Prognostic and Diagnostic Testing in Onco-Haematological Diseases in the Era of Novel Therapies: Current Updates and Future Perspectives

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: 30 June 2024 | Viewed by 1813

Special Issue Editor


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Guest Editor
Hematology Department, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy
Interests: lymphoma; molecular diagnostics; translational research; immunotherapies

Special Issue Information

Dear Colleagues,

The last decade has been characterized by a progressive shift in the treatment of onco-hematological malignancies from conventional radio- and chemotherapy to innovative and more personalized therapies, including those based on chimeric antigen receptor (CAR) T cells, bispecific T-cell engagers (BiTEs), and antibody–drug conjugates (ADCs). As a result, many patients achieve responses, including complete radiographical or pathological remission, thus implying that sensitive techniques need to be implemented to monitor minimal residual disease (MRD). Additionally, there is a need to assess whether the prognostic biomarkers and risk scoring systems that have been validated mainly in the chemo-immunotherapy era retain significance in the context of innovative and more personalized therapies.

For this to be conceivable, novel diagnostic and prognostic testing modalities have been developed mainly based on next-generation sequencing, digital droplet PCR, mass cytometry, and digital imaging to study both the tumor and the tumor microenvironment. Considerable data indicate, in fact, that the immune landscape of hematological malignancies defined through the integration of genomic, epigenomic, and transcriptomic data influence anti-tumor immune responses and patient outcomes.

Thus, the aim of this Special Issue is to provide a comprehensive overview of newly developed prognostic and diagnostic approaches and of their use in onco-hematology in the era of novel agents. Original research articles as well as reviews on these topics are welcomed.

Dr. Cristiana Carniti
Guest Editor

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Published Papers (1 paper)

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23 pages, 1131 KiB  
Review
Pediatric Bone Marrow Failure: A Broad Landscape in Need of Personalized Management
by Lotte T. W. Vissers, Mirjam van der Burg, Arjan C. Lankester, Frans J. W. Smiers, Marije Bartels and Alexander B. Mohseny
J. Clin. Med. 2023, 12(22), 7185; https://doi.org/10.3390/jcm12227185 - 20 Nov 2023
Viewed by 1526
Abstract
Irreversible severe bone marrow failure (BMF) is a life-threatening condition in pediatric patients. Most important causes are inherited bone marrow failure syndromes (IBMFSs) and (pre)malignant diseases, such as myelodysplastic syndrome (MDS) and (idiopathic) aplastic anemia (AA). Timely treatment is essential to prevent infections [...] Read more.
Irreversible severe bone marrow failure (BMF) is a life-threatening condition in pediatric patients. Most important causes are inherited bone marrow failure syndromes (IBMFSs) and (pre)malignant diseases, such as myelodysplastic syndrome (MDS) and (idiopathic) aplastic anemia (AA). Timely treatment is essential to prevent infections and bleeding complications and increase overall survival (OS). Allogeneic hematopoietic stem cell transplantation (HSCT) provides a cure for most types of BMF but cannot restore non-hematological defects. When using a matched sibling donor (MSD) or a matched unrelated donor (MUD), the OS after HSCT ranges between 60 and 90%. Due to the introduction of post-transplantation cyclophosphamide (PT-Cy) to prevent graft versus host disease (GVHD), alternative donor HSCT can reach similar survival rates. Although HSCT can restore ineffective hematopoiesis, it is not always used as a first-line therapy due to the severe risks associated with HSCT. Therefore, depending on the underlying cause, other treatment options might be preferred. Finally, for IBMFSs with an identified genetic etiology, gene therapy might provide a novel treatment strategy as it could bypass certain limitations of HSCT. However, gene therapy for most IBMFSs is still in its infancy. This review summarizes current clinical practices for pediatric BMF, including HSCT as well as other disease-specific treatment options. Full article
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