Ugochi O Ogu, Ayesha Mukhopadhyay, Kruti Patel, Marquita N Nelson, KayLee S Strahan, Lin Wu, Matthew P Smeltzer, Kenneth I Ataga
Hydroxyurea reduces the frequency of vaso-occlusive complications, increases hemoglobin, and decreases mortality in sickle cell disease (SCD). Although current guidelines recommend escalation to maximum tolerated dose (MTD), the use of fixed low-dose hydroxyurea is common in low-resource countries. We conducted a systematic review and meta-analysis to evaluate the efficacy of escalated doses versus fixed low-dose of hydroxyurea in adults with SCD. Nine studies were included in the quantitative synthesis, four evaluating fixed low-dose and five evaluating escalated doses of hydroxyurea...
November 29, 2023: European Journal of Haematology
Carly Rich, Koo Wilson, Jens Olsen, Mikkel Pedersen, Henrik Frederiksen
OBJECTIVES: To establish epidemiology, healthcare costs, and labor market attachment in patients with paroxysmal nocturnal hemoglobinuria (Pt-PNH) in Denmark. METHODS: Data were from Statistics Denmark and the Danish Health Data Authority national population registers (2005-2021). Descriptive baseline statistics characterized the Pt-PNH analytic population; ordinary least squares and adjusted Cox proportional hazards regressions measured outcomes in the Pt-PNH versus Danish general population matched comparators...
November 27, 2023: European Journal of Haematology
Anil Ananthaneni, Gaelen Shimkus, Francesca Weis, Eunice Adu-Dapaah, Rachaita Lakra, Poornima Ramadas, Samina Hayat
BACKGROUND: Adult-onset Still's disease (AOSD) is a rare systemic inflammatory disorder that is characterized by quotidian fevers, arthritis, and an evanescent rash. Occurrence of concurrent thrombotic microangiopathy (TMA) in AOSD is rare. The treatment aspects of TMA in AOSD are actively being debated. METHODS: Medline search using MeSH terms and snowballing yielded a total of 29 articles with co-occurrence of AOSD and thrombotic thrombocytopenic purpura (TTP) including our own...
November 23, 2023: European Journal of Haematology
Koo Wilson, Carly Rich, Zalmai Hakimi, Regina Horneff, Jesse Fishman, Jennifer Mellor, Lucy Earl, Yasmin Taylor, Alice Simons, Joe Conyers, Brian Mulherin, Elaine Majerus, Alexander Röth
OBJECTIVES: To describe real-world use/effectiveness of pegcetacoplan (PEG) in paroxysmal nocturnal haemoglobinuria (PNH). METHODS: Data were drawn from the Adelphi PNH Disease Specific Programme™, a cross-sectional survey conducted in France, Italy, Germany, Spain and the United States from January to November 2022. Patients had a confirmed PNH diagnosis and received PEG for ≥1 month. Physicians reported patient characteristics, treatment use/satisfaction and their perception of patients' fatigue and health-related quality of life (HRQoL)...
November 23, 2023: European Journal of Haematology
Louis Nevejan, Veerle Labarque, Nancy Boeckx
BACKGROUND: Barely two per million Belgian children/adolescents are diagnosed with chronic myeloid leukemia (CML) annually. In this retrospective study, we aimed to investigate the diagnostic features, clinical and laboratory characteristics, and treatment outcome of this rare entity. METHODS: Medical records of all pediatric CML patients (age ≤ 17 years) diagnosed at the University Hospitals Leuven between 1986 and 2021 were reviewed...
November 20, 2023: European Journal of Haematology
Damian A Laber, Parth C Patel, Constantine N Logothetis, Ankita K Patel, Michael Jaglal, Mintallah Haider, Nathan Visweshwar, Geetha Rajasekaran-Rathnakumar, Jennifer Eatrides
BACKGROUND: Complement-mediated thrombotic microangiopathy (CM-TMA), also called atypical hemolytic uremic syndrome (aHUS), is a difficult-to-diagnose rare disease that carries severe morbidity and mortality. Anti-C5 monoclonal antibodies (aC5-mab) are standard treatments, but large studies and long-term data are scarce. Here, we report our single institution experience to augment the knowledge of CM-TMA treated with aC5-mab therapy. METHODS: We aimed to assess the short and long-term effects of aC5-mab in patients diagnosed with CM-TMA treated outside of a clinical trial...
November 20, 2023: European Journal of Haematology
Fredrik H Schjesvold, Heinz Ludwig, Maria-Victoria Mateos, Alessandra Larocca, Haifaa Abdulhaq, Stefan Norin, Marcus Thuresson, Nicolaas A Bakker, Paul G Richardson, Pieter Sonneveld
Melphalan flufenamide (melflufen), a first-in-class alkylating peptide-drug conjugate, plus dexamethasone demonstrated superior progression-free survival (PFS), but not overall survival (OS), versus pomalidomide plus dexamethasone in relapsed/refractory multiple myeloma in the OCEAN study. Time to progression (TTP) <36 months after a prior autologous stem cell transplantation (ASCT) was a negative prognostic factor for OS with melflufen. This post hoc exploratory analysis evaluated patients refractory to prior alkylators (e...
November 15, 2023: European Journal of Haematology
Xiaohui Zhang, Kai Zhang, Jing Zhang, Wei Chang, Yunguo Zhao, Xiaohui Suo
OBJECTIVES: As a tumor suppressor gene, SOCS3 inhibits the growth of tumor cells by regulating JAK/STAT signaling pathway through negative feedback. This study aimed to investigate the biological function and mechanism of SOCS3 methylation mediated by DNMTs in the development of AML. METHODS: Bone marrow samples were collected from 70 AML patients and 20 healthy volunteers. The expression and methylation status of each gene were detected by RT-qPCR, western blot and MS-PCR, and the growth and apoptosis rate of leukemia cell lines were detected by CCK-8 and flow cytometry...
November 11, 2023: European Journal of Haematology
Vered Stavi, Nihar Desai, Fotios V Michelis, Dennis Dong Hwan Kim, Rajat Kumar, Jeffrey Howard Lipton, Arjun Datt Law
INTRODUCTION: Pneumocystis jirovecii pneumonia (PJP) is a rare complication of hematopoietic stem cell transplantation (HSCT). Primary prophylaxis for 6-12 months post-HSCT is the standard approach. However, there is no consensus regarding the optimal duration of prophylaxis. METHODS: We identified patients who developed PJP more than 1-year post-HSCT. All patients had previously received 12 months of PJP prophylaxis. PJP was diagnosed based on clinical findings and the detection of P...
November 10, 2023: European Journal of Haematology
Benjamin J Lee, Shawn P Griffin, Jean Doh, Alexandre Chan, Stefan O Ciurea, Deepa Jeyakumar, Angela G Fleischman, Kiran Naqvi, Nicholas R Pannunzio, Susan O'Brien, Piyanuch Kongtim
OBJECTIVE: There are significant disparities in outcomes among Hispanic patients with acute lymphoblastic leukemia (ALL). Recent studies have demonstrated favorable outcomes of pegaspargase-containing ALL regimens (PEG-CAR) in young adults however, outcomes in Hispanic ethnicity continue to be underreported. METHODS: We evaluated outcomes of newly diagnosed, adult B-cell ALL Hispanic and non-Hispanic patients consecutively treated with a PEG-CAR or HyperCVAD between January 2011 and November 2022...
November 6, 2023: European Journal of Haematology
Charles E Foucar, Daniel H Foley, Jessica Aldous, Patrick W Burke, Kristen R Pettit, Lydia L Benitez, Anthony J Perissinotti, Bernard L Marini, Philip Boonstra, Dale L Bixby
Aplastic anemia (AA) is a rare bone marrow failure disorder that is treated with either allogeneic stem cell transplant or immunosuppressive therapy (IST) consisting of antithymocyte globulin (ATG), cyclosporine (CSA), and eltrombopag. While outcomes are favorable in younger patients, older patients (>60) have significantly worse long-term survival. The dose of ATG is often reduced in older patients and those with multiple comorbidities given concerns for tolerability. The efficacy and safety of dose-attenuated IST in this population is largely undescribed...
November 6, 2023: European Journal of Haematology
Sarah Bonnet, Serge Carillo, Baptiste Legrand, Barbara Burroni, Thierry Lavabre-Bertrand, Guilhem Requirand, Nicolas Robert, Lea Fornero, Ahmed Al Mansoori, Jérôme Moreaux, Guillaume Cartron, Ludovic Gabellier, Charles Herbaux
We describe the case of a patient with extreme thrombocytosis whose evolution was rapidly fatal. No cause of secondary thrombocytosis was found. There was no sign of myelofibrosis but the megakaryocytes were small and dysplastic. The patient presented a calreticulin (CALR) variant in exon 3 (C105S), as well as concomitant mutations of ASXL1, U2AF1, and EZH2. This variant of CALR has never been described before, and after sorting, all identified mutations were found in myeloid cells but not in lymphoid cells...
November 2, 2023: European Journal of Haematology
Alexandre Theocharides, Heinz Gisslinger, Valerio De Stefano, Vincenzo Accurso, Alessandra Iurlo, Timothy Devos, Miklos Egyed, Eric Lippert, Regina Garcia Delgado, Nathan Cantoni, Anders E A Dahm, Damianos Sotiropoulos, Erik Houtsma, Aoife Smyth, Amir Iqbal, Paola Di Matteo, Mike Zuurman, Peter A W Te Boekhorst
BACKGROUND: Hydroxyurea (HU) is a commonly used first-line treatment in patients with polycythemia vera (PV). However, approximately 15%-24% of PV patients report intolerance and resistance to HU. METHODS: This phase IV, European, real-world, observational study assessed the efficacy and safety of ruxolitinib in PV patients who were resistant and/or intolerant to HU, with a 24-month follow-up. The primary objective was to describe the profile and disease burden of PV patients...
October 30, 2023: European Journal of Haematology
Prashanth Ashok Kumar, Krishna Ghimire, Elio Haroun, Joseph Kassab, Ludovic Saba, Teresa Gentile, Dibyendu Dutta, Seah H Lim
Allogeneic hematopoietic stem cell transplant (allo-HSCT) is increasingly being used in the United States (US) and across the world as a curative therapeutic option for patients with certain high-risk hematologic malignancies and non-malignant diseases. However, racial and ethnic disparities in utilization of the procedure and in outcome following transplant remain major problems. Racial and ethnic minority patients are consistently under-represented in the proportion of patients who undergo allo-HSCT in the US...
October 30, 2023: European Journal of Haematology
Irit Avivi, Tamir Shragai, Efrat Luttwak, Svetlana Trestman, Yael C Cohen
OBJECTIVES: To evaluate whether low-dose belantamab mafodotin (B-MAF) dosing results in lower toxicity and better overall outcome. METHODS: We retrospectively evaluated nine consecutive patients treated with low-dose (1.9 mg/kg) B-MAF. RESULTS: The median age was 70 years. Most patients were penta-refractory. Ocular toxicity was observed in 77.7%. Adverse events resulting in treatment delays were recorded in 9 out of 124 cycles being given...
October 26, 2023: European Journal of Haematology
Nicol Garcia-Pouton, Valentín Ortiz-Maldonado, Oliver Peyrony, Mariana Chumbita, Tommaso Francesco Aiello, Patricia Monzo-Gallo, Carlos Lopera, Pedro Puerta-Alcalde, Laura Magnano, Nuria Martinez-Cibrian, Cristina Pitart, Manel Juan, Julio Delgado, Carlos Fernandez De Larrea, Álex Soriano, Álvaro Urbano-Ispizua, Carolina Garcia-Vidal
BACKGROUND: We described the real-life epidemiology and causes of infections on the different therapy phases in patients undergoing chimeric antigen receptor (CAR) T-cells directed towards CD19+ or BCMA+ cells. METHODS: All consecutive patients receiving CAR T-cell therapy at our institution were prospectively followed-up. We performed various comparative analyses of all patients and subgroups with and without infections. RESULTS: Ninety-one adults mainly received CAR T-cell therapy for acute leukaemia (53%) and lymphoma (33%)...
October 25, 2023: European Journal of Haematology
Mark T Reding, María Teresa Álvarez-Román, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg
OBJECTIVES: To assess effectiveness and safety of damoctocog alfa pegol in interim analyses of the ongoing real-world hemophilia A HEM-POWR study. METHODS: HEM-POWR (NCT03932201) is a multinational Phase 4 prospective observational study. The primary objective was annualized bleeding rate (ABR) in previously treated patients (PTPs) with hemophilia A. Secondary objectives included adverse events and number of affected joints. RESULTS: At data cut-off (August 17, 2022), the safety analysis set included 268 patients and the full analysis set (FAS) included 161 patients...
October 24, 2023: European Journal of Haematology
Enrica Antonia Martino, Antonella Bruzzese, Caterina Labanca, Francesco Mendicino, Eugenio Lucia, Virginia Olivito, Antonino Neri, Fortunato Morabito, Ernesto Vigna, Massimo Gentile
Multiple myeloma (MM) is an incurable neoplasm characterized by significant morbidity and mortality. Despite advances in treatment, MM patients eventually experienced a relapse of the disease. Penta-drug refractory patients continue to be the hard core of relapsed/refractory (RR) settings. Teclistamab-cqyv is a humanized IgG4 antibody and a bispecific BCMA-director CD3 T-cell engager. It recruits endogenous T cells, by targeting CD3 receptors expressed on their surface, resulting in their activation against BCMA, an antigen expressed by plasma cells...
October 17, 2023: European Journal of Haematology
Radwan Massoud, Hassan Naim, Evgeny Klyuchnikov, Dietlinde Janson, Christine Wolschke, Francis Ayuk, Nicolaus Kröger
OBJECTIVE: Allogeneic stem cell transplantation (allo-SCT) may have a curative potential due to the graft versus lymphoma effect. In this study, we aimed to compare transplant outcomes between refractory-T-NHL (ref-NHL) and Chemosensitive-T-NHL (CS-T-NHL). MATERIALS AND METHODS: We retrospectively reviewed the records of 26 ref-NHL and 29 CS-T-NHL consecutive patients who underwent allo-SCT at our center and compared the transplant outcomes between the groups. RESULTS: All patients were heavily pretreated with 27% of patients relapsing post-auto-SCT and two patients in the ref-T-NHL post-allo-SCT...
October 16, 2023: European Journal of Haematology
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