Morgana Pinheiro Maux Lessa, Alexandre Soares Ferreira Junior, Margaret Graton, Erin Simon, Leila Ledbetter, Oluwatoyosi A Onwuemene
When de-novo immune-mediated thrombotic thrombocytopenic purpura (TTP) is diagnosed following an invasive procedure, clinical presentation patterns and outcomes are poorly defined. Therefore, in a systematic literature review of patients diagnosed with TTP following an invasive surgical or non-surgical procedure, we identified 19 studies reporting data on 25 patients. These data suggest that 1) TTP pathogenesis likely begins prior to the invasive procedure, 2) patients experience significant diagnostic delays, and 3) there is a high incidence of renal replacement therapy...
April 9, 2024: Blood Reviews
Yun Wang, Zhi-Jian Liang, Robert Peter Gale, Hua-Ze Liao, Jun Ma, Tie-Jun Gong, Ying-Qi Shao, Yang Liang
Chronic myeloid leukaemia (CML) is caused by BCR::ABL1. Tyrosine kinase-inhibitors (TKIs) are the initial therapy. Several organizations have reported milestones to evaluate response to initial TKI-therapy and suggest when a change of TKI should be considered. Achieving treatment-free remission (TFR) is increasingly recognized as the optimal therapy goal. Which TKI is the best initial therapy for which persons and what depth and duration of molecular remission is needed to achieve TFR are controversial. In this review we discuss these issues and suggest future research directions...
March 27, 2024: Blood Reviews
Dominic J Fowler-Shorten, Charlotte Hellmich, Matthew Markham, Kristian M Bowles, Stuart A Rushworth
B-cell lymphoma-2 (BCL-2) family proteins are fundamental regulators of the intrinsic apoptotic pathway which modulate cellular fate. In many haematological malignancies, overexpression of anti-apoptotic factors (BCL-2, BCL-XL and MCL-1) circumvent apoptosis. To address this cancer hallmark, a concerted effort has been made to induce apoptosis by inhibiting BCL-2 family proteins. A series of highly selective BCL-2 homology 3 (BH3) domain mimetics are in clinical use and in ongoing clinical trials for acute myeloid leukaemia (AML), chronic myeloid leukaemia (CML), chronic lymphocytic leukaemia (CLL), and multiple myeloma (MM)...
March 21, 2024: Blood Reviews
Paul Telfer, Kofi A Anie, Stella Kotsiopoulou, Laura Aiken, Stephen Hibbs, Carol Burt, Sara Stuart-Smith, Sanne Lugthart
The acute pain crisis (APC) is the commonest complication of sickle cell disease (SCD). Severe episodes may require treatment in hospital with strong opioid analgesic drugs, combined with additional supportive care measures. Guidelines for APC management have been produced over the past two decades gathering evidence from published studies, expert opinion, and patient perspective. Unfortunately, reports from multiple sources indicate that guidelines are often not followed, and that acute care in emergency departments and on acute medical wards is suboptimal...
March 19, 2024: Blood Reviews
Marija Dimitrievska, Dravie Bansal, Marta Vitale, John Strouboulis, Annarita Miccio, Kypros H Nicolaides, Sara El Hoss, Panicos Shangaris, Joanna Jacków-Malinowska
Recent advancements in gene editing illuminate new potential therapeutic approaches for Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in the β-globin gene. Despite the availability of several FDA-approved medications for symptomatic relief, allogeneic hematopoietic stem cell transplantation (HSCT) remains the sole curative option, underscoring a persistent need for novel treatments. This review delves into the growing field of gene editing, particularly the extensive research focused on curing haemoglobinopathies like SCD...
March 7, 2024: Blood Reviews
Geoffrey L Uy, Daniel J DeAngelo, Jay N Lozier, Dennis M Fisher, Brian A Jonas, John L Magnani, Pamela S Becker, Hillard M Lazarus, Ingrid G Winkler
E-selectin, a cytoadhesive glycoprotein, is expressed on venular endothelial cells and mediates leukocyte localization to inflamed endothelium, the first step in inflammatory cell extravasation into tissue. Constitutive marrow endothelial E-selectin expression also supports bone marrow hematopoiesis via NF-κB-mediated signaling. Correspondingly, E-selectin interaction with E-selectin ligand (sialyl Lewisx ) on acute myeloid leukemia (AML) cells leads to chemotherapy resistance in vivo. Uproleselan (GMI-1271) is a carbohydrate analog of sialyl Lewisx that blocks E-selectin binding...
February 28, 2024: Blood Reviews
Yasamin Abdu, Alaa Rahhal, Khalid Ahmed, Nada Adli, Mariam Abdou, Elrazi Awadelkarim Hamid Ali, Salam Al-Kindi, Mona Al Rasheed, Jaffer Altooq, Iheb Bougmiza, Mohamed A Yassin
This systematic review and meta-analysis aimed to provide guidance on preoperative blood transfusion strategies for patients with sickle cell disease (SCD). We included all randomized controlled and observational studies exploring the clinical outcomes of preoperative blood transfusion among patients with SCD compared to the conservative transfusion strategy until 14/09/2022. Sixteen studies involving 3486 participants were analysed. The findings revealed a significantly higher bleeding rate in patients who received preoperative transfusion than those who followed a conservative strategy (RR = 4...
February 18, 2024: Blood Reviews
Thomas M Kuczmarski, Lizabeth Roemer, Oreofe O Odejide
Patients with hematologic malignancies experience high rates of depression. These patients are vulnerable to depression throughout the disease trajectory, from diagnosis to survivorship, and at the end of life. In addition to the distressing nature of depression, it has substantial downstream effects including poor quality of life, increased risk of treatment complications, and worse survival. Therefore, systematic screening for depression and integration of robust psychological interventions for affected patients is crucial...
February 13, 2024: Blood Reviews
Maya Bizri, Rawan Koleilat, Nathalie Akiki, Reem Dergham, Alexandra Monica Mihailescu, Rayan Bou-Fakhredin, Khaled M Musallam, Ali T Taher
Advances in understanding the disease process in β-thalassemia supported development of various treatment strategies that resulted in improved survival. Improved survival, however, allowed multiple morbidities to manifest and cemented the need for frequent, lifelong treatment. This has directly impacted patients' health-related quality of life and opened the door for various psychiatric and cognitive disorders to potentially develop. In this review, we summarize available evidence on quality of life, depression and anxiety, suicidality, and cognitive impairment in adult patients with β-thalassemia while sharing our personal insights from experience in treating patients with both transfusion-dependent and non-transfusion-dependent forms...
February 8, 2024: Blood Reviews
Malgorzata Mikulska, Chiara Oltolini, Emanuela Zappulo, Michele Bartoletti, Anna Maria Frustaci, Andrea Visentin, Candida Vitale, Francesca R Mauro
CLL is associated with an increased risk of infectious complications. Treatment with BTK or BCL-2 inhibitors does not seem to increase significantly the risk of opportunistic infections, but the role of combination therapies including BTK and/or BCL-2 inhibitors remains to be established. Various infectious complications can be successfully prevented with appropriate risk management strategies. In this paper we reviewed the international guidelines on prevention and management of infectious complications in patients with CLL treated with BTK or BCL-2 inhibitors...
February 1, 2024: Blood Reviews
Sarah E Clarke, Kathryn A Fuller, Wendy N Erber
Multiple myeloma is a plasma cell neoplasm driven by primary (e.g. hyperdiploidy; IGH translocations) and secondary (e.g. 1q21 gains/amplifications; del(17p); MYC translocations) chromosomal events. These are important to detect as they influence prognosis, therapeutic response and disease survival. Currently, cytogenetic testing is most commonly performed by interphase fluorescence in situ hybridisation (FISH) on aspirated bone marrow samples. A number of variations to FISH methodology are available, including prior plasma cell enrichment and incorporation of immunophenotypic plasma cell identification...
March 2024: Blood Reviews
Antonino Carbone, Amy Chadburn, Annunziata Gloghini, Emanuela Vaccher, Mark Bower
Significant advances in the field of lymphoma have resulted in two recent classification proposals, the International Consensus Classification (ICC) and the 5th edition WHO. A few entities are categorized differently in the ICC compared to the WHO. Nowhere is this more apparent than the immunodeficiency lymphoproliferative disorders. The three previous versions of the WHO classification (3rd, 4th and revised 4th editions) and the ICC focused on four clinical settings in which these lesions arise for primary categorization...
March 2024: Blood Reviews
Azin Vakilpour, Bénédicte Lefebvre, Catherine Lai, Marielle Scherrer-Crosbie
Cancer survivors are at significant risk of cardiovascular (CV) morbidity and mortality; patients with hematologic malignancies have a higher rate of death due to heart failure compared to all other cancer subtypes. The majority of conventional hematologic cancer treatments is associated with increased risk of acute and long-term CV toxicity. The incidence of cancer therapy induced CV toxicity depends on the combination of patient characteristics and on the type, dose, and duration of the therapy. Early diagnosis of CV toxicity, appropriate referral, more specific cardiac monitoring follow-up and timely interventions in target patients can decrease the risk of CV adverse events, the interruption of oncological therapy, and improve the patient's prognosis...
March 2024: Blood Reviews
A Barrett, N Appleby, H Dreau, C P Fox, T Munir, T A Eyre
Richter transformation (RT) represents an aggressive histological transformation from chronic lymphocytic leukaemia, most often to a large B cell lymphoma. It is characterised by chemo-resistance and subsequent short survival. Drug development has struggled over recent years in light of the aggressive kinetics of the disease, lack of pivotal registrational trials and relative rarity of the phenomenon. In this review we will highlight the diagnostic and therapeutic challenges of managing patients with RT as well as taking a look to the future therapeutic landscape...
March 2024: Blood Reviews
Gunjan Shah, Sergio Giralt, Parastoo Dahi
Melphalan, has been a major component of myeloma therapy since the 1950s. In the context of hematopoietic cell transplantation (HCT), high dose melphalan (HDM) is the most common conditioning regimen used due to its potent anti-myeloma effects and manageable toxicities. Common toxicities associated with HDM include myelosuppression, gastrointestinal issues, and mucositis. Established approaches to reduce these toxicities encompass dose modification, nausea prophylaxis with 5HT3 receptor antagonists, cryotherapy, amifostine use, and growth factors...
March 2024: Blood Reviews
Anem Waheed, Jamile Shammo, David Dingli
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by complement-mediated hemolysis and thrombosis through the alternative pathway. The most common symptom of PNH is fatigue due to chronic anemia, which can negatively impact quality of life (QoL) and affect overall well-being. The currently approved therapies for PNH significantly limit intravascular hemolysis (IVH) and reduce the risk of thrombosis; however, they are associated with an infusion schedule that can become burdensome, and not all patients experience complete disease control...
March 2024: Blood Reviews
Grace H Tang, Michelle Sholzberg
Iron deficiency is the most common and widespread nutritional deficiency in the world. For women, the risk of iron deficiency and iron deficiency anemia increases due to iron demands during pregnancy and regular iron losses due to menstruation during reproductive years. These interrelated conditions are of public health concern as they are highly prevalent, and the negative consequences such as chronic fatigue, cognitive impairment and poor quality of life are broad and multifaceted. People of low socioeconomic status are at higher risk of iron deficiency due to low intake of expensive iron-rich foods, and decreased access to healthcare...
March 2024: Blood Reviews
Daniel J Chandra, Curtis A Lachowiez, Sanam Loghavi
The updated WHO 5th edition and ICC 2022 classification systems for AML aim to refine our diagnostic criteria and definitions of AML with deeper incorporation of cytogenetic and molecular aberrations. The two classification systems diverge, however, in numerous AML defining criteria and subclassifications, including the incorporation of blast enumeration and the integration of specific genomic mutations. These differences often create challenges for clinicians in not only establishing a diagnosis of AML, but also in determining the best treatment plan for patients...
March 2024: Blood Reviews
Rozeta Sokou, Stavroula Parastatidou, Aikaterini Konstantinidi, Andreas G Tsantes, Nicoletta Iacovidou, Daniele Piovani, Stefanos Bonovas, Argirios E Tsantes
The assessment of hemostatic disorders in neonates is crucial, but remains challenging for clinicians. Although the concept of developmental hemostasis is widely accepted among hemostasis specialists globally, it is probably under-recognized by clinicians and laboratory practitioners. In parallel with age-dependent hemostatic status maturation, comprehension of the differences between normal values is crucial for the accurate diagnosis of potential hemorrhagic and thrombotic disorders of the vulnerable neonatal population...
March 2024: Blood Reviews
H Witte, A Künstner, N Gebauer
The vast spectrum of aggressive B-cell non-Hodgkin neoplasms (B-NHL) encompasses several infrequent entities occurring in association with viral infections, posing diagnostic challenges for practitioners. In the emerging era of precision oncology, the molecular characterization of malignancies has acquired paramount significance. The pathophysiological comprehension of specific entities and the identification of targeted therapeutic options have seen rapid development. However, owing to their rarity, not all entities have undergone exhaustive molecular characterization...
January 20, 2024: Blood Reviews
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