Biblio

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2022
Albert MH, Slatter MA, Gennery AR, Güngör T, Bakunina K, Markovitch B, Hazelaar S, Sirait T, Courteille V, Aiuti A, et al. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis. Blood. 2022.
Schuetz C, Gerke J, Ege MJohannes, Walter JEszter, Kusters M, Worth AJJ, Kanakry JA, Dimitrova D, Wolska-Kusnierz B, Chen K, et al. Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCT. Blood. 2022.
Ottaviano G, Georgiadis C, Gkazi SAthina, Syed F, Zhan H, Etuk A, Preece R, Chu J, Kubat A, Adams S, et al. Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia. Sci Transl Med. 2022;14(668):eabq3010.
2021
Ottaviano G, Achini-Gutzwiller F, Kalwak K, Lanino E, Faraci M, Rao K, Chiesa R, Amrolia P, Bonanomi S, Rovelli A, et al. Impact of in vivo lymphodepletion on outcome in children with non-malignant disorders receiving peripheral blood stem cell transplantation. Transplant Cell Ther. 2021.
2020
Cesaro S, Pillon M, Sauer M, Smiers F, Faraci M, de Heredia CDíaz, Wynn R, Greil J, Locatelli F, Veys P, et al. Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Tra. Bone Marrow Transplant. 2020.
Willasch AManfred, Peters C, Sedlacek P, Dalle J-H, Kitra-Roussou V, Yeşilipek A, Wachowiak J, Lankester A, Prete A, Hamidieh AAli, et al. Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?-A multicenter EBMT-PDWP study. Bone Marrow Transplant. 2020.
Elfeky R, Lucchini G, Lum S-H, Ottaviano G, Builes N, Nademi Z, Battersby A, Flood T, Owens S, Cant AJ, et al. New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT. Blood Adv. 2020;4(11):2418-2429.
Lum SHan, Elfeky R, Achini FR, Margarit-Soler A, Cinicola B, Perez-Heras I, Nademi Z, Flood T, Cheetham T, Worth A, et al. Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency. J Clin Immunol. 2020.
Felber M, Steward CG, Kentouche K, Fasth A, Wynn RF, Zeilhofer U, Haunerdinger V, Volkmer B, Prader S, Gruhn B, et al. Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis. Blood Adv. 2020;4(9):1998-2010.
2019
Ferrua F, Galimberti S, Courteille V, Slatter MAnne, Booth C, Moshous D, Neven B, Blanche S, Laberko A, Shcherbina A, et al. Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-SCETIDE-PIDTC Study. J Allergy Clin Immunol. 2019.
Elfeky R, Lazareva A, Qasim W, Veys P. Immune reconstitution following hematopoietic stem cell transplantation using different stem cell sources. Expert Rev Clin Immunol. 2019:1-17.
Elfeky R, Shah RM, Unni MNm, Ottaviano G, Rao K, Chiesa R, Amrolia P, Worth A, Flood T, Abinun M, et al. New graft manipulation strategies improved outcome of mismatched stem cell transplantation in children with primary immunodeficiencies. J Allergy Clin Immunol. 2019.
2016
Chiesa R, Wynn RF, Veys P. Haematopoietic stem cell transplantation in inborn errors of metabolism. Curr Opin Hematol. 2016.
Al-Herz W, Chu JI, van der Spek J, Raghupathy R, Massaad MJ, Keles S, Biggs CM, Cockerton L, Chou J, Dbaibo G, et al. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2016.
2015
Boztug H, Sykora K-W, Slatter M, Zecca M, Veys P, Lankester A, Cant A, Skinner R, Wachowiak J, Glogova E, et al. European society for blood and marrow transplantation analysis of treosulfan conditioning before hematopoietic stem cell transplantation in children and adolescents with hematological malignancies. Pediatr Blood Cancer. 2015.
Panasiuk A, Nussey S, Veys P, Amrolia P, Rao K, Krawczuk-Rybak M, Leiper A. Gonadal function and fertility after stem cell transplantation in childhood: comparison of a reduced intensity conditioning regimen containing melphalan with a myeloablative regimen containing busulfan. Br J Haematol. 2015.
Lucchini G, Perales M-A, Veys P. Immune reconstitution after cord blood transplantation: peculiarities, clinical implications and management strategies. Cytotherapy. 2015;17(6):711-722.
Petrof G, Lwin SM, Martinez-Queipo M, Abdul-Wahab A, Tso S, Mellerio JE, Slaper-Cortenbach I, Boelens JJ, Tolar J, Veys P, et al. Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children With Recessive Dystrophic Epidermolysis Bullosa. J Invest Dermatol. 2015.
2014
Lee F, Luevano M, Veys P, Yong K, Madrigal A, Shaw BE, Saudemont A. The Effects of CAMPATH-1H on Cell Viability Do Not Correlate to the CD52 Density on the Cell Surface. PLoS One. 2014;9(7):e103254.
Hiwarkar P, Gajdosova E, Qasim W, Worth A, Breuer J, Chiesa R, Ridout D, Edelsten C, Moore A, Amrolia P, et al. Frequent occurrence of Cytomegalovirus retinitis during immune-reconstitution warrants regular ophthalmic screening in high-risk pediatric allogeneic hematopoietic stem cell transplant recipients. Clin Infect Dis. 2014.
2013
Zhan H, Gilmour K, Chan L, Farzaneh F, McNicol AMarie, Xu J-H, Adams S, Fehse B, Veys P, Thrasher A, et al. Production and First-in-Man Use of T Cells Engineered to Express a HSVTK-CD34 Sort-Suicide Gene. PLoS One. 2013;8(10):e77106.
Worth AJJ, Nikolajeva O, Chiesa R, Rao K, Veys P, Amrolia PJ. Successful stem cell transplant with antibody-based conditioning for XIAP deficiency with refractory hemophagocytic lymphohistiocytosis. Blood. 2013;121(24):4966-8.