Onco-hematology
ATS Guidelines on Invasive Pulmonary Aspergillosis and Antifungal Strategies in Critically Ill Adults
7 Jan, 2025 | 12:29h | UTCIntroduction: This summary provides an overview of a recent American Thoracic Society clinical practice guideline addressing two core questions in adult pulmonary and critical care practice. First, it examines whether combination therapy with a mold-active triazole (most data concern voriconazole, though newer agents such as isavuconazole or posaconazole may also be considered) plus an echinocandin (specifically caspofungin, micafungin, or anidulafungin) offers added benefit over mold-active triazole monotherapy for patients with proven or probable invasive pulmonary aspergillosis (IPA). Second, it evaluates whether routine use of prophylactic or empiric antifungal agents against Candida species is advisable in critically ill, nonneutropenic, nontransplant patients at risk of invasive candidiasis (IC). By synthesizing available evidence using the GRADE approach, this guideline aims to support clinicians in optimizing therapeutic strategies and improving patient outcomes in these complex infections.
Key Recommendations:
Initial Combination Therapy vs. Monotherapy for IPA
- For patients with proven or probable IPA, the guideline makes a conditional recommendation, meaning the best choice isn’t entirely clear. Both initial combination therapy (mold-active triazole + echinocandin) and monotherapy (mold-active triazole alone) are considered reasonable options.
- Evidence stems primarily from studies in hematologic malignancy (HM) or hematopoietic stem cell transplant (HSCT) recipients, with mixed findings in observational cohorts and a key randomized trial favoring combination therapy, particularly in a subgroup diagnosed by positive galactomannan assays.
- When critical illness or triazole resistance is a concern, combination therapy may be considered, but there is insufficient evidence to categorically endorse one approach over the other.
Prophylactic or Empiric Antifungal Therapy for Candida in Critically Ill Patients
- In nonneutropenic, nontransplant adult ICU patients at risk for IC, the guideline makes a conditional recommendation against routinely using prophylactic or empiric antifungal therapy. This means the benefits of withholding these treatments likely outweigh the risks, but there’s still some uncertainty.
- Low-quality evidence from multiple randomized controlled trials showed no significant mortality benefit in administering antifungals prophylactically or empirically compared with placebo.
- Although IC carries substantial morbidity and mortality, its overall incidence in this population remains low, and ongoing surveillance or targeted diagnostics may be preferable to universal antifungal administration.
Conclusion: The panel emphasizes that these recommendations should be applied with clinical judgment, especially in patients with severe disease, likely high fungal burden, or concerns for antifungal resistance. Combination therapy for IPA may be particularly relevant when critical illness or limited triazole efficacy is suspected. Meanwhile, prophylactic or empiric anti-Candida therapy in the broader ICU setting does not appear to substantially reduce mortality. Continued advances in rapid diagnostics, close monitoring of local resistance patterns, and new antifungal agents may further refine best practices. Future research should focus on validating these findings in diverse patient populations, exploring novel combination regimens, and establishing more precise risk assessments for IC in the ICU.
Reference: Epelbaum O, Marinelli T, Haydour Q, Pennington KM, Evans SE, Carmona EM, Husain S, Knox KS, Jarrett BJ, Azoulay E, Hope WW, and others. “Treatment of Invasive Pulmonary Aspergillosis and Preventive and Empirical Therapy for Invasive Candidiasis in Adult Pulmonary and Critical Care Patients: An Official American Thoracic Society Clinical Practice Guideline.” American Journal of Respiratory and Critical Care Medicine (2025). https://doi.org/10.1164/rccm.202410-2045ST
RCT: Daratumumab Monotherapy Prevents Progression in High-Risk Smoldering Multiple Myeloma
26 Dec, 2024 | 15:44h | UTCBackground: Smoldering multiple myeloma (SMM) is an asymptomatic plasma cell disorder that can progress to active multiple myeloma, especially when risk factors place patients in a high-risk subset. Although daratumumab has been approved for multiple myeloma, no treatments have been approved for high-risk SMM. This study (AQUILA) examined whether subcutaneous daratumumab could prevent or significantly delay progression to symptomatic myeloma.
Objective: To evaluate the effectiveness of subcutaneous daratumumab monotherapy versus active monitoring in prolonging time to disease progression (defined by SLiM–CRAB criteria) or death in patients with high-risk SMM.
Methods: In this open-label phase 3 trial, 390 patients with high-risk SMM were randomly assigned (1:1) to either daratumumab (1800 mg subcutaneously) or active monitoring. Daratumumab was administered weekly for cycles 1–2, every two weeks for cycles 3–6, and then every four weeks for up to 39 cycles (36 months) or until confirmed disease progression. Active monitoring followed the same schedule for disease evaluations without any specific therapy. The primary endpoint was progression-free survival (PFS), assessed by an independent committee. Secondary endpoints included overall survival, response rates, and time to subsequent therapy.
Results: After a median follow-up of 65.2 months, disease progression or death occurred in 34.5% of patients in the daratumumab group compared to 50.5% in the active-monitoring group (HR, 0.49; 95% CI, 0.36–0.67; p<0.001). At five years, the PFS rate was 63.1% with daratumumab and 40.8% with active monitoring. Overall survival was also higher in the daratumumab arm: 93.0% versus 86.9% at five years (HR for death, 0.52; 95% CI, 0.27–0.98). Treatment discontinuation due to adverse events was low (5.7%), and no new safety signals emerged. Grade 3 or 4 adverse events, primarily hypertension (5.7% vs. 4.6%), occurred at similar rates in both arms. Infections of grade 3 or 4 were more frequent with daratumumab (16.1% vs. 4.6%), including COVID-19 pneumonia, yet overall tolerability remained acceptable. Patient-reported outcomes, including quality-of-life measures, were largely preserved in both groups during the study.
Conclusions: Subcutaneous daratumumab monotherapy substantially delayed progression to symptomatic multiple myeloma and improved overall survival among patients with high-risk SMM. The safety profile was consistent with prior daratumumab studies, suggesting a favorable risk–benefit balance. Early intervention with daratumumab may thus alter the disease trajectory for select patients, sparing them from end-organ damage and improving long-term clinical outcomes.
Implications for Practice: While active monitoring has been the standard of care for high-risk SMM, these findings support early therapeutic intervention for patients with multiple high-risk features. Clinicians should remain cautious, however, when generalizing across different risk stratification models. Additional research on optimal treatment durations, combination strategies, and real-world outcomes will further refine patient selection and management of high-risk SMM.
Study Strengths and Limitations: This trial featured robust follow-up (median of over five years) and clear outcome definitions. However, the classification of high-risk features has evolved, and certain populations (e.g., Black patients) were underrepresented. These factors may limit the generalizability of the findings in broader clinical settings.
Future Research: Ongoing trials are investigating alternative dosing schedules, combination regimens (e.g., daratumumab-based quadruplets), and the role of minimal residual disease monitoring to optimize patient outcomes. Additional studies will clarify whether more intense or shorter treatments might maintain efficacy with fewer side effects.
Reference: Dimopoulos MA, Voorhees PM, Schjesvold F, Cohen YC, Hungria V, Sandhu I, Lindsay J, +29, for the AQUILA Investigators. Daratumumab or Active Monitoring for High-Risk Smoldering Multiple Myeloma. New England Journal of Medicine. 2024; DOI: http://doi.org/10.1056/NEJMoa2409029
Nonrandomized Phase 1b–2 Study: CAR T-cell Therapy Obecabtagene Autoleucel Effective with Low Severe Toxicity in Adult B-cell ALL
4 Dec, 2024 | 12:04h | UTCBackground: Relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) in adults has a poor prognosis, with current therapies often requiring allogeneic stem-cell transplantation for durable responses. CAR T-cell therapies targeting CD19 have shown promise but are associated with significant toxic effects.
Objective: To evaluate the efficacy and safety of obecabtagene autoleucel (obe-cel), a novel autologous 41BB-ζ anti-CD19 CAR T-cell therapy designed to reduce toxic effects and improve persistence, in adults with relapsed or refractory B-cell ALL.
Methods: In this nonrandomized, phase 1b–2 multicenter study, 127 adults aged 18 years or older with relapsed or refractory CD19-positive B-cell ALL received at least one infusion of obe-cel. The primary endpoint was overall remission (complete remission or complete remission with incomplete hematologic recovery) in cohort 2A (patients with morphologic disease). Secondary endpoints included event-free survival, overall survival, and safety assessments.
Results: Among the 94 patients in cohort 2A (median follow-up of 20.3 months), overall remission occurred in 77% (95% CI, 67–85%), with 55% achieving complete remission and 21% achieving complete remission with incomplete hematologic recovery. The prespecified null hypotheses were rejected (P < 0.001). In the total infused population, the median event-free survival was 11.9 months (95% CI, 8.0–22.1), and the median overall survival was 15.6 months (95% CI, 12.9–NE). Grade 3 or higher cytokine release syndrome occurred in 2.4% of patients, and grade 3 or higher immune effector cell–associated neurotoxicity syndrome (ICANS) occurred in 7.1%.
Conclusions: Obe-cel demonstrated a high incidence of durable responses with a low incidence of severe immune-related toxic effects in adults with relapsed or refractory B-cell ALL.
Implications for Practice: Obe-cel may offer an effective CAR T-cell therapy option with manageable toxicity for adult patients with relapsed or refractory B-cell ALL. However, long-term benefits and direct comparisons with existing therapies require further investigation. Clinicians should consider the potential advantages but remain cautious given the lack of randomized controlled data.
Study Strengths and Limitations: Strengths include the multicenter design and substantial patient population. Limitations involve the nonrandomized, single-arm design without a control group, and potential bias from patients who did not receive the infusion due to disease progression or manufacturing failures. Additionally, longer follow-up is needed to fully assess durability and late-onset toxicities.
Future Research: Further studies are warranted to compare obe-cel directly with standard therapies in randomized controlled trials, assess long-term outcomes, and explore its efficacy in earlier lines of treatment or in combination with other modalities.
Phase 2 RCT: Axatilimab Demonstrates Efficacy in Refractory Chronic GVHD by Targeting CSF1R-Dependent Macrophages
19 Sep, 2024 | 16:05h | UTCBackground: Chronic graft-versus-host disease (GVHD) is a significant long-term complication of allogeneic hematopoietic stem-cell transplantation, affecting approximately half of recipients and leading to substantial morbidity and mortality. Standard therapies often fail to induce durable responses in patients with refractory or recurrent disease. CSF1R-dependent monocytes and macrophages are key mediators of chronic GVHD, contributing to inflammation and fibrosis. Axatilimab, a CSF1R-blocking antibody, has shown promising activity in early studies.
Objective: To evaluate the efficacy and safety of axatilimab at three different doses in patients with recurrent or refractory chronic GVHD.
Methods: In this phase 2, multinational, randomized study (AGAVE-201), 241 patients aged ≥2 years with active chronic GVHD after at least two prior systemic therapies were randomized 1:1:1 to receive intravenous axatilimab at 0.3 mg/kg every 2 weeks (n=80), 1 mg/kg every 2 weeks (n=81), or 3 mg/kg every 4 weeks (n=80). Randomization was stratified by chronic GVHD severity and prior use of FDA-approved therapies (ibrutinib, ruxolitinib, or belumosudil). The primary endpoint was overall response rate (complete or partial response) within the first six cycles. The key secondary endpoint was a patient-reported reduction in symptom burden, defined as a decrease of more than 5 points on the modified Lee Symptom Scale (range 0–100).
Results: The overall response rate was 74% (95% CI, 63%–83%) in the 0.3 mg/kg group, 67% (95% CI, 55%–77%) in the 1 mg/kg group, and 50% (95% CI, 39%–61%) in the 3 mg/kg group, exceeding the predefined efficacy threshold in all groups. A clinically meaningful reduction in symptom burden was reported in 60%, 69%, and 41% of patients in the respective dose groups. Median time to response was less than 2 months across all groups. Organ-specific responses were observed in all affected organs, including skin, lungs, joints, and fascia.
The most common adverse events were dose-dependent transient laboratory abnormalities related to CSF1R blockade, such as elevations in liver enzymes and creatine kinase, which were not associated with clinical symptoms or end-organ damage. Periorbital edema occurred more frequently at higher doses. Adverse events leading to discontinuation occurred in 6% of patients in the 0.3 mg/kg group, 22% in the 1 mg/kg group, and 18% in the 3 mg/kg group. Serious infections were reported but were not dose-dependent.
Conclusions: Axatilimab demonstrated significant efficacy in patients with heavily pretreated recurrent or refractory chronic GVHD, with the highest response rates and best tolerability observed at the lowest dose tested (0.3 mg/kg every 2 weeks). Targeting CSF1R-dependent monocytes and macrophages may represent a novel therapeutic strategy in chronic GVHD.
Implications for Practice: Axatilimab offers a potential new treatment option for patients with chronic GVHD refractory to standard therapies, including those who have failed prior FDA-approved treatments. Clinicians should consider axatilimab as a therapeutic option while monitoring for transient laboratory abnormalities associated with CSF1R blockade. The lower dose appears to provide optimal efficacy with fewer adverse events.
Study Strengths and Limitations: Strengths include the randomized, multinational design and inclusion of patients with severe, refractory chronic GVHD who had received multiple prior therapies. Limitations include the lack of a comparator group, which may introduce outcome-reporting bias, and the small sizes of subgroups, limiting the generalizability of certain findings.
Future Research: Further studies are needed to confirm these results, assess long-term outcomes, and explore axatilimab in earlier lines of therapy and in combination with other treatments. Investigations into the use of axatilimab in other autoimmune diseases characterized by CSF1R-driven macrophage-mediated inflammation and fibrosis are also warranted.
RCT: Belantamab Mafodotin Combination Improves Progression-Free Survival in Relapsed/Refractory Multiple Myeloma Compared to Daratumumab-Based Therapy – N Engl J Med
18 Aug, 2024 | 19:03h | UTCStudy Design and Population: This phase 3, open-label, randomized controlled trial compared the efficacy and safety of belantamab mafodotin, bortezomib, and dexamethasone (BVd) versus daratumumab, bortezomib, and dexamethasone (DVd) in 494 patients with relapsed or refractory multiple myeloma after at least one prior therapy. Patients were randomly assigned to the BVd group (243) or the DVd group (251) and were followed for a median of 28.2 months.
Main Findings: The BVd regimen significantly improved median progression-free survival (36.6 months vs. 13.4 months; HR, 0.41; P<0.001) compared to the DVd regimen. Overall survival at 18 months was also higher in the BVd group (84% vs. 73%). The BVd group showed a higher rate of complete response or better plus MRD-negative status (25% vs. 10%). However, the BVd regimen was associated with a higher incidence of grade 3 or higher adverse events (95% vs. 78%), particularly ocular events.
Implications for Practice: BVd therapy offers a significant progression-free survival advantage over DVd in patients with relapsed or refractory multiple myeloma, though it is associated with a higher rate of serious adverse events, particularly ocular toxicity. These findings suggest BVd as a potent treatment option but highlight the need for careful monitoring and management of side effects, particularly eye-related complications.
RCT: Isatuximab Plus VRd Significantly Improves Progression-Free Survival in Newly Diagnosed Multiple Myeloma – N Engl J Med
18 Aug, 2024 | 18:11h | UTCStudy Design and Population: This international, open-label, phase 3 trial assessed the efficacy of adding isatuximab to the bortezomib, lenalidomide, and dexamethasone (VRd) regimen in 446 patients aged 18 to 80 years with newly diagnosed multiple myeloma who were ineligible for transplantation. Participants were randomized in a 3:2 ratio to receive either the isatuximab-VRd combination or VRd alone.
Main Findings: At a median follow-up of 59.7 months, the isatuximab-VRd group showed a significantly higher estimated progression-free survival at 60 months (63.2% vs. 45.2%; HR, 0.60; P<0.001) compared to the VRd group. Additionally, the isatuximab-VRd group had higher rates of complete response or better (74.7% vs. 64.1%; P=0.01) and MRD-negative status with a complete response (55.5% vs. 40.9%; P=0.003). Safety profiles were comparable between the two groups.
Implications for Practice: The addition of isatuximab to the standard VRd regimen significantly improves progression-free survival and response rates in newly diagnosed multiple myeloma patients who are ineligible for transplantation, without increasing serious adverse events. This suggests that isatuximab-VRd may be considered a new standard of care in this patient population.
RCT: Blinatumomab Improves Overall Survival in MRD-Negative Acute Lymphoblastic Leukemia Patients – N Engl J Med
11 Aug, 2024 | 12:53h | UTCStudy Design and Population: This phase 3 randomized clinical trial included 488 adults aged 30 to 70 with BCR::ABL1-negative B-cell precursor acute lymphoblastic leukemia (BCP-ALL) who achieved measurable residual disease (MRD)-negative remission after initial chemotherapy. The study compared the outcomes of patients receiving four cycles of blinatumomab alongside consolidation chemotherapy versus those receiving consolidation chemotherapy alone.
Main Findings: At a median follow-up of 43 months, the blinatumomab group demonstrated a significant improvement in overall survival (85% vs. 68% at 3 years) compared to the chemotherapy-only group, with a hazard ratio for death of 0.41 (95% CI, 0.23 to 0.73; P = 0.002). The 3-year relapse-free survival was also higher in the blinatumomab group (80% vs. 64%), with a hazard ratio for relapse or death of 0.53 (95% CI, 0.32 to 0.87). However, a higher incidence of neuropsychiatric events was noted in the blinatumomab group.
Implications for Practice: The addition of blinatumomab to consolidation chemotherapy significantly enhances overall and relapse-free survival in adult patients with MRD-negative BCP-ALL, suggesting its potential as a standard treatment approach for this population. Clinicians should monitor for neuropsychiatric side effects associated with blinatumomab use.
RCT: Ponatinib shows superior MRD-negative complete remission rates compared to Imatinib in newly diagnosed Philadelphia chromosome–positive acute lymphoblastic leukemia – JAMA
25 May, 2024 | 19:03h | UTCThis global phase 3 randomized clinical trial investigated the efficacy and safety of ponatinib versus imatinib in adults with newly diagnosed Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL). The study, conducted across 77 sites, enrolled 245 patients who were randomized in a 2:1 ratio to receive either ponatinib (30 mg/d) or imatinib (600 mg/d) alongside reduced-intensity chemotherapy. The primary endpoint of the trial was minimal residual disease–negative (MRD-negative) complete remission, assessed at the end of cycle 3. Results showed that ponatinib achieved a significantly higher MRD-negative complete remission rate of 34.4% compared to 16.7% with imatinib. Additionally, the safety profile between the two drugs was comparable, with arterial occlusive events being rare and similar across groups. These findings suggest ponatinib as a potentially preferable frontline therapy in this patient population due to its superior efficacy in achieving MRD-negative status without compromising safety.
Reference (link to abstract – $ for full-text):
RCT: Adding Ibrutinib to immunochemotherapy and autologous stem-cell transplantation improves PFS in young mantle cell lymphoma patients but increases toxicity – The Lancet
5 May, 2024 | 15:02h | UTCStudy Design and Population:
This study is a three-arm, randomized, open-label, phase 3 superiority trial named TRIANGLE, conducted across 165 centers in Europe and Israel. It aimed to compare the effectiveness of adding ibrutinib to standard immunochemotherapy, both with and without autologous stem-cell transplantation (ASCT), in 870 previously untreated mantle cell lymphoma patients aged 18-65 years, suitable for ASCT.
Main Findings:
After a median follow-up of 31 months, the arm with ibrutinib added to immunochemotherapy followed by ASCT (group A+I) demonstrated a 3-year failure-free survival rate of 88% compared to 72% in the standard immunochemotherapy plus ASCT group (group A). This indicates a significant improvement (hazard ratio 0.52; p=0.0008). Conversely, the efficacy of ASCT in the presence of ibrutinib (group A+I vs. group I, ibrutinib without ASCT) remains under analysis. Adverse events were more frequent and severe post-ASCT, particularly concerning hematological complications and infections.
Implications for Practice:
The results suggest that adding ibrutinib to first-line immunochemotherapy regimens significantly enhances clinical outcomes in younger patients with mantle cell lymphoma. However, the increased toxicity observed warrants careful patient monitoring, especially following ASCT. Further research is needed to evaluate the necessity and timing of ASCT in regimens containing ibrutinib.
Reference (link to free full-text):
Systematic Review: Daratumumab enhances survival in newly diagnosed multiple myeloma patients ineligible for transplant – Cochrane Library
4 May, 2024 | 13:46h | UTCStudy Design and Population:
This Cochrane review evaluates four open-label, two-arm randomized controlled trials involving 1,783 adults with newly diagnosed multiple myeloma ineligible for autologous stem cell transplant. The studies were conducted globally, mainly in middle- and high-income countries, with participants aged between 69 to 74 years. Adding Daratumumab, a CD38-targeting monoclonal antibody, to standard antineoplastic therapy was compared to standard antineoplastic therapy alone.
Main Findings:
Daratumumab significantly improved overall survival with a hazard ratio of 0.64 and increased progression-free survival with a hazard ratio of 0.48, based on moderate-certainty evidence from the studies. Quality of life showed a slight improvement, although the evidence was of low certainty. Treatment-related serious adverse events were more common in the daratumumab group, with a risk ratio of 1.18. However, there was no significant increase in adverse events of CTCAE grade ≥3, except for a higher risk of infections.
Implications for Practice:
The addition of daratumumab to standard antineoplastic therapy offers a potential benefit in overall survival and disease progression control for patients with multiple myeloma who are ineligible for transplant. These benefits must be weighed against the increased risk of serious adverse events and infections. Future studies could provide further insights, particularly into the long-term quality of life and management of side effects.
Reference (link to abstract – $ for full-text):
RCT | Early oral antibiotic switch in low-risk neutropenic sepsis shows mixed results
3 Aug, 2023 | 13:15h | UTC
Phase 1 Trial | NI006 shows potential for cardiac amyloid depletion in transthyretin amyloid cardiomyopathy
1 Aug, 2023 | 14:17h | UTCPhase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid – New England Journal of Medicine (link to abstract – $ for full-text)
Commentaries:
Amyloid Removal Looks Possible in ATTR Cardiomyopathy Imaging Trial – TCTMD
Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid – American College of cardiology
Phase 1–2 study | Liso-cel shows effectiveness against resistant CLL/SLL post BTK inhibitor, venetoclax treatment
20 Jul, 2023 | 10:58h | UTCLisocabtagene maraleucel in chronic lymphocytic leukaemia and small lymphocytic lymphoma (TRANSCEND CLL 004): a multicentre, open-label, single-arm, phase 1–2 study – The Lancet (link to abstract – $ for full-text)
News Release: Immunotherapy Improves Remission for Relapsed, Refractory Leukemia – Northwestern Medicine
Review | Guiding the use of invasive cardiac interventions in patients with advanced malignancies
17 Jul, 2023 | 13:23h | UTC
Clinical Trial Update | 5-year follow-up validates efficacy of sorafenib maintenance in FLT3-ITD AML
11 Jul, 2023 | 13:37h | UTCSorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trial – The Lancet Haematology (link to abstract – $ for full-text)
Commentary on Twitter
NEW: with over 5 years of follow-up, sorafenib maintenance is associated with improved outcomes compared w/ non-maintenance in patients w/ FLT3-ITD AML undergoing allogeneic HSCT in a multicentre, randomised, ph 3 trial #leusm #bmtsm https://t.co/JK190vS0Ql pic.twitter.com/SBP887hgEu
— The Lancet Haematology (@TheLancetHaem) July 5, 2023
Single-arm study | Pirtobrutinib exhibits efficacy in CLL patients after failure of covalent BTK inhibitor treatment
7 Jul, 2023 | 16:14h | UTCPirtobrutinib after a Covalent BTK Inhibitor in Chronic Lymphocytic Leukemia – New England Journal of Medicine (link to abstract – $ for full-text)
News release from the manufacturer: New England Journal of Medicine Publishes BRUIN Phase 1/2 Trial Data for Pirtobrutinib in BTK Inhibitor Pre-Treated Adult Patients with Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma – Lilly
Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management
5 Jul, 2023 | 01:00h | UTC
SR | Polyclonal anti‐thymocyte globulins for the prophylaxis of GVHD after allogeneic stem cell or bone marrow transplantation
3 Jul, 2023 | 14:16h | UTC
RCT | Luspatercept outperforms epoetin alfa in interim analysis, shows promise for lower-risk myelodysplastic syndromes
30 Jun, 2023 | 14:42h | UTCEfficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial – The Lancet (link to abstract – $ for full-text)
RCT | Cyclophosphamide-based regimen enhances GVHD-free survival after hematopoietic stem-cell transplantation
27 Jun, 2023 | 13:54h | UTCSummary: The article details a phase 3 trial comparing the efficacy of two graft-versus-host disease (GVHD) prophylactic regimens in hematologic cancer patients undergoing allogeneic hematopoietic stem-cell transplantation (HSCT). The experimental group received cyclophosphamide–tacrolimus–mycophenolate mofetil, and the standard group received tacrolimus–methotrexate. The patients, a total of 431, underwent HSCT from an HLA-matched related donor or a matched or 7/8 mismatched unrelated donor.
The primary end point was GVHD-free, relapse-free survival at 1 year. Results indicated a significantly higher incidence of this outcome in the experimental group (hazard ratio, 0.64; 95% confidence interval [CI], 0.49 to 0.83; P=0.001). At 1 year, adjusted GVHD-free, relapse-free survival was 52.7% (95% CI, 45.8 to 59.2) in the experimental group, compared to 34.9% (95% CI, 28.6 to 41.3) in the standard group.
Notably, patients in the experimental-prophylaxis group appeared to have less severe acute or chronic GVHD and a higher incidence of immunosuppression-free survival at 1 year. Overall survival, disease-free survival, relapse, transplantation-related death, and engraftment did not show a substantial difference between the groups. These results suggest that cyclophosphamide–tacrolimus–mycophenolate mofetil may offer a more effective prophylaxis against GVHD in HSCT patients.
Article: Post-Transplantation Cyclophosphamide-Based Graft-versus-Host Disease Prophylaxis – New England Journal of Medicine (link to abstract – $ for full-text)
News Release: Study Sets New Standard for Graft-Versus-Host Disease Prevention After Stem Cell Transplant – Johns Hopkins Medicine
Commentary on Twitter
In this trial, 1-year GVHD-free, relapse-free survival after stem-cell transplantation was 52.7% in the cyclophosphamide–tacrolimus–mycophenolate mofetil group and 34.9% in the tacrolimus–methotrexate group. https://t.co/wCdvP1LChu
— NEJM (@NEJM) June 21, 2023
Consensus Paper | Primary prophylaxis of invasive fungal diseases in patients with hematological malignancies
23 Jun, 2023 | 13:23h | UTC
Consensus Paper | Management of febrile neutropenia in pediatric hematology and oncology patients
20 Jun, 2023 | 12:38h | UTC
Review | Updates in immunohistochemistry for hematopoietic and lymphoid neoplasms
20 Jun, 2023 | 12:36h | UTC
RCT | Avapritinib superior to placebo in reducing symptoms, mast-cell burden in indolent systemic mastocytosis patients
20 Jun, 2023 | 12:34h | UTCAvapritinib versus Placebo in Indolent Systemic Mastocytosis – NEJM Evidence
Commentary on Twitter
“…in this 24-week blinded phase of the PIONEER trial, avapritinib demonstrated superiority to placebo in improving symptoms in patients with moderate to severe symptomatic ISM [indolent systemic mastocytosis]….”#Oncology #ClinicalTrials @StanfordDeptMed @ProfMaurer
— NEJM Evidence (@NEJMEvidence) June 15, 2023
Review | Antifungal stewardship interventions in patients with hematologic malignancies
16 Jun, 2023 | 13:47h | UTCAntifungal Stewardship Interventions in Patients with Hematologic Malignancies