Myelodysplastic syndromes/neoplasms (MDS) represent a biologically and clinically diverse group of myeloid malignancies marked by cytopenias, morphological dysplasia, and an inherent risk of progression to acute myeloid leukemia. Over the past two decades, the field has made significant advances in characterizing the molecular landscape of MDS, leading to refined classification systems to reflect the underlying genetic and biological diversity. In 2025, the treatment of MDS is increasingly individualized, guided by integrated clinical, cytogenetic, and molecular risk stratification tools. For lower-risk MDS, the treatment paradigm has evolved beyond erythropoiesis-stimulating agents (ESAs) with the introduction of novel effective agents such as luspatercept and imetelstat, as well as shortened schedules of hypomethylating agents (HMAs). For higher-risk disease, monotherapy with HMAs continue to be the standard of care as combination therapies of HMAs with novel agents have, to date, failed to redefine treatment paradigms. The recognition of precursor states like clonal hematopoiesis of indeterminate potential (CHIP) and the increasing use of molecular monitoring will hopefully enable earlier intervention/prevention strategies. This review provides a comprehensive overview of the current treatment approach for MDS, highlighting new classifications, prognostic tools, evolving therapeutic options, and ongoing challenges. We discuss evidence-based recommendations, treatment sequencing, and emerging clinical trials, with a focus on translating biological insights into improved outcomes for patients with MDS.
Tyrosine kinase inhibitors (TKIs) have significantly improved the survival of patients with chronic myeloid leukemia (CML), however, older patients are often underrepresented in pivotal trials. Approximately 20% of older adults never start treatment and face significant barriers to accomplish favorable outcomes. The treatment goal is to improve survival, prevent progression, and preserve quality of life. This is achieved through optimizing TKI doses and employing discontinuation strategies to attain treatment-free remission (TFR), a goal increasingly pursued by older patients. Imatinib may be favored as the front-line option for older individuals due to its side effect profile and cost. Bosutinib's favorable cardiovascular tolerability makes it a suitable second-line agent, but lower-dose dasatinib may likewise be an attractive option. The prevalence of comorbidities can preclude the use of second generation TKIs in some older patients. Optimal care for older patients with CML centers on personalized treatment, close monitoring, and proactive support.
Luspatercept has emerged as a novel therapy for anemia in transfusion-dependent (TD) lower-risk myelodysplastic syndromes (LR-MDS). This systematic review and meta-analysis aimed to evaluate the efficacy and safety of luspatercept in TD LR-MDS. Six databases were searched through March 2025 to find relevant material. Studies were screened and extracted by 2 independent authors. A total of 20 studies encompassing 3455 patients were included in the analysis. The pooled 8-week transfusion independence (TI) rate was 51.2% (95% confidence interval [CI], 39.9-60.4; I2 = 94.9%), with higher rates observed among ring sideroblast–positive (RS+) patients (57.8%; 95% CI, 47.4-67.7; I2 = 86%) and those with low transfusion burden (LTB; 72.9%; 95% CI, 60.4-82.6; I2 = 0%). The 12-week and 24-week TI rates were 57.0% (95% CI, 48.1-65.5; I2 = 90%) and 35.8% (95% CI, 28.7-43.6; I2 = 82.1%), respectively. Hematologic improvement–erythroid was achieved in 51.3% of patients (95% CI, 41.3%–61.2%; I2 = 93%). The most frequent adverse events were peripheral edema (17.8%; 95% CI, 11.4-26.8), diarrhea (15.6%; 95% CI, 8.2-27.7), and fatigue (11.4%; 95% CI, 5.4-22.6). Serious adverse events occurred in 28.0% of patients (95% CI, 12.8-50.7; I2 = 97.2%). Luspatercept is an effective and well-tolerated treatment for anemia in TD LR-MDS, especially in patients with RS+ and LTB. Its favorable safety profile and high TI rates, particularly in erythropoiesis-stimulating agent–naïve populations, support its use in the frontline setting.
PURPOSE: Medical podcast integration into hematology/oncology fellowship curricula remains largely unstudied. We conducted a multicenter cluster randomized trial to evaluate the educational impact of a podcast-based curriculum (PC) when delivered alongside standard didactics compared with standard didactics alone. METHODS: Twenty-seven US Accreditation Council for Graduate Medical Education–accredited hematology/oncology fellowship programs were randomized to receive either PC plus standard didactics (podcast arm, n = 14) or standard didactics alone (control arm, n = 13). The PC included curated podcast episodes and detailed show notes on breast cancer (BC), multiple myeloma (MM), bleeding disorders (BDs), and heparin-induced thrombocytopenia (HIT), developed by The Fellow on Call and Two Onc Docs . Fellows completed preintervention and postintervention comfort surveys (Likert scale 1-7) and a 16-item knowledge assessment. Coprimary end points were improvement in mean comfort (≥0.5 points) and knowledge (≥10%). Mixed-effects ANCOVA was used to adjust for baseline scores and program clustering. RESULTS: Of 420 eligible fellows, 221 (52%) completed baseline surveys and 186 (44%) completed the knowledge assessment. Ninety-nine fellows (53 podcast, 46 control) completed both postintervention assessments. Baseline comfort was similar across groups. The podcast arm demonstrated significantly greater adjusted mean improvements in comfort: BC (0.48; P < .01), MM (0.65; P < .01), BD (0.70; P < .01), and HIT (0.40; P < .01). Knowledge scores improved from 39.7% to 62.0% in the podcast arm versus 43.5% to 50.3% in the control arm (adjusted mean difference, 15.5%; P < .01). Most fellows in the podcast arm (83%) rated show notes as highly useful, and 89% planned continued podcast use. CONCLUSION: The PODCAST-HOF trial showed that adding curated podcasts and show notes significantly improved fellow comfort and knowledge. These findings support incorporating The Fellow on Call and Two Onc Docs as a recommended supplement to traditional fellowship didactics.
1 June 2019 in International Journal of Infectious Diseases
Severe malaria is an uncommon diagnosis in the United States. However, awareness of signs, symptoms, and treatment options is imperative in order to promptly initiate optimal therapy. False positive human immunodeficiency virus (HIV) results are rare in the setting of acute malaria infection and with the introduction of newer fourth-generation immunoassays. The Centers for Disease Control algorithms assist in confirming true HIV infection (Branson et al. 2014).
1 January 2017 in Respiratory Medicine Case Reports
With approximately 750 cases reported, Erdheim-Chester disease is an exceedingly rare histiocyte cell disorder. Affected sites typically include long bones, large vessels and central nervous system. However, cutaneous and pulmonary involvement can also occur. The diagnosis is ascertained by identification of foamy histiocytes positive for CD68, CD163, and factor XIIIa on immunoperoxidase staining. Recently published literature have described an association between Erdheim-Chester disease and BRAF V600E mutation. This finding prompted the investigation of therapeutic possibilities with BRAF inhibitors, successful agents against other BRAF mutation-positive diseases. Vemurafenib, a BRAF kinase inhibitor, has been shown to be effective in BRAF V600E mutation-positive malignancies, such as NSCLC and melanoma, as well as in several case reports of Erdheim-Chester disease. We report a case of Erdheim-Chester disease diagnosed at our institution, treated with vemurafenib.