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New Developments in the Field of Myeloid Neoplasms
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New Developments in the Field of Myeloid Neoplasms

A special issue of Journal of Clinical Medicine (ISSN 2077-0383).

Deadline for manuscript submissions: closed (31 March 2021) | Viewed by 12033

Special Issue Editor

Dr. Francesco Mannelli

E-Mail Website
Guest Editor
Centro Ricerca e Innovazione Malattie Mieloproliferative (CRIMM), AOU Careggi, Largo Brambilla 3, 50134 Firenze, Italy
Interests: acute myeloid leukemia; minimal residual disease; peripheral blast clearance; immunophenotyping; myeloproliferative neoplasms; systemic mastocytosis

Special Issue Information

Dear Colleagues,

The major aim of this Special Issue is to provide the reader with new developments in the field of myeloid neoplasms, ranging from incorporation of modern methodologies in diagnostics, prognostication and disease monitoring, to clinical management of challenging clinical pictures and advancement in therapeutic modalities.

Given the wide spectrum of specificities included within the label of “myeloid neoplasms”, we have selected some issues that are emerging as particularly relevant in the area.

We discuss the increasing availability of novel agents in acute myeloid leukemia that is making the choice of the most suitable treatment combinations progressively more difficult, as well as the adaptation on an individual basis upon actual sensitivity to treatment, and critical clinical decisions, such as allocation to allogeneic transplantation.

Significant progress has been made in myeloproliferative neoplasms (MPN), first in terms of diagnostic capability; the introduction of genetic data has also contributed to a significant amelioration of conventional disease risk stratification, especially in myelofibrosis. In this setting, we also propose a thorough revision of available evidence on the blast phase of MPN, an extremely challenging context.

In recent years, the achievement of deeper biological insight has also involved rare entities, such as systemic mastocytosis, initially inserted into the MPN category but eventually considered as a separate disease in view of its unique features. In this context, the wide variability of clinical manifestations and disease course demands an appropriate risk stratification, embedding more objective parameters such as comprehensive molecular profiles. Such advances in the knowledge of the disease eventually led to the development of targeted drugs, which are progressively extending the range of available therapeutic alternatives.

Dr. Francesco Mannelli
Guest Editor

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. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). 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

  • acute myeloid leukemia
  • minimal residual disease
  • myeloproliferative neoplasms
  • blast phase
  • targeted therapies
  • systemic mastocytosis

Published Papers (4 papers)

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Research

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12 pages, 1301 KiB  
Article
Venetoclax-Based Regimens for Relapsed/Refractory Acute Myeloid Leukemia in a Real-Life Setting: A Retrospective Single-Center Experience
by Matteo Piccini, Sofia Pilerci, Marta Merlini, Pietro Grieco, Barbara Scappini, Sara Bencini, Benedetta Peruzzi, Roberto Caporale, Leonardo Signori, Fabiana Pancani, Alessandro Maria Vannucchi and Giacomo Gianfaldoni
J. Clin. Med. 2021, 10(8), 1684; https://doi.org/10.3390/jcm10081684 - 14 Apr 2021
Cited by 20 | Viewed by 2920
Abstract
Relapsed/refractory (R/R) acute myeloid leukemia (AML) is a largely unmet medical need, owing to the lack of standardized, effective treatment approaches, resulting in an overall dismal outcome. The only curative option for R/R AML patients is allogeneic hematopoietic stem cell transplantation (HSCT) which [...] Read more.
Relapsed/refractory (R/R) acute myeloid leukemia (AML) is a largely unmet medical need, owing to the lack of standardized, effective treatment approaches, resulting in an overall dismal outcome. The only curative option for R/R AML patients is allogeneic hematopoietic stem cell transplantation (HSCT) which is only applicable in a fraction of patients due to the scarce efficacy and high toxicity of salvage regimens. Recently, a number of targeted agents with relatively favorable toxicity profiles have been explored in clinical trials for R/R AML patients. The Bcl-2 inhibitor venetoclax, in combination with hypomethylating agents or low dose cytarabine, has produced impressive results for newly diagnosed AML, while its role in R/R disease is not well defined yet. We retrospectively analyzed the clinical outcomes of 47 R/R AML patients treated with venetoclax-based regimens between March 2018 and December 2020 at our institution. Overall, we report a composite complete response rate of 55% with an overall acceptable toxicity profile. Outcomes were particularly favorable for NPM1 mutated patients, unlike for FLT3-ITD positive patients irrespective of NPM1 status. For patients treated with intention to transplant, the procedure could be finally performed in 54%. These findings suggest a role for venetoclax-based regimens in R/R AML patients and support the design of prospective studies. Full article
(This article belongs to the Special Issue New Developments in the Field of Myeloid Neoplasms)
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13 pages, 1304 KiB  
Article
Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis
by Aneta Szudy-Szczyrek, Oliwia Bachanek-Mitura, Tomasz Gromek, Karolina Chromik, Andrzej Mital, Michał Szczyrek, Witold Krupski, Justyna Szumiło, Zuzanna Kanduła, Grzegorz Helbig and Marek Hus
J. Clin. Med. 2021, 10(5), 1109; https://doi.org/10.3390/jcm10051109 - 7 Mar 2021
Cited by 11 | Viewed by 2752
Abstract
In April 2017 midostaurin was approved by the US Food and Drug Administration for the treatment of patients with aggressive systemic mastocytosis (ASM). So far, very limited real world data on its efficacy is available. Thirteen patients aged from 48 to 79 years, [...] Read more.
In April 2017 midostaurin was approved by the US Food and Drug Administration for the treatment of patients with aggressive systemic mastocytosis (ASM). So far, very limited real world data on its efficacy is available. Thirteen patients aged from 48 to 79 years, who received midostaurin in the early access program, were included in the study. Midostaurin was used both in first (n = 5) and subsequent lines of treatment (n = 8). The median duration of exposure was 9 months. Most patients (77%, n = 10) had a clinical improvement already as soon as the second month of therapy. Objective response was noted in 4 (50%) of eight evaluated patients. Among responders, we observed a decrease in serum tryptase level (median 74.14%) and bone marrow infiltration by mast cells (median 50%) in the sixth month of treatment. In one case, in the 10th month of treatment, allogenic stem cell transplantation was performed, achieving complete remission. Five patients died, three due to progression of disease, one in the course of secondary acute myeloid leukemia and one due to reasons not related to mastocytosis. Treatment is ongoing in seven patients. We found that midostaurin therapy is beneficial to patients with ASM. Full article
(This article belongs to the Special Issue New Developments in the Field of Myeloid Neoplasms)
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Review

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11 pages, 698 KiB  
Review
Bone Marrow Mastocytosis: A Diagnostic Challenge
by Roberta Zanotti, Ilaria Tanasi, Andrea Bernardelli, Giovanni Orsolini and Patrizia Bonadonna
J. Clin. Med. 2021, 10(7), 1420; https://doi.org/10.3390/jcm10071420 - 1 Apr 2021
Cited by 13 | Viewed by 3058
Abstract
Bone marrow mastocytosis (BMM) represents a provisional, indolent subvariant of systemic mastocytosis (SM). Utilizing WHO criteria, BMM requires bone marrow (BM) involvement and the absence of mastocytosis skin lesions. BMM is characterized by male sex prevalence, a slight increase of serum tryptase levels, [...] Read more.
Bone marrow mastocytosis (BMM) represents a provisional, indolent subvariant of systemic mastocytosis (SM). Utilizing WHO criteria, BMM requires bone marrow (BM) involvement and the absence of mastocytosis skin lesions. BMM is characterized by male sex prevalence, a slight increase of serum tryptase levels, low BM mast cells (MC) burden, and an indolent clinical course. BMM shows a strong correlation with severe anaphylaxis, mainly due to an IgE-mediated allergy to bee or wasp venom and, less frequently, to unexplained (idiopathic) anaphylaxis. Furthermore, BMM is often associated with osteoporosis which could be the only presenting symptom of the disease. BMM is an undervalued disease as serum tryptase levels are not routinely measured in the presence of unexplained osteoporosis or anaphylaxis. Moreover, BMM patients are often symptom-free except for severe allergic reactions. These factors, along with typical low BM MCs infiltration, may contribute to physicians overlooking BMM diagnosis, especially in medical centers that lack appropriately sensitive diagnostic techniques. This review highlights the need for a correct diagnostic pathway to diagnose BMM in patients with suspected symptoms but lacking typical skin lesions, even in the case of normal serum tryptase levels. Early diagnosis may prevent potential life-threatening anaphylaxis or severe skeletal complications. Full article
(This article belongs to the Special Issue New Developments in the Field of Myeloid Neoplasms)
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13 pages, 279 KiB  
Review
Acute Myeloid Leukemia Evolving from Myeloproliferative Neoplasms: Many Sides of a Challenging Disease
by Francesco Mannelli
J. Clin. Med. 2021, 10(3), 436; https://doi.org/10.3390/jcm10030436 - 23 Jan 2021
Cited by 13 | Viewed by 2720
Abstract
The evolution to blast phase is a frequently unpredictable and almost invariably fatal event in the course of myeloproliferative neoplasms. The molecular mechanisms underlying blast transformation have not been elucidated and the specific genetic and epigenetic events governing leukemogenesis remain unclear. The result [...] Read more.
The evolution to blast phase is a frequently unpredictable and almost invariably fatal event in the course of myeloproliferative neoplasms. The molecular mechanisms underlying blast transformation have not been elucidated and the specific genetic and epigenetic events governing leukemogenesis remain unclear. The result of the long-lasting dynamics, passing through progressive genetic steps, is the emergence of one or more clones often characterized by complex genetics, either at conventional karyotyping or at modern high-throughput sequencing analyses, with all clinical and prognostic correlates. The current therapeutic approaches are largely inadequate and incapable of modifying the inherent unfavorable outcome. In this perspective, the application of targeted strategies should aim to prevent the occurrence of leukemic evolution. At transformation, the crucial target of treatment should be the allocation to allogeneic transplant for eligible patients. With this in mind, novel combination treatments may provide useful bridging strategies, beyond potentially improving outcomes for patients who are not candidates for intensive approaches. Full article
(This article belongs to the Special Issue New Developments in the Field of Myeloid Neoplasms)
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