Доктор Robert Chiesa

Consultant in Bone Marrow Transplants and CAR-T cell therapy, BMT Specialty Lead
Dr Robert Chiesa

Обзор

Dr Robert Chiesa graduated in Medicine at the University of Milan, Italy and then he trained in paediatric haemato-oncology and stem cell transplant in Monza, where he worked on several research projects related to new drugs in relapsed ALL. 

After his specialisation in Paediatrics, he worked 2 years at the San Raffaele Institute in Milan, where he published in the field of stem cell transplant in thalassaemia and stem cell gene therapy in primary immunodeficiencies.

In 2009 he moved to the Bone Marrow Transplant Department at Great Ormond Street Hospital in London, where he works as consultant in Bone Marrow Transplant and CAR-T cell therapy. He has led on several clinical studies in malignant (B-ALL, T-ALL, AML) and non-malignant (chronic granulomatous disease, infantile osteopetrosis, X-linked adrenoleukodystrophy) diseases, as well as pharmacokinetics studies on Busulfan and Treosulfan. He runs the radio-immunotherapy (RIT) service with Yttrium-90 at GOSH, as part of the RIT phase 2 study. He leads on the stem cell transplant program in inborn errors of metabolism, and he has co-led the stem cell gene therapy trial in C-ALD. 

He is the clinical transplant lead on the allogeneic CAR-T cell therapy program in T-ALL and AML. He is the BMT Specialty Lead.

Владение языками:

  • Italian
  • English
  • Spanish

Квалификация и достижения

  • Radio-immunotherapy with Yttrium-90 labelled anti-CD66 monoclonal antibody prior to allogeneic haematopoietic stem cell transplant in children with leukaemia.
  • Allogeneic, base edited CD7 CAR-T cell therapy in T-ALL.
  • Allogeneic, base edited CD33 CAR-T cell therapy in AML.
  • Stem cell gene therapy in cerebral adrenoleukodystrophy.
  • Allogeneic haematopoietic stem cell transplant in inborn errors of metabolism. 
  • Allogeneic haematopoietic stem cell transplant in infantile osteopetrosis.
  • Pharmacokinetics of busulfan and treosulfan.
  • Treatment of transplant-associated thrombotic microangiopathy.
  • Radio-immunotherapy with Yttrium-90 labelled anti-CD66 monoclonal antibody prior to allogeneic haematopoietic stem cell transplant in children with leukaemia.
  • Allogeneic, base edited CD7 CAR-T cell therapy in T-ALL.
  • Allogeneic, base edited CD33 CAR-T cell therapy in AML.
  • Stem cell gene therapy in cerebral adrenoleukodystrophy.
  • Allogeneic haematopoietic stem cell transplant in inborn errors of metabolism. 
  • Allogeneic haematopoietic stem cell transplant in infantile osteopetrosis.
  • Pharmacokinetics of busulfan and treosulfan.
  • Treatment of transplant-associated thrombotic microangiopathy.
  • Specialisation in paediatrics with honour (UK CCST equivalent) 
  • Radio-immunotherapy with Yttrium-90 labelled anti-CD66 monoclonal antibody prior to allogeneic haematopoietic stem cell transplant in children with leukaemia.
  • Allogeneic, base edited CD7 CAR-T cell therapy in T-ALL.
  • Allogeneic, base edited CD33 CAR-T cell therapy in AML.
  • Stem cell gene therapy in cerebral adrenoleukodystrophy.
  • Allogeneic haematopoietic stem cell transplant in inborn errors of metabolism. 
  • Allogeneic haematopoietic stem cell transplant in infantile osteopetrosis.
  • Pharmacokinetics of busulfan and treosulfan.
  • Treatment of transplant-associated thrombotic microangiopathy.

Новости и публикации

Dr Chiesa has several publications in high impact journals, including the New England Journal of MedicineBlood and Nature Medicine.

1.    Arico M, Boccalatte MF, Silvestri D, Barisone E, Messina C, Chiesa R, Santoro N, Tamaro P, Lippi A, Gallisai D, Basso G, De Rossi G; Associazione Italiana di Ematologia ed Oncologia Pediatrica. Osteonecrosis: An emerging complication of intensive chemotherapy for childhood acute lymphoblastic leukemia. Haematologica. 2003 Jul;88(7):747-53.
2.    Rizzari C, Chiesa R., Citterio M., Coliva T., Vigano E., Conter V. Uso della PEG asparaginasi nell’età pediatrica. Haematologica. 2004 Oct; volume 89, supplemento n. 10.
3.    Masera G., Sala A., Locati M.G., Bonanomi S., Chiesa R., Marinoni M. Ematologia pediatrica e diritti fondamentali. Il progetto MASCOTA-MISPHO con l’America centrale e Latina. Assistenza infermieristica e ricerca, 2004, 23, 3.
4.    Rizzari C, Chiesa R, Locatelli F, Coliva T, Citterio M, Conter V. Late relapses in Childhood Acute Lymphoblastic Leukemia. Current Medical Literature Leukaemia & Lymphoma, Vol 12, number 2, 2005.
5.    Rizzari C, Citterio M, Zucchetti M, Conter V, Chiesa R, Colombini A, Malguzzi S, D’Incalci M. A pharmacological study on pegylated asparaginase used in front-line treatment of children with acute lymphoblastic leukemia. Haematologica. 2006 Jan; 91(1):24-31.
6.    Biral E, Chiesa R, Cappelli B, Roccia T, Frugnoli I, Noè A, Soliman C, Fiori R, Cursi L, Cattaneo F, Evangelio C, Miniero R, Ciceri F, Roncarolo MG, Marktel S. Multiple BM harvests in pediatric donors for thalassemic siblings: safety, efficacy and ethical issues. Bone Marrow Transplant. 2008 Sep;42(6):379-84
7.    Aiuti A, Brigida I, Ferrua F, Cappelli B, Chiesa R, Marktel S, Roncarolo MG. Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID.  Immunological Research 2009 Feb 18.
8.    Cappelli B, Chiesa R, Evangelio C, Biffi A, Roccia T, Frugnoli I, Biral E, Noè A, Fossati M, Finizio V, Miniero R, Napolitano S, Ferrua F, Soliman C, Ciceri F, Roncarolo MG, Marktel S. Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan. Br J Haematol. 2009 Nov;147(4):554-60.Frugnoli I, Cappelli B, Chiesa R, Biral E, Noè A, Evangelio C, Fossati M, Napolitano S, Ciceri F, Roncarolo MG, Marktel S. Escalating doses of donor lymphocytes for incipient graft rejection following SCT for thalassemia. Bone Marrow Transplant. 2009 Nov 2.
9.    Chiesa R, Cappelli B, Crocchiolo R, Frugnoli I, Biral E, Noè A, Evangelio C, Fossati M, Roccia T, Biffi A, Finizio V, Aiuti A, Broglia M, Bartoli A, Ciceri F, Roncarolo MG, Marktel S. Unpredictability of iv Busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplant for advanced beta thalassemia: Limited toxicity with a dose adjustment policy. Biol Blood Marrow Transplant. 2009 Dec 3.
10.    Marktel S, Napolitano S, Zino E, Cappelli B, Chiesa R, Poli F, Crocchiolo R, Ronchi P, Rossini S, Ciceri F, Roncarolo MG, Fleischhauer K. Platelet transfusion refractoriness in highly immunized beta thalassemia children undergoing stem cell transplantation. Pediatr Transplant. 2010 Jan 7.
11.    Trifari S, Scaramuzza S, Catucci M, Ponzoni M, Mollica L, Chiesa R, Cattaneo F, Lafouresse F, Caniglia M, Aiuti A, Villa A, Roncarolo MG, Dupré L. Revertant T lymphocytes in a Wiskott-Aldrich syndrome patient: analysis of function and distribution in lymphoid organs. The Journal of Allergy and Clinical Immunology, 2010 Feb; 125 (2): 439-448.
12.    Noè A, Cappelli B, Biffi A, Chiesa R, Frugnoli I, Biral E, Roncarolo MG, Ciceri F and Marktel S. High incidence of severe cyclosporine neurotoxicity in thalassemic children undergoing myeloablative hematopoietic stem cell transplantation: aggressive monitoring and prompt intervention ameliorates neurological outcome.  Italian Journal Pediatrics. 2010 Feb 6; 36 (1): 14.
13.    Fossati M, Cappelli B, Biral E, Chiesa R, Biffi A, Ossi C, Moro M, Cirillo DM, Clementi M, Soliman C, Ciceri F, Roncarolo MG, Fumagalli L, Marktel S. Fatal vancomycin and linezolid resistant Enterococcus faecium sepsis in a child undergoing allogeneic hematopoietic stem cell transplantation for beta thalassemia major. J Med Microbiol. 2010 Mar 18.
14.    Qasim W, Derniame S, Gilmour K, Chiesa R, Weber M, Adams S, Rao K, Amrolia P, Goulden N, Veys P, Gaspar H. Third-party virus-specific T cells eradicate adenoviraemia but trigger bystander graft-versus-host disease. Br J Haematol. 2011 Jul;154(1):150-3. 
15.    Worth A, Conyers R, Cohen J, Jagani M, Chiesa R, Rao K, Goulden N, Veys P, Amrolia PJ. Pre-emptive rituximab based on viraemia and T cell reconstitution: a highly effective strategy for the prevention of Epstein-Barr virus-associated lymphoproliferative disease following stem cell transplantation. Br J Haematol. 2011 Sep 13. 
16.    Pangrazio A, Cassani B, Guerrini MM, Crockett JC, Marrella V, Zammataro L, Strina D, Schulz A, Schlack C, Kornak U, Mellis DJ, Duthie A, Helfrich MH, Durandy A, Moshous D, Vellodi A, Chiesa R, Veys P, Iacono NL, Vezzoni P, Fischer A, Villa A, Sobacchi C. RANK-dependent autosomal recessive osteopetrosis: characterisation of 5 new cases with novel mutations. J Bone Mineral Research. 2011 Nov  9

17.    Chiesa R, Gilmour K, Qasim W, et al. Omission of in vivo T-cell depletion promotes rapid expansion of naïve CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant. British Journal Haematology.2012 January 9.
18.    Chiesa R, Veys P. Reduced intensity conditioning for allogeneic stem cell transplant in primary immune deficiencies. Expert Review Clinical Immunology. (2012). 2012 Mar, 8(3): 255-66.
19.    Sizzano F, Testi M, Zito L, Crocchiolo R, Troiano M, Mazzi B, Turchiano G, Torchio M, Pultrone C, Gregori S, Chiesa R, et al. Genotypes and haplotypes in the 3' untranslated region of the HLA-G gene and their association with clinical outcome of hematopoietic stem cell transplantation for beta-thalassemia. Tissue Antigens. 2012 May;79(5):326-32. 
20.    Bartelink IH, van Kesteren C, Boelens JJ, Egberts TC, Bierings MB, Cuvelier GD, Wynn RF, Slatter MA, Chiesa R, Danhof M, Knibbe CA. Predictive Performance of a Busulfan Pharmacokinetic Model in Children and Young Adults. Ther Drug Monit. 2012 Oct;34(5):574-583.
21.    Small TN, Qasim W, Friedrich W, Chiesa R, et al. Alternative donor SCT for the treatment of MHC Class II deficiency. Bone Marrow Transplant. 2012 Sep 24. 
22.    Zeilhofer U, Ashworth M, Amrolia P, Rao A, Chiesa R, et al. Pulmonary hypertension following haematopoietic stem cell transplantation for primary haemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2012 Nov 28. 
23.    Hiwarkar P, Gaspar HB, Gilmour K, Jagani M, Chiesa R, et al. Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients. Bone Marrow Transplant. 2012 Nov 26. 
24.    Worth AJ, Nikolajeva O, Chiesa R et al. Successful stem cell transplant with antibody-based conditioning for XIAP deficiency with refractory hemophagocytic lymphohistiocytosis. Blood. 2013 Jun 13;121(24):4966-8. 
25.    Jackson J, Titman P, Butler S, Bond K, Rao A, Veys P, Chiesa R, et al. Cognitive and psychosocial function post hematopoietic stem cell transplantation in children with hemophagocytic lymphohistiocytosis. Allergy Clin Immunol. 2013 Oct;132(4):889-895.e3. doi: 10.1016/j.jaci.2013.05.046. 
26.    CA Lindemans*, R Chiesa*, PJ Amrolia, et al. The impact of thymoglobulin prior to pediatric unrelated umbilical cord blood transplantation on immune-reconstitution and clinical outcome. Blood 2014 Jan 2; 123 (1): 126-32. *equal contribution.
27.    Hiwarkar P, Gajdosova E, Qasim W, Worth A, Breuer J, Chiesa R et al. Frequent occurrence of Cytomegalovirus retinitis during immune-reconstitution warrants regular ophthalmic screening in high-risk pediatric allogeneic hematopoietic stem cell transplant recipients. Clinical Infectious Diseases. 2014 Apr 3.
28.    Hassan A, Lee P, Maggina P, Xu JH, Moreira D, Slatter M, Nademi Z, Worth A, Adams S, Jones A, Cale C, Allwood Z, Rao K, Chiesa R, et al. Host natural killer immunity is a key indicator of permissiveness for donor cell engraftment in patients with severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Apr 30.


29.    Goodship THJ, Pinto F, Weston-Davies WH, Silva J, Nishimura JI, Nunn MA, Mackie I, Machin SJ, Palm L, Pryce JW, Chiesa R, et al. Use of the complement inhibitor Coversin to treat HSCT-associated TMA. Blood Adv. 2017 Jul 3;1(16):1254-1258. 
30.    Slatter MA, Rao K, Abd Hamid IJ, Nademi Z, Chiesa R, et al.Treosulfan and Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Children with Primary Immunodeficiency: UK Experience. Biol Blood Marrow Transplant. 2017.
31.    Shah RM, Elfeky R, Nademi Z, Qasim W, Amrolia P, Chiesa R, et al. T-cell receptor αβ+ and CD19+ cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency. J Allergy Clin Immunol. 2017 Aug 3. 
32.    Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Facchini M, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini C, Chiesa R, et al. Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy. Bone Marrow Transplantation. January 2018.
33.    Unni MNM, Elfeky R, Rao K, Nademi Z, Chiesa R et al. Non-PTLD Malignancy post HSCT in patients with Primary Immunodeficiency: UK experience. J Allergy Clin Immunol. 2018 Mar 7. 
34.    Elfeky RA, Furtado-Silva JM, Chiesa R et al. Umbilical cord blood transplantation without in vivo T-cell depletion for children with MHC class II deficiency. J Allergy Clin Immunol. 2018 Jan 20. 
35.    Ip W, Silva JMF, Gaspar H, Mitra A, Patel S, Rao K, Chiesa R, et al. Multicenter phase 1/2 application of adenovirus-specific T cells in high-risk pediatric patients after allogeneic stem cell transplantation. Cytotherapy. 2018 May 9. 
36.    Boonsathorn S, Cheng I, Kloprogge F, Alonso C, Lee C, Doncheva B, Booth J, ChiesaR, et al. Clinical Pharmacokinetics and Dose Recommendations for Posaconazole in Infants and Children. Clin Pharmacokinet. 2018 Apr 20. 
37.    M F Silva J, Ladomenou F, Carpenter B, Chandra S, Sedlacek P, Formankova R, Grandage V, Friswell M, Cant AJ, Nademi Z, Slatter MA, Gennery AR, Hambleton S, Flood TJ, Lucchini G, Chiesa R, et al. Allogeneic hematopoietic stem cell transplantation for severe, refractory juvenile idiopathic arthritis. Blood Adv. 2018 Apr 10;2(7):777-786. 
38.    Elfeky RA, Furtado-Silva JM, Chiesa R, et al. One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years. J Allergy Clin Immunol. 2018 Nov;142(5):1654-1656.e7. doi: 10.1016/j.jaci.2018.06.042. Epub 2018 Jul 25.
39.    Lucchini G, Marsh R, Gilmour K, Worth A, Nademi Z, Rao A, Booth C, Amrolia P, Silva J, Chiesa R, et al. Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis. Blood. 2018 Nov 8;132(19):2088-2096. doi: 10.1182/blood-2018-01-827485. 
40.    Gkazi AS, Margetts BK, Attenborough T, Mhaldien L, Standing JF, Oakes T, Heather JM, Booth J, Pasquet M, Chiesa R, et al. Clinical T Cell Receptor Repertoire Deep Sequencing and Analysis: An Application to Monitor Immune Reconstitution Following Cord Blood Transplantation. Front Immunol. 2018 Nov 5;9:2547. doi: 10.3389/fimmu.2018.02547. 
41.    Ottaviano G, Lucchini G, Breuer J, Furtado-Silva JM, Lazareva A, Ciocarlie O, Elfeky R, Rao K, Amrolia PJ, Veys P, Chiesa R. Delaying haematopoietic stem cell transplantation in children with viral respiratory infections reduces transplant-related mortality. Br J Haematol. 2019 Sep 30. doi: 10.1111/bjh.16216. 
42.    Ghorashian S, Kramer AM, Onuoha S, Wright G, Bartram J, Richardson R, Albon SJ, Casanovas-Company J, Castro F, Popova B, Villanueva K, Yeung J, Vetharoy W, Guvenel A, Wawrzyniecka PA, Mekkaoui L, Cheung GW, Pinner D, Chu J, Lucchini G, Silva J, Ciocarlie O, Lazareva A, Inglott S, Gilmour KC, Ahsan G, Ferrari M, Manzoor S, Champion K, Brooks T, Lopes A, Hackshaw A, Farzaneh F, Chiesa R, et al. Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL treated with a low-affinity CD19 CAR. Nat Med. 2019 Sep;25(9):1408-1414. doi: 10.1038/s41591-019-0549-5. 
43.    Elfeky R, Shah RM, Unni MNM, Ottaviano G, Rao K, Chiesa R, et al. New graft manipulation strategies improve the outcome of mismatched stem cell transplantation in children with primary immunodeficiencies. J Allergy Clin Immunol. 2019 Jul;144(1):280-293. 
44.    Ottaviano G, Chiesa R, Feuchtinger T, Vickers MA, Dickinson A, Gennery AR, Veys P, Todryk S. Adoptive T Cell Therapy Strategies for Viral Infections in Patients Receiving Haematopoietic Stem Cell Transplantation. Cells. 2019 Jan 14;8(1). 
45.    Chiesa R, Standing JF, Winter R, Nademi Z, Chu J, et al. Proposed therapeutic range of treosulfan in reduced toxicity pediatric allogeneic hematopoietic stem cell transplant conditioning: results from a prospective trial. Clin Pharmacol Ther. 2019 Nov 7. 
46.    Solans BP, Chiesa R, Doncheva B, Prunty H, Veys P, Trocóniz IF, Standing JF.Solans BP, et al. Modelling of neutrophil dynamics in children receiving busulfan or treosulfan for haematopoietic stem cell transplant conditioning.  Br J Clin Pharmacol. 2020 Feb 19;86(8):1537-49. doi: 10.1111/bcp.14260.
47.    Elfeky R, Lucchini G, Lum SH, Ottaviano G, Builes N, Nademi Z, Battersby A, Flood T, Owens S, Cant AJ, Young H, Greener S, Walsh P, Kavanagh D, Annavarapu S, Rao K, Amrolia P, Chiesa R, et al. New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT. Blood Adv. 2020 Jun 9;4(11):2418-2429. doi: 10.1182/bloodadvances.2019001315.
48.    Güngör T, Chiesa R. Cellular Therapies in Chronic Granulomatous Disease.  Front Pediatr. 2020 Jun 26;8:327. doi: 10.3389/fped.2020.00327. eCollection 2020.
49.    Chiesa R, Wang J, Blok HJ, Hazelaar S, et al. Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults.  Blood. 2020 Sep 3;136(10):1201-1211. doi: 10.1182/blood.2020005590.
50.    Gabelli M, Veys P, Chiesa R. Current status of umbilical cord blood transplantation in children. Br J Haematol. 2020 Sep;190(5):650-683. doi: 10.1111/bjh.16107. Epub 2019 Aug 13.
51.    Cordoba S, Onuoha S, Thomas S, Pignataro DS, Hough R, Ghorashian S, Vora A, Bonney D, Veys P, Rao K, Lucchini G, Chiesa R, Chu J, Clark L, Fung MM, Smith K, Peticone C, Al-Hajj M, Baldan V, Ferrari M, Srivastava S, Jha R, Arce Vargas F, Duffy K, Day W, Virgo P, Wheeler L, Hancock J, Farzaneh F, Domning S, Zhang Y, Khokhar NZ, Peddareddigari VGR, Wynn R, Pule M, Amrolia PJ. CAR T cells with dual targeting of CD19 and CD22 in pediatric and young adult patients with relapsed or refractory B cell acute lymphoblastic leukemia: a phase 1 trial. Nat Med. 2021 Oct;27(10):1797-1805. doi: 10.1038/s41591-021-01497-1. Epub 2021 Oct 12.
52.    Silva JMF, Jones A, Sibson K, Bibi S, Jeggo P, Woodbine L, Ahsan G, Gilmour KC, Rao K, Chiesa R, Lucchini G, Veys P, Worth A, Amrolia PJ. Haematopoietic Stem Cell Transplantation for DNA Ligase 1 Deficiency. J Clin Immunol. 2021 Jan;41(1):238-242. doi: 10.1007/s10875-020-00871-x. Epub 2020 Oct 6.
53.    Chiesa R, Bernardo ME. Haematopoietic stem cell gene therapy in inborn errors of metabolism. Br J Haematol. 2022 Jul;198(2):227-243. doi: 10.1111/bjh.18179. Epub 2022 May 10
54.    Mullanfiroze K, Lazareva A, Chu J, Williams L, Burridge S, Silva J, Chiesa R, Rao K, Lucchini G, Ghorashian S, O'Reilly M, Carpenter B, Grandage V, Hough R, Roddie C, Amrolia PJ. CD34+-selected stem cell boost can safely improve cytopenias following CAR T-cell therapy. Blood Adv. 2022 Aug 23;6(16):4715-4718. doi: 10.1182/bloodadvances.2022007572.
55.    Chiesa R, Boelens JJ, Duncan CN, Kühl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. .Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294.
56.    Albert MH, Slatter MA, Gennery AR, Güngör T, Bakunina K, Markovitch B, Hazelaar S, Sirait T, Courteille V, Aiuti A, Aleinikova OV, Balashov D, Bernardo ME, Bodova I, Bruno B, Cavazzana M, Chiesa R, Fischer A, Hauck F, Ifversen M, Kałwak K, Klein C, Kulagin A, Kupesiz A, Kuskonmaz B, Lindemans CA, Locatelli F, Lum SH, Maschan A, Meisel R, Moshous D, Porta F, Sauer MG, Sedlacek P, Schulz A, Suarez F, Vallée TC, Winiarski JH, Zecca M, Neven B, Veys P, Lankester AC. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis. Blood. 2022 Mar 31;139(13):2066-2079. doi: 10.1182/blood.2021014687.
57.    Chiesa R, Georgiadis C, Syed F, Zhan H, Etuk A, Gkazi SA, Preece R, Ottaviano G, Braybrook T, Chu J, Kubat A, Adams S, Thomas R, Gilmour K, O'Connor D, Vora A, Qasim W; Base-Edited CAR T Group. Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia. N Engl J Med. 2023 Jun 14. doi: 10.1056/NEJMoa2300709. 
58.    Ghorashian S, Lucchini G, Richardson R, Nguyen K, Terris C, Guvenel A, Oporto-Espuelas M, Yeung J, Pinner D, Chu J, Williams L, Ko KY, Walding C, Watts K, Inglott S, Thomas R, Connor C, Adams S, Gravett E, Gilmour K, Lal A, Kunaseelan S, Popova B, Lopes A, Ngai Y, Hackshaw A, Kokalaki E, Carulla MB, Mullanfiroze K, Lazareva A, Pavasovic V, Rao A, Bartram J, Vora A, Chiesa R, Silva J, Rao K, Bonney D, Wynn R, Pule M, Hough R, Amrolia PJ. CD19/CD22 targeting with cotransduced CAR T cells to prevent antigen-negative relapse after CAR T-cell therapy for B-cell ALL. Blood. 2024 Jan 11;143(2):118-123. doi: 10.1182/blood.2023020621.PMID: 37647647
59.    Gabelli M, Oporto-Espuelas M, Burridge S, Chu J, Farish S, Hedges E, Ware K, Williams L, Young L, Alajangi R, Ancliff P, Bartram J, Bonney D, Chenchara L, Chiesa R, Cugno C, Hodby K, Jalowiec KA, Lazareva A, Lucchini G, Mirci-Danicar OC, Mullanfiroze K, Pavasovic V, Rao A, Rao K, Riley L, Samarasinghe S, Shenton G, Silva J, Vora A, Hough R, Amrolia PJ, Ghorashian S.Maintenance therapy for early loss of B-cell aplasia after anti-CD19 CAR T-cell therapy. Blood Adv. 2024 Apr 23;8(8):1959-1963. doi: 0.1182/bloodadvances.2023011168.PMID: 37820111.
60.    Oporto Espuelas M, Burridge S, Kirkwood AA, Bonney D, Watts K, Shenton G, Jalowiec KA, O'Reilly MA, Roddie C, Castleton A, Clesham K, Nicholson E, Alajangi R, Prabhu S, George L, Uttenthal B, Gabelli M, Neill L, Besley C, Chaganti S, Wynn RF, Bartram J, Chiesa R, Lucchini G, Pavasovic V, Rao A, Rao K, Silva J, Samarasinghe S, Vora A, Clark P, Cummins M, Marks DI, Amrolia P, Hough R, Ghorashian S.Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme. Blood Cancer J. 2024 Apr 15;14(1):66. doi: 10.1038/s41408-024-01038-2.PMID: 38622139
61.    Bognàr T, Garcia-Rosa M, Lalmohamed A, Güngör T, Hauri-Hohl M, Prockop S, Oram L, Pai SY, Brooks J, Savic RM, Dvorak CC, Long-Boyle JR, Krajinovic M, Bittencourt H, Teyssier AC, Théorêt Y, Martinez C, Egberts TCG, Morales E, Slatter M, Cuvelier GDE, Chiesa R, Wynn RF, Coussons M, Cicalese MP, Ansari M, Long SE, Ebens CL, Lust H, Chaudhury S, Nath CE, Shaw PJ, Keogh SJ, van der Stoep MYEC, Bredius R, Lindemans CA, Boelens JJ, Bartelink IHAssociation of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity. Blood Adv. 2024 Oct 8;8(19):5137-5145. doi: 10.1182/bloodadvances.2024013275.PMID: 39074263
62.    Elfeky R, Builes N, Pearce RM, Kania SP, Nademi Z, Lucchini G, Chiesa R, Amrolia PJ, Sorror ML, Veys P, Rao K. Pediatric Adapted Risk index (PARI) to predict 2-year transplant related mortality post-HSCT in children.Blood Adv. 2024 Aug 2:bloodadvances.2024013484. doi: 10.1182/bloodadvances.2024013484. Online ahead of print.

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