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Article ; Online: HCT for CGD? Yes, and the sooner the better.

Morris, Emma C

2020 Volume 136, Issue 10, Page(s) 1121–1123

MeSH term(s)	Adult ; Child ; Granulomatous Disease, Chronic ; Hematopoietic Stem Cell Transplantation ; Humans ; NADPH Oxidases
Chemical Substances	NADPH Oxidases (EC 1.6.3.-)
Language	English
Publishing date	2020-09-11
Publishing country	United States
Document type	Journal Article ; Comment
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood.2020007891
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Zs.MO 364: Show issues

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Article ; Online: Engineered T cells Flt around their targets.

Morris, Emma C

Nature immunology

2020 Volume 21, Issue 8, Page(s) 831–832

MeSH term(s)	Dendritic Cells ; Epitopes ; Immunotherapy, Adoptive ; T-Lymphocytes ; Tomography, X-Ray Computed
Chemical Substances	Epitopes
Language	English
Publishing date	2020-07-09
Publishing country	United States
Document type	Journal Article ; Comment
ZDB-ID	2016987-5
ISSN	1529-2916 ; 1529-2908
ISSN (online)	1529-2916
ISSN	1529-2908
DOI	10.1038/s41590-020-0740-3
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Zs.A 5294: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
Zs.MG 42: Show issues

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Article ; Online: Allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency.

Morris, Emma C

Hematology. American Society of Hematology. Education Program

2020 Volume 2020, Issue 1, Page(s) 649–660

Abstract: With recent advances in genetic sequencing and its widespread adoption for clinical diagnostics, the identification of a primary immunodeficiency (PID) as the underlying cause of diseases presenting to hematologists including refractory autoimmunity, ... ...

Abstract	With recent advances in genetic sequencing and its widespread adoption for clinical diagnostics, the identification of a primary immunodeficiency (PID) as the underlying cause of diseases presenting to hematologists including refractory autoimmunity, cytopenias, immune dysregulation, and hematologic malignancy, is increasing, particularly in the adult population. Where the pathogenic genetic variants are restricted to the hematopoietic system, selected patients may benefit from allogeneic hematopoietic stem cell transplantation (allo-HSCT). Although it is generally accepted that early allo-HSCT (ie, in infancy or childhood) for PID is preferable, this is not always possible. The clinical phenotype of non-severe combined immune deficiency forms of PID can be very heterogeneous, in part because of the high number of genetic and functional defects affecting T, B, and natural killer cells, neutrophils, and/or antigen presentation. As a result, some patients have less severe disease manifestations in childhood and/or a later de novo presentation. For others, a delayed diagnosis, lack of a genetic diagnosis, or a previous lack of a suitable donor has precluded prior allo-HSCT. Specific issues which make transplantation for adult PID patients particularly challenging are discussed, including understanding the natural history of rare diseases and predicting outcome with conservative management alone; indications for and optimal timing of transplant; donor selection; conditioning regimens; and PID-specific transplant management. The role of gene therapy approaches as an alternative to allo-HSCT in high-risk monogenic PID is also discussed.
MeSH term(s)	Adult ; Donor Selection ; Female ; Genetic Therapy ; Hematopoietic Stem Cell Transplantation ; Humans ; Leukocytes/metabolism ; Male ; Primary Immunodeficiency Diseases/blood ; Primary Immunodeficiency Diseases/genetics ; Primary Immunodeficiency Diseases/therapy ; Transplantation, Homologous
Language	English
Publishing date	2020-12-04
Publishing country	United States
Document type	Case Reports ; Journal Article ; Review
ZDB-ID	2084287-9
ISSN	1520-4383 ; 1520-4391
ISSN (online)	1520-4383
ISSN	1520-4391
DOI	10.1182/hematology.2020000152
Database	MEDical Literature Analysis and Retrieval System OnLINE

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Article ; Online: Editing gene engineering to enhance function.

Morris, Emma C

Blood

2018 Volume 131, Issue 3, Page(s) 272–273

MeSH term(s)	Animals ; Clustered Regularly Interspaced Short Palindromic Repeats ; Gene Editing ; Genetic Engineering ; Receptors, Antigen, T-Cell ; T-Lymphocytes
Chemical Substances	Receptors, Antigen, T-Cell
Language	English
Publishing date	2018-01-18
Publishing country	United States
Document type	Journal Article ; Comment
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood-2017-11-816587
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Article ; Online: How I use allogeneic HSCT for adults with inborn errors of immunity.

Burns, Siobhan O / Morris, Emma C

Blood

2021 Volume 138, Issue 18, Page(s) 1666–1676

Abstract: Inborn errors of immunity (IEIs) are rare inherited disorders arising from monogenic germline mutations in genes that regulate the immune system. The majority of IEI are primary immunodeficiencies characterized by severe infection often associated with ... ...

Abstract	Inborn errors of immunity (IEIs) are rare inherited disorders arising from monogenic germline mutations in genes that regulate the immune system. The majority of IEI are primary immunodeficiencies characterized by severe infection often associated with autoimmunity, autoinflammation, and/or malignancy. Allogeneic hematopoietic stem cell transplant (HSCT) has been the corrective treatment of choice for many IEIs presenting with severe disease in early childhood, and experience has made this a successful and comparatively safe treatment in affected children. Early HSCT outcomes in adults were poor, resulting in extremely limited use worldwide. This is changing because of a combination of improved IEI diagnosis to inform patient selection, better understanding of the natural history of specific IEI, and improvements in transplant practice. Recently published HSCT outcomes for adults with IEIs have been comparable with pediatric data, making HSCT an important option for correction of clinically severe IEIs in adulthood. Here we discuss our practice for patient selection, timing of HSCT, donor selection and conditioning, peri- and post-HSCT management, and our approach to long-term follow-up. We stress the importance of multidisciplinary involvement in the complex decision-making process that we believe is required for successful outcomes in this rapidly emerging area.
MeSH term(s)	Adult ; Genetic Therapy ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Patient Selection ; Primary Immunodeficiency Diseases/genetics ; Primary Immunodeficiency Diseases/therapy ; Transplantation, Homologous/methods
Language	English
Publishing date	2021-06-02
Publishing country	United States
Document type	Journal Article
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood.2020008187
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Article: Allogeneic HSCT in Adolescents and Young Adults With Primary Immunodeficiencies.

Morris, Emma C / Albert, Michael H

Frontiers in pediatrics

2019 Volume 7, Page(s) 437

Abstract: Significant advances in hematopoietic transplantation over the past 20 years, have facilitated the safe transplantation of older adults with higher co-morbidities. In pediatric practice these advances have simultaneously improved outcomes for sicker ... ...

Abstract	Significant advances in hematopoietic transplantation over the past 20 years, have facilitated the safe transplantation of older adults with higher co-morbidities. In pediatric practice these advances have simultaneously improved outcomes for sicker children with complex, rare diseases including the primary immunodeficiencies, PID. With more widespread adoption of genetic sequencing, older patients with disease-causing mutations restricted to the hematopoietic system can be identified who may benefit from allogeneic hematopoietic stem cell transplantation (Allo-HSCT). Here we discuss the evidence for Allo-HSCT in adolescent and younger adults (AYAs) with PID.
Language	English
Publishing date	2019-10-24
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2711999-3
ISSN	2296-2360
ISSN	2296-2360
DOI	10.3389/fped.2019.00437
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Article ; Online: Cytokine release syndrome and associated neurotoxicity in cancer immunotherapy.

Morris, Emma C / Neelapu, Sattva S / Giavridis, Theodoros / Sadelain, Michel

Nature reviews. Immunology

2021 Volume 22, Issue 2, Page(s) 85–96

Abstract: A paradigm shift has recently occurred in the field of cancer therapeutics. Traditional anticancer agents, such as chemotherapy, radiotherapy and small-molecule drugs targeting specific signalling pathways, have been joined by cellular immunotherapies ... ...

Abstract	A paradigm shift has recently occurred in the field of cancer therapeutics. Traditional anticancer agents, such as chemotherapy, radiotherapy and small-molecule drugs targeting specific signalling pathways, have been joined by cellular immunotherapies based on T cell engineering. The rapid adoption of novel, patient-specific cellular therapies builds on scientific developments in tumour immunology, genetic engineering and cell manufacturing, best illustrated by the curative potential of chimeric antigen receptor (CAR) T cell therapy targeting CD19-expressing malignancies. However, the clinical benefit observed in many patients may come at a cost. In up to one-third of patients, significant toxicities occur that are directly associated with the induction of powerful immune effector responses. The most frequently observed immune-mediated toxicities are cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. This Review discusses our current understanding of their pathophysiology and clinical features, as well as the development of novel therapeutics for their prevention and/or management.
MeSH term(s)	Antigens, CD19 ; Cytokine Release Syndrome/etiology ; Humans ; Immunotherapy/adverse effects ; Immunotherapy, Adoptive/adverse effects ; Neoplasms/drug therapy ; Neurotoxicity Syndromes/drug therapy ; Neurotoxicity Syndromes/etiology ; Receptors, Antigen, T-Cell/genetics
Chemical Substances	Antigens, CD19 ; Receptors, Antigen, T-Cell
Language	English
Publishing date	2021-05-17
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Review
ZDB-ID	2062776-2
ISSN	1474-1741 ; 1474-1733
ISSN (online)	1474-1741
ISSN	1474-1733
DOI	10.1038/s41577-021-00547-6
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Zs.A 5565: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: T-cell receptor gene-modified cells: past promises, present methodologies and future challenges.

Tendeiro Rego, Rita / Morris, Emma C / Lowdell, Mark W

Cytotherapy

2019 Volume 21, Issue 3, Page(s) 341–357

Abstract: Immunotherapy constitutes an exciting and rapidly evolving field, and the demonstration that genetically modified T-cell receptors (TCRs) can be used to produce T-lymphocyte populations of desired specificity offers new opportunities for antigen-specific ...

Abstract	Immunotherapy constitutes an exciting and rapidly evolving field, and the demonstration that genetically modified T-cell receptors (TCRs) can be used to produce T-lymphocyte populations of desired specificity offers new opportunities for antigen-specific T-cell therapy. Overall, TCR-modified T cells have the ability to target a wide variety of self and non-self targets through the normal biology of a T cell. Although major histocompatibility complex (MHC)-restricted and dependent on co-receptors, genetically engineered TCRs still present a number of characteristics that ensure they are an important alternative strategy to chimeric antigen receptors (CARs), and high-affinity TCRs can now be successfully engineered with the potential to enhance therapeutic efficacy while minimizing adverse events. This review will focus on the main characteristics of TCR gene-modified cells, their potential clinical application and promise to the field of adoptive cell transfer (ACT), basic manufacturing procedures and characterization protocols and overall challenges that need to be overcome so that redirection of TCR specificity may be successfully translated into clinical practice, beyond early-phase clinical trials.
MeSH term(s)	Adoptive Transfer/methods ; Cell Survival ; Gene Editing/methods ; Genes, T-Cell Receptor/genetics ; Genetic Therapy/methods ; Genetic Vectors ; Humans ; Lentivirus/genetics ; Neoplasms/therapy ; Protein Engineering/methods ; Receptors, Antigen, T-Cell/genetics ; T-Lymphocytes/immunology ; Transduction, Genetic
Chemical Substances	Receptors, Antigen, T-Cell
Language	English
Publishing date	2019-01-14
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Review
ZDB-ID	2039821-9
ISSN	1477-2566 ; 1465-3249
ISSN (online)	1477-2566
ISSN	1465-3249
DOI	10.1016/j.jcyt.2018.12.002
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Article ; Online: Optimizing T-cell receptor gene therapy for hematologic malignancies.

Morris, Emma C / Stauss, Hans J

Blood

2016 Volume 127, Issue 26, Page(s) 3305–3311

Abstract: Recent advances in genetic engineering have enabled the delivery of clinical trials using patient T cells redirected to recognize tumor-associated antigens. The most dramatic results have been seen with T cells engineered to express a chimeric antigen ... ...

Abstract	Recent advances in genetic engineering have enabled the delivery of clinical trials using patient T cells redirected to recognize tumor-associated antigens. The most dramatic results have been seen with T cells engineered to express a chimeric antigen receptor (CAR) specific for CD19, a differentiation antigen expressed in B cells and B lineage malignancies. We propose that antigen expression in nonmalignant cells may contribute to the efficacy of T-cell therapy by maintaining effector function and promoting memory. Although CAR recognition is limited to cell surface structures, T-cell receptors (TCRs) can recognize intracellular proteins. This not only expands the range of tumor-associated self-antigens that are amenable for T-cell therapy, but also allows TCR targeting of the cancer mutagenome. We will highlight biological bottlenecks that potentially limit mutation-specific T-cell therapy and may require high-avidity TCRs that are capable of activating effector function when the concentrations of mutant peptides are low. Unexpectedly, modified TCRs with artificially high affinities function poorly in response to low concentration of cognate peptide but pose an increased safety risk as they may respond optimally to cross-reactive peptides. Recent gene-editing tools, such as transcription activator-like effector nucleases and clustered regularly interspaced short palindromic repeats, provide a platform to delete endogenous TCR and HLA genes, which removes alloreactivity and decreases immunogenicity of third-party T cells. This represents an important step toward generic off-the-shelf T-cell products that may be used in the future for the treatment of large numbers of patients.
MeSH term(s)	Adoptive Transfer/methods ; Animals ; Genetic Therapy/methods ; Hematologic Neoplasms/genetics ; Hematologic Neoplasms/therapy ; Humans ; Receptors, Antigen, T-Cell/genetics ; T-Lymphocytes/transplantation
Chemical Substances	Receptors, Antigen, T-Cell
Language	English
Publishing date	2016-05-20
Publishing country	United States
Document type	Journal Article ; Review ; Research Support, Non-U.S. Gov't
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood-2015-11-629071
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Article ; Online: Quality of Life and Social and Psychological Outcomes in Adulthood Following Allogeneic HSCT in Childhood for Inborn Errors of Immunity

Nicholson, Bethany / Goodman, Rupert / Day, James / Worth, Austen / Carpenter, Ben / Sandford, Kit / Morris, Emma C. / Burns, Siobhan O. / Ridout, Deborah / Titman, Penny / Campbell, Mari

J Clin Immunol. 2022 Oct., v. 42, no. 7, p. 1451-1460

2022 , Page(s) 1451–1460

Abstract: BACKGROUND: Hematopoietic stem cell transplant (HSCT) is well established as a corrective treatment for many inborn errors of immunity (IEIs) presenting in childhood. Due to improved techniques, more transplants are undertaken and patients are living ... ...

Abstract	BACKGROUND: Hematopoietic stem cell transplant (HSCT) is well established as a corrective treatment for many inborn errors of immunity (IEIs) presenting in childhood. Due to improved techniques, more transplants are undertaken and patients are living longer. However, long-term complications can significantly affect future health and quality of life. Previous research has focused on short-term medical outcomes and little is known about health or psychosocial outcomes in adulthood. OBJECTIVE: This project aimed to ascertain the long-term social and psychological outcomes for adults who underwent HSCT for IEI during childhood. METHODS: Adult patients, who had all undergone HSCT for IEI during childhood at two specialist immunology services at least 5 years previously, were invited to participate in the study. Questionnaires and practical tasks assessed their current functioning and circumstances. Information was also gathered from medical notes. Data was compared with population norms and a control group of participant-nominated siblings or friends. RESULTS: Eighty-three patients and 46 matched controls participated in the study. Patients reported significantly better physical health-related quality of life than the general population norm, but significantly worse than matched controls. Patient’s self-reported physical health status and the perceived impact of their physical health on everyday life were worse than matched controls and patients reported higher levels of anxiety and lower mood than the general population. For those where their IEI diagnosis was not associated with a learning disability, cognitive function was generally within the normal range. CONCLUSIONS: Patients who have had a HSCT in childhood report mixed psychosocial outcomes in adulthood. More research is needed to establish screening protocols and targeted interventions to maximize holistic outcomes. CLINICAL IMPLICATIONS: Screening for holistic needs and common mental health difficulties should be part of routine follow-up. Information should be provided to patients and families in order to support decision-making regarding progression to transplant and the early identification of any difficulties.
Keywords	adulthood ; adults ; anxiety ; childhood ; cognition ; decision making ; health status ; hematopoietic stem cells ; immunity ; mental health ; patients ; physical health ; quality of life
Language	English
Dates of publication	2022-10
Size	p. 1451-1460
Publishing place	Springer US
Document type	Article ; Online
ZDB-ID	779361-3
ISSN	1573-2592 ; 0271-9142
ISSN (online)	1573-2592
ISSN	0271-9142
DOI	10.1007/s10875-022-01286-6
Database	NAL-Catalogue (AGRICOLA)

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