Alessandro Bosi, Wilma Barcellini, Francesco Passamonti, Bruno Fattizzo
Despite recent advancements, treatment of cytopenia due to bone marrow failures (BMF) and myeloid neoplasms remains challenging. Androgens promote renewal and maturation of blood cells and may be beneficial in these forms. Here we report a systematic review of androgens use as single agent in hematologic conditions. Forty-six studies, mainly retrospective with various androgen types and doses, were included: 12 on acquired aplastic anemia (AA), 11 on inherited BMF, 17 on myelodysplastic syndromes (MDS), and 7 on myelofibrosis...
September 9, 2023: Blood Reviews
H P Eising, M C Punt, J C Leemans, M Y Bongers
BACKGROUND: Optimal peri-operative management for women with Von Willebrand disease (VWD) and heavy menstrual bleeding (HMB) remains undetermined. AIM AND METHODS: To evaluate (pre)operative management in relation to (post)operative bleeding after endometrial ablation (EA) and hysterectomy in VWD women with HMB by performing a database search between 1994 and 2023. RESULTS: Eleven cohort studies and 1 case-report were included, of overall 'low' quality, describing 691 operative procedures...
September 7, 2023: Blood Reviews
Enrico Santinelli, Maria Rosaria Pascale, Zhuoer Xie, Talha Badar, Maximilian F Stahl, Jan P Bewersdorf, Carmelo Gurnari, Amer M Zeidan
In recent years, the therapeutic landscape of myeloid malignancies has been completely revolutionized by the introduction of several new drugs, targeting molecular alterations or pathways crucial for leukemia cells survival. Particularly, many agents targeting apoptosis have been investigated in both pre-clinical and clinical studies. For instance, venetoclax, a pro-apoptotic agent active on BCL-2 signaling, has been successfully used in the treatment of acute myeloid leukemia (AML). The impressive results achieved in this context have made the apoptotic pathway an attractive target also in other myeloid neoplasms, translating the experience of AML...
August 29, 2023: Blood Reviews
Prashant Kapoor, S Vincent Rajkumar
Waldenström macroglobulinemia (WM) is a unique CD20+, B-cell non-Hodgkin lymphoma, characterized by lymphoplasmacytic infiltration of the bone marrow and circulating monoclonal immunoglobulin M. The clinical manifestations and outcomes of patients are highly variable. High-level evidence supports integration of monoclonal anti-CD20 antibody, rituximab, to the chemotherapy backbone to treat WM. However, its contemporary management has become more nuanced, with deeper understanding of the pathophysiology and incorporation of Bruton's tyrosine kinase (BTK) inhibitors to the treatment paradigm...
August 26, 2023: Blood Reviews
Maximilian Stahl, Jan Philipp Bewersdorf, Zhuoer Xie, Matteo Giovanni Della Porta, Rami Komrokji, Mina L Xu, Omar Abdel-Wahab, Justin Taylor, David P Steensma, Daniel T Starczynowski, Mikkael A Sekeres, Guillermo Sanz, David A Sallman, Gail J Roboz, Uwe Platzbecker, Mrinal M Patnaik, Eric Padron, Olatoyosi Odenike, Stephen D Nimer, Aziz Nazha, Ravi Majeti, Sanam Loghavi, Richard F Little, Alan F List, Tae Kon Kim, Christopher S Hourigan, Robert P Hasserjian, Stephanie Halene, Elizabeth A Griffiths, Steven D Gore, Peter Greenberg, Maria E Figueroa, Pierre Fenaux, Fabio Efficace, Amy E DeZern, Naval G Daver, Jane E Churpek, Hetty E Carraway, Rena Buckstein, Andrew M Brunner, Jacqueline Boultwood, Uma Borate, Rafael Bejar, John M Bennett, Andrew H Wei, Valeria Santini, Michael R Savona, Amer M Zeidan
The guidelines for classification, prognostication, and response assessment of myelodysplastic syndromes/neoplasms (MDS) have all recently been updated. In this report on behalf of the International Consortium for MDS (icMDS) we summarize these developments. We first critically examine the updated World Health Organization (WHO) classification and the International Consensus Classification (ICC) of MDS. We then compare traditional and molecularly based risk MDS risk assessment tools. Lastly, we discuss limitations of criteria in measuring therapeutic benefit and highlight how the International Working Group (IWG) 2018 and 2023 response criteria addressed these deficiencies and are endorsed by the icMDS...
August 19, 2023: Blood Reviews
D Santagata, M P Donadini, W Ageno
Anticoagulant drugs that are currently used to prevent and/or treat thrombosis have some limitations that hinder their ability to meet specific clinical requirements. While these drugs effectively reduce the rates of thrombotic events, they simultaneously increase the risk of bleeding. Moreover, their risk-to-benefit balance is problematic in some patients, such as those with severe chronic kidney disease or those at high bleeding risk. A novel anticoagulation method, FXI inhibition has emerged as a promising alternative...
August 10, 2023: Blood Reviews
Giovanni Di Minno, Gaia Spadarella, Nelson Mauro Maldonato, Natascia De Lucia, Giancarlo Castaman, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Anna Borrelli, Angelo Lupi, Marco Follino, Gerardo Guerrino, Filomena Morisco, Matteo Di Minno
In clinical medicine, shared decision making (SDM) is a well-recognized strategy to enhance engagement of both patients and clinicians in medical decisions. The success of liver-directed gene therapy (GT) to transform severe congenital haemophilia A (HA) from an incurable to a curable disease has launched a shift beyond current standards of treatment. However, GT acceptance remains low in the community of HA persons. We argue for both persons with haemophilia (PWH) and specialists in HA care including clinicians, as needing SDM-oriented educational programs devoted to GT...
August 1, 2023: Blood Reviews
Christina Darwish, Kyle Farina, Douglas Tremblay
Core binding factor acute myeloid leukemia (CBF AML), defined by t(8;21) or inv(16), is a subset of favorable risk AML. Despite its association with a high complete remission rate after induction and relatively good prognosis overall compared with other subtypes of AML, relapse risk after induction chemotherapy remains high. Optimizing treatment planning to promote recurrence free survival and increase the likelihood of survival after relapse is imperative to improving outcomes. Recent areas of research have included evaluation of the role of gemtuzumab in induction and consolidation, the relative benefit of increased cycles of high dose cytarabine in consolidation, the utility of hypomethylating agents and kinase inhibitors, and the most appropriate timing of stem cell transplant...
July 25, 2023: Blood Reviews
Noffar Bar, Ross S Firestone, Saad Z Usmani
Frontline therapy for multiple myeloma (MM) is evolving to include novel combinations that can achieve unprecedented deep response rates. Several treatment strategies exist, varying in induction regimen composition, use of transplant and or consolidation and maintenance. In this sea of different treatment permutations, the overarching theme is the powerful prognostic factors of disease risk and achievement of minimal residual disease (MRD) negativity. MM has significant inter-patient variability that requires treatment to be individualized...
July 14, 2023: Blood Reviews
Yevgen Chornenkyy, Takayuki Yamamoto, Hidetaka Hara, Sean R Stowell, Ionita Ghiran, Simon C Robson, David K C Cooper
Transfusion of allogeneic human red blood cell (hRBCs) is limited by supply and compatibility between individual donors and recipients. In situations where the blood supply is constrained or when no compatible RBCs are available, patients suffer. As a result, alternatives to hRBCs that complement existing RBC transfusion strategies are needed. Pig RBCs (pRBCs) could provide an alternative because of their abundant supply, and functional similarities to hRBCs. The ability to genetically modify pigs to limit pRBC immunogenicity and augment expression of human 'protective' proteins has provided major boosts to this research and opens up new therapeutic avenues...
July 14, 2023: Blood Reviews
Marika Bini Antunes, Sara Pinto Cardeal, Manuel Magalhães, Emídio Vale-Fernandes, Márcia Barreiro, Rosália Sá, Mário Sousa
Recent developments of assisted reproduction techniques turned possible to avoid the infertility consequences of oncologic treatments, but fertility preservation (FP) has been somewhat neglected in women with hematologic diseases undergoing gonadotoxic treatments. For these specific cases, the current options for FP include the cryopreservation of embryos, mature oocytes and ovarian tissue, and oocyte in-vitro maturation. We intend to make patients and clinicians aware of this important and relevant issue, and provide hematologists, assisted reproduction physicians and patients, with updated tools to guide decisions for FP...
July 13, 2023: Blood Reviews
Allison Mo, Matthew Poynton, Erica Wood, Jake Shortt, Susan J Brunskill, Carolyn Doree, Josie Sandercock, Nicholas Saadah, Edwin Luk, Simon J Stanworth, Zoe McQuilten
Anemia is common in Myelodysplastic Syndromes (MDS). Different anemia treatments have been tested in clinical studies, but the full impact on patients' health-related quality of life (HRQoL) and physical function is unknown. The main aim of this review was to assess whether improvements in anemia are associated with changes in HRQoL/physical function. Twenty-six full-text publications were identified, enrolling 2211 patients: nine randomized trials (RCTs), fourteen non-randomized studies of interventions and three cross-sectional studies...
July 13, 2023: Blood Reviews
Tomás José González-López, Drew Provan, Abelardo Bárez, Angel Bernardo-Gutiérrez, Silvia Bernat, Daniel Martínez-Carballeira, Isidro Jarque-Ramos, Inmaculada Soto, Reyes Jiménez-Bárcenas, Fernando Fernández-Fuertes
There are not many publications that provide a holistic view of the management of primary and secondary ITP as a whole, reflecting the similarities and differences between the two. Given the lack of major clinical trials, we believe that comprehensive reviews are much needed to guide the diagnosis and treatment of ITP today. Therefore, our review addresses the contemporary diagnosis and treatment of ITP in adult patients. With respect to primary ITP we especially focus on establishing the management of ITP based on the different and successive lines of treatment...
September 2023: Blood Reviews
David Kegyes, Diana Gulei, Rares Drula, Diana Cenariu, Bogdan Tigu, Delia Dima, Alina Tanase, Sorina Badelita, Anca-Dana Buzoianu, Stefan Ciurea, Gabriel Ghiaur, Evangelos Terpos, Aaron Ciechanover, Hermann Einsele, Ciprian Tomuleasa
Multiple myeloma (MM) is a malignant plasma cell disorder accounting for around 1.8% of all neoplastic diseases. Nowadays, clinicians have a broad arsenal of drugs at their disposal for the treatment of MM, such as proteasome inhibitors, immunomodulatory drugs, monoclonal antibodies, bispecific antibodies, CAR T-cell therapies and antibody-drug conjugates. In this paper we briefly highlight essential clinical elements relating to proteasome inhibitors, such as bortezomib, carfilzomib and ixazomib. Studies suggest that the early use of immunotherapy may improve outcomes significantly...
September 2023: Blood Reviews
Jeffrey Lantz, Craig A Portell, Emily C Ayers
Secondary involvement of the central nervous system (CNS) by diffuse large b-cell lymphoma (DLBCL) is a rare yet often catastrophic event for DLBCL patients. As standard first-line therapy for DLBCL with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) does not cross the blood-brain barrier, one approach to lessen the risk of CNS relapse has been to include additional agents, primarily methotrexate, directed at the CNS with standard R-CHOP although the timing, dose, and mode of administration differs widely across treating physicians...
September 2023: Blood Reviews
Yasmine St Martin, Joseph K Franz, Mounzer E Agha, Hillard M Lazarus
Since its FDA approval, chimeric antigen receptor (CAR)-T cell therapy is changing the landscape of the treatment algorithm for relapsed and refractory large cell lymphoma and multiple myeloma. While initially hailed as a game changer and received widely with great enthusiasm, the reality of treatment failure soon became a major disappointment. This situation left patients and clinicians alike wondering about the next treatment options. CAR-T cell therapy failure for aggressive lymphoma or multiple myeloma creates a very poor prognosis and the treatment options are very limited...
July 2023: Blood Reviews
Mark Juckett, Christopher Dandoy, Zachariah DeFilipp, Tamila L Kindwall-Keller, Stephen R Spellman, Celalettin Ustun, Bryce M Waldman, Daniel J Weisdorf, William A Wood, Mary M Horowitz, Linda J Burns, Nandita Khera
The field of hematopoietic cell transplantation and cell therapy (HCT/CT) is advancing rapidly to bring an ever-expanding collection of potentially curative therapies to patients with malignant and non-malignant diseases. The impact of these therapies depends on our ability to implement them as new evidence becomes available to advance the quality of care. There is often a long delay between evidence development and adoption of therapies based on that evidence into clinical practice. In this review, we describe the potential factors based on an implementation framework that could act as facilitators or barriers to adoption of therapies in the context of HCT/CT...
July 2023: Blood Reviews
Jean M Nunes, Douglas B Kell, Etheresia Pretorius
ME/CFS is a debilitating chronic condition that often develops after viral or bacterial infection. Insight from the study of Long COVID/Post Acute Sequelae of COVID-19 (PASC), the post-viral syndrome associated with SARS-CoV-2 infection, might prove to be useful for understanding pathophysiological mechanisms of ME/CFS. Disease presentation is similar between the two conditions, and a subset of Long COVID patients meet the diagnostic criteria for ME/CFS. Since Long COVID is characterized by significant vascular pathology - including endothelial dysfunction, coagulopathy, and vascular dysregulation - the question of whether or not the same biological abnormalities are of significance in ME/CFS arises...
July 2023: Blood Reviews
Jennifer A Marks, Xin Wang, Elena M Fenu, Adam Bagg, Catherine Lai
MDS and AML are clonal hematopoietic stem cell disorders of increasing incidence, having a variable prognosis based, among others, on co-occurring molecular abnormalities. TP53 mutations are frequently detected in these myeloid neoplasms and portend a poor prognosis with known therapeutic resistance. This article provides a timely review of the complexity of TP53 alterations, providing updates in diagnosis and prognosis based on new 2022 International Consensus Classification (ICC) and World Health Organization (WHO) guidelines...
July 2023: Blood Reviews
Stacey M Frumm, Shai Shimony, Richard M Stone, Daniel J DeAngelo, Jan Phillipp Bewersdorf, Amer M Zeidan, Maximilian Stahl
Approval of new agents to treat higher risk (HR) myelodysplastic syndrome (MDS) has stalled since the approval of DNA methyltransferase inhibitors (DNMTi). In addition, the options for patients with lower risk (LR) MDS who have high transfusion needs and do not harbor ring sideroblasts or 5q- syndrome are limited. Here, we review the current treatment landscape in MDS and identify areas of unmet need, such as treatment after failure of erythropoiesis-stimulating agents or DNMTis, TP53-mutated disease, and MDS with potentially targetable mutations...
July 2023: Blood Reviews
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