The clinical prognostic value of monitoring minimal residual disease (MRD) in acute myeloid leukemia (AML) patients undergoing nonintensive treatment remains insufficiently established. The aim of this work was to examine MRD status at various time points, highlighting the potential for pre-emptive therapy to improve patient outcomes.

Multiple myeloma (MM) is predominantly a disease of the elderly, but approximately 10% of patients are younger than 50 years at diagnosis.   METHODS: This study aimed to investigate the clinical characteristics, treatment outcomes, and prognostic factors in younger MM patients using retrospective data from the Balkan Myeloma Study Group registry.   RESULTS: A total of 350 patients under 50 years old were included, comprising 10.4% of the overall cohort. The study found that younger patients had lower rates of renal impairment and anemia but a higher incidence of lytic bone disease and adverse cytogenetics. Treatment regimens, including proteasome inhibitors and immunomodulatory agents, were comparable between younger and older patients, but younger patients had significantly better complete response rates and overall survival (OS). The 5- and 10-year OS rates were 76% and 64%, respectively, with a projected median OS exceeding 15 years. Factors such as anemia, hypercalcemia, and high-risk cytogenetics were associated with worse survival outcomes. Autologous stem cell transplantation (ASCT) emerged as a key contributor to improved progression-free survival (PFS) and OS.

Anti-CD19 CAR-T therapy has been a breakthrough in treatment of primary refractory or relapsed large B-cell lymphoma (r/r LBCL) and is poised to supplant previous second line of high dose chemotherapy and autologous stem cell transplantation (HDT/ASCT). However, in clinical practice, high risk patients with chemoimmunotherapy sensitive disease continue to receive salvage chemoimmunotherapy or cannot access CAR-T in a timely manner and thus may still proceed to HDT/ASCT. Little is known about clinical outcomes of CAR-T in patients who receive HDT/ASCT compared to those who are transplant-naïve.

Myelodysplastic syndromes/neoplasms (MDS) are a diverse group of clonal myeloid disorders. Advances in molecular technology lead to the development of new classification systems. However, large-scale epidemiological studies on MDS in Asian countries are currently scarce.

In vivo T-cell depletion with antithymocyte globulin (ATG), especially at high-doses has been shown to be associated with increased incidence of infections after allogeneic hematopoietic stem cell transplantation (HSCT). However, it remains unclear whether ATG, even at low-doses increases the risk of posttransplant infections in the high-risk HSCT setting.

To analyze the impact of tyrosine kinase inhibitor (TKI) maintenance therapy following allogeneic hematopoietic stem cell transplantation (HSCT) in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) on recurrence rates and prognosis for the 2 transcripts, p190 and p210.

Therapy-related myeloid neoplasms (t-MN), which include acute myeloid leukemia (t-AML), myelodysplastic syndrome (t-MDS), and myelodysplastic/myeloproliferative neoplasms are secondary malignancies occurring as a late complication following chemotherapy or radiation therapy for an antecedent disorder. Allogeneic hematopoietic stem cell transplant (allo-HCT) is a potentially curative treatment option in t-MN patients. This systematic review and meta-analysis aimed to explore the outcomes of allo-HCT in t-MN. Following PRISMA guidelines, a comprehensive literature search was performed on PubMed, Cochrane, and Clinicaltrials.gov. Survival data were extracted from Kaplan-Meier curves to calculate overall survival (OS) and disease-free survival (DFS) probabilities. A total of 7785 (t-AML: 67.3%, t-MDS: 26.5%, and mixed presentation: 6%) patients from 33 original studies reporting outcomes of allo-HCT in t-MN patients were included for analysis. The patients age ranged from 2 to 89 years, and 61.7% were female. The pooled median OS was 16.9 months (95% CI: 13.7-21.1), whereas the estimated mean OS was 46.0 months (95% CI: 42.1-49.6). The pooled median DFS was 8.8 months (95% CI: 7.4-11.2), and the mean DFS was 35.5 months (95% CI: 33.4-41.9). The pooled proportion of acute graft-versus-host disease (aGvHD) was 34% (95% CI: 0.35-0.45, I: 91.71%, P < .0001). Relapse of the myeloid neoplasm was the most common cause of mortality, followed by infections, relapse of the underlying disease, and GvHD. Despite these challenges, allo-HCT remains a potential treatment option with promising outcomes for carefully selected t-MN patients.

Measurement of 24- hour urine protein electrophoresis (PEP) and immunofixation (IFE) are part of standard diagnostic evaluation and monitoring of patients with suspected and diagnosed monoclonal plasma cell disorder for baseline evaluation of renal dysfunction or nephrotic syndrome. Measurement of 24-hour urine protein can however be time consuming and cumbersome and many centers have moved towards random urine protein measurements. The evidence for correlation between spot urine protein creatinine ratio (SUPC) and 24-hour urine protein measurements its scarce. Therefore, we have carried out a prospective study with a sample size of 40 multiple myeloma (MM) patients to demonstrate this correlation. Our results suggest a good correlation between SUPC and 24-hour urine protein measurements, suggesting SUPC testing is a reliable and easier alternative to 24-hour urine collection. These findings should now be confirmed in larger patient populations including early stage plasma cell disorders, light chain amyloidosis and symptomatic MM.

Immune effector cell-associated neurotoxicity syndrome (ICANS) can be a severe, life-threatening toxicity following CAR T-cell therapy. While currently evaluated by the immune effector cell-associated encephalopathy (ICE) score, not all patients have changes in their ICE score and not all signs and symptoms of neurotoxicity are captured.

To describe tyrosine kinase inhibitor (TKI) treatment patterns and analyze co-variates of TKI switch for chronic myeloid leukemia (CML) patients in a center from China.

High dose chemotherapy and autologous stem cell transplantation (HDT/ASCT) remains the preferred first line consolidation strategy for newly diagnosed multiple myeloma (MM). However, The role of HDT/ASCT in first relapse is uncertain in the context of novel therapies. This study evaluates real-world outcomes of MM patients in first relapse, focusing on the role of consolidative HDT/ASCT.

Invasive fungal disease (IFD) poses significant challenges for critically ill patients with hematological malignancies (HMs). However, there is limited research on the clinical characteristics, risk factors, and outcomes of IFD within this population.

This analysis explored real-world characteristics, treatment patterns and clinical outcomes in patients with relapsed or refractory multiple myeloma (RRMM) previously treated with lenalidomide and an anti-CD38 monoclonal antibody (mAb) and requiring subsequent treatment.

Multiple myeloma (MM) clinical management is challenging owing to its relapse and refractoriness to treatment. Understanding the treatment patterns and refractory dynamics is crucial for optimizing patient care. This study aimed to estimate the evolution of MM according to the treatment line and refractoriness status in Italy.

Multiple myeloma treatment has evolved rapidly with the development of novel targeted therapies. The paper outlines multiple myeloma epidemiology, current treatments, and recent advances, highlighting the role of bispecific antibodies. Brazilian authorities have approved 3 bispecific antibodies (teclistamab, elranatamab, and talquetamab) for relapsed/refractory multiple myeloma patients who have received at least three prior therapies. These therapies have shown promising efficacy in clinical trials, with 61%-74% overall response rates. However, access to these treatments varies significantly between Brazil's private and public healthcare systems. A panel of 6 Brazilian experts in multiple myeloma and bispecific antibody therapy convened for a three-day virtual conference organized and moderated by Americas Health Foundation. They addressed key questions regarding bispecific antibody therapy in multiple myeloma and developed consensus recommendations. While bispecific antibodies offer new hope for multiple myeloma patients, challenges remain in ensuring equitable access to these therapies. The paper discusses the sequencing of bispecific antibodies with other treatments, the management of adverse events, and the need for real-world data. It also highlights the disparities in multiple myeloma treatment between Brazil's public and private healthcare systems, emphasizing the need for targeted efforts to bridge this gap and improve outcomes for all multiple myeloma patients.

Chronic myeloid leukemia (CML) treatment has significantly evolved with the introduction of tyrosine kinase inhibitors. However, access to these treatments and outcomes vary globally. This study examines 2 decades of CML management in Colombia using the RENEHOC registry, focusing on TKI efficacy, safety, and healthcare system challenges.

'Standard of care' therapies for adult acute myeloid leukemia (AML) have yielded 5-year overall survival (OS) rates of 30%-45 %. Risk stratification and novel targeted therapies have improved 5-year OS rates to >75 % for certain groups in specialized centers.

Myelofibrosis (MF) is a rare myeloproliferative neoplasm characterized by progressive bone marrow fibrosis and splenomegaly. Ruxolitinib is the standard-of-care first-line treatment option for MF. This review summarizes real-world effectiveness and safety of ruxolitinib in more than 4500 patients with MF from real-world settings, including expanded-access and phase 4 trials, as well as registry, postmarketing, and retrospective studies in the 10 years since regulatory approval. Consistent with results from the phase 3 COMFORT trials, real-world evidence supports the effectiveness of ruxolitinib in improving splenomegaly and MF symptoms while significantly increasing overall survival. Real-world safety data have also been aligned with results from the COMFORT trials. Transient anemia, thrombocytopenia, and infections are the most frequently observed adverse events (AEs) but rarely required ruxolitinib discontinuation. Other nonhematologic AEs are generally mild, and grade ≥ 3 events rarely occur. Importantly, real-world evidence also supports the effectiveness of ruxolitinib in patient groups that were poorly represented in clinical trials, including those with lower-risk MF, those presenting with thrombocytopenia or anemia, and those who have previously discontinued ruxolitinib treatment. Finally, cost-effectiveness analyses show ruxolitinib to be cost-effective in Europe and North America. Taken together, real-world studies reinforce the efficacy, safety, and cost-effectiveness of ruxolitinib for the treatment of patients with MF, supporting results from prospective clinical trials. Furthermore, they demonstrate the clinical benefit of ruxolitinib in patient subgroups poorly represented in clinical trials and the value of dose modifications or re-treatment after interruptions to optimize outcomes.

BCR::ABL1-negative myelo-proliferative neoplasms (MPNs) are characterized by mutations in JAK2, CALR, or MPL. Usually these mutations are co-exclusive of each other and of BCR::ABL1. We reviewed clonal interactions in 177 subjects with mutations in JAK2, CALR, or MPL and BCR::ABL1 including JAK2/BCR::ABL1 (N = 142), CALR/BCR::ABL1 (N = 31), MPL/BCR::ABL1 (N = 3). Co-mutations can arise in the same clone or in different sub-clones. In this review we used clonality data, mutation sequencing and therapy response evaluation to address this question. We found that in subjects with JAK2/BCR::ABL1 co-mutations there is a complex, branched clonal evolution. In contrast, in subjects with CALR/BCR::ABL1, co-mutations are in different sub-clones. There are too few data in subjects with MPL/BCR::ABL1 to critically analyze. However, indirect methods for assessing clonality limit our conclusions. Understanding clonal architecture of MPNs with co-mutations is needed to understand the underlying biology and give appropriate therapy.

Hematopoietic stem cell transplantation (HSCT) is a potentially curative option for adults with acute lymphoblastic leukemia (ALL) who have achieved remission. This systematic review and meta-analysis compare the efficacy of total body irradiation (TBI) versus chemotherapy (CHT) based regimens for conditioning in adult ALL patients being prepared for HSCT. A comprehensive literature search was conducted in MEDLINE, Embase, the Cochrane Library, and relevant trial registries from their inception to August 2024. The inclusion criteria encompassed all randomized controlled trials (RCTs) and cohort studies that compared TBI with CHT as conditioning regimens prior to HSCT in adult patients with ALL. The primary outcomes assessed were overall survival (OS) and event-free survival (EFS). All statistical analyses were carried out using RevMan version 5.4, using a random-effects model. This meta-analysis includes 20 cohort studies and one RCT. The relative risk (RR) for OS was 1.37 (95% CI: 1.20-1.57), while the RR for EFS was 1.28 (95% CI: 1.15-1.43), highlighting the superior efficacy of TBI-based regimens in this patient population. TBI was also superior in terms of relapse rate (RR 0.71). The 2 regimens were comparable in terms of nonrelapse mortality, acute graft-versus-host disease (GVHD), and chronic GVHD. When used for conditioning prior to HSCT, TBI-based conditioning regimens demonstrate superior OS, EFS, and relapse outcomes compared to CHT-based regimens.