Latest Hematology research

Top 10 this week

Week of 1 November 2026

1. 1

   Iberdomide plus daratumumab and dexamethasone versus daratumumab, bortezomib, and dexamethasone in patients with relapsed or refractory multiple myeloma (EXCALIBER-RRMM): an open-label, randomised, controlled, phase 3 trial

   The Lancet. Oncology · Q1 journal · RCT · Evidence level 1 (High) · 25 September 2026

   In the first 420 patients analyzed, 41% receiving iberdomide, daratumumab, and dexamethasone achieved a complete response with no detectable minimal residual disease, versus 21% on daratumumab, bortezomib, and dexamethasone. Severe side effects were more common with iberdomide, especially neutropenia and infections; whether the response advantage improves progression-free survival remains unknown.

2. 2

   Low-dose, short-duration catheter-directed thrombolysis for acute intermediate-high-risk pulmonary embolism: the BETULA trial

   EuroIntervention : journal of EuroPCR in collaboration with the Working Group on Interventional Cardiology of the European Society of Cardiology · Q1 journal · RCT · Evidence level 1 (High) · 17 August 2026

   Low‑dose CDT improves RV/LV ratio in intermediate‑high‑risk PE

3. 3

   Decitabine plus busulfan-cyclophosphamide versus busulfan-cyclophosphamide myeloablative conditioning before allogeneic haematopoietic stem cell transplantation for reducing risk of relapse in adult KMT2A‑rearranged leukaemia in China: an open-label, multicentre, randomised phase 3 trial

   The Lancet. Haematology · Q1 journal · RCT · Evidence level 1 (High) · 1 November 2026

   In a randomized trial of 130 patients with KMT2A-rearranged leukemia undergoing stem cell transplant, adding decitabine to conditioning reduced 2-year relapse from 29% to 12%. Severe infections, graft-versus-host disease and gastrointestinal events were similar between groups, but the trial was conducted at eight centers in China; whether the benefit holds in other settings remains unknown.

4. 4

   Methylphenidate for Hematologic Cancer-Related Fatigue: A Randomized Clinical Trial

   Journal of clinical oncology : official journal of the American Society of Clinical Oncology · Q1 journal · RCT · Evidence level 1 (High) · 30 October 2026

   In a randomized crossover trial of 152 adults with hematologic cancer, fatigue scores improved by 8.9 points during six weeks of methylphenidate, compared with 3.9 points on placebo. Side effects were mostly mild but more common with methylphenidate, and the results may not apply to patients receiving recent chemotherapy or other psychotropic drugs.

5. 5

   Baseline Tumor Transcriptional Features and Event-Free Survival After Anti-CD19 CAR T-Cell Therapy with Axicabtagene Ciloleucel in Large B-Cell Lymphoma: An Exploratory Reanalysis of the Randomized ZUMA-7 Trial

   Medical sciences (Basel, Switzerland) · Q1 journal · RCT · Evidence level 1 (High) · 13 October 2026

   In an exploratory reanalysis of 134 patients treated with axicabtagene ciloleucel, eight of 25 prioritized tumor transcripts were associated with event-free survival, and higher B-cell gene expression generally tracked with longer survival without an event. The findings do not establish that these markers predict who benefits from CAR T; independent validation is needed.

6. 6

   Temporal trends in infectious risk among multiple myeloma patients treated with daratumumab: A meta-analysis

   British journal of haematology · Q1 journal · SR/MA of RCTs · Evidence level 1 (High) · 26 October 2026

   Across six randomized trials involving 2,989 patients, severe infections occurred in 29% of those receiving daratumumab, compared with 20% of controls. Infection rates peaked in the first three months, and recurrent infections were also more common; fewer than 20% of patients with severe respiratory infections received immunoglobulin replacement. The findings point to an early window for closer monitoring, but the best prevention strategy remains unclear.

7. 7

   Multi-theory model-based exercise for cognition, physical fitness, and quality of life in patients with lymphoma during chemotherapy: a randomized controlled trial

   Journal of cancer survivorship : research and practice · Q1 journal · RCT · Evidence level 1 (High) · 31 July 2026

   In a randomized trial of 86 adults with lymphoma, a theory-driven exercise regimen achieved 79% adherence and 88% retention, leading to significant gains in self-reported cognitive function, physical fitness and overall quality of life that persisted at least four weeks after the program ended. The findings suggest a feasible supportive-care option, though longer-term effects remain unknown.

8. 8

   A multimodal approach to sexual function after allogeneic hematopoietic stem cell transplantation: a randomized controlled trial

   Bone marrow transplantation · Q1 journal · RCT · Evidence level 1 (High) · 24 October 2026

   In a randomized trial after stem cell transplant, repeated assessments and nurse-led consultations did not significantly improve overall sexual function. They did improve sexual pain, communication with professionals, and partnership quality, and the approach was feasible; genital chronic graft-versus-host disease was identified in about one-quarter of participants. Whether these benefits last remains unknown.

9. 9

   Adaptive, Stepped-Care for Cancer-Related PTSD Symptoms in HCT Survivors: Results From a SMART Trial

   Psycho-oncology · Q1 journal · RCT · Evidence level 1 (High) · 1 August 2026

   In 477 hematopoietic cell transplant survivors with PTSD, about 60% improved after a 4-week self-guided app, but the app alone was no better than usual care. Patients who did not respond and were stepped up to therapist-delivered CBT showed the greatest and most lasting reductions in PTSD, depression, and anxiety. The findings support using digital tools as a first step, reserving intensive psychotherapy for non-responders.

10. 10

    Comparable efficacy of extraoral and intraoral photobiomodulation in preventing oral mucositis during high-dose methotrexate therapy in pediatric oncology: A single-blind randomized controlled trial

    Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · Q1 journal · RCT · Evidence level 1 (High) · 19 October 2026

    In this randomized trial of 155 children receiving high-dose methotrexate, mouth sores occurred in 30.5% with extraoral treatment and 41.1% with intraoral treatment, a difference that was not statistically significant; severity and pain also did not differ. Extraoral sessions took 30 seconds versus about 6 minutes, but the trial did not establish that the approaches are equivalent.

Paper of the day, last two weeks

• Overall Survival in Adults With Acute Myeloid Leukemia Initiating Quizartinib vs Midostaurin With Chemotherapy

  JAMA network open · Q1 journal · Retrospective cohort · Evidence level 3 (Low) · 2 November 2026

  Comparative real-world outcomes for FLT3-ITD AML regimens

• Dasatinib in pediatric patients with chronic myeloid leukemia in chronic phase: long-term efficacy and endocrine analysis

  Blood advances · Q1 journal · Non-randomized trial · Evidence level 2 (Moderate) · 5 November 2026

  In this phase 2 trial, after about eight years, 55% of newly diagnosed children had a deep molecular response; the rate was 38% among children whose leukemia resisted or did not tolerate imatinib. Height scores fell most in prepubertal children, while other reported endocrine effects were modest. The findings support durable disease control, but the growth effects warrant attention during long-term treatment.

• Distinct cellular dynamics following CD19 and CD7 CAR-T therapies revealed by high-dimensional CyTOF analysis and impact on clinical outcomes

  Journal of translational internal medicine · Q1 journal · Unclassified · Evidence level 5 (Expert Opinion) · 23 October 2026

  CAR-T immune recovery, toxicity, and survival outcomes

• Zanubrutinib Plus Obinutuzumab Versus Obinutuzumab Monotherapy in Patients with Relapsed or Refractory Follicular Lymphoma: Final Analysis of the Phase 2 ROSEWOOD Study

  Targeted oncology · Q1 journal · Non-randomized trial · Evidence level 2 (Moderate) · 3 November 2026

  In this randomized phase 2 study of heavily pretreated patients, the combination produced responses in 70% versus 44% with obinutuzumab alone after a median 34.6 months of follow-up. Median progression-free survival was 22.1 versus 10.3 months, but the overall survival difference was not statistically significant; longer follow-up is needed to clarify whether the response and disease-control gains extend survival.

• Comparative survival and time toxicity outcomes of CAR-T versus bispecific antibody therapies in relapsed/refractory FL

  Blood advances · Q1 journal · Retrospective cohort · Evidence level 3 (Low) · 30 October 2026

  In this 228-patient real-world study, median progression-free survival was not reached with CAR-T, versus about 19 months with bispecific antibodies, whether patients had two or at least three prior treatment lines. CAR-T involved 14 to 20 more health care contact days in the first year, while overall survival was similar; treatment choice still requires weighing disease control against time burden.

• Eltrombopag during induction chemotherapy in older patients with newly diagnosed acute myeloid leukemia is safe and reduces platelet transfusion requirements: The EPAG-2015 French Innovative Leukemia Organization phase 2 randomized trial

  Cancer · Q1 journal · Phase 2 RCT · Evidence level 2 (Moderate) · 1 November 2026

  In a randomized phase 2 trial of 110 older adults receiving intensive chemotherapy for acute myeloid leukemia, eltrombopag lowered the median number of platelet transfusions from 10 to eight, with no increase in severe adverse events. It did not improve overall survival, so the practical value is fewer transfusions; whether that benefit holds in a larger trial remains unknown.

• Neutrophil Engraftment Timing after Single-Unit Cord Blood Transplantation as a Prognostic Marker of Transplant Outcomes Influenced by GVHD Prophylaxis: A nationwide, retrospective study by the Japanese Society for Transplantation and Cellular Therapy

  Transplantation and cellular therapy · Q1 journal · Retrospective cohort · Evidence level 3 (Low) · 28 October 2026

  In 9,049 adults receiving single-unit cord blood transplants, survival was most favorable when neutrophils recovered between days 15 and 23. Recovery by day 14 tracked with severe graft-versus-host disease and worse outcomes mainly in patients given methotrexate-based prevention, while recovery on or after day 24 independently predicted worse survival, more non-relapse deaths and infections; this retrospective study supports engraftment timing as a risk marker, not proof of cause.

• Reduced-Intensity Haploidentical Bone Marrow Transplantation in Children with Severe Sickle Cell Disease: BMT CTN 1507

  Blood advances · Q1 journal · Non-randomized trial · Evidence level 2 (Moderate) · 28 October 2026

  In this phase 2 trial, 39 children with severe sickle cell disease received a transplant from a half-matched donor; 79% were alive and disease-free at two years, and overall survival was 95%. No new or worsening brain infarcts appeared in 22 children with follow-up MRI, but graft failure and graft-versus-host disease reduced event-free survival, and the single-arm study cannot establish comparative benefit.

• Clinical Experience of Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

  American journal of hematology · Q1 journal · Retrospective cohort · Evidence level 3 (Low) · 27 October 2026

  In this retrospective study of 151 people with paroxysmal nocturnal hemoglobinuria, mean hemoglobin rose from 104 to 127 g/L by three months, and 88% became transfusion independent. Responses were similar with or without a history of bone marrow failure; no thromboses or drug-related deaths occurred, but longer follow-up is needed to clarify long-term infection risk and effectiveness.

• Zamtocabtagene autoleucel, a tandem CD20-CD19-directed CAR T-cell therapy, for second line treatment of large B-cell lymphoma in patients ineligible for transplantation (DALY 2-EU): primary analysis of an open-label, multicentre, randomised phase 2 study

  The Lancet. Oncology · Q1 journal · Phase 2 RCT · Evidence level 2 (Moderate) · 1 November 2026

  In 160 transplant-ineligible patients with relapsed or refractory large B-cell lymphoma, second-line zamto-cel extended median event-free survival to 6.2 months, versus 2.5 months with rituximab-gemcitabine-oxaliplatin. Severe treatment-related side effects were common in both groups; zamto-cel also caused cytokine release syndrome and neurologic toxicity, and follow-up was 17 months, leaving longer-term benefit and risks to clarify.

• Short-Course Ruxolitinib Initiated at Neutrophil Engraftment for Prevention of Acute Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation: A Multicenter, Randomized, Open-Label, Controlled Phase II Trial

  Transplantation and cellular therapy · Q1 journal · Phase 2 RCT · Evidence level 2 (Moderate) · 24 October 2026

  In this randomized phase II trial, grade II to IV acute graft-versus-host disease by day 100 occurred in 14% of patients given 14 days of ruxolitinib at neutrophil engraftment, versus 34% with standard prevention alone - about one fewer case for every five treated. Severe side effects were similar, but survival was unchanged and larger trials are needed to confirm benefit and clarify long-term effects.

• Comparison of DOAC Versus VKA for Cerebral Venous Thrombosis: A Systematic Review and Meta-Analysis

  Stroke · Q1 journal · SR/MA of RCTs · Evidence level 1 (High) · 22 October 2026

  Across 14 studies involving 3,698 patients, direct oral anticoagulants and vitamin K antagonists had similar rates of recurrent clots, bleeding, good functional recovery and death. The results support DOACs as a reasonable treatment alternative, but the studies were small and pooled estimates do not establish that the drugs are equivalent.

Evidence summaries in Hematology

• Venous Thromboembolism
• Hemophilia A
• Amyloidosis
• Sickle Cell Disease
• Warfarin
• Aplastic Anemia and Pure Red Cell Aplasia
• Chronic Myeloid Leukemia
• Immune Effector Cell-Associated Neurotoxicity Syndrome
• Graft-Versus-Host Disease
• Hemophagocytic Lymphohistiocytosis
• Thrombotic Thrombocytopenic Purpura
• Multiple Myeloma
• Iron Deficiency Anemia
• Polycythemia Vera
• Hereditary Hemochromatosis
• Aplastic Anemia
• Apixaban