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HemaBites

HemaBites showcase hematology news & short commentaries on recent high-impact articles published in international journals. This blog will keep you up to date with the latest discoveries in the field of hematology as well as other related hematology news.

Wednesday, May 10, 2023

Donna Kirby, EHA/HemaSphere, The Hague, The Netherlands

 

Did you know that the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) publicly releases its monthly meeting highlights? These highlights include medicines recommended for approval, negative opinions on new medicines, recommendations on extensions of therapeutic indication, and more. We are pleased to alert you to this important information, and to specifically feature the hematology-related updates from the committee.

 

 In April 2023, the CHMP:

 - adopted a positive opinion, under conditional marketing authorization,  for Columvi* (glofitamab) a biosimilar medicine for the treatment of diffuse large B-cell lymphoma, an aggressive type of non-Hodgkin lymphoma, a cancer of the lymphatic system that can arise in lymph nodes or outside of the lymphatic system. The committee also adopted a positive opinion,  under conditional marketing authorisation, for Jaypirca* (pirtobrutinib) for the treatment of relapsed or refractory mantle cell lymphoma which develops when B-cells, a type of white blood cell that makes antibodies, become abnormal..

- the application for marketing authorization of Tidhesco* was withdrawn. This medicine was intended for the treatment of acute myeloid leukaemia. Tidhesco was a duplicate of Tibsovo. Both applications received a positive opinion on 23 February 2023.


You can access the full CHMP April 2023 meeting highlights freely; click here.​


*This product was designated as an orphan medicine during its development. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products (COMP) at time of approval determining whether the information available to date allows maintaining the medicine's orphan status and granting the medicine ten years of market exclusivity.


Saturday, April 15, 2023

Donna Kirby: EHA/HemaSphere, The Hague, The Netherlands

Are you aware that each month our Editor in Chief picks a few of our articles to call out as ‘Editors Highlights’ ? These are articles called out for extra notice: either for new data, exciting breakthroughs, novel use of technology, or general interest  We feature them in our HemaSphere monthly newsletter, as well as on LinkedIn and Twitter.

For your convenience, we have compiled a quarterly round up of the linked articles to catch you up on what you might have missed. 

January:

Pathogen-specific T-cells: Targeting Old Enemies and New Invaders in Transplantation and Beyond 

Yannaki E et al

Adoptive immunotherapy with pathogen-specific T cells (pSTs) can restore immunity and prevent or treat opportunistic infections/diseases post transplantation or even outside of this context. This could provide a platform to also target novel emerging pathogens, including SARS-COV-2. The journey of evolution of immunotherapy with pSTs against infections and virus-associated malignancies from the early steps of development up to clinical translation is presented in this review. Will adoptive immunotherapy with pSTs become a standard treatment in the future?

Outcome Prediction in Patients With Large B-cell Lymphoma Undergoing Chimeric Antigen Receptor T-cell Therapy   

Voltin et al

While the introduction of chimeric antigen receptor (CAR) T-cell therapy has led to a fundamental shift in the management of relapsed and refractory large B-cell lymphoma, our understanding of risk factors associated with non-response is still insufficient. Relevant patient and disease characteristics should be considered when managing patients with large B-cell lymphoma who are due to undergo CAR T-cell therapy, and this study shows that in this context, higher metabolic tumor volume and SUVmax as well as extra-nodal disease are associated with significantly inferior patient outcome.

February:

Hematopoietic Cell Autonomous Disruption of Hematopoiesis in a Germline Loss-of-function Mouse Model of RUNX1-FPD  

Ernst et al

RUNX1 familial platelet disorder (RUNX1-FPD) is caused by germline loss-of-function mutations in the RUNX1 gene, and is characterized by thrombocytopathy, thrombocytopenia, and an increased risk of developing hematologic malignancies mostly of myeloid origin. However, what are the potential contributions of ancillary, non-hematopoietic cells to the hematopoietic manifestations of this disease? Using a mouse model of RUNX1-FPD that mimicked impaired residual RUNX1 function, authors uncovered hematopoietic cell-intrinsic platelet deficiency, increased myeloid potential, and decreased lymphoid potential.

Impact of Ultra High-risk Genetics on Real-world Outcomes of Transplant-eligible Multiple Myeloma Patients                

Panopoulou et al

Ultra high-risk genetics predict early relapse and poor subsequent outcomes post standard care in autologous stem cell transplantation. This was further evaluated via a retrospective review of records of patients with newly diagnosed multiple myeloma (NDMM). Double hit genetics ≥2 adverse markers: t(4;14), t(14;16), t(14;20), gain(1q), and del(17p) were associated with a shortened progression-free survival (HR = 4.27, P < 0.001) and overall survival (HR = 4.01, P = 0.03), and characterized most early relapses. Results support the real-world utility of extended genetic profiling for improved risk prediction in patients with NDMM.

March:

Simultaneous Inhibition of PI3Kgamma and PI3Kdelta Deteriorates T-cell Function with Implications for Chronic Lymphocytic Leukemia  

Roessner et al​

The goal of this study is a comprehensive analysis of four PI3K inhibitors, idelalisib, umbralisib, duvelisib, and eganelisib, and their impact on CD4 and CD8 T-cell activation and function. By performing treatment studies with naïve and chronic lymphocytic leukemia-experienced T-cells of human or murine origin, authors show that inhibition of PI3K-delta and PI3K-gamma limits the activation and proliferation of T cells. Unexpectedly, the authors observed an additive effect of dual inhibition of PI3K-delta and PI3K-gamma on T-cell function.

Prediction of Nonrelapse Mortality in Patients With Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia Receiving Allogeneic Stem Cell Transplantaion With Posttransplantation Cyclophosphamide-based Graft Versus Host Disease Prophylaxis         

Versluis et al

In this study, authors develop and validate a novel, post-transplant cyclophosphamide (PTCY)-specific risk score model for nonrelapse mortality (NRM) prediction in patients with acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL) who receive PTCY-based graft vs host disease prophylaxis. This novel NRM risk score model better predicts NRM and overall survival at 2 years compared to existing models, which might be due to the specific toxicities of high-dose cyclophosphamide.


You can always find the latest issue here​, and as always, our content is fully open access. ​

Wednesday, April 12, 2023

Donna Kirby, EHA/HemaSphere, The Hague, The Netherlands

 

Did you know that the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) publicly releases its monthly meeting highlights? These highlights include medicines recommended for approval, negative opinions on new medicines, recommendations on extensions of therapeutic indication, and more. We are pleased to alert you to this important information, and to specifically feature the hematology-related updates from the committee.

 

 In March 2023, the CHMP:

 

 - adopted a positive opinion for Epysqli (eculizumab) a biosimilar medicine for the treatment of paroxysmal nocturnal haemoglobinuria, a rare disorder that leads to the premature destruction and impaired production of blood cells. The committee also adopted a positive opinion for generic medicine  Dabigatran Etexilate Accord (dabigatran etexilate) for the prevention of venous thromboembolic events.

 

-recommended extensions of indication for Breyanzi  used to treat large B cell lymphoma, Entresto and its duplicate Neparvis, Tenkasi, Ultomiris and Wegovy.  

 

- the application for marketing authorization of Feraheme was withdrawn.  Feraheme was intended for the intravenous treatment of iron deficiency anaemia.

 

You can access the full CHMP March 2023 meeting highlights freely; click here.





 


Tuesday, March 7, 2023

Donna Kirby: EHA/HemaSphere, The Hague, The Netherlands

Did you know that the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) publicly releases its monthly meeting highlights? These highlights include medicines recommended for approval, negative opinions on new medicines, recommendations on extensions of therapeutic indication, and more. We are pleased to alert you to this important information, and to specifically feature the hematology-related updates from the committee.

 

In February 2023, the CHMP:

 

 - adopted a positive opinion for Tibsovo* (ivosidenib) for the treatment of newly diagnosed acute myeloid leukemia, AML, a cancer of the blood and bone marrow, and for the treatment of locally advanced or metastatic cholangiocarcinoma, a type of cancer that forms in the bile ducts. The committee also adopted a positive opinion for its duplicate Tidhesco* (ivosidenib) for the treatment of newly diagnosed AML.

 

-gave a positive opinion to Vafseo (vadadustat) for the treatment of symptomatic anemia in adults with chronic kidney disease who are on maintenance dialysis.

 

-biosimilar medicine Bekemv (eculizumab) received a positive opinion for the treatment of paroxysmal nocturnal hemoglobinuria in adults and children. Paroxysmal nocturnal hemoglobinuria is a life-threatening genetic disease that causes the breakdown of red blood cells resulting in various medical complications. Bekemv is the first biosimilar approved for this active substance. Unlike the reference product, Bekemv is contraindicated in patients with hereditary fructose intolerance and in children below two years of age.

You can access the full CHMP February 2023 meeting highlights freely; click here.




 

*This product was designated an orphan medicine during its development. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products (COMP) at approval, determining whether the information available to date allows maintaining the medicine's orphan status and granting the medicine ten years of market exclusivity.


Wednesday, February 22, 2023

Donna Kirby, EHA/HemaSphere, The Hague, The Netherlands

7 days left!

Abstract submission is now open for the 28th Annual EHA Congress—or, EHA2023—held in person and livestreamed from June 8-11, 2023 in Frankfurt, Germany, with virtual days June 14-15 online. The submission deadline is March 1, 2023. Submit your abstract(s) now and showcase your work to an international group of experts - the submission page can be found here. Additionally, you may be eligible and can apply for a travel or participation grant when submitting your abstract.


Submit soon and profile your research. We are very proud to be publishing accepted EHA2023 abstracts as an official HemaSphere supplement this June.

 

Best wishes, everyone!