Gene Therapy Approaches for Sickle Cell Anemia
Sickle cell disease (SCD) has a widely variable clinical course; some of them are severely affected in childhood while in others major damage does not manifest until adulthood. There is no test that can be used to predict the disease course. The best predictor of SCD severity is a patient ’s course to date [1]. Patients’ quality of life is low and life expectancy is relatively short, nearly 50 years, even newly developed treatments such as iron chelating and disease modifying agents. The only curative treatment option is allogeneic stem cell transplantation (Allo-SCT), but only o ne fifth of the patients have matched s...
Source: Transfusion and Apheresis Science - February 24, 2023 Category: Hematology Authors: Turgay Ulas, Mehmet Sinan Dal Source Type: research
Extracorporeal photopheresis in conditions of autoimmunity
Autoimmune disorders (AID) are chronic conditions that involve the loss of immunological tolerance to self-antigens, and result in systemic or tissue-specific damage. Current treatment regimens for AID are often nonspecific and involve long-term administration of immunosuppressive agents such as corticosteroids, calcineurin inhibitors, and cytotoxic chemotherapeutics[1]. While effective, these medications render the patient vulnerable to opportunistic infections, as well as increasing long-term risk of secondary malignancy[2]. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 24, 2023 Category: Hematology Authors: Christopher Webb Tags: Review Source Type: research
Apheresis in pediatric patients: Current differences and difficulties
Pediatric patients require tailored therapy adjusted to their age and condition and someone to stand for their rights as they are politically vulnerable. Apheresis procedures performed on the pediatric patient group is challenging like elderly or pregnant patients. Differences from adult group and difficulties during the apheresis procedure may be discussed in four major groups: lack of evidence for apheresis in pediatrics, rapidly changing body weight and height alongside small proportions of newborns and infants, continuous embodiment and development of organ systems forcing health care providers to pay extra attention f...
Source: Transfusion and Apheresis Science - February 24, 2023 Category: Hematology Authors: Ba şak Adaklı Aksoy Source Type: research
COVID 19 convalescent plasma: is there still a place for CCP?
Convalescent plasma has been used for a long time for the treatment of various infectious diseases. The principle is to collect antibody-containing plasma from recovered patients and to transfuse the plasma to infectious patients thereby modifying their immune system. This approach was also used in the SARS-CoV-2 pandemic when no specific drugs were available for the treatment of the disease. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 24, 2023 Category: Hematology Authors: Rainer Moog Tags: Review Source Type: research
The role of plasmapheresis in the pulmonary-renal syndrome
A pulmonary renal syndrome is diagnosed when the combination of a rapid progressive glomerulonephritis (RPGN) and diffuse pulmonary hemorrhage (DAH) are present. Both are severe disease manifestations that are associated with a considerable morbidity and mortality. Most frequently, the underlying cause is a small vessel vasculitis due to anti-neutrophil cytoplasmatic antibody (ANCA) associated vasculitis (AAV) or anti-glomerular basement membrane disease. Other vasculitis ’s and connective tissue diseases such as Systemic Lupus Erythematosus and Rheumatoid Arthritis present the more rare causes of this syndrome. (Source:...
Source: Transfusion and Apheresis Science - February 24, 2023 Category: Hematology Authors: Kaatje le Poole, Hans Vrielink Tags: Review Source Type: research
Alirocumab in lipoprotein apheresis: a synergy for patients with high-Lp(a)
Until today Lipoprotein Apheresis (LA) is considered the most effective treatment for patients with high-Lp(a) and proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) are often combined with LA to dampen the rebound in lipoprotein concentrations.The aim of the present work is to evaluate the effect of dose-adjustment strategy for alirocumab in a small cohort of high-Lp(a) subjects with ischemic heart disease and in chronic LA treatment. Chronic LA effect on Lp(a) levels is a significant reduction in pre-LA Lp(a) concentrations compared to native Lp(a) value (118 [116-119] mg/dl vs 150 [137-155] mg/dl; p (Sour...
Source: Transfusion and Apheresis Science - February 21, 2023 Category: Hematology Authors: Francesco Sbrana, Federico Bigazzi, Andrea Ripoli, Beatrice Dal Pino Tags: Short Report Source Type: research
Does ferritin level affect the outcomes of autologous stem cell transplantation equally in all diseases?
In this study, 170 patients with measured ferritin levels within one month before transplantation who underwent ASCT with the diagnosis of MM or lymphoma were evaluated. The cut-off value of ferritin was determined as 500ng/mL to evaluate the transplant outcomes in both groups. The hematological recovery status/duration, febrile neutropenia rate, hospitalization time, transplant-related mortality (TRM) in the first 100 days, and OS were evaluated according to the ferritin level. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 16, 2023 Category: Hematology Authors: Ay şe Uysal, Mehmet Ali Erkurt, İrfan Kuku, Emin Kaya, Ahmet Sarıcı, İlhami Berber, Soykan Biçim, Emine Hidayet, Ahmet Kaya, Süleyman Arslan, Salih Cırık Source Type: research
Acute hemolytic transfusion reaction following ABO-mismatched platelet transfusion: two case reports
Acute hemolytic transfusion reaction following ABO-incompatible platelet transfusion: two case reportsAn ideal platelet transfusion should provide ABO identical platelet concentrate, and cross match compatibility is not routinely performed in the standard practices. However, ABO non identical platelet transfusions are not uncommon with the limited resources and short shelf life of platelet concentrate. Though rare, acute hemolytic transfusion reaction (AHTR) may occur following minor ABO-incompatible platelet transfusion. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 15, 2023 Category: Hematology Authors: Razan Hayati Zulkeflee, Mohd Nazri Hassan, Rosline Hassan, Nur Ilyia Syazwani Saidin, Zefarina Zulkafli, Marini Ramli, Marne Abdullah, Salfarina Iberahim, Wardah Roslan, Noor Haslina Mohd Noor, Wan Suriana Wan Ab Rahman Source Type: research
Pregnancy-induced severe hypertriglyceridemia effectively treated with therapeutic plasmapheresis
Hypertriglyceridemia is associated with significant morbidity during pregnancy. Hypertriglyceridemia-induced pancreatitis (HTGP) is associated with genetically determined dyslipidemia or a secondary condition such as diabetes, alcohol, pregnancy, or medication use. The lack of data on the safety of drugs to be used to decrease triglyceride levels during pregnancy dictates that other strategies must be chosen. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 14, 2023 Category: Hematology Authors: Gustavo de Carvalho Duarte, Lauren Farnden, Ruth C.E. Hughes, Huan Chan Tags: Case Report Source Type: research
Pregnancy-induced Severe Hypertriglyceridemia effectively treated with Therapeutic Plasmapheresis.
Hypertriglyceridemia is associated with significant morbidity during pregnancy. Hypertriglyceridemia-induced pancreatitis (HTGP) is associated with genetically determined dyslipidemia or a secondary condition such as diabetes, alcohol, pregnancy, or medication use. The lack of data on the safety of drugs to be used to decrease triglyceride levels during pregnancy dictates that other strategies must be chosen. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 14, 2023 Category: Hematology Authors: Gustavo de Carvalho Duarte, Lauren Farnden, Ruth C.E. Hughes, Huan Chan Tags: Case Report Source Type: research
Potential for prolongation of fibrinogen concentrates post-reconstitution
and ObjectivesReconstituted fibrinogen concentrate is considered stable for 8-24hours based on product monographs. Given the long half-life of fibrinogen in vivo (3-4 days), we hypothesized that reconstituted sterile fibrinogen protein would remain stable longer than 8-24hours. Extending the expiry date for reconstituted fibrinogen concentrate could decrease wastage and facilitate reconstitution in advance to minimize turnaround times. We performed a pilot study to define the stability of reconstituted fibrinogen concentrates over time. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 14, 2023 Category: Hematology Authors: Robert J. Guo, Tyler Smith, David Zamar, Jacqueline D. Trudeau, Andrew W. Shih Source Type: research
Caplacizumab as rescue therapy in refractory TTP involving neurologic features
The role of caplacizumab in the routine treatment of immune thrombotic thrombocytopenic purpura (iTTP) remains to be established. (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 10, 2023 Category: Hematology Authors: Minh-Ha Tran, Lisa X Lee, Yen Cao, Lan Vu, Zahra Pakbaz Tags: Case Report Source Type: research
Examining Participant Representation by Sex, Race, Ethnicity and Age in United States Blood Donation and Blood Transfusion Clinical Trials
Significant inequities based on sex, race, ethnicity, and age exist among participants in clinical trials dedicated to investigating medical disease states. While general demographic data regarding blood donors and blood transfusion recipients have been studied extensively, the demographics of participants involved in blood donation and blood transfusion clinical trials are unknown. We performed a cross-sectional analysis of United States (U.S.) -based interventional blood donation and blood transfusion clinical trials registered with Clinicaltrials.gov to ascertain the composition of participants ’ sex, race, ethnicity,...
Source: Transfusion and Apheresis Science - February 9, 2023 Category: Hematology Authors: Roshell Muir, Jeremy W. Jacobs, Laura E. Flores, Ariela L. Marshall, Eman Samih Ansari, Garrett S. Booth, Julie K. Silver Source Type: research
Perioperative Therapeutic Plasma Exchange in a Patient with Rare Factor XIII Inhibitor
We report a case of acquired Factor XIII inhibitor in a 75-yearold male with a suspicious left renal mass treated perioperatively with therapeutic plasma exchange (TPE). (Source: Transfusion and Apheresis Science)
Source: Transfusion and Apheresis Science - February 8, 2023 Category: Hematology Authors: Joshua Smith, Jared S. Bodine, Mark T. Cunningham, Kathleen Gooley, Frederick V. Plapp, Amitava Dasgupta, Zhan Ye Tags: Case Report Source Type: research
The Italian registry of therapeutic apheresis: year of activity 2021
In 2019, the Italian National Blood Center (NBC), at the request of the Italian Scientific Society of Haemapheresis and Cell Manipulation (SIdEM), included the Italian Registry of Therapeutic Apheresis (IRTA) in the Information System of Transfusion Services (SISTRA), whose activity is coordinated by the NBC.The IRTA provides institutions and scientific societies with a wide range of information including therapeutic procedures and outcomes of treated patients.The Italian National Health Service offers therapeutic apheresis for patients with various conditions, but it is mainly the patient with haematological and/or neurol...
Source: Transfusion and Apheresis Science - February 6, 2023 Category: Hematology Authors: Giustina de Silvestro, Liviana Catalano, Giuseppe Marano, Vanessa Piccinini, Livia Cannata, Angelo Ostuni, Vincenzo de Angelis Source Type: research
