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The Evolving Pharmacotherapeutic Landscape for the Treatment of Sickle Cell Disease.

Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated with global morbidity and early mortality. More effective and inexpensive therapies are needed. During the last five years, the landscape of the pharmacotherapy of SCD has changed dramatically. Currently, 54 drugs have been used or under consideration to use for the treatment of SCD. These fall into 3 categories: the first category includes the four drugs (Hydroxyurea, L-Glutamine, Crizanlizumab tmca and Voxelotor) that have been approved by the United States Food and Drug Administration (FDA) based on suc

The Role of Injectables in the Treatment and Prevention of Cancer-Associated Thrombosis.

Cancer-associated thrombosis (CAT) is a leading cause of death among patients with cancer. CAT can manifest itself as venous thromboembolism (VTE), in the form of deep vein thrombosis or pulmonary embolism, or arterial thromboembolism. The pathophysiology of CAT is complex and depends on cancer-, patient-, treatment- and biomarkers-related factors. Treatment of VTE in patients with cancer is complex and includes three major classes of anticoagulant agents: heparin and its derivatives, e.g., low molecular weight heparins, direct oral anticoagulants (DOACs), and vitamin K inhibitors. Given the t

Efficacy and Safety of a DOAC Compared to Unfractionated Heparin and A Low Molecular Weight Heparin in the Prevention of Thromboembolism in Hematopoietic Stem Cell Transplant Recipients.

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) patients are at risk of thromboembolic events, making thromboprophylaxis crucial. OBJECTIVES: This study aimed to compare apixaban, a direct factor Xa inhibitor (DOAC), with dalteparin and unfractionated heparin for thromboprophylaxis in HSCT recipients. The safety outcome included the assessment of hemorrhagic events. METHODS: In this open-label randomized clinical trial, 182 HSCT recipients were divided into three groups: Apixaban (n = 61, 2.5 mg two times a day), dalteparin (n = 59, 5000 IU daily), and unfractionated heparin (n =

A cost-utility and budget impact analysis of allogeneic hematopoietic stem cell transplantation for severe thalassemic patients in Thailand.

BackgroundHematopoietic stem cell transplantation (HSCT) is the only curative treatment available to severe thalassemic patients. The treatment, however, is very costly, particularly in the context of low and middle income countries, and no studies have been carried out to explore its economic justifiability. This study aimed to estimate the cost-utility of HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for severe thalassemia in Thailand, and to investigate the affordability of HSCT using a budget impact analysis.MethodsA Markov model was used to estimate t

Impact of epoetin beta on quality of life in patients with malignant disease.

This open-label, prospective study was conducted to compare the impact of epoetin beta vs standard care on quality of life (QoL) in anaemic patients with lymphoid or solid tumour malignancies. A total of 262 anaemic patients (haemoglobin [Hb]or=2 g dl(-1) increase in Hb level without need of transfusion after the initial 4 weeks of treatment. Baseline to final visit changes in SF-36 PCS, FACT-F and VAS scores were significantly greater with epoetin beta than with standard care (P<0.05); changes in FACT-An subscale score tended to be greater with epoetin beta (P=0.076). Epoetin beta significant

Immune Therapies for Myelodysplastic Syndromes and Acute Myeloid Leukemia.

Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are hematologic malignancies arising from the bone marrow. Despite recent advances in treating these diseases, patients with higher-risk MDS and AML continue to have a poor prognosis with limited survival. It has long been recognized that there is an immune component to the pathogenesis of MDS and AML, but until recently, immune therapies have played a limited role in treating these diseases. Immune suppressive therapy exhibits durable clinical responses in selected patients with MDS, but the question of which patients are most s

Cancer-associated stroke: Pathophysiology, detection and management (Review).

Numerous types of cancer have been shown to be associated with either ischemic or hemorrhagic stroke. In this review, the epidemiology and pathophysiology of stroke in cancer patients is discussed, while providing vital information on the diagnosis and management of patients with cancer and stroke. Cancer may mediate stroke pathophysiology either directly or via coagulation disorders that establish a state of hypercoagulation, as well as via infections. Cancer treatment options, such as chemotherapy, radiotherapy and surgery have all been shown to aggravate the risk of stroke as well. The clin

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… Generally, cerebrovascular hemorrhagic events are related to recalcitrance to platelet transfusions, to arterial hypertensions, low fibrinogen serum levels and, as previously described, to high-grade GVHD ( 109 ). … With the exception of myeloablative therapy in hematologic malignancies, chemotherapy for non-hematologic ones rarely requires platelet transfusion support ( 54 ). …

Endothelial cell function and endothelial-related disorders following haematopoietic cell transplantation.

Use of haematopoietic cell transplantation (HCT) in the treatment of haematologic and neoplastic diseases may lead to life-threatening complications that cause substantial morbidity and mortality if untreated. In addition to patient- and disease-related factors, toxicity associated with HCT puts patients at risk for complications that share a similar pathophysiology involving endothelial cells (ECs). Normally, the endothelium plays a role in maintaining homeostasis, including regulation of coagulation, vascular tone, permeability and inflammatory processes. When activated, ECs acquire cellular

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… Recent evidence suggests that inflammation can increase platelet adhesion to the endothelium and platelets can in turn amplify the inflammatory response. 79 Platelet transfusions have also been associated with a greater risk of IPS following myeloablative HCT. 80 Endothelial apoptosis occurs with T‐cell–mediated … The association between red blood cell and platelet transfusion and subsequently developing idiopathic pneumonia syndrome after hematopoietic stem cell transplantation. Transfusion. 2014;54:1071–80. 81. Gerbitz A, Nickoloff BJ, Olkiewicz K, Willmarth NE, Hildebrandt G, Liu C, et al. …

Редкое дерматологическое проявление фолликулярных смешений у пациента с множественной миеломой и конечной стадией почечной болезни при гемодиализе: отчет по делу.

Множественная миелома (ММ) — гематологическая порча, характеризующаяся клональным расширением злокачественных плазменных клеток в костном мозге, что приводит к различным системным осложнениям. Хотя резкие проявления MM редки, фолликулы представляют собой весьма специфический, но редкий дерматологический вывод, связанный с MM. В настоящем исследовании представлен пример 59-year-old Человек с MM, который демонстрировал фолликулярный акантоз и гиперкератоз в качестве скромных индикаторов злодеяний. Пациент с историей сцены 5 В ходе клинической оценки и лабораторных исследований был диагностирован

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… Blood and platelet transfusions were also administered to manage anemia and thrombocytopenia. Additionally, a sputum cytology specimen was processed and stained using Hetropoxa-Papanicolaou staining was performed at room temperature (20-25˚C) for 15 min for cytological evaluation. …

A Focus on CAR T-Cell Therapy and Bispecific Antibodies in Multiple Myeloma.

Significant strides have been made in the management of patients living with myeloma. However, patients with multiply relapsed or refractory multiple myeloma (MM) have a shorter overall survival; therefore, new treatments with novel mechanisms of action are needed in this patient population. Patients with relapsing disease require a full restaging workup, including whole body imaging to evaluate for extramedullary disease and lytic bone lesions, as well as bone marrow biopsy with fluorescence in situ hybridization to determine if the patient has any new chromosomal changes that are present. Th

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… Regular blood or platelet transfusions are often required for up to 3 or 4 weeks after VTD-PACE is given. If patients are in a community oncology setting, close communication with the referring center should be maintained ( Brigle, 2021 ; Gray, 2021 ; Lee, 2003 ). Case Study 1 Mr. … He was seen once weekly by the advanced practitioner (AP) and scheduled for possible red blood cell transfusions (if hemoglobin < 7.0 g/dL) or platelet transfusions (if platelet count < 10 µL). …

The Ratio of the Hemoglobin to Red Cell Distribution Width Combined with the Ratio of Platelets to Lymphocytes Can Predict the Survival of Patients with Gastric Cancer Liver Metastasis.

BackgroundHemoglobin/red cell distribution width (HR) and platelet/lymphocyte (PLR) ratios are considered effective prognostic markers in various cancers. We have proposed a new prognostic parameter: HR+PLR. The aim of this study is to explore the prognostic value of the HR+PLR scoring system in patients with gastric cancer liver metastasis.MethodsThis study retrospectively analyzed the clinical data of 306 patients with gastric cancer liver metastases admitted to our hospital from 2007 to 2014. According to the size of HR value and PLR value, we will divide the patients into three groups, nam

Thalidomide for refractory gastrointestinal bleeding from vascular malformations in patients with significant comorbidities.

BackgroundRefractory gastrointestinal bleeding (GIB) secondary to gastrointestinal vascular malformations (GIVM) such as gastrointestinal angiodysplasia (GIAD) and gastric antral vascular ectasia (GAVE) remains challenging to treat when endoscopic therapy fails. Recently thalidomide has been suggested as a treatment option for refractory GIB.AimTo determine the outcome of patients treated with thalidomide for refractory GIB due to GIVM.MethodsIRB approved, single center, retrospective review of electronic medical records from January 2012 to November 2018. Patients age > 18 years old, who had

Proceedings of the RAMI Intern Section Meeting.

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Evans syndrome: clinical perspectives, biological insights and treatment modalities.

Evans syndrome (ES) is a rare and chronic autoimmune disease characterized by autoimmune hemolytic anemia and immune thrombocytopenic purpura with a positive direct anti-human globulin test. It is classified as primary and secondary, with the frequency in patients with autoimmune hemolytic anemia being 37%-73%. It predominates in children, mainly due to primary immunodeficiencies or autoimmune lymphoproliferative syndrome. ES during pregnancy is associated with high fetal morbidity, including severe hemolysis and intracranial bleeding with neurological sequelae and death. The clinical presenta

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… Steroids with and without IVIG are recommended as front-line therapy. 11 , 12 , 85 Red blood cell/platelet transfusion is indicated only in severe symptomatic patients due to the risk of exacerbations. 11 First-line treatments Steroids It is postulated that steroids inhibit the ability of macrophages …

Myelodysplasia in VEXAS syndrome.

AbstractVEXAS (vacuoles, E1 enzyme, X linked, autoinflammatory, somatic) syndrome is a clonal hematoinflammatory disorder affecting older, predominantly male patients, characterized by systemic inflammation and progressive bone marrow failure. It is caused by somatic mutations in ubiquitin-activating enzyme E1 (UBA1), an X-linked gene, essential for initiating cellular ubiquitination. Loss-of-function mutations, most commonly M41 missense variants, lead to decreased expression of the cytoplasmic isoform, UBA1b, resulting in accumulation of misfolded proteins and activation of the endoplasmic r

Existing and emerging strategies for fertility preservation in female patients with leukemia.

Leukemia, the most common malignancy in childhood, affects individuals from prepubertal girl to women of reproductive age. It originates from immature hematopoietic cells in the bone marrow and disseminates systemically via the blood and lymphatic circulations. Although survival rates have improved substantially, standard treatments, including intensive chemotherapy and hematopoietic stem cell transplantation, often combined with total body irradiation, are highly gonadotoxic. Consequently, a significant proportion of survivors experience long-term endocrine dysfunction and infertility, with f

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… In clinical practice, platelet transfusions are frequently administered before the procedure to ensure that platelet counts reach a safe threshold. According to the American Academy of Family Physicians, prior to invasive procedures, platelet count should be above 40,000-50,000/µL. … Platelet transfusion: a clinical practice guideline from the AABB . Ann Internal Med . ( 2015 ) 162 : 205–13 . doi: 10.7326/m14-1589 . PMID: 25383671 149 Gauer RL Whitaker DJ . Thrombocytopenia: evaluation and management . …

Outcome of therapy-related myeloid neoplasms treated with azacitidine.

BackgroundTherapy-related myeloid neoplasms (t-MN), including myelodysplastic syndromes and acute myeloid leukemia (t-MDS and t-AML) are associated to clinical and biologic unfavorable prognostic features, including high levels of DNA methylation.MethodsWe retrospectively evaluated 50 t-MN patients (34 MDS and 16 AML) selected among all patients receiving azacitidine (AZA) at 10 Italian Hematology Centers. Patients had developed a t-MN at a median of 6.5 years (range 1.7- 29) after treatment of the primary tumor (hematological neoplasm, 27 patients; solid tumor, 23 patients).ResultsThe overall

Assessing the Influence of Different Comorbidities Indexes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in a Developing Country.

Although the application of Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI) has enabled better prediction of transplant-related mortality (TRM) in allogeneic hematopoietic stem cell transplants (AHSCT), data from developing countries are scarce. This study prospectively evaluated the HCT-CI and the Adult Comorbidity Evaluation (ACE-27), in its original and in a modified version, as predictors of post-transplant complications in adults undergoing a first related or unrelated AHSCT in Brazil. Both bone marrow (BM) and peripheral blood stem cells (PBSC) as graft sources wer

Impact of HLA locus mismatch on peripheral blood allogeneic hematopoietic stem cell transplantation from unrelated donors using an ATG-based GVHD prophylaxis strategy.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an important treatment option for hematologic diseases. However, limited studies have evaluated the prognosis of patients receiving single human leukocyte antigen (HLA) mismatched unrelated donor allo-HSCT (HLA 9/10 MMUD-HSCT) compared to those receiving fully matched unrelated donor allo-HSCT (10/10 MUD-HSCT). This study retrospectively analyzed 126 cases of unrelated donor allo-HSCT (URD-HSCT) at our center, in which anti-human thymocyte globulin (ATG, 7.5 mg/kg) was used as a graft-versus-host disease (GVHD) prophylaxis strat

T-Cell Redirecting Therapies in Multiple Myeloma: Pathogenesis and Management of Toxicities Beyond CRS and ICANS.

The introduction of chimeric antigen receptor T (CAR-T) cell and bispecific antibody (BsAb) therapies has revolutionized multiple myeloma (MM) treatment, offering exceptional efficacy, and culminating in recent regulatory approval. However, these therapies have brought unique toxicity challenges, manifesting not only with the well-established cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), but also with the emergence of other common and equally distinctive toxicities, including cytopenias, hypogammaglobulinemia, infections, and the rare but l