Nico Gagelmann: Selected clinical studies 2024 in hematology

Nico Gagelmann, Co-founder and Co-chair the European Society for Blood and Marrow Transplantation (EBMT) Trainee Committee, shared aa post on X:

“What a year 2024 in hematology! Here’s a thread on selected clinical studies we’ve seen this year.

Asciminib in Newly Diagnosed Chronic Myeloid Leukemia | NEJM

Authors: Andreas Hochhaus et al.

– asciminib showed superior efficacy and a favorable safety profile in patients with newly diagnosed chronic-phase CML
– BUT comparison with second-generation TKIs was not a primary objective!

Blinatumomab in Standard-Risk B-Cell Acute Lymphoblastic Leukemia in Children | NEJM

Authors: Sumit Gupta et al.

– 3-year DFS was 96% with blinatumomab and chemotherapy and 88% with chemotherapy alone
– Cytokine release syndrome, seizures, and sepsis of grade 3 or higher were rare

Sequential CD7 CAR-T and allogeneic BMT without GVHD prophylaxis | NEJM

Authors: Yongxian Hu et al.

– novel “all-in-one” strategy -combining sequential CD7 CAR-T and haploidentical BMT
– in CD7-positive leukemia or lymphoma
– enhances efficacy while minimizing the risk of GVHD

PERSEUS: Daratumumab, Bortezomib, Lenalidomide, and Dexamethasone for Multiple Myeloma | NEJM

Authors:  Pieter Sonneveld et al.

– simple: standard of care for all patients with newly diagnosed myeloma!
– if accessible and eligible, of course

Nivolumab+AVD in Advanced-Stage Classic Hodgkin’s Lymphoma

Authors: Alex F. Herrera et al.

– simple: new standard of care
– longer PFS than brentuximab vedotin+AVD in adolescents/adults with stage III or IV advanced-stage classic disease
– better side-effect profile!

Sincere congrats Jonathan W. Friedberg et al!

Belantamab Mafodotin, Bortezomib, and Dexamethasone for Multiple Myeloma | NEJM

Authors: Vania Hungria et al.

– compared with daratumumab, bortezomib and dexa, belantamab conferred a significant benefit with respect to among patients after at least one line of therapy
– revival of belantamab!

Daratumumab or Active Monitoring for High-Risk Smoldering Multiple Myeloma | NEJM

Authors: Meletios A. Dimopoulos et al.

– reduces risk of progression to active multiple myeloma by 51%
– 5-year PFS: 63% (vs. 41% in monitoring)
– Safety profile consistent
– Game-changer!

Congrats Vincent Rajkumar and colleagues!

Ibrutinib in Early-Stage Chronic Lymphocytic Leukemia: The Randomized, Placebo-Controlled Phase III CLL12 Trial | JCO ASCO

Authors: Petra Langerbeins et al.

– Ibrutinib delayed disease progression
– BUT, no survival benefit was demonstrated
– watch and wait remains standard of care!

Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101) | JCO ASCO

Authors: Ghayas C. Issa et al.

– febrile neutropenia (1/3), differentiation syndrome (16%), QTc prolongation (14%)
– CR + CRh rate was 23%
– 2/3 with no detectable residual disease

Linvoseltamab for Treatment of Relapsed/Refractory Multiple Myeloma | JCO ASCO

Authors: Naresh Bumma et al.

– 71% ORR, 50% CR or better
– median duration of response of 29.4 months
– CRS in 1/3
– response-adapted regimen enabled patients with VGPR or better to shift to once every 4-week dosing

Gilteritinib as Post-Transplant Maintenance for AML With Internal Tandem Duplication Mutation of FLT3 | JCO ASCO

Authors: Mark J. Levis

– overall improvement in RFS was not statistically significant
– RFS higher for detectable FLT3-ITD MRD pre- or post-HCT who received gilteritinib treatment

Azacitidine, Venetoclax, and Gilteritinib in Newly Diagnosed and Relapsed or Refractory FLT3-Mutated AML | JCO ASCO

Authors: Nicholas J. Short

– high rates of CR/CRi, deep FLT3molecular responses, and encouraging survival
– myelosuppression manageable with mitigative dosing strategies

Long-Term Results of the Dasatinib-Blinatumomab Protocol for Adult Philadelphia-Positive ALL | JCO ASCO

Authors: Robin Foà et al.

– chemotherapy-free induction/consolidation regimen induces durable long-term hematologic and molecular responses
– new era in the management of these patients!

Epcoritamab monotherapy in patients with relapsed or refractory follicular lymphoma (EPCORE NHL-1) | The Lancet Haematology

Authors: Kim M Linton et al.

– showed clinically meaningful activity in patients with multiply relapsed or refractory follicular lymphoma
– manageable safety profile

Tranexamic acid versus placebo to prevent bleeding in patients with haematological malignancies and severe thrombocytopenia | The Lancet Haematology

Authors: TREATT Trial Investigators

– study shows insufficient evidence to support routine prophylactic tranexamic acid in haematological malignancies and chemotherapy

That’s a wrap of selected clinical studies in hematology from 2024.

This list does not claim to be complete, and I am happy if you share your most important study, so that we all can learn as community.

Safe transfer to 2025 to all of you who love learning and blood.”

Nico Gagelmann is a physician and scientist who co-founded and co-chairs the European Society for Blood and Marrow Transplantation (EBMT) Trainee Committee, and he also serves as the chair of the EBMT subcommittee focused on CAR-T cell therapies for plasma cell disorders. His work is particularly impactful in the realm of CAR-T treatments for multiple myeloma, where he has contributed to advancing research and clinical approaches.