

[73]  T. Morio,et al.  Hematopoietic stem cell transplantation for 30 patients with primary immunodeficiency diseases: 20 years experience of a single team , 2006, Bone Marrow Transplantation.

[74]  P. Veys,et al.  Stem cell transplantation with reduced-intensity conditioning for hemophagocytic lymphohistiocytosis. , 2006, Blood.

[75]  Kohsuke Imai,et al.  Clinical course of patients with WASP gene mutations. , 2004, Blood.

[76]  A. Fischer,et al.  Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99 , 2003, The Lancet.

[77]  A. Fischer,et al.  Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000. , 2002, Blood.

[78]  R. Egeler,et al.  Treatment of hemophagocytic lymphohistiocytosis with HLH-94 immunochemotherapy and bone marrow transplantation. , 2002, Blood.

[79]  G. Leverger,et al.  Allogeneic bone marrow transplantation for erythrophagocytic lymphohistiocytosis. , 1986, The Journal of pediatrics.

[80]  H. Lohrmann The problem of permanent bone marrow damage after cytotoxic drug treatment. , 1984, Oncology.

[81]  R. Gatti,et al.  Immunological reconstitution of sex-linked lymphopenic immunological deficiency. , 1968, Lancet.