Literature DB >> 30590619

Hematopoietic Stem Cell Transplantation in Primary Immunodeficiencies Beyond Severe Combined Immunodeficiency.

Alexandra F Freeman1.   

Abstract

Hematopoietic stem cell transplantation (HSCT) has been the standard of care for infants with severe combined immunodeficiency (SCID) for several decades due to the dismal prognosis early in life without immune reconstitution. In recent years, as HSCT conditioning regimens and supportive care have greatly improved, HSCT is gaining in acceptance for more non-SCID primary immunodeficiencies (PIDs) and outside the early childhood period. In addition, potential donor options for non-SCID PIDs are expanding with increasing success for haploidentical donor transplants. In this brief report of a presentation at the PIDS-St. Jude 2018 conference, PIDs for which transplants are increasingly performed outside of early childhood will be discussed.

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Year:  2018        PMID: 30590619      PMCID: PMC6306013          DOI: 10.1093/jpids/piy114

Source DB:  PubMed          Journal:  J Pediatric Infect Dis Soc        ISSN: 2048-7193            Impact factor:   3.164


  15 in total

1.  Successful long-term immunologic reconstitution by allogeneic hematopoietic stem cell transplantation cures patients with autosomal dominant hyper-IgE syndrome.

Authors:  Evgenios Goussetis; Ioulia Peristeri; Vasiliki Kitra; Joanne Traeger-Synodinos; Maria Theodosaki; Katerina Psarra; Maria Kanariou; Fotini Tzortzatou-Stathopoulou; Eftichia Petrakou; Irene Fylaktou; Emmanuel Kanavakis; Stelios Graphakos
Journal:  J Allergy Clin Immunol       Date:  2010-07-02       Impact factor: 10.793

Review 2.  Diagnostic and therapeutic challenges in a child with complete interferon-γ receptor 1 deficiency.

Authors:  Peter Olbrich; Maria Teresa Martínez-Saavedra; José Maria Perez-Hurtado; Cristina Sanchez; Berta Sanchez; Caroline Deswarte; Ignacio Obando; Jean-Laurent Casanova; Carsten Speckmann; Jacinta Bustamante; Carlos Rodriguez-Gallego; Olaf Neth
Journal:  Pediatr Blood Cancer       Date:  2015-07-14       Impact factor: 3.167

3.  Matched related and unrelated donor hematopoietic stem cell transplantation for DOCK8 deficiency.

Authors:  Jennifer Cuellar-Rodriguez; Alexandra F Freeman; Jennifer Grossman; Helen Su; Mark Parta; Heardley Murdock; Nirali Shah; Catherine Bollard; Heidi H Kong; Niki Moutsopoulos; Kelly Stone; Juan Gea-Banacloche; Steven M Holland; Dennis D Hickstein
Journal:  Biol Blood Marrow Transplant       Date:  2015-01-27       Impact factor: 5.742

4.  Mutations in GATA2 are associated with the autosomal dominant and sporadic monocytopenia and mycobacterial infection (MonoMAC) syndrome.

Authors:  Amy P Hsu; Elizabeth P Sampaio; Javed Khan; Katherine R Calvo; Jacob E Lemieux; Smita Y Patel; David M Frucht; Donald C Vinh; Roger D Auth; Alexandra F Freeman; Kenneth N Olivier; Gulbu Uzel; Christa S Zerbe; Christine Spalding; Stefania Pittaluga; Mark Raffeld; Douglas B Kuhns; Li Ding; Michelle L Paulson; Beatriz E Marciano; Juan C Gea-Banacloche; Jordan S Orange; Jennifer Cuellar-Rodriguez; Dennis D Hickstein; Steven M Holland
Journal:  Blood       Date:  2011-06-13       Impact factor: 22.113

5.  Dedicator of cytokinesis 8 deficiency: a predisposition to sclerosing cholangitis.

Authors:  Tejshri Shah; Catherine Cale; Nedim Hadzic; Alison Jones
Journal:  Clin Immunol       Date:  2014-09-16       Impact factor: 3.969

6.  Bone marrow transplantation does not correct the hyper IgE syndrome.

Authors:  A R Gennery; T J Flood; M Abinun; A J Cant
Journal:  Bone Marrow Transplant       Date:  2000-06       Impact factor: 5.483

7.  DOCK8 deficiency: clinical and immunological phenotype and treatment options - a review of 136 patients.

Authors:  Susanne E Aydin; Sara Sebnem Kilic; Caner Aytekin; Ashish Kumar; Oscar Porras; Leena Kainulainen; Larysa Kostyuchenko; Ferah Genel; Necil Kütükcüler; Neslihan Karaca; Luis Gonzalez-Granado; Jordan Abbott; Daifulah Al-Zahrani; Nima Rezaei; Zeina Baz; Jens Thiel; Stephan Ehl; László Marodi; Jordan S Orange; Julie Sawalle-Belohradsky; Sevgi Keles; Steven M Holland; Özden Sanal; Deniz C Ayvaz; Ilhan Tezcan; Hamoud Al-Mousa; Zobaida Alsum; Abbas Hawwari; Ayse Metin; Susanne Matthes-Martin; Manfred Hönig; Ansgar Schulz; Capucine Picard; Vincent Barlogis; Andrew Gennery; Marianne Ifversen; Joris van Montfrans; Taco Kuijpers; Robbert Bredius; Gregor Dückers; Waleed Al-Herz; Sung-Yun Pai; Raif Geha; Gundula Notheis; Carl-Philipp Schwarze; Betül Tavil; Fatih Azik; Kirsten Bienemann; Bodo Grimbacher; Valerie Heinz; H Bobby Gaspar; Roland Aydin; Beate Hagl; Benjamin Gathmann; Bernd H Belohradsky; Hans D Ochs; Talal Chatila; Ellen D Renner; Helen Su; Alexandra F Freeman; Karin Engelhardt; Michael H Albert
Journal:  J Clin Immunol       Date:  2015-01-28       Impact factor: 8.317

8.  Combined immunodeficiency associated with DOCK8 mutations.

Authors:  Qian Zhang; Jeremiah C Davis; Ian T Lamborn; Alexandra F Freeman; Huie Jing; Amanda J Favreau; Helen F Matthews; Joie Davis; Maria L Turner; Gulbu Uzel; Steven M Holland; Helen C Su
Journal:  N Engl J Med       Date:  2009-09-23       Impact factor: 91.245

9.  Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation.

Authors:  Theresa Cole; Mark S Pearce; Andrew J Cant; Catherine M Cale; David Goldblatt; Andrew R Gennery
Journal:  J Allergy Clin Immunol       Date:  2013-07-16       Impact factor: 10.793

10.  CNS vasculitis and stroke as a complication of DOCK8 deficiency: a case report.

Authors:  Suzan A AlKhater
Journal:  BMC Neurol       Date:  2016-04-26       Impact factor: 2.474

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  6 in total

Review 1.  An appraisal of the Wilson & Jungner criteria in the context of genomic-based newborn screening for inborn errors of immunity.

Authors:  Jovanka R King; Luigi D Notarangelo; Lennart Hammarström
Journal:  J Allergy Clin Immunol       Date:  2021-02       Impact factor: 10.793

2.  Venetoclax/decitabine for a pediatric patient with chronic myelomonocytic leukemia.

Authors:  John C Molina; Julie M Asare; Laura Tuschong; Robert R West; Katherine R Calvo; Rebecca Persky; Alison M Boyce; Dima A Hammoud; Steven M Holland; Dennis Hickstein; Nirali N Shah
Journal:  Pediatr Blood Cancer       Date:  2020-12-27       Impact factor: 3.838

Review 3.  Considerations for hematopoietic stem cell transplantation in primary immunodeficiency disorders.

Authors:  Tatyana Gavrilova
Journal:  World J Transplant       Date:  2019-07-31

4.  Delaying haematopoietic stem cell transplantation in children with viral respiratory infections reduces transplant-related mortality.

Authors:  Giorgio Ottaviano; Giovanna Lucchini; Judith Breuer; Juliana M Furtado-Silva; Arina Lazareva; Oana Ciocarlie; Reem Elfeky; Kanchan Rao; Persis J Amrolia; Paul Veys; Robert Chiesa
Journal:  Br J Haematol       Date:  2019-09-30       Impact factor: 6.998

5.  Towards European harmonisation of healthcare for patients with rare immune disorders: outcome from the ERN RITA registries survey.

Authors:  Riccardo Papa; Andrew Cant; Christoph Klein; Mark A Little; Nico M Wulffraat; Marco Gattorno; Nicolino Ruperto
Journal:  Orphanet J Rare Dis       Date:  2020-01-30       Impact factor: 4.123

Review 6.  Defining and targeting patterns of T cell dysfunction in inborn errors of immunity.

Authors:  Jose S Campos; Sarah E Henrickson
Journal:  Front Immunol       Date:  2022-09-14       Impact factor: 8.786

  6 in total

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