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Pharmaceuticals
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Novel Therapeutics in Hematology
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Novel Therapeutics in Hematology

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: closed (20 February 2025) | Viewed by 2878

Special Issue Editor

Dr. Eleni Gavriilaki

E-Mail Website
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,

Advancements in hematology, the medical field focusing on the study of blood, blood-forming organs, and blood diseases, have led to the development of novel therapeutics aimed at improving treatment outcomes for patients. These novel treatments encompass a wide range of approaches, each designed to target specific diseases or conditions more precisely and with fewer side effects.

Therefore, this Special Issue aims to highlight basic, translational, and clinical research in the field and welcomes original research and review articles from physicians and scientists.

Dr. Eleni Gavriilaki
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. Pharmaceuticals is an international peer-reviewed open access monthly 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 2900 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

  • hematopoietic cell transplantation
  • cell therapy
  • acute myeloid leukemia
  • graft-versus-host disease
  • hematologic malignancies

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Published Papers (2 papers)

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13 pages, 1193 KiB  
Article
Safety and Efficacy of Extracorporeal Photopheresis for Acute and Chronic Graft-versus-Host Disease
by Eleni Gavriilaki, Eleni Papchianou, Giorgos Karavalakis, Ioannis Batsis, Alkistis Panteliadou, Andriana Lazaridou, Despina Mallouri, Varnavas Constantinou, Paraskevi Karvouni, Paschalis Evangelidis, Anna Papakonstantinou, Apostolia Papalexandri, Panayotis Kaloyannidis, Nikolaos Spyridis, Zoi Bousiou, Anna Vardi, Evangelia Yannaki, Damianos Sotiropoulos and Ioanna Sakellari
Pharmaceuticals 2024, 17(10), 1279; https://doi.org/10.3390/ph17101279 - 27 Sep 2024
Cited by 1 | Viewed by 1314
Abstract
Background/Objectives: Despite novel biological agents, steroid-dependent or -refractory graft-versus-host disease (GvHD) remains a severe complication of allogeneic hematopoietic cell transplantation (allo-HCT). Extracorporeal photopheresis (ECP) is an alternative, non-immunosuppressive treatment for patients with acute (aGvHD) or chronic (cGvHD) GvHD. The aim of this study [...] Read more.
Background/Objectives: Despite novel biological agents, steroid-dependent or -refractory graft-versus-host disease (GvHD) remains a severe complication of allogeneic hematopoietic cell transplantation (allo-HCT). Extracorporeal photopheresis (ECP) is an alternative, non-immunosuppressive treatment for patients with acute (aGvHD) or chronic (cGvHD) GvHD. The aim of this study was to investigate the safety and efficacy of ECP in the treatment of acute and chronic GvHD; Methods: We prospectively studied 112 patients with cGvHD who received one or more previous lines of treatment and 28 patients with steroid-dependent or refractory grade II-IV aGvHD post-alloHSCT. Results: In terms of severe aGvHD, most of the patients (19/28) responded to ECP treatment, while the five-year overall survival (OS) was 34%. After adjustment for several confounder factors, the reduction in immunosuppression (p = 0.026) and number of ECP sessions (p < 0.001) were associated with improved OS. Regarding chronic GvHD, only 19 patients failed to respond to ECP treatment; though significantly lower rates of response were presented in patients with visceral involvement (p = 0.037) and earlier post-transplant GVHD diagnosis (p = 0.001). Over a follow-up period of 45.2 [interquartile range (IQR): 5.6–345.1] months, the 5-year cumulative incidence (CI) of cGvHD-related mortality was 21.2% and was significantly reduced in patients with ECP response (p < 0.001), while the 5-year OS was 65.3%. Conclusions: Our results confirm the safety and efficacy of ECP in patients with GvHD and provide sufficient data for further investigation and the best combination drugs needed such that GvHD will not be the major barrier of allo-HCT in the near future. Full article
(This article belongs to the Special Issue Novel Therapeutics in Hematology)
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14 pages, 931 KiB  
Case Report
Delayed Onset of Thrombotic Microangiopathy (TMA) upon Prolonged Carfilzomib Therapy in Multiple Myeloma: A Case Report and Comprehensive Review
by Andrea Ceglédi, Ágnes Király, Andrea Várkonyi, Szabolcs Tasnády, Hajnalka Andrikovics, Mónika Fekete, Bálint G. Szabó, Zsuzsanna Szemlaky, Ágnes Szilágyi, György Sinkovits, Zoltán Prohászka, Marienn Réti and Gábor Mikala
Pharmaceuticals 2024, 17(12), 1722; https://doi.org/10.3390/ph17121722 - 20 Dec 2024
Cited by 1 | Viewed by 1101
Abstract
Background: Thrombotic microangiopathy (TMA) is a potentially life-threatening complication associated with carfilzomib, a proteasome inhibitor approved for treating multiple myeloma. TMA typically presents within the initial months of treatment; however, delayed onset is rare and poses significant diagnostic challenges. Methods: We conducted a [...] Read more.
Background: Thrombotic microangiopathy (TMA) is a potentially life-threatening complication associated with carfilzomib, a proteasome inhibitor approved for treating multiple myeloma. TMA typically presents within the initial months of treatment; however, delayed onset is rare and poses significant diagnostic challenges. Methods: We conducted a retrospective analysis of the medical records of a 47-year-old Caucasian woman diagnosed with IgA kappa myeloma who developed signs and symptoms consistent with TMA eleven months after the initiation of carfilzomib therapy and already in ongoing very good partial remission. Results: The clinical presentation included an acute onset of weakness, dizziness, somnolence, diffuse bruising, oliguria, jaundice, severe thrombocytopenia, and acute kidney injury. An immediate workup raised a strong suspicion for TMA, confirmed by laboratory findings of schistocytosis and complement activation. Following the immediate discontinuation of carfilzomib, the patient underwent 18 plasmapheresis (PEX) sessions and received supportive fresh frozen plasma transfusions, which resulted in the complete remission of TMA symptoms without the need for complement inhibitory therapy. Conclusions: The need for ongoing monitoring for TMA throughout carfilzomib therapy, regardless of treatment duration, is emphasized. Early diagnosis and intervention, including drug discontinuation and the timely initiation of PEX, are crucial for patient recovery. Full article
(This article belongs to the Special Issue Novel Therapeutics in Hematology)
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