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Medicina
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Therapeutic Strategies for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
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Therapeutic Strategies for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

A special issue of Medicina (ISSN 1648-9144). This special issue belongs to the section "Hematology and Immunology".

Deadline for manuscript submissions: closed (30 November 2023) | Viewed by 13420

Special Issue Editors

Dr. Carmelo Gurnari

E-Mail Website
Guest Editor
Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy
Interests: bone marrow failure; VEXAS; myeloid neoplasms
Dr. Simona Pagliuca

E-Mail Website
Guest Editor
Department of Clinical Hematology, CHRU Nancy, CEDEX, 54035 Nancy, France
Interests: bone marrow failure; immunology; HLA system

Special Issue Information

Dear Colleagues, 

Idiopathic Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) belong to the spectrum of immune-mediated bone marrow failure disorders (BMFs). The autoimmune attack by cytotoxic T lymphocytes to hematopoietic stem cells (HSCs) is the pathogenic hallmark of AA. The consequent reduction of the HSCs pool generates the classical AA clinical picture, dominated by peripheral cytopenias and an increased tendency for hemorrhagic and infectious complications. In the context of an AA immune attack, PIGA mutations may arise, ultimately generating the absence of glycosylphosphatidylinositol (GPI)-anchored protein on the membrane of all blood cells derived by the mutant clone. The absence of such GPI-linked proteins makes blood cells (particularly erythrocytes) vulnerable to complement-mediated lysis. This represents the genomic underpinning of PNH, whose clinical picture encompasses complement-mediated hemolytic anemia and thrombophilia. In the last decade, new evidence on disease pathobiology furthered our understanding of both AA and PNH, generating mechanistic clues on potential therapeutic opportunities. Particularly, the addition of eltrombopag in AA and newer complement inhibitors in PNH is revolutionizing the treatment landscape of BMFs. 

In this issue, we will consider articles across a wide spectrum of related areas, ranging from technical studies, review articles, meta-analyses, research articles (including observation and randomized), and case reports. 

Dr. Carmelo Gurnari
Dr. Simona Pagliuca
Guest Editors

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Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2200 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

  • aplastic anemia
  • paroxysmal nocturnal hemoglobinuria
  • immunosuppressive treatment
  • complement inhibitors

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Published Papers (4 papers)

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Review

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19 pages, 727 KiB  
Review
Safety Profile of Monoclonal Antibodies and Subsequent Drug Developments in the Treatment of Paroxysmal Nocturnal Hemoglobinuria
by Vasantha Mallenahalli Neeekantappa, Ashwin Kamath and Poovizhi Bharathi Rajaduraivelpandian
Medicina 2024, 60(3), 379; https://doi.org/10.3390/medicina60030379 - 24 Feb 2024
Cited by 1 | Viewed by 2913
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal stem cell disease characterized by intravascular hemolysis due to the targeting of affected red blood cells by the complement system. Eculizumab and ravulizumab are two monoclonal antibodies that inhibit the complement system’s components and have been [...] Read more.
Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal stem cell disease characterized by intravascular hemolysis due to the targeting of affected red blood cells by the complement system. Eculizumab and ravulizumab are two monoclonal antibodies that inhibit the complement system’s components and have been shown to significantly improve survival and quality of life. This review describes the role of these monoclonal antibodies in the treatment of PNH with an emphasis on their safety profile. The challenges in the use of these drugs and new drugs in various stages of drug development are also described, which may be helpful in addressing some of these challenges. Full article
(This article belongs to the Special Issue Therapeutic Strategies for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria)
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13 pages, 4082 KiB  
Review
All Roads Lead to Interferon-γ: From Known to Untraveled Pathways in Acquired Aplastic Anemia
by Bianca Serio, Valentina Giudice and Carmine Selleri
Medicina 2023, 59(12), 2170; https://doi.org/10.3390/medicina59122170 - 14 Dec 2023
Cited by 3 | Viewed by 2150
Abstract
Bone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated [...] Read more.
Bone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated autologous immune attacks against hematopoietic stem and progenitor cells (HSPCs). Interferon-γ (IFNγ), tumor necrosis factor-α, and Fas-ligand are historically linked to AA pathogenesis because they drive Th1 and cytotoxic T cell-mediated responses and can directly induce HSPC apoptosis and differentiation block. The use of omics technologies has amplified the amount of data at the single-cell level, and knowledge on AA, and new scenarios, have been opened on “old” point of view. In this review, we summarize the current state-of-art of the pathogenic role of IFNγ in AA from initial findings to novel evidence, such as the involvement of the HIF-1α pathway, and how this knowledge can be translated in clinical practice. Full article
(This article belongs to the Special Issue Therapeutic Strategies for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria)
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14 pages, 733 KiB  
Review
Paroxysmal Nocturnal Hemoglobinuria: Biology and Treatment
by Carlos Bravo-Perez, Luca Guarnera, Nakisha D. Williams and Valeria Visconte
Medicina 2023, 59(9), 1612; https://doi.org/10.3390/medicina59091612 - 6 Sep 2023
Cited by 8 | Viewed by 5331
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal hematopoietic disorder characterized by the lack of glycosylphosphatidylinositol-anchored proteins (GPI-APs) as a consequence of somatic mutations in the phosphatidylinositol glycan anchor biosynthesis class A (PIGA) gene. Clinical manifestations of PNH are intravascular hemolysis, [...] Read more.
Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal hematopoietic disorder characterized by the lack of glycosylphosphatidylinositol-anchored proteins (GPI-APs) as a consequence of somatic mutations in the phosphatidylinositol glycan anchor biosynthesis class A (PIGA) gene. Clinical manifestations of PNH are intravascular hemolysis, thrombophilia, and bone marrow failure. Treatment of PNH mainly relies on the use of complement-targeted therapy (C5 inhibitors), with the newest agents being explored against other factors involved in the complement cascade to alleviate unresolved intravascular hemolysis and extravascular hemolysis. This review summarizes the biology and current treatment strategies for PNH with the aim of reaching a general audience with an interest in hematologic disorders. Full article
(This article belongs to the Special Issue Therapeutic Strategies for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria)
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Other

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7 pages, 887 KiB  
Case Report
Shifting Paradigms: The Case of Autologous Reconstitution after an Upfront Matched Unrelated Hematopoietic Cell Transplantation for Severe Acquired Aplastic Anemia in a Child
by Cécile Pochon, Marion Lubnau and Simona Pagliuca
Medicina 2023, 59(11), 1890; https://doi.org/10.3390/medicina59111890 - 24 Oct 2023
Viewed by 2317
Abstract
During the last few years, the therapeutic landscape of idiopathic aplastic anemia (IAA) has been profoundly revolutionized by the increased use of alternative transplant procedures, such that today hematopoietic cell transplantation (HCT) from a matched unrelated donor (MUD) has been suggested as a [...] Read more.
During the last few years, the therapeutic landscape of idiopathic aplastic anemia (IAA) has been profoundly revolutionized by the increased use of alternative transplant procedures, such that today hematopoietic cell transplantation (HCT) from a matched unrelated donor (MUD) has been suggested as a possible first line strategy in pediatric patients with severe IAA, in the absence of a matched related donor. However, in this particular context, outcomes and early and long-term toxicities remain to be determined, as compared to non-transplant procedures. While prospective trials are ongoing, we report here the case of a 12-year-old boy with IAA, receiving an upfront bone marrow HCT from a MUD, who experienced early graft rejection associated with autologous hematological recovery, which could induce remission of his hemopathy. This case offers the opportunity to discuss the challenges associated with these new transplant paradigms and provides a brief review of the literature regarding the issue of autologous recoveries after allogeneic HCT in IAA. Full article
(This article belongs to the Special Issue Therapeutic Strategies for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria)
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