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Novel Insights into Hematopoietic Cell Transplantation and Cellular Therapies

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A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Immunology".

Deadline for manuscript submissions: closed (31 October 2022) | Viewed by 7733

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Special Issue Editors

Dr. Ioanna Sakellari

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Guest Editor

George Papanicolaou General Hospital, Thessaloniki, Greece
Interests: hematopoietic cell transplantation; cellular therapy; gene therapy; CAR-T; allogeneic; autologous; graft-versus-host disease; toxicity
Special Issues, Collections and Topics in MDPI journals

Dr. Eleni Gavriilaki

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Guest Editor

Second Propedeutic Department of Internal Medicine, Hippocration Hospital, Aristotle University of Thessaloniki, 54642 Thessaloniki, Greece
Interests: hematologic malignancies; thrombosis; complement; cellular therapy; lymphoma; myeloma; COVID-19
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Cellular therapies are the cornerstone of state-of-the-art treatment in hematologic malignant and non-malignant diseases. Although novel cellular therapies such as CAR (Chimeric Antigen Receptor)-T cells and gene therapy, have revolutionized the field, autologous and allogeneic hematopoietic cell transplantation remain key modalities in treatment algorithms.

Over the last few years, novel techniques in established modalities, such as alternative donors, reduced intensity or toxicity regimens, prophylaxis and treatment for graft-versus-host-disease, have shown significant improvements. Nevertheless, novel biomarkers, early predictors and treatments of life-threatening complications such as endothelial injury syndrome, as well as novel biologic agents for graft-versus-host-disease remain under investigation.

In parallel, novel cellular therapies have introduced novel toxicities, such as the cytokine release syndrome and neurotoxicity, with an emerging clinical need for early prediction and treatment. Additional efforts are made to improve their efficacy and expand their indications.

Therefore, this Special Issue aims to cover novel insights into both established and novel cellular therapies focusing on molecular advances.

Dr. Ioanna Sakellari
Dr. Eleni Gavriilaki
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

hematopoietic cell transplantation
cellular therapy
gene therapy
CAR-T
allogeneic
autologous
graft-versus-host disease
toxicity

Published Papers (3 papers)

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Research

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13 pages, 934 KiB

Open AccessArticle

Increased Intestinal Permeability and Stool Zonulin, Calprotectin and Beta-Defensin-2 Concentrations in Allogenic Hematopoietic Cell Transplantation Recipients

by Martyna Tyszka, Dominika Maciejewska-Markiewicz, Jarosław Biliński, Arkadiusz Lubas, Ewa Stachowska and Grzegorz W. Basak

Int. J. Mol. Sci. 2022, 23(24), 15962; https://doi.org/10.3390/ijms232415962 - 15 Dec 2022

Cited by 4 | Viewed by 1564

Abstract

Significant progress has been made in understanding the connection between intestinal barrier function and allogenic hematopoietic cell transplantation (allo-HCT) recipients’ outcomes. The purpose of this study was to further evaluate gut barrier permeability and other potential intestinal barrier disruption markers in the allo-HCT setting. Fifty-one patients were enrolled in the study. Intestinal permeability was assessed with the sugar absorption test and faecal concentrations of the zonulin, calprotectin and beta-defensin-2 levels in the peri-transplantation period. Most patients undergoing allo-HCT in our department had a disrupted intestinal barrier at the baseline, which was associated with older age and higher Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI). Regardless of this, we observed a further increase in gut barrier permeability after allo-HCT in most patients. However, there was no association between permeability assay and other markers (zonulin, calprotectin and beta-defensin-2). Patients with acute GVHD had significantly higher median calprotectin concentrations after allo-HCT compared with the patients without this complication. Our findings indicate that gut barrier damage develops prior to allo-HCT with progression after the procedure and precedes further complications, but did not prove other markers to be useful surrogates of intestinal permeability. Full article

(This article belongs to the Special Issue Novel Insights into Hematopoietic Cell Transplantation and Cellular Therapies)

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15 pages, 1702 KiB

Open AccessArticle

Anti-Thymocyte Globulin (ATG)-Free Nonmyeloablative Haploidentical PBSCT Plus Post-Transplantation Cyclophosphamide Is a Safe and Efficient Treatment Approach for Pediatric Acquired Aplastic Anemia

by Rong-Long Chen, Peng Peng Ip, Jy-juinn Shaw, Yun-Hsin Wang, Li-Hua Fan, Yi-Ling Shen, Nithila A. Joseph, Tsen-Erh Chen and Liuh-Yow Chen

Int. J. Mol. Sci. 2022, 23(23), 15192; https://doi.org/10.3390/ijms232315192 - 2 Dec 2022

Viewed by 3446

Abstract

Most cases of acquired aplastic anemia (AA) arise from autoimmune destruction of hematopoietic stem and progenitor cells. Human leukocyte antigen (HLA)-haploidentical nonmyeloablative hematopoietic stem cell transplantation (HSCT) plus post-transplantation cyclophosphamide (PTCy) is increasingly applied to salvage AA using bone marrow as graft and anti-thymocyte globulin (ATG) in conditioning. Herein, we characterize a cohort of twelve AA patients clinically and molecularly, six who possessed other immunological disorders (including two also carrying germline SAMD9L mutations). Each patient with SAMD9L mutation also carried an AA-related rare BCORL1 variant or CTLA4 p.T17A GG genotype, respectively, and both presented short telomere lengths. Six of the ten patients analyzed harbored AA-risky HLA polymorphisms. All patients recovered upon non-HSCT (n = 4) or HSCT (n = 8) treatments. Six of the eight HSCT-treated patients were subjected to a modified PTCy-based regimen involving freshly prepared peripheral blood stem cells (PBSC) as graft and exclusion of ATG. All patients were engrafted between post-transplantation days +13 and +18 and quickly reverted to normal life, displaying a sustained complete hematologic response and an absence of graft-versus-host disease. These outcomes indicate most AA cases, including of the SAMD9L-inherited subtype, are immune-mediated and the modified PTCy-based regimen we present is efficient and safe for salvage. Full article

(This article belongs to the Special Issue Novel Insights into Hematopoietic Cell Transplantation and Cellular Therapies)

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Review

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17 pages, 723 KiB

Open AccessReview

Molecular Advances in Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease

by Ioulia Mavrikou, Dimitrios Chatzidimitriou, Lemonia Skoura, Emmanouil Nikolousis, Ioanna Sakellari and Eleni Gavriilaki

Int. J. Mol. Sci. 2023, 24(6), 5620; https://doi.org/10.3390/ijms24065620 - 15 Mar 2023

Cited by 1 | Viewed by 2194

Abstract

Sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) detected in the liver has been considered a severe complication of hematopoietic stem cell transplantation (HSCT). SOS/VOD is characterized by hepatomegaly, right upper quadrant pain, jaundice, and ascites. The severe forms of the disease may result in multi-organ dysfunction (MOD) with a high mortality rate (>80%). The development of SOS/VOD can be rapid and unpredictable. Therefore, early identification and severity assessment is crucial in facilitating prompt diagnosis and timely treatment. Effective treatment and potential prophylaxis with defibrotide highlight the need for characterizing a sub-group of patients at high risk for SOS/VOD. Moreover, antibodies that are conjugated with calicheamicin, gemtuzumab, and inotuzumab ozogamicin, have led to renewed interest in this syndrome. Evaluation and management of serious adverse events associated with gemtuzumab and inotuzumab ozogamicin are recommended. We review hepatic-, transplant- and patient-related risk factors, criteria for diagnosis and grading classification, and SOS/VOD potential biomarkers. Furthermore, we examine pathogenesis, clinical presentation, diagnostic criteria, risk factors, prophylaxis, and treatment of SOS/VOD occurring post HSCT. Moreover, we aim to provide an up-to-date summary of molecular advances in the diagnosis and management of SOS/VOD. We performed a comprehensive review of the literature and examined the recently available data, mostly using the PubMed and Medline search engines for original articles published over the last decade. In the era of precision medicine, our review provides up-to-date knowledge of genetic or sera markers for SOS/VOD with the goal of identifying a subset of high-risk patients. Full article

(This article belongs to the Special Issue Novel Insights into Hematopoietic Cell Transplantation and Cellular Therapies)

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