Hematopoietic Stem Cell Transplantation Beyond Severe Combined Immunodeficiency: Seeking a Cure for Primary Immunodeficiency

被引：3

作者：

Mitchell, Richard ^{[1
,2
]}

机构：

[1] Sydney Childrens Hosp, Kids Canc Ctr, Level 1,High St, Randwick, NSW 2031, Australia

[2] Univ New South Wales, Sch Women & Childrens Hlth, Sydney, NSW, Australia

来源：

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE | 2019年 / 7卷 / 03期

关键词：

Primary immunodeficiency; Hematopoietic stem cell transplantation; Reduced intensity conditioning; CHRONIC GRANULOMATOUS-DISEASE; CORD BLOOD TRANSPLANTATION; WISKOTT-ALDRICH-SYNDROME; SINGLE-CENTER EXPERIENCE; HYPER-IGM SYNDROME; SEVERE CONGENITAL NEUTROPENIA; NERVOUS-SYSTEM INVOLVEMENT; DAILY INTRAVENOUS BUSULFAN; CD40 LIGAND DEFICIENCY; HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS;

D O I：

10.1016/j.jaip.2018.12.011

中图分类号：

R392 [医学免疫学];

学科分类号：

100102 ;

摘要：

Hematopoietic stem cell transplantation (HSCT) can provide definitive therapy for patients with primary immunodeficiency disease (PIDD). Modern HSCT techniques and supportive care have significantly improved outcomes for patients with PIDD. This review examines current HSCT practice for PIDD other than severe combined immunodeficiency, and explores indications, risks, and long-term outcomes for this group of challenging diseases. Crown Copyright (C) 2018 Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology

引用

页码：776 / 785

页数：10

共 123 条

[91] 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 [J].

Rao, Kanchan ;

Adams, Stuart ;

Qasim, Waseem ;

Allwood, Zoe ;

Worth, Austen ;

Silva, Juliana ;

Lucchini, Giovanna ;

Chiesa, Robert ;

Veys, Paul ;

Amrolia, Persis .

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2016, 138 (04) :1152-1160

[92] Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure [J].

Richardson, Paul G. ;

Riches, Marcie L. ;

Kernan, Nancy A. ;

Brochstein, Joel A. ;

Mineishi, Shin ;

Termuhlen, Amanda M. ;

Arai, Sally ;

Grupp, Stephan A. ;

Guinan, Eva C. ;

Martin, Paul L. ;

Steinbach, Gideon ;

Krishnan, Amrita ;

Nemecek, Eneida R. ;

Giralt, Sergio ;

Rodriguez, Tulio ;

Duerst, Reggie ;

Doyle, John ;

Antin, Joseph H. ;

Smith, Angela ;

Lehmann, Leslie ;

Champlin, Richard ;

Gillio, Alfred ;

Bajwa, Rajinder ;

D'Agostino, Ralph B., Sr. ;

Massaro, Joseph ;

Warren, Diane ;

Miloslavsky, Maja ;

Hume, Robin L. ;

Iacobelli, Massimo ;

Nejadnik, Bijan ;

Hannah, Alison L. ;

Soiffer, Robert J. .

BLOOD, 2016, 127 (13) :1656-1665

[93] Outcome of allogeneic stem cell transplantation in adults with common variable immunodeficiency [J].

Rizzi, Marta ;

Neumann, Carla ;

Fielding, Adele K. ;

Marks, Reinhard ;

Goldacker, Sigune ;

Thaventhiran, James ;

Tarzi, Michael D. ;

Schlesier, Michael ;

Salzer, Ulrich ;

Eibel, Hermann ;

Warnatz, Klaus ;

Finke, Juergen ;

Grimbacher, Bodo ;

Peter, Hans-Hartmut .

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2011, 128 (06) :1371-U783

[94] Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy [J].

Rosenberg, Philip S. ;

Zeidler, Cornelia ;

Bolyard, Audrey A. ;

Alter, Blanche P. ;

Bonilla, Mary A. ;

Boxer, Laurence A. ;

Dror, Yigal ;

Kinsey, Sally ;

Link, Daniel C. ;

Newburger, Peter E. ;

Shimamura, Akiko ;

Welte, Karl ;

Dale, David C. .

BRITISH JOURNAL OF HAEMATOLOGY, 2010, 150 (02) :196-199

[95] Inflammatory manifestations in chronic granulomatous disease (CGD) [J].

Rosenzweig, S. D. .

JOURNAL OF CLINICAL IMMUNOLOGY, 2008, 28 (Suppl 1) :S67-S72

[96] Cardiotoxicity and cardiomyopathy in children and young adult survivors of hematopoietic stem cell transplant [J].

Rotz, Seth J. ;

Ryan, Thomas D. ;

Hlavaty, Joel ;

George, Stephen A. ;

El-Bietar, Javier ;

Dandoy, Christopher E. .

PEDIATRIC BLOOD & CANCER, 2017, 64 (11)

[97] Clinical similarities and differences of patients with X-linked lymphoproliferative syndrome type 1 (XLP-1/SAP deficiency) versus type 2 (XLP-2/XIAP deficiency) [J].

Schmid, Jana Pachlopnik ;

Canioni, Danielle ;

Moshous, Despina ;

Touzot, Fabien ;

Mahlaoui, Nizar ;

Hauck, Fabian ;

Kanegane, Hirokazu ;

Lopez-Granados, Eduardo ;

Mejstrikova, Ester ;

Pellier, Isabelle ;

Galicier, Lionel ;

Galambrun, Claire ;

Barlogis, Vincent ;

Bordigoni, Pierre ;

Fourmaintraux, Alain ;

Hamidou, Mohamed ;

Dabadie, Alain ;

Le Deist, Francoise ;

Haerynck, Filomeen ;

Ouachee-Chardin, Marie ;

Rohrlich, Pierre ;

Stephan, Jean-Louis ;

Lenoir, Christelle ;

Rigaud, Stephanie ;

Lambert, Nathalie ;

Milili, Michele ;

Schiff, Claudin ;

Chapel, Helen ;

Picard, Capucine ;

de Saint Basile, Genevieve ;

Blanche, Stephane ;

Fischer, Alain ;

Latour, Sylvain .

BLOOD, 2011, 117 (05) :1522-1529

[98] Guideline for Primary Antifungal Prophylaxis for Pediatric Patients With Cancer or Hematopoietic Stem Cell Transplant Recipients [J].

Science, Michelle ;

Robinson, Paula D. ;

MacDonald, Tamara ;

Rassekh, Shahrad Rod ;

Dupuis, L. Lee ;

Sung, Lillian .

PEDIATRIC BLOOD & CANCER, 2014, 61 (03) :393-400

[99] Genetic, biochemical, and clinical features of chronic granulomatous disease [J].

Segal, BH ;

Leto, TL ;

Gallin, JI ;

Malech, HL ;

Holland, SM .

MEDICINE, 2000, 79 (03) :170-200

[100] Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft:: a survey of the European experience, 1985-2000 [J].

Seger, RA ;

Gungor, T ;

Belohradsky, BH ;

Blanche, S ;

Bordigoni, P ;

Di Bartolomeo, P ;

Flood, T ;

Landais, P ;

Müller, S ;

Ozsahin, H ;

Passwell, JH ;

Porta, F ;

Slavin, S ;

Wulffraat, N ;

Zintl, F ;

Nagler, A ;

Cant, A ;

Fischer, A .

BLOOD, 2002, 100 (13) :4344-4350

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